Wealthy people spend millions of dollars on their children for many things which have no benefit to others. This, unlike a starter home in Beverly Hills, has a chance of benefiting others through future research. I may not choose to donate to such a narrowly focused foundation but that was the donors' choice.
55
As long as knowledge is gained that can be used to help this and future patients, it is not a waste of resources. We can focus on many problems at once. The amount of spending on medical and scientific research is a pittance compared to the amount that is wasted on unnecessary wars and valueless pork barrel projects handed out to purchase senators' votes.
57
So Western allopathic medicine gets hailed for... doing what Chinese and Ayurvedic medicine have done for over two thousand years: tailoring treatment to the individual. But there's no mass-marketing profits in that, so forget it. It's not fair...
https://emcphd.wordpress.com
16
If it is inaccessible to the poor, it is neither radical nor revolutionary.
14
My daughter has a rare neurological condition with a few thousand known US cases (RTT). At 15, her lifetime medical costs (let alone our lost income from caring for her) has been in the millions. A one off cure, while expensive would be both cheaper and lead to a productive citizen.
49
Brilliant scientific work that hopefully at least thought flow chart wise can be helpful to development in general. Medicine while has come long way has even longer way to go and we are all dying here (though we don't focus on it) and can't get too much help to slow aging and myriad diseases/conditions. We should be all about this- On front page everyday should be some medical progress tidbit-. yes, the rich will get the vitality and youth first -right now we dont even have the quality which comes before quantity- In medicine we need both of course. And we can do so much better. The need for profit and less than optimal prioritization is slowing down development where the complexity will take years and even in innovation that will benefit all of us.
9
Life is never fair but endeavor is not about a cure but a scientific experiment. So long as there is no public fund is used, there shouldn't any problems with bio-ethics*
* I am no professional bio-ethicist - what constitutes one anyway? - but I am presenting an argument both rational and practical
9
Do you have much of an idea about where millions in 'public funding' goes? Have you looked at the costs of upgrades to foodball stadiums and such 'investments' (which are often not) which cities have wasted? Is that better use of your hard-earned cash? So you're ok with the research potentially ultimately benefitting you as long as you don't pay for it? And I guess you're ok with the scenario where if this was a close relative of yours, presumably you wouldn't have the millions it takes to push for more focused research to help?
The money didn't all come from some rich family - a lot was donated, or did you not read the story?
We all want something for free. The world owes us nothing, despite what youth (primarily in the US) may think.
8
I guess you only get the future you can pay for. Otherwise, you're just roadkill waiting for the Buick. What was the point, again?
8
This is news to me. Felt very happy reading the story since a child is at least improving from the worst unique medical condition. A life saved is much more than millions spent. Thanks a lot for this story.
24
What an Amazing mom of Mila. If only the spend on Guns and wars is used for medical drug research , there’s will be so many children helped and so many moms Blessed.
50
can we just not be happy for this little girl? Perhaps the techniques, if not precisely the same genetic sequence, will have a more general application.
63
I knew that I would read comments from scolds predictably wagging a finger about whether this Herculean effort was a waste of money that would have been better spent elsewhere. Oh please! Can people pull back from judging for a mili-second and grasp the enormity of what this mother and a brilliant scientist (and I'm sure the contribution of many others) have done for this child? (not to mention the next child like her) Instead of arguing how pointless their effort is, look at the grin on this child's face in the photo. I am in awe, astounded.
Addendum: Part of my job as a pharmacist is to write about new drugs. Whereas new drugs for rare diseases were uncommon in the past, they are a substantial segment of what I do today. I'm writing about Scenesse right now. It's the first drug ever for erythropoietic protoporphyria, an inherited disease that affects about 4000 people in the US. The Australian company that developed it started pursuing FDA approval in 2006 -- almost 15 years! They got to the finishing line AND (this is mind-blowing!) they intend to charge the same price for the drug in the US as in Europe. This is walking the walk and talking the talk. I'm not sure most Americans grasp how motivated and capable families and scientists are as a team to find cures for rare diseases. They are a powerful force for good that I support 100%.
101
@JEA where do you write? Is it publicly accessible? I'd be interested to hear more.
Thank you for the positive comment among the whingers.
19
This is a subset of a larger moral conundrum for those who sink hundreds of millions into treating or curing rare genetic diseases - the so called orphan diseases - that may affect hundreds or thousands instead of just one.
Is it morally defensible to sink that much money into staving off death for the few percent, when we look away from the world's many who die from very treatable diseases, ranging from polio or the flu to malnutrition and diarrhea?
To face more inconvenient facts, why do we put more research monies into HIV than into history's great killers - TB and malaria - who lurk at our doors?
The truth is that research is dictated by funding, and funding is dictated by the politics of the local, of people we know or can identify with. When malaria and TB epidemics rise again in Europe and the U.S. - and they will - perhaps the imperatives will change.
25
If among the tough questions is the risk of precipitating death what about the opposite risk of delaying death in a patient who is certain to die and mercifully so? Now that the parents have delayed for decades this poor girl's death, what further experiments are they planning to perform on her? I find this scientific "advance" nothing short of terrifying.
13
@Mickeyd : Unless I missed it, the article didn't state or imply that her life expectancy would be extended, only that her symptoms are less severe. If it's true that this drug will prolong her life, then I agree that it's terrifying to think of her living even longer in this hell, but I also note that it's a relief that she's suffering less at present.
Millions of old persons are kept alive, with poor quality of life, because medical protocols have changed -- drugs and devices exist and their use is standard. So this thorny ethical issue has been with us for some time, and it applies to far more than just people like this little girl.
22
What a tragic story. I am so sorry for the girl and her mother.....but the drug is only slowing down (slightly) the inevitable. Thousands of other kids could have benefited from that $3million......what is the "right thing" ???? Many kids with cancers could have been cured with that money....
Which is why, and I say this as a retired RN, our health system IS broken, it functions to further enrich the CEO's of big pharma, of hospital corporations and insurance companies.
We must have some form of Universal health care, as does every other developed nation in the world, for all citizens.
Why not take funds from the 1% (especially those like Amazon who haven't paid taxes in years), and the military/industrial complex? We could STILL destroy the world 10x over even doing that.
We must VOTE for those who will help this occur.
43
Often drug companies develop drug treatments for rare diseases under the support of the 1983 US Orphan Drug Act, yet “serendipitously” get these drugs subsequently approved or sold for off-label purposes or continue research for much more common diseases or symptoms. All for the good.
20
@Joel H
Agree, and that point should have been raised in the article. A rare diseases can inspire research in new directions and in novel approaches that if proven efficacious can be used to treat far more than one disease.
13
Back in 2018, the CBC ran a show about 'Glybera', "The Million Dollar Drug", which was the first government-approved gene therapy drug designed to treat lipoprotein lipase defficiency (LPLD). The drug was developed at UBC, and the rights to the drug were eventually acquired by an Italian pharmaceutical company. It was so expensive (1 million U$ for a single dose), that there was no market for it and it is now no longer available to the public.
https://newsinteractives.cbc.ca/longform/glybera
6
Hence eminent domain?
1
Hats off Dr. Yu and the whole team...kinda a miracle
9
It is called "proof of concept".
Eventually can be generalized.
17
There are likely millions of one-off monogenic disorders that will afflict children now and in the future. Regardless of the outcome in this case, researchers will learn from this experience and hopefully have better therapies in the future. The negative comments here are at once incomprehensible and ignorant with respect to how clinical research actually operates.
22
@LTJ LTJ, I have a PhD in Biophysics and worked in pharma. My comments againist this aren't incomprehensible, or ignorant of clinical research.
They are merely a statement that, this particular situation is probably not one that should be used as an example of responsible science. The resources used to implement this came at the expense of helping larger numbers of other peoppe, and the transferrability of what was learned in this experiment is extremely limited.
6
@David Konerding I don't think you can fairly say it was "at the expense of" something else - this was not a zero sum game.
I don't disagree with the overall sentiment, per se, but wasn't most of this crown funded? (I should go reread it).
Seemed it's the result of a good campaign benefitting from the human tendency to feel more sympathy with one hungry child, than two or a million.
10
The article states that Milas mother raised $3m, but it does not state that $3m was the total cost of developing the therapy.
5
Poor kid and her mom, as a parent I cannot imagine the suffering. Intellectually, one would argue that a one person medication is a bad idea as it is only for those who can afford it. One could also argue that this family is trying to avoid the inevitable, and that this child is likely to die a painful and slow death. But as a parent, I cannot judge. I cannot say that I would not do the same! Hope things do really improve for this child.
13
This is a tough one. But untold numbers of parents everyday make merciful decisions about ending treatment for their children who have bleak futures. I cannot condemn this parent's hope (and love) but I do worry about the limited futures and suffering that falls to the child.
