KIF1A.ORG Accelerates KAND Research at Christodoulou Lab

From KIF1A.ORG November 30, 2020November 30, 2020

KIF1A.ORG Community,

What if KIF1A treatment already existed? It could be on the shelves of your local pharmacy, or a drug created for a related disease like Parkinson’s or epilepsy. Are there FDA-approved treatments for other conditions that could bring relief to KAND patients living with progressive spasticity, uncontrolled seizures or painful neuropathy? We won’t know until we look. KIF1A.ORG is proud to announce our collaboration with the Christodoulou Lab to search for existing drugs that could immediately improve and save the lives of KAND warriors.

You may have heard about the concept of drug repurposing in the news as scientists around the world scrambled to discover treatment for COVID-19. In fact, the first FDA-approved treatment for COVID-19 is a repurposed drug. Or maybe you’ve heard of a success story in the rare disease world, like Dr. David Fajgenbaum, who found an existing drug to treat his own near-fatal rare disease in 2014.

Creating a new drug from scratch requires time and money that we don’t have as we race against the incessant clock of KIF1A. Every day we go without KAND treatment, our children lose skills and abilities they work so hard to gain.

Every day we go without KAND treatment, we risk losing another precious warrior.

Drug repurposing has its clear advantages: saving time and money. We bypass millions of dollars in drug development and years of clinical trials required to build a safety profile. While we continue to work with biotech partners such as Ionis and Ovid on gene-based therapies, our new collaboration with the Christodoulou Lab is the world’s first high-throughput targeted screening of potential repurposed FDA-approved drugs to treat KAND.    

The Christodoulou Lab has long been friends of KIF1A.ORG and actively collaborates with multiple members of the KIF1A.ORG Research Network. Dr. Simran Kaur of the Christodoulou Lab will devote 100% of her time to this KIF1A Drug Repurposing Project over the next year, with additional involvement from Dr. John Christodoulou, Dr. Nicole Van Bergen, and Dr. Alejandro Hidalgo-Gonzalez of Murdoch Children’s Research Institute (MCRI) in Australia. We send our deepest gratitude to each of them for joining our mission to bring treatment to this generation of people affected by KAND.

Learn more about this and other projects on our Funded Projects page. If you’re able, I hope you can support KIF1A.ORG on Giving Tuesday so we can continue to fund these critical projects. Donate to our Facebook fundraiser on December 1, or anytime of the year at kif1a.org/donate.

Relentlessly,
Kathryn Atchley
President, KIF1A.ORG