According to a report by BioSpace, a new treatment for SCID could be around the corner. Mustang Bio, Inc. (Mustang), and St. Jude Children’s Research Hospital recently announced a partnership towards the development of a first-in-class therapy for SCID. The new treatment is a form of gene therapy using the patient’s own cells and is currently being evaluated in trials. Keep reading to learn more or follow the original story here for further information.
What is SCID?
SCID stands for severe combined immunodeficiency. SCID is a primary immunodeficiency condition characterized by little or no immune response. As a result, a person experiences frequent infections from bacteria, fungi, or viruses. As a result of some patients requiring sterile conditions SCID is sometimes referred to colloquially as bubble boy disease.
Symptoms of SCID most commonly include frequent and recurring viral infections. Pneumocytosis pneumonia is a common occuring infection and indicator in children with SCID. Other symptoms include poor growth, eczema-like rashes, and chronic diarrhea.
Investigating Gene Therapy
Together with St. Jude, Mustang is evaluating a way to treat SCID using a patient’s own cells. The therapy uses a low dose of busulfan and modified blood-cells from the person with SCID. A lentiviral delivery system has been designed to carry the modified cells back into the patient’s body.
The collaborative therapy is currently being tested in a series of Phase 1/2 trials. Researchers conducting the trials are observing the treatment and its effects with infants under the age of two at centers across the country. A second Phase 1/2 trial at the National Institutes of Health is examining the way the treatment affects those over the age of two.
Thus far, eight patients in the under two group have been successfully treated with the new therapy. Six additional patients also showed a reconstituted immune system within three to four months of receiving treatment. Furthermore, researchers describe the therapy as being well tolerated. It does not appear as of yet to have any unexpected or major side effects.
One of the major differences between this new therapy and the ones that came before it involves the use of immunoglobulin. Some previous therapies have shown signs of success but were unable to encourage the reconstitution of B cells. This meant patients required intravenous infusions for the rest of their lives even with treatment. Mustang’s new treatment appears to start a recovery process of the entire immune system. This could free patients from the need for immunoglobulin therapies.
Mustang’s CEO and president, Manuel Litchman, MD, says his company continue to look forward to working with St. Jude. It his hope they will be able to proceed with clinical trials, and create something unique and long-term for patients of SCID.