Potential for new Huntington’s treatment sparks optimism for MDs
But US neurologists also express frustration with current lack of therapies
Neurologists in the U.S. are optimistic about the potential for a new treatment for Huntington’s disease, but emphasize the need for therapies that halt nerve degeneration before symptom onset, according to a report by Spherix Global Insights.
Most of the specialist physicians surveyed hoped for a gene therapy that would prevent nerve cell loss.
More than 80% “also expressed frustration with the absence of meaningful treatments to slow cognitive decline, a critical concern,” Spherix stated in a company press release.
In the report series, called Market Dynamix: Huntington’s Disease (U.S.) — an ongoing survey of neurologists about disease-modifying therapies (DMTs) for Huntington’s — the experts described their expectations for any new treatment being developed.
“Research from Market Dynamix: Huntington’s Disease (US) affirms neurologists’ optimism (perhaps buoyed by the dearth of therapies today) about effective disease-modifying treatments coming from biopharmaceutical pipelines within the next five years,” the release stated.
Nearly all Huntington’s symptoms have unmet treatment need, per neurologists
Huntington’s is a neurodegenerative disorder caused by excessive repeats of three nucleotides, or DNA’s building blocks, in the HTT gene. This results in the production of an abnormally long huntingtin protein that’s prone to form toxic clumps, triggering the loss of nerve cells in the brain.
The disease is marked by a wide range of physical and mental symptoms, including uncontrolled rapid, jerky, involuntary body movements called chorea, a loss of cognitive ability, and psychiatric problems. In many cases, those affected are initially unaware of these symptoms.
“A Huntington’s patient is almost a combination of Alzheimer’s, tardive dyskinesia [a drug-induced movement disorder], Parkinson’s — all in one. Basically, every symptom has an unmet need, save for perhaps chorea,” one general neurologist was quoted as saying in the press release.
Alzheimer’s and Parkinson’s are two neurodegenerative diseases, the first marked mostly by cognitive deficits and the second primarily by motor impairments. Tardive dyskinesia occurs as an uncommon side effect of certain medicines, and is characterized mainly by facial tics.
To date, there are no approved DMTs that can slow or stop disease progression. There is treatment available that can help Huntington’s patients manage certain symptoms, such as chorea and psychiatric issues, but these do not address the disease’s cause.
Given the many unmet needs of people with Huntington’s, Spherix sought insights from neurologists who help patients manage the condition.
Key goals are stopping neurodegeneration, slowing cognitive decline
According to the survey, more than 80% of neurologists said that stopping neurodegeneration before the onset of symptoms is a top unmet need in Huntington’s.
Moreover, an equal proportion of neurologists said they were frustrated with the lack of treatments aimed at slowing cognitive decline. This issue is particularly critical as patients often seek treatment at later stages because they are not fully aware of their symptoms.
“Usually, chorea in Huntington’s … isn’t something that patients always notice and certainly as a result isn’t always something that bothers them,” said one movement disorder specialist. According to this clinician, it’s often “family who says you’re moving all the time and it’s a problem and the patient isn’t actually disabled by it.”
Despite the numerous experimental Huntington’s therapies in the pipeline, fewer than 1 in 20 neurologists — less than 5% — were able to name one. Still, one-fifth were aware that gene therapy is in the works as a potential solution, and two-thirds were very interested in bringing such a therapy to market.
If there’s a way that pathology [disease mechanisms[ in the brain could be halted or stopped or altered genetically … you wouldn’t have the atrophy [wasting] … and the progression.
“Looking at the pathology [disease mechanisms], we know there’s a genetic mutation,” said a movement disorder specialist.
“The repeats, we know the accumulation, the huntingtin protein. If there’s a way that pathology in the brain could be halted or stopped or altered genetically where you would have lesser repeats, you wouldn’t have the atrophy [wasting] … and the progression,” this specialist said.
The neurologists also were asked about their familiarity with and interest in — and the clinical stage — of five experimental therapies under development. These included tominersen (Roche/Genentech), PTC518 (PTC Therapeutics), SAGE-718 (Sage Therapeutics), AMT-130 (UniQure Biopharma), and WVE-003 (Wave Life Science).
The specialists also naturally mentioned four other therapies in the pipeline: ANX005, from Annexon Bioscience; SRX246, from Azevan Pharmaceuticals; SOM3355, by SOM Biotech, and pepinemab, from Vaccinex.
This is Spherix’s first market landscape study on Huntington’s disease.
About the Author
