A glimmer of hope has emerged for those suffering from Huntington’s disease and spinocerebellar ataxia, with the announcement that the first patient has been dosed in a Phase 1/2a clinical trial for a new therapy. These incurable neurodegenerative diseases currently have no cure or effective treatment, but this new trial represents a potential breakthrough in the search for a solution. This exciting news brings hope for the millions of people that are affected and their families, and could potentially pave the way for future treatments.
Vico Therapeutics has commenced a clinical trial that will evaluate VO659, an investigational antisense oligonucleotide for the treatment of Huntington’s disease and spinocerebellar ataxia types 1 and 3. The trial has dosed the first patient in the phase 1/2a study, which will assess the safety and tolerance of multiple ascending doses of intrathecally administered VO659 in 71 individuals diagnosed with early manifest Huntington’s or mild to moderate spinocerebellar ataxia type 1 or 3. The therapy targets the CAG repeat expansion responsible for these diseases.
This drug candidate has demonstrated significant and dose-dependent reductions of mutant huntingtin protein in preclinical studies, as well as allele-preferential reductions of the protein in Huntington’s patient cell models. Similar reductions in mutant ATXN1 and mutant ATXN3 were observed in patient cell models of SCA1 and SCA3. These promising results suggest VO659 may represent a groundbreaking new treatment for patients suffering from these devastating disorders.
According to Scott Schobel, MD, chief medical officer at Vico, “We are encouraged by the continued progress of our development program and very pleased to announce the first patient dosed in this phase 1/2a study of VO659 in [Huntington’s disease], SCA1, and SCA3. VO659 is the first allele-preferential [antisense oligonucleotide] in clinical development with broad application to all CAG repeat expansion diseases.”
This technology has demonstrated the ability to silence genes that cause devastating disorders, such as the neurodegenerative diseases that afflict millions of people worldwide. Antisense oligonucleotides hold the potential to halt or even reverse debilitating genetic diseases, offering hope to patients who have been underserved by traditional therapeutics.
As more research is conducted in this field, it is expected that additional promising treatments will be developed that offer hope to patients with previously untreatable genetic conditions. Vico Therapeutics’ VO659 is an exciting and innovative step forward in this important area of research, providing much-needed hope to patients with Huntington’s disease and spinocerebellar ataxia types 1 and 3.
With the first patient successfully dosed in the phase 1/2a trial of a potential treatment for Huntington’s and spinocerebellar ataxia, there is hope for thousands of patients suffering from these devastating diseases. While there is still much work to be done in the clinical trial process, this milestone marks an important step forward in the quest for effective therapies. As researchers continue to explore new treatments, we can look to this progress as a symbol of hope for patients and their families everywhere.