13
I refuse to believe that the bench research that went into creating this drug will not prove beneficial to others with diseases yet untreated. If this nation weren't giving tax cuts to corporations and .01% ers we could cure and treat all diseases, fund all research. Bravo to everyone involved in helping this child.
40
@winchestereast : While you're right to decry this nation's screwy and anti-social tax code, you're being overly optimistic with regard to curing and treating all diseases.
For one thing, cures for all are unrealistic -- science isn't miracles-based -- and so costly that even truly, scupulously fair taxation wouldn't cover the cost.
For another, new diseases will continue to arise, in part because Nature will keep trying to cull the herd for as long as we continue to destroy the Earth.
But, yes, we need fair taxation and we need decent, single-payer medical care for every US resident -- we need a system like Canada's.
6
The reason to do this is to bring the cost down from $3M to $30k to $300.
15
Terminal illnesses in children have to be one of the most ethically difficult situations to parse in medical ethics, at least from my layperson's view. We're generally not accepting of euthanasia of children, as that's a lot of responsibility - and power, for that matter - to put in the hands of parents and doctors of those helpless children. For a child slowly slipping away, especially one who isn't old enough or is otherwise incapable of making their wishes known, and in a case like this where the parent is basically helpless to do anything themselves to ease their child's suffering, what choice does she have? If nothing else, hopefully she is able to make some lasting memories with her child.
15
"precipitating severe complications or death is not acceptable" except when the insurance company, or state tell you that since assisted suicide is legal where you live, it is the only alternative they'll pay for. First, the AMA needs to decide on a consistent life ethic. Second, health care is rationed everywhere. Either people with money, or who can raise the money get more, or high ranking public officials and politicians get more. It's never fair. Nowhere is it fair. But I definitely know that I don't want to live in a country where anybody has the right to tell a free citizen what he has to do for a living. Tim Yu lives free and is free to work on what he wants. Change that for him and everybody is up for grabs.
5
I would much rather see these medical stories than us giving more money and lives to countries that hate everything about us except our money .
24
@BorisRoberts , please keep in mind that U.S. foreign aid makes up a very small percentage of our national budget.
The question raised by this article leads this reader to ask why we have an inefficient, profit driven healthcare system that leads family members to resort to public fundraising campaigns.
35
This just in: a kid is a kid, and deserves help wherever they are. Feel free to discuss this with Jesus.
31
@Robert
Correct, but I THINK what BorisRoberts is saying is that WHY do families have to beg and plead to raise money for kids with cancer, bad burns, etc? Which they do.......not just kids with incurable conditions like the girl in the article, but with more "normal" but devastating conditions that CAN be saved but only with enough money. WHY is the onus on the parents to raise the money and do the hard work?
5
It doesn't really work. At best, it's dragging things out from a slow decline and death into an excruciatingly slow decline and death. The article doesn't do a very good job of being honest about this.
33
@A
Not necessarily. Yes, she will need the ASO for life, but in her case, that's better than certain death through intractable seizures.
This really IS the future of medicine. It may seem like a one-off deal now, but the same ASO can be used in other patients in the future. In the past, those have a) not been diagnosed and b) died an agonizing death.
ASOs will revolutionize neurological disease treatment.
5
I see absolutely no ethical dilemma here, and I applaud Mrs. Vitarello for doing everything in her power to take care of her daughter. Heaven for bid anything should happen to my two children; if it did, I would go to the ends of the earth to try to save them.
34
@David H
AMEN!
4
Whether or not this is a good idea for society as a whole, or makes sense on a global scale, you can hardly blame the mother for doing everything in her power to save her own child. She will probably fail, but at least she tried.
14
Sometimes you just have to let go.
Others say we could learn from this to develop cheaper options. I say there are many out there who have serious but treatable problems who go without due to finances and these resources would be better spent in these areas.
I, personally, would feel very guilty asking for contributions for this poor child when there are so many with needs who could be helped. ...and I would, also, fear, with this child's severe disabilities, the prospect of what her life would be like if I should die first.
There is a very fine line between love and selfishness.
26
@Blanche White what do you mean by let go? stop feeding? stop giving medicine? euthanize?
2
I can’t even conceive of this mother’s torment as she watches her child fade away in front of her. This story may be about scientific advancement and the privilege money can buy but ultimately it’s a universal story about mothers trying to keep their children alive and safe.
21
In medicine, most existential breakthroughs are long in coming and hard won. They are more and more dependent upon technology not hitherto available; solutions follow needs. The days of “discovering” drugs like penicillin are probably past, at least in most breadboxes.
Although this breakthrough, and we should not mistake this - it IS a major breakthrough, is incredibly expensive, the principles and processes developed will lead to more and more breakthroughs. Eventually, with new “super computers” (probably quantum) we will be able to chart individual genomes and analyze them for defects - AND MODIFY THEM in uterus or at the embryo stage - to eliminate many rare and most common genomic disorders.
I have had the pain and pleasure of working with hundreds of such patients over my career and I herald this possibility. It will not happen in my lifetime, but future generations will profit greatly from it.
A note: if humanity would eliminate wastes like war, pollution, poverty, illiteracy and Trump’s extraordinarily wasteful, mindless and tyrannical fascism and narcissistic governance and campaign waste, this would come to fruition in a short time. When Americans demand competence, humanity and excellence, anything is possible. The sooner we get rid of that evil sociopath, the sooner we can cure our children. Yes, another area where politics is critical - make wise choices! Think how all those millions it costs US for The Dotard to play golf could cure others!
25
We have tens of thousands of kids who’d be fine if they got their shots, checkups, glasses, a bit of behvioral therapy, removal of lead from their house, decent meals,and so on. That stuff’s cheap, and has enormous benefits.
And around the world, measles and rotavirus kill hindreds of thousands of kids a year. Also cheap, also easy to fix.
So while I feel for this family, and for Mina, this is an insane way to spend money that could literally save hundreds of kids.
27
@Robert
You're missing the point of the proof of concept that this is...
5
"Parents panhandle $3 Million from strangers to only minorly prolong their doomed child's suffering for absolutely no other scientific or medical benefit"
A fool and their money are soon parted. This doesn't change no matter the circumstances. Call me callous but this literally accomplishes nothing in the grand scheme of things, and actually likely causes NEGATIVE something. The kid is still deteriorating and can't articulate as well as before to boot, the mother doesn't have proper peace of mind, and no advances of note were made in science or medicine, other than making everybody ask "well what do we do now".
15
I'm sorry to say that I would rather be dead than living the severely diminished quality of life that Mila now has. The money would have been better spent on other medical causes that have much broader benefit. Yes, death at an early age is sometimes the proper solution, just as it is always the solution in old age. "Life is a poor player who struts and frets his hour upon the stage" ..... but that's not so of death. Thankfully.
21
@Steven
The other perspective is that they were brave and selfless enough to risk their child to see if it can help her - and yes, maybe it can be beneficial to others in the future. It's a horrible thing to have to choose but it is like deciding whether your terminally ill child will be an organ donor.
I get the impression that this mother would do anything in her power to minimise the girls' suffering.
2
It's hard to have an opinion on this. People spend more money than that on more frivolous things.
4
The Drug saved a life, so why do you write? Are we all so accustomed to the idea of death everywhere, that we find a saved life unusual?
4
@PATRICK :
the drug didn't actually "save a life".....she is terminally ill, but the drug did, apparently, slow things down.
6
Tough choice.
3
Is this Medical Ethics 101?
It would help to realize that post-WWII medical ethics evolved in with the example of the Nazi gnadentod program, which executed adults and children with diseases like mental illness or diabetes, in which their cost to society was greater than their economic contribution to society.
Hitler was inspired by the American eugenics programs in which mental defectives were sterilized under many state laws. As Justice Holmes wrote, "Three generations of imbeciles are enough".
What seemed like a good idea to rational economists turned out to be unpopular, and in Nazi Germany, that's saying something.
So these days, when you ask, "Is it worth keeping this blind, mentally retarded child alive at a lifetime cost of $3 million," you'll have a difficult time finding people willing to pull the plug. (Try Stephen Miller.)
As a practical matter, doctors give a lot of deference to the family. But there are limits -- if they decide that this is a case of "medical futility," they might pull the plug. These decisions are also influence by economics, to a degree that many medical ethicists would prefer to mumble through. Ariel Sharon, yes. Jahi McMath, no.
Giving them their due, Nazi executions -- morphine followed by potassium chloride -- were much more humane than modern executions in Texas.
1
@Norman :
In some cases, the Nazi's just allowed "defective" kids (think autism, down's syndrome, mental retardation, behavioral issues, deformities etc who were actually turned IN to be euthanized by their brainwashed parents, ) were allowed to develop pneumonia and not treated for it, so they died. NOT a "nice" way to die.
