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 ¡ª and the drug worked for all 10.
¡°That¡¯s the power of the ,¡± says , 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 ¡ª short for Poly (ADP-ribose) polymerase ¡ª 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.












