Crowley Family

John Crowley’s life is literally a Hollywood movie. His journey to save his children’s life was turned into the movie “Extraordinary Measures” starring Brendan Fraser and Harrison Ford, and his family has been featured in a number of magazine and newspaper articles, and in two books. John changed his career path to help find a cure for the disease that could end his children’s life, and to help others diagnosed with rare diseases, and along the way, has inspired many others with his leadership skills.

In March 1998, when John’s daughter Megan was 15 months old, she and her newborn brother, Patrick, were both diagnosed with Pompe disease. John and his wife Aileen were told their two younger children would not live past their second birthday.

Pompe disease is a neuromuscular disorder caused by a genetic mutation that prevents GAA (acid alpha-glucosidase) production. The result is a toxic buildup of glycogen which in turn causes a number of symptoms, including muscle weakness and swelling of vital organs, and ultimately, respiratory issues and heart failure. Symptoms can be managed with infusions of Lumizyme, which breaks down the glycogen, but it does not treat the cause of the buildup.

When John learned about the prognosis for his children, he felt he had to do more than just sit and watch their health decline. He was well educated, with a bachelor’s degree in foreign service from Georgetown University, a law degree from Notre Dame, and a Harvard MBA. His left his career in management consulting to take a position with a pharmaceutical company in 1998. In 2000, John co-found Novazyme Pharmaceuticals, a startup biotech, with glycobiologist, William Canfield, MD, PhD. Cash advances on credit cards and home equity line of credit on the Crowley’s home helped fund the start up. The gamble would be worth it in the long run.

Novazyme was bought by Genzyme within a couple of years. Genzyme has successfully received FDA approvals for treatments for rare diseases, and eventually created Myozyme, which was a life-saving medicine for Pompe disease. It was not an easy process for Megan and Patrick though. The Genzyme team thought they would have better success with infants to test the drug, and the Crowley children were already toddlers at this point.

Even when Genzyme agreed to test the drug on older children, the Crowley’s children did not quality. Finally, Genzyme saw an opportunity to create a clinical trial just for Megan and Patrick, because while both children have an identical genetic mutation, Patrick is affected more than Megan. This “sibling test” gave Genzyme the opportunity to study why the treatment was more effective for some children than others. First, John had to resign from the company in order to avoid any conflicts for Megan and Patrick to be eligible for a clinical trial.

“We hadn’t seen her [Megan] smile in two years. After the first couple of months, we started to notice that she was smiling again.” John shared, “That was the first sign to me that there was some hope.”

John and Aileen with Megan

John and Aileen Crowley with Megan at her undergraduate graduation at Notre Dame.

Within the first few months of treatment, Megan and Patrick gained muscle strength, and their enlarged hearts returned to the normal size. As the study theorized, Megan did respond better to the treatment than Patrick, but both showed signs of improvement. That life-saving drug, Myozyme, was redeveloped into Lumizyme, and now, more than 3,000 people diagnosed with Pompe worldwide receive the treatments.

John went on to found Amicus Therapeutics, a biotech company that develops treatments for those diagnosed with rare diseases, including new treatments for those with Pompe disease. He has been a passionate advocate for providing universal access to treatments for those with rare diseases, as well as championing needs for those with disabilities.

“In our view, nobody in the world should ever be denied our medicine,” John said. “So we have to figure out creative ways to get it to everybody.”

John was introduced to Global Genes by founder Nicole Boice in 2009 when she was in the planning stages for the non-profit. John and some other contributors helped Nicole to envision the final product as an umbrella organization in the rare disease community. John became a founding board member in 2010 when Global Genes was founded.

John told Nicole, “I love your passion, and I love the vision here. There’s an unbelievable opportunity to fill a huge void.”

As a researcher, John is still seeking new and better treatments through his work with Amicus, as a board member for Global Genes, and a former board member for the Make-A-Wish Foundation. As a father, John knows that his children are all adults now, and their medical decisions are their own to make. The Crowley’s oldest child, John Jr., was diagnosed with dyslexia, ADHD and Asperger’s syndrome as a child, so the Crowley family has had to navigate many diagnostic and treatment journeys throughout their kids’ childhoods.

“I think I did my job,” John said. “As a dad, I did what I had to do. I don’t think that makes me a hero.”

In addition to all of his work in the world of rare diseases and being a crusader for those living with disabilities, John has also become known as an authority in leadership, and has spoken to numerous groups about his experiences as a leader in challenging situations. Among his many speaking engagements, John spoke at the 2020 commencement at his alma mater, Notre Dame, at the Rothman Institute of Entrepreneurship at Fairleigh Dickinson University, and at the University of Georgia’s Terry College of Business for the Mason Public Leadership Seminar.

Pulitzer Prize-winning reporter, Geeta Anand, captured the Crowley’s odyssey from diagnosis to finding a treatment, first in a Washington Post article, then, in a book The Cure: How a Father Raised $100 Million–and Bucked the Medical Establishment–in a Quest to Save His Children. The book was the inspiration for the movie, Extraordinary Measures, starring Harrison Ford, Brendan Fraser and Keri Russell.

Imagine what the world would be like if John had not taken the extraordinary measures for his children to save their lives, and in turn affect the lives of thousands of others with Pompe disease, and all of the other lives of those diagnosed with rare diseases and living with disabilities that he has affected.

The Crowley Family at the premiere of Extraordinary Measures with Harrison Ford, Brendan Fraser & Keri Russell

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