Drug Development Global Program

Bringing malaria elimination within reach through population-wide drug-based strategies

By Zachary Clemence, Malaria Learning Series Intern at PATH

This post originally appeared on the Making Malaria History blog.

In a rural village, a group of health workers meet with a crowd of men, women, and children.

A group of health workers and a crowd of men, women, and children sit together in Sinafala village, Zambia. Photo: PATH/Gabe Bienczycki

The global scale-up of vector control and improved case management has helped bring malaria cases and deaths to record lows since 2000. Endemic countries and donors are now working toward a world without malaria. Since 2010, PATH, through the Malaria Control and Elimination Partnership in Africa (MACEPA), has been investigating ways to accelerate this process by using drugs to clear parasites from their human hosts. If these approaches, known as population-wide drug-based strategies, are effective, feasible, and affordable, they may reduce malaria transmission in the short term to a level where it could be eliminated through strong surveillance that allows for tracking and treating each case.

This week, PATH released a brief on findings from this research as the first installment of their new Malaria Learning Series. The Learning Series is a suite of reports on current topics in malaria science that will provide practitioners, policymakers, and donors with the most recent evidence for decision-making. Topics will include emerging tools and strategies, health system and surveillance innovations, and planning for elimination.

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Scientists as advocates: Behind the bench, in front of the camera

By Deborah Kidd, Senior Communications Officer, PATH

This post originally appeared on the DefeatDD blog.

Photo: PATH.

Photo: PATH.

A scientist has a mandate: objective, data-driven fact-finding; research with an open mind and a solid protocol. But a scientist is more, too. Behind the standards of practice and quality control checks are life experiences, a full range of emotions, motivations, and passions. Scientific evidence is key to confirming unequivocally that diarrhea still sickens and kills too many children; chronic illness stunts growth and development; and vaccines can prevent severe disease. But in telling the story of diarrheal disease—as a complement to figures, findings, and data points—we wanted to explore that other side of science, to get at the human stories behind these stats. We asked a few researchers to reflect.

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PATH in San Francisco, Robert Choy

PATH’s team in San Francisco works to advance transformative global health innovations to improve lives of women and children, primarily through our work in drug development. In this post, we learn more about Robert Choy, associate director for research and preclinical development for PATH’s Drug Development program.

Photo: Robert Choy.

Photo: Robert Choy.

In my role, I collaborate with colleagues at pharmaceutical companies, academic institutions, and contract research organizations around the world to design, analyze, and interpret experiments, which advance the development of lifesaving treatments for enteric pathogens such as Cryptosporidium and soil-transmitted helminths.

Do you have any pets? No, but I have two young sons, Lucas, six, and Xavier, three, who act like wild animals. 

Do you have any hidden talents? I have practiced and taught the martial art of Kokikai Aikido for over 20 years, so I can show you how to effortlessly throw someone across the room.

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The best health advice from Mom

Photo: PATH.

Photo: PATH.

Moms around the world have one crucial thing in common—there’s nothing more important to them than the health and well-being of their families. Every day, moms focus their time, energy, and resources on keeping kids protected from sickness and harm and teaching them the lessons needed to become healthy adults. In times of illness, few are more dedicated or powerful than Mom. Our team in San Francisco is lucky to count some really great moms on our side; below are some of the most important lessons we’ve learned from the moms in our lives.

I remember my mom always saying “a healthy child has no bounds.” Little did I realize this would hold true well into my adulthood. –Neha A., Commercialization Officer, Diagnostics

The most important lesson that Mom taught me about health is that it’s more than merely the absence of disease, and the health of communities and populations deserves as much attention as the health of individuals. David Shoultz, Global Program Leader, Drug Development and Devices and Tools

My Mom continues to emphasize proper hand washing. She enjoys teaching my daughters to sing “Happy Birthday” two times while scrubbing front and back of hands with soap (but be sure not to let the water run—we are in a drought after all!). –Bridget B., Senior Manager, Outreach and Development

The most important thing my mom did for me is to get me vaccinated. Continually updating my vaccines has helped keep me, and those around me, healthy! –Sarina A., Program Coordinator, Drug Development

Photo: Yvonne Sidell.

Photo: Yvonne Sidell.

My Mom, at 82, always says, “The good [food] you put inside will truly show on the outside,” and I couldn’t agree more… Treat yourself to healthy eating so your life can be as long and enjoyable as hers! –Yvonne S., Regulatory Officer, Drug Development

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GHTC briefing highlights critical role of US government in advancing global health innovation in an increasingly globalized world

By Kat Kelley, Senior Program Assistant, Global Health Technologies Coalition

This post originally appeared on the Global Health Technologies Coalition (GHTC) Breakthroughs blog.

