Through the enthusiasm generated by the Ice Bucket Challenge, many people now know that ALS is a degenerative neurological disease that currently affects over 30,000 Americans, and as it stands the disease is always fatal. Most people with ALS (PALS) are given only 2-5 years to live. When diagnosed, it is devastating.

Currently, there is only one drug available to treat ALS (Riluzole) and it is only able to extend life expectancy by a couple months. That's why the recent news about a small company named Genervon Biopharmaceuticals and their trial drug "GM604" has been so exciting for the Global ALS Community. While most trials have a hard time even finding a positive trend, the GM604 trial data supports the view that this drug could very well be is a game changer in the battle against ALS. There are multiple, statistically significant data. Not only in clinical data and results alone, but also correlated biomarkers data and results as well.

Genervon met with the FDA in February to make a potentially life-changing request on behalf of the ALS community; they asked the FDA to promote GM604 to the Accelerated Approval Program with Post-Marketing Phase 4 Requirements, so all ALS patients can have legal access to GM604 now. Under the FDA's Accelerated Approval Program, the treatments would be covered by health insurance. Although Genervon knows that this request might complicate their relationship with the FDA, they are taking a stand and doing everything they can to help the ALS community because it is the right thing to do. If the FDA does not grant Accelerated Approval, it will likely be 3 more years before patients are able to access this drug -- meaning that most people currently living with ALS will not live to see it reach market.

While acknowledging the paramount importance of public safety in the FDA’s drug approval process, we believe that ALS presents a compelling case for an exception to the usual process. We are asking you to help us appeal to the FDA through their sense of compassion for those of us with this dreadful illness. PALS don't have time to wait for another clinical trial to be completed.

By signing our petition, you'll be helping patients request that the FDA expedite the way potentially life-saving treatments are made accessible to PALS -- starting right now with GM604. Doing so could very likely mean a change in the course of ALS progression for tens of thousands of people currently diagnosed and for millions who will be diagnosed in the future.

It is incredibly important that we show the FDA how widespread the support for GM6 is -- not just within the ALS community but also with the general public who have been awakened to the harsh realities of ALS and support our work to find treatments and a cure. While we do not have an exact date of the Genervon meeting with the FDA, we do know this meeting could determine the fate of all ALS patients and their families.

There are two things you can do now before they meet that could help ensure that the decision comes down in our favor:

  1. Click here to tweet directly to the FDA and tell them you stand with the ALS community and support approval of GM6.
  2. Email the FDA directly to voice your support. The people at the FDA are the same as people all over the world. They have hearts. They feel empathy and compassion like everyone else. Share with them your personal stories of how ALS has affected your life, and explain why you feel that potentially life-saving treatments need to be made accessible to people with ALS now. Remind the FDA that GM604 has a very strong indication for efficacy and a huge indication of safety -- it's a "no-brainer".
  3. Please email: druginfo@fda.hhs.gov and copy FDA Commissioner Margaret Hamburg: margaret.hamburg@fda.hhs.gov & Dr. William Dunn: william.dunn@fda.hhs.gov & Dr. Stephen Ostroff: stephen.ostroff@fda.hhs.gov (Make sure to keep it polite and professional--remember we are asking for their help. Clicking the link above will open an email message in which you can type your own message).

You can keep track of the campaign on Twitter and Facebook.

Thank you.