I am dying of squamous cell carcinoma, and potential treatments are out of reach

This is my brother. I love you.<p>Anyone who has suffered through this disease or has a loved one that experienced it knows the feeling of helplessness.<p>More on Jake’s story below, and a link to the fundraiser to support him and his wife: <a href="https:&#x2F;&#x2F;www.gofundme.com&#x2F;f&#x2F;help-the-fight-against-cancer-with-jake-s" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.gofundme.com&#x2F;f&#x2F;help-the-fight-against-cancer-wit...</a><p>Be kind and good to your loved ones. Life is short and no one knows how much time we have left. While the FDA’s antiquated bureaucracy does no good, the true enemy is the disease itself. I believe in science and I hope that someday no one else will needlessly suffer as my brother has.<p>You be good. I love you.

I would hate to ask the author this, as it seems cruel, but is the FDA denying him access? As he points out, Moderna has two drug trials going. The FDA &quot;right to try&quot; page[0] clearly spells out that Moderna <i>could</i> give him unproven medicine currently in a clinical trial under the right to try laws. So Moderna could let him into one of the two studies, or give him the medicine under the right to try laws if it rejects him.<p>Likely, Moderna doesn&#x27;t want him to take the drugs because they will probably be ineffective this late in the cancer stage. It doesn&#x27;t want its treatment to be associated with his death.<p>[0]<a href="https:&#x2F;&#x2F;www.fda.gov&#x2F;patients&#x2F;learn-about-expanded-access-and-other-treatment-options&#x2F;right-try" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.fda.gov&#x2F;patients&#x2F;learn-about-expanded-access-and...</a>

I feel somewhat closer to this person because I was once in his position as a child fighting a neuroblastoma in 1981 and some how my mother found a way to get me on an experimental list which saved my life. Half of the kids that took the drug died by the time they were teens due to heart failure, obviously I wasn&#x27;t one of them, but am thankful for the chance to live a full life.<p>When the odds are against survival, there shouldn&#x27;t be any rules limiting treatment, if it means even a slightly higher chance of winning. That&#x27;s something our government has forgotten about or doesn&#x27;t care about. If I was in this persons shoes, I would make it as personal as possible against those in control of the FDA. Show up at their houses, work, kids soccer practice, get in their face and let them see what their inaction is doing in person.

Why don&#x27;t we just have a schedule&#x2F;tier system?<p>Category A - approved by the FDA, basically what we have right now<p>Category B - approved by some other established regulatory body (EU). Comes with all the warnings, doctors can write it off-label, insurance companies may not cover it but doctors can ask for a variance<p>Category C - approved by some non-OECD body. Insurance companies under no obligation to cover<p>Category D - experimental, this is stuff maybe still only in animal models, but pharmacies can still order and dispense it<p>Category E - experimental and basically limited run from pharmaceutical companies. These essentially need to be tailor-made or produced by a GMP kilo lab. There are plenty of drugs in this category - I worked on them - and the intended recipients are entirely animals, QA, and regulatory agencies. But maybe if some crazy S.R. Hadden type (billionaire in Contact) wants to guinea pig themselves, let em.<p>The latter category also opens the door for custom therapies (gene&#x2F;mRNA) that you basically can&#x27;t test the active pharmaceutical ingredient for efficacy on.

There are &quot;right to try&quot; laws both Federally and in 40+ states. It&#x27;s unfortunate the author doesn&#x27;t address those, I&#x27;m curious how they interact with his case.

I was an early investor about 20 years ago (nothing to write home about) for a company in this space. Small molecule therapy though, not MRNA.<p>Very slow going, as is all speculative biotech stuff but it made it to approvals on the vetinary side (dogs and horses) and is sold in the US for those purposes. However, I believe the human trials are somewhere around P2 currently for H&amp;N and other indications are even further back.<p>Only mentioning it because I still follow the annual reports, and I know that despite having no approvals they&#x27;ve treated a few patients via the various avenues that are available in Australia to people who have exhausted all other options.<p>The drug is tigilanol tiglate. It&#x27;s had some success to date, with some immune responses in distal tumors after the initial application.<p>There is plenty of published research available, so please don&#x27;t anyone take this comment as any kind of recommendation or advice.

