The Muscular Dystrophy Association (MDA) announced that it has awarded a human clinical trial grant totaling $750,000 to Massachusetts General Hospital. This important grant was catalysed by ALS ONE, a partnership between four institutional leaders in ALS treatment development: Massachusetts General Hospital (MGH), ALS Therapy Development Institute (ALS TDI), University of Massachusetts Medical School and Compassionate Care ALS (CCALS).
MDA research grant awarded to ALS ONE will fund exploration of Positron Emission Tomography (PET) imaging of inflammation in people with ALS
The ALS ONE partnership was developed to expedite treatments for ALS and develop novel approaches to improve care for those living with the disease. The new 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. The project, led by Nazem Atassi, M.D., MMSc, will assess the value of PET imaging in healthy people who carry a known ALS gene and in symptomatic people with early-stage ALS. Atassi is 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.
“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. “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.”
ALS is a progressive neurodegenerative disease that affects muscle-controlling nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which leads to total paralysis and death, usually within five years of diagnosis. One of the main roadblocks for ALS drug development is the lack of biomarkers that can signal drug efficacy in patients. With recent advances in the understanding of ALS and the exciting drug development pipeline, tools such as improved imaging biomarkers are needed to allow researchers to quickly test many treatments.
At Massachusetts General Hospital, researchers at the Neurological Clinical Research Institute (NCRI) plan to assess the ability of PET imaging to measure the amount of inflammation in the brain in people with ALS, as early data shows significantly increased inflammation in the motor regions in people with ALS. In previous studies it has been found that people with ALS who have more inflammation tend to be more advanced in their disease progression with more impaired function. Implementing this novel PET imaging technology potentially could reduce the duration of future trials from 12 to 3 months, and the required patient enrollment from 400 to 30 patients. This would add an enormous degree of efficiency to ALS drug development and the ability to test more treatments faster.
According to Atassi, the funding from MDA will enable his team at the NCRI to build on initial findings and allow for better characterisationof the onset and progression of brain inflammation in people with very early ALS symptoms and in people who carry one of the ALS genes but are asymptomatic. Additionally, this project will contribute to the growing body of evidence suggesting PET imaging can be used as a tool to accurately measure inflammation in the brains of people living with ALS. Ultimately, this biomarker will be available for any researcher or pharmaceutical company to use for drug development in ALS. Assembling a diverse cache of biomarker tools will help to encourage new biotech and pharma entrants to develop safe and effective treatments for people with ALS.
"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.”
Kevin Gosnell founded ALS ONE in January 2016 to expedite progress toward finding a treatment for ALS by 2020 while improving care now. Gosnell passed away in August 2016 due to complications from ALS, following his final meeting at which he spoke with MDA about partnership opportunities. The ALS ONE partnership has already placed resources and expertise into a powerful, coordinated team with aligned goals and the ability to move ideas and products quickly from inception to trial and from idea into care practice. Kevin’s vision continues today with this new funding commitment from MDA.