Nationwide Children’s Hospital, the site of a first-of-its-kind clinical trial in Batten disease, has dedicated its lobby in honor of the Charlotte and Gwenyth Gray Foundation to Cure Batten Disease.
Touted as one of the world’s largest and most comprehensive pediatric academic medical centers, the hospital is in its third year of an ongoing, foundation-funded investigational gene therapy trial for children with late infantile Batten CLN6 disease.
In the open label, Phase 1/2 first-in-human trial (NCT02725580), the adeno-associated virus 9 (AAV9) is used to transport a corrected CNL6 gene to patients with the CLN6 disorder. Through a lumbar puncture, the treatment is delivered once directly into the spinal cord.
The chief objective of the study, which includes 12 children, is to measure the safety and toxicity of the therapy. The foundation is underwriting the participants’ treatments, equipment, and two years of follow-up visits. It’s also funding further research for complementary treatments, particularly for children who have lost significant function.
Located in Columbus, Ohio, Nationwide has been designated a Center of Excellence by the Batten Disease Support and Research Association for its clinical and research history with the disease, and its ability to provide comprehensive care.
“The Gray family’s commitment is an example of their selfless and tireless efforts to help children and their families everywhere who are living with and fighting this unforgiving disease,” Steve Testa, president of the Nationwide Children’s Hospital Foundation, said at the lobby dedication, according to a news release.
Four years ago, sisters Charlotte and Gwenyth, then ages 4 and 2, respectively, were diagnosed with the rare form of the neurodegenerative disorder. Unwilling to accept the prognosis, their parents, Kristen and Gordon, established the foundation in search for a cure.
“As parents of two young children with Batten disease (CLN6), every time we enter the hospital doors and through this passageway, we walk in knowing we’re facing this battle alongside a world-leading team of pediatric neurologists and medical researchers working to fast-forward our mission to eradicate this destructive disease,” said Kristen Kaiser Gray.
“Today we’re celebrating the three-year anniversary of the start of the clinical trials which first began with our daughter Charlotte, and then Gwenyth was treated shortly thereafter,” she said. “There is much work to be done, with an urgent need for more research funding. But we remain hopeful and steadfast in our quest for the cure.”
Gordon Gray is a Hollywood producer whose film credits include “Secretariat,” “Miracle,” “The Rookie,” and “Invincible.” He’s currently producing actress Kate Winslet’s HBO series “Mare of Easttown,” and the movie “Wish List” starring Reese Witherspoon.
To date, the foundation has raised nearly $7 million of its $10-million goal. The capital goes to scientists studying innovations in not only gene therapy, but also small-molecule treatments, regenerative medicine, RNA modulating compounds, repurposed pharmaceuticals, and natural compounds.