A new model for developing cures is winning battles in the war against disease in laboratories, clinics and research centers around the world. The hallmarks of this movement are collaboration and the sharing of intellectual property in a wide-open manner that cuts against the grain of traditional for-profit drug discovery.

The new initiatives take many forms. Some target conditions ranging from Alzheimer’s to tuberculosis through alliances of foundations, nonprofits, universities, corporations and government agencies. Others make years of research available at the click of a mouse button to scientists around the world. Still others are carving out new financial and regulatory paths for bringing drugs to market at an accelerated pace.

All aim to break the cycle of frustration marked by the sharp decline in the number of new medicines that have been approved for use in recent years despite increased spending on research and development. Many new collaborations represent “very worthy skirmishes at the margins” of the pharmaceutical industry, says Garret FitzGerald, associate dean of the Institute for Translational Medicine and Therapeutics at the University of Pennsylvania Medical School.

Such efforts are needed, since simply “throwing more money at the problem won’t solve it,” says Terry Fadem, manager of the biosciences initiative at the Mack Center for Technological Innovation at Wharton. What’s required is “a significant change in the way we develop drugs.”

Spearheading the new initiatives are forward-looking organizations that bring their financial clout and strategic vision to the task of finding treatments. A look at some of these key groups and their trailblazing ventures:

Philanthropies and Nonprofits

The Bill & Melinda Gates Foundation:With $36 billion in assets, the Seattle, Washington-based foundation is one of the largest private philanthropies on earth and funds health and development projects around the world. It pumped $1.8 billion into global health programs in 2009 for projects ranging from drug discovery to family planning and the treatment of HIV/AIDS.

Among the groups backed by Gates is PATH, a Seattle nonprofit that used a $70 million grant to develop and deliver a meningitis vaccine to countries in sub-Saharan Africa in an unprecedently short time. The treatment was approved — or prequalified — by the World Health Organization in 2009 and introduced in Burkina Faso, Mali and Niger last December. “Never before had a new vaccine been prequalified and introduced at public-health scale within a year,” says Marc LaForce, who oversaw the project for PATH.

A key step was finding a company willing to develop and make a vaccine that would sell for less than 50 cents a dose, which was all that the impoverished countries could afford. A company called Serum Institute of India in Pune, India, took the job in 2004 and used technology transferred from the U.S. National Institutes of Health (NIH) to complete the work in five years. Plans now call for the vaccine to be given to more than 300 million individuals between the ages of one and 29 in 25 countries in Africa’s so-called Meningitis Belt.

In another Gates-funded effort, the San Francisco-based Institute for OneWorld Health took a novel approach to producing an affordable treatment for visceral leishmaniasis (VL), an infection of the spleen found in India and elsewhere that is transmitted by sand flies and can be fatal if left untreated. The institute, which styles itself the first U.S. nonprofit pharmaceutical company, found that an unused antibiotic that had gone off patent could work against VL. The drug went straight to phase III clinical trials — the final step before submission to regulators — since it had already been approved as an antibiotic. It is now approved for use in India and is to be clinically tested in Bangladesh and studied in combination with another drug in Nepal.

The Milken Institute:The 20-year-old brainchild of Michael Milken, a former investment banker who survived prostate cancer, is a nonprofit think tank that does research on issues ranging from health to energy and job creation. The institute’s FasterCures unit holds annual “Partnering for Cures” conferences that bring together researchers, investors, policy makers and others who seek to speed up drug development.

These sessions have produced some promising alliances. Among them: a partnership that enabled a biotechnology firm with an innovative cancer treatment to cross the so-called Valley of Death — the period between the discovery of a potential cure and clinical trials when research often dies for lack of funding. In this case, a source called the Cancer Vaccine Acceleration Fund agreed to provide cash and clinical test sites for the drug being developed by the biotech Tolerx based in Cambridge, Massachusetts. The partnership has “certainly accelerated” work on the treatment, says Christopher Merrill, senior director of business development for Tolerx, which launched a trial in December that might not otherwise have begun until 2012 or 2013.

Critical Path Institute (C-Path):Founded in 2005 in Phoenix, Arizona, this nonprofit serves as a catalyst for public-private partnerships to speed the development of drugs to combat major diseases. Current initiatives range from cofounding a global effort to find cures for tuberculosis to the release of a database gleaned from clinical trials involving more than 4,000 Alzheimer’s patients.

The tuberculosis initiative, called Critical Path to TB Drug Regimens, hopes to have treatments for the disease ready for submission to regulators in as few as six years. Making this possible are new FDA guidelines that let companies seek approval of treatments based on a combination of two or more drugs. More than one drug is needed to fight tuberculosis, says Thorir Bjornsson, who represents C-Path in the initiative, and readying any one drug for approval could take six years by itself. Research teams are currently working on nine separate compounds as part of the initiative, he notes, and “we may very well be one of the first, if not the first” to apply for approval of a combined treatment of the disease. 

