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2024 ADDF-Harrington + 2025 Harrington Scholar-Innovator Programs

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John Letterio, MD

John Letterio, MD

Case Western Reserve University

Disease Areas

Immunology, Neuroscience, Oncology


Advance a First-in-Class Small Molecule Therapeutic that Inhibits an Enzymatic Regulator of Pathways Essential for the Growth and Spread of Cancer

Scholar Profile

2017 Harrington Scholar-Innovator

The enzyme cyclin dependent kinase 5 (Cdk5) is very active in cancer cells, enabling them to evade detection by the immune system, resist the effects of chemotherapy drugs and spread throughout the body. The Letterio lab has developed MDX01, a highly specific inhibitor that disrupts distinct molecular interactions required for Cdk5 activation and function.

With support from Harrington Discovery Institute, the Letterio lab will advance studies of MDX01 through the preclinical stage and complete the formulation and characterization work necessary to pursue an Investigational New Drug application.

“Advances in science and medicine have enabled a giant leap forward in our understanding of the biology of rare diseases, like childhood cancer. We have an obligation to use that information to make a difference. That is what motivates me.”

“Cdk5 has emerged as an important therapeutic target in some of the most serious cancers affecting both adults and children, including brain tumors and cancers with a high propensity to spread or metastasize. However, we think the potential, broader impact of this effort is quite high as our own data also reveal a critical role for Cdk5 in forming immune memory, a function that predicts application of MDX01 in settings that include autoimmune disease and organ transplant.

We have had many physician-scientists calling, interested in how MDX01 could help patients affected by many diseases where Cdk5 is viewed as a therapeutic target.”

“We are very focused on the impact of this effort, as our advances in the laboratory only truly matter if they lead to better outcomes for the patients and families affected by these conditions.”

Source: Article from 2017-18 Annual Publication.

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