The Jews,( including children) Gypsies (including children) and Gay men killed of course were mostly shot and gassed, neither is very pleasant.
The gas chambers still have finger nail marks in them, where people tried until their last breath to escape but could not.
4
Take the trillion dollar tax cuts given to big business and spent it on health care!
10
@Kevin Bitz Sadly, given the current state of our healthcare system, the only beneficiaries would be giant hospital systems, Big Pharma, and insurance companies. A broken system would still be broken.
1
@BA
which is why, and I say this as a retired RN, our health system IS broken, it functions to further enrich the CEO's of big pharma, of hospital corporations and insurance companies as you rightly said.
We must have some form of Universal health care, as does every other developed nation in the world, for all citizens.
Why not take funds from the 1% (especially those like Amazon who haven't paid taxes in years), the military/industrial complex? We could STILL destroy the world 10x over even doing that.
We must VOTE for those who will help this occur.
4
Mila is just the first child to receive this drug. There will be others and one day a case will be caught early enough that a child will be cured instead of dying a slow and miserable death. That will be an outstanding outcome- miraculous even- though the outcome for Mila may not be that great.
9
@Andrew Wrong. This drug will only work for Mila, as it is designed for the extraneous DNA unique to Mila. No other children will benefit from this drug.
Other children with the same illness will need another $3 million (and some change) to get a drug designed for their unique DNA.
5
It's sad very sad, but Sorry, it's a high cost for a child or anyone that will never have a functional life, it this more for the parent or the child? Would you want to live a life like that because some one doesn't know when to let go, just think of that poor lady in a coma for all those years.
14
I cannot imagine what this child and her family are going through. I cannot fault what happened. I can wonder how many children's lives could have been saved with known treatments and access to that money. It's a real question of what to do with all of our resources. Someone benefits and someone doesn't. Being in the right place at the right time with the right resources is the answer. A question with no right or wrong answer.
3
Remember when a DNA profile cost $10,000? A cost accountant will tell you that most of the cost for this was because everything used to develop it was single use. If we paid for a new factory to build a single car, the price of that car would be in the tens of millions. Spread that cost over millions of cars and the cost per vehicle drops dramatically. Eventually artificial intelligence and future technologies will bring down the cost of custom treatments. Until then let's hope that prenatal care will detect these genetic abnormalities early enough in the pregnancy for the mother to consider whether or not to terminate the zygote.
7
@D. C. Miller: Teminate the zygote? There is no test in the world that can detect anomalies so early. I hope you're talking about detection early enough to abort the fetus ... if that right even exists beyond the current term of the Supreme Court.
2
@D. C. Miller “Until then let’s hope....”? By then, “terminating the zygote” may not be possible in this country for any reason at all, let alone because of a future of guaranteed suffering. (Unless, of course, you have money.)
3
I am curious.
Does Mila's father (unmentioned in the article) agree with Ms. Vitarello's approach? It would appear she lives with her mother and another child. The father is present in the video shown on the foundation site but is not mentioned in this article or her blog.
Looking at her foundation's website and her blog I am impressed with the heart tugging presentation but I cannot help but think that Ms. Vitarello is making it seem as if Mila is far better off than she appears and that her disease affects many others.
While the approach taken to help Mila may have wider application to other genetic issues it is not clear that is any of the money raised is being spent on treatments for anyone besides Mila. The basic approach is being touted as a path for others but how many others are on that path and how far have they gone?
I realize that one presents the best possible case when fundraising but would she have raised $3 million if she stated that this was dedicated to treatment for ONE case where even after treatment the patient was only getting worse - far worse than is shown on her site.
15
@cynicalskeptic Having followed this story for several years and read all the GoFundMe updates, hundreds of thousands of the money was indeed donated towards an effort for a broader clinical trial for more children and the disease in general. The campaign started off as being general for the disease and ended when the personalized opportunity arose and the other project languished. The story is a bit of an oversimplification. Its also likely they don't want to highlight the other contributions as the results have yet to be determined or the programs have not yet progressed enough.
1
The method will be used to develop drugs for other patients with related diseases. This will be mostly automated, bringing the cost (in expert people, as well as dollars) down. It will be like genomic sequencing, first done on one individual, and now almost routine.
8
It is not true that Mila's drug benefits only her. Every new technology is expensive, until its workings are better understood and systematized. The public value is in the method used to develop the medicine, which may become more efficient over time, lowering the cost and complexity of developing medicines for others in the future. Remember when determining a genome seemed impossibly expensive? It is routine and inexpensive today.
If custom drugs proved to be an exorbitantly costly dead end, the time may come to stop developing them. Until then, while their promise is being studied, we should patiently withhold judgement, lest we needlessly prevent a future of inexpensive, customized, more effective medical care.
1
I think, as a society, we should do everything we can for each member of it. They raised the money for this treatment, and the child is having some positive response. I also believe that this kind of research is not a zero-sum game. There will very likely be insights gained that can be exported to other types of treatments.
I think those who think solely in terms of the expense of this are missing the bigger picture. Just as there were so many people who thought the moon landings were a waste of time and money. I disagree.
Ultimately, people are driven to help those they love. To help mitigate their pain and suffering. And I don't think playing the hypothetical "that money could have gone elsewhere" argument is very productive in this case.
It is with military spending, etc, but not here.
7
@Chicago Guy
You have a warm heart which is to be commended. BUT we DO NOT have enough resources to help all the children with possibly terminal illnesses. (thinking mainly of kids with cancer). Many of them WOULD benefit greatly from the $3 million used to ?slow down? this child's terminal disease. MANY of the kids with cancer COULD be cured.
"Facts About Childhood Cancer - National Pediatric Cancer ...
https://nationalpcf.org › facts-about-childhood-cancer
43 children per day or 15,780 children per year are expected to be diagnosed with cancer. Only 4% of the billions of dollars that are annually spent on cancer research and treatments are directed towards treating childhood cancer. The average cost of a stay in a hospital for a child with cancer is $40,000."
3
Innovation occurs over time with multiple progressions in a field. Every case such as this will be documented and analyzed leading to new insights and technology. Many cases will be understood in the aggregate and will provide great leaps in science and curse for millions, not just one. One day, there may be a machine that analyzes then manufactures a chemical formulation that mimics a cure found in nature. within minutes for each patient.
4
Regrettably, my keyboard is cursed, not millions as appeared in my comment.
This case will lead to cures for millions.
It must be a Trump Republican anti health care keyboard.
2
Our country is going broke with our overpriced healthcare system and yet we are inventing even more expensive products for the industry to push at the public? Somebody at some point is going to have to resolve the cost-benefit dilemma and have an answer to the question: How high are we willing to go to treat a disease?
Perhaps the medical breakthrough we need is for someone to discover how to deliver basic healthcare to millions of people for a reasonable cost, and then we can turn our attention to the corner-case diseases.
14
One of the most common arguments against socialized medicine is that profit fuels innovation - In articles like this, with the passion and dedication of family and doctors to follow the science to a treatment in a rare condition that benefits only one person, it seems more to me that researchers and doctors would be free to pursue real innovation and medical advances if not in harness to the demands of the market that demands the next cosmetic mass produced and highly profitable drug. Take profit out of healthcare.
6
@Megan The the commentary above yours by Scott Werden. The same was said about the space program that gave us cordless tools and Velcro.
@Kathleen : I would rather live in a world without Velcro and cordless tools and in a world in which all money spent on the space program had instead been used to eradicate poverty -- which absolutely could be done, but hey, it's not a very sexy sell.
Scientific breakthroughs like this never have any benefit for the enormous, and growing, American underclass. And anyone who thinks the underclass isn't worth caring about should read a little history, to bear in mind what happens to societies in which income inequality spirals out of control.
Guillotine, anyone?
2
According to the article, there are 7000 rare diseases. Assuming they all cost, as this one did, 3 million dollars to research and produce, that’s 21 billion dollars for a drug for each of these diseases.
We spend more than 700 billion dollars on the military each year, engaged in useful projects such as blowing up villages in Afghanistan.
Perhaps we need to rethink our priorities.
55
@Serrated Thoughts
We spend about $3 trillion each year on medical care, 150 times this commentor's estimate of cures for all those rare diseases (most of which won't be developed for decades because the scientific problems are unsolved).
Much of the $3 trillion is spent on diseases that people bring on themselves, voluntarily, with bad habits they could avoid: smoking, eating junk food, alcohol, etc.
2
@Jonathan Katz
I just love the "blame the victim" pov....(sarcasm).....genetics plays a role, even in those diseases you call "lifestyle"....yes, no smoking, no drinking, exercising and eating "right" would stop a lot of diseases, but lets talk a minute about childhood cancer.
