Massachusetts General Hospital will test the application of positron emission tomography to see if it can accurately detect inflammation as a biomarker for the diagnosis of ALS, a progressive neurodegenerative disease.
The Boston research hospital’s work will be funded by a $750,000 human clinical trial grant from the Muscular Dystrophy Association. The impetus for the grant is a partnership between four leaders in ALS treatment development—Massachusetts General, ALS Therapy Development Institute, the University of Massachusetts Medical School and Compassionate Care ALS.
The four organizations are partnering to develop new approaches to improve care for those who have ALS.
The grant for PET imaging will assess the value of the technology in testing healthy people who carry a known ALS gene; it also will be tested in people who already are showing symptoms of early-stage ALS.
The project is intended “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,” says Nazem Atassi, MD, who is leading the project.
ALS affects muscle-controlling nerve cells in the brain—patients eventually lose the ability to initiate and control muscle movement, which leads to total paralysis and death. The development of drug treatments for the disease has been hampered by the inability to identify biomarkers that indicate drug efficacy in patients.
Researchers hope that PET imaging will enable the identification of these biomarkers and quicker tests of treatments. At Mass General, researchers will be assessing the ability of PET imaging to measure the amount of inflammation in the brains of patients with ALS; earlier studies have found that those with ALS showing signs of inflammation tend to be more advanced in the progression of the disease with more impaired function.
Researchers will test the use of PET imaging; they believe that its use in future trials can reduce the time it takes to complete them, to as little as three months from 12 months. Also, the use of PET imaging could trim the number of patients needed for trials, to 30 patients from current levels of 400 patients.
Ultimately, the use of PET imaging to identify potential biomarkers will aid researchers and pharmaceutical companies seeking to develop drugs for treating ALS, Atassi believes.
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