US neurologists are staking their hopes on gene therapy as a potential new treatment for Huntington’s Disease, according to a new market report.
However, those interviewed for the report, by Spherix Global Insights, also expressed frustration at the absence of available treatments to slow cognitive decline.
Huntington’s disease (HD) could be called the perfect storm of neurodegenerative diseases. It causes a loss of critical striatal neurons which affect not only motor function – causing symptoms like the hallmark chorea (rapid, jerky involuntary body movements) – but also cognitive and emotional function.
Patients lose motor control, experience psychiatric symptoms, and cognitive decline.
A neurologist’s perspective from a recent research study suggests: “A Huntington’s patient is almost a combination of Alzheimer’s, tardive dyskinesia, Parkinson’s – all in one. Basically, every symptom has an unmet need, save for perhaps- CHOREA.” (General Neurologist)
The only treatments for HD are so-called ‘symptomatics’, which address abnormal movements and some psychiatric symptoms, but do not alter the worsening of the disease or the myriad other symptoms.
Generic tetrabenazine has been used for years to help with abnormal movements and more recently, Teva’s Austedo (deutetrabenazine) was approved as another movement disorder alternative. But beyond that, the only other pharmaceutical interventions used for HD are antipsychotic and antidepressant therapies for emotional and behavioural symptoms.
Recognising the significant treatment unmet needs, Spherix Global Insights conducted a market landscape study, seeking insights from neurologists managing HD.
Neurologists answered questions about their familiarity, interest, and degree of advance over current treatments for five pipeline agents: Hoffman-La Roche/Genentech’s tominersen, PTC Therapeutics PTC518, Sage Therapeutics SAGE-718, UniQure Biopharma’s AMT-130, and Wave Life Sciences’ WVE-003. Additionally and unaided, neurologists also mentioned Annexon Biosciences’ ANX005, Azevan Pharmaceuticals’ SRX246, SOM Biotech’s SOM3355, and Vaccinex, Inc.’s pepinemab.
The findings revealed that over 80 per cent of neurologists agreed that a top unmet need in HD is to stop neurodegeneration prior to the onset of symptoms. Furthermore, an equal number of neurologists also expressed frustration with the absence of meaningful treatments to slow cognitive decline, a critical concern given patients’ delayed seeking of treatment due to a lack of insight into 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. Its family who says you’re moving all the time and it’s a problem and the patient isn’t actually disabled by it,” said one Movement Disorder’s Specialist.
Study results show that practicing neurologists have little to no knowledge of the assets in clinical development – less than one in twenty neurologists can name a specific HD therapy in development. However, one-fifth are aware that gene therapy, conceptually, is one of the potential solutions being studied for HD and two-thirds are very interested in bringing a gene therapy to market.
“Looking at the pathology, we know there’s a genetic mutation,” one said.
“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 we talked about and the progression in the caudate nucleus in the basal ganglia.”
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.
