In this issue of Quest, you'll find an eight-page pullout called "Gene Therapy Entering the Clinic." This special section reports that, pending only one more go-ahead from a government body, MDA-funded scientists will soon be injecting a corrected gene into a foot muscle of someone with a form of limb-girdle muscular dystrophy. This will be the first time any form of gene therapy for a neuromuscular disease has been tested on humans -- a historic development indeed.

In this space in December 1997, I announced the largest research grant MDA had ever made -- $3.2 million -- to the University of Pennsylvania's Institute for Human Gene Therapy. The safety trial that we expect to begin in a few weeks is the first "bench to bedside" result of the effort fueled by that grant. The Penn group is coordinating the work of MDA researchers at many institutions and, of course, is building on dozens of findings by MDA-supported scientists over virtually the past 50 years.

While this phase 1 trial is certainly a momentous step in MDA research, we understand that its purpose is primarily to see if the delivery of the gene through a particular vector causes any harm. If the safety trial, or "proof of principle" study, passes the first test, we'll take further steps, with our sights set on gene therapy treatments that may restore muscle strength or slow disease progression.

As articles in the supplement explain, limb-girdle dystrophy is the focus of our first gene therapy safety tests primarily for technical reasons. Efforts to develop gene therapy for Duchenne muscular dystrophy have encountered several obstacles. However, with the best scientific minds in the field turned toward this challenge, we're working diligently on a variety of potential therapies for Duchenne -- some involving gene therapy, some involving other equally promising avenues.

The families of those with Duchenne and other genetic neuromuscular diseases can rest assured that all the skills and resources that could effectively be put into play to end these disorders have indeed been mobilized. Testing gene therapy on children with Duchenne is one of our top priorities, and we'll proceed with it as soon as it's safe and responsible to do so.

By the way, we wouldn't be taking this very promising step today without the perseverance not only of our scientists, but also of the volunteers selected for testing and the friends of MDA whose loyalty over the years has enabled us to reach this point. For their faith and dedication, I thank them all.

Robert Ross
Senior Vice President & Executive Director