Tag Archives: neglected tropical diseases

Today is Rare Disease Day

On February 28, Rare Disease Day, more than 60 countries and hundreds of organizations come together to raise awareness of the plight of those afflicted with rare diseases. Although rare diseases affect more than 100 million people worldwide, there is limited public awareness and insufficient research funding to develop tools to prevent and treat these diseases.

This year, the theme of the day is “Rare Disorders Without Borders.” Advances in rare disease research are far more likely to succeed if teams of researchers from different countries pool resources, share findings and work together to find new solutions.

There are clear parallels between these rare diseases and neglected tropical diseases, such as Chagas disease and dengue fever, that are afflicting people throughout tropical regions and increasingly closer to home. These diseases are not widely known, which often leads to misdiagnoses and delayed treatment. This lack of awareness results in very little funding for research in widespread prevention and treatment. It is abundantly clear that more research is needed to better deal with these rare and neglected diseases.

On the Rare Disease Day website, you can find the Handprints Across America gallery of patients and their families. The disease names may be unfamiliar to you—scleroderma, Hermansky-Pudlak syndrome, Prader-Willi syndrome, Pompe disease, Leiber’s congenital amaurosis, and familial partial lipodystrophy Dunnigan syndrome just to name a few. But the stories paint a clear picture of the impact of rare diseases on families across the nation and around the world. More patient photos and stories can be found on Rare Disease Day’s Flickr photostream.

Research!America members like the National Organization of Rare Diseases (NORD) and the American Autoimmune Related Disease Association (AARDA), are celebrating this year’s Rare Disease Day in addition to marking the 30th anniversary of the Orphan Drug Act. The Orphan Drug Act provides financial incentives to encourage companies to develop treatments for small patient populations. Passage of the Orphan Drug Act by was successful in large part due to patient advocates and other organizations working together. However, research and advocacy are still needed for these rare and neglected diseases! Check out the Rare Disease Day page to see how you can support the rare disease community.

Other research and advocacy groups are also spreading the word about Rare Disease Day. Groups and individuals on Twitter are using the hashtag #rarediseaseday. Also see the newest blog post from National Institutes of Health Director Francis Collins, MD, PhD. The NIH is hosting the sixth annual Rare Disease Day on Feb. 28 and March 1. The two-day event will highlight the work of the NIH-funded researchers, agency partnerships and collaboration among researchers across the globe.

Task Force for Global Health Receives Grant for NTD Research

On February 5, the Task Force for Global Health, a non-profit based in Decatur, Georgia announced that it received a $28.8 million, five-year grant from the Gates Foundation for neglected tropical disease research. This funding will support the new Neglected Tropical Diseases Support Center and will allow the center to work with the NTD community to address challenges in implementing NTD control programs. The Center will focus primarily on operational research and will work to develop new solutions to increase the efficiency and cost-effectiveness of NTD interventions. The grant will also support the development of a “Coalition for Operational Research for NTDs,” which will allow for more collaboration between international NTD researchers. Awarded just over a year after the London Declaration on NTDs, this grant is an important step forward in the fight against NTDs as it “will support the research needed to eliminate and control these dreaded diseases.”

-Morgan McCloskey, global health intern

Report on Social and Economic Impact of NTDs

In November 2012, the Hudson Institute and the Global Network for Neglected Tropical Diseases released a Social and Economic Impact Review on Neglected Tropical Diseases. The report, which was the culmination of a comprehensive research and policy analysis study, outlined the economic and social impact of seven of the most common NTDs including lymphatic filariasis, onchocerciasis, trachoma, schistosomiasis, hookworm, ascariasis and trichuriasis. These diseases impose a huge economic burden by causing roughly 46-57 million years of healthy life lost due to premature death or years lived with a disability. The report also quantified the economic burden in terms of lost productivity caused by NTDs and highlighted the success of current treatment efforts. For example, trachoma, the world’s leading cause of preventable blindness, causes up to $5.3 billion in lost economic productivity each year while treatment efforts for lymphatic filariasis have saved over $24 billion in lost economic productivity.

The report argues that one of the most promising ways to treat many of these NTDs is mass drug administration (MDA), which involves treating entire populations with drugs for the seven most common NTDs. These MDA programs are also successful examples of critical public private partnerships. The combination of federal government investments in basic R&D and private sector investment in later stage R&D has produced crucial drugs that private sector companies are now donating in order to support mass drug administration programs.  These public private sector collaborations, combined with investments in research and development for new tools to control NTDs, remain one of the core recommendations from the report. Research!America will continue to advocate for federal government support for R&D for NTDs and will be working with the private sector to limit the economic devastation and healthy life years lost to these diseases.

