Founded on the Strength of a Parent’s Love

There are few things in the world more powerful than a parent’s love, and the fact that two Princeton children are alive today is a testament to their father’s determination to find a cure for their rare and nearly always fatal disease.

The Crowley family’s story was made into a feature movie, "Extraordinary Measures," featuring Brendan Fraser as John Crowley and Harrison Ford as the brilliant scientist who helps him in the race against time to save two of Crowley’s children, Megan and Patrick, who in 1998 were diagnosed with a genetic neuromuscular disorder called Pompe Disease.

In the course of pursuing the experimental trial that saved his children’s lives, and in his continuing efforts to help other families find cures for rare diseases, Crowley, of Princeton, became the driving force behind several biotech companies.

One is Amicus Therapeutics, Inc., a firm with Princeton roots now based in Cranbury.  Though Crowley remains active as chairman of the company’s board of directors, just last month Matthew Patterson took on the role of president and acting chief executive officer. 

Amicus Therapeutics employs 105 people in its Cranbury location as well as a small research team in San Diego and remains dedicated to developing medicines to treat rare genetic diseases often ignored by the larger research community.

“It’s a huge aspect of who we are as a company,” Patterson said. “It’s not just about the treatment of the disease symptoms; it’s about understanding the impact on someone’s life, how they can affect someone's functioning day-to-day, and spend time with family and friends that we take for granted. 

“As a company, you develop a relationship with the patient community that is different from companies that deal with the large diseases,” Patterson said.

Amicus is devoted to patient advocacy and helps patients and families navigate the daily challenges of living with a rare disease, finding external support services and staying up-to-date on clinical trials.

“A very unique and personal touch to how Amicus has grown is because of John and Aileen (Crowley’s) personal experiences, the strength of their conviction and love for their children,” Patterson said. “Megan is 14, and Patrick is 12, going on 13. They lived longer than they were supposed to because they received a treatment that became available after many years of research. It’s parents like the Crowleys who won’t accept the fact that there’s no research going on, people who will take out a second mortgage and raise the money themselves – these are the people who will make the difference and take their passion out into the community.” 

The company’s research currently is focused largely on three conditions — inherited lysosomal storage disorders — caused by an enzyme deficiency. Fabry Disease is estimated to affect 5,000 to 10,000 people worldwide; Gaucher Disease affects 8,000 to 10,000 people worldwide; and Pompe Disease, 5,000-10,000 people worldwide, including Megan and Patrick Crowley, who still live in Princeton.

 “Our mission is to build on our science and build on a number of different diseases,” Patterson said. “Our focus is the rare inherited diseases, but our research has broader applications. Parkinson’s, Alzheimer’s, these are really becoming very interesting subjects, particularly as baby boomers age and there is lots of investment in these areas for companies. In the course of our research on the rare diseases, we are learning so much that can have wider implications.”

While Amicus has occupied space at its Cedarbrook Drive campus since the spring of 2005, it was originally founded in the New Jersey Technology Center, the state-sponsored tech center on Route 1 in North Brunswick, with a handful of people before developing the momentum to grow and move to Cranbury.

“We are the perfect poster child for New Jersey business,” Patterson said. “With a little financial help early on, the state of New Jersey helped us succeed and that allowed us to stay here, flourish, create jobs, hire people from the area and create revenue for the state.”

Amicus Therapeutics was named one of NJBIZ’s 2009 Best Places to Work in New Jersey. 

“Cranbury has been a wonderful home for the company,” said Patterson. “A tremendous number of our employees are based in the Cranbury and Princeton areas. It’s a great location, close to all the highways and central to lots of the places we need to go around the state—New York, Washington, D.C. and Philadelphia—so our intention is to stay in the neighborhood and grow.” 

Patterson said his goal going into 2012 is to continue to hire and conduct good research that will result in new treatments. The company develops orally administered, small molecule drugs called pharmacological chaperones. There are no products approved yet, but Amicus is in late stage development for a treatment for Fabry Disease and has filed for approval with the FDA. It hopes to have an approved product in 2013 that will make money and help fund other programs.

Amicus Therapeutics became a publicly-traded company in 2007, but for now most of the company’s research is funded by venture capital and some grant money from patient and scientific research organizations.

Patterson said he goes to work every day motivated to ease the suffering of patients with rare diseases and to help their families.

“It’s a combination of knowing I’m working on something that could help people who could use it, the opportunity to work on innovative science and to work with a set of individuals at this company who are passionate, smart, energetic and want to make a difference," he said.