The three-year, $450,000 grant – OHSU's first from the New York-based trust – will support work by Markus Grompe, M.D., a professor in the OHSU School of Medicine Departments of Pediatrics and Molecular and Medical Genetics. Grompe has earned international acclaim for a diverse list of achievements in the areas of stem cell and gene therapy research, including breakthroughs in Fanconi anemia, hereditary liver diseases and liver regeneration.
The grant will enable Grompe to apply his expertise in liver genetics to correct the underlying cause of Type 1 diabetes: the failure of "beta cells" in the pancreas to produce the insulin our bodies need to convert sugars and other foods into energy. The research aims to replace these deficient pancreatic cells with liver cells that have been genetically reprogrammed to produce and secrete insulin. If successful, this research could relieve patients of the burdensome daily survival routine of blood sugar checks, insulin injections and strict dietary regimens.
Grompe's leadership in the study of liver progenitors – stem cells derived from liver and related tissues that shed light on how the liver develops and regenerates – provides the basis for the Leona M. and Harry B. Helmsley Charitable Trust-funded project. He said the grant will help move his research in a new direction.
"Currently my laboratory is mostly a 'liver lab,' and our focus in regenerative medicine has been on cell and gene therapy for liver diseases," he said. "This grant expands our horizons in seeking to use liver cells not only for the treatment of liver diseases but for the use of cell therapy to treat Type 1 diabetes. I'm very grateful for the opportunity this grant gives us to put our strengths together in a new way."
Grompe's project has two major phases. First, he will attempt to identify the easiest types of liver cells to reprogram into insulin-secreting pancreatic beta-cells. Candidates include the distinct-but-closely-related cells of liver itself, the bile ducts and gall bladder. Once the best cell type is identified, Grompe will work on perfecting methods to mass produce it in tissue culture. When available in sufficient quantities, the cells would be reprogrammed in vitro and ultimately transplanted into diabetic mice to test their insulin-producing efficiency.
The ultimate goal of the research is to scale up this system for the treatment of human patients with Type 1 diabetes. Grompe said he will be studying liver cells from both mice and humans, but noted that no tissue specimens will be removed from human patients specifically for this investigation. Rather, he said, the cells would be collected from otherwise-discarded tissues removed during routine liver or gall bladder surgeries.
"If this work is successful, it will lead to future treatments where diabetes patients donate their own liver or gall bladder cells for reprogramming into beta-cells," he said. "That eliminates any risk of immune rejection of transplanted cells."
The Leona M. and Harry B. Helmsley Charitable Trust provides funding for innovative and collaborative type 1 diabetes research as part of their focus in Health and Medical Research. The OHSU funding is part of the Helmsley Type 1 Diabetes Consortium, which recently awarded a total of more than $21 million in grants to 11 institutions and organizations.This recent funding is focused on advancing the understanding of type 1 diabetes, with the goal of developing therapeutic solutions for patients living with the disease until a cure can be found.
The award comes at a particularly opportune time for OHSU, said OHSU Foundation President Allan Price. "This grant provides a great backdrop for the launch of a major new OHSU/OHSU Foundation initiative to raise significant philanthropic support to fund diabetes research and care," Price said.
"In a state that's experienced a 35-percent jump in diabetes cases in just a decade, the need is urgent to attack this problem from several different angles. We're seeking philanthropic investment that will enable OHSU to leverage the individual efforts of 100 or more OHSU scientists doing diabetes-related research, just as the Harold Schnitzer Diabetes Health Center has accomplished for our clinical and outreach services," he said.
Oregon Health & Science University is the state's only health and research university and Oregon's only academic health center. OHSU is Portland's largest employer and the fourth largest in Oregon (excluding government). OHSU's size contributes to its ability to provide many services and community support activities not found anywhere else in the state. It serves patients from every corner of the state, and is a conduit for learning for more than 3,400 students and trainees. OHSU is the source of more than 200 community outreach programs that bring health and education services to every county in the state.
ABOUT MARKUS GROMPE, M.D.
Dr. Grompe is a professor in the OHSU Departments of Pediatrics and Molecular and Medical Genetics. He is also the director of the Papé Family Pediatric Research Institute and the Ray Hickey Chair, as well as the director of the Oregon Stem Cell Center.
ABOUT THE LEONA M. AND HARRY B. HELMSLEY CHARITABLE TRUST
The Leona M. and Harry B. Helmsley Trust, established in 1999, is administered by five trustees selected by Leona Helmsley as a continuation of Mr. and Mrs. Helmsley's generous giving through their lifetimes. The Trust supports a diverse range of organizations with a major focus on health and medical research, in addition to programs in human services, education and conservation. The Trust aspires to improve lives by supporting effective nonprofits. Earlier this year, the Trust announced $136 million in grants to charitable organizations across the United States and abroad.
The OHSU Foundation is a separate 501(c)(3) organization that exists to secure private philanthropic support for Oregon Health & Science University. The foundation raises funds from individuals, companies, foundations and organizations, and invests and manages gifts in accordance with donors' wishes.