Bottom line: Converting discoveries into new treatments at a faster rate
When a clinical trial for a new breast cancer drug launched recently, researchers estimated it would take at least two years to enroll the 20 patients needed to make the trial a success. However, within four months, half the patients had already enrolled — and the drug worked for all 10.
“That’s the power of the Moon Shots Program,” says Jennifer Litton, M.D., associate professor of Breast Medical Oncology and the trial’s principal investigator.
Accelerating the conversion of scientific knowledge into new treatments for patients is a priority of the program.
“What moon shots has done for my patients is cut the time it takes to go from a clinical trial idea to the time a person gets a new drug down from three years to about one. That’s huge,” says Litton, an experienced leader of clinical trials.
The drug, called talozoparib, is currently being tested in late-stage breast cancer patients who have BRCA1 and 2 gene mutations and whose disease has spread.
Those who test positive for BRCA mutations have a significantly higher risk of developing breast and ovarian cancers, as well as passing on the mutation to future generations.
Owned by Medivation Inc., talozoparib is what is known as a PARP inhibitor. PARP — short for Poly (ADP-ribose) polymerase — is an enzyme that helps repair damaged DNA. In cancer treatment, blocking PARP may prevent such repairs, causing the cells to die.
Litton’s trial uses a new targeted therapy for two months before proceeding to standard of care chemotherapy followed by surgery. The trial and accompanying moon shot studies will provide a detailed view of talozoparib in first-line use.
“In this and other studies, we’ve found that when companies know we have the moon shot behind us, we’ve been given access to drugs they won’t give to anyone else for investigator-initiated trials because we can promise them the trial will happen,” Litton says.
Not only does the moon shot provide funding for the study, it supplies a deep roster of scientists drawing on expertise from across the institution to provide the pharmaceutical corporation an unprecedented opportunity to learn about its drug.
“It’s low risk for them. And on top of that, they’re getting correlative study information they can’t get from other places now.”
The previously untreated patients enrolled in Litton’s trial have biopsies done before and after treatment. Moon shot co-leader Gordon Mills, M.D., Ph.D., chair of Systems Biology, will oversee genetic and molecular analysis of the tumors both before and after. Provost Helen Piwnica- Worms, M.D., Ph.D., will cultivate grafts of the human tumors in mouse models to further understand the mechanisms of the disease and drug.