Neglected tropical diseases are a group of chronic and disabling parasitic infections that primarily affect poor and underserved communities. These diseases affect more than 1 billion people globally, yet are rarely the target of new drug discovery efforts. According to the World Health Organization (WHO), global climate change will most likely alter the communities at greatest risk for these diseases, as changes in temperature and rainfall influence the distribution and life cycles of the insects that transmit these parasites. In some cases, insects and the parasites they carry have already begun emerging in regions where they were previously unheard of, including the United States.
These children live in Bangladesh, where UC San Diego researchers are leading a clinical trial to determine if a repurposed rheumatoid arthritis drug can be used treat amebiasis and giardiasis. Image source:
Leveraging its strengths in molecular biology, clinical research and pharmaceutical sciences, the University of California San Diego has now launched a new Center for Anti-Parasitic Drug Discovery and Development to address this unmet need in global health.
“These diseases are called ‘neglected’ because there is little economic incentive for the pharmaceutical industry to intervene,” said James McKerrow, MD, PhD, Distinguished Professor, dean of Skaggs School of Pharmacy and Pharmaceutical Sciences at UC San Diego and head of the new center. “Since we are a public university, we have a duty to meet that challenge.”
The center will involve 15 research and clinical faculty at UC San Diego, representing three schools and five departments — an unprecedented, interdisciplinary cadre of scientists dedicated to helping alleviate diseases of the poor worldwide, McKerrow said.
UC San Diego researchers use robotic technology to screen hundreds of thousands of chemical compounds from a variety of sources — including drugs abandoned by pharmaceutical companies and marine natural products — for their ability to kill parasites while leaving human cells unharmed. More than 10 disease-causing parasites are maintained and used for drug screening at UC San Diego. When promising drug precursors are identified, in-house experts can computationally and chemically optimize them, and further test the compounds in laboratory experiments and animal models of disease. This drug development pipeline is supported by UC San Diego Altman Clinical and Translational Research Institute, where researchers are able to carry out the entire process of drug discovery and development, including human clinical trials.
The robotic drug screening facility in Skaggs School of Pharmacy and Pharmaceutical Sciences at UC San Diego allows researchers to rapidly and reproducibly test hundreds of thousands of chemical compounds (potential drug precursors) for their abilities to kill the parasites that cause neglected tropical diseases.
With modest support to date, the center’s researchers are already leading advances in drug discovery and development for neglected parasitic diseases. For example, team members are developing a chemical compound for the treatment of malaria; working with pharmaceutical companies to develop potential drugs for Chagas Disease and schistosomiasis; and leading a clinical trial in Bangladesh to determine if a repurposed rheumatoid arthritis drug can be used treat amebiasis and giardiasis.
In addition, UC San Diego is the only place outside the Centers for Disease Control and Prevention (CDC) working with the animal model for Naegleria fowleri, a universally fatal brain-eating amoeba that can infect people swimming in warm freshwater worldwide, including the United States.
Now, thanks to seed funding from the UC San Diego Chancellor’s office, McKerrow and the center team can take the first steps to streamline these efforts and more rapidly move their initial findings along the drug pipeline.
“No other center is equipped to study these many organisms and target the diseases they cause,” McKerrow said. “No other entity — academic, government or industrial — covers the spectrum of parasite research and drug discovery from basic science to clinical trials at one site.”
The UC San Diego Center for Anti-Parasitic Drug Discovery and Development includes:
Skaggs School of Pharmacy and Pharmaceutical Sciences
- James McKerrow, PhD, MD
- Jair Lage de Siqueira-Neto, PhD
- Conor Caffrey, PhD
- Dionicio Siegel, PhD
- Carlo Ballatore, PhD
- Anthony O’Donoghue, PhD
- Ruben Abagyan, PhD
- Larissa Podust, PhD
- Anjan Debnath, PhD
- Jeremiah Momper, PharmD, PhD
School of Medicine
- Elizabeth Winzeler, PhD
- Sharon Reed, MD
- Joseph Vinetz, MD
- Lars Eckmann, MD
Division of Biological Sciences
Scripps Institution of Oceanography