Legislation Seeks Early Access to Promising Treatments for ALS, Similar Disorders

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by Forest Ray PhD |

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Two bills making their way through Congress are intended to improve access to medical therapies for those most in need, and in particular, those with amyotrophic lateral sclerosis (ALS).

Sen. Mike Braun, R-Ind., announced his plan to introduce a bill to hasten the regulatory process for making promising therapies available to patients who need them. The so-called Promising Pathway Act (aka Conditional Approval Act) seeks to amend the Federal Food, Drug, and Cosmetic Act to allow the U.S. Food and Drug Administration (FDA) to provisionally approve therapies that treat conditions in which the disease progresses rapidly and few, or no, treatment options exist.

U.S. Reps. Jeff Fortenberry, R-Neb., and Mike Quigley, D-Ill., announced their intent to introduce the bipartisan Accelerating Access to Critical Therapies for ALS Act into the House of Representatives. This legislation will make $75 million available for grants that fund early access to promising therapies for patients who have fast-progressing neurodegenerative and terminal diseases like ALS.

ALS is a progressive neurological disease characterized by the loss of motor neurons, the nerve cells needed to control movement. It affects roughly one in 300 people, most of whom are 60 or older.

Because patients typically live, on average, two to five years after they are diagnosed, they cannot afford to wait long for new treatment options. Access to promising therapies can be a game-changer for this population.

“[W]hen we find a cure for one of the fastest progressing neurodegenerative diseases, like ALS, we will unlock critical breakthroughs for other diseases like Parkinson’s, Alzheimer’s, Frontotemporal Dementia and beyond, which affect 50 million Americans each year,” Quigley said in a press release. “This legislation not only paves the way to curing a litany of diseases but provides a model for how we speed up our drug treatment pipeline and bring it into the 21st century.”

To that end, the Accelerating Access to Critical Therapies for ALS Act also calls for creating a Center of Excellence for Neurodegenerative Diseases at the FDA to streamline the development and approval of future therapies. The center will be modeled after the FDA’s Center of Excellence for Oncology that already has ushered in several significant discoveries in cancer research.

“Americans with life-threatening and terminal diseases don’t have time to waste on a bureaucratic journey through red tape and regulations, only to be denied access,” said Braun.

Together, these bills should provide better access to therapies for those living with ALS and other fast-progressing and terminal diseases.