A novel nanoparticle formulation of an existing PARP-1 inhibitor and an associated administration regimen has been developed for the treatment of Ewing Sarcoma. This technology allows the therapeutic treatment to be targeted to the tumors, resulting in reduced required doses and system toxicity.
Background:
Ewing Sarcoma is rare cancer of the bone that primarily affects adolescents and young adults. It occurs in approximately 3% of adolescents 10-19 years old and is considered an orphan disease. It has a fairly high survival rate if diagnosed before metastasis; however, those diagnosed after metastasis have a poor prognosis with a survival rate of only 25-30%. Current treatment options are limited and many patients do not respond to them.
The novel nanoparticle formulation has been tested in a xenograft mouse model of Ewing Sarcoma and has shown significant effectiveness with complete tumor disappearance without recurrence during the study duration (10 weeks). The nanoparticles can be used for an individual therapeutic or for combination treatment. The targeting of the nanoparticle to the tumor allows higher, lethal doses to be administered to the tumor, while the systemic doses are much lower than the current chemotherapy regimens. The result is increased tumor cytotoxicity but lower systemic side effects.
Commercial Applications & Advantages:
This nanoparticle technology has the following advantages to the current standard of care when treating Ewing Sarcoma:
- Targeted Action
- Reduced Toxicity
- Metastatic Efficacy - demonstrated effectiveness against low-survival, metastatic disease