Research

Pathway Grant Recipients

2020 Grant Recipients

Accelerator award

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Judith Agudo, PhD

Dana-Farber Cancer Institute, Boston, MA
Harnessing Immune Privilege Mechanisms from Stem Cells to Protect Stem Cells from Immune Attack
Grant # 1-20-ACE-08

Despite notable improvement in exogenous insulin therapy, people with diabetes often have difficulty adequately controlling their glucose levels, which can lead to serious complications. To definitively cure type 1 diabetes, the insulin-producing β-cells that were lost need to be replaced. Strategies to accomplish this, including transplantation of cells provided by donors or the conversion of stem cells, have seen limited success because the transplanted cells inevitably succumb to the same autoimmune attack that killed the original β-cells. Thus, it is of the utmost importance to develop strategies to preserve newly generated or transplanted insulin-producing cells.

Dr. Agudo’s Pathway to Stop Diabetes project will investigate such a strategy. Her laboratory recently discovered the existence of specialized stem cells in the skin and muscle that are somehow protected from attack by immune cells. The goal of her project is to determine the molecular underpinnings that allow these stem cells to be “cloaked” from activated immune cells and then to apply them to β-cells. In effect, this could indefinitely protect transplanted β-cells without the need for immunosuppressive drugs and could lead to better outcomes and reduced burden for people living with diabetes.

Visionary award

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Maxence V. Nachury, PhD

University of California, San Francisco
Regulation of Body Weight Homeostasis and β-Cell Function by Primary Cilia
Grant # 1-20-VSN-03

Nearly every cell in the human body possesses a sensory “antenna” that is used to sense changes occurring outside of the cell. These antennae are called primary cilia. In a group of rare genetic disorders dubbed ciliopathies, malfunction of the primary cilia results in profound obesity, kidney anomalies, vision loss, altered glucose tolerance, and a host of other symptoms. The range of symptoms present in the ciliopathies suggests a broad physiological importance for primary cilia. However, little is known about how primary cilia affect regulation of blood glucose and body weight.

Dr. Nachury’s Pathway to Stop Diabetes project seeks to determine the role primary cilia play in two distinct areas that are important to the development of obesity and type 2 diabetes. First, he will determine how primary cilia influence the processes that control appetite in the brain. Second, he will study how primary cilia affect the function of insulin-producing pancreatic β-cells. Ultimately, the goal of Dr. Nachury’s project is to determine whether primary cilia can be targeted therapeutically to improve treatments for people with diabetes.

Previous Award Recipients