The October issue of the Human Gene Therapy journal featured an article honoring the memory of Terry Horgan, a 27-year-old with Duchenne muscular dystrophy, who passed away after receiving a CRISPR-based treatment for his condition. An investigation into the case suggested that his death was likely due to an adverse effect of the viral vector used to deliver the treatment. The article titled “What we Owe Terry Horgan: Reflections from Providers, Family, and Scientists” is a touching collection of stories and recollections from the people who knew Terry best and the doctors and scientists that tried to help him.
The first piece was written by Terry Flotte, MD, one of the doctors who saw Terry at the Duchenne Muscular Dystrophy (DMD) center University of Massachusetts’ Chan Medical School. Flotte remembers evaluating Terry’s respiratory status during that initial meeting and making recommendations for his care. Years later, when the conversation of treating Terry with gene therapy came up, he would also offer advice and insights on treatment risks and preventing immune complications.
Reflecting on the case and its aftermath, Flotte writes “I deeply regret that the therapy did not work for him. I regret that neither I, nor any other member of the team, was smart enough to prevent or reverse the terrible consequences that ensued. But along with that regret, I also owe Terry many things. I owe it to him to get every possible bit of data from his case published and disseminated to all gene therapy scientists working in this field. I owe it to him to do my part in scientific discussions of how to overcome obstacles preventing older DMD patients from gaining the benefits of gene therapy. I owe it him to speak out on behalf of patients who are being left behind as gene therapy advances, because they are too old or too sick or have a disease that is too rare to make for a neat and easy gene therapy success story.”
In their contribution to the editorial, Angela Lek, PhD, vice president of research at the Muscular Dystrophy Association and Monkol Lek, PhD, an assistant professor of genetics at Yale University School of Medicine echoed similar sentiments. They worked on devising the CRISPR-based therapy that Terry received and on testing it in cells and then living mice. “The findings from Terry’s case as well as other recent deaths in gene therapies provide painful but valuable lessons that will help de-risk the translation of gene therapies for future patients,” they write. “Terry—thank you for being our astronaut and willing to brave the unknowns of safe travel for future generations. Wherever you are—we hope you are having the adventure of a lifetime, free from the constraints of your physical body.”
Fittingly, the editorial concludes with reflections from Terry’s brother, Richard Horgan. In his writing, Richard highlights happier times with the brother who did not let his disease define him. He talks about his brother’s many accomplishments including graduating at the top of his high school class and going on to study information science at Cornell University. He describes a young man with a big heart willing to help those around him and who remained hopeful that there would be a cure for his disease even as his body deteriorated. Richard started Cure Rare Disease with an eye towards harnessing the power of gene editing to help his brother. He reflects on the years-long process of working with scientists to develop a treatment for Terry and on the work the organization is doing now to continue advancing gene therapy and gene-editing technologies to help families dealing with rare and ultra-rare diseases.
Today, Cure Rare Disease is involved in over 15 drug development programs—ranging from rare mutations of DMD, to a growing number of limb-girdle muscular dystrophy subtypes, to distal myopathies and neurodegenerative diseases. And the organization works with collaborators around the world. In his commentary, Richard writes, “We are united across geographies, across race and socioeconomics in a shared journey to define a new future, regardless of the number of patients that an individual disease impacts and regardless of industry’s desire to help realize that future. This is my call to those with whom this message resonates—the small percentage of game changers this world has to offer—to join us. Together we can, we have, and we will continue to push that bleeding edge forward to capture all who have been forgotten.”
Read more about Cure Rare Disease here and a letter from Terry Horgan’s parents commemorating their son’s life and legacy here.
