The Cure
Have you ever imagined the life of kids with incurable diseases? If you were their parents, what is your life like at daily basis, and what would and could you do for your family? I barely know the life disabled people lead though I have seen millions of them, not to mention kids with incurable genetic diseases. The book “The Cure” gave me a good lesson.
“The Cure” illustrated the life of Crowley family. Along the mainstream of treating two kids, Megan and Patrick, who suffered from Pompe diseases, the book described the persistent fight of their father, John, who was a graduate from Harvard MBA program, a savvy fund raisers and CEO in a biotech company, for getting them treated in clinical trials; their mother, Aileen, who has been always caring and dedicating herself to her husband and kids, and their extended families who have been supportive and helpful.
Pompe disease is a rare progressive genetic disorder happening in infints, kids and adults. It is believed that the etiology of this disease is the lack of the enzyme in lysosome for degrading glycogen. Thus, while glycogen accumulated abnormally rather than being consumed as energy, the muscles will become very weak. The heart of patients will enlarge; the patients are able to neither walk nor run, and eventually confined to wheelchair. In some cases as described in the book, the only parts which can move in a patient are eye lid, thumb and mouth. They are expressionless and unable to speak clearly. Most of infant patients die before age 2. Alive patients usually lose respiratory function and can only breathe with the help of tracheotomy and ventilator. Since it is a genetic disorder, it could affect more than one member in a family, which is not uncommon in the book.
Megan was the first to be diagnosed as Pompe disease in Crowley family. During her hospital admission due to pneumonia, her little brother Patrick was also diagnosed. Mishap caught this family without any warnings. Since then, John persistently started his journey to fight for their treatment. He worked extremely hard for his kids and other Pompe patients and won opportunities for other kids. But his kids had been ruled out of clinical trials once and once again, either because of “conflict of interest” or they were older than the optimal treatment age suggested by clinical trial protocols. The hardships inspired and motivated John rather than hindering him. Eventually both Megan and Patrick were put in a clinical trial with the help of family, friends, scientists, physicians, and investors. John symbolized American spirits --- independence, hard working, optimism and belief in happy ending.
Needless to say, happy ending is not easy to reach. It usually follows the painful processes. In a family with two Pompe kids, the life was a mess, especially at the very beginning. Having patients at home could change everything such as family relationship and dynamics. Both John and Aileen suffered from physical exhaustion, mental struggle and financial burden. John, although positive, sometimes felt to quit, or at least run away from this miserable life, when he could barely see any hope. He even thought about divorcing Aileen since he believed she put all burden on him and she was not helpful. Relationship between John and extended family members changed too. John argued with his mother who always tried to assist. John fired family nurses one after another when he was at bad temper since he thought nurses were either lazy or incompetent. This actually was one of the biggest problems between John and Aileen, since Aileen desperately needed helpers. On the other hand, everybody loved the kids and tried to do the right things for them, which also bonded the family together. For instance, Aileen managed to take kids to Disney Land with the help of some volunteers and John’s colleagues. Grandparents accommodated John’s family when they desperately need help and played the role of temporary caregivers for the kids. Fortunately, the family dynamics and relationships, as well as the relationships between John and his colleagues and investors adapted to the direction of doing well to the sick kids, which led to a relatively happy ending, though not surprisingly, no one enjoyed the painstaking process.
Another reason the book is very intriguing is it portrayed some scientists, physician-scientists, as well as investors. Scientists are usually incompetent to raise money and translate their scientific discoveries into practical application. Investors, on the other hand, focus mainly on cost and profit. Sometimes neither of the two groups put patients on top of everything and meets their demand in a flexible way. John stood right in the core of science, money, and patients. One of the most impressive contributions of John was Lunch N Learn, i.e scientists and investors met with Pompe patients during lunch and listened to patients’ stories. The strong hope of being treated and leading a normal life, the devout belief in science motivated both scientists and investors to work harder and work together. Scientific research, a marathon rather than a sprint, needs to marry money in order to create invaluable therapies for patients.
In addition to John’s fighting, the graceful resilience of the patients and their families are also very inspiring. Megan, the little girl, never ever treated herself as a disable. She was very out- going and full of hopes to her future. Another girl patient in the book finished high school and college, and prepared for law school. If those sick kids can smile to their uncertain life with dreams, why can’t healthy ones?
While I was reading, I couldn’t resist thinking that John’s family was relatively rich, belonging to upper middle class. And the experience of studying in Harvard Business School has established him an invaluable network. The first fund he raised for the Children’s Pompe Fundation was through his Harvard classmates. What if his family was poor and he had not been one of Harvard alumni? What would he do? Could he do the same? Needless to say, economic status and useful network always play critical roles in decision making and direction of future life. People can be categorized based on their socioeconomic background. Poor people definitely perceive the world differently from those well-to-do. Fortunately, people are always ready to help, and meanwhile advocate more attention for those who are suffering.
P.S. A newly released movie "Extradinary Measures" is based on this story.
