FBR Smart Brief – July 18, 2018

A five-year, $42 million grant renewal from the NIH will enable the Tulane National Primate Research Center to continue searching for ways to prevent, treat and cure a host of devastating diseases, including Alzheimer's disease, HIV/AIDS and Zika virus. The funding will also support the center's regenerative medicine program and allow the expansion of research on drug abuse, neurodegenerative diseases and cardiovascular disease, according to a statement from Director Jay Rappaport.

LDL cholesterol levels declined by 30% to 60% and PCSK9 levels dropped by 45% to 84% in rhesus macaques that had undergone genome editing to inactivate the PCSK9 protein in their livers, researchers reported in Nature Biotechnology. Scientists engineered a meganuclease to recognize and inactivate the PCSK9 gene and delivered the enzyme via an adeno-associated virus, validating an approach they hope to test in humans.

Although circulating tumor cells can signal disease, their scarcity makes them difficult to use as a biomarker, but a paper in the journal Nature Biomedical Engineering describes technology that could make the approach more effective. Researchers were able to catch far more tumor cells than existing techniques allow using an inserted magnetized wire and magnetic nanoparticles, and after testing the method in pigs, they hope to move toward testing in humans.

Machine-learning software is as good as or better than animal studies in predicting some kinds of toxicity, including inhalation hazards and aquatic toxicity, researchers reported in Toxicological Sciences. The researchers built a machine-readable database with information on some 10,000 chemicals based on about 800,000 animal studies that can compare new chemicals to closely related known compounds and predict toxic effects.

A nanoparticle gel "backpack" bearing the cytokine IL-15 and attached to engineered chimeric antigen receptor T-cells eliminated signs of melanoma and glioblastoma in 60% of mice that received injections. The backpack activated more T-cells than free IL-15, and the mice tolerated high doses without adverse effects, the researchers reported in Nature Biotechnology.

Metronomic chemotherapy involves regular, frequent administration of low doses of cytotoxic drugs instead of periodic administration of high doses of cytotoxic drugs, and the protocol prevents tumor growth and metastasis by depriving tumors of blood, stabilizing disease, veterinary oncologist Sue Ettinger said during a presentation at the AVMA Convention 2018. The protocol minimizes side effects and damage to healthy cells, but it is not without risk, and more research is needed to determine optimal drugs and doses and ideal tumor targets, Dr. Ettinger said.

The online AVMA Animal Health Studies Database lists 371 studies for companion and agricultural animals in 17 fields of veterinary medicine, and the AVMA has enhanced the interface and is looking at ways to raise public awareness. Participating in veterinary clinical trials is a boon to animal and human health, says Dr. Sue VandeWoude, who serves on the AVMA Council on Research and as associate dean for research at Colorado State University College of Veterinary Medicine & Biomedical Sciences.

The FDA approved a treatment for smallpox that was shown to be effective in monkeys and rabbits infected with a similar virus, and the US government has stockpiled 2 million units of the drug in case of a bioweapon attack. The drug may also be developed for use against monkeypox and other infectious diseases.

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