Treatment of NF1-Driven Neurofibromas through VDR-Mediated Stromal Reprograming
Abstract
Virtually every patient diagnosed with neurofibromatosis is faced with the challenge of managing neurofibroma tumor growth. This can range from the emotional difficulties of cosmetically disfiguring dermal neurofibromas to the more painful growth of deep-tissue plexiform neurofibromas. Here we propose to take a radically new approach to target to neurofibromatosis-associated tumors by breaking down their stromal support network with a clinically approved class of drugs that target the vitamin D receptor (VDR). We will test if this VDR stromal remodeling therapy is sufficient to impact tumor growth on its own and explore its potential to work in combination with clinically approved MEK inhibitors. Of particular relevance for patients, VDR therapies are relatively safe for long-term treatment and are already available in the clinic, allowing this work to immediately translate into clinical trials.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2021
- Accession Number
- AD1164129
Entities
People
- Ronald M. Evans
Organizations
- Salk Institute for Biological Studies