"Facts About Childhood Cancer - National Pediatric Cancer ...
https://nationalpcf.org › facts-about-childhood-cancer
43 children per day or 15,780 children per year are expected to be diagnosed with cancer. Only 4% of the billions of dollars that are annually spent on cancer research and treatments are directed towards treating childhood cancer. The average cost of a stay in a hospital for a child with cancer is $40,000."
"Image result for Cure rate of childhood cancerwww.acco.org
Survival rates can range from almost 0% for cancers such as DIPG, a type of brain cancer, to as high as 90% for the most common type of childhood cancer known as Acute Lymphoblastic Leukemia (ALL). The average 5-year survival rate, not including children with ALL, is 80%.
Childhood Cancer Fact Library, 2018 - Coalition Against ...
https://cac2.org › childhood-cancer-fact-library-2018"
BUT it all costs money....lots of money. How far would the $3 million raised for this one child, NOT to cure her, but to slow down her symptoms, have gone?
2
@Jonathan Katz : Maybe take a break from blaming ill people to remember that
* obesity is linked to low income -- the cheapest, most filling foods are the least healthful and the highest in calories;
* incomes for the middle and lower classes have stagnated for the past 40 years, so that's a lot of people relying on a lot of garbage food;
* subsidies to American agribusiness mean that chemical-laden foods, which our bodies aren't designed to process and which are screwing up metabolism, are ubiquitous and cheap;
* air and water pollution, which none of us can control and which are worse in poor
neighborhoods, are changing our bodies in ways that we don't yet understand, and it's probable that metabolism is negatively affected by these changes (the average person now consumes about 5 grams of plastic -- the weight of a credit card -- per week because so much plastic is in our food and water supply); and
* we have approximately 1/10th the number of treatment slots we need for addiction treatment.
So maybe you'll want to support a robust, Canadian-type universal healthcare system that includes education and prevention, in order to reduce the costs of treatment. Or just keep blaming people who are struggling to do the best they can in a system that fails them daily.
Your choice.
9
Whether or not Mila is the only person who benefits from this drug, medical science benefits from this drug. The principles used in developing this medication improve our understanding and may lead to many additional drugs that may have broader use. Knowledge is powerful. The fact that an obscure problem was identified and treated does not mean that the knowledge gained can not be used to cure less obscure illnesses.
49
“What type of evidence is needed before exposing a human to a new drug? Even in rapidly progressing, fatal illnesses, precipitating severe complications or death is not acceptable...”
If I had a rapidly progressive fatal illness ( such as Creutzfeldt-Jakob Disease, or something similarly horrible) and was otherwise healthy and young-ish at baseline, I’d absolutely want to try a novel medication, even if the medication killed me. I mean, I’d die from the terrible disease anyway, so why not advance knowledge ....and maybe even hit the jackpot and be cured.
As long as the patient /family is aware that the experimental therapy might kill them faster than disease itself would - and they are okay with that - then go for it!
38
@GBR--From my reading of the article, it doesn't sound at all like Mila has any understanding of either her disease or the drug or indeed that she's even sick. There doesn't seem to be much evidence that she's cognizant of anything at all. This money was raised and the drug was developed because her mother wanted it, not because Mila did. It's impossible to know what Mila would have wanted. Besides, Mila is a minor and her consent isn't even required. It seems to me that an impartial child advocate should have been assigned to Mila to speak for her, much as is done in court cases. There's no mention in the article of whether or not Mila's wishes were considered at all. She is just being experimented on, without her knowledge, or apparently her consent.
13
@Ms. Pea - Great point and I 100% agree w you. My comment was meant more generally, in response to the passage I quoted as the start of my comment (from an FDA official. )
3
The tradition of allowing parents to decide about their children's best interests is to be abandoned, so all may benefit from the vast wisdom of the State?
5
Please explain in detail the difference between a drug and therapy in personalized medicine {"custom piece of RNA to block the effects of the extraneous DNA" vs. "CAR-T cancer therapies, while individualized, are not drugs").
What are the patent rules differences between a drug and a therapy regarding the above personalized medicine?
Okay, so what happened to the $3million?
1
@Joel H CAR-T therapies are whole live cells so they are biologics which are regulated by a different center of the FDA (CBER). Anything that is living , like a cell, or blood, virus or a bacteria (as in fecal transplants) is a biologic. Non-living things that are manufactured chemically such as small molecules like RNA and chemicals are drugs. Drugs are regulated by CDER. The regulations that cover these centers are different so it is a big deal when a treatment is cleared from each center.
12
@Joel H
Every person has T-cell lymphocytes that have the ability to fight cancer. In CAR-T treatment, a portion of a person's T-cells are modified to attack the specific cancer attacking the patient. The therapy is applicable to many different cancers, but the T-cells created are specific to the individual's cancer. It's a form of immunotherapy.
7
@Joel H CAR-T are specially engineered cells, created by genetically modifying native T-cells (a kind of immune cell) taken from the patient. This is currently a personalized therapy because the immune system will reject cells from other individuals, which means these engineered CAR-T cells must be made from cells taken from the patient who will be treated with them. It is not technically a drug because it is not a single molecule, but is instead composed of cells, which can replicate themselves in the body. This kind of therapy saved my husband’s life this summer when his engineered T-cells destroyed an aggressive B-cell (another kind of immune cell) lymphoma that was otherwise untreatable. His clinical trial treatment at Stanford Medical Center was funded partly by Kite Pharmaceutical, which engineered his cells, and partly by our Kaiser health insurance. We are immensely grateful for the federally funded research that led to this breakthrough treatment.
10
Is Mila being kept alive for her mother or for herself? Is there evidence that she enjoys life or even understands what life is? What future does she have? I can understand that her mother wants to hold on to Mila for as long as she can. But, when does the well-being and quality of life of the patient take priority? As hard as it is for families, sometimes they have to face the reality that what they want may not be in the best interest of their loved one.
44
@Ms. Pea We're all being "kept alive". None of us knows what future we have. Unless you want Mila euthanized, I don't understand your point.
4
@Ms. Pea
"What future does she have"?
The last photo shows Mila with a smile. That speaks to a certain quality of life that would be enough for me to do everything I could to give her another smile.
9
@Ms. Pea and who but their parents are qualified to make that choice? the state, religion, your perception of someone else's perceived value and enjoyment of existence?
Your comment lacks all empirical reasoning and seems more a projection of individual imagined fear of what one imagines it would be like to be in similar circumstance. None of us can see the whole picture of what a person's life is worth or decree what value or purpose it has.
8
Personalized medicine, like everything else in America, can be had if you have the money.
19
@Jacquie
Very true. In more socially advanced societies, however, where healthcare insurance is regulated as a greater good and innovative medicines are more generally accessible.
Our healthcare system is broken, not because of drug prices (which fuels medical innovation), but because of the middle-men, the private insurance companies that suck billions of dollars out of the system without returning any value or benefit to society.
7
@Jacquie
It's that way all over the world. So?
@Rea Tarr
No, actually untrue.
EVERY developed nation in the world has some form of universal health coverage for all citizens.
Read about the escalation of the cost of Insulin, required to keep diabetics alive......
"Quinn Nystrom, a leader of T1International's Minnesota chapter, said on May via Twitter that the price of insulin in the United States per vial was $320, while in Canada the same medication under a different name was $30.Jun 29, 2019
American caravan arrives in Canadian 'birthplace of insulin ...
https://www.reuters.com › article › us-canada-health-insulin › american-cara..."
2
It is likely that more efficient methods of drug development or other treatments for rare diseases will emerge in the future.
4
Faster please. This poor little girl.......
3
I scanned through the NEJM report. To me, this treatment wasn’t as effective as described in this report. 7 out of 11 measures didn’t improve. Her brain volume kept decreasing. I don’t think this report is as accurate as it should be and gives false hope for other patients. Social and ethical issues put aside.
72
@KO
The point of the article is that there are no other patients. The DNA fragment that was identified is unique to Mila. The RNA used to block the DNA is specific to Mila's abnormal DNA. and has no use for anyone else. The knowledge gained may be of profound use to others. Her response is significant. Correcting what caused the problem may not reverse existing damage.
8
@KO People have awakened from coma after decades. Miracles sometimes really happen, and hope is a blessing for the blessed people who care for those they love.
@KO
Would it be ethical to prevent the mother from buying treatment for her child? Should parents have to put it to the public to vote on before they make healthcare decisions for their children?
3
This the future of medicine — personalized treatments. Pioneering the method is always risky and expensive.
5
@J. R. Wakeland
Personalized medicine? for all? What world do you live on?
A good number - perhaps half - of the world's current population is not getting ANY regular medical care.
