Richard J. Bram, M.D., Ph.D.

Can you describe your drug development project?

We are performing high-throughput screening to discover new compounds that block a protein called cyclophilin B. In previous experiments, we discovered that many types of brain tumors rely on the cyclophilin B protein for their growth.

We plan to test compounds that bind specifically to cyclophilin B to see if they might lead to new medicines that could more effectively treat brain tumors than current therapies.

What motivated you to get involved in this work?

The treatment regimens for most pediatric cancers have been improving over the years, such that more than half of affected children can be cured. Unfortunately, most children with malignant brain tumors do not share in this favorable outcome. The majority with advanced stage gliomas will not survive. Additionally, even when children do survive, they often have devastating long-term toxicities from the surgery, radiation and chemotherapy treatments for brain tumors.

Clearly, improved modalities that have reduced side effects are needed for children with brain cancers.

What do you hope to accomplish with this project?

We hope to develop one or more new drugs that have improved efficacy and reduced toxicity for treatment of childhood brain tumors.

How has the CCaTS Drug Discovery and Development resource aided your work?

CCaTS funded the initial seed project to explore the feasibility of a high-throughput screen, and also introduced us to our new collaborators at the Sanford-Burnham Medical Research Institute, where the high-throughput screen is being performed. This seed grant allowed us to obtain data that was instrumental in obtaining National Institutes of Health R01 grant funding to support the project.