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STEM CELL GENE THERAPY FOR FATAL CHILDHOOD DISEASE READY FOR HUMAN TRIAL Scientists in Manchester, who have developed a stem cell gene therapy to reverse a fatal childhood illness, have agreed to work with a new therapeutics company to test it in a human trial. University and Central Manchester University Hospital NHS Foundation Trust (CMFT) researchers have developed the pioneering approach for Sanfilippo disease (also known as mucopolysaccharidosis type III or MPS III) – a genetic condition for which there is currently no effective treatment. The most common of the four types of Sanfilippo (type A) affects around 100 children in the UK, or one in 89,000 births, and it is this type that is targeted by the new treatment. Sanfilippo syndrome is caused by a lack of the SGSH enzyme, which helps to break down and recycle long chain sugars. This results in a build-up of sugars in the body, particularly in the brain. Children with Sanfilippo begin showing symptoms of hyperactivity, severe behavioural problems and miss developmental milestones as toddlers. As they get older they show symptoms similar to dementia, and most never achieve a mental age beyond two years. Later they will experience seizures and difficulties in walking and swallowing. It is invariably fatal, with most children dying around the age of 18 years. Following a licence agreement with Orchard Therapeutics, a new UK-based clinical-stage biotechnology company, the gene therapy developed in Manchester will be trialled in humans. The University's technology transfer company, UMI3, negotiated the terms of the major deal with Orchard Therapeutics. Dr Brian Bigger, who leads the Stem Cell and Neurotherapies Laboratory at the University and developed the technique in partnership with the Trust scientists said: "This license agreement with Orchard will allow us to take the technique we have developed to the next and crucial stage of trials in humans. We are hopeful that this treatment may help to treat the early onset dementia in these patients and saving children's lives. UMIP HIGHLIGHTS