There are no fundraising walks for Human African Trypanosomiasis (HAT or “sleeping sickness”) but a drug for this “orphan disease” has gone to clinical trial thanks in part to professor emeritus Cyrus Bacchi, PhD, and Nigel Yarlett, PhD, chair of chemistry and physical sciences in the Dyson College of Arts and Sciences at Pace University.
Drugs for Neglected Diseases initiative Project of the Year 2011 Award given to Pace professors
NEW YORK, NY, March 12, 2012 – There are no fundraising walks for Human African Trypanosomiasis (HAT or “sleeping sickness”) but a drug for this “orphan disease” has gone to clinical trial thanks in part to biology professor emeritus Cyrus Bacchi, PhD, and chemistry professor Nigel Yarlett, PhD, and their undergraduate students in the Dyson College of Arts and Sciences at Pace University.
The professors’ work out of Haskins Laboratories at Pace has been awarded the Project of the Year 2011 Award by the Drugs for Neglected Diseases initiative (DNDi) for their role in the development of the first new drug to go to clinical trial and the first new treatment for sleeping sickness since 1980. The award was given jointly to Scynexis Inc. of Research Triangle Park, NC, which synthesized the compounds, and Pace University which did initial testing in vivo. Several Pace chemistry and biology students assisted in animal testing of the drug, providing them with hands-on experience in drug development from the bench to in vivo studies. Haskins Labs identified 15 compounds that may work in humans.
Bacchi and Yarlett have devoted their careers to neglected diseases. The research of Haskins Labs on compounds to treat sleeping sickness was written about in The New York Times in 1985 and 2008. In one article the disease was called “fearsome but nearly forgotten because its victims are poor and obscure.” In another article one drug, eflornithine, the trademark name for DFMO discovered in 1980 at Pace, is mentioned saying it is a “miracle” the drug is available. The drug is still used as a first line clinical treatment for sleeping sickness.
About 150,000 people contract sleeping sickness each year, but 50 million people in 36 countries live in areas where they are at risk. During recent epidemics in the Democratic Republic of Congo, Angola, and Southern Sudan, prevalence has been as high as 50 percent. In some communities with high prevalence, the death rate from African sleeping sickness has exceeded that of HIV/AIDS. Nevertheless, there is no profit in it. Without outside funding and incentives, drug companies are not interested in developing treatments for such orphan diseases.
With funding from DNDi, which receives support from the Gates Foundation, the drug has been able to go to clinical trials. One quarter of a million dollars a year is going to support this research at Pace.
The researchers have identified a new series of compounds which are effective in curing mice and are now being tested on larger mammals. Phase I clinical toxicity studies are beginning on humans in France on one compound, SCYX7158. Volunteers who are not infected take the drug to see if humans can tolerate it and are monitored closely for side effects. If these studies are successful, this compound will go to Phase II clinical trials later this year in African villages with infected inhabitants who are cut off from most medical access.
“For the people living in these villages, this sort of sickness is just a way of life,” said Yarlett.
“Sleeping sickness” has been called by The New York Times “too benign a nickname” for human African trypanosomiasis, which is caused by a protozoan. The disease is characterized by two distinct stages, early stage or blood stream infection and late stage disease of the central nervous system. When the brain is affected, victims hallucinate and their behavior becomes erratic and sometimes violent. Victims may chase people with machetes, throw themselves into latrines and scream with pain at the touch of water. Near the end of the disease, they lapse into a state of listlessness followed by coma and death.
The Haskins Laboratories, which have been at Pace since 1970, have a long legacy of researching possible cures for diseases that are out of the public spotlight. “We work on things that aren’t stylish—not in vogue. And consequentially, things that aren’t typically funded to a great extent,” said Yarlett.
“Drugs were developed between 1920 and 1950 to treat sleeping sickness, but some of these drugs had an arsenic base and were toxic,” Yarlett said. “For about 10% of those being treated with these drugs, death occurred more quickly than it would have if they hadn’t been treated. These are the first new drugs developed to treat sleeping sickness in 30 years. We’re very excited.”
Workers at Haskins Labs are also developing a first line of treatment for a more global issue—cryptosporidiosis, a waterborne illness that causes chronic diarrhea. Its major impact has been among those with weakened immune systems, including those who are HIV positive, receiving cancer treatments, or those that have undergone organ transplants.
“Cryptosporidosis is one of the major causes of death in HIV positive people and currently there is nothing available to treat it,” Yarlett said.
Read the press release from DNDi here.
About Haskins Labs
The Haskins Laboratories was founded in 1935 at General Electrical and Union College by four young and innovative scientists, one of whom became its namesake, Caryl Haskins, a physicist and geneticist. In 1970 it split into two divisions, the Microbiology Division, under Seymour Hutner (one of the original scientists) affiliated with Pace University, and the Speech Recognition and Cognition Division affiliated with Yale University. It is funded by a number of sources, including the National Institutes of Health (in collaboration with the University of Texas Southwestern Medical Center in Dallas), Drugs for Neglected Diseases initiative (DNDi), and Genzyme Corp and works in collaboration with pharmaceutical companies Scynexis and Anacor.
For more information about the work being done at the Haskins Laboratories, click here.
About Pace University
For 105 years Pace has produced thinking professionals by providing high quality education for the professions on a firm base of liberal learning amid the advantages of the New York metropolitan area. A private university, Pace has campuses in New York City and Westchester County, New York, enrolling nearly 13,000 students in bachelor’s, master’s, and doctoral programs in its Lubin School of Business, Dyson College of Arts and Sciences, College of Health Professions, School of Education, School of Law, and Seidenberg School of Computer Science and Information Systems. www.pace.edu
Cara Cea, 914-906-9680, firstname.lastname@example.org