A promising future for Sickle Cell patients
Researchers from WorldCare Consortium® medical second opinion provider, Dana-Farber/Boston Children’s Cancer and Blood Disorder Center have developed groundbreaking gene therapy treatment to cure sickle cell disease. This groundbreaking treatment comes after 70 years of research into finding a way to silence the gene BCL11A. This means that we may soon have a treatment that allows patients to avoid the lengthy hassle of finding a matching donor and be their own donor .
Restoring fetal hemoglobin is important in sickle cell patients because after birth most people stop producing hemoglobin, its production is blocked by BCL11A. It has been known for decades that patients with sickle cell disease that have the ability to produce hemoglobin have much milder cases, in most cases these patients transition into making a mutated form of sickled hemoglobin as they become adults.
How does gene therapy work in this case? Well, the best way to explain it is to look at the example of Manny Johnson of Boston, 21 who previously required monthly blood transfusions to keep his condition under control. Dr. Vijay Sankaran, MD, Ph.D. of Dana-Farber and his team isolated Johnsons’ blood stem cells and exposed them to the gene therapy vector, a lentivirus instructed to knock down the BCL11A gene. Importantly this affected only the precursors of red blood cells. The next step was for Johnson to undergo chemotherapy to prepare for the gene-modified cells, which were later returned to him through intravenous infusion. As a result, Many’s blood dramatically increased production of fetal hemoglobin; after six months his hemoglobin blood levels are where doctors hoped and appeared to be free of his sickle cell condition.
Researchers led by David A Williams, MD, president Dana-Farber/Boston Children’s Hospital went on to engineer an optimized gene therapy vector and presented their clinical trial data at the 60th Annual American Society of Hematology (ASH) meeting in San Diego, California on December 1, 2018. With clinical trials still to be conducted, it looks like there is a real promise for gene therapy to treat patients suffering from sickle cell.
If you or a loved one have been diagnosed with sickle cell and would like a medical second opinion, please contact us today.