According to the latest news, scientists might have found a new potential drug to treat Huntington’s disease. It appears that Huntington’s disease might get a new treatment due to an innovative drug. Researchers claim the new drug was tested on monkeys and mice, showing incredible results. At the moment, experts’ next step is to test it on humans in a clinical trial to see its effectiveness. If the clinical trial proves to be successful, people with Huntington’s disease might have a real chance to a better life.
Huntington’s disease (HD) is an affliction which leads to progressive deterioration of nerve cells in the brain. It affects the functional abilities of an individual, generally causing movement, cognitive and psychiatric problems. HD is an inherited disorder; every child of a parent suffering from HD stands a 50-50 chance of carrying the gene.
Most commons symptoms are depression, personality issues, forgetfulness, involuntary movements, weight loss, slurred speech; eventually, HD leads to death. Currently, there are medical treatments to manage the symptoms. Nonetheless, there aren’t medications which could prevent the physical and behavioral deterioration related to the disease.
However, a group of scientists developed a drug which aims to attack the cause of this lethal disorder. The new potential drug to treat Huntington’s disease is called IONIS-HTTRx. Researchers explained is an antisense drug which can inhibit the developing of the huntingtin protein, by silencing the gene.
Laboratory tests on mice and monkeys proved these types of drugs delay the disease’s progression and reverse the disease phenotype. The motor functions of a mouse improved after receiving the treatment for one month. Two months after the treatment, its health was back to normal.
In another mouse the results showed an improvement after 8 weeks of treatment. Its health continued to improve after nine months after experts stopped the treatment. In monkey, the new drug reduced the huntingtin gene and protein in their central nervous system.
Monkey tests indicated a 50 percent reduction of the cortical huntingtin levels. Also, the caudate nucleus of the brain decreased by 15-20 percent. After getting such promising results, experts plan to conduct a human clinical study. The participants will receive the drug monthly, in four doses.
Scientists explained they will inject IONIS-HTTRx into the cerebral spinal fluid, using an intrathecal injection in the lumbar space. After this, they will assess both the safety and tolerability of the new drug in different doses. Moreover, researchers will look at the drug pharmacokinetics and evaluate the therapy’s effects according to certain biomarkers and clinical results.
Nevertheless, it could still take a couple of years before the new drug can be used for clinical practices. If it proves to be successful, Huntington’s disease might be cured and patients will have their health restored.
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