/** */

Condition Green

As many of you might already know, I was the late-stage PALS mentioned in the recent Genervon press release. I got interested in this drug some time ago, did some research on it and wrote a blog post about it. I had contacted the company, Genervon, to get information for my post. Thereafter, a dialogue was maintained regarding clinical trial status and future development plans. Being that I am a late stage PALS and still extremely active in awareness, advocacy, and science, they agreed to my request for compassionate use. It was another 9 months going through the process of authorization (mostly because my local hospital had never done anything like this before and together we created a new protocol).

During that time the Phase 2A results came out and I was given access to some of the data. Those, combined with my own experience, gave me the satisfaction that this drug was safe and quite likely effective. I share the concerns about trial size, but like all PALS am concerned for the time required to go through the usual phases of clinical trials. The clinical trial program actually has four parts:

  • Phase 1 – single dose usually in healthy subjects for gauging safety
  • Phase 2 – use in actual patients looking at safety and initial efficacy
  • Phase 3 – larger patient population with different doses, efficacy and SAEs
  • Phase 4 – market surveillance for adverse events

Not only does it take time to fully enroll and execute a large clinical trial but it takes even more time to secure the funding necessary to begin each phase. This is especially true in this current era of venture capital avoiding biotech investment.

I have helped launch other initiatives to get PALS access to experimental treatments. It is critical that patients get more than one or perhaps two chances at early access to treatment while they are newly diagnosed. Drugs that are possibly effective must be made broadly available to patients who are facing otherwise-certain death. Based on the safety and the indication of efficacy in GM6 (mainly borne of my personal experience), I got behind the effort to seek what FDA calls Accelerated Approval so that many more PALS can try it and see where it takes us. Accelerated Approval requires full data surveillance for efficacy, not just serious adverse events (SAEs). The efficacy data determines whether final approval is made. Basically, Accelerated Approval is like a Phase 3 where patients/insurance pay for participation. I believe all PALS would gladly participate in such a program.

If the wider data don’t support the continued use of GM6 I will be the first to admit it. But right now I believe GM6 has the capability to effectively treat ALS in a way no previous drug ever has. And I want to get that opportunity as quickly as possible to as many PALS as possible.

After publishing the press release and posting it on social media and online forums, another PALS started a petition to the FDA to demonstrate the support in the ALS Community for this Accelerated Approval. I would like to urge all who are concerned about ALS – PALS/CALS/Friends – to sign this petition and share it among your social circles. At that link you can sign the petition and post comments to be included with your name. You can also find links to email Senators who oversee FDA and proposed text for those messages.

It is imperative that the comments left on the petition signatures be respectful. FDA isn’t the enemy. They really would like nothing better than to approve a treatment for ALS but need the data to support it. I think we have the data because even though the population was small, the slope of decline as measured by the ALSFRS-R was reduced significantly during the short treatment window. Also, certain biomarker candidates were tracked and correlated with progression. Nevertheless, FDA has to be very careful with the precedent it sets so we as patients must be partners with them in these decisions.

My own experience with GM6 has been positive. The worst part of the entire project was getting the PICC line and the lumbar punctures for CSF samples to make biomarker measurements. I experienced absolutely no adverse events related to the drug. Insofar as benefits, I must admit that the small gains in function noted in the press release are most likely due to surviving neurons branching out new axon terminals to cover the neuromuscular junctions (NMJs) abandoned by the dying motor neurons affected by ALS. GM6 will NOT regrow dead motor neurons. However, it does induce healing in injured ones. In my case, I probably don’t have many injured motor neurons – most of mine are gone. But people who are more recently diagnosed have a higher chance of regaining some lost function in addition to stopping progression.

Based on the information I have seen and my own positive experience, along with the considerable (at best) delay in commencing a larger Phase 2 or 3 trial, I think GM6 deserves Accelerated Approval. I also think this could set a beneficial precedent for future drugs which show similar safety and efficacy signals in early trials. Hence my hope for GM6 getting into the larger population of PALS.

6 thoughts on “Condition Green”

  1. Thanks! We in the ALS Community must be our own advocates. Certain organizations with the mission statement of advocating for PALS are much too weak in their efforts on behalf of a population given 2-5 years to live.

  2. Thank you for your post. I am in decline but can barely walk. My FVC is 45%. I’d like to write the FDA to support accelerated approval while not being counter productive. Do you have the proper FDA contact(s) to whom I will correspond?

    1. To email the FDA, address the email to:
      druginfo@fda.hhs.gov
      Then cc these people at these FDA:
      margaret.hamburg@fda.hhs.gov
      william.dunn@fda.hhs.gov
      stephen.ostroff@fda.hhs.gov
      And cc these news shows:
      news@cbsnewyork.com
      Viewermail@newshour.org
      contact.nbcnews@nbcuni.com
      anderson.cooper@turner.com
      foxnewstips@foxnews.com
      To email Senators Alexander, Murray, and Paul:
      http://www.alexander.senate.gov/public/index.cfm/email
      http://www.murray.senate.gov/public/index.cfm/contactme
      http://www.paul.senate.gov/?p=contact
      To tweet the FDA: http://ctt.ec/uEdGb
      To tweet Senators: http://ctt.ec/j230_
      Next Step:
      Reach Out For Official Support From Organizations & Medical Professionals
      Here’s what we’ve been doing – we’ve been emailing and calling doctors and all kinds of organizations. We tell them:
      1) Our names and what our personal experience is with ALS. If you don’t have ALS or don’t know someone with ALS, just say you support the global ALS community.
      2) We say we working on promoting a campaign that is urging the U.S. FDA to promote a new ALS treatment to their Accelerated Approval Program.
      “There is a new experimental treatment for ALS developed by a innovative biopharmaceutical company, Genervon Pharmaceuticals. Their drug is “GM604”, which has shown to be safe and efficacious. Genervon has a meeting scheduled with the FDA sometime in the near future, and are going to ask the FDA to promote this treatment to the Accelerated Approval Program (AAP).
      3) We explain that we are seeking official support from medical professionals, advocacy organizations, and religious groups.
      4) We provide these links to the petition and the list of current organization that support the campaign.
      https://www.change.org/p/janet-woodcock-m-d-fda-accelerated…
      https://www.change.org/p/janet-woodcock-m-d-fda-a…/u/9342736
      5) We ask if we can add their name / their organization’s name to the list.
      6) We give this email address to respond to (you can too) gm6nick@gmail.com.
      As always, people can email Nick at gm6nick@gmail.com or Jehad Majed at jmajed@mac.com
      Your efforts are making this campaign very successful.
      Thank you,

  3. I am Anthonio Albert, I want to testify of how i got cured from ALS (Lou Gherigs Disease), I was diagnosed of this disease in April 2009, I was bedridden, i tried all possible means to get cured but all to no avail until i saw a post in a health forum about a healing herbal spell caster who casts spells to heal all kind of diseases including ALS, at first i doubted it but decided to give it a try, when i contacted this herbal spell caster via his email, he created a herbal spell portion and sent it to me through the courier service, when i recieved the herbal spell portion, i used it under his directives and i was totally cured from this deadly disease, I am well and healthy again, all thanks to this great herbal spell caster. Contact him for cure to any kind of deadly and incurable diseases via his email azizahealingtemple@gmail.com

Leave a Reply

Your email address will not be published. Required fields are marked *