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Find a Cure for VWM
Posted on March 24 2014
Imagine for a moment that a child you love, perhaps your son, daughter, granddaughter, or niece, was unexpectedly diagnosed with one of those “parent’s worst nightmare diseases.” In this instance, the monster disease is Leukodystrophy called Vanishing White Matter disease.
Vanishing White Matter disease (VWM), also known as Childhood Ataxia with Central Nervous System Hypomyelination (CACH), is a devastating condition that destroys myelin” the brain's white matter. Unfortunately, there is no known cure or form of treatment for this disease. Symptoms generally appear in young children who appear to be developing fairly normally. A diagnosis is usually made in the early years of life at around 2 to 6 years old. According to the United Leukodystrophy Foundation, www.ulf.org - http://ulf.org/what-is-leukodystrophy VWM/CACH is one of 40 known forms of leukodystrophy.
Like many of you, I was once unaware about VWM. I never heard of the disease and did the thought of it ever affecting my life never even cross my mind. But then in 2009, my niece was diagnosed with VWM Leukodystrophy, and ever since that moment I’ve been searching the web like crazy, day and night, trying to help my sister save her daughter. I can still remember clearly the day my sister told me about the MRI results, and how they showed signs of Leukodystrophy. I was devastated, and the more I read about Leukodystrophy the more horrified I would get. My heart sank when in one article I read stated, “These disorders are progressive, meaning that they tend to get worse throughout the life of the patient”. All Leukodystrophies are a result of problems with the growth or maintenance of the myelin sheath. However, there are many genes that are important in this process. Leukodystrophies also share some common characteristics with Multiple Sclerosis (MS). The symptoms of the individual Leukodystrophies may vary because of the differences in their genetic cause. Whereas Leukodystrophies are generally caused by a defect in one of the genes involved with the growth or maintenance of the myelin, MS is thought to be caused by an attack on the myelin by the body’s own immune system.
VWM/CACH is a recessive genetic disease, which occurs when both parents are carriers and each contributes an allele to the embryo. In other words, the individual diagnosed with it inherited one copy of the 'bad' gene from each parent, who also inherited the gene from one of their own parents. There is a 50:50 chance that any carrier will pass on either the good or the bad version to their children. Because the disease is so rare, it means that several people in a family can be carriers without ever knowing it. Once the genetic diagnosis is made it may be possible to screen family member who wishes to know if they are a carrier.
While there is research currently being conducted, there are no treatment options available for VWM disease. Prof. Orna Elroy-Stein from Tel-Aviv University is one of the few researchers in the world studying VWM disease. She is focused on research surrounding VWM disease and finding a cure for VWM. The process of finding a cure is very tedious and expensive, and consists of several phases. Scientific research is like putting together a huge jigsaw puzzle. It takes months and sometimes years. Currently however, there is no major funding dedicated to VWM research. Without funding there is no hope for a cure, which is why I’ve decided to stop waiting for an answer and put matters into my own hands. I can’t be a scientist finding a cure but I can be citizen scientist, so this is what I have to do, raise the money to fund the science. It’s what we all have to do. We don’t have a choice! We’re doing this not just for my niece, but for all kids with VWM. We’re at war with this disease. My dream is be that science and research makes VWM a condition people can live with. I want everyone with VWM to have the ability to live a full, meaningful life. That would be a gift!
Last year, July 2013, my sister and I organized “Find a Cure for VWM” research fundraiser which was a huge success. The success of the fundraiser would not have been possible without help of ALL our friends, family, the entire community and our VWM Dream Team! We all put our hearts and hard work into raising funds needed to "Find a Cure for VWM”!
If you are interested in making a tax-deductible donation to support Prof. Orna Elroy –Stein VWM research, you can do so through AFTAU. Make your check payable to: American Friends of Tel Aviv University (AFTAU) and mail to: 9715 Woods Drive # 1608Skokie, IL 60077-4447 Attn: Rick Kruger/Orna Elroy-Stein research for VWM. In advance, thank you for your support !