Breakthroughs Blog: NIH

In this guest post, Rachel Cohen—regional executive director of the Drugs for Neglected Diseases initiative (DNDi) North America—writes about the need for research to develop new health products for neglected tropical diseases (NTDs). This post originally appeared on the US Agency for International Development’s (USAID) Impact blog . The United States government and its country partners should be commended for the tremendous achievements in the fight against neglected tropical diseases (NTDs) as part of the USAID NTD Program. When it comes to research, the US government also plays a major role in NTDs: the National Institutes of Health is by far the largest funder of basic research for NTDs. The commitment to NTD implementation programs and research has spanned several presidential administrations, receiving widespread bipartisan support from both sides of the congressional aisle. However, not all NTD research is created equal. Beyond basic research, much more research and development (R&D), including late-stage product development, for new drugs, vaccines, and diagnostics is urgently needed for those NTDs where adequate tools do not exist. Without such new tools, disease control and elimination will not be possible for certain NTDs—greater investments in research are urgently needed. For example, when it comes to the filarial parasitic-worm diseases of river blindness (onchocerciasis) and elephantiasis (lymphatic filariasis), which together infect over 150 million people, the standard treatment of ivermectin, alone or in combination with albendazole, can lead to brain damage or death in people co-infected with loiasis (Loa loa, also called African eye worm). Also, current drugs kill only juvenile and not adult worms, which continue to infect, requiring repeated mass drug administrations. The Drugs for Neglected Diseases initiative (DNDi)—a nonprofit R&D organization—is looking to develop a new macrofilaricide (a drug targeting adult worms) to address this gap in NTD treatment. Of the 17 NTDs defined by the World Health Organization, DNDi focuses most of its attention on the three with the highest case fatality rates—African sleeping sickness, Chagas disease, and kala azar (visceral leishmaniasis, or black fever). These diseases are not yet included in the USAID NTD Program. Although cases of African sleeping sickness (human African trypanosomiasis) have dropped over recent years, this fatal disease continues to infect people in remote, conflict-ridden pockets of the Democratic Republic of Congo, among other hotspots. Diagnostic tools are inadequate—painful lumbar punctures must be performed—and the current treatment requires hospitalization and intravenous infusions, which are difficult in unstable, resource-poor settings. DNDi is therefore aiming to develop an easy-to-take oral pill for sleeping sickness, which can treat both stages of the disease and do away with the need for painful lumbar punctures. This would go a long way in helping eliminate the disease once and for all. While continuing to provide existing medicines for NTDs, greater commitment to developing new NTD treatments and other tools is sorely needed if disease control or elimination is to be achieved. Patients infected now with these neglected afflictions are anxiously waiting.

Since our last update on the latest budget action on the Hill, two significant events happened late last week in Congress and at the White House. First, the White House’s Office of Management and Budget (OMB) released a report on the potential impact of sequestration on federal agencies. Sequestration would require a reduction in government spending of $109 billion per year for nine years, beginning in January 2013. According to the report, most defense programs relevant to global health—including global health research and development (R&D) programs at the Department of Defense—would receive a 9.4 percent cut under sequestration. Non-defense programs—including most programs at the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), the US Food and Drug Administration (FDA), the State Department, and the US Agency for International Development (USAID)—that are not exempt from cuts would receive an 8.2 percent cut. It’s important to note that some parts of these programs’ funds are exempt from cuts. For instance, out of a total budget of $34.249 billion for the NIH in fiscal year (FY) 2013, the sequestration applies to $30.861 billion. The rest is exempt. As for the primary agencies that fund global health and R&D programming, the report finds that: The total cut to NIH would equal $2.529 billion. This would put NIH’s total funding for FY 2013 at $31.720 billion. At USAID, the Global Health Programs account—the account responsible for all of USAID’s global health R&D work—would be cut by $670 million, leaving the final global health program’s funding level at $7.498 billion. The FDA, which plays a key role in regulating global health products, would see a cut of $318 million, with a final funding level of $3.624 billion (not including funding from user fees). The CDC would see a cut of $470 million from its overall funding, with $464 million coming from non-defense activities funding. This leaves CDC with a final funding level of $6.861 billion. These predictions are based on current FY 2012 funding levels. Many of these agency programs are already dealing with looming cuts to their funding levels for next year under various congressional budget proposals. If FY 2013 appropriations bills in Congress reduce agencies’ funding even further, the sequestration cuts would be even steeper than what’s outlined in the OMB report. Second, the House of Representatives late last week approved a Continuing Resolution (CR) for FY 2013, in order to keep the government running through the end of March 2013. The House approved the measure by a vote of 329-91, with 70 Republicans and 21 Democrats in opposition. The bill now moves to the Senate and is expected to pass this week. The CR funds the government at $1.047 trillion, consistent with the Budget Control Act. It also includes a slight 0.6 percent increase across the board for all federal programs, essentially maintaining spending at current levels. The CR provides a total of $55.2 billion for International Affairs, slightly higher than the $54.9 billion current level and higher than either the House or Senate FY 2013 appropriations recommendations. House Appropriations Committee Chair Hal Rogers (R-KY) said that lawmakers will aim to finalize appropriations bills when they return to work after the November elections. “We intend to use the lame-duck session to the fullest extent,” he said, adding, “Just because this CR will last until March 27 of next year, we will not rest on our laurels until that time.”

