We are all familiar with the Talmudic saying, “Whoever saves a life, it is as if he has saved an entire world.” Sometimes, when we least expect it, that quote takes on new meaning.
Almost 30 years ago, Prof. Zohar Argov, Senior Physician in the Department of Neurology, and Prof. Stella Mitrani-Rosenbaum PhD, of Hadassah’s Goldyne Savad Institute of Gene Therapy, identified the genetic defect responsible for Hereditary Inclusion Body Myopathy (HIBM). HIBM is an incurable disease that affects Jews of Persian origin – and, it turns out, many others.
Little could Hadassah professors have imagined that their discovery would have implications for people among other ethnic groups and in countries as far away as Japan.
HIBM causes progressive muscle degeneration, which eventually leads to general disability and even total paralysis of the voluntary muscles. This orphan disease is so rare that medical professionals often can’t identify it since they are not familiar with the symptoms. Between 5 and 10 percent of the Persian community is thought to carry the defective gene.
“The condition was first identified in an ethnic cluster of Persian Jews, but we now know that the disease is a world-wide condition,” Prof. Argov says. “After we discovered the defective gene, suddenly patients with the same disease were identified in other countries and other communities around the world – especially in Japan.
“Previously, it was thought that the Japanese patients suffered from a different disorder. But when Hadassah discovered the gene defect, it became clear that these Japanese individuals suffered from the same condition, but with a different cluster of mutations.
“The Persian Jewish community of Los Angeles , especially the Malka and Haifa Chapters of Hadassah, has supported our HIBM research for many years,” Prof. Argov said. “Recently,” he continued “a young LA doctor unfortunately learned about the disease firsthand and decided to establish a foundation – the Neuromuscular Disease Foundation – to help support research.”
Last week, Hadassah Southern California’s Haifa and Malka Chapters and the Neuromuscular Disease Foundation joined forces to sponsor a special event to raise funds for the cure of Hereditary Inclusion Body Myopathy. The exciting cocktail and dinner event included a colorful fashion show and live entertainment, in true Hollywood style.
“This discovery is a classic example of the global benefit of Hadassah research,” said Prof. Shlomo Mor-Yosef to the more than 700 people present. “A single patient turns to Hadassah with a problem and our researchers work tirelessly to find a solution. In this case our solution helps the Iranian Jews who initially turned to us as well as Japanese patients and others around the world.”
“Today, thanks to Hadassah researchers’ discovery and the generous support of donors, we are able to prevent HIBM by a simple test,” Prof. Mor-Yosef said. “This is the power of research, the power of Hadassah.”
Currently, Hadassah’s team, including Prof. Argov and Dr. Mitrani-Rosenbaum, is embarking on an attempt to treat the condition using ‘virus mediated’ gene therapy, while they continue to investigate the mechanism of the disease and search for a cure.
Now, nearly three decades after the initial discovery, young couples can take advantage of diagnostic testing and genetic counseling – freeing themselves and their unborn children from the anguish of HIBM.
Looking to save just one life, Hadassah research has made it possible to save – if not an entire world – at least a small portion of its people. None of this would have happened without the intense involvement of Hadassah’s dedicated California volunteers. Last week’s gala evening was just the latest success in their tireless effort to involve people in supporting the cause. Coming together as a community – and as Hadassah – they continue to make a difference in peoples’ lives.
This is the special quality that connects Hadassah in Jerusalem with Hadassah in Los Angeles – and with people in far flung places, who didn’t even know what Hadassah is, let alone what we do.