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Trial Shows RNA Therapy Safe for Diabetes

Blood glucose test

(Peejhunt, Pixabay)

20 Feb. 2019. Results from a clinical trial show a new drug with synthetic messenger RNA is safe for people with diabetes, and has the potential to promote new blood vessel growth. Results of the trial testing a messenger RNA treatment made by biotechnology company Moderna Therapeutics in Cambridge, Massachusetts and drug maker AstraZeneca in London, appear in today’s issue of the journal Nature Communications.

The trial is testing the therapy code-named AZD8601 developed by AstraZeneca, based on Moderna’s technology that synthesizes messenger RNA, a nucleic acid based on the genetic code from DNA, and used by cells to produce the amino acids in proteins for cellular functions. Moderna manipulates the coding region in the messenger RNA chemistry to provide instructions for cells to produce proteins with specific therapeutic properties. Those coding instructions are contained in a standard package that appears in most cases like natural RNA to avoid triggering an immune response, and reach the desired cells where the therapeutic protein is needed.

In this case, AZD8601 codes for the protein vascular endothelial growth factor A, or VEGF-A, that promotes the growth and proliferation of blood vessels. Many people with type 2 diabetes, the form affecting 90 to 95 percent of people with the disease, develop complications where blood vessels are comprised, leading to heart disease, stroke, vision problems, and in some cases amputations. AZD8601 is being developed as a treatment for people with type 2 diabetes to promote blood vessel growth.

The early-stage clinical trial enrolled 42 men age 18 to 65 in Berlin, Germany with type 2 diabetes. The study, conducted by researchers from Sahlgrenska University Hospital in Gothenburg, Sweden and Karolinska Institutet, in Stockholm, first tested escalating doses of AZD8601 against a placebo. One group of participants received either AZD8601 or a placebo as intradermal injections into the forearm under the skin, and were assessed for adverse reactions. The other group received similar injections of AZD8601 or a placebo, but were instead evaluated for VEGF-A protein levels with skin microdialysis, and blood flow dynamics, as an indicator of blood vessel development, with laser imaging.

The results show the AZD8601 treatments are safe and well-tolerated, with only mild adverse injection-site reactions reported by participants. Individuals receiving AZD8601 also show higher VEGF-A concentrations from 4 to 24 hours after treatment compared to placebo recipients. And AZD8601 recipients display enhanced blood flow through skin layers than placebo recipients, both within 4 hours of treatment and 7 days later.

Kenneth Chien, a molecular and cell biologist at Karolinska Institutet and co-author of the Nature Communications paper, says in a Moderna statement, “I believe this is an important milestone in the field of mRNA therapeutics as it starts to address many questions regarding the safety and delivery of mRNA to human tissues, the duration and level of the protein that can be expressed and the ability of the technology to have a physiologic, measurable function over a prolonged period of time.” Chien is also a scientific founder of Moderna.

AstraZeneca in 2013 agreed to license Moderna messenger RNA therapies addressing up to 40 targets divided among metabolic and cardiac diseases and cancer, in a deal with a total potential value of $420 million. And also as reported by Science & Enterprise, Moderna in December 2018 issued the largest initial public stock offering ever for a biotechnology company, valued at $604 million.

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