Hunter Syndrome (MPSII) is a rare disease but the sufferer will slowly die before the age of 20. It occurs once in every 100,000 male births and causes damage to the brain and body. The damage to the gene causes the body to produce the enzyme iduronate-2-sulfatase (IDS) which is needed to keep the body's cells healthy.
Researchers at the University of Manchester have announced that a child with Hunter Syndrome has been successfully treated using a gene therapy technique. Oliver Chu, a child from the US, is the first recipient of the treatment and is one of the first five to be involved in a trial of a treatment for Hunter Syndrome that is being developed.
Oliver Chu and father – University of Manchester
Stem cells are taken from Oliver's blood and then the gene containing IDS is inserted into a viral shell. This is then re-introduced into the patient's body. The virus enters the stem cells and when it reaches the bone marrow it starts producing new white blood cells and each of them is expected to start producing the IDS enzyme.
After 9 months of treatment, Oliver showed positive progress and was able to speak and interact with children of his age. Although early signs show that this treatment is successful, the study will continue for two years to see its effectiveness. In addition to Oliver, four other children from America, Europe and Australia participated in this trial.
This gene therapy treatment can only prevent damage from occurring but cannot treat damage that has already occurred. Early detection before the age of two is required for this treatment to avoid permanent damage to the patient's body.
Among the symptoms of Hunter Syndrome are changes in physical characteristics, stiff limbs and a limp body. It can cause damage throughout the body, including the heart, liver, bones and joints and in the most serious cases can cause severe mental disorders and progressive neurological decline.
In addition to treating Hunter Syndrome, gene therapy techniques have also successfully treated Huntington's disease this year and sickle cell anemia in 2023.
