Park Slope boy with incurable disease helps raise awareness for ‘World Rare Disease Day’

Park Slope resident Zach Becker loves “Star Wars” and superheroes just like any other nine-year old. However, for much of his young life, Zach has been living with a rare and incurable disease that has kept him in and out of hospitals.  

Zach is planning to participate in a special awareness event at Carnegie Hall, which just happens to fall on the rarest day of the year: leap day, Saturday, Feb. 29. It honors the 25-30 million Americans currently struggling to get accurate disease diagnoses and treatments.

The event is open to the public and will highlight patient artists who raise awareness of their disease through their artwork. The Carnegie Hall event will be hosted by Rocket Pharmaceuticals, a clinical-stage company advancing genetic therapies for rare childhood disorders, in partnership with the RTW Charitable Foundation.

The theme of the event is “I am rare, hear me roar” to highlight the Roaring ‘20s, which was a decade that saw a lot of advancements in science and medicine. There is hope that the 2020s will be similar, with the potential for gene therapies to cure rare genetic diseases.

Zach was diagnosed at the age of five with a rare blood disorder called Fanconi anemia that can result in bone marrow failure and organ defects, and increase the risk of certain cancers.

In 2017, Zach had a bone marrow transplant surgery at Memorial Sloan Kettering Cancer Center, where the staff wkept his spirits up through his love for “Star Wars.” But, unfortunately, this isn’t the end — Zach’s disease is life-shortening and currently has no cure.

“Our son, Zach, was diagnosed with Fanconi anemia in August 2015,” Zach’s father Adam Becker told this paper. “The simplest way to describe Fanconi anemia is as a DNA repair issue; our cells all die and get replaced every day, but for people with Fanconi, the new cells don’t copy over correctly. That presents some short-term and long-term problems.”

Becker continued, “In the short-term, often before age 10, bone marrow often fails, leaving one with anemia and a compromised immune system. If this symptom can be cured with steroids or a grueling bone marrow transplant, then the individual is still left with the long-term prospect of cancer. Individuals with Fanconi anemia are hundreds of times more likely than the rest of us to develop cancer, and most get it in their 20s and 30s,” he added.

That’s why experts at Rocket Pharmaceuticals, headquartered in the Empire State Building — which, for the second year in a row, will be illuminated in pink, blue, purple and green for “World Rare Disease Day” — are working to develop what could be a first-of-its-kind therapy for Fanconi and other devastating rare diseases. The company hopes to connect patients to world-renowned disease experts.

According to Becker, there is hope on the horizon. “The good news is that thanks to financial support for organizations like Fanconi Anemia Research Fund and Be the Match, and hospitals with Fanconi anemia expertise like Memorial Sloan-Kettering Cancer Center, University of Minnesota and Cincinnati, bone marrow transplant success rates for Fanconi have risen from roughly 50 percent to more than 80 percent.

“That means that for the first time, we’re starting to see more children with Fanconi grow into young adults,” said Becker. “Our motto is, “Alone we are rare, together we are strong.”