On Tuesday, GHTC launched our seventh annual policy report through a congressional briefing and panel discussion. The critical role played by the US government in catalyzing global health research and development (R&D) was a recurring theme, as panelists discussed the interconnectedness of global and American health and emphasized the need for new health tools to protect against outbreaks and continue progress.

As moderator, GHTC Director Erin Will Morton opened the briefing, reminding attendees that despite the landmark progress achieved in global health over the past fifteen years, the US government must continue to invest in health R&D to achieve the next milestones in global health. “Goal setting works. And R&D for new technologies is key to achieving global health goals,” said Morton.

Morton emphasized how past investments have had a remarkable impact, highlighting that the neglected disease pipeline has grown from 365 products under development in 2012 to 485 in 2015. However, as the GHTC policy report points out, US funding for global health R&D has stagnated or declined since 2009, and with the exception of the emergency funding for Ebola, 2014 funding levels were down 13 percent. “Our dollars are best spent when [US] agencies are working closely together,” said Morton, highlighting three goals the US government should embrace to advance innovation, as detailed in the report, including the importance of sustainable, long-term funding and innovative financing mechanisms; improved coordination of global health R&D efforts across US agencies; and streamlined and strengthened regulatory pathways.

Panelist Dr. Peter Hotez—president of the Sabin Vaccine Institute and US science envoy—reiterated the need for long-term, sustainable funding for global health R&D, noting that the science to develop an Ebola vaccine was first published in 2013, but the technology sat there and was not advanced to product development because Ebola is a neglected disease and the technology offered little financial incentive to the private sector. He emphasized the role of US government investment in catalyzing health research, noting that when the US government “stepped in” and put up more than US$100 million to develop an Ebola vaccine, three leading pharmaceutical companies joined in.

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Drugs meant for the heart could save lives from diarrhea

By Robert Choy, Associate Director for Research and Preclinical Development, PATH

Woman holding an infant. Both are smiling.

Childhood diarrhea remains a leading cause of illness among young children. Sustained research and development of new treatments is needed to reduce mortality and morbidity. Photo: PATH/Doune Porter.

While substantial progress has been made in reducing childhood mortality in developing countries over the past two decades, UNICEF estimates that more than half a million children under five years old still die from diarrhea every year. For most people, this number is very difficult to fathom, so here is another way of describing the burden of diarrhea: Imagine three Boeing 747 airplanes filled with small children all crashed on January 1. Most likely, the world would be gripped by this tragedy. Now imagine three more planes filled with kids crashed on January 2. And again on January 3. How long would it be until people demanded action?

For more than 10 years, a major focus of our Drug Development program has been working to develop safe, affordable, and effective treatments for enteric and diarrheal diseases. One area of investigation has been a class of drugs that inhibit an enzyme called neprilysin, or NEP, which helps to regulate intestinal function. One drug in this class called racecadotril has been approved in France and is also used in several low- and middle-income countries to treat diarrhea, but it has never been widely adopted because of several shortcomings, including the need to take it at least three times per day and the fact that its performance in clinical trials has been mixed.

Partners focus on repurposing drug candidates meant for heart disease

In collaboration with the Center for World Health and Medicine (CWHM) at Saint Louis University, PATH sought to identify another NEP inhibitor that might be more effective than racecadotril. Fortunately for PATH, a number of pharmaceutical companies including Pfizer, Bristol-Myers Squibb, Sanofi, and Novartis have concurrently developed NEP inhibitors, but for reasons completely unrelated to diarrhea. In addition to its role in regulating intestinal function, neprilysin was also found to be involved in other biological processes, including the cardiovascular system. More than a dozen NEP inhibitors were tested in clinical trials for heart disease in the 1990s and early 2000s, but then this class of drugs was largely abandoned due to lack of efficacy.

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Life is impossible without water

By Erika Amaya, Communications Associate, PATH

On World Water Day, I see blue in an umbrella approach that can protect and save lives around the world.

Photo: PATH/Erika Amaya.

On World Water Day, I see blue in an umbrella: as in, an umbrella approach to protect and save lives.

In the last few years, the persistent drought in California has really driven home for me the vitality of clean, fresh water for the health and well-being of people everywhere. From society-level needs like sanitation and irrigation for agriculture, to individual needs like staying hydrated and handwashing, life is impossible without water.