-Chris Bennet, Senior Manager of Global Health R&D Advocacy

Briefing on the Social and Economic Impact of NTDs

On January 17, the Hudson Institute and the Global Network for Neglected Tropical Diseases held a briefing event to discuss their recently released report, Social and Economic Impact Review on Neglected Tropical Diseases. In addition to negative health outcomes, the report highlights the social and economic costs of these deadly diseases and argues that NTD control and elimination programs are a cost effective public health measure. For example, Michael Kremer, Gates professor of Developing Societies at Harvard University, discussed de-worming as an extremely cost effective development intervention. Several studies around the world, including in the southern United States, have shown that de-worming is worth our money and attention as it can lead to increased labor outputs, higher wages and better test scores among students.

Panelists at the event also paid tribute to many organizations that have altered the landscape of NTDs: the Rockefeller Foundation, whose campaign against hookworm has had a long standing effect in the American South and pharmaceutical company Merck & Co., who made an unprecedented commitment in 1987 to donate the drug Mectizan for as long as necessary for the control of onchocerciasis (river blindness). Other pharmaceutical companies have followed suit and drug donation programs are now being administered around the world. Of course, these programs would not be possible without collaborative partnerships between a host of public and private sector entities, from multilateral and government agencies to local on-the-ground operations. In addition to transforming the NTD landscape, lessons learned from these public private partnerships and other NTD control efforts have helped to inform other global health programs around the world.

Finally, Ellen Nagler, CEO of the END Fund, discussed the Fund’s private philanthropy model that allows the private sector to invest in NTD interventions for maximum impact. The END Fund provides capital resources and capacity to collaborate with governments and existing organizations to scale up treatments for individuals most at risk. Fifty cents per person to treat the seven diseases affecting 90% of the world’s poorest is a powerful return on investment. Nagler concluded that in order to raise the money necessary to reach our goals and eliminate these diseases, a lot more people will need to be educated about NTDs and their impact throughout the world. Please read Research!America’s summary of the report  in tomorrow’s post.

-Jennifer Chow, Director of Global Health R&D Advocacy

NTD Highlights of the Week: January 18th

On January 16, Uniting to Combat NTDs released “From Promises to Progress,” the first annual report on the London Declaration on NTDs. The report details the progress made by global partners that signed onto the London Declaration one year ago. Notable successes include leading pharmaceutical companies donating treatments for 100% of drug requests in endemic countries and the development of new NTD control plans in over forty countries. The past year has also seen regulatory approval for two new NTD diagnostics: a new test for lymphatic filariasis and the first rapid test for sleeping sickness developed by the Foundation for Innovative New Diagnostics.

Alongside this report, WHO also launched its second NTD report, “Sustaining the Drive to Overcome the Global Impact of Neglected Tropical Diseases.” The report targets two diseases for global eradication: guinea worm disease by 2015 and yaws by 2020. WHO also reports successes in preventive treatment, noting that 711 million people received treatment for at least one NTD in 2010 and projecting that these treatments will continue to reach more individuals in the future.  However, there is still significant work to be done. Diseases like African sleeping sickness and leishmaniasis remain extremely difficult and costly to treat. There has been a 30-fold increase in dengue in the past 50 years and there is the potential for a global dengue epidemic, but we lack the appropriate tools to control and treat the virus. Whether it is scientific research to develop new drugs or operational research to develop the most effective control plans, additional investment in NTD research is crucial. Despite these challenges, Dr. Chan, Director-General of WHO, says that “the prospects for success have never been so strong.” The more we can raise awareness about these diseases that primarily affect the 1.4 billion people under poverty, the more we can do to mobilize resources for the global fight to combat NTDs. We want to make sure we continue turning prospects into actual success.

-Morgan McCloskey, global health intern

NTD Highlights of the Week: January 4th

As we ring in the New Year, 2013 promises to be an exciting time to be involved in the fight to raise support and awareness for neglected tropical diseases. As the world becomes more interconnected and global warming changes disease patterns, NTDs are increasingly spreading across borders – including right here at home. For example, Slate recently published an article addressing the return of dengue in the United States. In the past few years, dengue has sickened hundreds in Florida and other southern states. Experts warn that the combination of the virus, a lack of immunity to dengue and widespread mosquitoes provide the perfect storm of conditions for larger dengue outbreaks in the U.S.

