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Comment by ekianjo | Hacker News Reader

> The idea that BrainStorm could make a profit from NurOwn before the treatment is proven effective and approved by the FDA is a bad look for the company and the entire pharmaceutical industry. It reeks of opportunism, even when couched in compassionate rhetoric.

If anything we need more experiments like that. If you have no treatments left to try and you are dying, being able to try something that is not 100% proven but that has some potential is better than nothing. It's not like selling snake oil or something, most therapies have clear identified pathways and targets based on actual fundamental research.

Companies are hardly going to make tons of money on unapproved therapies anyway, because the word will get out if x patients tried it and had no benefits.

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> being able to try something that is not 100% proven but that has some potential is better than nothing

Most groundbreaking new treatment don't cure you forever, they only prolong your life for 6 month or 1 year. (After more test and improvement, they extend the time more, hopefully many years.) So don't expect too mush from an unproven treatment.

So a more realistic alternative is

A) No unproven treatment, and try to have a good pain relieve treatment and good emotional support for the family

B) Spend all your money in an unproven treatment and left a broken family, and

- 1%? chance of 6 more months of life

- 50%? chance of no side effects

- 49%? chance of nasty side effect of the treatment that makes your small time on Earth worse

For a real example, there is a somewhat popular but ineffective treatment for some types of breast cancer that include bone marrow transplant, and living with a transplanted bone marrow is a problem. https://www.nejm.org/doi/full/10.1056/NEJMbkrev58584

> It has been estimated that more than 30,000 women in the United States with breast cancer received bone marrow transplants between 1985 and 1998, at the cost of millions of dollars, considerable adverse effects, and treatment-related mortality rates of 3 to 15%.

>There’s little evidence that ALS patients, especially those with advanced disease, will benefit from NurOwn. In BrainStorm’s Phase 2 study, NurOwn was unable to slow the progression of ALS compared with a placebo. The company turned to a responder analysis to eke out a signal of efficacy, which it’s now trying to confirm in the Phase 3 study.

Here, you seemed to have missed this part.

The author of the article also seems to imply that FDA approval is the only process by which a treatment could be "proven effective." There are many treatments that are already available in Europe but spend years waiting for FDA approval.

There's also the fact that even when a drug does successfully pass through the FDA testing process, there can be a period of up to a year between the drug being successful in Phase 3 trials and actually getting approved for prescription. If you're a terminally ill patient, a 12 month waiting period between "proven in phase 3 trial" and "actually finally getting the FDA's rubber stamp" can be a long time.

The FDA allows "compassionate use" exceptions for this type of thing and generally grants them. The point of the approval process (and the entire FDA) is to prevent charlatans peddling dangerous and ineffective therapies and drugs to desperate people for profit.

Which is what this article is about: you can have no choices, but here, pay us a ton of money for UnprovenDrug(TM)! It might work according to nobody but us.

You're making the classic misstep of assuming a Pascal's wager type situation - as though there's only ever 1 unproven option out there so you might as well, when in reality there's many and getting scammed out of all your money on one means you can't try others which actually might work.

It used to be the FDAs job to monitor and report if unapproved therapies (which could be used with a waiver) worked or not. Now, there’s no one to provide that role, so the best you’ll get is anecdotal evidence. Removing the FDA from this process doesn’t help.

> Companies are hardly going to make tons of money on unapproved therapies anyway, because the word will get out if x patients tried it and had no benefits.

That seems wildly optimistic to me. Companies, especially pharma, will always find ways to 'make tons of money'. The second part of the sentence assumes a level of disclosure (from the companies) and rationality (on the part of average people), that I don't believe exists in the world. There are plenty of examples to back that up, especially people's willingness to believe self-serving 'facts'.

Okay, so instead of charing someone desperate $300K for this unapproved treatment, why not just relax the rules for clinical trials for end of life treatments? This still matches the intent of compassionate care, still collects structured data that can be explored and gives people the right to try anything.

I also think the "word will get out" argument doesn't really wash. If every other option says you'll die, a miracle is worth a shot.

something that is not 100% proven but that has some potential is better than nothing

This is not necessarily true, and can certainly be worse than nothing.

> If anything we need more experiments like that.

How does reducing the number to the wealthy help here? In Germany at least, your insurance will surely not pay for this.

Margins are so high in this sector, why should they prey on the hopeless to turn a bit more profit, when they'll make huge ones anyways once the drug is finalized?

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