Breakthrough mitochondrial-based therapies for neurological diseases.
Imagine a world in which doctors can diagnose and treat patients before the tremors, the dementia, or the seizures from neurological diseases like Rett Syndrome, Alzheimer’s, and Parkinson’s take control. This is a world that Lucy Therapeutics is working to realize.
For Amy Ripka, the company’s founder, any significant progress in developing treatments for neurological diseases will require non-traditional methods. The siloed manner through which disease targets are normally identified is potentially why such treatments remain elusive, even after decades of research. And it’s why Ripka is taking another approach — she is selecting drug targets based on a deep understanding of the crossover chemical and biological interplay at work in these diseases.
This strategy led Ripka to a therapeutic target that mirrors the complexities of the diseases themselves: the mitochondria. She has linked neurodegenerative disease to dysfunctional mitochondria in neurons and is pioneering a new class of treatments designed to address such dysfunction.
The insights underlying Lucy Therapeutics’ drug discovery platform give it a substantial advantage, especially when one considers the neuroscience industry’s limited success identifying targets. These insights also unlock the door to a biomarker that would enable early, pre-symptomatic diagnosis.
Ripka’s approach requires her to read exhaustively beyond her field and to understand how to apply her disparate insights in an integrated fashion. Finding such a cross-functional scientist is not common; finding one who is pioneering potential curative treatments is downright rare.
Training and continued performance work as a classical violinist provided Ripka with an unusual basis for her scientific perspective. “As a professional violinist, you are routinely asked to read extremely complicated music with very little rehearsal,” she notes. “To be effective you must be able to simultaneously play the notes, interpret all bowing and dynamic instructions, monitor the conductor’s gestures, deal with unforeseen events on stage, as well as produce a sound which blends with your colleagues. And do all of this in a matter of microseconds.” Ripka continues, “Learning how to do this well allowed me to see the value of using this same approach of integration in the neuroscience field.”
Ripka has deep experience in the complementary realms of big pharma, biotech, and contract research organizations. After receiving a PhD in chemistry from the University of Wisconsin-Madison, she went on to work in the lab of Nobel Laureate K. Barry Sharpless at The Scripps Research Institute, followed by a career that included time at Bristol-Myers Squibb, EnVivo Pharmaceuticals, and WuXi AppTec, among others.
This variety of professional roles taught Ripka to approach the traditional drug discovery process in an untraditional way. She saw the inefficiencies in today’s discovery pathways first hand — the work spent pursuing a solution without the realization that a scientist in a similar, but seemingly unrelated field had already trod that ground and gained valuable data. Ripka notes that these pathways lack “cross-fertilization,” or the interdisciplinary flow of knowledge in pursuit of a singular goal.
Similar to the web of neurons that they impair, neurological diseases like Parkinson’s, Alzheimer’s, and Rett Syndrome are complex and mysterious things. Ripka’s insights into the root of these illnesses will guide the team at Lucy Therapeutics to lay bare such mysteries and reveal treatments that will fundamentally change our relationships with some of our most devastating and unpredictable diseases.