A Cancer Conundrum: Too Many Drug Trials, Too Few Patients

My son has Melanoma. He already had 6 cycles of Pembrolizumab from Merck. He still has cancer. Any new cancer treatment for Melanoma, from other drug companies?

https://www.nytimes.com/2017/08/12/health/cancer-drug-trials-encounter-a...
This article caught my eye as does anything involving a potential cure to cancer. Cancer is one of the highest reasons for death amongst Americans Today, so anything that involves a possible cure is something that interest me. My family does not have a high risk of cancer and those in my family who did suffer from Cancer were thankfully treated and cured. Although, my Aunt just recently passed away about two years ago from cancer. Everyday, I wish that her medicine would've cured her. This potential break through for cancer is great!

What do all cancer cells have in common? They all metabolize fuel for energy in a faulty manner, say Dr. Thomas Seyfried, BU cell biologist, and lots of other smart scientists. How to attack? Target cancer cell metabolism and the underlying pathways by which cells communicate messages about their own fate. https://eatandbeatcancer.com/2017/08/06/a-love-letter-to-john-mccain-sey...

Let's not get cancer in the first place, a vegan diet will dramatically decrease your risk for getting cancer. But instead our government which wants us to get sick subsidizes and promotes the food that gets us sick. Watch"forks over knives" and read the China study.

It felt like I was reading about a shark feeding frenzy. So what happens when the food runs out, the sharks keep on eating each other? Sounds good to me. Capitalism is really very stupid.

This is yet another problem with our for-profit health care system. It is driven primarily by greed, not concern for human welfare. We don't need umpteen "new" anti-PD1 antibody therapies, each of which will be priced even higher than the previously approved and outrageously priced drugs already on the market. Here are a few simple steps that would help considerably: (1) require that the FDA approve only drugs that are significantly better than existing drugs (not such better than a nothing); (2) permit Medicare to negotiate competitive drug prices; (3) don't require Medicare to pay for any and all drugs under patent (and therefore priced to the max), only those significantly better than the best generic. The insurance companies generally follow what Medicare does.

I remember reading that there was a gene, I think it was called P-51 that essentially killed mutant cancer cells. So for a patient to have cancer they needed to have damage to a gene that causes unrestricted multiplication and also a damaged P-51 killer gene.
Why don't they go after repairing the P-51 killer gene? Seems that would take care of all of the cancers. Maybe this is too simple and there is more to it I don't know about...

In response to this article I understood that the cancer drugs may have been effective although they are not for the right people is like making a cure for a disease that no longer exists,It doesn't really make sense. My greatest concern is that these new breakthrough treatments is that some of them are being made purely of profit rather than it's true goal, helping the patient. So while the drugs may work for a few indivaduals with the mutation that would work great for them but instead we should work on the others as well, because we shouldn't be looking across countries to search for patients, although I have high respect for companies like Genentech and I understand they are hard at work I just fear maybe not now but possibly later all of the medical field will be monopolized, however I am truly excited for the future that immunooncology will bring and I am so confident in its success that I even see it as my own possible future career What better way to kill cancer than to target it specifically. It truly is a new frontier in medicine, although that is its own weakness because not many people would want to take the risk in an experimental trial if there life is on the line but if it is the only chance they have its well worth it. After all every treatment started out as a experiment just like immunooncology Take proton therapy for example, a focused beam of radiation, could just as well be a new possibility to fighting cancer, medicene will evolve and improve.

The obvious answer is for patients to sell to the highest bidder. Get on EBay y'awl.

science : noun

1a. false altruism for profit.

1b. profit.

2. egoism run amok.

3. lies and conflation of relevance, esp. via the use of "statistics", for profit.

4. science? hahahahaha....

5. a marketing ploy for various businesses.

I'm baffled why genetic sequencing costs $5000 and I suspect it's because that's the price they're stating. Are we to think that the procedure costs 4999 dollars including labor and maintenance of the high tech equipment and they're pocketing a buck?
Maybe I'm missing something but companies like 23 and Me charge $199 for a complete analysis of all 23 chromosomes. They're not deciphering the nucleotide bases one by one, they're using templates to search for matching pieces on the test person's DNA, both good and bad. Surely the same thing could be done with searching for a particular mutation in a cancer. Admittedly at this point 23 and Me is losing money as the product is their "loss leader" while they attempt to exponentially enlarge their database. But they're not losing thousands for every individual who signs up. And cancer contains far fewer genes that need to be searched.
Sometimes it seems the whole thing is a ruse. Also remember 5000 dollars is chump change compared to the overall cost of treating most cancers, unless you die quickly.

