The University of Tennessee Graduate School of Medicine, Knoxville

Amyloidosis Research Team Receives SMARTT Grant for Clinical Trial

There are approximately 30 diseases that are associated with the buildup of a waxy material composed of protein fibrils and sugar-like molecules — amyloid — in various body organs. Most widely known and studied is amyloid found in the brains of patients with Alzheimer’s disease; but, less commonly, amyloid may also deposit in the heart, liver, kidneys and other organs in certain patients with multiple myeloma or light chain amyloidosis.

The rarer forms of amyloid disease are difficult to diagnose early because they are often unrecognized and the patients have a vast array of diverse symptoms. At present there is no approved method in the US for imaging the disease in these patients, something that would aid in definitive diagnosis and assist doctors in monitoring patients with these devastating, often fatal disorders.

To address this clinical problem, researchers in the Amyloidosis and Cancer Theranostics Program (ACTP) and Molecular Imaging and Translational Research Program (MITRP) have been developing new tools and techniques, through partnerships with Oak Ridge National Laboratory and Siemens, to effectively image amyloid in the body organs using radioactive proteins. The most important step in the development of this new technique is to perform a Phase I clinical trial with patients suffering from these disorders. Preparation for the clinical trial involves performing standard toxicology studies, manufacturing sufficient protein for the trial, and applying for approval from the Food and Drug Administration (FDA).

The ACTP team, led by Jonathan Wall, PhD, has been awarded funding and support from the Science Moving towArds Research Translation and Therapy (SMARTT) Program to help make the clinical trial a reality.

The SMARTT program, available through the National Heart, Lung, and Blood Institute at the National Institutes of Health, is designed to accelerate translation of research from bench to bedside. To accomplish this mission, the SMARTT program provides regulatory support, manufacturing, and toxicology services to qualified projects.

With support from the SMARTT program, the ACTP team, in collaboration with the MITRP and oncologists Ronald Lands, MD, Associate Professor of Medicine, David Aljadir, MD, and John Bell, MD, Professor of Surgery, is applying to the FDA for approval of an imaging clinical trial using a novel amyloid-targeting agent called peptide p5+14. Also, SMARTT plans to provide more than 500 patient doses of the peptide and may also support important safety studies in preparation for the clinical trial at The University of Tennessee Medical Center.