Genervon - GM6 - An opportunity to communicate with the FDA

[GregK]

Indeed it is. My apologies.

 

[rknt50a]

Interesting point/counterpoint...

ALS patients press FDA for quick access to controversial biotech drug - The Washington Post

The bottom line for me is that the status quo is not acceptable.

 

[Nikki J]

It was unfortunate that Winston Ko of Genervon was quoted as saying " even if it does not work... they have something" I hope that was grossly out of context. We have had too many experiences of false hope

 

[jennyinfrance]

Excuse me GregK - how rude! I am not a something - I am a real person living with ALS and TRYING to help myself by researching as much as I can and sharing news with other sufferers. No one else is going to help me. Yes I do live in France but if GM604 is passed by the FDA we can access it here in Europe - that's my reason for joining the campaign to get it passed. What a cynical person you are. I think I'll stick to twitter where I have made some good friends - where we share information and don't get insulted for doing so.

I don't care if you think I don't exist but please be a bit more sensitive to others' feelings in future. I have enough grief in my life already without being insulted by you.

Cordialement

Jenny Dean
Dordogne
France

 

[GregK]

Wow Jenny - this is actually the first post where you sound like a human. Which I've mentioned many times, not just this one. Which also added to my suspicion that you were a bot.

Sorry you were insulted. But damn, you got some really thin skin!

 

[jennyinfrance]

Hi Greg

Yes I have very thin skin at the moment and I'm a bit feisty. Maybe I did go a bit overboard.

 

[4tloml]

It was unfortunate that Winston Ko of Genervon was quoted as saying " even if it does not work... they have something" I hope that was grossly out of context. We have had too many experiences of false hope

Nikki, I hoped it was, too (out of context), when I read the article. But it sure seemed a cavalier approach in any context. Winston Ko would certainly not be my first choice of people to head up a company with such a promising drug, but I guess we'll take it where we can get it, as long as the drug works! Such hope.

 

[13yearsago]

Someone here can enlighten me as to why Pharmaceutical companies cannot take their business and research overseas and have their drugs approved in other countries that might not be as strict as US? It would seem to me that a person with ALS would go anywhere to have a treatment that shows promise. Perhaps that is a lot more complex than I imagine? Excuse my candidness; just wondering about that

 

[Nikki J]

If they remain unapproved in the US then they can not make the profits they would get here. And while some would ( some do) travel for treatment it is impractical for many especially if it requires treatments over time

 

[lgelb]

Today's Boston Business Journal piece on the controversy -- they interviewed a couple of PALS. From my biopharma past, I found Steve Perrin's argument that the "right to try" would constrain ALS rx R&D overall to be the reddest of herrings.

 

[mcwtlg]

For some odd reason it did not come up when I searched, not sure why. I visit a lot of forums on a variety of subjects and know to search and should have been wary when my search pulled up nothing on the site. Personally if I could have deleted the post myself I would have, once I realized my mistake.

 

[4tloml]

Oh, Laurie, I so agree with you! And thank you for the link--we (my PALS and I) had missed that article.

Steve Perrin, CEO of the Cambridge-based nonprofit ALS Therapy Development Institute — a researcher whose life is devoted to helping find a cure for ALS — does not believe the trial data in support of GM604 is anywhere near sufficient to warrant approval. He argues that any patient who starts taking GM604 would automatically be excluded from other drug trials, meaning that if it turns out the drug doesn’t work, those patients are irreversibly harmed.​

Perrin pretty much admits the battle is about who is getting what patients into their own trial. As we look over the selection criteria for clinical trials, my husband, who has diabetes, peripheral neuropathy and takes blood pressure medication, would be excluded from most on that basis, and from others because he is not willing to go without his bipap for the duration of a trial when we know how beneficial it has been. He feels he has nothing to lose by adding GM604, but he has a lot to lose by foregoing the medication and equipment doing so much for him already. He should have a choice. There is a host of ALS patients Perrin would leave in the cold, "irreversibly harmed," to ensure he kept his pool of trial candidates, many of whom would never meet his selection criteria to begin with!

 

[lgelb]

There has been alot of media coverage since my last post. I don't want to sound like I'm "sold" on the Genervon data -- it's very scant and the company has a checkered history, though many of us do know Eric V., the compassionate use case, which was prob the point of selecting him.

However, the "right to try," though unlikely to leapfrog the FDA's regulatory mandate any time soon, would not keep biopharmas from developing in ALS any more than it has in cancer and AIDS. Moreover, expanded access could surface new prognostic/therapeutic insights more quickly; and, as you say, 4tloml, the inclusion criteria for controlled trials (and this is not unique to ALS) disqualify most with significant concomitant conditions; my husband was among them.

That said, the Kos are not doing themselves any favors w/ their over-the-top communication and uncertain regulatory strategy, which does not speak well to their ability to ever bring this therapy to market. But all this only reinforces the need for new approaches, of which there have been few under the ALS research oligopoly in which TDI is a leading player. That's the real tragedy -- a horrific disease, a don't-harsh-our-paradigm response. Partnering w/ the FDA and communication pullthrough toward improved endpoints, surrogate markers and adaptive trial/early experience designs would be a lot more constructive.

 

[rknt50a]

http://als-advocacy.blogspot.com/2015/04/time-out-its-time-to-listen-to-adults.html

 

[MaxEidswick]

>Fact: Traditional Phase 3 trials are dreadfully expensive; therefore, "promising" drug candidates sit on shelves while people with ALS die.

>We have a failed process. It takes too damned long.

Ditto that!