Join together with other Jewish families and help save children's lives.
| | | | | This Hanukkah, I’m hoping that you’ll help me make a miracle happen. You may have seen the article about my family in the Forward. I’m Samantha, and I need your help to save my sister’s life.
Lindsay was born with Canavan Disease, highly prevalent in Ashkenazi Jews. In fact, one out of 40 carry this disease, but carriers don’t display symptoms. My parents, both carriers, had no idea. Canavan children can’t walk or talk. They are severely handicapped, and face early death.
Predicted not to live past 5, Lindsay was the first person in the world treated with gene therapy for a brain disease in 1996, making scientific history, thanks to the tireless work of the Canavan Research Foundation and terrific scientists.
Since then, 14 children have been treated with gene therapy. In long term follow up, the treatment was shown to create myelin in the brain and decrease atrophy, a scientific miracle.
The benchmark Canavan gene therapy has been widely cited and implemented worldwide by other investigators. It has informed trials all over the world on other neurodegenerative diseases, resulting in the first gene therapy for Parkinson’s disease, among others.
Two decades later, Lindsay is still fighting. The little girl they said should have died long ago defied all odds- Lindsay just turned 25. But she still cannot walk, or talk, or light the Hanukkah candles. Last year, she almost died. But she defiantly persists. I think that she has stuck around because she knows that we are fighting as hard as we can to cure her. | |  | | Yoel and Yael Ilinestky of San Francisco, twins who suffer from Canavan Disease. | | | Lindsay is not the only Canavan child. Every week, I hear from another parent with a newly diagnosed child. Their voices in agony, they always have the same question- is there hope?
Thanks to the work of the Canavan Research Foundation and the scientists we’ve supported, I’m proud to say there is. After 25 years of research, the scientific team has finally engineered a new gene therapy treatment that has been proven to cure Canavan disease in animal models. Next year, 10 children are slated to receive the gene therapy, and after that, it will become available to every Canavan child– but it won’t happen without your help.
The gene therapy incurs staggering costs. We need to raise another 1.6 million dollars by the springtime to pay for the trial. If every person receiving this email chipped in $20, we would be able to pay for the gene therapy entirely.
We are also looking for a few good people who care about bringing medical science to a new place who would be interested in learning more - please email me directly at skarlin@canavan.org. You can meet more Canavan families in this video.
As you light the Hanukkah candles this year, can I count on you to help me bring about a miracle?
Thank you for your generosity, and warmest holiday wishes!
Samantha Karlin
Canavan Research Foundation
www.canavan.org
skarlin@canavan.org | | | | | | | |
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