Huntington disease is an illness which is inherited and is caused by a faulty gene in your DNA. This condition damages certain nerve cells in your brain causing behavioural changes, restriction of movement and cognitive issues. The disease is caused by a defect in the 4th chromosome known for making the protein Huntington which is vital for development in the brain, however the genetic error corrupts the production of the protein causing it to become a killer of brain cells. The illness is usually common in people ages 30 to 50, however if someone develops it at a younger age then it is known as juvenile Huntington disease.
There has been a breakthrough in research that could essentially correct the cause of the neurodegenerative disease. Research conducted by University College London predicts that there is now hope that the disease can be stopped. The treatment discovered is designed to help silence the gene by four steps: the instructions for making huntingtin are locked away inside the DNA in a cell’s nucleus, those blueprints have to be carried to a cell’s protein-making factories and that job is done by a short strand of genetic code, called messenger RNA; the drug kills the messenger; it is an engineered piece of genetic code that is the precise mirror image of the messenger, known as an antisense oligonucleotide. It binds to the RNA and neutralises it.
On the trial, 46 patients had the drug injected into the fluid that cleanses the brain and spinal cord. The process was carried out at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery in London. Doctors were not certain on what would happen. One fear was the injections could have instigated fatal meningitis. But the first in-human trial showed the drug was safe, well tolerated by patients and crucially reduced the levels of huntingtin in the brain.
Research professionals say that this new publication of research can pave the way for many other genetic illnesses. There is still a way to go to improve research and test it on a larger group of patients, but this marks the start of a change for many people battling the illness.
Sources: The BBC, NHS. Huntington Disease Association