The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has granted a 2018 Therapeutic Pipeline Award to Progenra to support the company’s development of small molecule treatment candidates for Parkinson’s disease.
“Progenra is pleased to have been selected by The Michael J. Fox Foundation to contribute to bringing Parkinson’s and other major neurodegenerative diseases under control by introducing new therapeutic agents,” Tauseef Butt, Progenra’s president and CEO, said in a press release.
The company’s goal is to find new medicines that target enzymes in the ubiquitin proteasome system (UPS). The UPS has been implicated in neurodegenerative diseases, based on the observation of protein deposits tagged with ubiquitin — a marker for degradation in cellular structures called proteasomes — in affected neurons.
Research has shown that impaired UPS function, due to the accumulation of aggregation-prone proteins, delays the degradation of substrates important in cellular processes such as signaling and apoptosis — which refers to “programmed” cell death, rather than death caused by injury — ultimately leading to neurodegeneration.
“We believe that augmenting the activity of a native enzyme known to be beneficial in combating neurodegeneration will provide both mechanistic information and the potential for breakthrough treatment,” Butt stated. “We look forward to working with the many excellent researchers in the foundation’s consortium.”
MJFF’s Therapeutic Pipeline Program grants awards to projects of clinical utility for patients and to proposals believed to have the potential to alter disease course and/or significantly improve treatment over the current standards of care.
Both industry and academic investigators may apply by proposing new strategies or clinically safe therapies used in other diseases. The program covers development from preclinical studies to clinical trials, which includes pharmacological and non-pharmacological approaches such as gene therapy, biological, surgical, and non-invasive methods.
In addition, MJFF’s Target Advancement Program aims to overcome the need for well-validated targets in the disease process. It seeks to foster critical target validation studies that may ultimately speed up subsequent therapy development. The program also supports continuing work on established targets — those already showing links to the disease in patients.