Treatments for neurological diseases have been developing rapidly in the modern era. Despite the development of new medicines and therapeutic techniques for many conditions, no cure has been found for Huntington’s disease. According to a July 10th report on raredr.com, however, Huntintgton’s treatment may soon change as well. There are several advances which appear both promising and soon-to-be-realized. Keep reading to learn more, or follow the original source here for more information.
What is Huntington’s Disease?
Huntington’s disease is a progressive neurological condition affecting the brain. Huntington’s disease affects between three to seven people in about every 100,000. Onset of Huntington’s typically occurs between the ages of 30 and 40 with an average lifespan of 15 to 20 years after the first symptoms appear.
Huntington’s disease is caused by a mutation on the HTT gene. It is passed on by a genetic process in which at least one parent must be a carrier of the HTT gene. Each child of a parent carrying the HTT gene has a 50% chance of inheriting the gene.
Common symptoms of Huntington’s disease include personality changes, decline in cognitive ability, and a loss of motor control.
Old Becomes New
One area of research that seems to be changing the landscape of Huntington’s disease treatment is repurposing existing drugs. This involves the use of drugs already approved to treat other conditions to treat a different condition.
Researchers are currently studying a drug known as KD3010 which has shown promise in mice studies of Huntington’s disease. KD3010 was originally intended to treat diabetes but never gained approval in that context. KD3010 is unique in the way it interacts with a transcription factor known as PPAR Delta, and is able to cross the blood-brain barrier.
Researchers suspect that KD3010 may be more effective on its own than a current treatment known as bexarotene. There is also some hope that the two drugs can be used in combination to create a more ideal treatment with a lower dosage.
Gene Editing and Immunotherapy
CRISPR technology is currently being tested on a wide variety of conditions. It is no surprise then that a new enzyme is being tested with CRISPR which may allow for the treatment of Huntington’s disease. The new enzyme would be able to increase the accuracy of the gene editing technique. This would allow scientists to turn off the gene responsible for Huntington’s without affecting other important DNA nearby.
Another potential treatment could come in the form of immunotherapy. A New York biotech company known as Vaccinex is currently testing an antibody coded VX15 in what is referred to as the SIGNAL trial. The antibody affects a molecule scientists believe is a major part of Huntington’s disease. So far, results seem to indicate that VX15 is able to prevent some of the progressive brain abnormalities caused by Huntington’s.
The Big Deal with ASOs
Perhaps one of the largest developments, however, is the use of antisense oligonucleotides (ASOs) for Huntington’s disease treatment. ASOs are a form of DNA molecule which attaches itself to RNA responsible for Huntington’s disease. Due to the large number of the molecules in this treatment, ASOs are administered via lumbar puncture.
A Phase 1 trial has begun to show how a monthly dose of ASOs lowers Huntington’s protein levels in the body. Increasing the dosage over a four month span appears to affect the patient positively.
A similar clinical trail examining ASOs in the treatment of spinal muscular atrophy caused Huntington’s researchers to be even more excited about the prospect of a new treatment. The trial for a drug called nusinersen was actually stopped because the positive affects of the ASO treatment were obvious enough that it was important for all patients to receive treatment. The drug recently received FDA approval. This ASO is deliver in the same manner as the ones being tested for Huntington’s disease. Some researchers view this development with great optimism.