UN predicts cure for hemophilia by year 2000
By Stephanie Nebehay
GENEVA (Reuter): The World Health Organization (WHO) predicted recently there would be a cure for hemophilia, using gene therapy, by the year 2000.
Gene therapy is the insertion into cells of a synthetic, normal gene to replace the defective one that causes hemophilia, the most frequent inherited blood disorder.
Transmitted by mothers to their sons, the disease can cause a life-threatening tendency towards excessive bleeding for the estimated 100,000 to 350,000 male sufferers worldwide.
A cure would also remove the risk to people being treated for the disease of being contaminated via blood transfusions with the virus that causes Acquired Immune Deficiency Syndrome (AIDS).
The U.N. agency said the conclusion that a cure was "within reach" was made after three-day talks in Geneva by international experts on hemophilia, which affects one male in every 10,000.
"The first cases of cure of the disease through the technique of gene therapy will be achieved, given essential further developments, by the end of the decade, the experts predict," the WHO said in a statement.
"Their predictions for the first gene therapy cures for the disorder are based on their assessments of the latest scientific progress in the field," it added.
The problem of providing care for hemophilia patients in developing countries will remain a major challenge until scientific advances become affordable to all, the WHO added.
Research is being carried out mainly in Britain, the United States and Japan, according to the agency.
The experts meeting at WHO headquarters were from Australia, India, Italy, Sweden, the United Kingdom and the United States.
Pier Mannucci, head of the WHO collaborating center for hemophilia and related disorders, chaired the talks.
Permanent cure
The WHO quoted him as saying: "The availability of safer, plasma-derived coagulation factor concentrates, and those produced by genetic engineering will provide reasonably good treatment until gene therapy becomes available as a permanent cure."
Hemophilia is currently treated by the transfusion of blood products which contain the clotting factors that are deficient in such patients.
However, blood products not thoroughly screened can sometimes transmit viruses such as hepatitis and "in rare instances" the human immunodeficiency virus (HIV) that is believed to cause AIDS, according to the agency.
Scandals over AIDS-tainted blood supplies have erupted in France and Germany, outraging hemophiliacs and others.
In France, two former health officials were jailed last year over the contamination of hundreds of hemophiliacs in 1985 with AIDS-tainted blood. About 400 of the more than 1,250 infected hemophiliacs have died.
Three German firms have been shut down on suspicion they failed to make sure their blood products were free from HIV.