The ALS Association

Ice Bucket Challenge Progress
Brian Frederick
The ALS Association
(202) 464-8612



FDA Issues Draft Guidance for ALS Drug Development

Agency credits ALS Association proposed guidance, which was funded by Ice Bucket Challenge funds

Washington, D.C. (February 15, 2018) – The ALS Association applauded the Food and Drug Association’s (FDA) announcement Thursday of its proposed industry guidance for the development of drugs to treat ALS. The FDA’s draft guidance is informed by The ALS Association’s first patient-focused guidance for ALS drug development that was submitted to the FDA in late 2017 and that was funded with donations from the ALS Ice Bucket Challenge.

“Despite the availability of some approved therapies, there is an urgent need to identify additional effective treatments for patients with ALS. We’ve been honored to work with the ALS Association to advance these goals. The ALS Association put together a comprehensive proposed draft guidance of their own, funded by the famous ‘ice bucket challenge,’” FDA Commissioner Scott Gottlieb said in a memo released Thursday.

The FDA guidance and the ALS patient-focused guidance complement each other and will work in tandem to provide a roadmap to help researchers in academic institutions and pharmaceutical companies navigate the drug development process. The goal of both these documents is to increase the efficiency, predictability and speed of the drug development process, including clinical trials, and lead to a more effective and earlier assessment of efficacy.

“We want to commend the FDA for moving forward with the first-ever guidance on drug development for people with ALS,” said Calaneet Balas, president and CEO of The ALS Association. “The FDA guidance and the patient-focused guidance both have the potential to speed up the process of drug development, reduce costs, and create incentives for the industry to enter the ALS market and develop new treatments.”

The ALS Association will be submitting comments on the FDA’s draft guidance and encourages others to do so, as well. The proposed guidance will be available for public comment for 60 days after it is placed in the public register. The final FDA guidance could change from the proposal to reflect feedback from the public. The proposed FDA guidance has not been officially published, but it can be viewed here.

The ALS Association worked with more than 100 participants, including industry, clinicians and researchers, the National Institutes of Health, the Centers for Disease Control and Prevention, and people with ALS and their families to develop the proposed patient-focused guidance.

“We are grateful to the many stakeholders who helped draft the patient-focused guidance document, especially people living with ALS,” Balas said. “We also appreciate the commitment of FDA leadership, including Dr. Scott Gottlieb, Commissioner of the Food and Drugs Administration; Janet Woodcock, Director of the Center for Drug Evaluation and Research and Dr. Billy Dunn, Director, Office of Drug Evaluation Division for Neurology Products. We will continue to push and support the FDA to expand its array of projects to accelerate therapies to people with ALS to meet the urgent need for ALS treatments and a cure.

“This is an important step, but it’s critical that our advocates and allies remain engaged during the next few weeks and weigh in during the public comment period to make sure that the community keeps driving this process forward,” Balas added.

About ALS
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population.

About The ALS Association
The ALS Association is the only national nonprofit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

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