The independent panel that is advising Oxford GlycoSciences on the compassionate use of OGT 918 met on Monday 13th March 2000. After reviewing the available information from studies in animals and man and a lengthy discussion, it was decided that it was inappropriate to release OGT on an individual patient basis for compassionate use. The panel advised that any use of OGT 918 should be as a controlled trial so that any potential benefits and the safety could be assessed properly. However, at the present time, there was insufficient information about the pharmacokinetics, efficacy and safety of OGT 918 in children to be able to make any firm recommendations. Decisions about the compassionate use in adults will await the outcome of current trials.
Further meetings are to be held when more details are available.
The first clinical trials have now been completed on patients with Type 1 Gaucher's Disease and the results published in Lancet. A second Gaucher's trial is presently underway as is a trial on Fabray's disease. The agreement was reached, in part, because of the research which has already been done with NPC mice. No date has been set for these clinical trials. Researchers are working with Oxford GlycoSciences to develop the appropriate protocols which will govern the trials.