As many of you know, our flagship gene therapy trial, designed to test the safety of gene transfer in limb-girdle muscular dystrophy, has been suspended by the Food and Drug Administration since January. The suspension, which includes several other gene therapy trials, stems from the death in September of a young man taking part in a gene therapy study for a metabolic disorder known as ornithine transcarbamylase deficiency at the University of Pennsylvania in Philadelphia.

The OTC deficiency trial had nothing to do with our own trial except that both were conducted under the auspices of the Institute for Human Gene Therapy at Penn. (The actual injections in our muscular dystrophy trial were being given at Ohio State University in Columbus.)

In the months since the tragic death in the OTC trial, Penn's institute has come under intense scrutiny by the FDA and the National Institutes of Health. At the same time, these agencies have themselves been the subject of scrutiny for possible lapses in carrying out their oversight responsibilities.

I want to reassure the families served by MDA that we're exploring every avenue to make possible the continuation of our first gene therapy trial and the start of others like it - either through the University of Pennsylvania or elsewhere.

I can also assure you that MDA has never and will never fund a clinical trial in which the well-being of anyone participating is jeopardized, no matter how exciting the research is or how promising the potential treatment seems. Two people have been injected with genes for sarcoglycan proteins in our muscular dystrophy trial, and neither has experienced any problems whatsoever.

MDA is now looking at the track records of medical centers at which so-called viral vectors (modified viruses used to carry genes into cells) can be produced to see which center can produce the best and the safest vectors - and do it the soonest. If the institute at Penn can satisfy the FDA's requirements for safe oversight of trials and can resume producing the needed vectors for our LGMD trial, we may keep our trial at Penn's institute. If not, we'll take another direction, possibly involving other institutions and maybe (at least eventually) other ways to deliver genes.

Stem cells, for example, can be thought of as gene therapy vectors (see "Renewing Muscles and Nerves"). In fact, the rapidly evolving field of stem cell transfer may make obsolete some of the forms of gene therapy we've considered up until now.

The future direction of gene transfer strategies remains to be revealed. But you have my word: MDA will do everything in its power to continue its pursuit of treatments and cures for neuromuscular disorders - and to do it as quickly and safely as is humanly possible.