The Bill & Melinda Gates Foundation has donated $1.5 million to Boston Children’s Hospital to develop a gene therapy for sickle cell disease, with the goal of making the drug technology more widely available in regions of the world with high rates of the condition.
Boston’s Children Hospital has received a $1.5m donation to develop a gene therapy for SCD, which will make the technology more readily available in Africa. The donation is from the Bill & Melinda Gates Foundation, which has also made a lot of grants for research into the eradication of malaria. Mosquito-borne Malaria is one of the biggest threats to sickle cell health in developing countries.
In gene therapy, blood stem cells are removed from the body, modified to replace or add new genes and re-infused into the patient.
The Gates Foundation and Boston Children’s Hospital are aiming to develop treatments that could be infused directly into patients.
Project leader Dr. David A. Williams, chief scientific officer and senior vice president of Boston Children’s Hospital and President, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center has this to say:
‘We will look at new technologies for introducing the therapeutic gene into stem cells that could help standardize gene therapies and make them more available and affordable.
‘While gene therapies are currently confined to a few research hospitals in the U.S. and other developed countries, our long-term goal is to make this treatment available to patients in developing countries.’
The Gates-Boston Children’s initiative builds on an ongoing clinical trial by Dana-Farber/Boston Children’s, in which researchers are testing a gene therapy designed to suppress a certain sickle gene and allow sickle cell patients to make normal red blood cells.