One aspect of the problem here is the difficulty in running a clinical trial, particularly at the recruitment stage. The covid-19 trials all had a surfeit of participants because of a pandemic, but with modern cancer treatment trials the qualification requirements significantly cut down on the eligible population.<p>This, in itself, isn&#x27;t a huge obstacle. The problem is the state of healthcare data systems. It&#x27;s next to impossible to perform high-quality search (even by individuals approved to do so by the IRB). The state of the art in most places is regex searching in SQL.<p>This is something we have the power to contribute to. Bringing modern search capabilities to important datasets like health (while maintaining HIPAA-conpliance) is a much better use of engineering time than mining spyware data for creepy insights...<p>[Disclosure: I contributed heavily to one of the major medical search products on the market. We dealt with organisations that expended tens of thousands of dollars and many months per candidate for recruitment. Using some very straightforward IR tech we literally found all their candidates in a few minutes, plus many more. But there is so much more to do!]

Read the rest of his blog; it’s wonderful and I’m devastated that someone I followed for years is going to vanish.<p>Thank you for giving us all some of your time on this earth.

I wonder - both in this specific case, and in general - if anyone here on HN has enough media&#x2F;political reach to spread this message.<p>This case reminds me a lot of the FAA realizing that safety restrictions for toddlers would <i>increase</i> deaths as more people would drive. (<a href="https:&#x2F;&#x2F;www.ntsb.gov&#x2F;news&#x2F;events&#x2F;Documents&#x2F;child_safety-Claussen.pdf" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.ntsb.gov&#x2F;news&#x2F;events&#x2F;Documents&#x2F;child_safety-Clau...</a>, <a href="https:&#x2F;&#x2F;www.ucsf.edu&#x2F;news&#x2F;2003&#x2F;10&#x2F;97119&#x2F;airline-infant-safety-seat-rule-could-cause-more-deaths-it-prevents" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.ucsf.edu&#x2F;news&#x2F;2003&#x2F;10&#x2F;97119&#x2F;airline-infant-safet...</a>)

There seems to be a growing sentiment that <i>FDA delenda est</i>, but if we don&#x27;t fix the underlying problem that led to this situation, it will just come back.<p>As the author says, no one ever blames the FDA for the people it failed to save. But approve something without the certainty that it isn&#x27;t potentially going to kill someone, and there will be hell to your doorstep.<p>Even if you want the experimental treatment, you cannot get it. Killing someone in an attempt to help is deeply unethical, while merely letting them die is not your fault. This is sometimes also known as the Copenhagen interpretation of Ethics.

The author seems to forget that entering a trial doesn&#x27;t necessarily give you access to the treatment. You have as many chances to be in the control group receiving a placebo.<p>And as bad, sad and frustrating as it sounds to someone in a desperate situation, it is for the greater good of many more human beings, protecting us from snake oil.

There&#x27;s a very easy regulatory solution: Roll the drug approval process back to the way things were done prior to 1962. Back then, safety testing was all that was required. Efficacy testing -- which is difficult, expensive, arguably unethical in itself, and in some cases effectively impossible -- was not required. Drugs cost ~20-50x less to bring to market, and were brought to market faster. Indeed the 40s and 50s are still known as the pharmaceutical industry&#x27;s &quot;Golden Age.&quot; And it wasn&#x27;t only because of low-hanging fruit.<p>Pair this paradigm with extensive postmarketing surveillance and periodic reviews for efficacy in a patient population. These could be done at two years, five years, and eight years -- and approval automatically rescinded if safety issues arise or efficacy is close to null.<p>Give people with fatal diseases a &quot;right to try&quot; drugs that haven&#x27;t passed safety testing -- and use that data.<p>Ban drug advertising in public-facing media.<p>Simple as.

Hopefully he makes it into a clinical trial. In case anyone is interested in reading what trial options are available for this type of cancer you can find the trials here: <a href="https:&#x2F;&#x2F;www.clinicaltrials.gov&#x2F;search?cond=Squamous%20Cell%20Carcinoma%20of%20the%20Head%20and%20Neck" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.clinicaltrials.gov&#x2F;search?cond=Squamous%20Cell%2...</a>

What I don&#x27;t understand is why the FDA doesn&#x27;t automatically rubber stamp approvals on medicine approved in Canada and the European Union. We all know their medicine is good - so why can&#x27;t I get it?!

This quote sounds like self interest in the article, but there is more to it:<p>&gt; If anything goes wrong,” he argued, “think how bad it will look that we approved the drug so quickly“<p>People who take an approved drug are relying on the result and authority of a scientific process — across all drugs. Some scary results and people may start to fear the process, which may end up killing more people.<p>That said I’m sympathetic to his plight and think that he sounds like a candidate for the compassionate use program. Any result would not help approval as who in such a program would volunteer to be randomized into a placebo arm? They’d just plead in a prior human administration disclosure section of any NDA.<p>(Also, pragmatically, immunotherapy grate nets are customized and extremely expensive. Who will underwrite it? Not the drug company who would have nothing to gain, not the insurance company, and I doubt the patient could afford it).<p>It’s a sad story all around.