The future of medicine seems to be LESS care for those that supposedly can afford it. That has been our experience.
7
As long as there are more people who get PhDs in biology than there are academic institutions to accommodate them or research dollars to fund them, there should be no objection to private dollars pursuing custom solutions for individuals that can come up with it. If it keeps more biologists working. Great.
A waste of money? If it's private, and given voluntarily, you could just as easily argue that that money would have been spent on more clothing or restaurants as on curing another disease.
Perhaps the gov't could facilitate establishing a connection between people with rare diseases and people who might be able to help, by having a federally funded website.
7
@DebbieR : You're mistakenly assuming that private money is somehow sacrosanct -- that people have it only by dint of hard work, whereas they have it because of a hideously skewed tax code and because of rich people's anti-union activism.
In the US, 1/10th of the top 1 percent have as much wealth as the bottom 80% of the population -- roughly 30,000 have as much as roughly 240,000,000. That's not only mind-boggling, it's an invitation to a bloody revolution.
Let's return to the Eisenhower-era tax rates -- remember good ole GOP Ike, and the broader prosperity of his era? -- and let's implement a Canadian-style universal healthcare system. Then we can debate how money should be spent on medical research, and where it should come from.
What is expensive and custom today becomes cheap and affordable tomorrow. The very rich fund this technology because they can. The poor wait for it’s development. That’s how any technology has worked since the beginning of time. It’s not right or wrong, just the way it is and always will be.
20
@Gabel spoken like a true american. just the way it is?? How can poor people living shorter lives be RIGHT? Because something has always been so, makes it RIGHT? How utterly appaling. From a country which spends billions in military, more then the next 19 in the list combined.
I am in Canada and, speaking with my US friends on healthcare, and the cognitive dissonance is mind blowing. The very concept of paying for healthcare is alien here. The peace of mind it brings, priceless.
My son was in intensive care for 100+ days. It cost me nothing. It cost the system 200k. It coulw have cost 500k+ in the states, with its inflated artificial costing. My family would have been ruined.
4
@Gabel Yes, that is why million dollar homes from twenty years ago can now be built for $200k. O, wait.
2
America must make a Sophie's Choice decision and it will be heartbreaking: which treatments will be public burdens and which will be private burdens. The ability of science to cure disease is a scientific challenge. The funding of such research and treatment is an economic challenge. They overlap but are not the same issue.
Even socialized medicine will not solve the dilemma, because no conceivable socialized medical model will provide funding for this type of situation.
12
@Mel To me it appears that a lot of drug research is funded on the backs of the US taxpayer anyway, without any socialized medicine in name.
A huge argument from a lot of pharma apologists is that drugs cost more in America because we have to fund all of the high risk Pharma R&D- if that is really the case then we should just nationalize the entire industry.
In effect drug prices are high in America because Pharma companies keep raising prices and we keep buying them at the higher prices.
19
@Hugh G
Yes, gauging does occur, but risk adjusted issues are real. If investors can't get at least the same amount as, say, hula hoops- they will invest in hoola hoops.
"The government can nationalize the industry". Aside from the legal impracticality of this, there has never been fully government run drug development that has ever worked. Even the Europeans & Japanese maintain private companies.
Even governmental run drug development would run into the same issues that plague private development: For, say, every 10 or so drugs in development, only one succeeds. This means whoever invest in the 10 drugs needs to be compensated by the one that succeeds. The government will have to come up with that same risk adjusted money as well- and there is no assurance that it will be more efficient.
There are also differences in the generic and patented market. 90% of the problems are in the generic market, where regulations can be as brutal as necessary since developmental cost are already paid for. Insulin and Epipens fall in this category. Problems here to need to be resolved perhaps by revising the patent code, using large scale negotiation and unlimted importing from approved countries.
Calling people who have real questions about some of the radical reforms "apologist", is rather dissapointing. As someone with several illiness, I personally don't need people questioning my motivations, or destroying the pipeline of drug development with untested economic theories.
1
@Nerka
I too have a life threatening chronic disease, very expensive meds required for it.
I do NOT see universal health coverage, which every other developed nation has, as an "untested economic theory"
For instance
"Quinn Nystrom, a leader of T1International's Minnesota chapter, said on May via Twitter that the price of insulin in the United States per vial was $320, while in Canada the same medication under a different name was $30.Jun 29, 2019
American caravan arrives in Canadian 'birthplace of insulin ...
https://www.reuters.com › article › us-canada-health-insulin › american-cara..."
1
The goal should be that by 2035 individualized drugs and therapies should be cheap and common. If that is the goal we need the US FDA, the government, and a lot of private (and yes rich people) to step up today and get the ball moving.
Restricting this either due to a false sense of interfering nannyism or because the sheer expense gives it the illusion of unfair will prevent every day people from access in the future.
For perspective: remember for a poor child in a third-world country a 10,000 USD drug is the same as a 3 million USD drug. Completely and totally out of reach.
7
So much love and compassion for Mila and her family. She has an incredible mother and advocate. We are cheering you on. Thank you for sharing your story.
18
@Vera One more thing. I can't help but imagine how Ms. Vitarello would feel if she read many of the comments here, people telling her she should have let her child die when she had the opportunity not to. It is privilege for sure, but I haven't met a parent who would not take the opportunity to try to help their child. We leave in her hands, trusting her as a mother, to decide what is right for her family. I can imagine where the mind would wander from that statement -- I am not an anti-vaxer. Quite the contrary. But a story like this begs your compassion, and asks you to see that a person trying to save her child is a person who deserves our trust.
We are in a trustless, judgmental place culturally, and I hope that we can work together to change that trend toward trust and compassion.
I also hear in these comments that many people are suffering and asking why they don't get the same level of care. You deserve it -- everyone deserves extreme care and dignity. Let's use our effort and energy to extend the care demonstrated here to everyone.
11
@Vera :
sorry but to be honest, if you read the article, ALL the medication is doing is slowing down SOME of her symptoms. She is still helpless, can't speak and is terminally ill.
HOW many kids with cancer (some forms of which are now curable) would have been helped with this same $3 million? We just don't have unlimited resources.
2
@RLiss You're right, there's a lot that could be done to help others with that money. I am also buying the arguments that research like this could help a lot more people down the road, even if it only helps one person now. Who knows what cancer treatments seemed niche and exorbitant a decade ago that are now treating many more? We don't really know.
And for a mother caring for her child, watching that child go from dozen(s?) of seizures per day down to zero to six is a pretty meaningful quality of life improvement.
1
When a new drug or procedure is successful, greed takes over and seals the fate of how it will benefit society.
Yes, to some extent unique and customized medicine can benefit a lot of people but medicine in the USA has the end goal of profits.
Excessive profits is the real disease in this country.
I won't be surprised if pharmaceutical companies have already gotten their lawyers involved in patenting this particular process that benefited Mila.
4
It is beyond wonderful that medicine has advanced to the point where the cause of a specific disease or genetic problem can be diagnosed and possibly treated by creating RNA that can correct the protein formation problem here.
This is an advance that potentially can help many.
The question then is raised as to how limited resources should be expended; which is the question raised by this article. There already is in place a principle called triage that states how one should proceed when resources are limited and cannot provide for everyone.
Triage would dictate that the limited resources should be applied where there recovery or at least some functionality will be possible and perhaps the number who can benefit from a particular treatment should be a primary consideration so that if the same effort can be used in helping 100 instead of 1 than help the 100.
More importantly, in the current political climate in which the Rs are in court pressing to have the ACA declared unconstitutional and ended; where 20-30 million more are uninsured in the last 2 years & illusory skinny insurance policies that provide almost no coverage are foisted upon the gullible it is impossible to advocate for individual tragedies while ignoring mass suffering.
Stalin said the death of 1 is a tragedy while the deaths of many is a statistic. I believe we must focus on the many so the many are not reduced to a statistic even though Mila's story is heartbreaking. This world is not perfect or kind.
5
We are just at the beginning of being able to understand individualized therapies. This is a step in the direction of being able to make a drug/medication/treatment regimen that works perfectly well for a specific person. This is amazing. Our scientists need more support for this kind of research so it can be used far and wide. Thank you beautiful Mila.
11
As one of what seems to be a fairly large number of people with a "rare" disease subsequent to taking a statin drug, this article is of particular interest to me.
The US has no compulsory system for reporting adverse effects of medications, so "rare" isn't founded on anything but intuition. I don't get out much now, but I've happened to meet three other people locally with painful, permanent, and progressive statin-triggered diseases. We've all lost the ability to walk more than shuffling around the house. Two of the others are MDs. Maybe not so rare.
At least one statin-triggered disease has been diagnosed: Statin-Associated Autoimmune Myopathy, NEJM. Also, there is a DNA test (about $100) to determine if one is up to 17.5 times more likely to have problems from a statin.
