Thalidomide as a Initial Therapy for Early-Stage Myeloma

Dr. Rajkumar, Drug, Early Stage Investigator, Mayo Clinic, Oncology, Rare Disease, Success Stories

Principal Investigator: Dr. Vincent Rajkumar

Disease: Multiple Myeloma

Research Description: In 2002, Cures Within Reach funded Dr. Vincent Rajkumar at Mayo Clinic to test thalidomide, used in leprosy, as a treatment for the rare blood cancer multiple myeloma after his earlier studies demonstrated that it helped prevent the growth of new blood vessels, a key mechanism in cancer cell growth. ​In 2003, Dr. Rajkumar published the results of this Phase II clinical trial, showing that thalidomide had significant potential as part of initial therapy for asymptomatic early-stage multiple myeloma to delay progression to symptomatic disease. Roughly 33% of trial participants had a partial response to therapy (at least a 50% reduction of a multiple myeloma-linked protein) and another 33% had a minor response (between 25-49% reduction). In addition, this was accompanied by improvements in bone marrow plasma cells. Within 2 years of Dr. Rajkumar’s publication, physicians began prescribing thalidomide earlier in the course of multiple myeloma treatment.

Completed Research

Mayo Clinic

Success Story

Read more about this success here.