A new drug for Huntington’s disease has shown “enormously significant” results according to researchers.

The Phase I trial of a new drug for Huntington’s yielded positive results, suggesting that the experimental drug could potentially be the first to treat this dreaded genetic illness.

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Researchers from the University College London discovered the treatment that would be capable of suppressing the toxic protein build-up in the brain which causes Huntington’s disease. Should this new drug passed all the necessary clinical trials, it will be deemed as the biggest breakthrough in the field of neurodegenerative study in five decades.

“For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s disease.” – Sarah Tabrizi

What is Huntington’s Disease?

Huntington’s disease is considered as one of the most devastating diseases today. The illness is a genetically inherited, progressive brain disorder that causes affected individuals to lose control of their movements and cognitive abilities which often lead to severe emotional problems.

To date, there is no known cure for Huntington’s. It is fatal and can affect anyone regardless of gender or age–children and adults alike. Many describe the symptoms of HD as having ALS, Parkinson’s, and Alzheimer’s diseases all at once.

Recent statistics show that approximately 3 to 7 out of 100,000 people of European ancestry are affected by HD. In the United States alone, one person in every 10,000 Americans or around 30,000 people are suffering from the condition, and over 150,000 others are at risk of developing the disease.

Some of the key symptoms of Huntington’s that are most likely to occur between the ages of 30 and 50 include:

  • personality changes, mood swings, and depression
  • problems with memory and judgment
  • unsteady walk and uncontrollable movements
  • difficulty speaking and swallowing, and weight loss

New Drug for Huntington’s Brings New Hope

The allegedly new drug for Huntington’s was developed by UCL researchers in collaboration with other scientists from over half a dozen places in Europe and Canada. The drug, called IONIS-HTTRx, is said to be the first drug to target the cause of HD.

How the new drug for Huntington's works
How the new drug for Huntington’s works | UCL via BBC | www.bbc.com

To explain further, HD is caused by a faulty gene on chromosome 4 of a person’s DNA. This gene is referred to as the huntingtin gene.

Normally, the huntingtin gene contains the instruction for creating a protein called huntingtin which is significant for the development of the brain. The instructions are carried by a short strand of genetic code or the messenger RNA. Now, because the gene is corrupted, it turns the protein into a killer of brain cells.

The drug works by delivering a small piece of genetic material that sticks to the RNA which in turn prevents it from building the toxic proteins.

During the drug’s phase 1 trial, Professor Sarah Tabrizi, the research lead and Director of UCL’s Huntington’s Disease Centre, and her team injected the drug into the fluid that bathes the brain and spinal cord of 46 patients.

Prof Sarah Tabrizi
Prof. Sarah Tabrizi, founder and director of the UCL Huntington’s Disease Centre | iris.ucl.as.uk

The procedure was reportedly conducted at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery in London. In an interview with BBC, Tabrizi said:

“I’ve been seeing patients in clinic for nearly 20 years, I’ve seen many of my patients over that time die. For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s disease. This is of groundbreaking importance for patients and families.”

The new drug for Huntington’s created a ripple of excitement in the medical community, with experts referring to it as both historic and phenomenal. Some cited that what makes the newfound drug interesting is the fact that it might hopefully reverse the progression of the disease.

“What’s really interesting in animal studies, if you stop the production of the mutant protein, not only does progression stop, but the brain starts to heal itself,” Christopher Ross, Director of the Huntington’s Disease Center at Johns Hopkins Medicine, said. “Which means there might be improvement.”

On the other hand, Blair Leavitt, a University of British Columbia researcher and the lead investigator for the North American group that conducted the study, said:

“This is the culmination of 20 years of work for all of us. To see that result and to know what it means — it means we’ve taken a really good promising step.”

The full details of the trial will be published in a scientific journal next year. The research was supported by Ionis Pharmaceuticals.

Are we going to see this new drug for Huntington’s helping millions of people around the world anytime soon? Will it be affordable enough for the masses to avail? What do you think?

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