A La Jolla researcher has received a prestigious grant to fund his groundbreaking work in the battle against diabetes.
Dr. Mattias von Herrath of the La Jolla Institute of Allergy and Immunology was selected May 15 as a recipient of the Juvenile Diabetes Research Foundation' s second annual Scholar Awards. Von Herrath was selected as one of six recipients of the award, which includes research funding of $250,000 annually for up to five years.
"The Scholar Award is designed to encourage and support innovative, high-risk and high-reward, paradigm-shifting, challenging research," said Richard Insel, vice president of research for the Juvenile Diabetes Research Foundation. "To fulfill its mission and find a cure fast, JDRF depends on the creativity and excellence of individual scientists like Dr. von Herrath."
The foundation is the world's largest donor to type 1 diabetes research, a disease that strikes children suddenly, makes them insulin-dependent for life and carries the constant threat of devastating complications. Since its inception, the foundation has provided more than $1 billion to diabetes research worldwide.
Type 1 diabetes, also known as insulin-dependent diabetes or juvenile diabetes, occurs when the immune system mistakenly destroys the insulin-producing beta cells of the pancreas, which are key to controlling blood sugar levels.
Von Herrath was chosen as a recipient of this year's Scholar Award for his proposal to obtain detailed images of the cells involved in the immune response in the pancreas of an animal that has type 1 diabetes. To acquire the information necessary and fully understand what takes place when the islets cells of the pancreas are being attacked, von Herrath plans to monitor the islets through live microscopy imaging.
Von Herrath is internationally recognized as an expert on the molecular basis of type 1 diabetes. Last April, he was the leader of a research team at the Institute of Allergy and Immunology that published a major finding about a promising combinatorial treatment approach to the disease.
The study combined two therapies already being tested individually in human clinical trials. When used in conjunction, the two methods produced better efficacy, longer-lasting results and fewer side effects in pre-clinical trials in mice than either therapy has shown alone in human studies.
Von Herrath's approach focuses on teaching the immune system to tolerate, rather than attack, the insulin-producing beta cells of the pancreas. By injecting the anti-CD3 antibody, which calms the immune system and lessens the attack on the beta cells, along with a piece of the nasal proinsulin peptide, which acts like a vaccine and induces a special cell type called "regulatory cells" that can actively and specifically protect beta cells, von Herrath was able to stop the immune system's destruction of the beta cells in mice.
Combining the two therapies created a strong synergy, von Herrath said.
"The combinatorial approach doubled the efficacy in laboratory mice, with fewer side effects than using either one alone," he said.
The study represented the first time a combinatorial treatment approach using a vaccine strategy had ever been tried with type 1 diabetes. Von Herrath noted that the technique showed long-lasting results.
"The diabetes never recurred in the lifespan of the mice," he said. Insel also expressed hope that the approach could change how diabetes is treated.
"This combination approach is worth evaluating in human type 1 diabetes to increase both the overall efficacy of the treatment and the duration of the beneficial effect," Insel said.
Type 1 diabetes sufferers inject insulin daily to control blood sugar levels. However, complete control is difficult to achieve.
"(Von Herrath's therapy) is particularly attractive because, if successful in humans, it could replace current insulin-injection treatments altogether, which often cannot prevent the long-term detrimental effects of diabetes."
The therapy is designed for those in the early stages of diabetes, who have not yet had a significant amount of beta cell destruction.
"Since the complications from high blood sugar levels worsen with time, we are hopeful that this therapy can reverse the disease in patients before they have too much multi-organ damage," von Herrath said.
The La Jolla Institute for Allergy and Immunology, located on UCSD's East Campus, is a nonprofit medical research center dedicated to increasing knowledge and improving human health through studies of the immune system. Its research staff includes more than 100 Ph.Ds.
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