C-Path supports innovative ALS, neurodegenerative disease research with new grant

Critical Path Institute's (C-Path) Translational Therapeutics Accelerator (TRxA) announced today a new research grant awarded to Daniela C. Zarnescu, Ph.D., Professor of Cellular and Molecular Physiology at Penn State University College of Medicine, in collaboration with Jon Njardarson, Ph.D., Professor at the University of Arizona. This $250,000 grant will support their innovative project titled "Novel Small Molecules with Neuroprotective Potential in ALS and Related Neurodegenerative Diseases."

Neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), occur when nerve cells in the brain or peripheral nervous system progressively lose structure and/or function and ultimately die. Significant challenges remain in research and drug development for rare neurodegenerative diseases and advances are needed to improve the lives of those affected. 

Dr. Zarnescu aims to identify and develop new small molecules that have the potential to protect nerve cells from degeneration. In response to receiving the grant, she expressed her enthusiasm, "I am honored to receive this grant from TRxA, alongside my collaborator Dr. Jon Njardarson. Together, we will explore new avenues in neuroprotective therapies for ALS and related diseases. This funding will accelerate our efforts to discover novel treatments that can impact the lives of patients living with these challenging conditions."

TRxA Executive Director, Maaike Everts, Ph.D., commented on the significance of the grant, "We are thrilled to support Dr. Zarnescu and Dr. Njardason's pioneering research through TRxA. Their collaborative approach aligns with C-Path's mission to accelerate the development of therapies for devastating diseases like ALS. This grant underscores our commitment to advance scientific discovery that can translate into tangible benefits for patients."

The grant is consistent with C-Path's broader commitment to foster collaboration among scientists and stakeholders by expediting the development of safe and effective therapies in neurodegenerative diseases. This is complimentary to C-Path's Critical Path for Rare Neurodegenerative Diseases Consortium (CP-RND) which is focused on increasing the field's understanding of disease pathogenesis and natural history by quantifying disease progression. These efforts include evaluating potential biomarkers from patient-level data and optimizing clinical trial design, to improve the efficiency and success of drug development.

Dr. Zarnescu's TRxA-funded project will benefit from interactions with CP-RND, since thinking through the translational and regulatory path of a potential new drug ensures early-stage drug discovery efforts are as effective as possible.

Through programs like TRxA and CP-RND, C-Path continues to play a vital role in bridging the gap between scientific innovation and patient impact.

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