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Today we are thrilled to announce that the U.S. Food and Drug Administration (FDA) approved rucaparib, a new medication to treat some patients with advanced prostate cancer. PCF science is proud to have been involved since the beginning, in every stage of the research that lead to this development. This is a historical first, in what PCF hopes will be a long line of personalized medicines for prostate cancer.

As with most FDA approvals, the journey from idea to approval is long and thorough. In 2015, the PCF-SU2C International Prostate Cancer Dream Team published a landmark study demonstrating that about a third of mCRPC (metastatic castration-resistant prostate cancer) cases have mutations in certain genes, including about 13% of prostate cancer patients with a BRCA2 mutation in their tumor. This study was momentous, providing the motivation to test PARP inhibitors such as rucaparib in prostate cancers.

The PCF Dream Team’s findings (73 researchers in all) ignited a race among pharmaceutical companies to develop precision medications for prostate cancer. The PCF Team quickly initiated a Phase II clinical trial of a similar drug called olaparib, which demonstrated anti-tumor activity in mCRPC patients with DDR (DNA Damage Repair) gene alterations.

Rucaparib is one of a class of drugs called PARP inhibitors and is already FDA-approved for the treatment of other cancers, including ovarian cancer. This is the first approval for a PARP inhibitor in the treatment of prostate cancer. It represents a groundbreaking advance in the care of patients with advanced disease, for whom solutions are urgently needed. One of the benefits of rucaparib is that it has fewer side effects than chemotherapy. This drug, a pill taken by mouth, is approved for patients whose prostate cancer has spread outside of the prostate and has developed resistance to hormone therapy and who also have specific genetic changes.

What is PARP and why is it such an important target for prostate cancer? Early data that PARP could be the key to finding treatments for prostate cancer came from a PCF-funded team led by Dr. Karen Knudsen of Thomas Jefferson University. Dr. Knudsen’s team proved that PARP is a driver of prostate cancer and that PARP inhibitors can suppress prostate tumor growth and progression. When DNA (your genetic code) is damaged and not repaired properly, a cell may become cancer. The genes called BRCA1 and BRCA2, which first became well-known for breast and ovarian cancer by Angelina Jolie’s public campaign, are also important in prostate cancer. Rucaparib exploits a lethal vulnerability in tumors with mutations in these genes, essentially eliminating prostate cancer’s ability to survive.

Today’s approval of rucaparib is based on results from a phase 2 clinical trial led by PCF Young Investigator Dr. Wassim Abida of Memorial Sloan Kettering Cancer Center, which was presented at the 2019 European Society of Medical Oncology (ESMO) Congress.

“This is a leap forward in helping men with mCRPC.” says PCF CEO Dr. Jonathan Simons. “PCF is proud to have funded the patient genomics research that catapulted rucaparib into global clinical trials. This kind of DNA-driven precision medicine is the future of treatment and brings us one step closer to our mission to eliminate all death and suffering from prostate cancer”

More information on this approval can be found here.