Huntington’s disease is characterized by the progressive degeneration of nerve cells in the brain.
As a result of the breakdown of these very important cells, the body and mind of a Huntington’s patient often develops movement, thinking and psychiatric disorders. The negative impact Huntington’s disease has on a patient’s functional movements is substantial, but many don’t experience symptoms until they reach the age of 30, sometimes 40.
Treatment options are available for Huntington’s disease patients, but these medications are currently unable to alleviate the physical, mental and behavioral degeneration that is associated with the inherited disease.
Just as frustrating as developing an effective treatment for Huntington’s is identifying disease progression in patients. It is often extremely difficult for researchers to determine Huntginton’s disease progression in clinical trials—but there may be some hope.
Recently, in an article published in JAMA Neurology, researchers have proposed the use of a new measure to help determine disease progression before performing a motor diagnosis.
Early diagnosis of Huntington’s can often be done by early genetic screening, as it is an inherited disease. A more official diagnosis of Huntington’s, however, is determined by the evaluation of motor skills through the Unified Huntington’s Disease Rating Scale (UHDRS). This rating scale is almost always able to detect if the motor symptoms a person displays are caused by Huntington’s disease.
The problem with this is when researchers tried to apply the method to clinical trials. Motor diagnosis failed to be an effective technique to measure treatment effectiveness because Huntington’s disease is a slow and progressive illness. Thus, significant motor events often did not happen during the length of an average clinical trial.
Recently, however, a group of international researchers have tried to remedy this issue. They decided to look into progression-free survival (PFS) as an alternative measurement of Huntington’s progression.
In their report, researchers wrote, “Progression-free survival is defined as the time elapsed from treatment initiation to the first event of HD progression or motor diagnosis, whichever comes first.”
For this study, the researchers went through almost seven years of follow-up data that were recorded between January 2008 and November 2014. They also tested the PFS technique for Huntington’s disease progression in a group of 366. The conclusion of their research led them to the conclusion that PFS determination could be used to determine Huntington’s disease progression.
This news is super hopeful for the future of Huntington’s disease research, as this information will definitely come up in future clinical trials. Researchers note that “This approach is especially appealing when a researcher wants to examine whether a treatment delays motor onset.”
It’s possible that this PFS insight could lead to a lot of new Huntington’s disease information that will be crucial to finding more effective treatments for patients. To read more about this research, check out Huntington’s Disease News here..