KIF1A.ORG Funds Expanded Natural History and Endpoint Enabling Study at The Chung Lab to Strengthen Clinical Development of KAND Therapeutics

NEW YORK, Feb. 28, 2022 /PRNewswire/ -- KIF1A.ORG, Inc., a 501(c)(3) nonprofit organization dedicated to development of therapeutics for KIF1A Associated Neurological Disorder (KAND), announced today increased funding to discover and develop disease-modifying therapeutics for KAND.

The KAND Natural History Study was launched in 2017 in collaboration with the Chung Lab at Columbia University, and led by Wendy Chung, MD, PhD. KIF1A.ORG will increase funding in 2022 to initiate the KOALA Study (KIF1A Outcome measures, Assessments, Longitudinal And endpoints Study). In addition to expanding the natural history of KAND, this study will investigate KIF1A-appropriate baseline and longitudinal assessments through known and validated instruments and scales. The KOALA Study will identify standardized assessments and measures to be used in clinical development of KAND therapeutics. Findings and data generated from the KOALA Study will be made freely available to the scientific community.

KIF1A Associated Neurological Disorder (KAND) is a rare neurodegenerative condition caused by variants in KIF1A, a gene that encodes an anterograde neuronal microtubule motor protein. People affected by KAND experience cognitive impairment; cerebellar atrophy; ataxia; spastic paraplegia; optic nerve atrophy; cortical vision impairment; peripheral neuropathy and epilepsy. Most KAND patients require 24/7 supervision and care. There are currently no approved treatments for KAND.

"KIF1A.ORG's continued collaboration with the Chung Lab is vital for the scientific community to understand and implement standardized endpoints for KAND clinical trials. The goal of the KOALA Study, and all KIF1A.ORG initiatives, is to enable rapid research and development opportunities for KIF1A Associated Neurological Disorder," said Luke Rosen, Founder of KIF1A.ORG. "Every day matters for KAND families, and our community is eager to collaborate with groups that work with extreme focus and urgency. The KOALA Study is one of many initiatives to accelerate patient-focused protocol design, and to rapidly enroll clinical trials. Our call to action is for industry partners to utilize this critical study, and to power efficient therapeutic development for KAND."

Building upon the existing natural history, severity scale and genotype/phenotype study (Boyle et al., 2021), the Chung Lab will further investigate and measure multiple clinical domains including motor, cognition, epilepsy and vision in patients living with KIF1A Associated Neurological Disorder.

"Every individual with KAND is unique, and the KOALA Study will allow us to assess changes that happen over time so we can look for a change in trajectory with a treatment."
-Wendy Chung, MD, PhD., Kennedy Family Professor of Pediatrics and Medicine and Chief, Clinical Genetics at Columbia University

About KIF1A.ORG: Formed in 2017, KIF1A.ORG exists to improve the quality of life for families affected by KIF1A Associated Neurological Disorder (KAND), and to relentlessly drive research initiatives leading to treatments and cures. Our mission is to accelerate research, and rapidly support development of therapeutics for this generation of KAND patients.

In addition to funding from KIF1A.ORG, this study is made possible through support from NIH and other generous collaborators.

CONTACT:
Kathryn Atchley, President, KIF1A.ORG, Inc.
Email: [email protected]

SOURCE KIF1A.ORG