Pharmaceutical Business review

Breakthrough in treatment for MDS patients

The study, from the H Lee Moffitt Cancer Center & Research Institute and sponsored by Celgene, evaluated response in 148 patients. The data published found that more than 75% of patients reduced their need for transfusions and two-thirds were completely freed from the need for transfusions.

Most significantly, in 45 percent of patients, there was no detectable trace of the cancer. After two years of follow-up, patients continue to respond and do well on treatment, representing a breakthrough in treatment for MDS patients.

“These new, landmark data demonstrate that Revlimid in some cases, eliminates all signs of the cancer’s genetic cause, an abnormality on the chromosome 5, can reduce or even eliminate the need for transfusions in many patients with MDS, and after two years these responses have continued to hold,” said Dr Alan List, professor of oncology and medicine at Moffitt and lead investigator of the study. “It is very rewarding to see patients treated with Revlimid, living three or four years transfusion free and living with a better quality of life.”

MDS, a cancer in which the bone marrow fails to make enough functioning blood cells, affects 300,000 people worldwide, killing 60,000 to 70,000 a year. MDS patients suffer from anemia and fatigue and need whole-body blood transfusions as much as twice a month. Repeated transfusions can lead to a toxic buildup called “iron overload” that severely damages the heart, liver and pancreas, and patients eventually succumb to the disease.