You want a patient for a trial? I'm available. I had prostate cancer but two and a half years after surgery, and a T4 stage, may PSA is undetectable. I've already had both tumor sequencing and whole genome sequencing and I own my data. I'll share it. So bring it on. But....

I don't want to wait 'til cancer recurs, see? One reason trials fail is that the patients in them are almost dead to start with. Why is that? Protocol. I'm not interested in a protocol that requires becoming metastatic. I want an alternative to ADT and I want it before I need it. But Phrma and the doctors who serve it are an obstacle, not a pathway. I am the customer here. Without people like me, you guys take off the white coats, shed the arrogance and get jobs that pay less than $300K. So who's up for a little real patient-centered medicine? I'm sick of the rest of you.

[email protected]

I recently heard a lecture about the immunotherapy. Many questions refers tô autoimmune diseases. It turned out this theory may help patients with autoimmune but the research is not open to these patients. Why not ? I asked.
As a person suffering from autoimmune I am willing to try anything. The FDA APPROVAL FOR MEDICATION for disease other than cancer is slower. Trials takes years and do not guarantee profit.
Hey , pharmasudicals, research centers and FDA beaurocats. We, people with auto immune diseases are suffering. Think about us.

We used to have extremely efficient medical/drug trials in this country- back in the mid and late 60's. I know because a grandparent of mine from a small rural town in the midwest was included in a very important oncologic clinical treatment/drug trial located at a large public university sixty miles away. Funded by- ta da- grants from the NIH.

The problem now, as a few readers here are intelligent enough to suggest, is the complete lack of-- shared data, be it in the form of a database of patients seeking to assist in possibly curing their disease and helping science, but also a level of shared cooperation among groups of researchers. Because right now? It's all about secrecy. Why? You guessed it; the almighty dollar.
And that is because private equity is the driver of what used to be grant funding by - the government funded NIH. No one will open up their databases because- no one wants to share their research because their private equity benefactors won't allow them. It's really that simple. And tragic. Unless the government gets back into the business of cancer research funding at the levels it used to support- I don't see much hope for change. Sadly.

Okay, here is a thought that might be way off-base, but instead of first subjecting patients to known cures that involve surgery, chemotherapy, expensive pharmaceuticals, and radiation, try these focused and personalized immunotherapies as a first line/primary therapy under strict experimental rules after DNA analyses of cancer cells, etc.

Secondly, it seems to me that immunotherapy is or should be working towards therapies that modify the individual's immune system through genetic manipulation and large scale growth of immune factors to be re-infused into the patient that may do away with the business model of expensive drugs.

Capitolism is not the best incentive to further science. At some point governments working together could make this process go faster and be more effective. Like public roads and global warming, it takes group action to make progress with these issues. Plus keeping in mind that people with serious life threatening illness are likely to try experimental drugs, that might be ineffective or even will hasten their death is some cases. The profit motive is basically making research target the most commom, most profitable cures or something like this immune therapy which is easy to make 1000 variations. It will take a few public health scares before we take the reins away from purely for profit drug research.

Curing cancer and killing a multi billion dollar industry is nearly laughable. Yes, the drug companies stand to gain but the jobless physicians will not just give up their income. And their income is very good. They will tell how much they have to pay back in college loans and how they will lay off their staff and on and on. Come on folks, get a grip here.

I couldnt even finish reading this article. It became clear that the motive behind big pHARMa's clinical trials is profit, not cure. Cured patients dont make money. So how about finding what causes cancer in the first place? Oh, right, people already have but you will never hear about them here. Psst, cancer is a metabolic disorder, on one level.

