Researchers funded by The ALS Association, through donations from the ALS Ice Bucket Challenge, discovered new evidence on the role that mutant TDP-43 plays in development and progression of the disease. This important work sheds light on novel aspects of TDP-43 biology and provides valuable tools to gain insight into early stages of ALS disease progression and could lead to the development of new therapies.

Treeway, a biotech company in the Netherlands with a mission to develop a cure for ALS, recently announced promising results of its phase I trial of its lead drug, TW001. TW001 is an oral formulation of edaravone. Radicava®, the most recently FDA approved drug to treat ALS, is an intravenous version of edaravone. The study showed that TW001 was well tolerated and stable, while improving bioavailability compared to Radicava.

unded by The ALS Association, NeuroBANK™ is the patient-centric platform for clinical research for numerous Association programs developed by the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital. The platform received the Best Practices Award for Personalized & Translational Medicine at the 2018 Bio-IT World Conference and Expo in Boston earlier this month.

A recent study led by Dr. Bjorn Oskarsson from the Mayo Clinic Jacksonville and supported by The ALS Association demonstrated that mexiletine, a drug approved by the U.S. Food and Drug Administration, reduced the frequency and severity of muscle cramps in people with ALS compared to a placebo.

The ALS Association is happy to continue our tradition of supporting bright, young scientists in ALS research through our Milton Safenowitz Postdoctoral Fellowship Program. These awards encourage young scientists to enter and, importantly, to remain in the ALS field.

The ALS Association is happy to continue our tradition of supporting bright, young scientists in ALS research through our Milton Safenowitz Postdoctoral Fellowship Program. These awards encourage young scientists to enter and, importantly, to remain in the ALS field.

Biohaven Pharmaceuticals, Inc. initiated an expanded access program (EAP) of BHV-0223 drug, a sublingual, lower dose formulation of Rilutek® (riluzole). The drug uses a Zydis® orally dissolving tablet technology, which does not require swallowing or additional fluids. Riluzole is the first drug approved by the U.S. Food and Drug Administration to treat ALS. It prolongs life approximately three months.

Researchers around the world working together for treatments and a cure for ALS are a main reason why we’re on the verge of changing the nature of the disease forever. As part of National ALS Awareness Month, we sat down with two ALS researchers at Johns Hopkins University in Baltimore who are funded by The ALS Association.

The 70th Annual American Academy of Neurology meeting, held in Los Angeles last week, provided an opportunity to check in on antisense therapies and the continued dividends from The ALS Association’s early investment in the technology.

At last week’s 70th Annual American Academy of Neurology (AAN) Meeting in Los Angeles, neuroscientists and neurologists from all over the world came together to present their important work and learn from each other. We are proud that many top ALS researchers we support attended and presented a wide range of research, on topics ranging from environmental risk factors to work leading up to clinical trials.

Neuraltus Pharmaceuticals, Inc. reported disappointing results from its phase II clinical trial studying the impact of an immune regulator, NP001, in people living with ALS who had increased levels of inflammation. The company presented the negative results during the 70th Annual American Academy of Neurology Meeting in Los Angeles.

Dr. Timothy Miller, the David Clayson Professor of Neurology from the Washington University School of Medicine in St. Louis accepted the 2018 Sheila Essey Award for ALS Research April 23 at the American Academy of Neurology 70th Annual Meeting in Los Angeles.

To make cell characteristics visible to the human eye, even under a microscope, scientists normally use chemicals that can kill the very cells they want to observe. Dr. Steven Finkbeiner, director and senior investigator at the Gladstone Institutes in San Francisco (pictured above), recently teamed with computer scientists at Google for a groundbreaking new study funded by The ALS Association Neuro Collaborative through ALS Ice Bucket Challenge donations.

Late last month, Congress passed a $1.3 trillion fiscal year 2018 Consolidated Appropriations spending bill that included a $3 billion increase to funding for medical research to the National Institutes of Health (NIH). Funding to the NIH has increased to $37 billion, the largest bump they have seen in years.

Researchers at Cedars-Sinai in Los Angeles, funded by The ALS Association with ALS Ice Bucket Challenge donations, are dedicated to finding unique avenues for treating ALS. Using animal models, they transplanted specially engineered neural cells into the motor cortex of the brain, the area responsible for muscle movement.

Lauren Sciences LLC, a private biotechnology company in New York, N.Y., recently received a third grant from The ALS Association to support the continued development of LAUR-301, Lauren Sciences’ novel V-Smart Nanomedicine designed specifically for ALS.

Today, we are happy to be joined by ALS clinician scientist Dr. Peter Creigh from the University of Rochester in Rochester, N.Y., the recipient of the 2018 Clinician Research Training Fellowship in ALS Research. The fellowship is given by The ALS Association, in partnership with the American Academy of Neurology (AAN).

The ALS Association is happy to continue our tradition of supporting bright, young scientists in ALS research through our Milton Safenowitz Postdoctoral Fellowship Program. These awards encourage young scientists to enter and, importantly, to remain in the ALS field.

The ALS Association is committed to helping improve clinical trial design, in order to increase trial efficiency that will more quickly lead to effective therapeutics. We awarded Dr. David Ennist and colleagues at Origent Data Sciences, Inc. two grants to support research exploring how machine learning algorithms, a type of computational tool, can optimize clinical trial design. Dr. Ennist’s work, recently published in the journal Annals of Clinical Trial and Translational Neurology, looks closely at optimizing patient randomization into clinical trials.

UPDATE: As of March 14, 2019, enrollment for the CENTAUR Phase II clinical trial has been completed. The CENTAUR phase II clinical drug trial, sponsored by Amylyx Pharmaceuticals and funded by ALS Ice Bucket Challenge donations, began an open-label extension, giving people with ALS who completed their trial period an opportunity to continue taking the drug, AMX0035.