By Kate Marino
The U.S. Food and Drug Administration recently approved two gene therapies to treat sickle cell disease in people ages 12 and older a gigantic step toward curing this often-debilitating disease. Children's Hospital of Richmond at VCU and VCU Medical Center were involved in the clinical trial that led to the approval of these products.
India Sisler, M.D., is interim division chief and clinical director of the Division of Hematology and Oncology and medical director of the pediatric comprehensive sickle cell center at CHoR. She explains how the new therapy works and why its approval is monumental.
Using a process called peripheral blood stem cell collection, we isolate and pull out the patients stem cells. We then send those cells to a company that specializes in gene modification so they can change the way those cells produce hemoglobin. We work to knock the patients bone marrow out completely and give them their own stem cells back with the modified hemoglobin gene.
This is a multi-step process that involves meticulous planning and special care for the patient along the way.
While treatment is only currently available to this certain subset of patients, our hope is that it will become more widely available in the near future. Clinical trials in children under 12 are still in process.
As we see in many patients, as Walter got older he was having a lot of complications and pain, which made daily life difficult. What made him a great candidate was his motivation to improve his health in order to be the social, active young man he wanted to be. He was all-in and hes doing so well now. Every time we see him, hes almost in tears telling us the things hes able to do and the new lease on life he feels like he has.
At this point Walter is done with treatment and cured of sickle cell. He will follow up with our transplant team so we can monitor him for any side effects every couple months for now, then well space out to a year. We dont really know what to expect 20 years from now since this a new treatment, but its so promising and were thrilled to see Walter thriving.
Beth Krieger, M.D, a pediatric hematology and oncology specialist at CHoR, conducted advanced fellowship training with leading experts in the field of bone marrow transplant and cellular therapies at University of Minnesota, where she did a significant amount of transplant work for non-cancerous conditions. Her knowledge and expertise have been instrumental in Walters care during this study and in bringing curative therapy to CHoR.
Our sickle cell teams work hand-in-hand on a daily basis to help patients transition seamlessly from pediatric to adult care, so were accustomed to collaborating and know it is beneficial for everyone.
Two different sickle cell gene therapy products were actually FDA approved the one we trialed and another one and we are working to begin offering both of them here. Our hope is also that many more patients will qualify in the near future and that we can begin curing people of sickle cell disease without them having to experience the extreme pain and other side effects of vaso-occlusive crises.
Similar gene therapy has also been FDA approved for beta thalassemia, another red blood cell disease. Its much less common than sickle cell, but we have patients undergoing this clinical treatment and it has the potential to be equally life changing.
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Promising new sickle cell therapy trialed at Children's Hospital of Richmond at VCU and VCU Medical Center - VCU Health