Open-Source Research

Alzheimer’s Disease Neuroimaging Initiative (ADNI):A major hurdle to medical advances against Alzheimer’s is the difficulty of recognizing symptoms of the disease in their earliest stages. Since 2004 this groundbreaking initiative has made brain scans, plasma, spinal fluid biomarkers and other data collected from some 800 individuals at sites in the United States and Canada freely available on the Internet to Alzheimer’s researchers everywhere. Biomarkers are substances or measurements that can indicate the presence of disease. Based at the University of California, San Francisco, the five-year project was funded by grants from NIH and other agencies, as well as from drug makers and foundations, and has now been extended to 2015.

The project has accelerated understanding of Alzheimer’s disease through standardized collections of biomarker and clinical data from subjects whose conditions range from healthy to mildly impaired to those suffering from Alzheimer’s. “For years we’ve been bedeviled by the wide variations” in lab tests that are designed to provide evidence of the onset and progression of Alzheimer’s disease, says John Q. Trojanowski of the University of Pennsylvania School of Medicine. He and medical school colleague Leslie M. Shaw head a section of the initiative that looks for signs of the disease in blood and bodily fluids gathered from the cerebrospinal fluid that circulates around the brain and spinal cord.

The wealth of new data has helped Trojanowski zero in on evidence of the onset of Alzheimer’s in subjects before they manifest clinical symptoms of the disease. “The variation in biomarkers has been reduced from 30% to 40% down to 10%,” says Trojanowski. “This is a big advance. Our dream is to be able to have biomarkers” as clear as a blood-pressure measure that can signal hypertension so it can be treated before it leads to a heart attack or stroke. “We hope to develop similar predictive tests so that we can tell someone at 50 that he will have Alzheimer’s at 70,” and give treatment to prevent or ameliorate it, Trojanowski says.

Such open-source efforts are “terrific,” says the Mack Center’s Fadem. “ADNI creates an opportunity to study a large and significant body of patients,” he notes, and is a “phenomenally good” collaborative project.

New Directions for Industry

Eli Lilly:The company has been a leader among Big Pharma companies in innovative drug development. In 2002, the Indianapolis, Indiana-based drug maker formed a unit called Chorus that aims to speed discovery by swiftly identifying compounds with the best chance of becoming cures. Chorus designs early trials to show if a drug candidate can work in humans — a process called seeking proof of concept — and outsources the testing to a network of clinical providers. This eliminates the traditional costly and cumbersome step of screening myriad compounds in-house. Only those molecules that get a strong thumbs-up are then put through later-stage trials and submitted to regulators for approval.

The result? In December Lilly launched phase III trials for a treatment for rheumatoid arthritis and lupus, diseases of the joints that are often found together. Getting to phase III took one-third less time and money than the industry standard, says Christine Van Marter, communications director for Lilly research laboratories, thanks to the Chorus groundwork. Lilly is so pleased with Chorus that it plans to funnel 80% of its new compounds through the unit for early-stage screening.

New Government Initiatives

NIH National Center for Advancing Translational Sciences: In its biggest new launch in years, the NIH in December unveiled plans for a center to help streamline the process of turning promising discoveries into cures. The NIH, which currently operates 27 centers and institutes and is the world’s largest funder of medical research, said the new center could open its doors as soon as October when the government’s next fiscal year begins. The new center will bring together universities, drug makers, government agencies, nonprofits and others to pool their resources and development insights.

The goal is to get the drug pipeline flowing again. “Development of new treatments is kind of stuck at the moment despite big investments,” says Arthur H. Rubenstein, dean of the University of Pennsylvania School of Medicine and head of an NIH panel that recommended the new center. “This is an attempt by a lot of interested people to find a way to make the [development] process more efficient and end barriers to succeeding. Everyone agrees the paradigm is broken.”

The new center will house two additional newcomers to the NIH — the Cures Accleration Network (CAN) and Therapeutics for Rare and Neglected Diseases (TRND) — that aim to speed up drug discovery. The U.S. Congress included CAN in the 2010 health care reform act to award grants to support “revolutionary advances” in medical research. Meanwhile, Congress launched TRND in 2009 to work with researchers seeking treatments for conditions like hookworm and sickle cell anemia that have received relatively little attention from big pharmaceutical companies.

What impact will all these public and private ventures have on the task of creating new cures for people around the world? “Every one of these efforts is trying to address a specific need in the market,” says Fadem. “They could become a template for future drug development.”