The new therapy, which will build on the preclinical work of Professor Gaspar and colleagues supported by the NIHR Great Ormond Street Hospital (GOSH) Biomedical Research Centre, could potentially cure patients of a rare but deadly inherited disease.
FHL-2 is caused by errors in the perforin gene which leads to failure of key immune cells known as T-cells to kill invaders - usually bacteria, fungi, or cancer cells. As a result, the disease is severely life-limiting if untreated, and the only existing therapy – bone marrow transplant – can cause significant complications especially if no suitable donor is available.
A number of genetic diseases resulting from mutations in single genes have been successfully treated in recent years using ex vivo gene therapy based on a lentiviral vector. Using a similar approach, Professor Gaspar and colleagues at GOSH and the UCL Great Ormond Street Institute for Child Health have demonstrated in a number of preclinical experiments that their approach has the potential to correct this devastating disease.
The funding provided by UCLTF is intended to support completion of the final preclinical steps (including biodistribution and toxicology studies) leading to a first-in-man, proof-of-concept clinical trial at GOSH with the potential to save the lives of children with FHL-2.