Parts of this problem are part of what a company a friend of mine works for is trying to solve:<p><a href="https:&#x2F;&#x2F;xcures.com&#x2F;" rel="nofollow noreferrer">https:&#x2F;&#x2F;xcures.com&#x2F;</a><p>Part of what they do is they match patients with clinical trials for whom they would be candidates and then facilitate the process on an on-going basis. Apparently this bypasses a number of bureaucratic hurdles which can be faced by patients; for example a patient receiving care from a particular research institution might not be informed about trials being conducted by other, non-affiliated institutions.<p>Naturally, there&#x27;s rather more than this to them, but this part sticks out in my mind.<p>And finally, take what I&#x27;m saying with a bit of a grain of salt because I am remembering from casual dinner-time discussions that I had some time ago with my friend. My memory might be bad, they may have changed what they do since I last talked with my friend about it, etc.

My Dad was dying of IPF and I was always on the hunt for anything that may help. Of course there are countless quack &quot;cures&quot; but I came across a study that said metformin may help. Metformin is what people with type 2 diabetes use to help to control blood lipids among other things. My Mom actually takes metformin since she has type 2 diabetes. My Dad&#x27;s doctor wouldn&#x27;t even discuss it and dismissed it outright.<p>Dad died in 2021 and I find it difficult to think metformin may have helped him live a more comfortable life. I know it&#x27;s not a cure but the research seems to indicate a significant improvement for anyone with IPF taking metformin along with anti-fibrotic medications which Dad did take. We should have just used Mom&#x27;s metformin really Dad had nothing to lose but it&#x27;s hard to see that at the time.

I read this, and while I felt immense sadness, I will obviously move on with my life soon enough and forget about it. I wondered, if I were ever in the same situation, and someone read what I wrote before dying, they might empathize for a bit, but then they&#x27;d move on as well.<p>I just really wish that there&#x27;s something beyond death, that there&#x27;s something out there, and that all the suffering and unfairness and randomness in life is not for nothing. I hope we someday truly figure out what the fuck we are doing here in this universe. And if not that, at least we reach a point in biology where we have the ability to solve a lot of problems of the mind and body and make life better for everyone.

There was a drug called Rapamycin discussed in this Radiolab episode <a href="https:&#x2F;&#x2F;radiolab.org&#x2F;podcast&#x2F;dirty-drug-and-ice-cream-tub" rel="nofollow noreferrer">https:&#x2F;&#x2F;radiolab.org&#x2F;podcast&#x2F;dirty-drug-and-ice-cream-tub</a><p>The drug supposedly helped delay the doctor&#x27;s cancer and extend his lifespan until he stopped taking it, then it came back &quot;with a vengeance&quot; and he passed away. He wanted to stop taking it to verify whether it was actually working or not.<p>I wonder if it might help this person? He might have to be on it for life though.<p>Don&#x27;t take my word for it though, this is FAR from my field of expertise.

If we block someones ability to try novel procedures, how is that any different from blocking their right to assisted suicide?

There is a clear problem, and a good idea here. (i.e. Right to Try) What needs to happen to make this move forward?<p>I am getting so mentally exhausted learning about a specific problem, and then experiencing the helplessness of not knowing how to push on driving towards some mutually agreeable resolution.<p>Add in the fact that there is no way to seemingly coordinate the push towards a resolution. Individuals taking action without coordination feels just the same as taking no action at all. Are there any tools to coordinate the push and keep track of progress?

I saw both of my parents go through cancer (they both, so far, survived), my wife has MS - so I was quite interested in the &quot;perspective&quot; treatments.<p>You have the whole usual bunch of idiots that will advertise magnetism and cosmic rays to cure you, then new treatments that may or may not be available in your country (but available in others) and finally the trial ones.<p>I discussed at length with an acquittance of mine who works in the clinic trial branch of a pharma and she first gave me the usual double blind trials explanation (which I am aware of), but then could not really answer why the pharma companies insist in not having people, for free, that would test if the treatment is <i>not obviously lethal</i> (or heavily impacting).<p>If I was faced with the perspective of dying soon (or going through something like Alzheimer) I could not care less if the treatment is fatal to me. Again: not that is not working (this is something one can assess statistically through normal trials) but that it is clearly harmful.<p>This would at least be a clear indication of &quot;don&#x27;t try it&quot; (or &quot;be very careful monitoring for this and that&quot;)) if there are enough patients who react badly

