University of South Florida Conducts Huntington Clinical Trial Exploring Gene Splicing with Roche/Ionis Investigational Drug

Aug 10, 2019 | Huntington's Disease, Ionis, RG6042, Roche

Genetic Medicine with dna isolated on white. 3d rendering

The University of South Florida is conducting a phase III clinical trial for a drug that targets genetic cause of Huntington’s disease (HD) which can be thought of as having a mix of Parkinson’s, Lou Gehrig’s disease and Alzheimer’s disease. Follow the link for a Health News’ interview of neurologist Dr. Juan Sanchez-Ramos about this new clinical trial in Florida.

University of South Florida (USF) clinical investigator Dr. Juan Sanchez-Ramos gets interviewed by multimedia journalist Daylina Miller of Health News WJCT.

Huntington’s Disease

Huntington’s disease (HD), also known as Huntington’s chorea, is an inherited disorder that results in the death of brain cells. The earliest symptoms are often subtle problems with mood or mental abilities. A general lack of coordination and an unsteady gait often follow. As the disease advances, uncoordinated, jerky body movements become more apparent. Physical abilities gradually worsen until coordinated movement becomes difficult and the person is unable to talk. Mental abilities generally decline into dementia. The specific symptoms vary somewhat between people. Symptoms usually begin between 30 and 50 years of age, but can start at any age. The disease may develop earlier in life in each successive generation. About eight percent of cases start before the age of 20 years and typically present with symptoms more similar to Parkinson’s disease. People with HD often underestimate the degree of their problems.

The Study

The clinical trial is testing what’s being called the “most advanced HD therapy in development, RG6042.” Recent research reveals that gene-silencing treatment, which is being administered through a spinal tap in the trial, reduced the amount of the illness-inducing protein in a study. The study is fully enrolled.

The study is sponsored by Swiss-based Roche. The study’s investigators (USF is one of the multiple sites) and the sponsor is evaluating the long-term safety and tolerability of RG6042 in participants who have completed other Roche or Genentech sponsored studies in the Huntington’s disease (HD) in the development program for RG6042. The study targeted 950 participants and is scheduled to end in 2023.

What is RG6042?

Recently Huntington’s Disease News covered the study of RG6042. The original developer is Ionis. The compound is an antisense drug designed to reduce the production of huntingtin (HTT) protein, which is the genetic cause of Huntington’s disease. HD is caused by the expansion of the CAG trinucleotide sequence in the HTT gene, which produces a toxic protein that progressively destroys neurons in the brain. As a result, HD patients experience progressive loss of mental faculties and physical control as the disease progresses. The European Medicines Agency has granted orphan drug designation to Ionis HTTrx for the treatment of patients with HD.

Roche and Ionis (Isis Pharmaceuticals) Partnership

In 2013 Roche and Isis Pharmaceuticals announced the partnership to develop the drug to address Huntington’s disease. San Diego based, the firm changed its name to Ionis

University of South Florida Lead Investigator

Dr. Juan Sanchez-Ramos 


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