Author Interviews, Cost of Health Care, Genetic Research, Hematology, JAMA / 22.03.2021 Interview with: Patrick DeMartino MD Pediatric Hematology and Oncology Fellow Doernbecher Children's Hospital Oregon Health & Science University What is the background for this study? Response: Dozens of gene therapies are expected to be on the market within a decade or so. Much has been written about the high prices of the therapies currently on the market (exceeding $1 million). However, only a small number of patients are eligible for these existing therapies each year. Gene therapy for sickle cell disease (SCD) appears promising and would potentially apply to a relatively large number of individuals in the U.S. We sought to explore potential affordability challenges associated with a gene therapy for SCD. (more…)
Author Interviews, Hematology, JAMA, Stem Cells, Transplantation / 03.07.2014

John F. Tisdale, Interview with: Dr. John Tisdale MD Molecular and Clinical Hematology Branch National Institute of Diabetes and Digestive and Kidney Diseases, National Heart, Lung, and Blood Institute, Bethesda, Maryland MedicalResearch: What are the main findings of the study? Dr. Tisdale: Using a nonmyeloablative allogeneic HLA-match peripheral blood stem cell transplantation strategy aimed at tolerance induction, we were able to revert the phenotype in 26 of 30 adult patients with severe sickle cell disease ranging in age from 16 to 65 years. In contrast to standard transplantation strategies which rely on high doses of chemo and/or radiotherapy after which the entire bone marrow and blood system is replaced by that of the donor, our patients had a mixture of their own and that of their donor. This procedure was well tolerated, with no non-relapse mortality, and led to complete replacement of red blood cells by that of the donor in successfully engrafted patients. This replacement resulted in decreases in pain, pain medication usage, hospitalizations, and improvements in organ function. (more…)