SEP 04, 2018 08:56 PM PDT

Treating Symptoms of Huntington Disease

WRITTEN BY: Nouran Amin

Credit: Indiana Public Media

According to a new study published in the Proceedings of the National Academy of Sciences (PNAS), a genetic disorder known as Huntington's disease may occur much earlier in life than previously believed, and a new drug may control the disease. "This could be a good start to developing new promising treatments for Huntington's disease, treatments that could be administered even before signs of the illness appear," said Alexander Osmand, a researcher in the Department of Biochemistry and Cellular and Molecular Biology at the University of Tennessee, Knoxville, and co-author of the study.

Huntington’s disease leads to the progressive deterioration of both mental and physical abilities and is the result of a mutation in the huntingtin gene; a gene possessed by all humans and necessary for healthy development. When the huntingtin gene is mutated, it “causes the rapid decay of specific neurons, eventually resulting in death”. According to the Huntington's Disease Society of America, an estimated 30,000 Americans suffer from the disease and a 2013 study found that “treatment can cost anywhere from $4,947 to $37,495 annually depending on the severity of the condition”.

Almost always, the symptoms of Huntington's disease are not present until an adult reaches their 30s or 40s. However, researchers have discovered that an array of symptoms may be exhibited much earlier. Although these early symptoms are not clearly defined than the later, more debilitating symptoms, they could be useful to health care providers when further screening is needed.

The researchers exposed several litters of mice engineered to carry the human huntingtin gene to four different dose regimens of the drug, Panobinostat, which was used in the treatment of multiple cancers. The researchers believe that this particular drug is responsible for regulating gene expression, which is an important step towards treating individuals with Huntington's disease. Additionally, researchers examined mice behavior by studying their vocalization, startle response, and risk-taking behavior. Such behavioral abnormalities provided evidence that the effects of the disease were present prior to the “full mutation commonly associated with Huntington's”.

Even though treating patients with Panobinostat cannot reverse the gene mutation completely, multiple clinical trials have shown that the drug may inhibit gene changes implicated in the expression of the disease. Until now physicians were able to provide treatment to only a few of the symptoms associated with Huntington's disease including depression, mood swings, and involuntary movement. However, while these treatments make Huntington's disease a bit bearable, they do nothing to eradicate the disease itself.

Watch the video below to learn more about Huntington's disease:

 

Source: University of Tennessee at Knoxville, Proceedings of the National Academy of Sciences

About the Author
  • Nouran enjoys writing on various topics including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
OCT 15, 2019
Drug Discovery & Development
OCT 15, 2019
Effective Treatment for Ulveal Melanoma
Uveal melanoma is a rare and aggressive type of melanoma that targets the eye and affects an estimated 2,500 people in the United States annually. Nearly h...
OCT 15, 2019
Drug Discovery & Development
OCT 15, 2019
Novel Therapy Shows Promise for Acute Migraines
In a study published in The New England Journal of Medicine, a novel drug, by the name of rimegepant, was found to eliminate pain and reduce characteristic...
OCT 15, 2019
Cell & Molecular Biology
OCT 15, 2019
Stopping Metastasis After Breast Cancer Surgery
Cancer is most deadly when it metastasizes, which can happen after tumor-removal surgery. It's a serious challenge for many patients....
OCT 15, 2019
Drug Discovery & Development
OCT 15, 2019
MDMA Treats Social Anxiety in Autistic Adults
People with autism are more likely than those without to develop social anxiety (Bejerot: 2014). Meanwhile, current anti-anxiety medications and therapeuti...
OCT 15, 2019
Genetics & Genomics
OCT 15, 2019
New Gene Editing Technology to Combat Hereditary Diseases
Until recently, many types of gene-editing methods were unable to target critical parts of DNA. Now however, researchers at the Salk Institute have develop...
OCT 15, 2019
Drug Discovery & Development
OCT 15, 2019
Why Drugs that Work on Mice Don't Work on Humans
Mice are used as the frontline for drug testing for brain disorders. Why? Their brains are supposed to have much of the same functioning as ours. Moreover,...
Loading Comments...