The Cure for Biotechnology (Mis)adventures
I recently read The Cure, Geeta Anand’s account of John Crowley’s effort to develop a treatment for the rare debilitating disease affecting his children. The story was the basis for the 2010 movie Extraordinary Measures.
Crowley’s children Megan and Patrick were diagnosed with Pompe disease, a genetic metabolic disorder that causes muscle weakening, heart enlargement, and severe respiratory difficulties. Both children are ventilator dependent by the age of two. In Pompe disease, alpha-glucosidase, an enzyme that breaks down glycogen is deficient or absent. Glycogen is a complex carbohydrate molecule that stores energy for use in the body. Alpha glucosidase is found in the lysosomes of cells. Lysosomes are the “garbage bins” of cells, primarily responsible for processing cellular waste. Anyway, since the enzyme is deficient, glycogen builds up in cells and causes muscle weakening.
Megan and Patrick have a life expectancy of about five years. Crowley, desperate to save his kids, begins meeting with Pompe researchers. He eventually becomes CEO of Novazyme, a company formed to manufacture the missing enzyme. He eventually negotiates the sale of Novazyme to Genzyme and continues to work with the Pompe team to get the enzyme in clinical trials.
Crowley is stubborn, charismatic, and absolutely determined to get his kids in clinical trials before they succumb to the disease. I’d like to address a couple issues I had with his behavior in the book.
1. Indiscriminate research grants. Crowley’s initial tactic is to form a nonprofit, the Children’s Pompe Foundation, and raise research funds. The foundation impressively raises $750,000 in eight months. However, he makes all the decisions about awarding grant money himself, and solely based on what the researchers say about their expediency to be approved for clinical trials. Crowley, a director of marketing at Bristol-Myers Squibb, acknowledged science is not his strong suit. I certainly didn’t expect Crowley to retrain as a biochemist, but he should have had a scientific advisor in the Foundation to assess research feasibility. The Komen Foundation and the Tourette’s Syndrome Association award research grants, and both have scientific advisory boards.
Granted, the field of Pompe researchers is small, but his ignorance about the research specifics cause problems for Crowley when he becomes CEO of Novazyme. He takes the job fully believing the company is his best chance into getting a Pompe treatment into clinical trials. At a meeting with venture capitalists, the potential investors point out the planned manufacturing process would never be approved by the FDA-something Crowley didn’t realize.
2. Conflict of Interest. In order to motivate the Novazyme staff, Crowley brings in Pompe patients and hosts lunchtime seminars on the disease. A number of venture capitalists question Novazyme’s ability to practice good science since Crowley is far from objective in the outcome of Novazyme’s drug development. Being in the senior management of Novazyme (and later Genzyme) actually is detrimental in getting Megan and Patrick in clinical trials, and I have mixed feelings on Crowley getting involved as intimately as he does getting his children treated. He wasn’t well qualified to be a CEO and had a steep learning curve. Taking the Novazyme job undoubtedly gave him a sense of control. I did like the lunchtime seminars. I think it gives a sense of urgency to the research being done. It’s easy to focus on the small details of a project on a day to day basis-this technique isn’t working, the cells are contaminated-that it’s easy to forget the end goal, and that people’s lives are depending on what you are able to accomplish.
Overall, I enjoyed the book. Anand tells the story well, and it took large amounts of willpower to not skip to the end and find out if Megan and Patrick were treated. I also learned a lot about venture capital investment!
Although it’s a couple days since Solstice and the start of Haunakkah, I hope you all have a great holiday!