Photo of John “Chip” Tilton
John “Chip” Tilton

Case Western Reserve’s John “Chip” Tilton receives prestigious Hartwell Foundation Award for research on enzyme-replacement therapy

Case Western Reserve named among The Hartwell Foundation’s Top Ten Centers of Biomedical Research

Inborn errors of metabolism are rare single gene defects that involve mutations of enzyme proteins in key metabolic pathways. Many mutations are lethal to children at birth or in early childhood and are often associated with profound developmental abnormalities, cognitive impairments, seizures and blindness.

Enzyme-replacement therapy could compensate functionally for a missing or defective enzyme, but such a therapy requires overcoming the technical barriers to delivery of the required enzyme into the affected cells.

John “Chip” Tilton, associate professor in the Department of Nutrition at Case Western Reserve University School of Medicine, maintains a research focus on understanding what makes cells susceptible to viral infection, but has more recently shifted to repurposing viruses for intracellular delivery of therapeutic proteins.

The Hartwell Foundation has again selected Case Western Reserve University among its 2018 Top Ten Centers of Biomedical Research, making it eligible to participate in the foundation’s competition for Individual Biomedical Research Awards.

The 2018 Hartwell Top 10 Research Centers include, in alphabetical order:

Case Western Reserve University

Cornell University

Duke University

Johns Hopkins University

St Jude Children’s Research Hospital

University of California, Davis

University of California, San Diego

University of Texas Southwestern Medical Center

University of Virginia

University of Wisconsin-Madison

The Hartwell Foundation invites each Top Ten Center to nominate up to three individuals for a Hartwell Individual Biomedical Research Award. The foundation will select at least 10 individuals to receive a 2018 Hartwell Individual Biomedical Research Award, with an announcement in April 2019.

For each nominee selected for funding, the sponsoring participating institution will qualify to receive a Hartwell Fellowship to fund one postdoctoral candidate in biomedical research who exemplifies the foundation’s values. The fellowships offer support for two years at $50,000 direct cost per year to individuals in the early stage of their career by enabling them to pursue further specialized research training as part of their professional development.

In selecting each research center of excellence, The Hartwell Foundation takes into account the shared values the institution has with the foundation relating to children’s health, the presence of an associated medical school and biomedical engineering program and the quality and scope of ongoing biomedical research.

The foundation also considers the institutional commitment to support collaboration, provide encouragement and extend technical support to the investigator, especially as related to translational approaches and technology transfer that could promote rapid clinical application of research results. Selection of an institution for participation in any given year does not guarantee selection in a subsequent year.

Tilton, also director of immunobiology at the School of Medicine’s Center for Proteomics and Bioinformatics, now has a new round of financial support for that work.

Tilton is one of just 12 researchers nationally to be recognized for a Biomedical Research Award from The Hartwell Foundation. The Memphis-based philanthropic institution invited 17 prestigious institutions to nominate individuals to compete for the award, which is given annually for early-stage, innovative and cutting-edge biomedical research with the potential to benefit children of the United States.

As a Hartwell Investigator, Tilton and his lab will receive support for three years at $100,000 in direct costs per year for his research titled, “Enzyme Replacement Therapy for Inborn Errors of Metabolism.”

“It’s a really important award for me because my lab is pivoting from studying viruses to redirecting viral particles for therapeutic purposes,” he said. “The goal of my Hartwell project is enzyme-replacement therapy using  a new method for moving enzymes across biological membranes and into the cytoplasm, mitochondria or nucleus of a living cell. This is early-stage research and the Hartwell award will support development of the technology.”

“John’s strategy is to co-opt the natural biology of a virus to spread and infect tissues within the host for a therapeutic benefit,” said Fred Dombrose, president of The Hartwell Foundation. “It is an innovative approach that offers hope for those patients and their families who suffer from the devastating effects of inborn errors of metabolism and must endure limited medical options.”

Tilton was a clinical research fellow at the National Institutes of Health and a senior research investigator at the University of Pennsylvania before starting his lab at Case Western Reserve seven years ago.

“Dr. Tilton’s potentially transformative technology could be life-saving for vulnerable infants and children by delivering critical missing proteins as a therapy for metabolic illnesses,” said Lynn Singer, deputy provost and vice president for academic affairs.

Each year, The Hartwell Foundation invites a limited number of institutions in the United States to hold an internal open competition to nominate candidates from their faculty who are involved in early-stage, innovative and cutting-edge biomedical research that has not yet qualified for significant funding from outside sources.

The award-winning proposals for 2017 represent technology from medicine and biomedical engineering, in research areas that include cancer, medical devices, medical diagnostics, molecular biology, neurobiology, physiology and tissue-engineering.

Tilton proposes to use engineered virus-like particles to deliver missing proteins into the cells of patients suffering from genetic diseases caused by mutations in genes that encode for metabolic enzymes and proteins. If successful, his approach will lay the foundation for a strategic, enabling method for other diseases that require delivery of proteins or specific drugs into the cells of living tissue.

For more information, contact Bill Lubinger at