Alzheimer's Researchers Have Five New Genes to Ponder

From fiercebiotech.com

New insights into the most baffling diseases of our day often, frustratingly, go in stops and starts. But when it comes to understanding Alzheimer's disease, the discovery of five more genes thought to be responsible for the degenerative brain disease is considered a huge leap forward. The discoveries do not mean that a cure is on the horizon, but it does mean that scientists are a step closer in understanding the role our genes play in this debilitating, costly disease and could eventually lead to new methods of detecting Alzheimer's much earlier in life. The next step is more research into how all these genes work together to produce Alzheimer's.

First, here's the news: In what is being billed as the largest study of its kind, a consortium of 44 research institutions identified four new genes that indicate Alzheimer's disease risk. Another group of investigators from the United States and Europe reported a fifth gene. This news doubles the numbers of genes now thought to contribute to Alzheimer's disease.

Bloomberg reports that the new discoveries raise the risks for Alzheimer's by 15 percent or less, so they are not necessarily markers for the disease. However, they are helping researchers "piece together the jigsaw and gain new understanding," lead researcher Julie Williams told Bloomberg.

Rudolph Tanzi, a neurology professor at Harvard Medical School, told Bloomberg that he is most excited by one particular newfound Alzheimer's gene: CD33. It might be responsible for a failure of the body to eliminate as much beta amyloid as it should. Beta amyloid buildup has long been thought to play a key role in degradation of nerve cells in the brain.

Still, it will be a decade or more before any of these discoveries will translate into drugs, but it has the Alzheimer's research community buzzing over new avenues of possible inquiry. The research can be found in the journal Nature Genetics.

Melanoma-seeking Nanoparticle Enters Testing

From Fiercebiotech.com

Researchers are targeting melanoma with a nanoparticle that's been in development for more than 10 years. The nanoparticle is designed to not only hunt down cancer, but also to highlight its spread through the body. Successfully tested in animals, the nanoparticle will be tried in five melanoma patients this year.

Once melanoma spreads beyond the skin, it's difficult to find and treat. "There's never been a targeted therapy for melanoma," says Michelle Bradbury, a radiologist at Memorial Sloan-Kettering who's leading the clinical trial. If the nanoparticle can track melanoma's spread, it could make a big difference in treating that cancer.

But Bradbury and her team are also hoping that the nanoparticle can serve as an optical imaging agent to help find cancer-containing lymph nodes. That would allow surgeons to remove all the cancer with as little cutting as possible.

This development uses nanoparticle technology to help find cancer cells. Much optimism in the cancer community surrounds using nanoparticles to treat cancer, perhaps one day even eliminating the whole-body poisoning approach of current chemotherapy regimens. MIT recently opened a $100 million cancer research center, funded by billionaire David Koch, with one of its goals being the development of nanoparticle delivery systems for cancer treatements.

Read more in the MIT Technology Review.

New Breast Cancer Drug Found Deep in the Sea

From the Wall Street Journal

Amid a dry spell for breakthrough cancer drugs, recent U.S. approval of Eisai Co.'s Halaven represents some vindication for a small group of researchers who believe, contrary to recent pharmaceutical fashion, that molecules from nature hold promise against hard-to-treat diseases.

The Food and Drug Administration's approval of Halaven in November for treating late-stage breast cancer was a triumph of chemistry and tenacious research. Its path, extending nearly three decades from the first studies, demonstrates not only potential benefits but also some of the hurdles in the hunt within nature's bounty for drugs of the future.

Primitive creatures developed many clever ways to kill each other after billions of years of evolution, and some can be turned to human use. "Weapons of mass destruction are alive and well on a coral reef," says David Newman of the National Cancer Institute, who has studied the subject for decades.

Halaven derives from halichondrin B, a substance identified in a black sponge that lives off the coast of Japan. Studies showed it has a powerful effect on tumors, blocking cell division in a way that scientists hadn't previously thought possible.

Yoshito Kishi, a Harvard University chemistry professor, synthesized halichondrin B with funding from the National Cancer Institute. His work galvanized researchers at Eisai of Japan, who identified the active part of the molecule and, working with Dr. Kishi, went on to create the drug. Chemists say Halaven is among the most complex small-molecule drugs ever made commercially.

Read the full story.

Gut Bacteria May Protect From Allergy And Autoimmunity

Scientists at the University of Tokyo have discovered that specific gut-dwelling bacteria control key immune cells that combat allergies and autoimmune diseases. The paper describing the findings was published in late December in the journal Science, and technology originating from the work is being advanced by the start-up Vedanta Biosciences. 

Dr. Kenya Honda and colleagues initially showed that mice raised in sterile conditions had deficient levels of regulatory T cells in their colon compared to mice colonized with microbes.  "Regulatory T cells are the peace-keepers of the immune system," says Dr. Alexander Rudensky, tri-institutional Professor at the Memorial Sloan-Kettering Institute, the Rockefeller University, and Cornell University, whose previous work helped describe this specialized subset of T cells. "When these cells are missing, the body has trouble putting the brakes on excessive immune responses."

The team of researchers led by Honda then zeroed in on a subset of gut microbes belonging to the Clostridia class that were responsible for triggering production of regulatory T cells. Feeding these "Good Clostridia" to mice prevented the development of allergies and inflammatory bowel disease.  "For millions of years, we have co-evolved with our gut microbes, communicating in an ancient language that may hold critical clues about how autoimmune and infectious diseases develop," said Dr. Ruslan Medzhitov, Professor of Immunobiology at Yale, who authored pioneering studies of the innate immune system."Researchers like Dr. Honda are making important strides to begin decoding this complex language."

This newly understood beneficial role of certain species of Clostridia may be reminiscent of the opposite effects on health of "good" and "bad" cholesterol.  While some species are harmful pathogens, such asC. difficile, many other Clostridia species are harmless or even beneficial as illustrated by Honda's work."These surprising findings suggest we should revisit how we view this class of microbes," said Dr. Dan Littman, Professor of Molecular Immunology at NYU, whose previous work shed light on the development of Th17 cells in response to microbial signals. Dr. Littman added that Honda's findings "open up new treatment options for preventing and treating a number of immune-related disorders."

Honda's paper is the latest of a series of recent high profile publications that have followed the launch of the Human Microbiome Project by the NIH, and of similar international efforts aimed at exploring the roles of the human microbiome in health and disease. "The human microbiome is an exciting area of research that may enable a completely new class of therapies," said Dr. Ben Shapiro, Chairman and Senior Partner of PureTech Ventures and former EVP Worldwide Basic & External Research at Merck. "PureTech has made a significant commitment to translating research related to the microbiome, and we have a number of exciting technologies and programs under development."

A Single Vaccine Cure for Rheumatoid Arthritis?

A team of scientists at Newcastle University is preparing a small human trial to test a one-shot approach to curing rheumatoid arthritis with a vaccine devised from a patient's white blood cells. The cells are taken from the volunteers and manipulated with a recipe of chemicals, steroids and Vitamin D so they can suppress the immune system rather than spur it. And a single jab could be enough to suppress the auto-immune response that triggers rheumatoid arthritis.

"This is an important potential cure. It is possible one injection could switch off the abnormal immune response," Prof Alan Silman, medical director of the charity Arthritis Research Campaign, which funded the research, told the Telegraph. "If it works it could reverse the disease and stop further episodes."

See the source article.