Health and Me

Ishita Roy

Three-Year-Old Boy With Rare Hunter Syndrome Makes History As Breakthrough Gene Therapy Halts His Disorder

A three-year-old boy from California has stunned doctors with his rapid progress after receiving a world-first <span>gene therapy</span> for <span>Hunter syndrome</span>, a rare inherited condition that causes progressive damage to the body and brain. The treatment was carried out at the Royal Manchester Children’s Hospital and offers new hope for families who have long lived with the devastating effects of the disease.

<h2><span rel="noindex">What Is Hunter Syndrome</span>?</h2>

Hunter syndrome is caused by a faulty gene that prevents patients from producing an enzyme needed to clear certain molecules from cells. Children appear healthy at birth but start showing symptoms around age two. The condition affects the heart, liver, bones and brain and is often described as a form of childhood dementia. Most severely affected patients rarely live beyond their teenage years.

<h2>How Doctors Tried to Halt the Disease</h2>

Before treatment, Oliver Chu could not produce the crucial enzyme, reports BBC. Conventional therapy required weekly infusions that only slowed physical decline and could not protect the brain. Doctors in Manchester decided to attempt a one-time gene therapy, something never tried before for this condition.

Stem cells were collected from Oliver’s blood in December 2024 and sent to a specialist laboratory at Great Ormond Street Hospital in London. Scientists inserted a working copy of the missing gene into a harmless virus. The virus delivered the corrected gene into Oliver’s stem cells, allowing them to produce the missing enzyme once infused back into his body. Researchers also modified the gene to help the enzyme travel more easily into the brain, a major challenge in previous treatments.

In February 2025, Oliver returned to Manchester for the infusion. His corrected stem cells, about 125 million of them, arrived frozen in a cryopreservation tank. After several safety checks, nurses slowly injected two doses into a catheter in his chest. The entire process took minutes, but the hope behind it was enormous. Once the infusion was complete, Oliver and his mother returned to California to wait for results.

By May, the effects were becoming clear. Oliver’s speech had improved, his mobility had increased, and his parents described him as “brighter” and “healthier.” Most significantly, he no longer needed the expensive weekly enzyme infusions. His body had begun producing the enzyme on its own.

His older brother Skyler, who also has Hunter syndrome, accompanied the family to Manchester during this follow-up visit. Their parents now hope Skyler may also qualify for the therapy one day.

By late August, nine months after treatment, doctors confirmed that Oliver was producing far above normal levels of the missing enzyme. He was gaining new words, moving more easily and showing continued cognitive progress. Professor Simon Jones, who co-leads the trial, praised Oliver’s development but noted that researchers still need long-term data.

Five boys from the United States, Europe and Australia are enrolled in the ongoing trial. No UK patients qualified because most were diagnosed too late. Participants will be monitored for at least two years to assess effectiveness and safety.

The gene therapy almost never reached patients. Researchers at the University of Manchester had spent over 15 years developing it but nearly lost funding when their biotech partner withdrew. A last-minute contribution of 2.5 million pounds from the medical charity LifeArc saved the trial and allowed children like Oliver to join.

His parents say they are “eternally grateful” and feel his life has been “reset.” For them, the treatment represents not just medical progress but a future filled with possibilities.

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A three-year-old boy from California has stunned doctors with his rapid progress after receiving a world-first gene therapy for Hunter syndrome, a rare inherited condition that causes progressive damage to the body and brain. The treatment was carried out at the Royal Manchester Children’s Hospital and offers new hope for families who have long lived with the devastating effects of the disease.What Is Hunter Syndrome?Hunter syndrome is caused by a faulty gene that prevents patients from producing an enzyme needed to clear certain molecules from cells. Children appear healthy at birth but start showing symptoms around age two. The condition affects the heart, liver, bones and brain and is often described as a form of childhood dementia. Most severely affected patients rarely live beyond their teenage years.How Doctors Tried to Halt the DiseaseBefore treatment, Oliver Chu could not produce the crucial enzyme, reports BBC. Conventional therapy required weekly infusions that only slowed physical decline and could not protect the brain. Doctors in Manchester decided to attempt a one-time gene therapy, something never tried before for this condition.Stem cells were collected from Oliver’s blood in December 2024 and sent to a specialist laboratory at Great Ormond Street Hospital in London. Scientists inserted a working copy of the missing gene into a harmless virus. The virus delivered the corrected gene into Oliver’s stem cells, allowing them to produce the missing enzyme once infused back into his body. Researchers also modified the gene to help the enzyme travel more easily into the brain, a major challenge in previous treatments.The First InfusionIn February 2025, Oliver returned to Manchester for the infusion. His corrected stem cells, about 125 million of them, arrived frozen in a cryopreservation tank. After several safety checks, nurses slowly injected two doses into a catheter in his chest. The entire process took minutes, but the hope behind it was enormous. Once the infusion was complete, Oliver and his mother returned to California to wait for results.Signs of ImprovementBy May, the effects were becoming clear. Oliver’s speech had improved, his mobility had increased, and his parents described him as “brighter” and “healthier.” Most significantly, he no longer needed the expensive weekly enzyme infusions. His body had begun producing the enzyme on its own.His older brother Skyler, who also has Hunter syndrome, accompanied the family to Manchester during this follow-up visit. Their parents now hope Skyler may also qualify for the therapy one day.A Year That Changed EverythingBy late August, nine months after treatment, doctors confirmed that Oliver was producing far above normal levels of the missing enzyme. He was gaining new words, moving more easily and showing continued cognitive progress. Professor Simon Jones, who co-leads the trial, praised Oliver’s development but noted that researchers still need long-term data.Five boys from the United States, Europe and Australia are enrolled in the ongoing trial. No UK patients qualified because most were diagnosed too late. Participants will be monitored for at least two years to assess effectiveness and safety.A Trial That Nearly CollapsedThe gene therapy almost never reached patients. Researchers at the University of Manchester had spent over 15 years developing it but nearly lost funding when their biotech partner withdrew. A last-minute contribution of 2.5 million pounds from the medical charity LifeArc saved the trial and allowed children like Oliver to join.His parents say they are “eternally grateful” and feel his life has been “reset.” For them, the treatment represents not just medical progress but a future filled with possibilities.