Partnership: The CPGM is among the first of its kind with a pediatric focus, providing clinical genomics services and genomic research in rare, inherited diseases in children. The KIDDRC, supported by the National Institute for Child Health and Human Development (NICHD) for nearly 50 years, conducts research aimed at the causes, prevention and treatment of intellectual and related developmental disabilities. Building upon these regional strengths and capabilities in rare disease genomic medicine and intellectual and developmental research, IAMI collaborated with CPGM and KIDDR to develop the Gene-to-Drug-to-Children (GDC) strategy. 

Success Story: The GDC strategy allows researchers to characterize the biology of a genomic discovery, identify and validate drug targets, discover new agents, exploit opportunities to repurpose existing drugs and advance promising new treatments to children suffering from rare diseases. With more than 7,500 single gene disorders reported, GDC provides a framework for selecting initial rare disease projects to demonstrate proof of concept based on unmet medical need, patient value and feasibility criteria and characteristics. Frontiers: Heartland Institute for Clinical and Translational Research patient and community engagement lead, Dr. Kim Kimminnau, Kelly Ranallo, co-founder and president of the Turner Syndrome Global Alliance and IAMI director Scott Weir Pharm.D., Ph.D., have brought together Frontiers partners University of Kansas Medical Center, CMH, University of Missouri-Kansas City, the Stowers Institute for Medical Research, the Global Genes Project, the Turner Syndrome Global Alliance and the Kansas City area chapter of the Cystic Fibrosis Foundation to rally the Kansas City region in support of T1 through T4 rare disease translational research.

Partnership: The TAG works directly with investigators from Frontiers, other Clinical and Translational Science Award hubs and National Cancer Institute Cancer Centers to create high potential projects at the chemistry/biology interface and advance these projects to in vivo preclinical proof of principle.

Success Story: TAG builds upon the drug discovery and development infrastructure within KU, created in part through participation in the National Institutes of Health-funded Molecular Libraries and NCI Experimental Therapeutics Programs to assist teams of basic and clinical (disease expert) researchers in drug discovery and development. TAG is comprised of a multi-disciplinary, multi-organizational team of experts that integrates project management, assay development, medicinal chemistry, drug delivery, pharmacokinetics and pharmacodynamics and clinical pharmacology disciplines into the drug discovery process.

Learn more about TAG.

Cultivating Medical Technology: IAMI's multi-disciplinary, multi-organizational project teams create rich, diverse scientific environments for translational scientists to foster innovation, enhance learning (not only of their own discipline but gaining knowledge of other disciplines in the process), create opportunities to engage patients in the development, demonstration and dissemination process and build entrepreneurship skills necessary to improve the quality of translational research conducted. In 2009, IAMI established an entrepreneurship training and education program available to graduate students in biomedical sciences and business administration, as well as postdoctoral trainees at KU Medical Center.

Success story: Two former IAMI Fellows, Karthik Ramachandaran, Ph.D., and Joshua Sestak, Ph.D., have created and currently lead successful Kansas-based startup companies, Likarda LLC and Orion Biosciences, Inc., respectively. Both startup companies have spun out medical innovation technologies supported by IAMI. Likarda, a One Health biotechnology company, has raised private capital investment and is currently generating service-based revenues. Orion has secured private investment and a Phase I Small Business Innovation Research grant and is currently advancing its first drug product.

IAMI took this opportunity to focus on reformulating and repurposing approved drugs. This created rich collaboration across the University of Kansas campus, the Kansas City community and the nation.

Creating a fast track to new treatment is the pathway the Institute is developing within the motivational need to help people and improve patient care. 

Children: New drug therapies

• Acute lymphoblastic leukemia (ALL) is a type of leukemia most common in children. The Institute leaders and clinicians from Children's Mercy Hospital in Kansas City have collaborated to open a pediatric clinical trial for ALL remission patients. Through this partnership, the Institute leaders reformulated a new drug therapy for children that clinicians at Children's Mercy Hospital were seeking to treat their young patients.
• The Institute also reformulated Hydroxyurea, a chemotherapy agent used for many years to treat people with certain cancers for use in treating children with sickle-cell disease, most often presented in children. The Institute in collaboration with the Institute for Pediatric Innovation has reformulated Enalapril, an ACE inhibitor, into a drug therapy for children.

"What motivates us (the Institute) is not to develop the next big blockbuster drug but to apply our expertise to these problems and help children," Scott Weir, IAMI director. 

Adult: New drug therapies

• Acute myeloid leukemia (AML), present in adults, is the most common acute leukemia.  The Institute provided services to repurpose an antifungal; drug, Ciclopirox, as a new option to cancer patients in clinical trials.  Leukemia & Lymphoma Society (LLS) and the Ontario Cancer Institute were partners in this effort and continue to collaborate with IAMI.  Additionally, Tigecycline, an antibiotic, has also been repurposed for AML patients and is in clinical trials. LLS and the Ontario Cancer Institute are partners for Tigecycline as well.
• The Learning Collaborative (TLC) project, is a partnership with the National Institutes of Health and the Leukemia & Lymphoma Society and is serving as a national model for academic, government and non-profit collaboration.  Thus far, the partnership has advanced auranofin, an off-patent drug approved fro the treatment of rheumatoid arthritis, to clinical evaluation for the treatment of chronic lymphocytic leukemia (CLL).  CLL is the most common type of leukemia.
• And lastly, Melphalan was licensed to a Kansas startup, Cydex.  Cydex applied platform drug delivery technology that was licensed from KU to address formulation shortcomings of the marketed formulation. Melphalan is designed to deliver a new treatment for multiple myeloma adult patients.