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

read the published articles in Nature and Haematologica describing the clinical trial results

Success Story

Read more about this success here.