Principle Investigator: Dr. S. Vincent Rajkumar

Research Institution: Mayo Clinic

Disease: Multiple Myeloma

Research Description: Multiple myeloma is a blood cancer that originates in the bone marrow, resulting in bone damage, impaired kidney function and ultimately death. For many years, treatment options for these patients were limited and the prognosis was poor, with most patients succumbing in 2-3 years from diagnosis. Attention had turned to the drug thalidomide because it appeared to target a pathway known to keep myeloma cells alive. Thalidomide had been withdrawn from the market because of serious side effects discovered when it was first introduced as a treatment for morning sickness, but had subsequently been approved to treat leprosy. With support from Cures Within Reach funders, Dr. Rajkumar repurposed thalidomide in a human clinical trial for the earliest stages of multiple myeloma. Within the first 24 months of publication of Dr. Rajkumar’s research, clinicians began to prescribe thalidomide off-label for their multiple myeloma patients, and within four years, the FDA approved thalidomide, in combination with the steroid dexamethasone, for multiple myeloma. The pharmaceutical company Celgene then developed the next generation version of thalidomide, leading to a better, more effective treatment with fewer side effects. To date, over 500,000 patients worldwide have used thalidomide for multiple myeloma and other blood cancers, increasing their length and quality of life.

CWR funding role: Primary funder

Publication: Leukemia;  Mayo Clinic Proceedings

Since publication:

→ Data leveraged into larger philanthropic or government funding

→ Repurposed treatment awarded FDA approval for multiple myeloma

→ Commercial development and FDA approval of future generation versions of thalidomide

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