Camps & education | First-grade best friends raise $1 million for rare disease

A fundraising campaign started quietly by two first-graders two years ago to help find a cure for a rare genetic disease recently passed the $1 million mark, with donations streaming in from across the United States and around the world.

The million-dollar achievement has been celebrated in the media as the perfect feel-good story, but the trigger for this global effort was a somber diagnosis at the birth of Jonah Pournazarian.

The playful, redheaded youngster was born weighing 4 pounds and with an extremely rare metabolic malfunction known as Glycogen Storage Disease, or GSD.

GSD predominantly afflicts children, targeting different parts of the body. Jonah’s case is identified as Type 1b, in which glucose is stored in the liver and “can’t get out,” says his doctor. (Type 1a of the disease affects mainly Ashkenazi Jewish kids.)

Because Type 1b of GSD is so rare — fewer than 100 children in the United States have been identified as having it — medical researchers and potential grant givers have long ignored it.

One 6-year-old wasn’t willing to do that, however.

Dylan Siegel (left) and Jonah Pournazarian  photo/courtesy david siegel

Dylan Siegel, now 8, was Jonah’s best friend at the Wise School, affiliated with Stephen Wise Temple, a large Reform congregation in West Los Angeles. And Dylan wanted to do something to help.

When he heard his mother talk about an effort to raise money among temple members to support the work of a leading GSD researcher, Dylan said he, too, wanted to give some money.

As his mom, Debra Siegel, recalls, “I suggested to Dylan that he set up a lemonade stand, but he said he wanted to write a book.” She took her son’s plan as a childhood fantasy, but the next day Dylan presented his parents with the finished product.

The cover of the richly illustrated 14-page booklet reads “Chocolate Bar by Dylan Siegel,” and the tone is set in the first entry, which reads, “I like to go to Disney Land. That is so Chocolate Bar.”

Other “Chocolate Bar” (read “awesome”) experiences recalled by the young author-illustrator include going to the swimming pool, aquarium and bowling alley, ending with: “I like to help my friends, that is the biggest Chocolate Bar.”

After completing the book, Dylan kept pushing the project, drafting his marketing-consultant father for the production phase, with an initial print run of 200 copies.

At the temple’s Mitzvah Day, the two boys and their parents sold enough autographed books (at $20 each) and $5 chocolate bars (donated by a neighborhood market) to raise about $7,000.

Augmenting the sales force were the boys’ two teachers, Orlee Raymond and Kimberly Snyder, sporting T-shirts that read “1st Grade Is So Chocolate Bar.”

In late 2012, the L.A. Jewish Journal ran an article about Jonah and Dylan and their mission to help find a cure for GSD. The article came to the attention of an NBC producer, who asked Chelsea Clinton, then doing feature segments for the network, to look into the story. She did, and the story aired on the national NBC evening news a short time later.

Amid the media’s generally gloomy string of disaster news, the “Chocolate Bar” segment resonated with viewers. Other major TV networks, newspapers and social media spread the story across the globe, and the results have been spectacular.

By early last month, more than 25,000 “Chocolate Bar” books had been sold, according to David Siegel, Dylan’s father and pro bono coordinator of the project. Donations have come from all 50 states and from 60 countries across the globe. Overseas, letters and money came from as far away as India, Kuwait, Nigeria, Slovakia, Mongolia, United Arab Emirates and Thailand.

Every dollar supports the GSD research of Dr. David A. Weinstein, initially at the Harvard Medical School and now at the University of Florida in Gainesville, where he directs the largest GSD treatment and research program in the world.

The disease was almost always fatal until 1971, when researchers developed the first effective therapy for GSD. A major breakthrough came a decade later with the discovery of a simple procedure of cornstarch injected through a surgically implanted feeding tube. However, the prescribed doses had to be administered every three hours; missing just one dose could lead to a hospital stay or even death.

Weinstein’s research, entirely underwritten by the “Chocolate Bar” campaign, has been able to extend the intervals between feedings, and his aim is to give his patients (and their parents) full nights of uninterrupted sleep.

In the long run, Weinstein is looking toward gene therapy as a cure, something that has been successful in dogs. He hopes to start trials on humans as soon as the federal Food and Drug Administration gives the green light.

Weinstein, who visits Israel frequently on a collaborative project at Sheba Hospital, is upbeat about Jonah’s future. “Our treatment is working,” he said,  “and I expect Jonah to do very well.”

To learn more about the campaign, see www.chocolatebarbook.com.

Tom Tugend

JTA Los Angeles correspondent