Regulators are, in general, too sheltered and disconnected from the impact of their actions.<p>Incentives matter. When you get to make decisions that impact others, but not feel any of the costs associated with that, you do not have the correct incentives. I hope the staff of FDA read this and can’t sleep tonight. We can hope they feel some emotional pain, even if it is only some small subset of the pain they have and continue to cause to others.<p><i>FDA delenda est.</i>

I think the consideration that an article like this misses is the unintended consequences of speeding a process up &#x2F; reducing the criteria for review, approval, etc.<p>I think we can all see incidents of where some oversight process is loosened because of some legitimate desire to help some, but then you learn that the bad &#x2F; incompetent &#x2F; exploitative actors come out of the woodwork to test and take advantage of the changing of rules. Not even saying intentionally, but just by sheer numbers, the change you made allowed things that were being held back by (hopefully proper) regulation to now happen.<p>Anyway, not saying that the FDA and other bodies couldn&#x27;t move faster -- that is always the case. And there will always be examples of people&#x2F;cases stuck in the cracks with legitimately sad situations.<p>But I would hope that people reading such stories think about why there is a process. It&#x27;s not like the FDA&#x27;s purpose in life is to stop people from benefitting from new treatments. It&#x27;s to prevent the flood of bad effects of companies&#x2F;individuals from being able to say they offer some drug that doesn&#x27;t work in the way it says it does.

Some FDA APPROVED cancer medication cause many severe side effects, for example, Opdivo&#x2F;Nivolumab[1], my father used it and it seems to help, but caused other long term side effects, which seems will never pass. 1: <a href="https:&#x2F;&#x2F;www.opdivo.com&#x2F;potential-side-effects" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.opdivo.com&#x2F;potential-side-effects</a>

Another topic for discussion related to this (and I hope we can be detached enough to discuss the intellectual side of this impersonally):<p>Even if the drug&#x2F;treatment method were approved, how much would this cost, would the author even be able to pay for &#x2F; have the insurance company pay for the treatment?<p>And by the way, what limits are there on the price of a drug that an insurance company or the government will cough up the money to pay, for one person&#x27;s extension to life for a couple years?<p>If someone has a very rare or just very advanced cancer, how much should the rest of us (as individuals paying taxes&#x2F;premiums&#x2F;etc) be on the hook for paying for last ditch efforts to prolong that person&#x27;s life?<p>These are genuine questions I think are legitimate to ask, if not in polite conversation, then at least at the level of policymaking bodies in govt or insurance companies. I&#x27;m sure that people in the UK are quite familiar with this concept or debate.<p>You cannot just say you&#x27;ll pay whatever it takes to save someone&#x27;s life no matter what the circumstances.

I really sympathize with the author and feel his position has great moral clarity.<p>Wish there was some country or state where you could try more freely.

I get that the FDA is bad but how come there is no country anywhere on earth where these treatments are legal and available?

I hope more and more medicine&#x2F;science moves outside the reach of FDA. If someone is willing to pay for a treatment (or recreational product), and is reasonably informed&#x2F;competent to decide the risks, and it&#x27;s not a risk to anyone else, go for it. If someone tries to prevent consensual commerce (possibly while wearing a silly uniform or carrying a printed ID badge from a state), violate the law if on balance it&#x27;s worth it to do so, and if it were literally my life at risk, I&#x27;d use whatever force required to accomplish the transaction. Hopefully treatment would be on the table before I lost the ability to run a carbine properly if needed.<p>Medical tourism is an excellent opportunity for &quot;network state&quot; and state alternatives. I already get virtually all elective medical care outside the US for commercial and service quality reasons, despite having US insurance.

