First of all, please watch the Fulginiti Family video. It’s incredibly well done and very powerful: http://ellentube.com/videos/1_71rpj16z Now to address some important misconceptions about the anticipated FDA decision that we are expecting before the end of April 2015. We want to be sure everyone understands the Accelerated Approval Program and what the FDA decision will mean. In 2012, Congress passed the Food and Drug Administration Safety Innovations Act (FDASIA). Section 901 of FDASIA amends the Federal Food, Drug, and Cosmetic Act (FD&C Act) to allow the FDA to base accelerated approval for drugs for serious conditions that fill an unmet medical need. This was done because, as we know, it can take many years to learn whether a drug actually provides a "clinical benefit", which is proof that the drug has real and measurable effect on how a patient survives, feels, or functions. So, instead of a drug or treatment having to prove that it can cure a disease or extend someone’s life, the FDA bases Accelerate Approval on whether or not the drug has an effect on something called a "surrogate end point” or an “intermediate clinical endpoint". Don't let that term stop you from reading. A surrogate or intermediate clinical endpoint is just a big way of saying, "a laboratory measurement, x-ray image, physical sign, or some other measure that is thought to predict clinical benefit. For example, instead of having to wait to learn if a drug actually extends survival for cancer patients, the FDA may approve a drug based on evidence that the drug shrinks tumors, because tumor shrinkage is considered reasonably likely to predict a real clinical benefit. In this example, tumor shrinkage is the surrogate endpoint and FDA approval based upon tumor shrinkage can occur far sooner than waiting to learn whether patients actually lived longer. Let's put this in the context of Genervon and GM604. The researchers at MGH and Columbia measured improvements for patients in the GM604 clinical trial who received treatment. The test that Genervon is asking to be used as a surrogate endpoint is called Forced Vital Capacity (FVC). FVC is the total amount of air exhaled after a full inhalation. Patients with ALS experience continual decrease in FVC as their disease develops. Remember that GM604 was tested on 8 ALS patients and the patients were followed for 12 weeks. While that time period is too short to prove that GM604 has the ability to save or extend someone’s life, is not too short to show that there was a measurement of improvement. The FDA now has to agree to accept FVC as a surrogate endpoint and agree that the clinical trials preformed by MGH and Columbia were “adequate and well controlled” as required by the FD&C Act. If the FDA accepts Genervon's application for Accelerated Approval, studies known as phase 4 confirmatory trials will have to be performed as patients gain access to GM604. If trials ultimately fail to verify clinical benefit or do not demonstrate sufficient clinical benefit to justify the risks associated with the drug, the FDA can pull GM604 off the market. If GM604 ultimately does prove to verify clinical benefit, then the FDA grants regular approval. Please watch this short video: http://www.fda.gov/Drugs/ResourcesForYou/HealthProfessionals/ucm313768.htm. This brings us to our next and most important topic: What does it mean to accept or deny Genervon's application for Accelerated Approval with phase 4 requirements (post-marketing requirements) of GM604? The FDA decision to accept the application for GM604 will not mean that Accelerated Approval will happen or that it will even happen soon. It only means that the FDA has agreed to consider Accelerated Approval. Before you get mad at the FDA, remember that it is the responsibility of Congress to direct and lead the FDA. If this process sounds unreasonable, it is the job of Congress to fix it. The FDA isn't going to police themselves into making their jobs harder. This is also one of the reasons why our campaign targets Senators with our emails. We are effectively telling the FDA watchdogs that something has to change at the FDA now in order to help get a viable treatment option to ALS patients. The FDA decision that we expect at the end of this month will: 1) Accept the application for GM604 and then they will begin to look very close at the Accelerated Approval Program (AAP) as a possible move forward for GM604. If the FDA accepts the application, they have a set number of months to complete that review, either 6 months if they grant a priority review, or 10 if they don’t. 2) Deny the application for AAP. Meaning they will not consider getting GM604 anytime soon and more clinical trials will have to take place 3) The FDA could ask Genervon for more time to consider the application for GM604. The FDA can do as it pleases - remember Congress is ultimately responsible here. Our campaign urging the FDA to apply AAP to GM604 has been extremely powerful and the FDA is listening to us. We must keep up the pressure so they accept the application 1st and ultimately enter GM604 into the Accelerated Approval Program. Stand with us. Keep your voices and emails going. Pay no attention to naysayers who never read the science and data that Genervon made available to the public or those that don't understand the 1992 Accelerated Approval Program was designed to address the needs of communities just like ours -- patients with no viable treatment options for a terminal illness that is extremely cruel and unforgiving. If someone speaks down to you remember to tell them "Why we believe Accelerated Approval is the better option for GM604". https://www.change.org/p/lisa-murkowski-fda-accelerated-approval-of-genervon-s-gm604-for-use-in-als/u/10369781. Be tenacious. So now you see why the rally in Washington, DC on May 11th is SO VERY IMPORTANT: No matter what the FDA decides, we will have reason to continue our campaign for treatment options NOW. We need ALL of you! Together we can change the status quo and start saving the current generation of people with ALS. Please register today for the rally at: http://www.meetup.com/ALS-Faster-Drug-Approval/events/221529700/ Please check out Genervon’s latest press release, “GM604 in a Nutshell”. It gives a very good understanding of GM604 -- even if you’re not a scientist. http://www.genervon.com/genervon/about_pressreleases.php Don’t forget to check us out and Like us on Facebook. It is the best way to stay on top of the campaign: https://www.facebook.com/gm604forals Remember, we're in this together, Anthony Carbajal, Bethany Wolfe, Jehad Majed (jmajed@mac.com), Matthew Bellina, Nick Grillo (gm6nick@gmail.com), and the Torrino Family