As a practicing diabetes clinician caring for children and teens, my job is to keep tight control over your blood sugars and you in the best physical condition to avoid complications…until we find a cure. I try to take a proactive stance so that when we find a cure, my patients are ready One of my biggest dreams is to actually be able to administer the cure (or interrelated cures) to my patients. I am always reading medical journals and information from families and kids about the latest research. I want to talk about two promising research trials: one that is actually taking place in newly diagnosed type 1 humans and the latest exciting breakthrough in mice that was published online last week in the journal Nature (a prestigious medical journal).
In my opinion, curing or preventing the relentless destruction of pancreatic Beta cells secondary to those killer cells (see blog on autoimmune diseases) will need several interlocking approaches. The first approach proven very effective in newly diagnosed children with diabetes involves the administration of monoclonal antibodies intravenously, which attack and neutralize those pesky killer cells that destroy your pancreatic islet cells. This study, championed by a former colleague, has advanced monoclonal antibody trials into pharmaceutical research throughout the world. The best news is that the antibodies truly work to knock out the killer cells, but continued research must be done to determine the amount, timing, and duration of the new drug (of monoclonal antibodies) that is necessary to induce remission of the pancreatic beta cells. By repeated administrations of the monoclonal antibody mixtures, Beta cells continue to produce insulin that essentially prolongs the honeymoon period. Once the medication is stopped, the killer cells begin to knock off the remaining islet cells. One of the next steps will be to determine just how long patients can receive these monoclonal antibodies over the years. Can they be given indefinitely? It is important to note that there are very strict inclusion criteria (a list of requirements for children with new onset diabetes that have to be fulfilled before a child can be included in the study; such as weight criteria, previous illnesses, time since diagnosis, etc.). If you know someone who has been recently diagnosed, they should see if they are interested in this research called the PROTÃ‰GÃ‰ Trial. Keep in mind that the study is very intensive, including two weeks of daily intravenous injections conducted at various institutions throughout the country.
The second study published last week in Nature was actually initially brought to my attention by the mother of one of my teens with type 1 diabetes. One of the coolest aspects of this study is that it bypasses the use of stem cells, which is a controversial topic in terms of the use of embryos as the source of these stem cells. In this study, mice are the subjects. It is important to realize that through evolution mice and humans share a significant number of genes. Therefore research conducted on mice is the first step before studying humans. In this study, scientists at Harvard took a mouse pancreatic non-islet cell that normally functions to digest food (an exocrine cell) and by gene transfer changed it into a BETA ISLET cell that could produce insulin (endocrine cell). The scientists inserted special genes (DNA), thought to be able to switch the non-islet cell into an insulin-producing cell, into a virus and then injected the virus into the mouse. The virus spread in the mouse and a portion of non-islet cells were transformed into islet cells, which then secreted insulin. The diabetic mice were not cured; but they did produce effective amounts of insulin that lowered their blood sugars! These findings are very exciting because they did not require the use of stem cells and typical cells in the pancreas could be coaxed to produce insulin. There are major concerns in terms of safety especially since a virus is used to introduce these genes. No one knows if the virus will cause future problems to these mice.
It is my belief that a combination of these approaches will eventually be employed in the treatment of type 1 diabetes–to keep those pancreatic beta cells alive to produce insulin by preventing their destruction (the ProtÃ©gÃ© trial) and increase the number of insulin producing cells by converting typical digestive pancreatic cells into islet cells (the new mouse study). This is a very exciting time for the creation of innovative approaches to save those precious pancreatic islet cells! I will keep you posted with new developments.