The long-anticipated clinical trial of a drug that could potentially stop Huntington’s disease at its genetic roots and perhaps someday even prevent the disorder in presymptomatic HD gene carriers like me could start by the middle of 2015.
If successful, the trial could result in a drug in five or six years.
Officials at Carlsbad, CA-based Isis Pharmaceuticals, Inc., in an interview with me on August 22, said that the Phase I trial for their drug, ISIS-HTTRx, likely will start by the second quarter of 2015, as long as the company receives regulatory approvals and fulfills other standard requirements for trials.
ISIS-HTTRx is an antisense oligonucleotide (ASO), a synthetic strand of DNA that silences, or turns off, the messenger RNA that makes proteins as coded by the DNA. If ISIS-HTTRx works as intended, it would reduce the production of the huntingtin protein in brain cells, reduce damage to the brain, and reduce or even eliminate HD symptoms.
ISIS-HTTRx is the company’s internal name for the drug, which will later receive a generic scientific name and, if it reaches the market, a commercial name. HTT is scientific shorthand for the huntingtin gene, messenger RNA, and protein. Rx is shorthand for a medical prescription.
Isis is also conducting standard toxicological studies of the drug in non-human primates to assure that it will not cause harm to humans. A Phase I trial tests for safety and tolerability. Researchers can make observations about the drug’s efficacy but must then conduct Phase II and Phase III trials, which involve more people, to demonstrate whether the drug really works.
Isis is planning the trial with the Swiss pharmaceutical giant Roche, vastly experienced in clinical trials and staffed with specialists in neurological disorders. Last year the two entered a partnership that included a $30 million infusion of funds into the Isis preparations for the clinical trial.
The trial will involve 36 early-stage Huntington’s patients at four to six sites in Canada and Europe. If Phase II occurs, the companies would extend the study to the U.S.
Only recently did Isis, a world leader in ASO science and technology, settle on ISIS-HTTRx.
You can watch my brief report from Isis headquarters in the video below. Soon I will provide a detailed report on the ISIS-HTTRx clinical trial project.
Source: At Risk for Huntington’s Disease