The treatment, which consists of the injection of a drug, is the first developed to reduce the production of the mutant toxic huntingtin, the underlying cause of the disease.
For the first time, a drug under development has been shown to slow the progression of the degenerative Huntington ‘s disease, which is genetic in origin and has devastating effects, the Pharma Web site said on Monday.
The treatment, which consists of the injection of a drug, is the first developed to reduce the production of the mutant toxic huntingtin, the underlying cause of the disease, according to a statement from Ionis, the pharmaceutical company that is developing it.
Although still in a state of clinical development, IONIS-HTTRx – the official name of the drug – is presented as the first innovation of its kind in efforts to contain this pathology.
“The key is now to move quickly to a larger testing process to see if it slows the progress of the disease,” said Sarah Tabrizi, the lead researcher for the project.
Innovation offers an alternative for all who suffer from Huntington’s disease, regardless of the individual mutation of each, making it “unique. ”
In the tests performed with the remedy, the researchers observed a reduction of the huntingtin protein. The drug was also safe and well tolerated, which led to its continued development.
There is currently no treatment to correct Huntington’s disease and the drugs focus on attenuating and controlling their symptoms.
Huntington’s disease is genetic, rare and neurodegenerative, and causes deterioration of mental abilities and physical control.
The condition usually appears in people between the ages of 30 and 50 to continue to worsen for a period of 10 to 25 years. In addition, the disease affects 30,000 people and threatens another 200,000 in the United States alone, as the company recalled in the statement.
The treatment license was recently acquired by Roche, which will be in charge of finalizing its development and its subsequent commercialization after the payment of US $ 45 million.
In the first half of 2018, both Roche and Ionis, the two pharmacists involved in drug development, are expected to present the results of their studies in scientific journals.