The U.S. Food and Drug Administration (FDA) has unveiled a new plan to treat rare neurodegenerative diseases. Most prominently, the five-year plan targets amyotrophic lateral sclerosis, also known as Lou Gehrig's Disease or ALS.

A neurodegenerative disease is a condition that occurs when the nerve cells of the brain or peripheral nervous system start to lose function and die. The most commonly known neurodegenerative diseases are Alzheimer’s disease and Parkinson’s disease. While more common neurodegenerative conditions can be devastating, many of them have some form of treatment. According to a statement from FDA Commissioner Robert M. Califf, there are “very few effective therapeutic options available” for patients with rarer neurodegenerative diseases.

One such uncommon neurodegenerative condition is ALS. According to the ALS Association, the disease is a progressive neurodegenerative condition that affects nerve cells in the brain and spinal cord. ALS causes scarring in the spinal cord around areas where muscle nerves die. When these muscle nerves die, the brain loses the ability to affect them, leading to the inability to speak, move, or breathe.

The stated goal of the FDA’s five-year plan is twofold. First, the plan aims to “Foster the development of safe and effective drugs that improve or extend, or both, the lives of people living with ALS and other rare neurodegenerative diseases.” Second, the plan will enable access to investigational drugs for treating or preventing ALS or other rare neurodegenerative diseases.

In order to accomplish these goals, the FDA is partnering with the National Institutes of Health, the Department of Health and Human Services, and any outside organizations that the agency may need. The FDA is authorized to award grants and contracts to public or private entities that engage in research and development of interventions intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases.

By the end of fiscal year 2022, the FDA intends to establish a “Rare Neurodegenerative Diseases Task Force” and establish partnerships for researching these conditions. The agency also outlined ongoing plans to develop scientific strategies tailored to the research of these diseases. The ALS science strategy has been specifically devised to better characterize the progressions, predictive markers, and natural history contributions of the disease.

The FDA also intends to reduce “barriers and burdens” faced by populations to facilitate participation in clinical trials and access to new drugs. Another effort to help the project is the agency’s intent to enhance clinical infrastructure to more efficiently select therapeutic candidates for trials and more optimally designed clinical trials. These efforts in turn will improve access to clinical trials, streamline operation, and reduce the resources needed to develop new drugs.

Commissioner Califf commented on the plan, stating, “This action plan, especially including the use of public-private partnerships and direct involvement of patients, will ensure the FDA is working toward meeting the task set forth by Congress to enhance the quality of life for those suffering by facilitating access to new therapies.”