In the United States alone, over 30,000 people are living with amyotrophic lateral sclerosis, or ALS.
ALS, also commonly referred to as Lou Gehrig's disease, is a progressive disease in which the motor neurons that control muscle movement begin to fail. After not being innervated for a length of time, the muscles begin to atrophy and lose function. Eventually, breathing is affected and the person dies, typically two to five years after the diagnosis.
The outlook for people with ALS is grim, and there is only one approved drug currently on the market. Unfortunately, it only prolongs the inevitable by a couple of months, and isn’t a real solution.
A small, California-based biopharmaceutical company called Genervon has been developing a drug known as GM604 that has shown amazing promise in clinical trials to dramatically preserve quality of life for those with ALS. Because of the FDA's lengthy drug approval process, many people currently living with ALS will likely be dead before the drug hits the market.
It generally takes about 15 years for a drug to complete the necessary development and testing needed to be approved by the FDA. This allows researchers and physicians to more fully understand the safety and efficacy of the drug. While the system isn't always perfect, it does do a good job of making sure that only safe and helpful drugs get prescribed to patients.
However, there are certain circumstances that will allow the FDA to fast-track approval of drugs, bringing them to market much more quickly.
Genervon applied for expedited approval back in February, and the FDA will be making a decision in April. Nick Grillo, who has been diagnosed with ALS, started a petition to urge the FDA to do Accelerated Approval on the drug for the people suffering just like he is. Since starting his petition, 4 additional similar petitions have been linked together for one common goal.
"Living with ALS is a nightmare," Grillo said in an email to A Plus. "Until now, I have experienced illness as 'you get sick, you get better'. But, with ALS you just watch yourself slowly become more and more paralysed - a shell of the person you once were."
While the creators of the petition state that they understand and respect the reason that drug approvals take so long, this isn't a matter where they can afford to wait any longer.
On Wednesday March 25, ALS advocates will be going to Washington, DC in order to meet with members of Congress, present their petition and urge for GM604 to get Accelerated Approval.
"This campaign is not only about GM604, it's also about getting experimental drugs to patients who are terminally ill with no treatment options," Jehad Majed, who is petitioning on behalf of his brother, told A Plus. "It's about speeding up the trial process for diseases like ALS were patients don't have much time."
Take a minute to learn more about ALS and why those with the disease, and their families, are begging for help.
"Through this petition process and our advocacy efforts I've met so many people from every walk of life; avid runners, people who like to travel, stay at home moms, young professionals like Anthony, and brilliant, positive and loving people just like my uncle," Bethany Wolfe explained to A Plus.
Wolfe became active in ALS advocacy when her uncle was diagnosed last year, and started one of the petitions that was linked in to Grillo's.
"Before this happened, I didn't realize how many people deal with the heartbreak and struggle that comes with this illness. I didn't know that it is terminal and that to-date there is only one other approved drug to combat the degenerative effects. I just didn't know anything about it."