For the first time ever, a drug trial has been shown to reduce the number of harmful proteins that are the cause of Huntington’s disease.
The hope is that this drug could be the key to slowing and potentially even stopping the debilitating disease in its tracks.
Huntington’s disease is a genetic disorder that affects around 1 in every 10,000 people by damaging nerve cells in the brain. There is currently no cure and no way to slow the progression of the disease.
After a full decade in pre-clinical development, the first human trial of the drug began in 2015 and included some 46 patients.
Injected directly into spinal fluid, the drug has been shown to actually lower the levels of the toxic huntingtins protein that devastates the nervous system.
Professor Tabrizi, Director of the UCL Huntington’s Disease Centre and IONIS-HTTRx Global Chief Investigator, said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families.”
Huntington’s is a hereditary disease that is caused by a fault in your DNA and affects the body’s nervous system.
The error is found in the huntingtin gene that tells the body to produce the huntingtin protein.
Normally this protein is vital for the development of the brain however the error in the DNA tells the protein to actually attack brain cells rather than encourage their growth.
How does the new drug work?
The drug known as IONIS-HTTRx is injected directly into the spinal fluid of the patient, this allows the drug to be sent straight to the brain.
Once it reaches the brain the drug gets to work in killing something called a mRNA. This is in effect the messenger which tells the body to create the toxic version of the huntingtin’s protein.
By killing the messenger the drug can slow the progress of the disease.
“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated.” explains Professor Tabrizi.
“The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”
What happens next?
Thanks to the success of the drug, the next stage will be a much larger trial. Currently the results are based on a very small sample stage (just 46 patients) so the next step will be making sure that it has the same dependable results across all patients suffering from the disease and to then measure the effects depending on the advancement of the disease.
While no doctors are calling this a cure, they are cautiously optimistic. The success of the human trials echoes the earlier animal trials which not only showed a slowing of the disease but even some recovery of motor function.