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Perspective: Advancing Hope for Andrew and Countless Others

By Nate Plasman

This February, I had the opportunity to travel to Capitol Hill to participate in the Institute for Gene Therapy’s Congressional Briefing. I don’t come from a scientific or particularly political background – addressing members of Congress and their staff in Washington, DC was not something I ever envisioned myself doing. The reason I made the trip was to talk about my son and my family’s journey with gene therapy.

Andrew Gene Plasman was born on July 1, 2014. His middle name is in fact Gene, spelled G-E-N-E. Occasionally I like to get a little theatrical and say, “Now from Lombard, Illinois, put your hands together for Andrew Gene Therapy Plasman!” Now in second grade, Andrew gets a little embarrassed when I do that, but it’s a truly perfect name for our son who has pioneered many firsts for our family and beyond.

Andrew seemed to be a healthy little boy for the first year or so of his life, but as time went on, we noticed that he just seemed “floppier” than other children his age. My wife Sara was diligent from the start about researching possible causes, taking him to doctor’s appointments, and enrolling him in occupational, speech, and physical therapy. Just after Andrew’s second birthday, he was diagnosed with Duchenne muscular dystrophy (DMD).

DMD is a genetic disorder characterized by progressive muscle degeneration and is predominant in boys. In the past, most people with DMD did not live beyond their early 20s. If Andrew had been born a few decades before, the doctors would have told us that there was nothing to be done. Our son would be in a wheelchair between the ages of eight and 10, and we would bury him before he turned 16. We are so fortunate that today that is not our reality.

Soon after Andrew’s diagnosis, we were introduced to the promise that gene therapy holds in treating young boys with DMD. With the support of our doctors, nine months after we initially reached out, we were incredibly fortunate that Andrew was selected for a clinical trial. This ushered in a new chapter of our lives.

At about five years old, Andrew understandably didn’t grasp the promise that this opportunity offered him, and our family. When the day came for him to receive his infusion, he was more focused on Paw Patrol than on the magnitude of what was about to happen. Ninety minutes went by, and Andrew’s infusion was complete. We knew that in this particular clinical trial Andrew would either receive the gene therapy at week one or week 49.

Just a few weeks later, we knew in our hearts and from our observations that he had received the therapy.

Now in second grade, Andrew has made incredible progress; progress that would have been impossible without gene therapy. Andrew interacts and plays with his classmates like a young boy should. In fact, we’ve heard from his teachers that in his classroom of 24 second graders, he is the only one who knows he has DMD.

Being able to share our experience with Congressional staff members – to help demonstrate just how incredible the science of gene therapy is – and give just one example of how significantly gene therapies can change lives was a very important opportunity for me to share on behalf of Andrew and our family. Leaders in Congress must move with tremendous urgency to ensure that policy is keeping up with the quickly progressing science. For the families facing a DMD diagnosis, every day, week, and month means so much. The window for treatment and for arresting the progression of the disease is short. We must make sure that innovative new therapies for this disease, and countless other orphan diseases, can quickly make their way through the manufacturing pipeline to patients who need them.

I hope that policymakers, researchers, manufacturers, and all others in this space can come together for patients and their families, and not just for the DMD community but for all patients and families who are living in hope of any treatment, but especially one as transformative as gene therapy. IGT’s briefing gave us an opportunity to have a meaningful conversation on the expansive value of gene therapy and I hope this momentum continues to build.

We see our son walking across the stage to get his high school diploma in 2033. We see him going to college, having a career, hopefully falling in love, and starting a family of his own. This is a whole new paradigm shift for genetic diseases. Because of gene therapy, a new day is dawning. Our son Andrew is proof of that.


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