Rare Disease Report

Six-Figure Grant Expected to Assist Researchers in Finding ALS Biomarkers

MAY 07, 2018
Mathew Shanley
A human clinical trial grant totaling $750,000 was awarded this morning by the Muscular Dystrophy Association (MDA) to Massachusetts General Hospital, with the hope that it will accelerate biomarker research for amyotrophic lateral sclerosis (ALS).

The grant was augmented by ALS ONE, a partnership between 4 institutional leaders in ALS treatment development established to expedite treatments for ALS and develop novel approaches to improve care for those living with the disease. The partnership is between: Massachusetts General Hospital, ALS Therapy Development Institute, University of Massachusetts Medical School and Compassionate Care ALS.

The MDA grant will fund the exploration of the potential for positron emission tomography (PET) imaging of inflammation to serve as a biomarker for ALS diagnosis and clinical trials.

Nazem Atassi, M.D., MMSc, a 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, will lead the project. He and his team of researchers intend to assess the value of PET imaging in healthy people who carry an identified ALS gene, and in symptomatic people with early-stage ALS.

"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," Atassi said in a press release. "Real scientific advances in rare diseases rely heavily on the integration of the research community. Organizations such as MDA and ALS ONE are the strongest links between all key stakeholders including the patients, academic institutions, philanthropy and industry, and we are incredibly grateful for their support."

In ALS, a general lack of biomarkers to signal drug efficacy in patients has served as a major roadblock in the development of novel therapies. It is believed by the MDA and ALS ONE that with recent advances in the understanding of ALS and the drug development pipeline, tools like improved imaging biomarkers are necessary to permit researchers to test several treatments more quickly and effectively.

Early data shows significantly increased inflammation in the motor regions in people with ALS, and previous studies have shown that people with ALS who have more inflammation are more likely to be advanced in their disease progression and with further impaired function. The team of researchers at the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital intend to study the ability of PET imaging to measure the amount of inflammation in the brains of people with ALS.

Implementing a new PET imaging technology could potentially reduce the duration of future trials from 12 months to 3 months, and the required patient enrollment from 400 patients to 3 patients. These breakthroughs would significantly enhance the degree of efficiency to ALS drug development.

Attasi believes that the new grant from the MDA will enable his team to add to initial findings and allow for better characterization of the onset and progression of brain inflammation in people with very early ALS symptoms and in people who care one of the ALS genes but are asymptomatic.

"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, Ph.D. "We are grateful to ALS ONE and Dr. Atassi for their continued commitment to expedite the path to find treatments for ALS."

The project is expected to contribute to the ever-growing body of evidence that suggests PET imaging can be used as a tool to accurately measure inflammation in the brains of people living with ALS.

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