The undruggables: Collaboration aims to uncover new therapies for neurodegenerative disorders

Biopharmaceutical company PhoreMost has entered into a research collaboration with the laboratory of Professor David Rubinsztein at the University of Cambridge.

Professor Rubinsztein is professor of Molecular Neurogenetics and deputy director of the Cambridge Institute for Medical Research. He also holds the appointment of lead academic scientist of the Alzheimer’s Research UK Cambridge Drug Discovery Institute.

PhoreMost, which is focused on drugging the ‘undruggable’ will be studying with Professor Rubinsztein, translating essential cellular mechanisms of neurodegenerative disorders into novel therapeutic strategies for diseases such as Parkinson’s and Alzheimer’s.

Professor Rubinsztein’s laboratory is a world leader in cell autophagy, which is a natural pathway that monitors and degrades defective proteins in healthy cells. Therapeutic regulation of this pathway has the promise to reverse or prevent the deposition of aggregation-prone proteins in nerve cells that lead to neurodegenerative diseases.

Unfortunately, few druggable targets have yet been discovered that modulate the autophagy pathway. To address this, PhoreMost will work with autophagy models from Professor Rubinsztein’s lab.

The company will use its SiteSeeker technology, which is able to identify druggable sites on targets. The technology will screen for hidden targets and drug sites that are the most relevant for treating neurodegenerative disorders. The studies could potentially reveal novel insights for previously unknown autophagy signaling components, which could suggest innovative starting points for new drug treatments.

Dr Chris Torrance, CEO of PhoreMost said: “We are very excited to be joining forces with Professor Rubinsztein, whose expert insights into neurodegenerative diseases will enable PhoreMost to expand into this important yet therapeutically unmet disease area of aging, and thereby complement our growing oncology discovery pipeline. This collaboration with the Rubinsztein research team fits our scientific and corporate mantra: find and drug the best disease targets, even if they are currently considered impossible, and engage closely with experts to solve the problem.”

Professor Rubinsztein said: “Autophagy is a fundamental protein quality control process in cells. It has the potential to significantly impact several neurodegenerative disorders, if we can recruit it to degrade intracellular, disease-causing, aggregate-prone proteins in the brain.  Combing our cutting-edge research and cell models in this area with innovative techniques like SiteSeeker will enable us to exploit the full depth of targetable disease biology that is present in this pathway.”