To safeguard their children, parents will go to any lengths, especially when they are sick. Did you know that although his children were diagnosed with an incurable illness, John Crowley never gave up and found a means to save them?
John Crowley’s two young children were diagnosed with an incurable genetic condition that would kill them in less than a year. He refused to accept this and, with no prior training, started a biotech company that pioneered an experimental enzyme therapy that saved their lives.
John Crowley’s Mission to Help His Children
Megan Crowley, then 15 months old, was diagnosed with Pompe illness on Friday the 13th, 1999. Patrick, her infant brother, was born a few weeks later with the same diagnosis.
Even the doctor who diagnosed the two children was unfamiliar with the illness. He had never handled a Pompe patient before that March day in 1998. He advised their parents, John and Aileen Crowley, to go home and enjoy their time with their young family, as Megan’s time would likely be nine months longer.
A little study was done on Pompe, and no cure was available. Like many other children born with this neuromuscular genetic disorder, Megan was not expected to live past her second birthday.
Crowley, the CEO of Amicus Therapeutics, which he founded in 2002, said in an interview with Pompe Disease News that he decided on determination. He quit a job in finance to pursue a career in pharmaceuticals and has worked for many biotech and pharmaceutical businesses over the last 20 years, including Bristol-Myers Squibb, Sanofi Genzyme, and his first startup, Novozymes. He was working on medicines for his children, Megan, 22, a senior at Notre Dame, and Patrick, 20, and those suffering from uncommon diseases with limited therapeutic choices.
(Source: Pompe Disease News)
The Enzyme Replacement Therapy
When Crowley set out to learn everything he could about Pompe, the few academics who were working on it were experimenting with two different treatment options: enzyme replacement therapy and immunotherapy (ERT)
In 2000, Crowley co-founded Novazyme with William Canfield, MD, Ph.D., a glycobiologist, to discover an ERT that could stabilize Pompe by replacing its missing or inadequate enzyme. He sold the company to Genzyme, as Sanofi Genzyme was then known a year later, to continue to develop the potential treatment before going on with an internally designed ERT. As a senior vice president, Crowley will oversee the company’s global Pompe program.
Infants with Pompe symptoms who emerged before a year were chosen as the optimum candidates for testing the most promising ERT in the program; these children had a worse prognosis than Crowley’s children.
Crowley had hoped that his children would be included in the trial, which began in late 2001, but they were not among the eight enrolled patients. The Testing treatment is in the form of alglucosidase alfa.
Crowley made another firm decision after seeing his children become profoundly weaker: he resigned as senior vice president. Days later, Genzyme approved a two-patient sibling trial of the same alglucosidase alfa intravenous medication that would include his children solely. In January 2003, Megan and Patrick received their first injections.
That saved their lives. It mended their hearts and made them stronger for a while.John Crowley
(Source: Pompe Disease News)