In the first gene therapy of its kind in humans, a clinical trial has begun at Nationwide Children’s Hospital in Columbus Ohio to evaluate a new investigative treatment for victims of a rare neurodegenerative disorder, Batten disease CLN6.
The study is funded by The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease (www.curebatten.org), which was launched in June 2015 by film producer Gordon Gray and his wife Kristen after their two young daughters were diagnosed with the fatal childhood disease.
Crowd-funding raised US$3.5million to support the trial, which was fast-tracked by doctors from various institutions who have collaborated on the project. Led by Drs. Brian Kaspar, Jerry Mendell and Emily De Los Reyes, the hospital’s clinical team enrolled the first patient, five-year old Charlotte Gray and additional patients are being recruited.
The study employs the AAV9 virus, which is not known to cause adverse effects in humans and is able to deliver a replacement CLN6 gene to the brain. The malfunctioning of the mutated CLN6 gene is what causes Batten disease, and the replacement gene appears to help restore the mutated gene’s functionality, allowing cells to purge built-up wastes and restore balance in the brain.
Find out more at curebatten.org.