Many clinical trials for Huntington’s disease care are underway, with the hope of one day finding treatment to slow or stop disease progression.
Huntington’s disease is a progressive neurological disorder for which there are currently no disease-modifying therapies. Current treatments for Huntington’s disease aim to minimize the effects of symptoms and improve quality of life but do not slow the progressive loss of nerve cells in the brain.
However, research into better therapies for Huntington’s disease is underway, which may one day change the way healthcare professionals treat this condition.
This article examines how new and emerging research aims to change the way doctors treat Huntington’s disease. Experts share the advancements they find most exciting and their thoughts on the potential for a cure for Huntington’s disease.
Currently, there are no treatments available for Huntington’s disease that change the course of the disease.
“The three main pillars of management include lifestyle changes, pharmacologic management, and rehabilitation services, such as physical, occupational, and speech therapy,” said Emily Forbes, DO, director of the Huntington’s Disease Society of American (HDSA) Center of Excellence at the University of Colorado.
“Individuals with HD may be seen in a multidisciplinary setting, where additional services from genetic counselors, social workers, and neuropalliative specialists may provide resources, such as support groups or home care options, to support family and care partners,” said Forbes.
“The treatment is symptomatic and supportive,” noted Kyan Younes, MD, a neurologist at Stanford University School of Medicine.
As Younes explained, current treatment options target the effects of Huntington’s disease that impact well-being, including motor (movement) symptoms, mental health concerns, cognitive health concerns, and physical symptoms. Current treatment does not target the underlying causes of disease.
There are many clinical trials currently underway investigating new ways to treat Huntington’s disease, including trials for both disease-modifying therapies and treatments for better management of symptoms.
“A recently concluded trial demonstrated a once-a-day medication, valbenazine, can improve chorea symptoms in Huntington’s disease and is currently awaiting FDA approval,” noted Forbes. “Once available, this will be another option to help manage movements in those with bothersome chorea.”
Valbenazine is already approved to treat tardive dyskinesia, a movement disorder similar to chorea.
Other types of treatments under investigation include:
- gene therapy
- dietary interventions
- deep brain stimulation
- stem cell therapy
“The most exciting are the trials that reduce the mHTT protein,” added Younes.
mHTT is the name given to the Huntington protein mutation, which is responsible for the effects that lead to the death of nerve cells in Huntington’s disease. This protein mutation results from an extended string of CAG repeats within the HTT gene. These repeats lead to the production of an atypical protein that can interfere with the functioning of nerve cells. mHTT is responsible for many of the effects observed in Huntington’s disease.
Younes is most excited about trials investigating genetic techniques that interfere with the production of mHTT within the cell, such as RNA interference, antisense oligonucleotide approaches, and gene editing.
Forbes and Younes suggested that as research into different treatment options progresses, there will be an opportunity to help relieve symptoms caused by Huntington’s disease rather than just manage them.
“Once a disease-modifying therapy is developed, treatment would ideally be initiated early in the disease course, potentially prior to symptom onset,” explained Forbes. “The goal is to slow down the course of Huntington’s disease, preferably preventing its progression entirely.”
Younes indicated there might even be an opportunity to proactively start treatment for people who carry the genetic mutation that causes Huntington’s disease. This means doctors could prevent symptoms before they even begin.
“In that case, predictive testing [testing a person before they have any symptoms] would become more common, as those with a family history of Huntington’s disease can know their status and be ready to initiate treatment once early signs of the disease become apparent, or potentially prior to any symptom onset,” added Forbes.
How soon is the future?
While Forbes is optimistic about the future of Huntington’s disease care, she acknowledged that it might take a while to see these changes implemented in the clinic.
“In the last few years, the Huntington’s disease community has experienced the disappointment of clinical trials not ending the way we hoped,” she explained. “I think it is important to remember that even if a trial does not provide the results we hoped for, it still gives us valuable information, which will move the field forward and continue to bring us closer to a cure.”
Younes also noted that scientists halted several clinical trials involving Huntington’s disease treatments early as they worked to understand the results.
“While there is good reason to be optimistic, it will take years before a trial results in a clinically available disease-modifying treatment,” noted Forbes.
Both Forbes and Younes, along with
“I think there is good reason to be optimistic about this, and it is more a question of when there will be a cure,” said Forbes.
“Because all cases of Huntington’s disease are caused by the same genetic mutation, there is a very clear therapeutic target,” she explained. “There are currently various trials underway targeting the cause of Huntington’s disease from numerous different angles. I think the effort and dedication of researchers and those who participate in clinical trials continues to advance our understanding of Huntington’s disease and brings us closer to a cure.”
Although no disease-modifying therapies currently exist to slow or halt the progression of Huntington’s disease, many new types of treatment are under investigation that may offer hope for the future.
“Huntington’s disease clinical trial news could be an emotional roller coaster,” acknowledged Younes. “Clinical trials take a long time, but this long process is necessary to find an effective treatment.”
“New strategies targeting DNA and RNA in Huntington’s disease engender new hope that a successful strategy will emerge, but there is much work ahead,” he concluded.