Objective
The objective of this research is to develop new ways to protect and regenerate the insulin-producing cells in the pancreas, called beta cells, which are destroyed by the immune system in people with Type 1 Diabetes (T1D). Currently, there is no cure for T1D, and treatments primarily focus on managing blood sugar levels with insulin injections. However, this does not stop the disease from progressing or prevent long-term complications. This research aims to change that by finding ways to protect beta cells from immune attacks and help them survive and function better. By focusing on a protein called Renalase (RNLS) that is linked to beta cell stress and destruction, we will create and test new drug compounds that specifically target this protein to reduce its harmful effects. If successful, these compounds could lead to new treatments that not only prevent beta cell loss but also promote their regeneration, offering a more effective approach to treating T1D and potentially reducing or eliminating the need for insulin therapy. This approach combines innovative lab techniques, advanced mouse models that mimic human T1D, and rigorous testing to ensure safety and effectiveness, paving the way for future human clinical trials and a hopeful new direction in diabetes care.
Background Rationale
Type 1 Diabetes (T1D) is a condition where the immune system, which normally protects the body from harmful invaders, mistakenly attacks the insulin-producing beta cells in the pancreas. Insulin is essential for regulating blood sugar, and without it, people with T1D must rely on insulin injections to manage their blood sugar levels. Even with treatment, managing T1D is difficult, and many people still face long-term health problems. Researchers are looking for new ways to protect beta cells from this immune attack, and our project focuses on a protein called Renalase (RNLS). RNLS might play a key role in how the immune system mistakenly targets and destroys beta cells. By studying how RNLS affects the interaction between beta cells and immune cells, we hope to find new ways to stop or reduce this immune attack. We are also testing whether blocking RNLS can protect beta cells in models that closely mimic human T1D. For this, we use special "humanized" mice that have been modified to have a human-like immune system, which allows us to see if blocking RNLS can reduce beta cell destruction in a way that would work in people. If successful, this research could lead to new treatments that help protect beta cells, potentially reducing the need for insulin therapy and improving the quality of life for people with T1D.
Description of Project
This research is focused on finding new ways to treat Type 1 diabetes (T1D), a condition where the body’s immune system mistakenly attacks the cells in the pancreas that produce insulin, a hormone essential for regulating blood sugar levels. Current treatments, like insulin injections, do not stop the disease from progressing or fully prevent complications. The study aims to develop a new type of therapy that could protect the insulin-producing cells from being destroyed, potentially reducing the need for frequent insulin doses and preventing diabetes-related health issues. One key target of this research is a protein called Renalase (RNLS), which is believed to play a role in the immune attack on pancreatic cells. We plan to study how blocking the activity of RNLS could protect these vital cells and improve their function. To do this, they will create and test new drug-like molecules that specifically inhibit RNLS, using both cell models and mouse models of diabetes. The goal is to identify compounds that are effective in protecting insulin-producing cells from immune destruction without causing harmful side effects. Additionally, the study will explore how different types of immune cells interact with pancreatic cells in both healthy and diabetic conditions. The research team will use innovative models, including mice that have been engineered to carry human immune cells, to better understand these interactions in a living system that closely mimics human disease. This approach could provide valuable insights into how T1D develops and help identify new targets for therapy. If successful, this research could pave the way for new treatments that not only manage blood sugar levels but also directly protect the body’s ability to produce insulin. This would represent a significant advancement in diabetes care, potentially improving the quality of life for millions of people affected by T1D.
Anticipated Outcome
The anticipated outcome of our research is to find a new way to protect the insulin-producing beta cells in people with Type 1 Diabetes (T1D) from being attacked by their immune systems. We hope that by understanding how a protein called Renalase (RNLS) influences this immune attack, we can develop new treatments that prevent or slow down the destruction of beta cells. If successful, this approach could lead to therapies that help people with T1D maintain some of their natural insulin production, potentially reducing their dependence on insulin injections. Ultimately, our goal is to improve blood sugar control and reduce the risk of long-term complications, making life easier and healthier for people living with T1D. We also expect that our findings will pave the way for developing safer and more effective treatments for T1D and may help us understand and treat other autoimmune diseases.
Relevance to T1D
Our research is highly relevant to people with Type 1 Diabetes (T1D) because it aims to address the core issue of the disease: the immune system's attack on insulin-producing beta cells in the pancreas. In T1D, the body mistakenly destroys these cells, leading to a lack of insulin, which is crucial for controlling blood sugar levels. Current treatments mainly focus on managing blood sugar through insulin injections or pumps, but they don’t stop the ongoing damage to beta cells. Our study is exploring new ways to protect these cells from immune attack, potentially preserving their function for longer. This could reduce the need for frequent insulin doses, lower the risk of complications, and improve overall quality of life. If successful, our work could lead to new therapies that target the disease at its source, offering a significant advancement in how T1D is treated and managed.