Manchester Researchers Offer New Hope for Children with Rare Genetic Disorder

Daily Health

25/11/2025

A new stem cell gene therapy created at the University of Manchester gives children who have Hunter syndrome - a rare, severe inherited disease - a chance for a longer, healthier life. Early findings from a clinical trial show clear benefit - one young patient, Ollie Chu, has already improved markedly after receiving the single treatment.

Key points

The first ever stem-cell gene therapy trial for Hunter syndrome is running in Manchester.
The treatment repairs the genetic fault that causes the disease letting the body make the enzyme it lacks.
First data show the therapy is safe, works and could transform life for affected children.

What Hunter syndrome is

Hunter syndrome also called mucopolysaccharidosis type II (MPS II), is an inherited disease in which a vital enzyme is missing. Without this enzyme, complex sugar molecules accumulate in organs and tissues. The buildup leads to stiff joints hearing loss, heart plus breathing problems, slowed development and loss of mental skills that resembles childhood dementia. Many patients live only 10 - 20 years.

The new therapy

Until now, the only option has been Elaprase, a weekly enzyme drip that costs a great deal and must continue for life - it barely slows the loss of mental function. The Manchester team has developed a single dose gene therapy that may cure the disease. Doctors remove the child's own blood forming stem cells, correct the faulty gene in the laboratory but also return the cells through a vein. The altered stem cells release large amounts of the missing enzyme and travel into the brain, a step earlier treatments could not achieve.

First results

Ollie Chu, a three-year-old boy from California, became the first child treated in the study at Royal Manchester Children's Hospital. Several months after the infusion he has recovered well and has advanced in many areas. His parents say he no longer needs weekly Elaprase. Blood tests now show very high levels of the enzyme that was previously almost absent, strong evidence that the therapy is active.

What specialists say

Professor Rob Wynn, Consultant Paediatric Haematologist at the hospital, states that gene therapy is safer as well as more effective than donor cell treatment because it uses the patient's own cells and removes the need for a donor. He adds that the same approach could help many other inherited diseases. Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease, shares this view saying the breakthrough could open the door to similar treatments and give hope to numerous families.

Team effort

The study brings together the University of Manchester, Manchester University NHS Foundation Trust, the University of Edinburgh besides Great Ormond Street Hospital. The University of Manchester or the charity LifeArc share the costs. Ollie is the first of five children with Hunter syndrome enrolled in the trial, which seeks to provide a more effective and possibly life saving option for this serious rare disease.

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