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John F. Crowley, President & CEO of the Biotechnology Innovation Organization (BIO), highlighted the crucial themes of “innovation, access, and advocacy” during his keynote speech at the June 25 American Cancer Society Cancer Action Network (ACS CAN) 17th Annual Research & Health Equity Breakfast in Boston. He drew from his personal journey, sharing his family’s struggle with Pompe disease—a rare condition that lacked treatment when his daughter and later his son were diagnosed. His story of transforming heartbreak into hope exemplified the spirit of determination that drives advancements in cancer treatment and biomedical research.
‘Settle on determination’
Crowley shared his own family’s struggle against rare disease as he spoke of “delivering hope” to patients with cancer.
He recalled the time more than two decades ago when he learned of his daughter’s diagnosis with Pompe—a condition with no treatment at the time. Crowley’s son would soon be diagnosed, as well.
“You go through the shock, the grief, the denial, the anger. And settle on determination,” he said.
The news was delivered by “a very caring neurologist with a social worker. He described the diagnosis, and he ended with. ‘I’m sorry, but there’s nothing we can do,’ ” Crowley recalled. “So that is what launched us into this whole world of biomedical research. Ultimately, patient advocacy and hope.”
Crowley started a small biotech and ended up working with Henry Tamir at Genzyme in Boston. Their Pompe disease division eventually produced a treatment.
“In January of 2003, by the time they had become quite weak and quite sick, on ventilators and in wheelchairs, our Megan and our Patrick were patients 27 and 28 in a clinical study on a lifelong, every-other-week, enzyme replacement therapy,” Crowley said. “By that point, the left side of their heart was three times enlarged, and it was the most life-threatening aspect of their disease. Within six infusions, their hearts went back down to normal size. Their function became normal—and it saved their lives.”
Innovation
“When we talk about innovation, we describe it as a virtuous circle and you put people living with these diseases—call them patients, whatever you will—put them at the center and think about what it takes to make newer and better medicines,” said Crowley.
“It takes oftentimes our great universities, it takes the NIH; it takes entrepreneurs; it takes the ability to take an enormous amount of risk with money and with careers, with reputation, with lives—ultimately takes larger companies; takes the FDA; takes an entire international system; manufacturers, consultants, bankers, advisors, all of that,” he continued.
“Even then, it’s one of the rarest things we can do in any industry: To go from an idea to a molecule, to a medicine that works as safe and effective in clinical studies and to ultimately get it approved,” he noted.
Access
Access “really means identifying and breaking down barriers, understanding why people don’t have access to these medicines,” Crowley said. “Is it awareness? Is it health equity? Look at the enormous disparities and outcomes of people in communities of color and underprivileged areas.”
Crowley praised work to eliminate barriers by another speaker at the ACS CAN breakfast, Massachusetts State Rep. Meghan Kilcoyne (D-Worcester). Her proposed legislation would increase coverage for biomarker testing.
Biomarkers are making a difference in controlling cancers, as well as rare conditions like Pompe disease.
When his daughter Megan was born, nobody knew she had Pompe disease. She wouldn’t be diagnosed for more than a year, said Crowley. Today, however, Pompe is diagnosed right away with a biomarker.
Biomarkers are important in cancer diagnosis and treatment, as well: “So many cancers, with early intervention, can be effectively and highly successfully treated.”
But access is critically important.
BIO will be looking at insurance and specifically “why in the world do we have co-pays for medicines for cancer,” Crowley said. “Let’s take a hard look at some of the economics that may be preventing people from getting access.”
Advocacy
Advocacy involves “people being their own champions and bringing together an entire community,” Crowley said.
He recalled his time at Novazyme and Genzyme—”the first companies to have departments of patient advocacy and have full-time employees who work with individuals, with families, with foundations, with communities, not for profits.” Those employees “were the conscience of the organization, but they also taught each of us in a very, very passionate and very genuine way to live this mission.”
Those working in biotech need “to have that patient mindset,” to understand the importance of the work that’s being done.
“I would ask everybody—everybody in our organization, everybody in our industry, and each of you—to think when you are going through the rigors of drug development that’s so darn hard,” Crowley said. “I’d ask you to think if you had that disease, if you had that cancer. Or you’re the mom or a dad of a child with that disease. What would you do?”
“In cancer and so many other disorders, the words that I described earlier—when the doctor said, ‘I’m sorry; there’s nothing we can do’ —that’s just not acceptable anymore,” Crowley said.
