Incentives rise for TB drugs

Infected man spurs debate over payoff for new medicines

June 23, 2007

Tuberculosis has been flying under the American radar for decades now, as it has lost its status as a top killer in the U.S. and a particular problem in Baltimore. But when an infected Atlanta man launched a public health panic last month, it raised the deadly disease's profile anew.

There were about 14,000 TB cases in the country in 2005. But worldwide, particularly in the poorest regions of sub-Saharan Africa, there are 14 million people living with the deadly disease, which kills 2 million of them annually and is estimated to cost developing countries $12 billion a year. And it's morphing into new, drug-resistant forms, causing increasing concern among medical professionals.

Even so, no new TB drugs have been introduced in 30 years, in large part, some say, because pharmaceutical companies haven't seen blockbuster commercial potential.

The renewed attention to the disease, given the Atlanta case, is fueling a debate among some pharmaceutical companies and nonprofit advocates about how much money there is in treating TB and whether discussion about its limited potential is stifling drug development.

Diseases such as malaria and TB are relatively rare in the United States and other developed countries, but prevalent in poor parts of the world. That has led nonprofit foundations to step forward to fill in the gaps. The TB Alliance, for example, was created in 2000 and is considered a leader in the resurgence of research and development of TB drugs.

But Carol Nacy, chief executive of Rockville's Sequella Inc., believes TB drug development has been neglected because too many people assume that treating it is not a big moneymaker. Her company is one of a handful of drugmakers working to develop therapeutics for TB less because new treatments are needed and more because they say it's good for business.

"Is it a Viagra market? Probably not," she acknowledges. "Nobody wants to make a billion with TB, but you can make a reasonable return on your investment."

Nacy and some others -- industry analysts and other business professionals -- counter the claims of organizations like the TB Alliance over the monetary value of developing tuberculosis treatments.

They say there's an unrecognized market potential of between $350 million and $7 billion for each new drug. And until that's acknowledged, they argue, new cures won't reach the market because businesses won't develop them, new biotechs (like Nacy's) won't get funded by investors, and millions of people around the world will continue to die from a simple bacterium.

The TB Alliance, however, identifies only a fragmented market of about $315 million. That situation has helped it raise $200 million in donations for its work, the main goal of which is spurring new treatments. If the organization didn't exist, the TB Alliance said, few would be working in the space.

"There's never been the perception of a commercially viable marketplace that would induce companies to invest in research and development," said Al Hinman, a former CNN medical correspondent who now acts as a spokesman for the TB Alliance. Still, he recognized the need for company participation: They're the ones who find the new potential drug candidates.

Tuberculosis, which destroys body tissue, particularly in the lungs, was once a focus of the drug industry -- about 60 years ago when it was among the deadliest diseases in the United States and there wasn't a cure. But as antibiotic treatments were developed, its local prevalence fell, along with its place as a business booster.

Baltimore has actually led the country in TB control during the past 30 years, with rates falling from 50 cases per 100,000 people in the late 1970s to fewer than 10 cases per 100,000 today.

That would all be just fine if the current drugs were working well, but they're not.

"[They] were developed more than 40 years ago and are not working globally," Dr. Richard E. Chaisson, a Johns Hopkins University School of Medicine professor and director of the JH Center for Tuberculosis Research, wrote in an e-mail interview.

Today's standard treatment, though effective, requires taking a cumbersome and months-long combination of four antibiotics. It's a feat many patients never achieve simply because of the inconvenience, which means that the disease not only lives on, but has become stronger.

About 20 percent of all new TB cases are considered MDR, or "multidrug resistant," rendering standard therapy useless. Ten percent of those -- including Andrew Speaker's case -- are XDR, as in extensively drug resistant.

Speaker is the Atlanta lawyer who hopped a plane to Europe for his honeymoon last month, launching health officials into a public panic over potential spreading of the disease.

Speaker is now being treated in the United State, and he is expected to have surgery to remove an infected area about the size of a tennis ball from one of his lungs.

"New drugs are need[ed] to shorten the time required to cure TB and to provide treatment to those with drug-resistant TB," wrote Dr. Chaisson, who was attending TB conferences in France last week. "We also need new drugs that are compatible with HIV treatments, as many people with TB also have HIV and require treatment for both diseases."

Tuberculosis kills a person every 15 seconds, according to the World Health Organization. It's the second-leading infectious killer of adults worldwide, and it's the top infectious killer of people with HIV.

One-third of the planet's population is thought infected with the TB bacterium, though most cases, including 35 million in the United States, are considered "latent" -- inactive and symptom-free. Those with latent TB are more likely to develop active TB, however, unless they receive medication.

Such numbers add up to a potential $7.4 billion industry for new and improved drugs, along with another $1.7 billion for tests to diagnose TB -- according to a report released this month by Kalorama Information, the publishing division of Rockville's MarketResearch.com, a clearinghouse for industry data.

"I think most of the market figures are under-reported or underestimated," the report's author, Kenneth Krul, said in an e-mail interview. "Underestimate the market and you are likely to not put the effort into it that is necessary to develop it."

Krul's report contrasts with the $315 million May estimate by the TB Alliance, which looked at the earnings promise for the current generic drugs on the market in 10 countries and concluded it was fragmented with "limited commercial market potential."

That confirms a need for the TB Alliance to step in and stir things up, spokesman Hinman said.

"[We're] in it to make sure there is leadership. When we were created, no one was driving [research and development] for new and faster drugs," he said. "We bring together the public and private sector, we bring together big pharma, smaller biotechs, academia, government researchers."

There is more going on in TB drug development now than there has been in decades, and Hinman said that was because of the TB Alliance's efforts and philanthropic pharmaceutical companies, not necessarily because of any recognition of a viable market.

Many of the pharmaceutical companies doing research and development on TB medications say they have a social responsibility to do so.

Sanofi-aventis, for example, includes the disease in its "Access to Medicines" program, which makes treatments available to underprivileged populations under a tiered pricing plan with an overall "no profit-no loss" policy.

Not having access to medicine is "unacceptable," said Dr. Robert Sebbag, vice president of the Access to Medicines program, which is based in France. "We have decided it's our social responsibility" to provide treatments, particularly in "undeveloped countries."

But he also sees a viable market in the disease. Sebbag sits somewhere between Sequella and the TB Alliance, believing that the charitable attention focused on the disease -- and others including malaria and HIV -- has helped raise the earnings potential, in particular by providing a new source of funding through partnerships.

The TB Alliance is paying to send several tentative treatments through clinical trials, licensing the drugs from pharmaceutical companies, including Chiron Corp., or partnering with companies in some other way. An agreement with Bayer AG says the company will donate its drug and the TB Alliance will manage the trials.

Sequella, however, has partnered with the National Institutes of Health to develop a TB treatment known as SQ109, which is in early-stage clinical testing and hoped to be a better and faster treatment than what's available.

It's a "promising candidate" and one of at least nine tuberculosis drugs in clinical testing today, Anthony S. Fauci, director of the National Institute of Allergy and Infectious Diseases within the National Institutes of Health, told a Senate committee last month.

The TB Alliance is connected with at least three of the drugs in testing, but not Sequella's. Though the company has struggled at times to attract funding, Nacy said, it has also declined to work with the TB Alliance.

"I think my drug has value," Nacy said. "I'm not going to give it away."