Mr. John Crowley is a remarkable man. He is a father of two children with Pompe’s disease, a rare and terminal neuromuscular disorder, and a biotechnology executive and entrepreneur. There was no treatment for Pompe’s disease when his children were diagnosed with Pompe’s disease. Instead of being depressed and crying, Mr. Crowley looked for a scientist who could potentially develop a drug for Pompe’e disease, established a biotech with him, created a drug, and saved his children’s lives. Because his children were treated at the later stage of the disease, they are still suffering from complications of the disease, but they are alive. Now he is looking for a better treatment to save his children and other patients with Pompe’s disease.
His partner, Dr. William Canfield, MD, PhD, was trained at Washington University Medical Center together with my colleague in the Department of Medicine. The story of Mr. Crowley and Dr. Canfield got the attention of Harrison Ford, and he created a film, Extraordinary Measures. Harrison Ford played a role of Dr. Canfield and Brendan Fraser played a role of Mr. Crowley. We can read their story in a book, The Cure. This book was written by a Pulitzer Prize-winning journalist Geeta Anand. I was inspired and encouraged by their story a few years ago, and still read this book from time to time.
Mr. John Crowley said, “I just didn’t want to get years or decades down the road and regret not having done something. I wanted to know, for our peace of mind and the peace of mind of everybody who loved the kids, that – even if things turned out badly – we had done everything we could, everything humanly possible. I just did what I thought I had to do.” He is an extraordinary man, and I met many people like Mr. Crowley in the past ten years through my quest to find a treatment for Wolfram syndrome.