Caraway Receives MJFF Grant to Explore New Therapeutic Strategy for Parkinson’s

Caraway Therapeutics has been awarded a research grant from The Michael J. Fox Foundation to further investigate a lysosomal ion channel that could be key in developing a new class of therapeutics for Parkinson’s disease.

“Receiving this grant from The Michael J. Fox Foundation is extremely validating for our science, and we are honored to work with the organization,” Martin Williams, CEO of Caraway Therapeutics, said in a press release. “Our unique drug discovery platform, electrophysiology capabilities and expertise in lysosomal function will allow us to rapidly advance this program towards the clinic.”

Parkinson’s disease is characterized by the loss of neurons, or nerve cells, that produce the neurotransmitter dopamine — a chemical “messenger” that is involved, among other things, in the control of movement.

Although the specific causes of Parkinson’s are still not fully understood, genetic factors have been described as important contributors to the onset of the disease.

Defects in protein degradation inside lysosomes — cell compartments responsible for digesting and recycling different types of molecules — have been implicated in Parkinson’s development and progression.

Caraway’s targeted approaches combine genetic data and unique biological understanding to discover small molecules that activate neuronal autophagy — the mechanism by which cells remove unnecessary or dysfunctional components — and lysosomal function, thereby accelerating the clearance of toxic materials and defective cellular components.

Rare genetic variants in the TMEM175 gene, which provides instructions for making a lysosomal ion channel, have been linked to both Parkinson’s incidence and age of disease onset. While decreased TMEM175 function reduces lysosomal efficiency in neurons, overexpression (higher-than-normal levels) of TMEM175 promotes neuronal health.

“Our approach is to activate this potassium channel to restore lysosomal function. Lysosomal dysfunction is emerging as a driver of [Parkinson’s] pathology in both familial and idiopathic forms of disease. We believe that TMEM175 agonists could be an important way to alleviate this dysfunction,” said Magdalene Moran, PhD, Caraway’s chief scientific officer.

With this new grant, Caraway will be able to further investigate the role of TMEM175 in cellular models of disease and evaluate the utility of TMEM175 agonists as therapeutics for Parkinson’s disease. (Agonists are compounds that bind to a receptor and activate it, mimicking a biological response.)

“We are glad to support Caraway’s investigation of TMEM175 as a novel therapeutic target toward our goal of a world without Parkinson’s,” said Marco Baptista, PhD, vice president of research programs at The Michael J. Fox Foundation.