We know the diseases exist, and we know statin drugs trigger them. We know that there is a relationship, at least in some cases, with at least one gene (SLCO1B1).
With 35 million or so people taking statins, it's certain that there are thousands of us with permanent, and sometimes extraordinarily debilitating and painful diseases.
As wonderful as it is (and it may even lead to help for people like us), the custom drug in the column is helping one person. I wonder why there is no equivalent research into treating, perhaps even curing!, the horrible diseases we have. Yes, we're older, but we had at least 1/3 of normal life span ahead of us.
Are you one of us? StatinStories.com to help.
7
@sing75 I took statins for a short period and realized that the side effects were far beyond what I was willing to undergo.
3
@Ernest Montague Please send the details to statinstories.com. I only took for 7 months, but that was enough to trigger a permanent disease. We all differ genetically. Let's try to learn something from what's happened to us.
I shouldn't have even read this since having a child of my own.
I am grateful to everyone who donated to this cause. I realize that the money could save however many babies farther away. But do we really want to live in a world where people always act based on those calculations?
My heart goes out to Ms. Vitarello and I hope Mila's recovery outstrips all her hopes.
27
What stood out for me in this article was the quote from the mother “She is starting not to respond to things that made her laugh or smile,”. In addition the child has lost the few words she had. At what cost do we keep a child alive but in doing so gravely diminish the quality of their life.
82
That people would donate money to this lost cause rather than simple treatments for thousands of children dying in the third world of preventable diseases says a lot about the psychology of empathy. They see a picture of a white child and a relatable mother and give; they see images of poor children of another race and their wallets stay closed. They figure it’s someone else’s problem to solve, and they won’t get the same fulfillment because they can’t fix the whole problem.
26
@sedanchair We have no idea who donated, and in what large amounts. They could have been mostly family and/or community members coming to the aid of a friend and neighbor. And we also have no idea what other charities any of these people contribute to. It's gratifying that people were willing to give so much to try to save this child. The fact that millions of others still need help need not diminish the good done here.
11
@MD No, your point is invalid. There are studies on this, people are bad at extending empathy to large groups or comprehensive problems. This was a waste of money, a disgrace to medicine and a thumb in the eye of people who would benefit from treatment (there was no benefit here, only the extension of life without consciousness).
2
@sedanchair I have a question, couldn't your logic be used whenever someone donates to fund someone with a terminal illness? Let's say when you get a GoFund me request for a colleague to fund medical expenses/therapies for their child (or themselves) with terminal cancer -- where you are really talking about extension of life vs. curing. Do you just respond to those with "sorry, your kid is going to die anyway, so I'm instead donating to help cure a preventable disease"? I would guess you (or many people, at least) still donate to their friend or colleague. And many of them may also donate to organizations doing work re preventable diseases in the third world or elsewhere. I don't think you can assume it is racism that drives their donation to help someone's child. Maybe it just isn't always so simple to say that you do not want to help a friend save their child -- even if you might think you might make different care decisions if it was your own child or loved one.
3
Journalism is both words and pictures.
This article feels like an advertisement in favor of personalized drugs. The images feel very professional.
Why isn't there also an image of a poor child in a hospital who also has a rare disease but no funding?
I feel for Mila and her mother (especially after seeing I saw these touching images) but when I close my eyes, I see the massive divide between the institutions that cater to the ultra wealthy, and the rest of us.
43
While i applaud the science, it is a monumental waste considering that the end outcome of the disease will be fatal. the money is better spent elsewhere.
22
@Rhsmd1 The money went to help research the entire process, which can help other people in the future as well.
7
@Rhsmd1
"a monumental waste considering that the end outcome of the disease will be fatal"
Life is a fatal disease.
...Andrew
2
I'm a nurse at a large hospital. Reading this story made me cringe. I see a parent forcing her child to suffer a horrible life that isn't truly a life. From my career caring for people including those at the end of their life I can tell you from my heart: Some things are worse than death. This looks like one of them.
196
@navybrat
Mila is not your child. If you wish to make a different decision for yours, that's fine. But each of us must make the best decision we can as regards our own. That you would make a different one does not take away the ability of others to make that choice.
"Some things are worse than death." Are you advocating euthanasia?
7
@navybrat
In times past the village priest would say 'Let them go to God'.
14
@navybrat I don't know. It's extraordinarily difficult to make that judgment for someone else.
4
Over $3,000,000 to maybe keep 1 kid alive who after treatment
* is still blind
* still can not move
* has lost the last few words of her vocabulary
* is becoming non-responsive - “She is starting not to respond to things that made her laugh or smile,
*and remains severely disabled.
She will need 24/7 care the rest of her life - at a time when nursing homes of that care level run $100,000+ a year.
I wouldn't keep a dog alive in that condition. I would be doing it for myself -not the animal
Now think about the costs of doing that for 10,000 or 40,000 patients x $3,000,000
$30,000,000,000 for just 10,000 people or $120,000,000,000 billion for 40,000.
Total healthcare spending in the US in 2017 was $13,500,000,000,000 -about $10500 per person.
That $3,000,000 on 1 patient would have paid the healthcare costs for 285 people & such costs for 40,000 patients equals care for 11,400,000 people
1% of the US population is 3,290,000 -10,000 or 40,000 patients are less than 1/329 - 4/329th of 1% of the US
In the real world a couple in their 70s near Seattle were a murder-suicide because they could not afford the copays deductibles under Medicare for the wife's illness. In 2016 they had filed for bankruptcy due to medical bills -owed $212,000 after 6/7 years of Medicare. In 2019 they died because they could not pay the medical bills coming in.
No Go-fund-me for them -they just died. What was spent on 1 kid would have paid their Medicare copays for 99 years.
168
@AnnS It's tough being a rational person in an emotiionally driven world. Just saying what you said above can make people incredibly angry because they feel like you are being sensitive. But I think this is an incredibly important point: we should focus on global optimization, not local optimizations that make us feel good about one person being "saved" (and as you point out, it's hardly saving the person, just signing up the family for a huge expense for the rest of her life).
50
You're right. I wouldn't want my child, my parent, my cat, or myself kept alive this way.
But if the child serves as a useful experiment, and researchers can apply what they've learned to other diseases, then keeping the child alive these months might have been worth it. I suppose her parents are learning now to let go.
Like you, I think that there is a time we all have to let go of creatures we love even when doing so breaks our hearts.
13
@David Konerding local optimizations need to be done before global ones can even be considered.
Ford may have revolutionized the production line and made cars more 'global' for all, but that's ignoring all the 'local' prototypes that also certaintly came with initial high investments. This is a prototype for medicine in the future that one family was able to invest in.
8
There are many in the world that have to pay out of pocket and do not have medical reimbursement. They face this issue every day. Many countries in the EU will not reimburse CAR-T therapies today.
It is a useless question to ask if it is "fair" to receive these more cutting edge technologies. We should ask, "how do we drive down cost of novel therapeutics so that they are accessible".
The fact that her parents could raise money for this treatment, and the scientific developments demonstrated here are incredible.
11
This child’s need to evaluate wether they are doing this for her or themselves. It appears the quality of life of this child is very poor.
28
@Paulie By all measures her "quality of life" is much, much better after taking this medication.
8
@Paulie It's the life she has and she appears to want it. I don't understand begrudging it. No one is asking you to live this way.
2
So what is exactly your “solution” here then? Euthanasia? The mother should have just let her child die a natural death (in I don’t know how many years)? She should have not tried a new treatment that she hoped (all parents ultimately hope for a cure, even if they are realistic about the chances) would cure her daughter?
I agree that some diseases can be “worse than death” but does that mean you can’t even try? What about the next patient with a similar disease? That’s how medicine advances. It may not save you because it comes too late, but the knowledge gained may save the next person.
4
A millions of dollars miracle cure for one child who may succumb regardless? What about the millions of other children dying? Sorry to be cold about this because I do have daughters -- but this seems the height of our self-absorbed narcissistic culture.
21
@Mary Rivkatot There is nothing less self absorbing than wanting your child to live and thrive. You obviously missed the boat on personal responsibility and compassion for one's family.
12
@Mary Rivkatot -- While I'm a mother and am unsure I would follow this path if (god forbid) I had to confront it -- it is NOT "narcissistic" to want your child to live.
13
@Nature Voter Compassion is fine, but realize that many other people with less severe injuries will now have worse health outcomes because of the immense resources.
We should make our health decisions rationally and globally, for the good of the most people. That's more compassionate, actually.
