Team Charlie attended the 2024 Batten Disease Research Conference and met the researchers behind Charlie's CLN1 gene therapy.
Rare disease is a hard business, and for patients and families, a hard life. But it’s not impossible, and it’s not hopeless. There are many tireless scientists, providers, and families working towards the common goal of making the world a better place for rare disease children. Over the last few days, we saw that in action.
This week, Team Charlie sponsored and attended the 2024 NCL (Batten Disease) International Research Conference in Chicago. The event, organized by Our Promise To Nicholas Foundation, was attended by more than 100 of the top Batten Disease researchers, scientists, and healthcare providers from around the world. Also in attendance was Congressman Bill Foster, who came to learn more about Batten Disease from the best in the world.
Charlie even made an appearance at the conference, where he was finally able to meet Sharon King of Taylor's Tale: Fighting Batten Disease, and Dr. Steven Gray, who initiated and developed the only gene therapy that has been developed for the ultra-rare CLN1 variant of Batten Disease. While Sharon lost her beloved Taylor in 2018, before the therapy development could be completed, she pressed on so that other children could someday benefit from the tireless work that went into developing the CLN1 gene therapy.
Last year, the hard work of Taylor's Tale and Dr. Gray finally went from theoretical to reality when Dr. Elizabeth Berry-Kravis of Rush University Medical Center, who along with her research director Kendall Robbins, skipped countless nights of sleep to formulate and submit an application for experimental approval to the FDA. Just months later, the FDA approved Charlie to become the first human ever dosed with a gene therapy for CLN1, which was delivered into his spine and brain. Of course, we knew it was risky being the first, but so too did we know the consequences of not trying.
Less than a year later, Charlie received emergency approval from the FDA to receive the same CLN1 gene therapy injected into the eye, a remarkable feat of unprecedented speed, brought about by the determined work of Dr. Elizabeth Berry-Kravis, who presented an innovative method for obtaining emergency FDA approval by presenting data from a different disease as the model for dosing and delivery.
While the FDA has not yet approved the CLN1 gene therapy for clinical use, we are hopeful that Charlie will pave the way for eventual full FDA approval, so that more children with this devastating disease can someday receive treatment.
Much love and gratitude to everyone who has stood by us on this journey, and more importantly, Charlie.
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