SOAR-NPC is comprised of family foundations and leading NPC research scientists who work with an extensive network of other scientific, industry, and government collaborators. Together, we have developed a focused approach, working expeditiously to identify new disease targets and biomarkers and to test promising compounds, moving any therapeutic agents with significant impact on disease progression towards clinical trial.
In collaboration with many scientists, individuals, and other organizations within the NPC community, SOAR is working on several therapeutic approaches to treat NPC. Compounds, in different stages of development, include:
- VTS-270 (a specific formulation of hydroxypropyl-beta-cyclodextrin) is currently in a Phase 2b/3 clinical trial sponsored by Vtesse, Inc. Read more about the trial.
- Vorinostat is in a proof-of-concept Phase 1 clinical trial. The Ara Parseghian Medical Research Foundation (APMRF) contributed extensively to funding of preclinical work and pushing Vorinostat forward into clinical trial. Read more about the trial.
- Gene therapy is being pursued to introduce functional copies of the NPC1 gene (DNA) into cells. Approximately 95% of NPC patients have a problem with the NPC1 gene.
- Scientists are screening drug compounds already FDA approved for use in humans to treat other diseases, a strategy which can significantly accelerate clinical trial entry.
- Evaluation of new drug compounds that affect proteostasis – the network of proteins that help stabilize or increase the expression of NPC mutant proteins such that some NPC protein is available to do its job – is underway.
- Investigation into new therapeutic leads and approaches that can be applied to NPC disease is always in progress.
Over the past eight years, SOAR has made significant progress in developing a drug pipeline for NPC therapeutics. Additionally, SOAR scientists have identified disease biomarkers to promote earlier diagnosis of the disease and facilitate clinical testing of new therapies. See Our Milestones below for additional information on SOAR’s progress.