ATLANTA — A Bar Mitzvah is a coming of age celebration, and while most kids look forward to getting some pocket money on their big day, an Atlanta boy is asking for donations to support research for his sister’s debilitating genetic disease.
Natanel Gold’s sister, 11-year-old Shaindy, loves music, pancakes and ice cream. She also has Mucolipidosis Type IV (ML4), a rare neurological disease that creates debilitating delays in childhood motor and mental skill development. Right now, there is no treatment and no cure.
Shaindy was diagnosed with ML4 when she was just 18 months old, Natanel posted on Fundrazr. He has already raised over $75,000 in her honor.
“Shaindy is unable to walk or talk, her brain works differently than hours, she is almost blind, and she will have a shortened life span,” 13-year-old Natanel wrote on his fundraising page. “Instead of giving me a gift for my Bar-Mitzvah, it would mean much more to me if you would please make a generous donation to the ML4 Foundation to fund ongoing ML4 gene therapy research.”
Randy Gold, Natanel and Shaindy’s father, said the family has been actively raising money for ML4 research for years.
“At a very young age as Natanel saw my wife Caroline and I become catalysts for research to find a cure for ML4, he recognized that each person, using their own unique talents, can make a difference for Shaindy and other kids with ML4,” Randy said.
Natanel came up with the idea to start the fundraiser for Shaindy while preparing for his Bar Mitzvah last year. Invitations to his party included a card asking for donations in lieu of gifts.
In the card, Natanel wrote that Shaindy is “the most special person in the world to me, and hearing her laugh makes me happy. It’s a blessing to be her only brother and protect and pray for her every day.”
“Natanel delivered a powerful and moving speech … about his sister … and the importance of friendship. When he was done, the congregation gave him a standing ovation,” Randy said. “I think the ovation sums up what we hope others will take away from Nat’s efforts.”
The National Institute of Health estimates ML4 occurs in one in every 40,000 people – with around two-thirds of those affected having Ashkenazi Jewish ancestry. The disease is so rare that researchers believe ML4 might be due to a single gene deformity – raising the hopes that it can be cured through gene therapy.
Randy said that he and Caroline told Natanel he might be able to raise $5,000, maybe $10,000 at the most. To date, he has raised over $75,000 for ML4 research.
“The response to Nat’s fundraising request continues to remind us how lucky we are to be surrounded by so many wonderful people,” Randy said. “We were particularly touched by the number of people we don’t even know who made donations when they saw the post on Facebook. The response from people we didn’t know impressed upon Nat that there are so many caring and generous people in the world.”
Eleven-year-old Shaindy has all the characteristics of children with the disease, Randy said. She is expected to never be able to walk or talk and has the mental development of an 18-month old child. Most children with ML4 end up going blind by the time they are 12 years old and have a shortened lifespan.
“Shaindy is 11 years old and has all of the characteristics that doctors describe in ML4 kids,” Randy said. “Often parents of children with ML4 notice their child isn’t developing properly or reaching expected milestones after birth. Too often, kids with ML4 are not specifically tested for ML4 and are misdiagnosed as having Cerebral Palsy because of the similarities in the physically expression of the disease.”
All of the money raised by Natanel directly supports treatment-focused research at Massachusetts General Hospital, where Harvard researchers and others are working to bring gene therapy to Shaindy and other children with the disease. There is a lot of promise around a new, FDA-approved drug that helps restore neuron function in the brain – according to the ML4 Foundation.
In celebrating new hope for Shaindy’s future, the Gold family is proud of Natanel, a young man with big dreams to change the world.
“The fundraising success is incredibly important for our ability to improve Shaindy’s life, but to us what is even more important is to understand that everyone regardless of age, religion, etc. can make an impact on the world in positive ways,” Randy said. “There is a lot of good in the world, lots of joy in the world and if we can remember that and hold on to those feelings in dark difficult moments, we can all make the world a better place.”