Staff Writers

Editor's Note: This is the last of a two-part series on neglected tropical diseases. Part one ran in Thursday's issue.

Neglected tropical diseases affect close to half the world's population. Lacking venture capital, it is the half that needs innovative funding strategies if it is to see new drugs for its afflictions.

One factor leading to the neglect of certain diseases is that they occur in rural areas of the developing world, which means that they are that much more remote from being a threat to Westerners. "There's no travelers' market, there's no military market" - which are the opportunities that entice companies to focus on malaria and tuberculosis, said Peter Hotez, chairman of the department of microbiology and tropical medicine at George Washington University.

But business models, and success stories, do exist.

"There's an enormous amount of non-dilutive financing available for work on neglected diseases today," said Christopher Earl, president and CEO of BIO Ventures for Global Health (BVGH). Such money, which could be used both for R&D and product purchases, is available from governments, public-private partnerships, product development partnerships, international agencies, non-governmental organizations and philanthropic groups.

Earl said that by employing "sound business strategies," biopharma companies should be able to use their drug discovery platforms to generate products for neglected tropical diseases. Everyone acknowledges the unmet medical need, so the industry needs to recognize the market pull that's taking shape.

One example of success that has arisen with philanthropic backing as a pull mechanism is the Institute for OneWorld Health, a nonprofit pharmaceutical organization based in San Francisco.

"We're trying to develop a funding model and an R&D model to get medicines to the poorest of the poor in the world," explained David Brown, head of drug discovery for the institute. Obviously that doesn't fit more typical investor-driven models, he said, because "nobody expects a return in the usual sense." Instead, the expectation is to save lives in the developing world.

Founded at the beginning of the decade and run by longtime drug industry executives, the organization recently received approval for paromomycin, a long-approved antibiotic typically used for intestinal infections, to treat visceral leishmaniasis in India. The frequently lethal parasitic disease is especially endemic in the northern Indian state of Bihar.

"We've proven we can actually do what a big company does," Brown said, "within this model."

He couldn't put a finger on a for-profit business making a go of it in this field, but he did note that OneWorld Health is partnered with many large pharmaceutical firms for certain "in kind help, applying their resources to help us," which is in addition to such companies' benevolent donations of drugs to the poor.

Brown also sounded optimistic about the future for treating neglected tropical diseases, with multiple high-profile philanthropic efforts on the map these days to back efforts such as the OneWorld Health model. "There's been a big change in the last three years," he said of the momentum. "The timing is perfect."

The Sabin Vaccine Institute is using a similar strategy to develop a hookworm vaccine. Existing drugs could do a lot to alleviate the burden of many neglected tropical diseases. But hookworm, which affects more than half a billion people globally, is usually reacquired through the soil within a year of a deworming drug regimen - and repeated regimens can lead to drug resistance.

For this reason, the Sabin Vaccine Institute is working on vaccines as an alternative strategy to drugs. Hotez, who is on the Institute's scientific advisory council, joked that "it's a guaranteed money-losing company - it's like those guys in [the Broadway hit] The Producers."

In September, the Institute announced that it had received a $13.8 million grant from the Bill and Melinda Gates Foundation, which it will use to develop a vaccine that targets the adult stage of the hookworm. A complementary vaccine targeting the larval stages has already been tested in Phase I trials, and testing is slated to continue in rural Brazil.

Hotez, whose energy is apparently boundless, will also serve as editor-in-chief for the open-access journal PLoS Neglected Tropical Diseases, which the Public Library of Science (PLoS) is launching next year. PLoS NTD Consulting Editor Gavin Yamey told BioWorld Today that currently "there is not a journal at all devoted to these diseases - and that's another form of neglect."

The PLoS hopes its NTD journal will help bring attention to neglected tropical diseases by two mechanisms. Providing a high-quality publishing outlet makes it that much more attractive to do research into such diseases from a career perspective. But more directly, and importantly, the fact that all PLoS journals are open-access means that the research will be accessible to scientists in countries that may ultimately have the best confluence of factors to vanquish these diseases. These are the so-called "innovative developing countries" where neglected tropical diseases exist, but which have managed to build up a research infrastructure to some degree. The list includes a dozen Asian, African, and Latin American countries such as China, India, Mexico, Cuba, South Africa and Senegal.

Such countries have an obvious interest in making neglected tropical diseases a research priority, but they also have far fewer resources than the West. "A typical journal article reprint costs $30 to $40," Yamey said, which is higher than the annual health budget per person in some of the poorest countries that are hard-hit by NTDs, and a tidy sum even in most innovative developing countries, which are not the poorest of the poor. An open-access journal, he said, could help with a crucial goal: "to build the capacity for those in countries with endemic diseases to tackle these diseases themselves."