Crowdfunding for life
Activist parents propel Tel Aviv University scientist to find cure for devastating disease.
This Rare Diseases Day (February 28, 2017) will be a particularly hopeful one for families of children suffering from Vanishing White Matter (VWM), a rare and fatal brain disease.
Prof. Orna Elroy-Stein, a cell biologist at Tel Aviv University (TAU), recently reached a breakthrough in her research on the disease, identifying several drugs that cured affected cells in lab experiments….
This milestone is the fruit of a unique marriage between Elroy-Stein and a group of unrelenting families from around the world, who accelerated her research through intensive grassroots fundraising and activism.
“This is a very exciting phase,” says Elroy-Stein. “It proves that all of the efforts by the families have helped us. More importantly, it gives all of us hope.”
Yet, the glimmer of optimism comes with a redoubled sense of urgency. Most children with VWM don’t survive their teens, and every advance in Elroy-Stein’s research has the potential to save another member of the global VWM community.
Vanishing White Matter is a genetic neurodegenerative disease, with less than 1,000 cases reported around the world.
The “white matter” is myelin, fatty tissue that insulates and protects nerve fibers in the brain and the cells that produce the tissue itself. Myelin recedes in everyone following physiological stress, such as a virus or head trauma, but in healthy people it regenerates itself, and the body continues to function normally. People with VWM have a genetic mutation that causes myelin to form less accurately and regenerate more slowly. Symptoms usually appear in the toddler years, when regular childhood occurrences, such as the flu or bumps to the head, cause myelin recession, and the children never fully recover. As a result of additional stress events, the clinical symptoms intensify and the nervous system eventually collapses, leading to coma and death.
Image credit tau.ac.il