The Muscular Dystrophy Association Awards Human Trial Grant for ALS Research

The Muscular Dystrophy Association (MDA) has awarded Massachusetts General Hospital (MGH) a $750,000 human clinical trial grant for the development of treatments for amyotrophic lateral sclerosis (ALS), known as Lou Gehrig's disease, according to a press release.

ALS ONE, a partnership among leaders in ALS treatment—MGH, ALS Therapy Development Institute, the University of Massachusetts Medical School, and Compassionate Care ALS—developed the grant. This partnership was created in January 2016 to expedite ALS treatments and to develop new approaches to improve ALS care.

The MDA grant intends to specifically fund research that explores the use of positron emission tomography (PET) imaging of inflammation to serve as a biomarker for ALS diagnosis and clinical trials, according to the press release.

“This project is poised to change the paradigm of ALS drug development and have a direct impact on the design of future treatment trials for both familial and sporadic ALS,” said Nazem Atassi, MD, MMSc, member of the ALS ONE Science Team, Associate Director for the Neurological Clinical Research Institute at Massachusetts General Hospital, and Associate Professor of Neurology at Harvard Medical School.

Patients with ALS often lose the ability to control their muscle movements leading to total paralysis and death, typically within 5 years of diagnosis. One of the challenges with ALS drug development is the lack of existing biomarkers that demonstrate the efficacy of the drugs in patients.

MGH’s Neurological Clinical Research Institute is assessing the ability of PET imaging in order to measure the inflammation in the brain of ALS patients; previous studies have shown ALS patients with more inflammation tend to have greater disease progression. The novel PET imaging that the grant would fund could potentially decrease the duration of future trials from 12 months to just 3 months and decrease the required patient enrollment from 400 to just 30; therefore, increasing the efficiency of ALS drug development.

Atassi explained that the funding can allow researchers to better characterize the onset and progression of brain inflammation in patients with early ALS symptoms and in people who are asymptomatic but carry ALS genes.

"At MDA, we are hopeful that this project will build upon recent advances in biomarker development for ALS to not only accelerate drug development, but also provide meaningful insights into the course of inflammation in people with ALS," said MDA Scientific Program Officer Amanda Haidet-Phillips, PhD.

The development and use of biomarker tools will encourage new biotech pharma entrants to develop more safe and effective treatments for people with ALS, MDA stated.