5
For those who want more scientific detail, Science magazine had a story which was more technical but not yet incomprehensible.
https://www.sciencemag.org/news/2018/10/tailormade-drug-developed-record-time-may-save-girl-fatal-brain-disease
7
In the US, most graduate biomedical education programs are free and include a stipend for living expenses and, at least in NYC, also access to housing. Nonetheless, we face a shortage of qualified, committed, and motivated applicants able and willing to undertake the lifelong commitment to pursuing innovative science in the currently challenging funding environment. The current restrictions on training foreign-borne students adds to the shortage.
7
@David L the shortage is market driven and has been for decades. NIH funding levels are close to single digit; chances of getting a tenure track faculty job are very small; for physician scientists, the lure of private practice income and not having to write grants is not insubstantial. altruism and passion for science are a large part of why people pursue biomedical science, but sometimes that's just not enough. then, there's this whole thing about respect for academics and higher ed. not really there any more......the model needs to change.
17
@David L, I used to work in biological research. The lack of money and jobs drove me out. The politics were crazy and getting a grant was next to impossible back in the 1980s unless one was an established researcher. We can thank St. Ronald Reagan for this situation. He and other political figures have no understanding of how science works and they don't care. As far as they are concerned only the "elites" understand science and that alone makes it not worth funding.
23
@David L Mother of a PhD in molecular and structural biology here. Both his PhD and generous stipend were paid by the National Science Foundation and he did research in a national laboratory. After his post-doc 6 years ago, he has never worked in his field. He said there were no research jobs in academia due to Republican budget cuts and private companies do not want to hire PhD's- they prefer to hire BS's and train them in the company's proprietary systems. He said that even during grad school the majority of his time was spent grant money-grubbing not doing research. He said the only applications for money that seemed to get approved were ones that mentioned the research could lead to a cure for AIDS. Our current grad school system leaves the committed, qualified students burned out and highly motivated to find another line of work.
9
As a parent, I would do anything for my child. Sometimes that means letting go.
183
I agree that sometimes you have to let go but wouldn’t you even try once? How many treatments would you try until you quit? What if the drug had cured her, would you think the same way?
8
@Patricia I've had to actually make this decision. The condition my twin daughters had is universally fatal. The longest lived child with the condition was three, suffered massive grand mal seizures multiple times a day and required massive medical interventions to survive as long as she did. The doctors could not tell me if our babies even dreamed. They were never conscious.
After two weeks in the NICU, we received the diagnosis. We took them home, along with hospice and let them go. Even had they discovered a cure the next day, it was too late for my daughters, Eleanor and Quinn to have had anything resembling a normal life. The damage was done.
Sometimes, no matter how painful it is, there is no control over a situation, no miracle cure and no options. The only compassionate response is to let go.
78
Mila is the first one with this illness to be treated. There will be another but if that child is lucky s/he will be able to receive treatment before the illness destroys his/her life. The next child might be able to have a more normal life. That's what the first case does for the second case once a treatment that helps is found.
55
Mika obviously came back from the brink of death but she still bears many scars (physical and emotional). Part of me wonder what kind of life she’d be able to lead. It sounds miserable and joyless. Was she actually saved by the medicine, or did we just extend her suffering? It’s a lot harder to make these judgments with children than adults but I think we should also think about quality of life when discuss healthcare. We often see this with the elderly where when a lifesaving procedure still results in loss of purpose, a natural death is often the more merciful option.
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@NKM How do you know she is emotionally scarred?
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@NKM It isn't for us to decide. We have no evidence one way or the other how this child's life will go. We can't arbitrarily start saying that because someone MIGHT not have the quality of life we preseve as good enough then they aren't worth saving. Also, by going down this path or discovery for this disease, there may be breakthroughs for other discoveries, either in procedures or in outcomes.
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@Teddi
actually as a society it is for us to decide. When medicare4all comes about( it will one day), you had better get ready for rationing of care.
there is NO way that the US can continue to pay outlandish prices for treatments for those who have no hope of survival.
the same can be said for the seniors with limited life expectancy who want hip, knee and back surgeries. say goodbye to dialysis.
Sadly this girl ,who has value to her personal family unit, has no value to society. is she going to contribute financially or educationally to society? No. from an actuarial point of view, she has no value.
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At first glance it would not seem to be viable for government-funded or private health insurers to spend $3MM to develop a custom drug for every person that needs one. On the other hand, I imagine that Medicaid or Medicare do spend upwards of that on persons that require 24-hour nurses or aides or numerous expensive treatments, such as those with severe disabilities or who are in long-term comas, over the course of their lives. If the drugs could save on those expenses, they may wind up being significantly cheaper than the development cost.
Also, the enormous cost may be largely due to the fact that the technology is in its infancy - think of how expensive the first computers were - and could come down significantly as it is refined. So, while it certainly seems unfair that it is only available to a very lucky few at this point (like so many other things in life), the silver lining may be the knowledge acquired in doing so helps make them more widely available.
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@DJG This is a good way of phrasing he payor question. One has to analyze the heatlcare economics of treatment vs. no treatment.
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While it might now seem a disproportionate amount of money to improve a single life, you have to put this in the historical perspective and realize that it is pioneer work.
The sequencing of the first human genome took 13 years, a cooperation of most industrialized nations and over 3 billion dollars. Today a human genome can be sequenced in less than a day for less than 1000 dollars.
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@Hans Rupp yes but the genome mapping helps more than one person. this is selfish treatment designed to placate the parents and their inability to let go.
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@MMS that's how the smallpox vaccination started. Edward Jenner noticed that milkmaids who had had cowpox didn't contract smallpox. He vaccinated one little boy whose mother was frightened he'd die if he got smallpox. The little boy lived, acquired the necessary immunity, and we entered a new age in vaccination.
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@hen3ry :
true, but Jenner knew that if it worked it would save thousands and thousands of lives, which it did.
see: "Image result for smallpox death tollourworldindata.org
The worldwide death toll was staggering and continued well into the twentieth century, where mortality has been estimated at 300 to 500 million. This number vastly exceeds the combined total of deaths in all world wars. In the United States, more than 100,000 cases of smallpox were recorded in 1921.
Epidemics of the Past: Smallpox: 12,000 Years of Terror
https://www.infoplease.com › math-science › health › diseases › epidemics-o..."
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If we have the money to make war, poison water, blot the skies and ruin the earth, we have the money to make so-called miracle cures.
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@Tom or we do not because of those other priorities it is a question of choice, perhaps political choice
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As a scientist and mother of a child with a rare genetic disease this type of work is incredibly important. The research done here can be applied to other genetic disorders, expands our knowledge of genomics, and it changed the outcome for that child. It is unfortunate that in this golden age of genomics that the US government is not prioritizing funding for more work in rare disease in pediatrics. There is a personalized medicine initiative, but like most research, it focuses on issues associated with our older population. The wealthy and well connected have taken it upon themselves to solve funding gaps. I know families that have benefited from this that could not afford or have the connections to do so. If your child suffered from a debilitating disease you would not be claiming Darwinism, you would do everything possible to help them.
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And better even to experiment on this one child -- for that in essence is what's being done -- than keep my poor late aunt, in her 90s, alive and completely helpless and demented, for years. She didn't know who I was, who she was.
Bless the two aides who fed, wiped, and bathed my aunt and took her to hospital when necessary (or not); but that was a lot of money could have been better spent on medical care for children. Multiply such spending by millions; and she wasn't even hooked up to
machines the way so many others are.
The best thing I can say is that she died in her own bedroom.
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Nothing on the face of the Earth is more important. Little girls are sacred.
5
Is there a price for a human life?
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@David
"The Environmental Protection Agency set the value of a life at $9.1 million in 2010. The Food and Drug Administration, on the other hand, came up with a value of $7.9 million and finally, the Transportation Department said it was around $6 million."
-various sources
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@David yes. We use it to make all sort of decisions, especially regulatory ones. $129000 give or take, though sometimes it can be a lot higher for quality adjusted, with figures over a million. We make all sorts of decisions we know will cost lives or don't do things to save lives because of the cost.
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@David This isn't the answer you're looking for, but many rational economists would base your value on your predicted lifetime earnings, cast back into current dollars. (https://escholarship.org/uc/item/82d0550k). You can think of it another way- divide the total amount of money in the economy by the number of people. The number can't be larger than that, otherwise you'd go bankrupt spending all your money on health care.
The better question is, given a large country with a healthy economy and advanced medical research, how much money should be allocated to each problem (cause of death or suffering) given we have a fixed limit on research budget? We spend most of our money on cancer and heart disease already, and those are the two largest causes of death by disease in the US.
One imagines, in a healthy portfolio you allocate most of your resources towards the most common and addressable problems, but hopefully have enough money left to address rare cases like this. The question is- if you only have $10 to spend, if you could spend $10 to save 100 people from cancer when 1 person dies of a rare disease, or $10 to save one person of a rare disease while 100 die of cancer, or spend $9 to save 90 people from cancer and $1 to help the one person with a rare disease?
