The world's most neglected diseasesBMJ 2002; 325 doi: https://doi.org/10.1136/bmj.325.7357.176 (Published 27 July 2002) Cite this as: BMJ 2002;325:176
All rapid responses
Allow me to make some comments on your recent editorial on the
world´s most neglected diseases. As my company happens to be the main
provider of medicines used to combat African sleeping sickness, I would
like to provide you and your readership with some precisions about the
general situation and to correct some misunderstandings raised in your
The statement that "current drugs are in scarce supply" is unfounded.
Even before the agreement signed last year between Aventis and WHO, the
three major drugs used against African sleeping sickness melarsoprol,
pentamidine and eflornithine have been available. Pentamidine had been
donated free of charge to WHO for more than ten years
by Rhone-Poulenc, melarsoprol was never in shortage and marketed at an
affordable price. Through WHO, even eflornithine was available to patients
I would also like to comment on the history of the clinical
development of eflornithine, which, in the public, is sometimes presented
in a misleading way.
Eflornithine was discovered in the 1970's by the Merrell
International Research Center. The molecule was part of the
company's research program on cancer and anti-infectives. In 1981, the
Merrell company was acquired by Dow Chemical, Merrell Dow Pharmaceuticals
was created. In 1989, Marion Merrell Dow was formed from the merger of
Marion Laboratories and Merrell Dow. In 1995, a merger between Hoechst AG,
Marion Merrell Dow and Roussel Uclaf created Hoechst Marion Roussel,
which, at the end of 1999, was part of the merger to form Aventis,
together with Rhône-Poulenc Rorer.
In 1979, Cyrus Baachi, a biochemist with Haskins Laboratories at New
York´s Pace University, discovered that the molecule cured mice with
trypanosome infection. The company, while pursuing the development of the
product against cancer, accepted to finance the development according to
FDA standards for the sleeping sickness indication, collaborating with WHO
to give access to patients in African endemic countries. The product was
registered as an orphan drug with the FDA in 1990, then in the endemic
countries from on 1993. This achievement was
publicly praised by Dr Nakajima, then Director General of WHO.
Since sleeping sickness was not the target of activities of the
company, it had been agreed in 1989, that, once
eflornithine was registered, the company would care for a batch of
product, equivalent to a two years demand, and hand over the patent rights
to WHO. Meanwhile, the organisation should identify a producer for the
Unfortunately, this approach was not successful. No other manufacturer
could be identified. In order not to create a shortage at WHO, the company
arranged for new batches which were produced in 1995 and 1997.
When Aventis was formed, the new company happened to have the three
major products to treat African sleeping sickness in its combined
portfolio. It decided to propose to WHO a joint initiative on this "most
neglected disease". First contacts were made in May 2000, long before an
eflornithine-based cosmetic product was launched on the US market. These
negotiations led to the agreement signed on May 3rd, 2001, between Aventis
and the WHO. >
At the end of 2000, Médécins Sans Frontières got to hear about the
U.S. launch of Vaniqa, a facial cream to treat female hirsutism. They
approached Bristol Myers Squibb to come to an arrangement in favour of
sleeping sickness patients in Africa. However, BMS did not dispose
of the technological process to make injectable eflornithine. Since
Aventis was already in discussion with WHO, BMS announced to donate
eflornithine active ingredient for the first year of the sleeping sickness
plus a donation in cash to WHO.
From the above, it should become clear that there is a difference
between facts and public perception: It was not the arrival of a cosmetic
product on the US market which changed the overall situation. The decisive
change was the creation of Aventis, a company with a portfolio apt
to propose to WHO a major program to jointly combat African sleeping
sickness in the most endemic countries. The program is now in its second
year, to the satisfaction of WHO, of MSF, and of Aventis.
Dr. Robert G. Geursen, PhD, MD
Head of Corporate Public Policy
D-65926 Frankfurt am Main
Competing interests: No competing interests
Gavin Yamey is to be commended on his article detailing lack of
research on treatments for Infectious Diseases which cause great mortality
in developing countries. There are other diseases which, although not
associated with a high mortality, cause considerable morbidity and result
in blighted lives and a high communal economic burden.
Mycobacterium ulcerans Infection, often called "The Buruli Ulcer", has now
displaced leprosy in many places in west Africa to become the second most
important mycobacterial infection of immunocompetent man. Thousands of
patients in endemic areas are infected annually. Untreated patients
develop hideous scars, contractures, loss of limbs following necessary
amputation, loss of breasts in women, blindness and chronic bone
infections. Treatment at an advanced stage of infection requires weeks or
months of hospitalisation, repeated surgery and wound dressings with loss
of schooling, as well as loss of domestic and agricultural activity. The
infection responds poorly to existing antibiotic regimes and vaccine
prevention (BCG) is of limited efficacy.
There is lack of support for development of new drugs or vaccines to treat
the disease, even lack of support for trials of existing drugs in
treatment regimes which when added to surgery may be of benefit in
preventing recurrences or bone infection.
I ask that your Editor to consider morbidity as well as mortality when
assessing the burden of Infectious Diseases in developing countries and to
add Mycobacterium ulcerans Infection to the list of neglected diseases.
Although they may never read it, your editorial is extremely relevant to
those who suffer from these diseases.
Competing interests: No competing interests