As the spread of NTDs adds urgency to the fight, scientists continue to work every day to develop innovative ideas to combat NTDs. In a trial experiment in Africa, researchers are testing the ability of prawns to combat schistosomiasis. A parasitic disease that can be fatal, schistosomiasis is spread through water snails. Prawns are the primary consumers of snails, so researchers hope that re-introducing prawns to rivers at the African test site will help decrease transmission of the disease. In addition to innovative experiments, every week there are reports of new scientific breakthroughs that will help save lives.  Just last week, the FDA approved a drug to fight drug resistant tuberculosis, the first new drug for the disease in over four decades. Developed by Johnson & Johnson, the drug cures patients in less time than older treatment options. It is these kinds of innovations and breakthroughs that demonstrate the power of research investments and the importance of research for global efforts to eliminate neglected diseases. Be sure to check back soon for new NTD highlights!

Morgan McCloskey, global health intern

Lives in the Balance

On December 13 and 14, the global health community gathered at the Mount Sinai School of Medicine in New York for a conference called “Lives in the Balance: Delivering Medical Innovations for Neglected Patients and Populations.” Hosted by Mount Sinai Global Health, Doctors Without Borders (MSF), and Drugs for Neglected Diseases initiative (DNDi), the conference aimed to spur innovation for new tools to combat neglected diseases.

Several key themes emerged from the conference. First, there is a ‘fatal imbalance’ between the burden of neglected disease and medical innovations to combat these illnesses. Neglected diseases affect more than 1.4 billion people worldwide and account for nearly 11% of the global disease burden. In contrast, MSF’s Jean-Herve Bradol, MD, pointed out that of 850 new therapeutic products approved in the past decade, only 4.4% were for neglected diseases. Furthermore, only 1.4% of 148,445 clinical trials were for neglected diseases. To solve this problem, many presenters agreed that there must be a new global framework for global health R&D. A new framework would place an emphasis on public financing and some called for all countries to pledge 0.01% GDP to government-funded R&D.

In addition to reforming the global R&D system, several panelists mentioned the importance of improving access. This means not only improving access to medicines among neglected patients but improving information sharing and access to essential compounds among researchers. The idea of access must also be built into the beginning of the research process. For example, considering storage temperatures or dosage early on in the R&D process will help to ensure that the new tools being developed can be easily utilized in the field.

The conference has put a spotlight on the need for more research to combat neglected diseases. Diseases that once only existed in the developing world are becoming an increasingly large threat in Europe and in the U.S. Cases of multi-drug resistant TB are on the rise, and we need much better treatment options to cure patients. Only two drugs are currently available to treat Chagas disease; both were developed more than 35 years ago, have toxic side effects and are not effective in all patients. It is more important than ever before that governments, philanthropic groups and the private sector come together to help reform the global R&D system, improve access and find new tools to help neglected patients and populations around the world. Many have called this time, these partnerships and current innovations in modern science an unprecedented opportunity for neglected disease R&D. Others are frustrated by the seeming regression in global health due to non transformative “stopgap” efforts, citing drug resistant TB as an example. Both may be right. With unified advocacy to raise awareness and engage political and civic will for NTDs, we can successfully channel both the frustration and the opportunity ahead of us.

Report Highlights Need for More R&D Funding for Neglected Diseases

On December 3, Policy Cures released its fifth annual G-FINDER report, a comprehensive survey of funding for research and development for neglected diseases. The report tracks global public, private and philanthropic investments into R&D for 31 diseases, including HIV/AIDS, tuberculosis, malaria and NTDs.  In positive news, this year’s report shows that total funding has actually increased by $443 million since 2007.

The report demonstrates that government funding, which accounts for over two-thirds of all investment, is increasingly going toward basic academic research, rather than product development. Research!America believes it is vital that the entire research pipeline be fully funded. Basic research will help us understand the best ways to tackle these neglected diseases and give us a better understanding of disease and the human body.  However, we also need robust funding for the development of urgently needed tools like drugs, vaccines and diagnostics. This urgency is worsened by the fact that private and philanthropic investments in product development for NTDs have also decreased.

