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THE BIOWORLD BIOME: Our Habitat for All Things Science

AACR 2014
SAN DIEGO – At a session on Application of Nanotechnology to the Treatment of Cancer Patients, the presenters showed multiple ways in which nanotechnology could improve cancer treatment and diagnostics. If, that is, the technology can make it onto pharma's priority list. Moderator Mark Davis kicked off the session by pointing out that the idea of nanoparticles itself is not new. But scientific advances have increased the understanding of multiple different functions nanoparticles can have to optimize drug delivery. "Now people know how to make these multiple functions work together properly," he told the audience, citing the example of Bind Biosciences Inc.'s BIND-014, which has optimized multiple functions simultaneously.
AACR 2014
SAN DIEGO – At the American Association for Cancer Research annual meeting, Lynda Chin had a blunt reminder for her audience. "No matter how much data you have," she said, "drugs, tests and devices are the only thing patients will benefit from." At her talk on Big Data and Technology: Transforming Cancer Care and Research, Chin, who is chair of genomic medicine and scientific director of the Institute for Applied Cancer Science of the MD Anderson Cancer Center described her hopes for getting from big data to such drugs, tests and devices.

LONDON – Work on a mouse model representing a rare inherited disease that affects the heart muscle suggests that gene therapy to replace a missing protein may one day be possible in the clinic, for some conditions at least. A team of French researchers used a specific adeno-associated virus that is known to enter heart muscle cells to evaluate how gene therapy could help patients with the neurodegenerative disease, Friedreich’s ataxia. Affecting just one in 50,000 births, Friedreich’s ataxia causes problems with balance and movement. It usually starts in adolescence, with muscle weakness progressing until patients are unable to walk or live independently.

AACR 2014
SAN DIEGO – Results presented at the of the American Association for Cancer Research (AACR) annual meeting on Sunday from the phase II PALOMA-1 trial of CDK inhibitor palbociclib (Pfizer Inc.) led to plenty of buzz that Pfizer Inc. might file for accelerated approval for the drug, despite the lack of an overall survival (OS) benefit to date. Researchers reported at the plenary session that treatment with the palbociclib doubled progression-free survival of women with metastatic estrogen receptor-positive (ER+) breast cancer in an open-label phase II trial when the drug was added to the standard treatment with the estrogen synthesis inhibitor letrozole.
HONG KONG — A new influenza A vaccine has been shown to be capable of inducing protective immune responses against multiple influenza A viruses in mice. Researchers from the School of Public Health at the University of Hong Kong (HKU) collaborated with U.S. National Cancer Institute (NCI) scientists to develop the new vaccine, which they described in the April 1, 2014, edition of the Proceedings of the National Academy of Sciences as a breakthrough for conventional seasonal flu vaccines that protect against only one or possibly a few viral subtypes.
THE CONTRATRIAN
Researchers have identified a biochemical pathway that destabilizes spines in knock-in mice bearing a protein linked to some cases of familial Alzheimer’s disease (AD). The work suggests a novel therapeutic approach to AD and age-related memory loss that is independent of the amyloid-beta processing approaches at the oft-broken heart of current drug trials. The findings were published in the April 2, 2014, issue of Neuron. Senior author Ilya Bezprozvanny, who is at the University of Texas Southwestern Medical Center, told BioWorld Today that beyond their practical possibilities, the results support his contention that there is a lot more to AD than amyloid-beta.
LONDON – The discovery of an enzyme that is vital to the survival of cancer cells, but which normal cells do not seem to need at all, is pointing to entirely new ways of treating cancer. Screening studies already have identified small-molecule inhibitors of the enzyme, and the Swedish team of researchers who made the discovery hopes to begin clinical trials with those compounds next year. So far, studies suggest that inhibiting the enzyme causes all types of cancer cells to die. It seems unlikely that the inhibitors will have side effects on a scale seen with most chemotherapy drugs.
GD TO T2D
Researchers have identified a metabolite, the fatty acid CMPF, which is starkly elevated in the blood plasma of pregnant women with gestational diabetes and in both women and men with type 2 diabetes. CMPF could possibly be both a biomarker for and a therapeutic target against diabetes. But the study, which was published in the April 1, 2014, issue of Cell Metabolism, also showed that it was only one of many metabolites that differed in normal individuals and diabetics, leading senior author Michael Wheeler to assert that diabetes is “overly simplified as a disorder of blood sugar levels and insulin secretion.” “Our studies,” Wheeler told BioWorld Today, “would suggest that diabetes is a disease of nutritional imbalance.”
HONG KONG — Publication of the first comprehensive map of gene activity throughout the human body should play a key role in the identification of those genes involved in disease and lead to the development of new personalized treatments. The 5th edition of the Functional Annotation of the Mammalian genome (FANTOM5) project, a large international consortium led by the Riken Institute in Japan, provides the first overview of the highly complex networks regulating gene expression across the wide variety of human cell types. The researchers published their findings in two landmark reports in the March 26, 2014, issue of Nature and in 16 related articles in 10 other leading academic journals.

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