"Anti-vaccine" for diabetes
DNA vaccine for type 1 diabetes has been successfully tested on humans
Medical Daily News: DNA Vaccine Sends Type 1 Diabetes Into Reverse, Does Well In Clinical TrialA group of American and Dutch scientists has developed a genetically engineered "reverse-acting vaccine" designed for the treatment of type 1 diabetes (insulin-dependent), and successfully conducted the first phase of its clinical trials.
Unlike conventional vaccines, BHT-3021 does not activate, but suppresses the patient's immune system, thereby restoring normal insulin biosynthesis. The work was published in the journal Science Translational Medicine (Roep et al., Plasmid-Encoded Proinsulin Preserves C-Peptide While Specifically Reducing Proinsulin-Specific CD8+ T Cells in Type 1 Diabetes).
The pathogenesis of type 1 diabetes is based on the insufficiency of insulin production by beta cells of the islets of Langerhans of the pancreas, caused by their destruction under the influence of an autoimmune process. The main target of the attack by immune killer cells - CD8–positive T-lymphocytes - is proinsulin, an insulin precursor.
In order to reduce the hyperactivity of the immune system and protect beta cells, the authors, specialists from Stanford (USA) and Leiden (Netherlands) universities, developed using genetic engineering methods the BHT-3021 vaccine, which is a ring DNA molecule (plasmid) that plays the role of a vector for the delivery of the genetic code of proinsulin. After getting into the tissues and biological fluids of the body, BHT-3021 "takes the hit" – distracts the attention of killer cells, thus generally reducing their activity, and at the same time does not affect the rest of the immune system. As a result, beta cells restore their ability to synthesize insulin.
The first phase of clinical trials of BHT-3021, which has previously proven its effectiveness on an animal model, involved 80 patients over the age of 18 who were diagnosed with type 1 diabetes in the last five years. Half of them received intramuscular injections of BHT-3021 weekly for 12 weeks, and the other half received a placebo.
After this period, the group receiving the vaccine demonstrated an increase in the level of C-peptides in the blood, a biomarker indicating the restoration of beta cell function. No serious side effects were recorded in any of the participants.
BHT-3021 is still far from commercial use. The license for it was obtained by the California-based biotech company Tolerance, which intends to continue clinical trials of the vaccine on a wider range of patients. It is expected that 200 young people who have been diagnosed with insulin-dependent diabetes will take part in them. Scientists want to test whether BHT-3021 can slow down or stop the development of the disease at an early stage.
It is believed that type 1 diabetes affects about 17 million inhabitants of the planet. Most often, young people get sick with it – children, teenagers and adults under 30 years old.
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