Thanks to El Niño, we recently had a downpour in San Francisco, which led me to dust off a white and blue umbrella that hasn’t seen much action in recent years. While my US-based colleagues in Seattle and DC are used to all kinds of weather, the rain was a bit novel for me and my sidekick Bogie. (In case you missed it, Bogie has been featured on DefeatDD before—in the 2016 #TravelingPoo calendar!)

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Why is malnutrition so hard to treat?

By William A. Petri, Jr., Chief, Division of Infectious Diseases & International Health, University of Virginia

Photo: William A. Petri, Jr.

Photo: William A. Petri, Jr.

For a number of years, I have worked with my colleague Rashidul Haque in Dhaka, Bangladesh, studying the health of infants in urban slums. There, we have studied the problems of infections, vaccine failures, and impaired growth and development. In this setting, a typical child is infected with four different enteric pathogens by the age of one month and sickened with three to four episodes of diarrhea. In the first year of life, a child will experience an equal number of acute respiratory infections.

One of the striking things to me has been the development of malnutrition in the children that we study. While only one in ten is born malnourished, nearly half are stunted (i.e., two standard deviations short for age, a measure of long-term malnutrition) by two years of age. A few years ago, one of my fellows, Cynthia Snider, suggested we intervene and treat this malnutrition, rather than observing from the sidelines. I agreed, and together with Rashidul, we instituted a program of supplemental feeding and nutritional counseling. Mothers and their malnourished children came into our clinic each morning where they were taught about nutrition and responsive feeding. In addition, the mothers gave their children complementary feedings with a rice and soy-based food containing vitamins and micronutrients. To our surprise, this approach resulted in almost zero impact. As I learned later, it was not that we were doing anything wrong; failure to respond is the rule and not the exception.

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PATH in San Francisco, Yvonne Sidell

Photo: PATH/Erika Amaya.

Photo: PATH/Erika Amaya.

PATH’s team in San Francisco works to advance transformative global health innovations to improve lives of women and children, primarily through our work in drug development. In this post, we learn more about Yvonne Sidell, regulatory program officer for PATH’s Drug Development program.

In my role, I assist the drug development team with regulatory guidance for projects to remain compliant with mandatory regulations. My duties include regulatory intelligence, preparation of Investigational New Drug and New Drug Applications, clinical trial safety reporting, electronic submissions, regulatory strategy, and various functions of quality assurance.

How were you introduced to PATH? I was on the OneWorld Health (OWH) team for five years before we became a part of PATH in 2012. During my time with OWH, I was involved in documentation management and quality assurance, transitioning into my current role in regulatory affairs, which I immensely enjoy. As part of the same global health community, I had been familiar with PATH’s work for a long time.

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Scientific gathering puts spotlight on Cryptosporidium

A full room of meeting attendees for Cryptosporidium symposium watches a presenter in front of PowerPoint slides.

Meeting participants from around the world gathered for a full day of presentations and discussions at the Symposium for Innovative Therapeutics on Cryptosporidium held in San Francisco. Photo: PATH/Erika Amaya.

As a leading cause of diarrheal disease among young children living in low-resource settings, Cryptosporidium not only kills, but it can leave survivors suffering from chronic malnutrition and long-term consequences, including growth stunting and deficits in cognitive development. Data from the Global Enteric Multicenter Study (GEMS) in 2013 and A Global Network for the Study of Malnutrition and Enteric Diseases (MAL-ED) in 2014 demonstrated the prominent role that Cryptosporidium plays in child health, but research, funding, and development of needed drugs has lagged behind. Currently, only one drug is approved for treatment, and new therapies are urgently needed.

In January, we hosted the Symposium on Innovative Therapeutics for Cryptosporidium, an international gathering of academic researchers, pharmaceutical professionals, and global stakeholders to highlight the work currently underway on this lethal parasite and speed progress toward new treatments and expanded access to existing therapies. Below are a few of the key moments at the meeting that shed light on the current state of Cryptosporidium research and development, in addition to opportunities for further progress and collaboration:

David Shoultz, PATH, welcomed the audience and provided an introduction to our work on enteric and diarrheal diseases, which is focused on the total effects of these illnesses in children. He presented ACCORD, our proposed approach to help accelerate the development of new therapeutics for Cryptosporidium, and encouraged anyone interested in learning more to get in touch with our team.

ACCORD intends to bring together pharmaceutical companies, research institutions, product development partnerships, and funders to accelerate research and development of needed Cryptosporidium drugs to reduce child mortality and morbidity. Figure: PATH.

ACCORD intends to bring together pharmaceutical companies, research institutions, product development partnerships, and funders to accelerate research and development of needed Cryptosporidium drugs to reduce child mortality and morbidity. Click for larger image. Figure: PATH.

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