In 2001, Dr. Victoria Hale approached the foundation seeking support for a risky new venture she had started the year before, shortly after leaving the successful biotechnology company Genentech. Dr. Hale’s venture, named the Institute for OneWorld Health, was a first in the United States—a nonprofit pharmaceutical firm. Dr. Hale explained that iOWH would focus exclusively on developing new medicines for “diseases of poverty” and would be driven by “global need rather than financial opportunity.”
When Dr. Hale approached us, she had just found her first drug candidate: an antibiotic called paromomycin that she believed could be repurposed as a cure for VL. Equally important, she had found that the government of India was eager to partner with her.
Paromomycin had been patented about 50 years before by an Italian pharmaceutical firm for treating infections unrelated to VL, but the drug had fallen into disuse when new products came to market. Only one small facility in Malta was still producing it and was thinking about closing down production.
In the late 1980s, Kenyan physician Dr. Charles Chunge was able to obtain a modest quantity of the drug and, with the support of the World Health Organization (WHO), tested it as a potential cure for VL. His study found that paromomycin was effective in curing all 53 patients who received it. Additional small studies in India produced similarly positive results. The WHO reviewed the results of these studies but was not able to secure the funding to follow them up.
That is where Dr. Hale picked up the challenge. In order to bring paromomycin to market for this new use, her firm would have to 1) work with the WHO, the government of India, and private researchers to organize and carry out a large and expensive clinical-trial program and 2) navigate India’s challenging regulatory-approval process.
In May 2002, the foundation approved the first of three grants to finance this effort. iOWH then initiated the largest-ever clinical trial for VL with the help of Indian doctors who had been treating this disease for decades.
The clinical trial proved more difficult and time-consuming than we anticipated. As newcomers to the global-health field, the foundation and iOWH both had short track records and were met with hesitation by people on the ground in India as well as by international health organizations. For example, local political figures were not eager to have outsiders shine a spotlight on problems in their communities—even in the context of an offer to help address those problems. More surprising was the fact that an international agency was reluctant to share important data from previous paromomycin trials. (For more on these challenges, see “Key Lessons,” below.)
But when iOWH completed the trial and the results came in, they confirmed iOWH’s greatest hopes for the drug: Paromomycin cured 95 percent of the 667 patients in the trial, without causing significant side effects.
Dr. Hale and her team are now working to secure regulatory approval for the drug in India. This month (June 2006), iOWH will hand-deliver a final “dossier” of more than 7,000 pages to the office of the Drug Controller General of India. They hope to receive approval by this fall. If all goes well, they may present a similar dossier to the U.S. Food and Drug Administration (FDA). An endorsement from the FDA would carry weight with health authorities in all the countries where VL exists.
Next: Results