Zbigniew Wszolek, MD, Mayo Clinic, Jacksonville, FD, discusses IGNITE, a Phase II study evaluating the use of VGL101 for the treatment of patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). ALSP is a rare, fatal, autosomal-dominant neurodegenerative microgliopathy caused by a CSF1R gene mutation. The standard therapy for ALSP is bone marrow transplant. More than 20 cases have been treated, with mixed results, although earlier treatment has had more promising results. As TREM2 regulates CSF1R, it is hypothesized that TREM2 activation by VGL101 could stimulate CSF1R and prevent loss of function. The interim results from the trial are promising, with no severe adverse events and positive results seen on MRI measures and disease-related fluid biomarkers. This interview took place at the European Academy of Neurology (EAN) Annual Meeting 2024 in Helsinki, Finland.
These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.
