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New Results From HEALEY ALS Platform Trial Show Promise for Treating ALS

New Results From HEALEY ALS Platform Trial Show Promise for Treating ALS

MONTREAL, Oct. 18, 2023 /PRNewswire/ -- One of the central challenges of Amyotrophic Lateral Sclerosis ALS research is finding ways to slow the progression of the relentlessly degenerative brain disease. Recently, based on results of the HEALEY ALS Platform Trial, two experimental medications plan to move to phase 3 clinical trials. This means the drugs will now be tested on a larger sample population to confirm promising early results that showed they can slow the progression of ALS. Phase 3 trials are the last step before drugs are submitted for FDA approval.

Merit Cudkowicz, MD, chair of neurology and director of the Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, revealed the results from the recent HEALEY Platform Trials and spoke about the promising growth in the number of available ALS treatments over the past decade at the 26th World Congress of Neurology. There are currently five approved drugs on the market for ALS as opposed to one 10 years ago, and the HEALEY ALS Platform Trial is accelerating the search for more treatments by enabling researchers to test multiple drugs simultaneously.

"It has been a game changer to have all these new treatments," said Dr. Cudkowicz. "It used to be that when we saw people with ALS, there wasn't a lot we could do to treat the disease. Now, all of a sudden, there is actually a lot we can do that has a direct impact on people's quality of life. There is still a great need for ALS research to better understand the underlying biologies and to develop more targeted treatments. There is a large community of scientists, clinicians, foundations and people living with ALS working together to solve ALS."

HEALEY ALS Platform Trial Accelerates Clinical Trials by Testing Multiple Treatments at Once
The HEALEY ALS Platform Trial is a first-of-its-kind effort to test multiple experimental treatments simultaneously and on a rolling basis. The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital reports that the HEALEY Platform Trial reduces the cost of research by 30% and decreases the average length of trials by 50%, allowing scientists to test a wider range of drugs much quicker than traditional clinical trials.

The HEALEY ALS Platform Trial began in 2020 and has since completed 5 rounds of clinical trials with two upcoming trials actively recruiting participants.

Proposed Biomarker Advances Research Into New ALS Treatments
The growing number of approved ALS therapies is in part due to a better understanding of the causes of the disease, including the recent discovery of a proposed biomarker for ALS called neurofilament. A biomarker is something doctors can test for in order to confirm the presence of a disease. Neurofilament is a protein that is important in the structure and function of nerves and their connections to each other and muscles. When ALS begins to damage these nerve fibers, neurofilament breaks down and can be detected in the blood and spinal fluid.

This helps doctors identify whether a medication is working and makes it much easier and quicker for researchers to do clinical trials of new therapies. "It offers a way to tell whether your drug might work or not [by studying] fewer people in a shorter time, and that accelerates progress in the field," said Dr. Cudkowicz.

Neurofilament is currently the best proposed biomarker with many more in development. More research is needed to fully understand what it means and how to interpret changes in the blood and spinal fluid levels. However, researchers are already using neurofilament as a way to test the effectiveness of certain ALS medications. In fact, the Food and Drug Administration gave accelerated approval to a drug for a genetic form of ALS in April based on its ability to significantly lower levels of neurofilament during clinical trials.

Gene Therapy Offers Scientists a Way to "Turn Off" ALS-Causing Genes
Advancements in gene therapy are also allowing scientists to slow the progression of some types of ALS by deactivating the genes that cause the disease. Dr. Cudkowicz hopes this will lead to similar treatments for other types of ALS in the near future.

"The ability to turn on and turn off genes in the human brain and spinal cord is relatively new, but it is very powerful," said Dr. Cudkowicz. "While these approaches can help the 10 to 20% of people who have a gene that causes ALS, it can also be very helpful for people who don't carry a gene. The same tools that can turn genes on and off to treat the genetic form can be used for the non-genetic form. In fact, this is already happening in trials in ALS"

This growing understanding of ALS is also driving more research across the field. In all, there are currently over 50 active trials targeting specific aspects of the disease, including symptoms, diagnosis and treatment.

"ALS is one of the most serious illnesses because it strikes people at all ages," said Dr. Cudkowicz. "Everyone in the ALS field is inspired to do something and to do it rigorously, with speed and urgency. We have enough knowledge about the biology of the disease, and we're having some successes [with treatment]—it finally feels like now is the time when we could turn this illness into something that's manageable."

Visit to learn more about Dr. Cudkowicz and all the featured research at this year's WCN.

About the World Congress of Neurology

The World Federation of Neurology's World Congress of Neurology brings together leading neuroscientists and public health experts to turn research into action and emphasize the importance of brain health across the globe. The 26th biennial conference was held in Montreal from October 15 to 19, 2023, and was co-hosted by the Canadian Neurological Society (CNS).

About the World Federation of Neurology

With support from its 123 national Member Societies, the World Federation of Neurology fosters quality neurology and brain health worldwide by promoting neurological education and training, emphasizing under-resourced areas of the world. As a non-state actor in official relations, WFN supports the World Health Organization (WHO) efforts to give everyone an equal chance to live a healthy life. With Member Societies around the globe, WFN unites the world's neurologists to ensure quality neurology and advocate for people to have better brain health. Learn more about the World Federation of Neurology at

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