Revolutionary gene therapy offers hope for Huntington’s Disease patients
Doctors have made a groundbreaking advancement in treating Huntington’s disease with a pioneering gene therapy that could slow the disease progression by 75%.
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For the first time, doctors have successfully treated Huntington’s disease, one of the most devastating genetic conditions known to medicine.
Often described as a cruel combination of dementia, Parkinson’s, and motor neurone disease, Huntington’s gradually destroys brain cells, robs people of their independence, and ultimately proves fatal.
Now, a pioneering gene therapy has offered patients a future once thought impossible.
The trial, which involved 29 patients in Japan and Europe, showed that the new treatment slowed the progression of the disease by an average of 75%.
In practical terms, the decline expected in just one year would instead take four. Researchers believe this could translate into decades of better quality life for those affected.
The treatment
The treatment involves highly complex neurosurgery lasting up to 18 hours.
During the procedure, a harmless virus engineered with a tailored DNA sequence is infused deep into two areas of the brain, the caudate nucleus and the putamen - guided by real-time MRI scans.
Once delivered, the virus acts as a carrier, turning neurons into miniature factories that produce microRNA.
This genetic material disables the messenger RNA responsible for creating the toxic mutant huntingtin protein, which normally destroys brain cells.
Treatment results
The therapy, developed by the company uniQure, has not yet been published in full but the preliminary data is compelling.
Patients not only showed a dramatic slowing of symptoms affecting movement, memory and daily function, but also biochemical evidence that brain cells were being preserved.
Levels of neurofilaments in spinal fluid – a marker of brain cell death – actually dropped, despite normally rising as the disease progresses.
Researchers at University College London, who helped lead the study, said the results exceeded all expectations. They reported that some patients who had been medically retired were able to return to work, while others expected to need wheelchairs remained mobile.
Although some participants experienced headaches and confusion due to inflammation from the viral vector, these were manageable and resolved with treatment.
What causes Huntington’s disease?
Huntington’s disease is caused by a mutation in the huntingtin gene. If one parent carries the faulty gene, each child has a 50% chance of inheriting it.
Symptoms typically emerge in a person’s 30s or 40s, and most patients do not survive more than two decades after diagnosis. Until now, treatment options have only managed symptoms, offering no real hope of altering the course of the disease.
Experts believe that, if given before symptoms appear, this new therapy could even prevent the disease from developing at all.
While the treatment is expected to be very costly, neurologists say it represents the most significant step forward in Huntington’s research to date.
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