Promising Huntington’s Therapy Candidate Discussed at Hereditary Disease Foundation Meeting

About 300 international scientists and industry leaders recently gathered in Cambridge, Massachusetts, for the Hereditary Disease Foundation‘s 10th biennial “Milton Wexler Celebration of Life” Symposium, to promote collaborations that can take scientific findings from the laboratory into the lives of patients as treatments.

One of the highlights of the meeting, according to the foundation, was a discussion on the development of Ionis Pharmaceuticals‘ drug candidate IONIS-HTT, now in Phase 1 and 2 clinical trials (NCT02519036) still recruiting participants in the United Kingdom, Germany, and Canada.

Huntington’s Disease News has been following the development of the therapy, which is designed to reduce the production of huntingtin, which in its mutant form aggregates and kills neurons in the brain.

IONIS-HTT builds on a technology that is still far from mainstream. An antisense molecule, essentially the mirror image of one of the DNA strands in the gene, binds to the huntingtin gene, preventing the formation of the mRNA molecule that acts as a mediator in the process of making a protein from the gene.

Expectations of the drug are high, but so far, only results from mice studies are available, and the human trials will determine whether the drugs work in patients as well.

“There are brilliant scientists from around the world working to conquer Huntington’s disease and other brain bandits,” Nancy S. Wexler, president of the Hereditary Disease Foundation, said in a news release, adding that funding and support from the foundation that these scientists receive have led to breakthroughs in the past.

“The exciting research presented at this year’s symposium suggests that we are on the brink of making new discoveries that will bring hope and healing to the millions of patients and families in the United States and worldwide who are affected by brain disorders,” Wexler said.

The Hereditary Disease Foundation has long held Huntington’s disease as a major focus and was involved in organizing the 1983 research mapping the region of the DNA linked to Huntington’s, which a decade later allowed scientists — also supported by the foundation — to identify the huntingtin gene.