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The US Food and Drug Administration has announced it will give priority review to a new treatment for multi-drug resistant tuberculosis. The announcement came after the drugmaker applied to the European Medicines Agency for approval. Nils Daulaire, director of the Office of Global Affairs at the US Department of Health and Human Services, writes about the agency’s first-ever global health strategy. Reuters recently examined several scientific breakthroughs in HIV and AIDS in recent years, and how the global financial crisis could impact progress against the disease. An article looks at how the US presidential candidates would handle foreign aid and international development if elected in November. A related piece examines the social, political, and financial issues at play ahead of the presidential election and their implications for domestic and global health programs.

On the second day of the XIX International AIDS Conference, the need for a host of new tools to prevent and treat HIV and AIDS has already made it to the forefront of the agenda. Of particular note has been the resounding call for a vaccine to prevent HIV, with leading policymakers, scientists, and advocates saying that the world will never reach the end of AIDS without this powerful tool. In a session that focused on improving the effectiveness and efficacy in the HIV and AIDS response, Senator Lindsey Graham (R-SC) said that a vaccine is desperately needed to help make a sizable dent in the pandemic. Graham’s call for a vaccine was echoed by Bill Gates from the Bill & Melinda Gates Foundation. The “ultimate tool” in the fight against HIV and AIDS “will be a vaccine,” Gates said, adding that the US government has been the largest supporter of efforts to develop and vaccine “by far.” He also said that microbicide gels and rings will be powerful prevention products. Without a number of these new tools, the world cannot “talk seriously about moving toward the end” of the pandemic. “We will get these tools, but only if we stay the course.” Later in the afternoon, National Institutes of Health (NIH) Director Francis Collins and Jon Cohen from Science magazine chaired a panel that looked at what lies ahead in HIV and AIDS science. Gary Nabel, director of the NIH’s Vaccine Research Center, said “we’ve never been in a more exciting position” in the search for an HIV and AIDS vaccine. He added that recent discoveries related to broadly neutralizing antibodies over the past two years have made HIV vaccine research “like a phoenix rising from the ashes.” When sharing their hopes for the status of HIV and AIDS research, several panelists also stressed the urgent need to develop new HIV prevention and treatment options. Nabel said that by 2020, he expects researchers will be “in the midst of efficacy trials for an HIV vaccine candidate.” Robert Siliciano of the Johns Hopkins University School of Medicine stressed that by 2020, scientists will be leading clinical trials for new drugs that attack HIV reservoirs. And Dawn Averitt-Bridge of the Well Project said that by 2020, she hopes the world has effective microbicides to prevent HIV, a functional cure for the disease, and that “research makes it into the lives of the people and families we all care so much about.” Cohen ended the session with a call for young people to become the “next generation of scientists” leading HIV and AIDS research. “I’m looking to the young people, because that’s where the revolution is going to happen.” Kim Lufkin is the GHTC’s communications officer.