AUB Thalassemia Expert Helps Drug Win FDA Approval

by MainGate Staff
Fall 2021

Dr. Ali Taher, professor of medicine at AUBMC’s Division of Hematology and Oncology.

In clinics across Lebanon and around the world, there is a certain kind of patient whose visible signs of suffering—pale, yellowish skin, distorted facial bone structure, bulging abdomen, shortness of breath, swollen legs—are the mark of a devastating and potentially fatal genetic disease called beta thalassemia. It is caused by a congenital defect in the globin protein, a component of hemoglobin, which lets our red blood cells carry oxygen. People who inherit two copies of the faulty gene experience a breakdown in their red blood cells, leading to severe anemia.

It is one of the most common genetic diseases in the world, particularly in the Mediterranean region, and thus, a major focus for industry-backed medical research. One of the world’s leading scientists in the field of thalassemia research is Dr. Ali Taher, professor of medicine at AUBMC’s Division of Hematology and Oncology. He is among a cadre of physician-scholars regularly called upon to lead and colead groundbreaking research into new treatment and management protocols aimed at improving beta thalassemia patient health and quality of life.

Recently, Taher was a coinvestigator for an international clinical trial, called BELIEVE, that evaluated the effect of luspatercept, marketed in the United States as Reblozyl, on reducing the need for blood transfusions in transfusion-dependent beta thalassemia patients. The results of the study, conducted by Bristol Myers Squibb and published in the New England Journal of Medicine, showed that luspatercept significantly reduced the demand for blood transfusions compared with a placebo.

Based on data from the BELIEVE trial, in November 2019 the US Food and Drug Administration (FDA) approved luspatercept for the treatment of adult patients with beta thalassemia who require regular red blood cell transfusions. The European Medicines Agency (EMA) followed suit in June 2020.

Lowering the transfusion demand is a boon for both patients and the healthcare system. “Blood transfusions can spread infections and lead to accumulation of excess iron in the body, which can lead to increased clinical complications and death if not treated. They also require patients to come to the hospital on a monthly basis indefinitely since diagnosis, which can considerably affect their quality of life. Securing transfusions on such a regular basis also causes a logistic and economic burden on the healthcare system,” explained Taher. The scientific community was looking to find a replacement for transfusions to address these challenges.

Currently, Taher is helping to shepherd the results of a sister international clinical trial (BEYOND) that he led as principal investigator. That trial showed luspatercept’s efficacy in improving hemoglobin levels in non-transfusion dependent beta thalassemia patients. “In this special transfusion-independent population of beta thalassemia patients, low hemoglobin levels can lead to serious, often irreversible complications and poor quality of life.” Taher recently presented data from the trial—which is currently being used to file for regulatory approvals in the US and Europe—at the Presidential Symposium of the European Hematology Association (EHA) Congress.