A groundbreaking new drug is offering hope for Huntington’s disease: an inherited neurodegenerative disease that currently has no cure.
While the average person’s DNA produces healthy huntingtin proteins that nourish brain development, Huntington’s disease is caused by a faulty gene that makes toxic proteins instead. These deadly proteins then poison the nervous system and cause loss of motor control, memory, and speech.
While there are therapies that exist to help Huntington’s patient cope with the degenerative symptoms, patients typically die about 10 years after diagnosis.
In a new study released by the University College London, however, 46 patients with Huntington’s Disease were enrolled in the first human trial for an experimental drug called IONIS-HTTRx.
The drug, which was administered through the patients’ spinal fluid, was not only shown to be safe and well-tolerated, but it also successfully lowered the level of harmful huntingtin protein in the brain’s nervous system.
Professor Tabrizi, Director of the UCL Huntington’s Disease Centre said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families. For the first time, a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated.
“The key now is to move quickly to a larger trial to test whether the drug slows disease progression,” she added.
Moving forward, the research team will conduct further testing on how drastically the drug can alter the progression of the disease. While they are not yet calling this drug a cure, they say that it is the most groundbreaking development in the disease’s treatment in over 50 years.
Patients who have already received the drug will be able to continue taking it as a treatment for their disease in the coming years.
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