drug discovery

Nature Outlook: Amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS) is a devastating disease with poorly understood causes and no known cure. But research is slowly beginning to bring hope to those affected. This Outlook discusses topics such as: how genetic and epidemiological research are beginning to reveal the secrets of ALS; new drugs and other treatments that are finally becoming available; and the lessons that the ALS Ice Bucket Challenge offers for funding disease research. Read more in this Nature Outlook I edited.

Faster, deeper, smaller—the rise of antibody-like scaffolds

In early May the pharmaceutical giant AstraZeneca completed a deal with Boston-based Pieris Pharmaceuticals worth up to $2.1 billion to bring Pieris’ anticalin asthma drug PRS-060, an engineered protein that mimics antibodies, to the clinic. And on June 1, Bicycle Therapeutics in Cambridge, UK, pulled in $52 million in a series B funding round with several high-profile investors to continue developing its bicycle peptides for a variety of cancer types.

Those are just two of the wide variety of protein scaffold drugs currently in development. “There’s a whole zoo of non-antibody scaffolds out there,” says Daniel Christ, an immunologist at the Garvan Institute of Medical Research in Sydney, Australia. Read more in Nature Biotechnology.

Rhinovirus vaccine development is about more than fighting colds

Rhinovirus, the pathogen behind the common cold, can cause severe, acute lung disease in children and those with underlying respiratory conditions. Since the 1970s, vaccine development has been hindered by the presence of numerous virus serotypes and the lack of a good animal model to test vaccine candidates. However, several different research groups are now making good progress on rhinovirus vaccines, using a variety of different techniques.

The researchers working on a vaccine for rhinovirus, the infection that causes the common cold, are all clear on one important point — they’re not trying to cure the sniffles.

“Rhinovirus is more than just a nuisance,” says Martin Moore, a virologist at Emory University in Atlanta, Georgia. “Sniffles in adults are not why we are doing this.” Read more in The Pharmaceutical Journal.

Solithromycin rejection chills antibiotic sector

The US Food and Drug Administration (FDA) in December rejected the new antibiotic solithromycin over liver toxicity fears, putting the future of the drug in doubt and sending a chill through companies working on novel antimicrobials. “The problems with solithromycin are going to hit the whole sector hard,” says Lloyd Czaplewski, director of Chemical Biology Ventures, a pharmaceutical R&D consultancy in Oxford, UK. “It could put people off getting into an area where we really need some successes.” Read more in Nature Biotechnology.

Outlook: Multiple sclerosis

Multiple sclerosis is a devastating disease that induces the body’s own immune system to eat away at the central nervous system, slowly robbing patients of their physical mobility. It is also mysterious. Despite years of research, the cause remains elusive, and treatments are few and far between. But new research to find the causes and provide innovative treatments means that progress, although still slow, is beginning to speed up. Read more in this Nature Outlook that I edited.

Nature Outlook: Kidney cancer

Kidney cancer has long flown under the radar despite being one of the top-ten cancer killers worldwide. It lacks the research spotlight and public awareness of other cancers that can help to drive new discoveries. It remains hard to detect, difficult to treat and poorly understood. But that is starting to change as researchers dig into the mysteries surrounding the disease. Read more in this Nature Outlook that I edited.

A ray of hope amid antibiotic gloom

As antibiotic resistance continues to threaten the treatment of various infections, researchers are looking for new ways to supplement and in some cases replace failing antimicrobial drugs.

When it comes to tackling infections, we’ve had it pretty good for the past 90 years. The development of antibiotics has turned many previously deadly infections into mere inconveniences, but it couldn’t last forever. Slowly, bacteria have fought back, developing resistance to many of the most effective drugs. In the United States alone, around 2 million people are infected with resistant strains of bacteria each year, and at least 23,000 of these patients die.

“We’re at the end of the first antibiotic era,” says Lloyd Czaplewski, founder of Chemical Biology Ventures, an R&D consultancy based in Oxfordshire. “There might not be any new classes of drugs to discover.”

New ideas are needed. And while most researchers and pharmaceutical companies have all but given up on developing new antibiotics, work is racing ahead on alternative therapies, with an aim to extend the life of existing drugs, or replace them altogether. Read more in The Pharmaceutical Journal.

Data sharing: Access all areas

Advocates say that open science will be good for innovation. One neuroscience institute plans to put that to the test.

In the cut-throat world of early-stage clinical development, where aggressive defence of data and intellectual property is thought to be key to amassing profits, one academic institute is opting out.

Over the next five years, McGill University’s Montreal Neurological Institute and Hospital (the Neuro) in Canada will conduct a radical experiment in open science. It will make all results, data and publications from its research free to access, will require collaborators to do the same, and, perhaps most surprisingly, will not pursue patents on any of its discoveries. Read more in Nature.

Profile: The Scripps Research Institute under new leadership

Steve Kay, the new president of The Scripps Research Institute, has big plans for the venerable biomedical research institution. Alongside incoming CEO Peter Shultz, he hopes to extend the institute’s mission beyond basic science to incorporate more translational work, in a complete bench-to-bedside approach. “If you can couple the strength in basic sciences of an institute like Scripps with real pipeline capability to make drug candidates, then you could make a really unique institute that is centred around therapeutics for unmet medical needs”, he says.

The principal way in which this transformation will be accomplished is through close cooperation with the California Institute for Biomedical Research (Calibr), an independent drug discovery institute of which Shultz is the director. Kay says he and Shultz want to “align, anneal, and eventually merge” the two institutes to create one with complete pipeline capabilities from basic discovery research to early-stage clinical work. “We want to join the drug discovery capability of Calibr with the brain trust at Scripps”, says Kay. Read more in The Lancet.

Drug development: The treasure chest

Pharmaceutical research into the chemicals found in cannabis has so far supplied only one licensed medicine. But scientists think there could be hundreds more.

The annual meeting of the International Cannabinoid Research Society (ICRS) is a highly unusual scientific conference. It has been closed to all media since its inception 25 years ago, lending an air of mystery to the gathering of researchers who study the unique chemicals found in cannabis.

In a relaxation of the organization’s long-standing policy, ICRS permitted Nature reporters to attend this year’s conference, which was hosted by Acadia University in the tiny Canadian town of Wolfville, Nova Scotia. The tight-knit group of researchers are bound together by onerous government restrictions on their subject, and by their sufferance of lingering suspicions from other scientists that they are a bunch of hippies trying to get an illicit drug legalized.

“The status of cannabis as an illegal substance makes it difficult for some people to take it seriously,” concedes Mark Ware, a pain specialist at McGill University in Montreal, Canada, who focuses on the analgesic properties of cannabis. Read more in Nature.