What is WAS
Wiskott-Aldrich Syndrome (WAS) is a very rare life-threatening genetic disorder that affects mostly male infants. Approximatly four out of one million male infants worldwide are diadgnosed with WAS (e.g. less than 20 in Israel and less than 30,000 patients worldwide). WAS affects the immune system, as well as the platelets, which are the blood cells that prevent excessive bleeding.
WAS is a children disease, since without treatment the average life expectancy is less than 5 years. We believe that each and every one of our WAS boys all over the world has the right to live normal and meaningful life without having the daily fear of bleeding or getting infections. Currently, the only approved treatment that may lead to complete recovery is Bone Marrow Transplantation (BMT). BMT is a complicated and dangerous process. Mortality rate is 10-15% and about 30% of the treated patients get complications related to the BMT process. In many countries BMT is not available for the children since a suitable BMT donor was not found. From the early 2000’s, gene therapy has emerged as a potential cure, and several clinical trials are in progress in Europe and in U.S. However, leukemia has been discovered in children who took part in the trials so far, leading to the conclusion that this treatment approach is not safe yet.