C-Path grant aids work into way of protecting nerve cells in ALS
Goal is to move molecules developed by university scientists into clinical tests
A researcher at Penn State University has received a $250,000 grant for an innovative project that seeks to identify and develop small molecules with the potential to protect nerve cells in neurodegenerative conditions such as amyotrophic lateral sclerosis (ALS).
The grant was awarded by the nonprofit Critical Path Institute (C-Path), through its Translational Therapeutics Accelerator program (TRxA), which seeks to advance the movement of discoveries made in an academic environment to the clinic. The funding will cover direct and indirect project costs for up to one year.
Titled “Novel Small Molecules with Neuroprotective Potential in ALS and Related Neurodegenerative Diseases,” the work will be led by Daniela C. Zarnescu, PhD, at Penn State University, in collaboration with Jon Njardarson, PhD, at the University of Arizona.
Penn State scientist focused on mechanisms of nerve cell degeneration
“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,” Zarnescu, a professor in the department of cellular and molecular physiology, said in a C-Path press release. “This funding will accelerate our efforts to discover novel treatments that can impact the lives of patients living with these challenging conditions.”
ALS is marked by the progressive damage and death of motor neurons, the nerve cells that control voluntary movements. This nerve degeneration leads to a progressive loss of muscle control, a hallmark disease symptom.
While there are some approved treatments for ALS, their ability to slow progression and extend survival is still very limited. Advances are needed to improve patients’ lives and outcomes.
Zarnescu’s work has focused on understanding the mechanisms that drive the degeneration of nerve cells, using the fruit fly as an ALS model. Meanwhile, Njardarson specializes in finding new chemical strategies to create molecules with unique biological activities. By joining efforts, the scientists seek to develop therapies that may address those unmet needs in ALS and potentially in other neurodegenerative conditions.
“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,” said Maikee Everts, PhD, executive director of TRxA.
Academic discoveries often don’t move into advanced therapy testing
Currently, most molecules with therapeutic potential discovered in academia fail to advance into preclinical and clinical testing, a process called translational science. This gap frequently is referred to as the “valley of death,” according to C-Path, a public-private partnership established in 2005.
C-Path’s mission is to lead and foster collaborations that advance potential treatments to the next stage of development, with the goal of creating better therapies for people.
The nonprofit’s TRxA program supports scientists in the early stages of drug development, helping them with funding and in defining optimal approaches for taking cutting-edge therapies from the lab to patients.
Zarnescu’s project, in particular, will benefit from interactions with C-Path’s Critical Path for Rare Neurodegenerative Diseases Consortium, which aims to boost the understanding of neurodegenerative conditions by quantifying disease progression.
Together, scientists will brainstorm on future clinical studies and on regulatory paths, should a candidate therapy show promise in the early studies.
“This grant underscores our commitment to advance scientific discovery that can translate into tangible benefits for patients,” Everts said.
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