Breakthroughs Blog: NIH

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The Food and Drug Administration has approved the use of a new diagnostic test developed by the Centers for Disease Control and Prevention for earlier detection of dengue fever in the United States. Research!America has launched a new ad campaign that promotes US federal investments in driving research for new global health tools. A recent article in the Journal of Medical Microbiology discusses some of the scientific, financial, and ethical challenges in vaccine development. A new report by BIO Ventures for Global Health and the Biotechnology Industry Organization found that small- and medium-sized biotechnology companies are tackling the development of treatments, diagnostics, and other tools for neglected diseases. Congressman Edward Markey (D-Mass.) calls for a “detailed understanding of the harm” that mandatory cuts in the US federal budget to agencies such as the National Institutes of Health “pose to vital health programs upon which millions of Americans around the nation rely.”

Combating preventable child deaths worldwide is an “investment that will pay dividends for years to come,” Deputy National Security Advisor Dennis McDonough said last week at a major conference at Georgetown University. At the conference, representatives from over 80 countries met to pledge their support for an exciting new global campaign for child survival. Hosted by the United States, UNICEF, and the governments of Ethiopia and India, the summit convened high-ranking government officials, members of the faith community, advocates, and private sector representatives. Over the last 50 years, child mortality rates have dropped by 70 percent. However, 7.6 million children under age five still die each year—far too many. In 2010, 57 out of every 1,000 children died before age five. The goal of the new program is to reduce that global rate to 15 deaths per 1,000 children by 2035. Under the program, UNICEF will house a small secretariat that will coordinate global activities to increase access to existing technologies and improve treatment guidelines, while partner countries like the United States will implement five strategic shifts to accelerate progress. As US agencies work with their partners to implement a new child health program, they must invest in R&D for new global health technologies. It’s critical that the program include a dedicated and distinct focus on global investments in research and development (R&D) for new tools to combat global child deaths, we still lack the vaccines, drugs, and other technologies needed to save children’s lives around the world. Indeed, research and innovation for new tools have been a huge factor in the success made against child mortality in the past 20 years, and must be prioritized by the new program. Several speakers at the summit praised the impact that technologies like the polio vaccine, oral rehydration solution to treat diarrhea, the MenAfriVac™ meningitis vaccine, and others have made in saving the lives of millions of children. Imagine if we had never invented a polio vaccine, or malaria drugs for children, or diagnostic tools for tuberculosis—what would global child mortality rates be today? A historic commitment to child health R&D helped create the tools that have already made a huge dent in global childhood deaths. For this reason, we should not only increase access to these existing technologies—the US Government and global partners must also continue to support R&D for the next generation of health products for children. If they don’t, the world will never reach the goals set by the new program. Fortunately, several US officials stressed the role of R&D in this effort last week. Secretary of State Hillary Clinton emphasized the commitment of the US Government to the development of new tools, saying, “We will support innovative research into new life-saving cost-effective measures.” Health and Human Services Secretary Kathleen Sebelius echoed the US role in basic research to support global health programs. Additionally, health officials from countries such as Bangladesh, Pakistan, and Nigeria highlighted the immense impact of global health technologies on their national child health programs. These achievements can be furthered by R&D-focused programs supported by the US Government, such as the second round of the Saving Lives at Birth program at the US Agency for International Development (USAID) and a joint USAID/National Institutes of Health initiative (NIH) that will provide grants to researchers in developing countries. As USAID and other agencies work with their partners to implement this important new program, they must invest in R&D for new global health technologies and plan for their delivery. USAID, NIH, and several other US agencies have a history of investment in innovation and are experts in catalyzing new research for huge impact. If we truly want to make the amazing progress we are promising to the next generation, the US must continue to expand its investment in global health R&D.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. Secretary of State Hillary Clinton and US Agency for International Development Administrator Rajiv Shah were joined by more than 700 leaders from the private sector, government, and civil society for a major conference devoted to preventing childhood deaths worldwide. "Amid these financial threats and budgetary realities, it is inevitable that some will question the role of the United States in global development," Sen. Richard Lugar (R-IN) said at the conference. "But I would assert this morning that development assistance, when properly administered, remains a bargain for US national security and for our own economic and moral standing in the world." Five new pharmaceutical companies have joined a National Institutes of Health (NIH) program that will pair researchers with certain pharmaceutical industry compounds to help scientists explore new treatment options for patients. The program, called Discovering New Therapeutic Uses for Existing Molecules, is run by the NIH's National Center for Advancing Translational Sciences (NCATS). The US Food and Drug Administration (FDA) has decided to delay its final decision on whether the drug Truvada, made by Gilead Sciences, can be used by certain high-risk, HIV-negative people as a preventive measure against HIV. The FDA says its decision will be announced by September 14, 2012. A Baltimore Sun letter-to-the-editor argues that there are more people living with tuberculosis (TB) “now than at any time in history.” The letter adds that prevention with “new vaccines is the only way to truly make TB a thing of history.”

Late last week, the National Center for Advancing Translational Science (NCATS) at the National Institutes of Health (NIH) unveiled a pilot program that aims to speed the creation of new drugs. Under the program, three pharmaceutical companies have agreed to make dozens of their failed compounds available to researchers, who will investigate if the compounds can be re-purposed into successful treatments for other diseases. The HIV and AIDS drug AZT, which was born out of an unsuccessful cancer treatment, has been cited as a prime example of the potential for this new program. At the launch event, NIH Director Francis Collins highlighted that the NIH needs to “speed the pace at which we are turning discoveries into better health outcomes.” He also emphasized the urgent need for new cures for patients suffering without any available treatment options. With NCATS, an exciting potential exists for new tools to combat neglected global health diseases. Photo credit: PATH The new program embodies the public-private partnership model. In 2013, $20 million in US Government grants from the NIH will fund researchers to work with compounds provided by private-sector pharmaceutical companies in pursuit of new treatments. As of now, over 20 compounds have been committed by the three companies. Kathy Hudson, acting deputy director of NCATS, hopes that many more will be available by the time the program releases its first Request for Applications (RFA) next month. No information is currently available from NCATS on what compounds will be available, or what diseases the program hopes to tackle. We won’t have that information until the RFA is public. In setting his goals for NIH at the beginning of his tenure, Collins stated that global health would be a top priority for NIH as an organization. Now that NCATS—which focuses on spurring drug development for neglected diseases of the developing world, as well as rare diseases—is established and this new program has been launched, an exciting potential exists for new tools to combat neglected global health diseases. Since its inception, NCATS has unfortunately been slow to take up initiatives that target global health diseases, as many of the NCATS programs announced thus far focus primarily on rare diseases that affect US populations. Rare diseases absolutely deserve funding, support, and targeted research—as do global health diseases. As shown in our recently released report, the United States leads the world in global health research and development. Under this leadership, who knows what groundbreaking cures for global health diseases could be created with the compounds included in the new program? At the press conference, patient advocate Michael Manganiello spoke enthusiastically about his experience as a patient saved by AZT. “Without partnerships between the NIH and industry, I would not be here to give this statement today,” he said. HIV and AIDS is just one example of the range of global health diseases that remain in urgent need of new tools. Think of how far we have advanced from a time when there were no treatments for HIV and AIDS, and think of what progress could be made in other neglected diseases under this new program. With this new therapeutics program, NIH and NCATS have created an exciting, innovative model for collaboration between the public and private sectors. They should be applauded for it. Now NCATS must ensure that research for neglected diseases is encouraged and supported through this initiative. NCATS has the opportunity to jump start innovation that will one day save lives and create better health outcomes—an opportunity that should be shared with patients around the world.