Jump-Start on Slow Trek to Treatment for a Disease

Last month, the Bill and Melinda Gates Foundation donated $19 million to the Drugs for Neglected Diseases Initiative to further one of its goals: finding a new drug for African sleeping sickness.
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But the gift spotlights just how tricky the search for new treatments can be when the disease is fearsome but nearly forgotten because its victims are poor and obscure.
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“Sleeping sickness” is too benign a nickname for human African trypanosomiasis, which is caused by a protozoan spread by biting tsetse flies. When the parasites enter the brain, victims hallucinate wildly. They have been known to chase neighbors with machetes, throw themselves into latrines and scream with pain at the touch of water. Only at the end do they lapse into a lassitude so great that they cannot eat, followed by coma and death.

About 150,000 people contract the disease each year, but 50 million people in 36 countries live in areas where they are at risk.

The best treatment now is eflornithine, sometimes called the resurrection drug because it can pull the dying out of comas.

It is almost a miracle that eflornithine is available. It was discovered in 1980 at Pace University in New York. By early 2000, the last 7,500 doses in the world were running out. The patentholder, a precursor of the drug maker Sanofi-Aventis, abandoned it in 1995 because it had not lived up to its anticancer potential. Then, in late 2000, plans to make a topical form emerged. It was the key ingredient in Vaniqa, a cream to prevent facial hair in women.

After critics accused Sanofi-Aventis of catering to vain rich women while letting poor Africans die, the company agreed to make an injectable form of the drug and now gives it free to the World Health Organization and Doctors Without Borders.

But in rural Africa, eflornithine is very hard to use. Patients need intravenous infusions four times a day for two weeks. When a “hospital” is a row of iron beds under a thatched roof, and the “nursing staff” is mostly relatives of the sick who sleep on the floor, round-the-clock treatment is hard. There might be no night nurse to insert an IV line.

For that reason, many countries do not adopt it. They still use the drug melarsoprol, which, Dr. Pécoul said, “is not effective and sometimes kills.”