San Francisco, Calif. - July 7, 2003 - The Institute for OneWorld Health, the first nonprofit pharmaceutical company in the United States, announced today it has begun Phase III testing of paromomycin to cure one of the world's deadliest parasitic diseases, kala azar, also known as visceral leishmaniasis (VL). Demonstrating a new use for an old medicine, OneWorld Health is developing paromomycin as an affordable and cost-effective life-long cure for VL. Through a $4.2 million grant from the Bill & Melinda Gates Foundation, OneWorld Health is collaborating with the Tropical Disease Research Programme of the World Health Organization (WHO/TDR) in a 670-patient Phase III multicenter clinical trial in Bihar, India.
Following completion of the trial, the goal is to seek regulatory approval of the drug for VL, particularly in India, which has the largest burden for this disease in the world. Paromomycin is an off-patent medicine that was previously approved by the Food and Drug Administration and marketed in the U.S. as a broad-spectrum antibiotic.
Transmitted by the bite of a sand fly, VL is a deadly vector (insect)-borne disease that attacks the internal organs. An estimated 1.5 million people worldwide are currently infected; 200 million people are at risk of acquiring VL; and at least 200,000 people die annually. The disease affects the poorest of the poor, who cannot afford expensive drugs. With the exception of malaria, VL kills more people than any other parasitic disease. While most Westerners have never heard of visceral leishmaniasis, if a disease killed the equivalent number of people in the U.S., it would be the third largest killer after heart disease and cancer, and would cause more deaths than stroke.
"It's not every day one can say an affordable cure for a deadly disease may be imminent, and we believe our approach will be successful," said Dr. Victoria Hale, founder and CEO of OneWorld Health. "This drug appears to be equivalent to a therapeutic vaccine since patients cured of VL develop life-long immunity to the parasite. This Phase III trial is the largest ever for VL and will be conducted under the most rigorous regulatory standards. Medicines like paromomycin are an example of how new cures for neglected infectious diseases can be developed quickly and cost effectively. We will continue our mission to identify late-stage drugs for other diseases and will collaborate with organizations to develop medicines for people in need."
"The development of paromomycin will expand the global toolbox to fight underfunded, neglected, deadly diseases," said Dr. Carlos Morel, director of TDR. "The trial also builds on years of support by WHO/TDR to strengthen research capacity and infrastructure in the Bihar region in partnership with the Indian Council for Medical Research."
OneWorld Health has signed a collaborative agreement with WHO/TDR for paromomycin and will develop and register paromomycin as a 21-day treatment and cure for VL in collaboration with a suitable pharmaceutical manufacturer. Current therapies for VL generally have toxic side effects, face growing parasite resistance or are unaffordable for the vast majority of people afflicted by the disease. Paromomycin should gain regulatory approval in India by 2005, making it a valuable tool to eliminate the disease from the Indian subcontinent.
An estimated 500,000 new cases of visceral leishmaniasis arise worldwide annually. More than 90 percent of cases occur in seven countries: India, Bangladesh, Brazil, Ethiopia, Kenya, Nepal, and Sudan. The visceral form of leishmaniasis, left untreated, is uniformly fatal, and is of growing public health concern since its incidence is increasing rapidly among patients with HIV infection.
OneWorld Health will work with international development, manufacturing and distribution partners. In addition to WHO/TDR (Switzerland), they include the International Dispensary Association (the Netherlands); the Indian Council for Medical Research and four kala-azar centers of excellence in Bihar, India (Kala-azar Medical Research Centre, Muzzafarpur; Rajendra Memorial Research Institute, Patna; Kalazar Research Centres, Muzzafarpur and Patna); Thammasat University (Thailand); and the Forum for Ethical Review Committees in Asia and the Western Pacific (Thailand).
About the Institute for OneWorld Health
The Institute for OneWorld Health, the first nonprofit pharmaceutical company in the U.S., advances global health by developing new affordable medicines for infectious diseases that disproportionately affect people in the developing world. OneWorld Health accomplishes this through an entrepreneurial business model in which its scientists identify promising drug leads and drive their development from pre-clinical studies to clinical trials through regulatory approval. The Institute for OneWorld Health, headquartered in San Francisco, Calif., is a tax-exempt 501(c) (3), U.S. corporation (www.oneworldhealth.org).