Adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)/CSF1R related leukoencephalopathy
From the article – Vigil Neuroscience Announces First Subject Enrolled in a Natural History Study of Patients with Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP) – access: https://www.businesswire.com/news/home/20210915005126/en/Vigil-Neuroscience-Announces-First-Subject-Enrolled-in-a-Natural-History-Study-of-Patients-with-Adult-Onset-Leukoencephalopathy-with-Axonal-Spheroids-and-Pigmented-Glia-ALSP
Adult-onset leukoencephalopathy with axonal spheroids and pigments (ALSP) is a rare neurodegenerative condition caused by mutation in the gene CSF1R. The mutation leads of dysfunction of a type of cell in the central nervous system (i.e., the brain and spinal cord), known as microglia. This microglial dysfunction leads to damage to the nerve cells of the CNS, resulting in neurological symptoms in patients affected by ALSP. Currently, there are no approved treatments for ALSP, and more generally, a large unmet need for treatment for other neurological diseases thought to be characterised by microglial dysfunction, including Alzheimer’s Disease. Vigil Neuroscience is a pharmaceutical company focussed on developing therapeutics specifically targeting this cell type. The compound VGL101, currently undergoing development with the view of shortly obtaining ‘Investigational New Drug’ (IND) status from the FDA, is one such therapeutic. The scientific rationale behind VGL101 is that the cellular pathways in microglia which become disrupted following mutation of CSFR1 may also be activated by a protein known as TREM2. VGL101 stimulates TREM2, thus compensating for CSFR1 loss. Obtaining IND status will mean that Vigil Neuroscience will be able to begin conducting clinical trials with the aim of establishing whether VGL101 could be a viable treatment option for ALSP.
Prior to any clinical trials beginning with VGL101 in this patient group however, Vigil Neuroscience have launched a prospective ‘natural history’ study of ALSP, which was designed in collaboration with patients, physicians and other advocates. Researchers running the study will follow a number of individuals with ALSP across the US, Canada and Europe. The aim of this study is to better understand how ALSP progresses, with the view of identifying relevant clinical measures which can later be used to assess the impact of VGL101 treatment. Researchers will evaluate the results of imaging and other clinical tests ALSP patients undergo, with the view of determining, for example, which biomarkers (a measurable indicator of a state of health or disease in a person) will be most useful to incorporate into a future VGL101 clinical trial.