Research Roundup: What we’re reading this week

Recently, a US Food and Drug Administration (FDA) panel recommended that the HIV and AIDS drug Truvada be approved for HIV prevention among certain groups at risk of infection. Known as pre-exposure prophylaxis (PrEP), this prevention method involves HIV-negative individuals taking antiretroviral drugs to prevent HIV infection. This recommendation from the FDA panel does not oblige the agency to follow suit; however, it often does. The Truvada hearing is illustrative of the important role that the FDA plays in the research, review, regulation, and licensure of global health technologies—both those that are brand new, as well as reviewing new uses for current tools. Indeed, the FDA has a vast amount of expertise that it can leverage to help address regulatory issues in the developing world, where countries often lack the expertise or resources to appropriately review new health tools. In addition to the regulation and review of health products, the FDA also plays a small but important role in research and development (R&D) for global health. Starting in 2010, the FDA engaged in R&D funding for new drugs to combat tuberculosis (TB) though its work with the Critical Path Institute. The Critical Path to TB Drug Regimens brings together leading pharmaceutical companies and other drug developers, global regulatory agencies, and nongovernmental organizations—like GHTC member the Global Alliance for TB Drug Development—to facilitate the development and availability of new TB treatments. In addition to the regulation and review of health products, the FDA also plays a small but important role in research and development (R&D) for global health. Photo credit: PATH/Evelyn Hockstein FDA clearly recognizes the importance of its position in the global regulatory world, as evidenced by its recent Pathway to Global Product Safety and Quality report. It’s equally important that Congress recognize the FDA’s increasing role in global health, and that it works with the agency to provide the funding needed to sustain and expand these activities. This week, the House Agriculture, FDA, and Related Agencies Appropriations Subcommittee will debate its version of fiscal year (FY) 2013 appropriations legislation. And recently both the House and the Senate approved very similar versions of the Food and Drug Administration Safety and Innovation Act (FDASI), including reauthorizations of the Prescription Drug User Fee and Medical Device User Fee programs – which provide a large chunk of FDA’s operating expenses, and must be reauthorized by September 30th. Included in FDASI is the Generating Antibiotic Incentives Now (GAIN) Act, a provision which would extend data exclusivity for new antibiotics to combat drug resistant bacteria—including multi-drug resistant TB—by five years. While incentives that engage the private sector in product development are urgently needed, data exclusivity alone may not solve the gap in antibiotics R&D, and should be combined with several other incentive mechanisms in order to truly leverage the knowledge and resources of the industry. The legislation does request a study be conducted outlining the use and effectiveness of such incentives, a positive step in the right direction. The next few months are a critical time for all of FDA’s programs—and also an excellent opportunity for Congress to make smart reforms that enhance the best of what FDA does, including its work to support global health research and development. Ashley Bennett is the GHTC’s senior policy associate.

In this guest post, Jamie Elizabeth Rosen—media & communications manager at Aeras—discusses a new effort to develop a novel tuberculosis (TB) vaccine candidate. Dr. Steven Reed is the founder, president, and chief scientific officer of the Infectious Disease Research Institute (IDRI), located in Seattle, Washington. IDRI is a 120-person nonprofit biotech committed to applying innovative science to the research and development of products to prevent, detect, and treat infectious diseases of poverty. My organization—Aeras, a nonprofit biotech focused on developing vaccines against TB—recently announced with IDRI a collaboration to conduct joint development activities with respect to IDRI’s novel TB vaccine candidate, called ID93 + GLA-SE. The collaboration was formed to accelerate the development of ID93 + GLA-SE by combining resources and expertise. IDRI’s internal development programs focus on three neglected infectious diseases—leishmaniasis, TB, and leprosy. But the organization has also collaborated or provided access to its vaccine technologies for a much wider range of infectious diseases and conditions, including malaria, pandemic influenza, HIV, hookworm, and Chagas. I asked Dr. Reed why IDRI got involved in TB. “IDRI started working on tuberculosis vaccines 20 years ago as an unmet need, and I have been working on TB since graduate school,” he said. “So we had knowledge of the disease and this question, and nobody else was addressing it.” Dr. Steven Reed, founder, president, and chief scientific officer at IDRI Although TB continues to claim the lives of more men, women, and children than any other infectious disease except HIV and AIDS, efforts to develop TB vaccines in clinical trials stalled for nearly 80 years. Part of the reason is that a vaccine against TB was invented and rolled out in 1921. But Bacille Calmette-Guerin (BCG), the most widely used vaccine in the world, has done little to stem the TB epidemic globally. While it helps prevent severe forms of TB in children, it is not very effective against adult pulmonary TB, the most common and contagious form of the disease. Only in the past decade has momentum in clinical development of new vaccines accelerated its pace. ID93 + GLA-SE is designed to boost protection afforded by BCG. It is slated to join a dozen vaccine candidates that are now undergoing clinical trials to test their safety and efficacy in protecting people from TB. Vaccine development is particularly complex for TB, so scientists are testing multiple candidates simultaneously so they can deliver new vaccines to protect the people who are most at risk for TB as soon as possible. I asked Dr. Reed why this vaccine candidate holds promise. “The main thing is that ID93 + GLA-SE is designed to boost the current vaccine, BCG. Secondly, the intellectual property for this vaccine candidate is held by IDRI and available for developing country manufacturers. Thirdly, it’s easily scaled and can be affordable. It has to be. So there’s no question in terms of making enough doses at an affordable price, which is not true for most live vaccines. Fourthly, it has a very extensive safety and efficacy data package and TB models.” The vaccine candidate contains four proteins recognized in both latent and active TB. One of every three people worldwide is already infected with latent TB, a dormant state of the disease that can reside in the body for years and will ultimately make about 10 percent of people who have it sick. The vaccine also contains the GLA adjuvant to enhance immune response. ID93 + GLA-SE has already been tested pre-clinically and, pending approval, will enter its first Phase I clinical trial in the United States this year. “We funded this program with over $35 million dollars from NIH [the National Institutes of Health] over the last 15 years,” said Dr. Reed. “All of the preclinical development is done with federal funds, with some industry support. We think working with Aeras will speed the process and integrate us a little more into the community of other vaccines that are in development.” Dr. Reed has a PhD in microbiology and immunology from the University of Montana, which served as a reference lab for BCG—the current TB vaccine—in the 1970s. He started working in global health as a Cornell University Medical College faculty member, and he worked in Brazil for five years in the 1980s. He developed the first recombinant TB vaccine to enter clinical trials, M72, which is now licensed to GlaxoSmithKline. In addition to vaccines, IDRI is working on other new tools to combat TB. “We’re working very closely with some excellent diagnostic manufacturers to make TB diagnostic tests, as well as with the Lilly TB Drug Discovery Initiative and the TB Drug Accelerator to identify and develop new TB drug targets,” Dr. Reed said.