Clinical Trials to Begin for Possible Sickle Cell Cure
Punam Malik, MD, director of Molecular and Gene Therapy Program and associate director of the Comprehensive Sickle Cell Program at Cincinnati Children’s.
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About one in every 500 African-Americans are born with sickle cell anemia. In the United States, sickle cell affects 70,000 to 100,000 people. Worldwide, sickle cell affects millions. People with sickle-cell disease live with years of pain and fatigue. They also face greater risks of stroke, organ failure, bone loss and blindness. Sickle cell treatments include bone marrow transplants, but matched donors are only available to 15% of patients and these therapies often come with immune side effects.
After about a decade of groundwork, scientists led by Punam Malik, MD, director of Molecular and Gene Therapy Program and associate director of the Comprehensive Sickle Cell Program at Cincinnati Children’s Hospital Medical Center, have developed a gene therapy approach that appears to prevent sickling deformation of red blood cells that characterize sickle cell disease. The gene therapy, derived from a patient’s own stem cells, would be delivered back to the patient via a virus engineered so that it can deliver the genes but not cause disease.
Testing so far has involved sickle cell disease mice and lab tests involving human tissue. If federal regulators approve, Dr. Malik plans to recruit 10 adults with sickle cell for the first phase of clinical testing, which could begin as early as fall of next year.
The plan is to begin early-stage clinical trial next year using this genetic therapy approach at Cincinnati Children’s Hospital. As with any early phase trial, to establish safety and feasibility of the gene therapy approach is the first goal or endpoint. If the results of the gene transfer trial show that this approach is feasible and safe, then the next phase of clinical testing would be aimed at efficacy or ‘curing’ the disease. Fully testing the new gene therapy could take another five years.
