Gene Therapies Have the Potential to Cure Sickle Cell Disease But Medical Experts Still Have Concerns About Potential Risks
Sickle cell disease or any chronic illness for that matter can take a major toll, not only on a patient’s physical health, but on their mental wellness, too. But it doesn’t stop there because it also impacts families and significant others, who only want to see their loved one healthy and happy.
Many health professionals are working to cure sickle cell disease (SCD) in the future and they’re making significant progress on that front. Treatments for the hereditary blood disorder are expanding and one of the most remarkable and promising developments is the advancement in gene therapy.
Sickle Cell Foundation of Georgia CEO, Tabatha McGee, also the host of the foundation’s new TV series “Sickle Cell: Beyond the Pain,” focused on gene therapy in a recent show and learned more about it from two noted experts.
Dr. James Eckman is the founder and medical director of the Sickle Cell Center at Grady Hospital where he’s served for 35 years, and his colleague Dr. Morgan McLemore works in hematology and oncology at Emory University School of Medicine. He’s board-certified in hematology and internal medicine.
Gene therapy is “a really exciting new development,” Eckman told McGee.
“They’ve been working on molecular genetics of hemoglobin for years. It actually was a prototype for the science we developed to understand genetics and the NIH made a commitment that it will be one of the first diseases to benefit from that knowledge,” he explained.
“Right now, we have at least two or three different approaches that are FDA-approved to cure sickle cell disease with gene therapy. So, it’s a really exciting new frontier. The short-term results are excellent,” the doctor added.
But McLemore urged caution when it comes to gene therapies.
“These therapies while are very exciting and short-term results are really great. The approval caught a lot of people off guard. We’re not really scaled-up to do this at the present time,” he explained.
“There are only a few centers that offering it currently. We’re in the process through Emory University of offering it, but we’re probably a year away.”
McLemore explained that because these therapies are commercial products there are regulations that have to be put into place first before the university can offer any them.
“And there are some concerns about long term complications from this. Albeit it’s a small percentage. There are increased risks associated with it and we have to kind of gauge carefully who we offer this to,” McLemore said.
“While I am excited for this and it will benefit a substantial number of people, it will probably not be something available to all currently,” he added.
But genetic research has transformed the understanding and treatment of sickle cell disease and this progress offers hope more than ever before that as the research continues to advance, the prospect of a definitive cure for SCD becomes increasingly more likely.
-By Shelby Lin Erdman