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Scientists have created a powerful new tool to calculate a person’s inherited risks for heart disease, breast cancer, type 2 diabetes, chronic inflammatory bowel disease, and atrial fibrillation...By surveying changes in DNA at 6.6 million places in the human genome, investigators at the Broad Institute and Harvard University were able to identify many more people at risk than do the usual genetic tests, which take into account very few genes...The researchers are now building a website that will allow anyone to upload genetic data from a company like 23andMe or Ancestry.com. Users will receive risk scores for the five aforementioned diseases. A risk score, including obtaining the genetic data, should cost less than $100...The study began because there was general agreement among researchers that many common diseases are linked not to one mutation, but rather to thousands or millions of mutations...scientists have cataloged more than 6 million tiny changes in DNA that slightly affect the chances that people will get various diseases. Each of those genetic alterations has such a small effect—approximately a 1 percent increase or decrease in a person’s odds of getting a disease — that it would not be helpful to test for each one in isolation...But...to combine data on all of the small DNA changes to construct an individual risk score. To do that, the researchers needed a new algorithm that would weigh the significance of the variations in the genes.

Scientists often fail to publicly register plans for clinical trials or to publish the results, and the outcomes they do share may mask instances when new treatments are unsafe or ineffective, a small study suggests...One goal of asking scientists to register clinical trial plans in public research databases is to highlight the main objective of experiments and make it easy to see whether a tested treatment achieved this goal when results are published, the study authors note in JAMA...But more than one-third of the 113 clinical trials researchers examined for the study were never registered. Only 64 of the trials, or 57 percent, were published...One in five trials didn’t define a primary outcome, or clear protocols for determining if the tested treatment had achieved its main goal...Doctors rely on published evidence to guide patient care decisions, while researchers use the published literature to guide which promising areas of inquiry to pursue...Unpublished trials tend to be the ones that found treatments didn’t work or weren’t safe...Published results, meanwhile, tend to highlight successful experiments...

An eye for an eye, a tooth for a tooth — a life for a lab book?...In the past few months, China has announced two new crackdowns on research misconduct — one of which could lead to executions for scientists who doctor their data...Scientists have been sounding alarms for years about the integrity of research in China. One recent survey estimated that 40 percent of biomedical papers by Chinese scholars were tainted by misconduct. Funding bodies there have in the past announced efforts to crack down on fraud, including clawing back money from scientists who cheat on their grants...Chinese...Ministry of Science and Technology proclaimed a "no tolerance" policy for research misconduct — although it’s not clear what that might look like...the mass retractions "seriously harmed the international reputation of our country’s scientific research and the dignity of Chinese scientists at large."...courts approved a new policy calling for stiff prison sentences for researchers who fabricate data in studies that lead to drug approvals. If the misconduct ends up harming people, then the punishment on the table even includes the death penalty...

The industry is readying for a leap into a new age of complex therapies, as major advances seem mere steps away from market approval. Regenerative cell-based therapies, CAR-T and immuno-oncology combinations are just some of the fields where researchers are reaching for new heights that could alter the treatment paradigm. Elaborate manufacturing and rising drug costs, however, loom as deep chasms to cross.

• CAR-T cutting it close
• Combos, to name a few
• A PD-1 backbone?
• Targeted therapy: Is that still a thing?
• NASH players
• State of Alzheimer’s
• Moving fast in Zika
• An eye on complexity
• The verdict: A pipeline of puzzles

...As researchers blaze the meandering and thorny path toward curative treatments, a clearing is visible on the horizon. But to get there, the industry must confront a daunting chasm—making the previous generation’s small molecule-to-antibody transition look like an easy stride across a tame stream....

MIT researchers, working with scientists from Brigham and Women’s Hospital, have developed a new way to power and communicate with devices implanted deep within the human body. Such devices could be used to deliver drugs, monitor conditions inside the body, or treat disease by stimulating the brain with electricity or light...The implants are powered by radio frequency waves, which can safely pass through human tissues...in animals, the researchers showed that the waves can power devices located 10 centimeters deep in tissue, from a distance of 1 meter...Even though these tiny implantable devices have no batteries, we can now communicate with them from a distance outside the body. This opens up entirely new types of medical applications...An overarching aspiration is that regulators will provide input to the design and may incorporate framework elements and learnings into regulatory programs. .