How horrible and frustrating. There is a fast-track approval procedure described here, I wonder why it doesn&#x27;t apply to these treatments?<p><a href="https:&#x2F;&#x2F;www.fda.gov&#x2F;patients&#x2F;fast-track-breakthrough-therapy-accelerated-approval-priority-review&#x2F;fast-track" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.fda.gov&#x2F;patients&#x2F;fast-track-breakthrough-therapy...</a><p>There is some important context for the following comment:<p><i>“If anything goes wrong,” he argued, “think how bad it will look that we approved the drug so quickly.”</i><p>In the 1980s, when the comment was made, there were still people at the FDA who remembered the Thalidomide disaster, which would have been a lot worse had the FDA approved the drug. In the U.S. several thousand women took thalidomide during the clinical trials, and some doctors took it, too:<p><i>In one case, a doctor had been using thalidomide himself and prescribing it to his wife. In addition to the wife’s loss of vision, the doctor mentioned peripheral neuritis, nerve pain that is a side effect of thalidomide.<p>The other report is even more alarming — a nurse had given birth to a baby without arms or legs and, as a registered nurse, “she may have had access to the item.”</i> (1)<p>Other countries including Canada, Taiwan, Japan, and West Germany <i>did</i> approve the drug or allowed it to be sold.<p><i>On December 2 1961, the drug was taken of the German and British markets, after several doctors brought up concerns as it appeared more and more plausible that thalidomide, when taken by pregnant women, was responsible for severe birth defects. Thought the Government of Canada was informed of these suspicions about the possible teratogenic effects of thalidomide, we had to wait until March 2 1962 for the Canadian authorities to react and, in their turn, withdraw thalidomide from the market. As unbelievable as it can appear, thalidomide was legally available in Canada for three full months after being withdrawn from its origin country.</i> (2)<p>1. <a href="https:&#x2F;&#x2F;www.nytimes.com&#x2F;2020&#x2F;03&#x2F;23&#x2F;health&#x2F;thalidomide-fda-documents.html" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.nytimes.com&#x2F;2020&#x2F;03&#x2F;23&#x2F;health&#x2F;thalidomide-fda-do...</a><p>2. <a href="https:&#x2F;&#x2F;thalidomide.ca&#x2F;en&#x2F;the-canadian-tragedy&#x2F;" rel="nofollow noreferrer">https:&#x2F;&#x2F;thalidomide.ca&#x2F;en&#x2F;the-canadian-tragedy&#x2F;</a>

There should be a universal law in human rights stating anyone can take anything into their own bodies and governments can never, ever prevent them.<p>Governments&#x2F;regulators deciding what you can put into your body is beyond ridiculous: applies to all substances and drugs.<p>Inform about the potential risks and effects: sure.<p>Prevent: never.

I am so sorry for what&#x27;s happening to this man, and I hope he finds peace in his last days.<p>the economics, ethics, and effectiveness around early approvals are really difficult to manage. companies have a strong incentive to get earlier approvals. but once the cat is out of the bag, it&#x27;s hard to get it back in. if a sub-standard drug makes it to market and gains wide adoption, it makes later drug trials of more effective drugs really hard. where&#x27;s the outrage for potential beneficiaries of better therapeutics?<p>I don&#x27;t mean to say this man and others shouldn&#x27;t get a chance at a hail mary when they&#x27;re staring down death. just, it&#x27;s risky business for others in the future. I hope the best for him and others.

I don’t know why, but this was heart breaking to read. I don’t think it was meant to be.

I’m not a Dr., I’m not recommending anything, but you might want to look into the following:<p>I see the FDA approved drug cemiplimab-rwlc for squamous cell. <a href="https:&#x2F;&#x2F;www.skincancer.org&#x2F;blog&#x2F;new-treatment-for-advanced-cutaneous-squamous-cell-carcinoma&#x2F;" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.skincancer.org&#x2F;blog&#x2F;new-treatment-for-advanced-c...</a><p>And if your willing to consider alternative therapies there is fenbendazole(see joe tippens protocol). It’s a dog dewormer and is available at pet stores as panacur-c.<p>Also see Albendazole, Flubendazole.

Things don&#x27;t happen in a vacuum. It’s understandable that people want access to the experimental medicine for themselves or their loved ones when the fear of god has been put into them. When one has nothing to loose, what’s there to loose?<p>Unfortunately it’s our dignity (or at least others diagnosed with „x remaining months“). As soon as this becomes normalized, the moral impetus will change that those wo didn’t make it, probably weren’t brave enough to try the new stuff.

(not an MD) The FDA does allow for compassionate use of experimental drugs. My understanding the many early stage drugs are tested this way, but access is limited to trial centers. The author should try search drug trials database to see there&#x27;s a group that is recruiting: <a href="https:&#x2F;&#x2F;classic.clinicaltrials.gov&#x2F;ct2&#x2F;results?cond=Head+Neck+Neoplasms&amp;term=vaccine&amp;cntry=&amp;state=&amp;city=&amp;dist=" rel="nofollow noreferrer">https:&#x2F;&#x2F;classic.clinicaltrials.gov&#x2F;ct2&#x2F;results?cond=Head+Nec...</a> My brother is a physician who is working with patients in drug trials. He had the very good fortune to see an experimental drug successfully treat a very sick, critical patient who was declared a candidate for &quot;compassionate use&quot;.

This seems bad. Help me understand.<p>In what ways does <i>always</i> letting individuals choose to take an experimental drug not work, if we make sure they know what is known and what is not? They are desperate, and that&#x27;s a horrible place from where to make a decision, but dying is a really bad alternative.<p>What are the pitfalls that would allow for this to not always be the option? How would this go more horribly wrong than just letting these people die?

