Researchers in the United Kingdom have developed a stem cell gene therapy that may be able to treat a fatal childhood disease. Sanfilippo disease, also known as mucopolysaccharidosis III, is a rare genetic condition that affects about 1 in 70,000 babies.
The new stem cell gene therapy, developed by researchers from the University of Manchester and Central Manchester University Hospital NHS Foundation Trust (CMFT), is about to enter human trials.
There are four forms of Sanfilippo disease. The new treatment is targeted at “type A” of the disease, which affects about 100 children in the UK (1 in 89,000 births). Type A Sanfilippo disease is caused by a deficiency of the SGSH enzyme, which breaks down long chain sugars. Without enough of the enzyme, sugars build up in the body and brain.
Symptoms of the disease include behavioural problems, slow development, hyperactivity and later on — dementia-like symptoms. Typically, children will only achieve a mental age of 2 and pass away before the age of 18.
UK-based biotechnology company Orchard Therapeutics will be involved in the human trial which is set to begin later this year. The company is very experienced with stem cell gene therapy and is excited to proceed with the project.
If researchers can address single gene diseases like Sanfilippo with stem cell gene therapy, it opens up the way to treat many other conditions including neuro-metabolic disorders.
The stem cell gene therapy works by correcting problems with the patient’s stem cells. Modified stem cells are transplanted into the patient’s bone marrow, where they begin to produce cells that do not carry the genetic defect. The new stem cells go on to produce the missing enzyme that the body needs to correctly process long-chain sugars.
Life Sciences Minister, George Freeman MP says of the research: “This new gene therapy has the potential to change the lives of children with this dreadful condition, and will undoubtedly help to cement the UK’s position as a world-leader in medical research.”
Source: Stem cell gene therapy for fatal childhood disease ready for human trial
{{cta(‘010124f3-c9bc-4a23-b9fc-74953e6288c9’)}}