Once again,as wit the development of drugs for HIV/AIDS, the problem is the serious demands of trial protocols written for a very, very heterogenous study population. Most of the control arms in these studies - typically standard of care or comparison to existing, suboptimal therapies that can cause serious and irreversible harm, death or extreme loss of QOL provide options that only a demented masochist would chose. In many cases surgery is not eve offered first because it is unprofitable for both hospital and pharmaceutical companies. Until you have been threatened with incarceration or detention by a desperate clinical trial coordinator you don't realize just how accurate the statement by Dr. Herbst is. Just remember, one cannot uncut a nerve or muscle and with the exception of the liver organs just don't grow back. After being in this field for nearly 30 years I can safely say you will never receive what you have been promised because doing so would mandate that any company has to open their books - if they intend to charge for single use/compassionate IND - or set aside your data points. That will never be done.You're lucky if an actual Informed Consent Process takes place. Imagine Divine in a lab coat with a huge syringe and shackles asking , "Does anybody want to die for the art of pharmaceutical industry profits?" Besides, most people will most likely not even be able to pay for the drug if approved.

"Genetic sequencing costs about $5,000, and insurers rarely pay."

That's seems like a small price to pay for valuable data collection. Each one of those genetic profiles could be input into IBM "Watson" and analyzed. Again- the insurance companies are let off the hook for being greedy, selfish and stupid.

The drug companies should see that it is in their best interest to work together on drugs that allegedly treat rare cancers. They could divide the cost of searching for and testing the rare patients. Does it actually cost $5000 to DNA test the samples or is that the usually inflated price that labs charge for this service? Even though patients live outside the areas of the research centers and are treated by local doctors, that is no reason that they should not be able to participate in a study of a new drug if they qualify. They could use overnight delivery for samples and drugs.
Each drug company and center wants to hog all the profits for itself even when it may not be in its best interest to do so. If they pooled their knowledge, resources and patients, they would be able to realize some profits sooner. Yes, the profits would be smaller, but so are the costs. They could at least pretend they are acting in the patients' best interest.

Further evidence that a profit-based healthcare system does not serve the public interest. Drug and insurance companies are not motivated to serve the public, only to use us as ATMs for their profits.

In reality, the hysteria over research/big pharma/capitalistic greed needs to calm down.
More people are living way longer with almost any type of cancer diagnosed Millions are cured.

Let's keep that in perspective. The sky is not falling. Obviously, research/big pharma/capitalistic greed have been getting quite a lot right.
I know someone whose child died at age 5 in 1972 of leukemia; today, the survival rate for that cancer in children is over 90%.

I was a lab rat in the Biovax ID immunotherapy trial at NIH in Bethesda Md.
Got kicked out.
The idea was to pretreat you with PACE (prednisolone, doxorubicin, cyclophosphamide, etoposide) for 9 months.
To gain a remission.
The Biovax ID was to clean up the cancer that might be lurking with a targeted immunotherapy. And create a durable remission
To test for remission, a harvest of a lymph node was needed and they blew it.
It appeared by cat scan that the cancer had come back, so out I went.
I had been receiving infusions of the placebo( it was a stage 2 trial and you needed a base to compare against).
I had been promised a compassionate exemption by Dr. Stergio Angelos, a director of Biovax. He withdrew it.
Remember that in these trials the idea is to get the drug to market, and anything that is an impediment to success will be removed. That means you sugar.
Never sign up for a stage two trial unless you are guaranteed a gold ring, in writing, reviewed by an attorney.
I was one of those extremely fortunate individuals who responded magically to Retuximab. A mono clonal antibody. Ten years and counting. People like myself should be studied to see why the drug worked so well. But that might negatively impact future sales, so that is a fantasy.

Unfortunately this is not a problem for women with cancer. We have a crisis in caring for women with gynecologic malignancy due to a lack of open trials and public funding for clinical research. Patients are eager to enroll in clinical trials funded by industry but these trials are much smaller/limited. Lack of public funding for cancer clinical trials means we are left with clinical trials designed for pharmaceutical companies to gain indications for new markets but not always to answer questions about how to care for cancer patients.

It is very difficult to get the genetic testing that should be standard at this point, even for patients at major cancer hospitals, which test only certain groups of patients with characteristics useful for specific research questions. Patients insisting that removed tumors and other body tissue be extensively and thoroughly tested (to rule in/various therapies, learn more about risks, know more about behavior of specific cancers, etc.)
get a resounding "no." Cancer's centers of excellence believe it's necessary to triage whose genes are looked at, to conserve their own resources, a decision that hurts patients kept in the dark and society as a whole. The more we know about cancer (in its myriad forms), its causes, symptoms, behaviors, and the impact of various treatments and their side effects, the better information patients and doctors will have to make truly well-informed decisions aiming for the best possible outcomes.

