July 2020 Foundation eNews

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HEALEY ALS Platform Clinical Trial Update

Our Les Turner ALS Center at Northwestern Medicine is a participating site for the first-ever ALS platform trial.

The clinical drug trial, led by the Sean M. Healey & AMG Center at Massachusetts General Hospital, aims to “accelerate the path to new ALS therapies by testing multiple treatments at once, reducing the cost of research by 30%, decreasing the trials time by 50% and increasing patient participation by 67%.”

Study leaders have adapted the trial to comply with COVID-19 precautions and are giving a webinar TODAY at 4 pm CST to share updates on the status of the trial. Sign up for the webinar here>> And learn more about the platform trial on our website>>

P.S. Stay tuned for the launch of the HEALEY ALS Platform Trial page on our website!


Advocating for Easier Access to Therapies

The Les Turner ALS Foundation has joined with the ALS community in support of two pieces of legislation – the Promising Pathway Act (S. 3872) and the Accelerating Access to Critical Therapies for ALS Act (H. 7071), which is co-sponsored by one of our Illinois members of Congress, Rep. Mike Quigley (IL-5).

“We are grateful to partner with patients and fellow patient advocacy groups to advance these initiatives, which are desperately needed,” said Foundation CEO Andrea Pauls Backman.

“One of the pillars of our mission is to advance scientific research into the causes, treatment and prevention of ALS, and we believe these bills, co-produced with people living with ALS, will provide earlier and easier access to promising therapies that could ultimately lead to a cure.” Learn more about the bills here>>


ALS Mutation Disrupts Cell-Cleaning Process

A multi-institutional collaborative study of the ALS gene mutation UBQLN2 has successfully utilized human cell lines and a mouse model, demonstrating additional defects in the body’s ability to remove unnecessary or dysfunctional cells. This paper was published in Proceedings of the National Academy of Sciences of the U.S. (PNAS).

“The discovery of the UBQLN2 gene mutation in my laboratory in 2011 was a key step,” shared Teepu Siddique, MD, DSc, co-author of the paper and member of our Les Turner ALS Center at Northwestern Medicine. “UBQLN2 is an important component of the two key protein recycling and systems in neurons and other cells.”

UBQLN2 may provide a promising target for the development of new therapeutics by focusing on restoring acidification defects to treat ALS and other neurogenerative diseases, like Alzheimer’s, Parkinson’s and Down Syndrome. Check out the full publication here>>