Incredible news out of the UK with the announcement that the first Sanfilippo patient has been treated in a clinical trial with a gene modified bone marrow stem cell transplant.
The Sanfilippo Type A patient was dosed with Orchard Therapeutics' OTL-201 gene therapy in a proof-of-concept clinical trial. The trial will enrol 5 children with Sanfilippo Type A under 24 months of age who will be followed for a minimum of three years.
This trial involves taking the patient's own stem cells from the bone marrow, using a virus to deliver a healthy copy of the faulty gene into these cells, and then transplanting them back into the body.
In May 2019 it was announced that a two-year-old boy with Sanfilippo Type A received this experimental therapy in Manchester, under what is called a “Specials” licence, but this recent news is of the first treatment within the formal clinical trial.
This news brings great hope to families, at a time when many Sanfilippo research projects and clinical trials are being delayed by the COVID-19 crisis.