Clinical trial availability is part of this. In an era with vast databases tracking a great deal of everything we do, and bar codes on everything from blood samples to lemons, we can certainly have all clinical trials in a common data base so that the total menu of medical options is not a secret to most doctors and patients.

Creating such a database would require some restructuring of the pharma business model but that, too, certainly can be done and right now there is a rare consensus that it urgently needs to be done.

First there is the complaint that drug prices are too high and then there is the complaint that there is too much me-too competition. If we assume that competition drives down prices....???

We are faced with a true dilemma. The focus on and excitement over targeted therapeutics and precision medicine has been based upon the expectation that such therapies will be more effective and less toxic. To some extent this has been true, but as the article demonstrates, these targeted therapies may be so precise that only a small fraction of patients with any given cancer type may be eligible for such treatments. While it may take fewer patients to demonstrate effectiveness if the results are dramatically positive, it often takes thousands of patients on a drug to understand the potential adverse effects of a drug, some of which occur rarely, but which may be lethal. The adult oncology world should take a lesson from pediatric oncology that has long dealt with uncommon cancers in children. Most children with such cancers are treated in regional cancer centers and usually enrolled in national or international clinical trials, in order to learn from every patient. This approach could work for rare genetic subtypes of adult cancers. As to how to solve the economic problem faced by the pharmaceutical industry, namely having such small populations of potential customers for each specific treatment, I haven't a clue.

What happened to the "cancer moonshot?" One of the objectives was to tamp down competition and somehow encourage researchers and drug companies to pool their knowledge instead of fighting to be first so they can make a gazillion dollars before everybody else. And it's just weird to me that "cancer patients treated outside of academic centers do not have their tumors sequenced." This is data utterly lost. What are we actually spending our moonshot dollars on?

Maybe the moonshot was never actually funded, I don't know, but come on, people, get your acts together.

Well, here's the other reason that there aren't enough patients...insurance companies routinely deny coverage for experimental trials, because they know that if they deny immediately and the family has to appeal, the chances are (for some cancers, like pancreatitis) the patient's disease will have progressed to the point that s/he is no longer a candidate for the trial. This just happened in my own family despite my family member having the gold standard of insurance. Just in the amount of time it took for the trial to approve him to pay out of pocket while appeal was being 'processed', he became ineligible due to health changes. My family member is an otherwise healthy 45 year old. But we'll pay for expensive, chronic disease care for diabetics for 25 years even if the patients make zero effort to make lifestyle changes to improve their health. Unconscionable.

It boggles the mind that every hospital in the United States (community and academic medical centers) does not have a database in which all patients are anonymously entered so researchers can reach out and inquire if say Patient G will agree to a clinical trial. Would not something along these lines begin to alleviate this patient shortage?

Life saving drug trials should be coordinated by the Nation Institutes of Health, and President Obama recognized this by having Joe Biden coordinate a Cancer Moonshot. Under President Trump, the NIH would see it's funding slashed by billions of dollars. Leaving cancer research to private, for profit industry is like leaving healthcare to for profit insurance companies, those people who will cost the most for care will be left behind. This is how pre-existing condition patients (those that cost more) got left behind before that ACA. The only path to a cure for cancer is one that coordinates the efforts of those doing the research and that should include universities and other nations as well as the pharmaceutical industry.

Another illustration of our broken medical industry. Billions spent on developing therapies of dubious effectiveness, or dubious advancement over existing drugs, is a social and economic waste. Praying on the hopes of cancer patients and their families is just plain evil.

Ever notice that some of the drug adds talk about an opportunity for a longer life while stating that the longer life may amount to weeks? Perhaps the adds should also reveal the size of the study.

Spend more money on new antibiotics instead.

Small patient populations have long been the challenge for patient accruals in pediatric cancers (12 primary diseases with hundreds of mutations), where there are often just a few hundred patients diagnosed in the US annually with the same type of cancer. I concur with himillermd; we need to develop an international clinical trial network in order to find enough patients for trials affecting such a small slice of patients. EU, UK, CAN, AU are strong partners, but we must tap in to Asian countries, as well. The advent of IO and targeted therapies is driving the FDA/EMA to reevaluate their criteria for trials, and hopefully, to open more international trials.

If the US government wanted to help American citizens they would REFORM the Pharmaceutical industry. There would be price caps on drugs. Salary caps on executives. Improved FDA approval processes. Improved transfer of US government funded research that is often given to pharma companies for little or no $. The taxpayers paid for the university research and than when the Drug gets given to the Pharma companies they pay again in a 10X or more markup.