I&#x27;m sorry this is happening to you. Thank you for fighting to improve SCC outcomes for yourself and others. Please keep us updated with your progress.

Not having some level of “right to try” is abhorrent.<p>Having said that there are two elements that I’m not quite sure how to adjudicate 1) where does one draw the line with regards to how sick someone needs to be to qualify for right to try, and 2) how do ensure that patients are actually getting a treatment with some resonance likelihood of success as opposed to some snake oil?

I thought the recent New Yorker article on FDA&#x27;s drug approval policies, &quot;When Dying Patients Want Unproven Drugs&quot;, was balanced, insightful, and well-written. It covers Thalidomide, and focuses on ALS and HIV. <a href="https:&#x2F;&#x2F;archive.is&#x2F;u6vmI" rel="nofollow noreferrer">https:&#x2F;&#x2F;archive.is&#x2F;u6vmI</a>

&gt; think how bad it will look that we approved the drug so quickly<p>The media (both traditional ans social) owns a large chunk of the underlying blame, due to their rush to create simplistic narratives in the wake of anything that &quot;goes wrong&quot; without nuance or examination of tradeoffs.

Unclear to me why clinical trials seem unavailable to him, which is how he would gain access to these treatments. The major cancer centers generally will have options for his exact situation, though sometimes they require he finish conventional and salvage treatments first.

Highly relevant: research on ways to accelerate the medical-trial process. Here is one example from JASA: <a href="https:&#x2F;&#x2F;doi.org&#x2F;10.1198&#x2F;016214504000001790" rel="nofollow noreferrer">https:&#x2F;&#x2F;doi.org&#x2F;10.1198&#x2F;016214504000001790</a>.

This is blaming the FDA for lack of treatment, not the recentness of the treatment.<p>That all the MRNA treatments they refer to are in trials tells you everything you need to know: we don&#x27;t know which work, we don&#x27;t know how effective they are, and we don&#x27;t know what they work on.<p>It is possible to enroll in trials for these treatments, but as they say they &quot;may&quot; be denied as they&#x27;re so far along. But that&#x27;s what you want: if a trial takes someone on who would be highly likely to die <i>even if the treatment works</i> then depending on the trial size it&#x27;s possible that that one patient might skew the results such that the treatment is denied, or alternatively, another person who applied for the trial gets denied even though they had a higher likelihood of survival.<p>I can understand it being incredibly hard for this guy, but we have been through the alternative:<p>* Snake oil treatments: drugs that do nothing but bankrupt people<p>* Actively harmful treatments: drugs that literally make things worse, while bankrupting people<p>* Paid trial scams: you can pay to be part of a trial, which immediately allows for the above two despite a regulatory environment that ostensibly requires trials.<p>etc<p>Things like the FDA exist in response to prior actions, and once they&#x27;ve existed for a while, people forget that the only reason that they don&#x27;t seem necessary, is because they are there. Much like the &quot;unnecessary&quot; financial regulations that were removed, and immediately resulted in banks creating the Great Recession.<p>Hence, it is not possible for the FDA to create a &quot;patients can be &#x27;treated&#x27; with untested treatments&quot; loophole that is not trivially exploitable by the kinds of people that resulted in the FDA existing in the first place.

Ok, so how do we fix this? Can we put this community into action to fix this problem?<p>This is after all a group full of hackers. I don’t know this domain area, but isn’t there anything we can start or do to change this obviously stupid and immoral system?

