The podcast episode titled “Stem Cell Gene Therapy Clinical Trial to eliminate Sickle Cell Disease” from Regen Med Global features Dr. Donald Kohn, a UCLA Professor for the Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics; Molecular & Medical Pharmacology.
Dr. Kohn begins by sharing his background, including his education and his interest in research and science. He explains that he has been working on gene therapy for about 40 years, with a focus on developing techniques to treat human blood diseases.
He then provides an overview of sickle cell disease, a genetic disease caused by a specific mutation in one of the genes that makes up the hemoglobin molecule. Patients with sickle cell disease experience progressive organ problems due to repeated sickle crises, which can be very disruptive to their lives.
Dr. Kohn discusses the clinical trial, which aims to increase fetal hemoglobin to prevent sickling. The trial involves taking bone marrow stem cells from the patient, adding a copy of the beta globin gene that’s been modified in the laboratory to be like fetal globin and prevent sickling, and then transplanting the cells back into the patient.
The procedure involves collecting stem cells from the patient’s bloodstream, adding the virus carrying the anti-sickling gene to the cells, and then freezing the cells. After a series of tests, the cells are given back to the patient following chemotherapy. The cells then start making red cells that don’t sickle.
Dr. Kohn shares that the first patient was treated in 2015, but the results were not successful. However, after modifying the trial, they treated a second patient in 2020 who has done quite well. The patient, a man in his mid-20s, has not had any sickle crises since the treatment and has a good number of cells in his blood and bone marrow with the gene they put in.
The treatment is intended to be a one-time procedure, with the hope of providing a lifetime of improved health. Ideal candidates for the trial are young adults who have had a lot of problems from sickle cell disease but still have good heart, lung, kidney, and liver function.
Dr. Kohn also discusses the risks and concerns associated with the trial, including the potential for infertility due to the high-dose chemotherapy, the risk from the chemotherapy itself, and the use of a virus derived from the HIV/AIDS virus to put the gene into the cells.
The podcast concludes with Dr. Kohn expressing his excitement about the progress in gene therapy and its potential to treat a variety of diseases. He acknowledges the economic and financial challenges of making these treatments available but remains hopeful that these can be overcome.
Watch full episode below: