Neglected Diseases Update

Sleeping sickness is estimated to cause 48,000 to 100,000 deaths a year in Sub-Saharan Africa. Effective disease control has been hampered by lack of safe oral drug treatments. Friday, Essential.org’s IPhealth listserv emailed a revised news article from SciDev.Net about a breakthrough cure. The email, prefaced by the Drugs for Neglected Diseases initiative (DNDi), stated that, although DNDi did not agree with the original SciDev.net article, the new drug combination (eflornithine and nifurtimox) appears to be efficacious against the Trypanosoma brucei parasite [Jan 10, “Calls for fast access to sleeping sickness drug“].

According to Girardo Priotto of Doctors Without Borders, “The situation is so desperate in the field that we are not happy with two more years of waiting for the final results of the current trial, so we are looking for ways of extending access to this treatment through additional studies.”…

Thousands of people each year are diagnosed with advanced-stage sleeping sickness, which is fatal if not treated. Current treatment, with the drug melarsoprol, itself causes the death of around six per cent of patients. In addition, some patients are also resistant.

No data were released in this report and it remains unclear how efficacious the new treatment regime may be. ClinicTrials.gov details the study design and collaborators.

More broadly in the neglected diseases field, there is resurgent interest in drug development: from discovery to trials to regulatory approval. OneWorldHealth is a well publicized example [see Oct 8th post “Non Profit Rx Venture“] and several others including Essential.org, DNDi, and the Tropical Disease Initiative [check out the 50 min video on Shannon’s Blog], each of which specialize in some link on the drug development chain. The World Health Organization and many high profile partners are also putting more muscle into the neglected diseases fight. Check out the October Preventive Chemotherapy in Human Helminthiasis news release, quoted here:

“Preventive chemotherapy does not necessarily stop infection taking place but it can help to reduce transmission,” the Director of the UN World Health Organization, Department for the Control of Neglected Tropical Diseases, Lorenzo Savioli, said. “The benefit of preventive chemotherapy is that it immediately improves health and prevents irreversible disease in adults.”The approach contained in a newly published manual, Preventive Chemotherapy in Human Helminthiasis, focuses on using a set of low-cost or free drugs to simultaneously treat the four most common diseases caused by worms and afflicting over 1 billion people: river blindness (onchocerciasis), elephantiasis (lymphatic filariasis), chistosomiasis, and soil-transmitted helminthiasis. The cost: as low as 40 cents per person per year.

“In the same way as we protect people against a number of vaccine-preventable diseases throughout their lives, the regular and coordinated use of a few drugs can protect people against worm-induced disease, improving children’s performance at school and the economic productivity of adults,” Mr. Savioli said.

The new approach provides a critical first step in combining treatment for diseases which, although different, require common resources and delivery strategies for control or elimination. The second key component brings together for the first time dozens of agencies, non-governmental organizations (NGOs), pharmaceutical companies and others into a coordinated assault on neglected diseases.

The diseases’ impact can be measured in the impaired growth and development of children, complications during pregnancies, underweight babies, significant and sometimes disabling disfigurements, blindness, social stigma, and reduced economic productivity and household incomes.

These effects can be dramatically reduced by using highly effective drugs of proven quality and excellent safety record - the majority donated free by companies or costing less than $0.40 per person per year, including the cost of the drugs and their delivery.

Another recent development in neglected diseases drug discovery by British researchers [Guardian story, hat tip to Innovation blog] has huge potential cost-savings but, at least according to the Innovation blog, the technique “effectively amounts to a sophisticated form of reverse engineering that skirts patent laws and this is not the same as conducting original drug development.” Definitely a story to watch…