This story is from BostonGlobe.com, the only place for complete digital access to the Globe.
One of the company’s first successful drugswas developed based on work spurred by a father looking to cure his children of Pompe disease—a story chronicled in the Harrison Ford movie, “Extraordinary Measures,’’ based on a book by Wall Street Journal reporter and former Globe staffer Geeta Anand.
“Being patient-centric isn’t just talking about patients,’’ said Jamie Ring, the company’s senior director of global patient advocacy. “It’s a company’s willingness to be challenged by patient communities [and when issues arise] being willing to change course.’’
Patients and their advocacy groups also played a large role in the development of a highly effective treatment for a rare form of cystic fibrosis by Vertex Pharmaceuticals of Cambridge. The nonprofit Cystic Fibrosis Foundation provided the company with millions of dollars—much of it raised by Boston businessmanJoe O’Donnell, whose son died of cystic fibrosis—to help finance research, development, and clinical trials.
The drug, Kalydeco, was approved by the federal Food and Drug Administration this year.
The Michael J. Fox Foundation is following a similar path, providing money to biopharmaceutical companies to accelerate the development of new drugs. The foundation has benefited from the high profile of its founder, television and movie star Michael J. Fox, who has Parkinson’s, said Todd Sherer, the foundation’s chief executive.
“Michael’s profile was really important in getting attention to Parkinson’s, not only drawing research funding, but researchers,’’ Sherer said.
Patients and their families are becoming not only partners with drug companies, but also political activists. Breast cancer survivor Lorraine Heidke-McCartin and her husband Phil McCartin, for example, lead a group of activists pushing federal regulators to speed up the approval process for experimental cancer treatments.
On Tuesday, the McCartins organized a demonstration that drew about two dozen people outside the BIO convention at the Boston Convention & Exhibition Center to protest FDA policies that they say keep cancer victims from new drugs that could save their lives. The biotech and pharmaceutical industries have also been critical of how long it can take to approve new drugs.
“We want flexible treatment and informed choice,’’ said McCartin, whose wife was diagnosed with an aggressive form of breast cancer in 2006.
An FDA spokeswoman said the agency has sped up its drug approval process in the last several years, while still ensuring the drugs are safe.
Still, even at its fastest, drug development can take years. Martin and Taylor, whose sons have hemophilia, launched their firm, Inspiration Biopharmaceuticals, in 2006, but haven’t yet brought a drug to market.
The firm, which employs about 60, hopes to have its first product on the market this year or next, and is conducting clinical trials on a second treatment.
Taylor laughed a bit as he recalled his journey to this point: learning about his son’s condition, making friends and acquaintances in the hemophilia research world, and finally partnering with the Ipsen Group of France to pursue the drug treatments the company is developing.
And how does his son feel about his dad’s endeavor?
“My son [still] can’t believe it, of course,’’ Taylor said.