Blaming the FDA is popular but the problem is not the FDA itself. Drug manufacturers are part of the problem.<p>1. When the FDA has fast-tracked drugs, drug makers have taken advantage of this to hide data, lie, and refuse to comply with timelines for proving those drugs work. <a href="https:&#x2F;&#x2F;www.npr.org&#x2F;sections&#x2F;health-shots&#x2F;2022&#x2F;07&#x2F;22&#x2F;1110830985&#x2F;drugmakers-are-slow-to-prove-medicines-that-got-a-fast-track-to-market-really-wo" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.npr.org&#x2F;sections&#x2F;health-shots&#x2F;2022&#x2F;07&#x2F;22&#x2F;1110830...</a><p>2. When these accelerated drugs are found to not work or cause harm, the manufacturers continue to push for them and the FDA has tremendous trouble trying to their approval revoked. <a href="https:&#x2F;&#x2F;apnews.com&#x2F;article&#x2F;science-health-medication-business-premature-births-a6032ee4b00ee507335892e9f3ce466b" rel="nofollow noreferrer">https:&#x2F;&#x2F;apnews.com&#x2F;article&#x2F;science-health-medication-busines...</a><p>3. Accelerated drug approval gets mired in advertising. The general public has no idea how to judge if a drug works or not. Plenty of people believe that homeopathy works. The FDA gets massive pushback when trying to take a drug that is worthless away, because people think it works. This hurts us all and creates an incentive for companies to hide data and deceive customers.<p>4. The FDA is as fast or faster than the Canadian or EU equivalents. The FDA is not specifically slow, bureaucracy everywhere has become much slower because there are no incentives to be fast but countless incentives to be slow.<p>5. Lawsuits. It&#x27;s easy to say &quot;I&#x27;m hurting, I&#x27;ll take anything&quot;. But, then, there are rights you cannot give away. If a drug harms you, you will sue. If it kills you, your family will sue. The whole system is bogged down by lawsuits with massive disproportionate payouts. The early biotech companies and scientists involved often cannot deal with even a mistaken lawsuit.<p>We need a much more comprehensive overhaul of the system, starting with open access laws to all data pertaining to any drugs that are on the market (no manufacturer can keep anything secret for a drug that people take), an FDA that has much more authority and much less industry capture, medical tort reform, to create a system where in exchange it actually makes sense to provide fast tracked approval.

As other comments have pointed out, I&#x27;m not sure why the author can&#x27;t access experimental therapies. Clinical trials or even compassionate use program (prior to FDA approval) are very common.

When a gov org cares about how it will &quot;look&quot; over wether it kills you - you live under tyranny.<p>A common example why government doesn&#x27;t solve problems well. Because it&#x27;s an absolute governing law, there&#x27;s nowhere else to turn to. This creates an industry of snake oil that preys upon people both as regulated medicine and homeopathic remedies. In practice vertical integration is a requirement for economies of scale. This means all medicines must be created by singular centralized corporations. These corps buy out the regulatory and now we have an incredibly inefficient and locked out market that prevents innovation.<p>All orgs should be sunset after 20 years by law. Repeal the FDA, replace it.

You should also try immunotherapy (pembrolizumab) with chemo if your CPS is &gt;1% before experimental early phase trials.

Jake, even in the face of declining health, don’t despair. I know it’s difficult but please, don’t lose hope.

The reality of cancer is a bit paralysing to anyone who has had direct exposure to it and there is nothing really overstated about it in the common sense of culture. Everyone’s fight is different, because there are so many paths to take and they keep changing as research advances, but the time available is limited. Besides being restricted, cancer drugs are also notoriously expensive, but for the more self-determined individual there is the option of off-shore pharmacies (many based out of India) and perhaps even Chinese chemical labs (anything on AliBaba must be assumed to be completely unreliable; however, some of the labs which are referenced by scientific papers as suppliers are actually happy to do business after only a little bit of social engineering). Unfortunately this approach is limited to relatively simple pharmacological interventions; anything that requires complex medical procedures (including palliative care) and has to happen in a hospital will require normal legal compliance and the cooperation of a doctor. There are stories of people who have gone far and beyond to enrol into cutting edge clinical trials [1] – usually with the energetic support of a loved one. In those cases, even with the consent of a doctor, the logistics and expenses aren’t trivial. There is much to say in general about the questionable role of institutions who gate-keep access to medical procedures under the pretext of protecting reckless consumers – but it wouldn’t feel right to steer the conversation too much away from cancer.<p>&gt; This makes it nearly impossible for patients to find an appropriate clinical trial, discouraging all but the most stubborn–people like Mike Hindt, and people like Stephanie Florence. By her own admission, Florence, 44, a photographer living in Lewiston, Idaho, had to “bulldoze” her way into a trial by being persistent to the point of obnoxiousness.<p>&gt; When she found out she was a candidate for a new trial, she also learned another hard truth: trials don’t come to patients. Patients have to go to the trials.<p>&gt; In August, she and her husband drove from Raleigh to Boston, despite having no place to stay. After a week in a hotel room paid for by their daughter, they wrote about their circumstances on a community blog. Several people in the area offered the couple rent-free housing during Price’s treatment. In order to pay for food and other expenses, the couple are trying to sell their home, and Price’s husband, laid off from his job at IBM, took a couple of part-time shifts a week at the outdoor-apparel store REI.<p>[1] <a href="https:&#x2F;&#x2F;time.com&#x2F;4270345&#x2F;immunotherapy-pembro-clinical-trials-cancer&#x2F;" rel="nofollow noreferrer">https:&#x2F;&#x2F;time.com&#x2F;4270345&#x2F;immunotherapy-pembro-clinical-trial...</a>

This could change if there was enough political pressure. Easier said then done, but it should be done.

