Sponsor link https://www.ctf.org/research/drug-discovery-initiative-registered-report...
DRUG DISCOVERY INITIATIVE (DDI)
The goal of the CTF Drug Discovery Initiative (DDI) program is to stimulate NF drug discovery by funding researchers proposing to investigate novel or repurposed therapies for NF or to develop tools that support such research. Proposals are expected to be short and concentrated on obtaining key preliminary data needed to quickly advance to the next step of drug discovery.
Specifically, the aims of the DDI program are to -
- support early stage testing of therapeutic compounds for the treatment of NF
- support generation of new in vitro or in vivo model systems for NF
NF causes a range of central and peripheral nervous system tumors (benign and malignant), bone abnormalities, learning disabilities, pain, vascular complications, and other manifestations. The progress of NF is unpredictable and often presents a chronic lifelong burden to the affected person. There is a need for drug management but, other than Koselugo (selumetinib) for pediatric NF1 plexiform neurofibromas, there are no effective therapies for the diverse manifestations of NF. The signaling pathways affected in NF are common to many other tumor disorders and many existing drugs developed for these disorders could be repurposed for NF.