Because NTDs disproportionately affect the “bottom billion” or individuals earning less than $1.25 per day, there is essentially no market demand for new NTD tools, so the private sector is unlikely to fund these projects. Dr. Moran, director of Policy Cures, believes that governments must step up to the plate, saying that “if [governments] want products for neglected diseases, they must fund product development as well as basic research.”

-Morgan McCloskey, global health intern

Research!America Releases New NTD Fact Sheet

Brian Vastag, science reporter at The Washington Post, recently found himself infected with the very disease he had been reporting on for months: West Nile virus. Detailing the raging fevers, interrupted sleep patterns and tingling in his arms and feet, he called himself a “West Nile zombie.” Brian’s story highlights the importance of research to develop new prevention, diagnostic and treatment methods for West Nile virus. It took eight weeks and several doctors before an infectious-disease specialist was able to correctly diagnose him with West Nile. Once diagnosed, he still had to endure the fevers, joint aches, headaches and interrupted work and simply wait for the virus to go away on its own. Beyond the agonizing symptoms of the disease, it was estimated that in 2002, West Nile cost the United States about $200 million in direct medical costs. Considering 2012 is on record to be the deadliest year we’ve seen, one can only imagine the medical costs of West Nile this year. As Brian points out in the article, we must acknowledge the true costs of these diseases. Research to develop a West Nile vaccine would not only save lives, but would save millions of dollars in future health care costs and lost worker productivity. Similarly, better diagnostic and treatment options would allow doctors to identify the virus sooner and more effectively treat patients.

In an effort to increase awareness about the importance of research for West Nile and other neglected tropical diseases, Research!America has released a new fact sheet called “NTDs in the United States.” The fact sheet details the burden of NTDs here at home and highlights important NTD research activities in the U.S. To see the fact sheet and learn more about NTDs, please visit www.researchamerica.org/gh_ntds.

-Morgan McCloskey, global health intern

West Nile Spike Reminds Us That Global Health Research Benefits Americans, Too

As reported in the Washington Post, the number of West Nile virus cases in the U.S. is on the rise. Traditionally a disease that affects people in Africa, the Middle East and Asia, 48 states in the U.S. have reported cases in 2012 alone. Nearly 2,000 cases and 87 deaths, including one Wednesday in DC, have been reported overall. The West Nile virus, a neglected tropical disease or NTD, can cause flu-like symptoms or, in severe cases, even brain damage.

Peter Hotez, MD, PhD, director of the Texas-based product development partnership Sabin Vaccine Institute, recently wrote a New York Times op-ed addressing the increasing thread of West Nile right here in the U.S., “Tropical Disease: The New Plague of Poverty.” As Hotez points out, West Nile is just one of several NTDs that have a presence in the United States. Dengue fever, another virus transmitted through mosquitoes, has been reported in Texas, Florida and Hawaii. A recent estimate finds that 300,000 individuals in the U.S. have Chagas disease, an infection transmitted through insects that can cause heart failure and even sudden death. These NTDs pose an immediate threat to the health of Americans, particularly in impoverished areas of the South where poorer sanitation and drainage systems allow NTD “carriers” to thrive.

NTDs can go undocumented for long periods of time, can be extremely debilitating and have inflicted a large toll on peoples’ health and economic stability around the world. NTDs paint an increasingly troubling picture for American health. Toxic and ineffective, or in some cases no treatments, exist for many of these NTDs, and better surveillance and monitoring is desperately needed. With little financial incentive, private companies are reluctant to invest in this research. However, there is hope. Federally funded researchers at the National Institutes of Health have identified a new drug that has the potential to treat Chagas disease. Additional clinical trials will determine its safety and efficacy for widespread use. Bloomberg recently reported that another PDP, the Dengue Vaccine Initiative, has developed four dengue vaccines that are currently undergoing clinical trials. Continuing to fund this type of research and development is critical to ensuring that the promise of these vaccines becomes a reality.

In an effort to raise awareness about the importance of funding for NTD research, Research!America hosted a joint forum this summer entitled “Global Health Research and Development and the Hidden Burden of Neglected Tropical Diseases in Texas.”  Additional support, including robust federal funding, will result in new prevention and treatment methods that are urgently needed not only to improve the health of individuals around the world, but right here in our own backyards. Please let your congressional representative know that even in today’s tough economic environment, funding for global health and NTD research must be a higher national priority.

A video of Research!America’s Texas forum on neglected tropical diseases is available here.