Research Report Brings Hope for Cure for Type 1 Diabetes

Pediatrics researchers at The Children's Hospital of Philadelphia and McGill University in Montreal announced that they have identified a gene variant that raises a child's risk for Diabetes Mellitus, commonly referred to as Type 1 diabetes.

The results of a study co-authored by doctors Hakon Hakonarson and Constantin Polychronakos appeared July 15 in an advance online letter in the journal Nature.

Currently, approximately 3 out of every 1000 children born within the United States is diagnosed with Type 1 Diabetes. While there is no cure for Diabetes currently, treatments for this disease are very restricted, can be costly long term and do not necessarily reduce the risk of long term complications. I had an opportunity to speak with one of the coauthors today, Dr. Hakon Hakonarson from Children's Hospital of Philadelphia.

The focus of the conversation was current treatment methods for Type 1 diabetes, previous treatment methods as well as the long term implications of his research. According to Dr. Hakonarson, current treatment methods are limited to, "monitoring and insulin injections." Because Type 1 diabetes is essentially caused by the body attacking the pancreas, causing it to not produce enough insulin to counter sugar levels in the body, at present there is "very little that can be done other then the monitoring of sugar levels, and certainly there is no cure at this time." When asked about prior procedures regarding pancreatic transplants as a treatment method, Dr. Hakonarson responded, "Pancreatic transplant has been tried in the past with little success," adding, "the pancreas is a very difficult organ."

Regarding what the current report can mean for diabetic treatment, Dr. Hakonarson said the study in labs and on animals shows that they are able to stop the "Killer Cells" from attacking the pancreas. This treatment method, if successful in clinical trials, is designed to be, "a preventative gene therapy for newborns and infants who have a family history of Type 1 Diabetes."

While this research is viewed by Dr. Hakonarson as a preventative treatment, he did not rule out the possibility that in the future, it could also be used for existing patients with Type 1 Diabetes. However, he said "that is much more difficult to do."

According to a report by William H Lamb MD, from Bishop Aukland UK, long term complications of Type 1 Diabetes are: Retinopathy, Cataracts, Hypertension, Progressive renal failure, Early coronary artery disease, Peripheral vascular disease, Neuropathy and increased risk of infection

While not currently not in clinical trials, the study was based off of the genomes of 1,046 children with Type 1 diabetes. These patient samples were collected from pediatric diabetes clinics in Philadelphia and additionally in four Canadian cities. The report itself can be read at: http://www.nature.com/nature/journal/vaop/ncurrent/abs/nature06010.html Sources: Children's Hospital of Philadelphia press release
Phone interview with Dr. Hakon Hakonarson of Children's Hospital of

Philadelphia
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