Life&Culture

First trial of Huntington's disease drug a success

First trial of Huntington's disease drug a success

Prof Sarah Tabrizi, executive of University College London's Huntington's Disease Center who drove the stage 1 trial, said the outcomes were "past what I'd ever trusted ..."

Sarah Tabrizi, the lead researcher and director of the Huntington's Disease Centre at UCL, said: "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die".

Meanwhile, the BBC is reporting that the defect that causes the neurodegenerative disease Huntington's has been corrected in patients for the first time.

Huntington's symptoms typically manifest in middle age and include involuntary movements, changes in personality, mood swings and dementia-like cognitive problems. Some people die within a decade of diagnosis.

"A large portion of our patients realize what's in their future", said Ed Wild, a UCL researcher and specialist neurologist at the National Hospital for Neurology and Neurosurgery in London, who controlled the medication in the trial.

The mutant Huntington's gene has instructions for cells to create the toxic protein called huntingtin. By doing this, the drug destroys the messenger molecule before the damaged proteins form.

The trials began in late 2015, when 46 patients with early Huntington's disease started their treatment with the experimental drug.

After being given the drug, the concentration of harmful protein in the spinal cord fluid dropped significantly and in proportion with the strength of the dose. This is coupled with the fact that the drug had no adverse effects and was seemingly safe.

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An experimental drug lowered levels of toxic proteins in the brains of people with Huntington's disease, researchers report.

Huntington's disease progressively robs you of your mental and physical faculties.

The trial was too small, and not long enough, to show whether patients' clinical symptoms improved, but Roche is now expected to launch a major trial aimed at testing this. It has been described as Parkinson's and Alzheimer's rolled into one - parts of the patient's brain die as it progresses, leading to incapacitation and eventually death. "One day we need to keep the disease".

The surprising achievement raises the tempting probability that a comparative approach may work for other degenerative cerebrum issue. Or, you can leave the gene alone, and alter the way the body codes for the misshapen protein.

As hoped, IONIS-HTTRx produced significant, dose-dependent lowering of the level of mutant huntingtin - the first time the protein known to cause Huntington's has been lowered in the nervous system of patients. Konopaske, who was not associated with the study, has been closely following the research around the use of drugs that suppress the production of mutated huntingtin in mice.

"Obviously, there will be much enthusiasm into whether it can be connected to the treatment of other neurodegenerative diseases, as Alzheimer's", she included.

She told the BBC: "The case for these is not as clear-cut as for Huntington's disease, they are more complex and less well understood". At present around 10,000 people in United Kingdom and 30,000 people in America are living with Huntington's disease.


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