“Biotechnology is a big word with a simple meaning--hope.” John Crowley, CALBIO 2012 Keynote Speaker, CEO of Amicus Therapeutics, Father of two children with Pompe disease
The biotechnology industry is in trouble. And one place industry leaders are finding inspiration is in reconnecting with patients.
“If it’s right for patients, it’s almost always right for your business,” stated George Scangos, CEO of Biogen Idec in his opening keynote speech to the crowd at CALBIO 2012. The conference, produced for the first time by California’s two biggest industry organizations, BayBio and BIOCOM, focused on the theme that the needs of patients must be the driving force for the industry.
The John Crowley Story
Few can tell a more remarkable story about patient driven medicine than John Crowley, a keynote speaker at this week’s conference.
In 1998, two of Crowley’s children were diagnosed with Pompe Disease, a fatal disease. At the time John Crowley was just 31 and consulting for financial institutions. Borrowing $100,000 on his home and 401(k) plan, he started his own company, Novazyme, devoted to finding a cure for his two children. And he found success. Novazyme developed an enzyme that showed promise, and Mr. Crowley sold the company to Genzyme for $137.5 million, one of the heftiest sums ever for a product untested in humans.
As it turned out, getting the product tested on his own kids wasn’t as easy as you might think for the entrepreneur and developer of the cure. Genzyme’s CEO, Henri Termeer, thought a father’s love would prove a powerful catalyst for the program, and Crowley was made a VP over the Pompe program. Still it took several years to finally bring the drug to his two failing children.
“It ripped me apart,” Crowley is quoted in a WSJ article in 2003. “Many times, I’d be talking aloud about programs and budgets, and at the back of my mind be thinking, ‘Oh my God, this is not good for Megan and Patrick.”
A powerful story for the role of patients to drive innovation, Crowley eventually was able to have the two children enrolled in a trial, and the enzyme he’d developed saved his kids from certain death.
“Hard not to get teary eyes hearing John Crowley talking about his kids at #calbio2012,” tweeted SF Business Journal’s Ron Leuty, reflecting the mood in the room packed with about 1,000 attendees.
The Crowley story is one about the depth of a father’s love, but also about success in the bio industry. (The story also made it to Hollywood in a CBS movie, Extraordinary Measures, with Harrison Ford acting and producing.)
When Drug Research Is Personal
Not everyone’s story is as dramatic as Crowley’s. But the theme of drug research being powered by patients and relatives of patients is an ongoing and strong theme.
On hand for lunch the second day was Representative Brian Bilbray, a U.S. Congressman representing California’s 50th district that includes some of San Diego. In a Q & A session with BIO’s CEO, James Greenwood, Rep. Bilbray talked about the impact of his being father of a 25 year old daughter recently diagnosed with melanoma. “It does affect me and my work,” he said. “Brianna (his daughter) is becoming the patient advocate in Congress. We changed regulations during the AIDS crisis and we can do it again.”
Melinda Cathbert is an entrepreneur, student, and mother of a son with autism. She volunteered at the conference, and I caught up with her after a panel. She offered some insight into why patients or relatives of patients can lead business to success.
Melinda is the founder of a biomarker company focused on autism, SmartDx. Melinda said that as a mother of an affected child she “had a unique perspective. There’s so many unintended consequences that come up [in business] that you have to have extra insight.” Melinda feels that watching a disease at close range gives just that insight.
Melinda is a driven woman. She showed me an app she has developed (still in alpha phase) which will help consumers make healthy changes to their daily habits. I suggested she go to Steve Burrill or another VC firm. It wasn’t but a few hours, and I saw her with Mr. Burrill showing off her app.
“Many folks say, I’m not going to get my career bogged down with a problem when a drug doesn’t work out, I’m moving on. But you have to hang in there long enough to solve the big issues. I have a twenty year commitment to the field of autism. I’m not going to jump ship.”
John Crowley didn’t jump ship, and it saved his kids lives.
Patient Advocacy and Disease Foundations
A major component of the conference was the presentations and panels populated by members of various disease foundations, including The Michael J. Fox Foundation, The Leukemia and Lymphoma Society, Susan G. Komen For The Cure, and others. More and more these foundations are become major contributors to R & D research.
The Myelin Repair Foundation has supported the discovery of 24 new research tools to advance research for potential multiple sclerosis therapeutics. The Melanoma Research Alliance awarded over $30 million to 73 research programs.
Disease foundations are supported by large patient networks. Like relatives of patients, they could be said to have a longer term commitment to a disease than say a big pharma or biotech. In addition, the foundations can help promote reform at the FDA on behalf of patients.
Dr. Robert Fisher is an advisory board member for The Epilepsy Therapy Project at Stanford University. In a panel on the role of patients in drug discovery, Dr. Fisher pointed to a growing trend of more sensitivity to the patients on the part of drug companies.
“It used to be as soon as the trial was over, it’s bye bye to the patient,” said Dr. Fisher. Even though the medication was helping the patient, drug companies would tell them “check back in three or four years.” Now Dr. Fisher says that for an academic research institute such as Stanford to sign off on the IRB, there must be more commitment to the patient. In addition, Dr. Fisher pointed out that more and more patients are now included on FDA panels. “Regulators listen more to patients than doctors,” he asserted.
This year is the first time BayBio and BIOCOM have come together for their annual conference. Together, they represent by far the largest concentration of biotechnology on the planet. CALBIO is the result of the pooled resources of the two large industry organizations and delivered a concentrated message. This year that message was simple.
Biotechnology equals hope.
“It reminded us why we all do what we do,” Gail Maderis, CEO of BayBio, said to me on the way out. Gail herself was diagnosed five years ago with MS.