Using a Skin Cancer Drug to Improve Current Treatment in a Rare Blood Cancer, Myelofibrosis

Principal Investigator: Dr. Kristen Pettit

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Disease: Myelofibrosis

Research Description: Myelofibrosis (MF) is a rare, aggressive bone marrow cancer causing excessive scar tissue or fibrosis to form in the blood marrow, resulting in bone marrow failure, massively enlarged spleen, significant symptoms and impaired quality of life for patients. Ruxolitinib is the only approved drug available to treat MF, and this treatment improves symptoms for about half of patients. For those who do not respond or eventually develop resistance, there is no second-line treatment option. Dr. Pettit and her team believe that cobimetinib, a drug currently approved to treat melanoma, may help overcome this resistance and at the same time may improve fibrosis.  They plan to conduct a clinical trial combining cobimetinib with ruxolitinib for patients whose MF has progressed on ruxolitinib alone. This study will test the safety of using these two drugs in combination for MF, and will identify the optimal dose of cobimetinib to use in this combination.

Funding Partners: Goldman Philanthropic Partnerships

CWR funding role: Participating funder