Oct 28, 2002
By Richard Woodman
Researchers announced plans on Monday for a major trial of a controversial stem cell therapy in patients with severe multiple sclerosis who have not responded to conventional treatment.
Professor Giovanni Mancardi from the University of Genoa in Italy said up to 240 people in 30 centres would take part, even though early studies suggest that the new therapy carries a high mortality risk as well as possible clinical benefits.
"The mortality is important and cannot be eliminated. The justification is that there is a population of MS patients who worsen rapidly in spite of conventional therapy," he told Reuters Health.
Dr. Mancardi told the European Federation of Neurological Societies congress in Vienna that a study of 16 patients he had carried out in Italy suggested that this method can suppress inflammation in severe MS for at least 2 years.
The therapy involves autologous haematopoietic stem cell transplantation, in which patients' own stored stem cells are reimplanted after drug treatment to knock out the immune system in order to stabilise the course of MS.
The mortality rate normally expected with this procedure is around 5%.
Dr. Mancardi said that in his study of 16 patients, MRI scans showed a complete stop of inflammatory activity sustained with time. The median follow up was 24 months, ranging from 5 to 42 months. Also, he said, "Quality of life improved. Clinically, 14 cases remained stable or slightly improved while two cases resumed worsening after nine and 36 months respectively."
The statement announcing the findings admitted the therapy was a "high risk strategy with experts divided over whether it is appropriate to treat a non-fatal condition in this way". Dr. Mancardi said: "We realise it is a very aggressive form of treatment but for some patients it could be the only possible option."
He said plans for the major new trial would be approved by the European Bone Marrow Transplantation Society, probably before the end of October, though various ethical committees also had to give consent.
The countries so far expected to take part were Spain, Switzerland, The Netherlands, Sweden, Poland, Italy, Greece, and Turkey.
A spokesman for Britain's MS Society said: "Research into treatments
for people with very severe MS is to be encouraged. This is a new therapy
with acknowledged risks so we must be cautious about raising expectations."
© 2002 Reuters Ltd