Understanding and Treating MDS
This article was originally published in the Spring 2011 issue of Columbia Medicine magazine.
An internationally known specialist in the blood disorder myelodysplastic syndrome, Azra Raza, MD, has been recruited to P&S and has launched the MDS Center to provide patients with the latest treatments and access to the newest clinical trials. Columbia’s MDS team cares for about 300 patients, making it the largest individual MDS program in the country.
Dr. Raza was professor of medicine and chief of hematology at the University of Massachusetts until she was recruited to St. Vincent Comprehensive Cancer Center.
MDS encompasses a group of bone marrow disorders marked by ineffective function of a myeloid stem cell, which is responsible for producing about 500 billion red cells, white cells, and platelets in the body each day. When the number and quality of the stem cells decline, production of blood cells becomes disorderly and ineffective and patients become severely anemic. In about one-third of patients, the disease transforms within months or a few years into acute myelogenous leukemia, one of the most prevalent and aggressive forms of leukemia.
Because MDS is relatively rare—only 10,000 to 15,000 new cases are diagnosed each year in the United States—most hematologists see only a handful of patients a year, not enough to conduct clinical trials or detailed molecular studies. The MDS Center at Columbia, recognized as a Center of Excellence by the MDS Foundation, sees patients who travel from all over the country to receive the latest therapies and to participate in research studies.
While conducting MDS research and treating patients for more than two decades, Dr. Raza developed a tissue repository containing more than 50,000 samples from MDS patients. “Our group is unique in our single-minded dedication to this field,” says Dr. Raza, professor of clinical medicine-oncology. “Since we began our work in the 1980s, we have built a national registry with a large enough patient population that enables us to conduct meaningful clinical trials. And we have vast experience in treating patients.”
Several cutting edge clinical trials are ongoing at the MDS Center, including those for patients with early-stage disease who are being treated with natural substances such as coenzyme Q10 and turmeric/ginger. Patients also travel to the center to participate in molecular and genetic studies of their bone marrow, which help shed light on their particular form of MDS and allow for individualized therapies.
Research studies at the MDS Center are always done with the goal of finding improved therapies, Dr. Raza says, and her work has already led to new treatments. Dr. Raza was the first to discover that MDS is caused by the premature death of bone marrow cells and that cell death is caused primarily by the inflammatory cytokine, TNF-alpha. Based on those findings, she pioneered the use of several anti-TNF drugs, including thalidomide. The success of thalidomide in a subset of MDS patients, as shown by her team, resulted in FDA approval of the drug Revlimid for MDS patients with certain chromosomal abnormalities.
“We use a very rational approach with evidence-based medicine to find ways to use existing drugs better and to identify new targets for drug development,” Dr. Raza says. “This is our mission and what I have dedicated my life to.”