In a 'ground-breaking' development, scientists have developed a drug that can treat Huntington's disease by lowering the level of the harmful protein in the nervous system.
Huntington's disease is characterised by the progressive loss of nerve cells in the brain. This fatal disorder is caused by a hereditary defect in a single gene.
IONIS-HTTRx successfully lowered the level of the harmful huntingtin protein in the nervous system.
The first human trial of huntingtin-lowering drug began in late 2015 by researchers from the University College London in the UK.
The trial involved 46 patients with early Huntington's disease at nine study centres in the UK, Germany and Canada.
Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain.
As the phase 1/2a trial progressed, the dose of IONIS- HTTRx was increased several times according to the ascending- dose trial design.
IONIS-HTTRx was well-tolerated by the trial participants and its safety profile supported further testing in patients.
"The results of this trial are of ground-breaking importance for Huntington's disease patients and families," said Sarah Tabrizi, from the University College London.
"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," Tabrizi said.
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