By: Shelagh McNally for Diabetes1
An innovative delivery method is offering hope to millions of people suffering from diabetes and may free them from the routine of daily injections. Researchers at the Baylor University Center and Baylor Research Institute in Texas have developed a new technique for getting gene-therapy into the pancreas.
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UTMD (ultrasound-targeted microbubble destruction) delivers tiny gas filled microbubbles of insulin to the pancreas via the bloodstream. An ultrasound beam is used to burst the bubbles and release the insulin. UTMD is proving to be much less invasive and much less damaging than other therapies. One major obstacle for treating diabetes has been the remote location of the pancreas – beneath the stomach – making it difficult to deliver therapeutic genes. Injecting the microbubbles directly seems to solve that problem.
“It’s very clever,” said Dr. Bob Goldstein, chief scientific officer of the Juvenile Diabetes Research Foundation. “Short of sticking a needle directly into your pancreas and delivering a gene, how am I going to deliver something harmlessly, so it doesn't hurt you and it gets to where I want it to go? That's what's special about this.”
Researchers started off by injecting rats with bubbles containing the human insulin gene and found that the rat’s blood sugar had been lowered significantly. Hexokinase1, the gene that regulates insulin production, was also successfully delivered using these bubbles and resulted in increased blood insulin as well decreased blood sugar. The pancreas appeared to be unharmed.
"Not only was their blood sugar lowered, but there was no evidence of any damage to the pancreas," said Paul Grayburn, M.D., principal investigator of the study. "Other forms of gene therapy are usually invasive and unlike the UTMD technique, do not target the tissues and organs specifically.
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Currently Type 1 diabetes must inject insulin daily to keep their blood sugar levels balanced as well as following nutritional guidelines. Type 1 is an inherited disease occurring when the body turns off its own insulin producing cells and is unable to process glucose. Type 2, the more common diabetes is an obesity-linked form of the disease where pancreatic cells lose sensitivity to insulin. People, especially those with Type 1 diabetes are at risk for heart disease, blindness, nerve damage and kidney damage. There are more than one million Americans with Type 1 and 18 million with Type 2 diabetes.
The UTMD is a promising new therapy but is still in early research stages. “It is still a long way to go to prove safe and efficacious of gene therapy on large animals before human tests,” said study co-author Dr. Jiahuan Ding, director of the Molecular Genetics Lab at Baylor University Medical Center in Dallas. Dr. Grayburn and his team are starting to research insulin-producing cells in human patients and are also looking at UTMD technique for delivering therapeutic gene agents to other organs as well.
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