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BRCA Mutations: What’s New in Clinical Trials?

BioMarin, a biopharmaceutical company, is launching a clinical trial to evaluate BMN 673, a potential first best-in-class PARP (poly ADP-ribose polymerase) inhibitor. This inhibitor is for patients with hereditary forms of breast cancer, specifically deleterious gBRCA mutation, in women with locally advanced and metastatic breast cancer. A PARP inhibitor is a targeted therapy, which interferes with a cancer cell’s ability to repair DNA and may lead to death of cancer cells. Because it is a “targeted” therapy, the drug goes after only the cancer-related cells rather than all cells.

The National Breast Cancer Coalition (NBCC), a nonprofit organization committed to the eradication of breast cancer, is a collaborating partner in the BMN 673 research. YSC, as a board member of NBCC, supports their efforts. NBCC’s partnership decisions on clinical trials follow rigorous criteria including significance of the research question, rigor of study design and ethical conduct of the trial among others. The BioMarin clinical trial proposed for BMN 673 meets those criteria. The BioMarin trial is unique because the team includes advocates who have received training through NBCC’s intensive science training program, Project LEAD®. Tracy Leduc and I attended the investigators’ meeting for this trial in Miami last week. The team truly cares about finding better, less toxic ways to fight breast cancer. The Phase 1 and 2 results of this trial show a high likelihood of success.

"BioMarin wants to make big differences in the lives of patients. We are honored to include a seat at the table for our colleagues from NBCC. Our efforts are best served by hearing their voice from the beginning and taking action together," said Hank Fuchs, Chief Medical Officer of BioMarin.

"We look forward to conducting a world-class study in collaboration with the finest clinical investigators in the world to evaluate the safety and efficacy of BMN 673 in the metastatic setting. Our advocates are poised to change the world through our actions. Ultimately, we hope to prevent women from getting breast cancer and prevent it from spreading outside the breast," Fran Visco, NBCC President added.

The BioMarin trial is underway and recruiting women ages 18 and older with a gBRCA mutation, who have experienced metastasis or a local recurrence after treatment for their initial diagnosis. For more information on the trial and how to enroll, please visit

About the National Breast Cancer Coalition (NBCC)
NBCC is dedicated to knowing how to end breast cancer by January 1, 2020 through the power of grassroots action and advocacy. NBCC supports increasing funding for innovative breast cancer research; monitors how those funds are spent; expands access to quality health care for all; and ensures that trained advocates influence all decision making in breast cancer. Join NBCC, learn more and take action. Visit

About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. For additional information, please visit

Joy Simha, diagnosed with Stage 2B breast cancer in 1994, is one of the three Co-Founders of YSC.  She is a former YSC Board Member and has represented YSC on the Board of Directors of the National Breast Cancer Coalition (NBCC). Joy graduated NBCC's Project LEAD®  in 1998 and has served as an educated consumer advocate as a member of the Integration Panel of the Department of Defense Breast Cancer Research Program since 2009.

Most recently Joy was co-chair of YSC’s 2013 Research Think Tank in order to insure researchers and educated advocates worked together to define the research priorities that would make the greatest impact in the quantity and quality of life for young women affected by breast cancer.  She currently lives and works as a writer and consultant out of her home in New Jersey.