NEW YORK - November 15 - Preliminary results from a study conducted by the international medical humanitarian organization Doctors Without Borders/Médecins Sans Frontières (MSF) show that significantly shorter and simplified treatment of African sleeping sickness (human African trypanosomiasis — HAT) could be possible in the near future. Announcing the study today at the 55th annual meeting of the American Society of Tropical Medicine and Hygiene in Atlanta, the organization stressed that measures must be undertaken to ensure that this treatment can be employed up as soon as possible at country level, and that older, toxic treatments are phased out."
As part of a larger study being conducted by MSF and the non-profit product development partnership, Drugs for Neglected Diseases initiative (DNDi), the preliminary results from 103 patients in the Republic of Congo demonstrate that a combination therapy of eflornithine and nifurtimox is a safe and effective alternative to currently used monotherapies. Eflornithine, currently the preferred first-line therapy for treating sleeping sickness, is only available to 20 percent of people stricken with sleeping sickness, most of whom are treated by MSF. Remaining patients continue to be treated with melarsoprol, an arsenic derivative used since the 1940s that is lethal for five percent of those treated.
"Due to its toxicity, we need to see a phasing out of melarsoprol as the primary drug for treating sleeping sickness," said Dr. Gerardo Priotto, principal investigator of the study. "We have now shown that combining eflornithine with nifurtimox makes it possible to simplify and make safer the treatment of sleeping sickness. Countries should not settle for a drug that kills one out of every twenty patients."
Doctors sometimes refer to eflornithine the "resurrection drug," as it can pull patients with sleeping sickness out of a comatose state. However, treating patients with eflornithine alone is fairly complex, requiring infusions every six hours over a period of two weeks, which demands steady nursing care and places a significant burden on patients. When combined with nifurtimox tablets for 10 days, as the study shows, patients only require one week of infusions twice a day. Therefore, treatment is significantly simplified.
"Success in scaling up the use of eflornithine-based therapies depends on national health authorities and the World Health Organization (WHO) working together to improve the capacity of nursing care and to provide free infusion kits," said Dr. Unni Karunakara, Medical Director of MSF`s Access to Essential Medicines Campaign. "WHO also needs to ensure the easy accessibility to nifurtimox, so that the immediate needs of HAT patients can be met by shorter treatment through combination therapy."
While this combination is expected to represent an improved treatment for patients, it is not ideal; the treatment is still relatively complicated to administer and requires close patient monitoring. Hence, major efforts are needed to bring truly innovative drugs into the pipeline. As a consequence, the current DNDi HAT portfolio is focused on the early discovery stage of drug research & development. By focusing on discovery today, DNDi aims to ensure that two new drug candidates for clinical testing are delivered by the end of 2011.
The WHO estimates that 50,000-70,000 people in sub-Saharan Africa are infected with sleeping sickness each year. MSF has been treating patients with the disease for 20 years, since it opened its first project in northern Uganda in 1986. In total, MSF has screened over 2.4 million people for the disease and has treated over 43,000. MSF remains a major provider of sleeping sickness treatment with six programs in all of the significantly affected countries; MSF's share of the total caseload was 18% in 2004, and MSF is currently responsible for the efficient supply and distribution of all HAT drugs used in the world today.