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As someone with a rare disease, one of the seven thousand diseases, having a treatment for one of my cohort, would open the way for treatment for many. Looking only at the cost is to be blind to other reasons to develop a one of a kind treatment. Creating anything starts with one and then that is modified until a final version is completed. But anywhere in the development a branch can be made to solve some other issue.
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"But there are nowhere near enough researchers to make custom drugs for all who might want them."
There is a lot of people who want to be researchers actually, but it is very hard for even qualified people to get in to schools that can train them to be good researchers. Too many schools turn down too many qualified applicants. As the world's population increases, diseases will too, and so will the need of doctors and researchers. Maybe it's time for the academia to adapt and make education available to more and more people who are willing to contribute.
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As someone who spent the necessary years getting my doctorate, I disagree wholeheartedly. Programs and labs will often bend over backwards for qualified graduate students who do not meet an arbitrary admissions metric. These “qualified” people who have trouble getting into programs likely aren’t qualified at all and we do not need average people attempting to develop treatments that require above average intellect.
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@Petunia, There's little point in training more people if there is no funding for research. Current funding levels are insufficient to sustain the existing research enterprise. To expand development efforts as suggested, NIH funding would have to be increased substantially from its current $40B and permanently (no one is going to spend years training if funding for future work is uncertain).
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@Dr. Trey I know plenty of people who possess the 'above average intellect' that would make embarking on a PhD path feasible. I also know that many of them are not financially able to pursue that path (because of debt from Undergrad and Master's programs.) Making these educational opportunities available to more people would benefit all of us. Not to promote the unqualified, but to promote the qualified but unable.
America continues to disadvantage the bright who are not economically privileged.
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As a geneticist, I can say that personalized medicine is the way forward - while it seems costly and inefficient now, successes in this field will generate the scientific infrastructure and precedent to generate personalized cures for *everyone* at an affordable price.
It seems every day we see another promising cure for cancer or rare disease that works in a few small trials but fails to be significant in larger studies. One reason these cures do not work for the larger population is that they may only work for small sections of the population! Identifying these small sections and what drugs will work with their specific genes and environment will bring a revolution in treatment efficacy.
There is a clear risk, however, for these medical miracles to further increase the health and lifestyle gap between those with means and those without. We cannot blindly push the boundaries of personalized medicine without considering the social effects of the distribution of this technology.
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@Samuel
I don't think it will ever become efficient.
Crizotinib, a targeted therapy on the market for years which has been trumpeted as a triumph of personalized medicine only works on a small subset of lung cancer patients is still $100,000 dollars a year per patient . That's more than double the median gross, pre-tax salary in our country, and it's a drug that actually can be used on a substantial, if relatively rare, number of patients.
It's been on the market and at scale for years. No, it will not magically become reasonably inexpensive without massive structural changes to incentives, and that's a drug that doesn't work on only one person.
I've worked in the biomedical industry long enough to know better.
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@db - What makes Crizotinib so expensive? The ingredients? The procedure needed to manufacture it? The process involved in providing it to the patient? Paying off the costs of the original research? Drug company desire for outsize profit? I'm wondering just why it hasn't come down in price.
3
If these 1-off treatments advance the science towards better treatments for a wider class of patients, I see no issue with providing them. Mila would almost certainly have died in another year or so according to what I've read online, and if donors find an appeal worthwhile, I see no standing for naysayers.
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@kwb There is plenty of standing for naysayers, including the fact that a health system — and charitable giving — should focus its resources and talents on providing the best care for the greatest number. How many children going without insulin, or any number of necessary treatments, could have been helped with that $3 million? Feeling pity for one child should not blind us to the needs of millions.
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Someone has to be a guinea pig, if I may use that expression. Science is not wasted.
3
How much of the research behind Milasen is open source and able to be replicated by other researchers, for other diseases? And how much is reserved as the intellectual property of Dr. Yu or his hospital or some LLC set up to manage the potential profits of customized medicine?
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@Doc Billingsley I think the technologies for these DNA oligos are published and available for those that have the means to access them. There just aren’t enough scientists and doctors (and money)
6
Who cares? If Dr. Yu spent the time developing it then it belongs to Dr. Yu. Everyone else can feel free to develop something similar if they do not like it.
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@Dr. Trey I spent time in a postdoc and as a researcher developing technological treatments. I retired from my academic job so that the intellectual property would belong to me and not the University. I don't want to gouge people that can benefit from this technology. I also want to provide a reasonably priced product that gives me a reasonable profit. I have not forgotten that a private medical college and a state university gave me a place to do my work. I benefited from these environments that were supported by tax payer dollars. You remind me of some of the people university administrators I worked with who only wanted to enrich themselves.
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I’m very sorry for this child’s plight but this is not a viable solution. The treatment may be prolonging her life and her suffering.
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When there is a new development in medicine, in this case a drug to treat a neurological genetic disorder, information is also learnt to benefit other patients in the future. Therefore, it is not wasted money. Unfortunately, in our society billions are wasted every day (just think about the costs of the political campaigns) and this expense should not be criticized, but seen just as an example to be imitated by more in the near future.
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The long-term solution may be to identify talented youngsters at an early age and pay for their entire college and post-grad work, the stipulation being they go into medical/scientific research. Society needs a steady and dedicated supply so that orphan diseases can be diagnosed and hopefully treated in the same manner we've learned to deal with cancer, heart disease and
cystic fibrosis. Relying on crowd funding and overworked research staff is not the way a civilized society deals with it's
sick children.
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@Tribal Elder
Superb idea! This has already been achieved (at least to some extent) via the federally-funded Medical Scientist Training Program (MSTP). Those of us fortunate to have been selected for this prestigious program were funded through a combined MD/PhD program and have spent our careers focused on medical research.
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@Tribal Elder
We *used* to do that. We stopped.
If you go to the main entrance of City College in New York City, you will see a wall of photos of CCNY graduates who went on to win Nobel prizes and similar accomplishments. In their biographies, they say that they never could have done it if CCNY had not been free. When I was an undergraduate I got a *scholarship* to pay my expenses. They *paid me* to study science.
In 1976, CCNY started to charge tuition, a trend that was matched by state universities around the country. With free-market rhetoric, legislators have turned universities into paying businesses.
Bernie Sanders recently said that the cost of free state university tuition would be $70 billion. The recent increase in military spending was $80 billion.
According to Science magazine, the Chinese are *ahead* of us in many areas of biomedical research, such as embryonic stem cell research.
War or biomedical research, America. It's our choice.
9
This is a Sophie's choice decision. Everyone wants their child to be normal, grow and thrive but what are the costs to society to spend three million on just one child when their are billions of children starving and dying because they can't get basic and cheap vaccines for very preventable child illnesses? I am grateful not to be in this situation.
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@Sasha Love I struggle with this as well. There is something that feels profoundly ammoral about spending $3 million on development of a drug alone (not to mention the many millions more which will be spent on care) to maintain a very low standard of life for one child, simply because of where she was born. Meanwhile, many children worldwide are dying of preventable diseases for want of a $50 investment in their health. There are many productive adults (mostly of color, incidentally) with Type I diabetes in this country who die because their struggles aren't emotionally resonant enough to raise millions on GoFundMe. Patients dependent on Indian Health Services and rural health care systems die daily because there are no doctors on duty or no drugs in stock, or because they don't have transportation funds (sometimes under $20) available to reach specialist treatment appointments. I understand the emotional appeal involved, but I believe we as a society need to challenge ourselves more and examine the ethics and morals of this sort of resource allocation.
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@Ariel
You might find the example of a country with socialized medicine instructive.
In the UK, the National Health Service calculates the value of a year of functional life at about $70,000. They pay for treatments that will preserve life or function for $70,000 a year or less, but don't pay for treatments that cost more than $70,000 a year.
This has led to many testy negotiations with American pharmaceutical companies.
The government can override these decisions. In practice, it's difficult to deny treatment to children who are dying of a curable disease, and the cost of most treatments usually goes down with time anyway.
As the NYT reported, David Gratzer of the Manhattan Institute claimed that in England, they wouldn't have treated Rudolph Giuliani's prostate cancer because he was too old. As with many conservative myths, this was false.
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@Sasha Love Very likely the approval hinged upon other factors in that particular field of medicine that the three million spent developing the one drug helped to pay for. The decision makers may be aware that there (is) a good chance in this or a related field of medical research that could eventually reveal a half dollar when only putting in this effort to find the nickel the little girl needs. Remember that it was alchemy that really got the ball rolling on what eventually became modern chemistry. Or that guy from way back with that thing who only went digging for water but struck oil. Same deal.
4