April 23, 2024

Huntington’s Disease Treatment: Current Options and Ongoing Research

Huntington’s disease is a genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities usually during their prime working years, between ages 30 and 50. There is currently no cure for this devastating disease, but researchers are making steady progress in developing new treatment approaches. In this article, we will explore some of the latest advances in Huntington’s disease treatment and outlook for the future.

Symptoms and Causes
The core symptoms of Huntington’s disease include movement abnormalities such as chorea (involuntary, irregular movements), psychiatric issues such as mood swings and cognitive decline. These symptoms worsen progressively with time and the disease is ultimately fatal. Huntington’s disease is caused by an expanded repetitive section of DNA on the Huntington gene which produces a mutant Huntingtin protein that causes progressive nerve cell death in certain areas of the brain like the basal ganglia. The mutation is inherited in an autosomal dominant manner, meaning offspring of an affected individual have a 50% chance of inheriting the mutation.

Current Treatment Approaches
There is currently no treatment that can stop or reverse the progression of Huntington’s disease. Available treatments currently only target individual symptoms to provide relief. Movement abnormalities are treated with medications such as dopamine blockers which help reduce chorea movements. Antidepressants are used for mood symptoms and antipsychotics for psychiatric problems. Other symptomatic treatments include speech, occupational and physical therapy which help maintain motor skills and independence for as long as possible. Lifestyle modifications like a healthy balanced diet, exercise, stress reduction also help some individuals cope better.

Gene Silencing Drugs
One of the most promising new treatment approaches is gene silencing therapy which aims to directly target the root cause of the disease. These drugs use oligonucleotides or small interfering RNAs to reduce the production of mutant Huntingtin (mHTT) protein by binding to its messenger RNA (mRNA) and preventing its translation into protein. Some gene silencing candidates like IONIS-HTTRx are already in late stage clinical trials. Early results are encouraging with significant reductions in mHTT levels observed without serious adverse effects so far. Researchers are hopeful this approach may be disease-modifying if administered at early presymptomatic stages.

Stem Cell Therapy
Another area of active research is stem cell therapy to potentially replace dying nerve cells in Huntington’s disease. In animal model studies, transplantation of human neural stem cells into the striatum has shown they can survive, migrate appropriately and differentiate into medium spiny projection neurons – the kind of brain cells most affected in Huntington’s. Further studies are exploring the use of induced pluripotent stem cells which are generated by reprogramming adult cells into an embryonic stem cell like state with unique potential for personalized cell therapy. Clinical trials evaluating the safety and efficacy of transplanting stem cell derived neurons in HD patients are still underway.

Lifestyle Management and Clinical Trials
While new disease-modifying treatments are being developed, integrating lifestyle management practices can help maximize quality of life and motor function for HD patients. A combination of physical and cognitive exercises tailored to individual abilities is recommended. A balanced diet, stress reduction techniques, avoiding smoking and excessive alcohol also support overall health. Enrolling in clinical drug trials also gives people access to cutting edge investigational treatments. Several Phase 2 and 3 trials are currently recruiting for gene silencing drugs, antioxidants, anti-inflammatories and other promising therapies.

Outlook for the Future
Significant progress is being made in developing more effective symptomatic treatments and disease-modifying strategies for Huntington’s disease. If ongoing gene silencing drug trials prove successful, they may provide the first approved medical treatment that can delay or slow disease progression. Stem cell therapy also holds promise to replace lost neurons. With continued research support and clinical trial participation, more personalized medicines tailored to an individual’s disease stage may become available in the near future. While a complete cure still remains elusive, advancing treatments are offering new hope to HD families worldwide.

*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraaged AI tools to mine informaation and compile it