FDA Challenges Stem Cell Companies As Patients Run Out Of Time

The issue is not really whether these treatments work or not. Every new treatment is by necessity unproven and more risky than an established one. Some people are early adopters or terminal patients with high risk-tolerances; others would rather suffer for a while than chance a drug that might make things worse.<p>Both of those are fine as preferences. The trouble arises because every US citizen is forced to have the global minimum of risk-tolerances across the population. It would be as if you could not try a development version till it became user-friendly enough for your grandparents. Specifically, in the US, you can't be an early adopter: the FDA does not allow citizens to opt-out unless they leave the borders of the USA. Indeed, in Cowan vs. US (1998) it successfully sued in federal court to prevent a dying AIDS patient from trying an experimental drug:<p><a href="http://www.leagle.com/xmlResult.aspx?page=4&#38;xmldoc=199812405FSupp2d1235_11084.xml&#38;docbase=CSLWAR2-1986-2006&#38;SizeDisp=7" rel="nofollow">http://www.leagle.com/xmlResult.aspx?page=4&#38;xmldoc=19981...</a><p><pre><code> Plaintiff requests that Dr. Davis be authorized to inject Plaintiff with the with the experimental goat neutralizing antibody drug [1] and that the FDA be enjoined from interfering with Dr. Davis' treatment of Plaintiff. ... The Court is sympathetic to Plaintiff's situation. However, the law is very clear, and under the current statutes and regulations, Plaintiff's physician may not administer the goat neutralizing antibody drug absent prior approval of the FDA. In Court, Plaintiff argued that he should have the right to take whatever treatment he wishes due to his terminal condition regardless of whether the FDA approves the treatment as effective or safe, and that to prohibit him from taking the treatment he wishes violates his rights under the US Constitution. ... This Court is in no way criticizing the intentions of Plaintiff and his physician or the potential effectiveness of the proposed treatment. Plaintiff's physician should pursue approval of his Investigational New Drug application as quickly as possible. Plaintiff's doctor must obtain appropriate approval through the proper regulatory authorities. As much as this Court may empathize with Plaintiff, the authority to provide some type of exemptions for individuals such as Plaintiff rests with Congress and not with this Court. </code></pre> Plaintiff was denied. Plaintiff died. Who knows whether the drug would have saved him, but he wasn't given the chance to try. Occasionally, if you have <i>tremendous</i> political connections, you can get a waiver, like Fred Baron:<p><a href="http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-bill-clinton-help-fred-baron-get-tysabri/" rel="nofollow">http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-b...</a><p><pre><code> “We did a safety review, consulted with experts on PML, and worked closely with FDA to come up with a risk management program that allowed us to bring it back on the market in a way that limited its use,” a Biogen spokeswoman told the Law Blog. The plan prohibits giving Tysabri for unapproved uses. Biogen Idec is running an early-stage trial of the drug in multiple myeloma, but Baron doesn’t meet the criteria to participate. Baron’s a prominent donor to the Democratic party, and many of his powerful friends, including Lance Armstrong and Bill Clinton, made appeals on his behalf. And the family agreed not to sue if anything goes wrong. Ultimately, his doctors at the Mayo Clinic worked directly with the FDA to find a “legal basis” for giving Baron Tysabri. The deal was announced on Baron’s son’s blog late yesterday. </code></pre> So, if you are wealthy you can travel outside the US to opt-out (though US-based companies will usually not administer treatment for fear of getting on the FDA's bad side). And if you are politically connected you can sometimes get an experimental treatment, like Baron.<p>But this is not really optimal. If you are an academic, you accept the concept of QC/quality checking, but you aren't stuck with just one journal to submit to. You can revise and resubmit somewhere else. And if you are an end-user, you don't <i>have</i> to take a reviewer's opinion into account when choosing between movies, books, bikes, or virtually any other physical good with star ratings on Amazon.com. Except for drugs. Then you, as the end-user, cannot opt-out and take the FDA's opinions with a grain of salt. In part this is because the FDA will sue you directly. In part it is because companies that even think of trying this route will get slapped by the FDA for trying to game the system, and subsequently find their approvals slowed or (nowadays) outright denied.<p>We need to carve out a jurisdiction where patients and entrepreneurs alike are free to take informed risks, recognizing up front that sick people do die in medicine, and also recognizing that society already allows people to take incredible risks in other contexts (joining the military, bungee jumping, walking tightropes). Whether that new jurisdiction is Singapore, or Estonia, or a seastead, or a medical cruise ship, or something else is to be determined. But that has to be the goal.<p>[1] In case a "goat antibody" sounds weird to you, it's a common thing in molecular biology. Google it, or see for example Thermo's web page: <a href="http://www.pierce-antibodies.com/custom-antibodies/goat-antibody-production-protocols.cfm" rel="nofollow">http://www.pierce-antibodies.com/custom-antibodies/goat-anti...</a>

It seems like this is what the FDA is designed to do.<p>Cost of FDA: someone might miss out on a beneficial treatment because it has not been reviewed by the FDA (and may never be, given the cost of FDA approval).<p>Benefit of FDA: prevents charlatans from fleecing medically-ignorant people (which is pretty much everyone) of their life savings just before they die, which might be better spent on proven treatments or left as inheritance.<p>At some point, we decided that the benefits were worth the costs.<p>Unfortunately, the benefits tend to be obvious and the costs tend to be more hidden. That usually results in overdoing it (in this case, perhaps overburdening the development and use of new treatments).

What's the difference between buying hard disk and getting a medical therapy?<p>* You can ask for a chargeback.<p>* You get a one year guaranty.<p>* You are not desperate.<p>* If it goes wrong, it's almost sure you aren't going to die for this.<p>Let's suppose that someone tries to sell a homeopathic "hard" disk: The information is stored homeopathically in water and read later. Is it a possible business model?<p>It's important that someone control the medical procedures and control that the effectively work. There have been a lot of horror stories of medical procedures that were totally ineffective of even dangerous. A similar case, with less emotional charge, is the need of regulation of airplanes.<p>&#62; <i>Wilkinson tells NPR's Laura Sullivan she got better immediately — 11 of her 25 MS-related symptoms have either improved or completely disappeared.</i><p>I hope that it's true, but I have a lot of doubts.<p>* How were these improvements measured? Self-reported or an independent test?<p>* Is this case reported in a medical journal?<p>* Can be only the placebo effect?<p>* Is this only a spontaneous temporal remission?<p>* Is she taking another treatment?

It is shit like this that makes me what to shut down the FDA and ban anybody who has every worked there from having a job again.<p>Isn't it enough that sick people have to suffer from their sickness? Does these nazies have to destroy their only chance of a better life?<p>If it turns out that the cure doesn't work, then let the estate sue the company -- don't go around ruining peoples life just to increase your own power.