November 25, 2010
Novel treatment for neuroblastoma and other tumors in the central nervous system.
- Induces cell death in tumor cells while having no negative effects on surrounding neurons.
- Slows the rate of neuroblastoma tumor growth.
Emory researchers have shown that progesterone caused significant cell death in tumor cells while remaining absolutely safe in primary cortical neurons. After a single treatment of progesterone a significant decrease in cell survival on human neuroblastoma was observed in vitro. Cell death of human neuroblastoma and glioblastoma cells increased after repeated treatments of progesterone. However no cell death was observed in primary cortical neurons at any concentration of progesterone even after six days of treatment. In addition, progesterone slowed the growth rate of neuroblastoma tumors in vivo, specifically within a mouse xenograft model of neuroblastoma.
Neuroblastoma is often treated with surgery and chemotherapy. However it frequently recurs in high-risk cases and eventually progresses and proves lethal in more than 70% of cases. The majority of survivors have long-term effects from the high drug doses necessary for treatment. Despite a number of clinical trials, a safe and effective drug that avoids the adverse side effects of very high drug doses has not yet been found. Progesterone has a high safety profile, shows limited side effects in current clinical testing for brain injury, and is easy and safe to administer. Because of its overall safety and effectiveness on tumors, progesterone offers a potential treatment for neuroblastoma and other central nervous system tumors like glioblastoma.
Developmental Stage & Potential Market
- Proof of principle has been effectively demonstrated in rodent models.
- The use of Progesterone for the treatment of traumatic brain injury is currently in Phase III trials.
- Between 20-50% of high-risk cases of neuroblastoma do not respond adequately to chemotherapy treatment and are progressive, refractory, or recurrent.