A research team led by scientists at UC San Francisco has developed a computational method to systematically probe massive amounts of open-access data to discover new ways to use drugs, including some that have already been approved for other uses...The method enables scientists to bypass the usual experiments in biological specimens and to instead do computational analyses, using open-access data to match FDA-approved drugs and other existing compounds to the molecular fingerprints of diseases like cancer. The specificity of the links between these drugs and the diseases they are predicted to be able to treat holds the potential to target drugs in ways that minimize side effects, overcome resistance and reveal more clearly how both the drugs and the diseases are working...Our hope is that ultimately our computational approach can be broadly applied, not only to cancer, but also to other diseases where molecular data exist, and that it will speed up drug discovery in diseases with high unmet needs...I’m (Bin Chen, PhD) most excited about the possibilities for applying this approach to individual patients to prescribe the best drug for each...

A comprehensive map of all existing drugs and how they work, created from information from several huge datasets, could be used to kick-start the next wave of drug discovery. Not only could scientists use the map to suggest promising new targets for diseases as diverse as cancer, mental illness and inflammatory conditions, it could also provide invaluable insight into where existing drugs can be used to treat different diseases...Jointly led by scientists at the Institute of Cancer Research...the research brought together vast amounts of information from datasets including the canSAR database...the ChEMBL database...the...DrugCentral database to create a map of all 1,578 licensed drugs and their mechanisms of action...Dr Bissan Al-Lazikani, explains how this first-of-its-kind map could help tackle one of the biggest challenges facing the drug discovery field: how do you innovate without taking risks that make the whole system unviable?

• Why did you decide that a comprehensive map of all existing drugs and their mechanisms of action was needed for the drug discovery community?
• How did you put the map together? It must have been quite a task!
• What were the most interesting findings of the latest study?
• What are the biggest challenges facing the drug discovery field at present and how will your comprehensive map help overcome these?

Scientists around the globe nowadays regularly take to the internet to scrutinize research after it’s been published — including to run their own analyses of the data and spot mistakes or fraud...And as interest in this so-called post-publication peer review has swelled, one lawyer argues, biotech and pharma companies would do well to take note. If companies and their investors aren’t reading these sites, they may be the last to know when industry-funded research is called into question...The most prominent forum for these discussions is PubPeer, where anyone can create an anonymous account and comment on published research papers. Many of the posts on PubPeer point out instances of plagiarism and duplicated or manipulated images — all of which represent retractable offenses and serious scientific misconduct. But posters often note erroneous data, flawed methodology, or even just sloppy thinking that somehow escaped the scrutiny of peer reviewers before the article was published.

An evolutionary biologist at the University of Houston has published new calculations that indicate no more than 25 percent of the human genome is functional. That is in stark contrast to suggestions by scientists with the ENCODE project that as much as 80 percent of the genome is functional...In work published online in Genome Biology and Evolution, Dan Graur reports the functional portion of the human genome probably falls between 10 percent and 15 percent, with an upper limit of 25 percent. The rest is so-called junk DNA, or useless but harmless DNA…this new study...will help to refocus the science of human genomics...“We need to know the functional fraction of the human genome in order to focus biomedical research on the parts that can be used to prevent and cure disease,” he said. “There is no need to sequence everything under the sun. We need only to sequence the sections we know are functional.”

Scientists have mapped all 1,578 licensed drugs licensed by the US Food and Drug Administration according to their mechanisms of action to help researchers visualize the ‘uncharted waters’ where they may find future treatments...Scientists at The Institute of Cancer Research in London...extracted data from their own drug database, as well as databases at European Bioinformatics Institute in Cambridge and the University of New Mexico. They matched each drug with prescribing information and data from published scientific papers to build up a picture of how each existing medicine works. The analysis, published in Nature Reviews Drug Discovery (A comprehensive map of molecular drug targets) reveals that there are just 667 unique human proteins targeted by existing approved drugs (or only 3.5% of the estimated 20,000 human proteins), and a further 189 drug targets in pathogenic organisms...This new map of drugs, created through the latest computational analytical technologies, will enhance our ability to use rational, data-driven approaches to identify the most promising future targets and treatment combinations for the next generation of cancer and other diseases...