Boy with rare condition amazes doctors after world-first gene therapy
20 hours ago
- #Rare Diseases
- #Gene Therapy
- #Medical Breakthrough
- Three-year-old Oliver Chu is the first person with Hunter syndrome (MPSII) to receive groundbreaking gene therapy.
- Hunter syndrome is a rare, inherited condition causing progressive damage to the body and brain, often fatal before age 20.
- The gene therapy involves modifying Oliver's stem cells to produce the missing enzyme, IDS, crucial for breaking down harmful sugar molecules.
- Oliver's modified cells were infused back into his body, aiming to halt the disease's progression in a one-off treatment.
- Nine months post-treatment, Oliver is thriving, producing the missing enzyme, and showing significant improvements in speech, mobility, and cognitive development.
- The trial, initially at risk due to funding issues, was saved by a £2.5m grant from British charity LifeArc.
- Five boys worldwide, including Oliver, are part of the trial, with hopes to expand the treatment to others, including Oliver's brother Skyler.
- The same gene therapy approach is being tested for other rare genetic disorders like Hurler syndrome and Sanfilippo syndrome.