Spare the condokescenes, spoilt he action. Less wasteful comments, more letters to representatives.

Courage and best of luck for jake, his closed ones and anybody hit by hard to cure diseases.

Trump got a federal Right to Try experimental drugs act passed in 2018:<p><a href="https:&#x2F;&#x2F;www.ncbi.nlm.nih.gov&#x2F;pmc&#x2F;articles&#x2F;PMC7309195&#x2F;" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.ncbi.nlm.nih.gov&#x2F;pmc&#x2F;articles&#x2F;PMC7309195&#x2F;</a><p>Not sure how this intersects with the author&#x27;s reality. It is sad to see article after article spewing nothing but hatred towards Trump --almost purely from an ideological perspective-- when something like this could help so many.<p>This, in more ways than one, demonstrates how difficult of a problem this is. When people&#x27;s lives become game pieces in political battles, the people lose and politicians, well, use them to score points towards their own career objectives.<p>I read in a comment that the author lives in NY, where, apparently, Right to Try isn&#x27;t available. If find this interesting when a federal law has been in place since 2018.<p>This is one of the things that can be perplexing about the US system of government. Here we have a state preventing people from having access to treatments that could materially affect their illness when federal laws allow it.<p>While I do understand the many advantages of the independence granted to states by the US constitution, sometimes it feels like the US has devolved into a fifty regions pretending to be united as a nation when they are actually not and, as a result, end-up conspiring to damage the very societies they claim to protect.<p>Education is another example of this. All nations with excellent systems of education (and the results to prove it) have national-level planning, management and standards. In the US, not only is our system of education fragmented at the State level. Our schools are run by fucking unions organized as districts, each with their own axe to grind. It is no surprise the results are what they are.<p>We somehow manage to extend some of this &quot;excellence&quot; (sarcasm) into every level of healthcare.

According to wikipedia, many types of squamous cell carcinomas are associated with HPV infection. [0]<p>Another area where the US has lagged which would prevent a lot of these cancers from ever developing in the first place is HPV vaccination. Last I heard it was only females who were being routinely vaccinated, and relatively recently at that. We should really be prioritizing eradication of HPV.<p>[0] <a href="https:&#x2F;&#x2F;en.wikipedia.org&#x2F;wiki&#x2F;Squamous_cell_carcinoma#By_body_location" rel="nofollow noreferrer">https:&#x2F;&#x2F;en.wikipedia.org&#x2F;wiki&#x2F;Squamous_cell_carcinoma#By_bod...</a>

separate efficacy testing from safety testing

For the record, FDA has fast track &#x2F; breakthrough therapy options:<p><a href="https:&#x2F;&#x2F;www.fda.gov&#x2F;patients&#x2F;learn-about-drug-and-device-approvals&#x2F;fast-track-breakthrough-therapy-accelerated-approval-priority-review" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.fda.gov&#x2F;patients&#x2F;learn-about-drug-and-device-app...</a><p>Biden&#x27;s Moonshot program is trying to drive money to support new innovations:<p><a href="https:&#x2F;&#x2F;www.whitehouse.gov&#x2F;cancermoonshot&#x2F;" rel="nofollow noreferrer">https:&#x2F;&#x2F;www.whitehouse.gov&#x2F;cancermoonshot&#x2F;</a>

There&#x27;s a wild book from 2006 called Overdose (by Richard Epstein), that makes a more or less libertarian argument for deregulation of the drug industry. What the book misses is that before the FDA and the Pure Food and Drug Act is that anyone who thought they could make a buck by poisoning as many people as possible did exactly that. And, especially given the &quot;tort reform&quot; prevalent in red states, there is fairly minimal disincentive for corporations to kill people in the US.

There is a religion. In it, good people are wealthy. Bad people, less so.<p>It&#x27;s a popular religion among the well-to-do. As you might imagine.

Must feel especially shitty to have your freedom curtailed like this, looking at the probable end of your life.

Anyone more knowledgeable know why they can&#x27;t file for a single patient IND, assuming the vaccine is even acquirable?

There is no 100% correct answer here. As soon as you speed up the process, there are still going to be people that just miss out, and then we will have people saying 4 months is too long, it should take 2 months, or let AI approve it, etc. Desperation skews perspective.<p>The issue with having a standardized approach to people getting experimental drugs is it opens up its own kind of mini-market unless the implication is they would be required to be free by law. And desperation is not the right mindset to make law or rational decisions.<p>Not trying to sound heartless, its just not as easy as saying &quot;go faster&quot;.