Making a Research-Inspired Breakthrough a Treatment Reality
Jim Wells, PhD, describes his lab in the Division of Developmental Biology at Cincinnati Children's as having a "basic science component with a spin toward applied biomedicine." His work in understanding basic endocrine pancreas development is one illustration of how scientific research shows translational potential for the treatment of childhood diseases such as Type 1 diabetes.
The general aim of Dr. Wells' work is to understand the molecular mechanisms underlying the development of organs, such as the pancreas. By identifying the genes and signaling mechanisms involved in pancreatic development, Dr. Wells hopes to use this information to coax the immature cells involved in organogenesis into becoming pancreatic cells to treat human diseases of the pancreas like diabetes.
Dr. Wells and his colleagues are studying two approaches, the first of which is to help regenerate cells that were destroyed by disease. By understanding how insulin-producing cells are normally regenerated during the lifespan of an organism, it may be possible to develop therapies to stimulate regeneration of insulin cells in patients with Type 1 diabetes.
Work in the Division of Developmental Biology illustrates how scientific research shows translational potential for treating childhood diseases.
The second approach is to grow pancreatic cells in a dish for surgical transplantation into patients. In some studies, researchers have transplanted beta cells from a donated pancreas into a patient. In some cases, says Dr. Wells, this technique has resulted in a patient being able to go years without insulin. This approach is promising, but, with around 2 million people in this country with Type 1 diabetes and enough donated pancreatic cells for only about 2,000 patients, it is not a viable solution. Dr. Wells' lab, however, is striving to learn how to create pancreatic cells in a dish from immature pluripotent cells. With an understanding of how pancreatic cells are made, Dr. Wells is attempting to take this information and, by exposing the pluripotent cells to the same signaling cues, coax these cells in a step-wise manner into becoming insulin-producing beta cells. There are certainly several key steps that an immature cell must take to become a functional insulin-producing beta cell, and it is the goal of Dr. Wells and his staff to recreate these steps in a culture dish. He estimates that his lab at Cincinnati Children's and others around the world are at least halfway to making this research-inspired breakthrough a treatment reality.
