In theory, a company called Avant Immunotherapeutics Inc. may have a solution for the problem of cholera, a resurgent disease that kills young children in poor countries. Avant is working on a vaccine that could offer years of protection.
But it's anybody's guess whether that vaccine will ever reach the people who need it most.
Avant, of Needham, Mass., is supplying doses for early trials in Bangladesh, but the small biotechnology company isn't sure it can find money for the large-scale tests that would prove the vaccine's usefulness in protecting poor youngsters.
"It's just a crying shame," said Una S. Ryan, who runs Avant.
But an answer to this kind of problem might be at hand.
In an ambitious new plan to be unveiled this week, the American biotechnology industry plans to join forces with two of the country's leading foundations in an effort to get new medical technologies to the world's poorest people.
The annual convention of the Biotechnology Industry Organization, a Washington trade group, is scheduled to begin today in San Francisco. In one of its most significant moves in years, the group, known as BIO, is establishing a new organization with help from the Rockefeller Foundation and the Bill and Melinda Gates Foundation.
The not-for-profit Washington group, BIO Ventures for Global Health, to be announced Tuesday, will be dedicated to solving the business problems that small biotechnology companies confront in trying to create products for poor countries.
The group itself will be relatively small, 10 or 20 employees, but its aim is to put together a string of deals, costing from $3 million to $15 million apiece, through which foundations and other charitable donors would help finance development of new products like vaccines that could be deployed overseas.
The group will also cultivate expertise to help small companies pick their way through the complex business issues associated with using cutting-edge genetic technology to develop products that have to be sold overseas at very low prices.
"We are thinking of this as a bold experiment," said Wendy Taylor, who was hired to be the group's new chief executive as plans took shape in Washington over the past 18 months. "These biotech chief executives are entrepreneurs, and they'd love to be able to come up with the world's first malaria vaccine or HIV vaccine. They need not only the money to move the projects forward, but tools to help them think about operating in these markets."
BIO Ventures for Global Health is part of a broader push in the world of public health, backed with serious money from the Gates and Rockefeller foundations, to solve a series of vexing business issues that have prevented vaccines and other lifesaving medical technologies from reaching the poor.
The problem is not so much funding the basic science that might provide needed medical technologies. Congress has for many years been willing to finance work on foreign diseases that don't afflict the United States. The National Institutes of Health and U.S. military doctors have used that money to produce several important scientific insights, and even potential vaccines.
But public-health experts have realized, to their dismay, that an NIH scientist holding up a test tube in Bethesda and shouting, "Eureka!" doesn't begin to solve medical problems in poor countries. A multitude of logistical, scientific and business problems stands between that test tube and a sick child in, say, Kenya or Nepal.
For starters, the government itself doesn't have much ability to manufacture medicines and has generally done a poor job when it has tried, experts said. So the job has fallen to private industry. The task can involve spending tens or hundreds of millions to run large-scale tests, build factories, develop complex manufacturing lines, ensure rigorous quality control -- and do it all under stringent cost controls that mean the vaccine or drug can be sold at a profit.
With drugs intended for rich countries, firms can tap venture capitalists or stock investors to finance the work, since there's a promise of profit -- often big profit -- at the end. But that hasn't worked nearly as well for products meant partly or entirely for poor countries.
There's so little money involved that many private investors simply aren't interested. The health budgets of the world's poorest countries, added together, don't equal the sales of a single blockbuster rich-country drug for ulcers or heart disease.
The problem has grown more acute in recent years as the genetic research pouring from the world's laboratories has opened new horizons. It is now an article of faith among many scientists that they will be able to create vaccines against humanity's great scourges: malaria, tuberculosis and other diseases that kill millions every year in poor countries. But can they get those products onto the market and into the villages where they are needed most?
Much of the necessary technology is being developed not in big drug companies, which can afford a certain amount of charity, but in small, scrappy American biotechnology companies. These companies, whose work is based entirely on genetic knowledge, are the source of much of the innovation in medicine today, but they are often tiny outfits with fewer than 100 employees.
Few of those companies have figured out business plans that might allow them to take a product all the way through the development cycle and put it on the market for use in poor countries.
That's where BIO Ventures for Global Health aims to come in. In some ways, the group is built on a model that the Rockefeller and Gates foundations have already spent years developing.
The foundations have financed several piecemeal ventures aimed at tackling the business problems -- usually by directly subsidizing some of the testing and development work that private investors won't finance. Several of these ventures, such as initiatives to create new malaria medicines or AIDS vaccines, have achieved research milestones, though none has yet put a major product on the market.
Although the organizers of BIO Ventures for Global Health don't argue with the piecemeal approach, they say they see a need for a more systematic effort to identify potentially useful technologies, help companies devise business plans and persuade foundations and other donors to put up big money to finance the work.
The Gates and Rockefeller foundations were persuaded enough by the argument to contribute more than $1 million for start-up costs. "We are fully behind it," said Richard D. Klausner, director of global health for the Gates Foundation. "We believe there's a pressing need for products for the developing world for global health. It's an enormous set of opportunities, particularly for small companies."
It is unclear how much money will be forthcoming from his foundation or other donors. Perhaps the biggest single need is financing for late-stage trials that can provide definitive evidence that a new drug or vaccine works. Such tests can cost hundreds of millions.
The Gates foundation, with more than $20 billion in assets, has that kind of money, but Klausner emphasized that each proposed deal will be subjected to rigorous scrutiny to make sure it serves charitable ends. "I don't want to be pinned down" about the sums the Gates foundation might commit, he said.
Although some medicines might find markets only in poor countries, others have dual uses in the developed and developing worlds. That could be the case with Avant's cholera vaccine.
Vaccines already exist for cholera, an intestinal ailment spread through poor sanitation that can kill children rapidly. But the vaccines are so mediocre, offering only a few months of protection at best, that they aren't widely used. Early studies suggest Avant's vaccine might work better.
Aside from its obvious potential in countries afflicted by cholera outbreaks, the vaccine could find a paying market among travelers or aid workers worried about cholera. That means there's some chance private investors can be persuaded to finance part of the work, if foundations also finance a chunk of it.
Early tests in Bangladesh have drawn support from a vaccine institute backed by Gates foundation money, but far more expensive tests loom.
Avant is likely to be one of the first in line when BIO Ventures for Global Health opens its doors.
"If I could get help that would allow me quicker access to my real markets, where I could charge a traveler $50 or something, I am quite willing to give away my rights and see a plant in a developing country make our vaccines" for low-profit overseas markets, Ryan said. "We are willing to do some charity here, in exchange for something that would actually move our products forward."