NEW YORK – Biotechnology company Vigil Neuroscience on Monday said it has partnered with InformedDNA and PreventionGenetics to offer free genetic testing and counseling to patients diagnosed with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
Approximately 10,000 people in the US have ALSP, which is caused by a mutation in the CSF1R gene. A similar number of patients in Europe and Japan also have the disease, which causes cognitive, neuropsychiatric, and motor dysfunction. The symptoms show up typically in individuals in their 40s and progress quickly, and after diagnosis, patients tend to live on average six to seven years.
There are currently no approved treatments for ALSP, and access to genetic testing, which is critical for diagnoses, is limited. By advancing the ALSPAware program with genetic counseling services provider InformedDNA and genetic testing provider PreventionGenetics, which is part of Exact Sciences, Vigil is hoping to improve genetic testing and counseling access for patients suspected of having ALSP, who could also be eligible for the company's precision neurodegenerative drug trials.
Vigil's lead therapy, the TREM2-targeting monoclonal antibody VGL101, is undergoing a Phase II proof-of-concept trial in ALSP patients. The Watertown, Massachusetts-based firm is also conducting investigational new drug application-enabling studies for a TREM2 agonist in common neurodegenerative diseases due to microglial dysfunction, focusing initially on Alzheimer's disease in genetically defined subpopulations.
"Limited access to genetic testing and appropriate counseling has led to diagnostic challenges affecting not only those living with ALSP, but also the healthcare providers hoping to treat the disease," Vigil CEO and President Ivana Magovčević-Liebisch said in a statement, estimating that only a third of ALSP patients have the correct diagnosis when their symptoms first manifest. "We believe that everyone deserves access to the services necessary to make the diagnostic journey as efficient and convenient as possible, and we are proud to be taking this important step forward in supporting the ALSP community."