If the US government wanted to help terminally ill Americans they would make it easier for them to participate in these trials. a national registry maybe of Trial Drugs available, the pros and cons of the meds and a simple WAIVER from liability for the patient to sign. Also allow these poor people access to Marijauana, Canadian Meds(at much lower prices). My friend had to wait weeks to get into a drug testing trial for her terminal cancer. It was an anxious time for the family.

If Pharma companies had to pay a fairer share of the research of their drugs....there wouldn't be 1000 versions of similar drug in testing. If you make it a casino where 1 big hit makes BILLIONS they are going to keep trying to find the winner. And maybe that is a good thing...

Why not to speak also of inhexpensive cancer primary prevention, based on bedside recognizing Oncological Terrain-Dependent, Inherited Ral Risk, diagnosed with a common stethoscope from birth, and removed by cheap Reconstructing Mitochondrial Quantum Therapy? For instance, notoriously all patients involved by pancreas cancer, termed erroneously as the silent killer, inexorably suffering from terrible pain. If NYTimes will decide to publish an epochal Editorial on malignancy primary prevention, aiming to stop its growing epidemic. I will provide my MEDLINE articles and books, as valuable documentation.

Easy peasy. Want to find patients? Offer to do a genetic profile on cancer patients. No need to sequence the ENTIRE genome for $5,000. Just areas that effect the cancer and the drugs. Companies could even GIVE this information to patients so they could join the appropriate study - this one thing would benefit everybody involved. Patients would have more info about their cancers and more options. The price of sequencing would be shared and go down. And companies would be able to find their target group of study participants.

This is the fundamental example of why for-profit is not always the best selector for investment in science.

The opposite of the benefit of allocating resource through capitalism is the tragedy of the commons. Each person trying to maximize his own profit and usage causes the whole system to work inefficiently, wasting valuable resource and often using up the resource, with a net loss of good to the whole of society.

Drug trials use a scarce resource - sick people - to find a way to help future sick people. Capitalistic competition is inefficient in this scenario. We don't have enough resource to find out which trial will scramble to the top of the heap.

Sometimes allocating resources might best be undertaken outside of the market.

Very very interesting. I am in a trial of a disease that uses FDA prior approved Cancer drugs ( 2001). The medical profession is hot on trials, as a step forward, of evidence based therapies, instead of all the therapy reversals, the profession encounters, for prior methods," This should work" These trails are supposed to provide benefit from a database of the experience of others.They are time consuming, and a one way street, of a database , commoners probably wouldn't understand, and Physicians have trouble explaining in laymen's language. Anyway drugs boosting your own immune system sounds logical .

Maybe the reason clinical trials can't find patients is that patients are waking up to the fact that there is a 50 percent chance they will be assigned to the "control group" where they will receive no treatment at all. It is highly unethical in my view to conduct research in this way. If a person has a life-threatening illness and there are treatments available, they should be treated.

Another partial remedy to the shortage of clinical trial subjects -- and the resulting delays in obtaining regulatory approval -- would be reciprocity of approvals between FDA and its equivalent counterparts: http://www.nationalreview.com/article/444199/prescription-drug-price-con.... Thus, a new immunotherapy or targeted therapy approved in, say, the EU, Canada or Australia, would automatically be available in the U.S.

Cancer research sponsored with billions of dollars by the pharmaceutical industry has been biased. They still believe that cancer is triggered by the mutation of nuclear DNA that seems to be fundamentally wrong. A new group of academic scientists like Dr. Thomas Seyfried or Dr. Dominic D'Agostino without pharmaceutical sponsorship have been working on least costly cancer treatment using ketosis and hyperbaric oxygen therapy. They have conformed a Nobel Prized theory (1932) of Dr. Otto Warburg that cancer is a mitochondrial metabolic disease instead of gene mutation. Their cancer therapy doesn't require expensive and toxic medicines or toxic radiation therapy or potentially dangerous surgery. This therapy using ketogenic diet and hyperbaric chamber doesn't excite the "cancer industry". However, this is the new trend to treat a group of metabolic diseases like Alzheimer's or type-2 diabetes. They all apply life style change, particularly, ketogenic diet, nutrient supplementation, fast, exercise with strict guidance by doctors.