You’ve probably heard the saying, “the journey of a thousand miles begins with a single step.” That’s very true, but it’s also true that getting to that first step can be a journey all its own. Families living with Huntington’s disease can perhaps relate to this better than anyone.
For years, this genetic condition has cut a heartbreaking path through the lives of generations of families, with each carrier of the impacted gene knowing every time they have a child, that child carries a 50/50 chance of inheriting the gene.
That inheritance is a grim one.
People who test positive for Huntington’s disease experience a steady degradation of cognitive, psychological, and motor ability as mutated copies of the protein huntingtin attack neurons in the brain.
For years doctors have only been able to treat the symptoms. Outside of a cure, researchers and Huntington’s families alike have been on a journey to find some way to slow disease progression or reverse its damage.
That’s the destination, but the first step on the journey has been a long time coming. However, a group of researchers from Ionis Pharmaceuticals, the CHDI Foundation, Roche Pharmaceuticals, and the University of California San Diego may have finally taken that step.
The team has been working on a new drug—IONIS-HTTRx—to treat the genetic causes of Huntington’s disease.
The drug is an “antisense treatment” that works to block the production of the protein responsible for neuron destruction. In animal tests, the drugs has been effective and safe in mice and monkeys. In mice, the drug seemed to temporarily slow progression and even reverse some of the motor deficits associated with the disease. Monkeys who received the drug by spinal canal injection showed a 50% reduction in cortical huntingtin levels.
Based on these promising results, clinical trials in humans are now underway. Researchers caution that a viable, commercially-available drug is still years away.
If this truly is the first step towards that destination, a thousand miles is still a hell of a journey—especially for anyone who’s living with Huntington’s disease today. But at least now they and their families are that one step closer.
Find out more about the IONIS-HTTRx results, and then visit the CHDI Foundation to learn more about Huntington’s disease.
