“For the past two weeks you have been reading about a bad break. Yet today I consider myself the luckiest man on the face of the earth.” Lou Gehrig spoke to a Yankee Stadium crowd of 61, 808 on July 4, 1939 following the publicization of his amyotrophic lateral sclerosis (“ALS”) diagnosis. As a result of Gehrig’s diagnosis and subsequent retirement from baseball, ALS became a well-known disease thanks to the man who considered it to be “a bad break.”
For approximately 20,000 Americans currently living with ALS, the disease is more than “a bad break.” For those with ALS, they must live with the progressive loss of control over their bodies. Voluntary movements such as walking, talking, breathing, and chewing become difficult. The nerve cells that control these voluntary movements are attacked and killed by the disease. As it progresses, ALS weakens and paralyzes the muscles, eventually leading to death. There are currently no cures for ALS.
In response to ALS and other neurodegenerative diseases, President Biden signed the “Accelerating Access to Critical Therapies for ALS Act” (ACT for ALS) into law on December 23, 2021. The ACT for ALS requires that the Food and Drug Administration (“the FDA”) publish and implement a 5-year action to foster drug development and facilitate access to investigational drugs for ALS and other rare neurodegenerative diseases.” Additionally, the ACT for ALS also requires the FDA to award grants or contracts to public and private entities to fund research and developments of interventions meant to prevent, diagnose, mitigate, treat, or cure ALS and other rare neurodegenerative diseases in children and adults.
The action plan that FDA developed per ACT for ALS outlines that FDA will engage in the following: establish the FDA Rare Neurodegenerative Diseases Task Force (FY 22), establish the Public-Private Partnership for Rare Neurodegenerative Diseases (FY 22), develop disease-specific science strategies (FY 22 – FY 26), and leverage ongoing FDA regulatory science efforts. The science strategies FDA developed for ALS addresses current obstacles and challenges to the development of ALS drugs.
On September 29, 2022, FDA approved the new drug Relyvrio (sodium phenylbutyrate/taurursodiol) for treatment of ALS. The drug causes a slower rate of decline and a longer overall survival for patients. It can be taken orally or through a feeding tube alongside a snack or meal. The drug’s efficacy was demonstrated in a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study. Patients received either Relyvrio or a placebo, with those receiving the drug experiencing a slower rate of decline than those who received the placebo.
The ACT for ALS appears to be doing its job: spurring more treatment options for patients with ALS and more development for therapies for neurodegenerative diseases. For those experiencing “a bad break,” there is now more hope for the future as more research is incentivized to find treatments for ALS and other rare neurodegenerative diseases.