The Book of the Week is “The Cure” by Geeta Anand, published in 2008. This ebook tells the emotional, suspenseful story of how a family coped with three disabled children, two of whom were suffering from a genetic disease for which a cure is yet to be found.
In the late 1990′s, John Crowley’s daughter and son were both diagnosed with Pompe disease, a muscle disorder. Patients, with varying severity, “have imperfectly produced acid alpha-glucosidase enzyme” which results in paralysis, obstructed breathing, and, if left untreated, death before the age of five.
Even though Crowley possessed the personality, talents, skills, education and privileged background that one would think would allow him access to a life-saving enzyme to save his children, he had to face numerous obstacles. The father naturally fell into the role of entrepreneur to do so. His wife provided invaluable emotional support and around-the-clock care of the children with the help of nurses; not to mention the running of the household.
Nevertheless, lots of genetic and environmental luck determines whether patients become fully cured and/or whether the quality of their lives improves significantly, or whether they die– even when they are sufficiently fortunate to take part in a trial of a new life-saving medicine. Death would be inevitable without the medicine.
Every patient is different. There are many different criteria the U.S. Food and Drug Administration considers when deciding whether to approve particular medical products for sale. Money plays a major part in whether a new product ever sees the light of day. A young medical research company raises funds through venture capitalists, and because the whole operation carries extremely high risks, if the company achieves success– the rewards, fittingly, are also extremely high.
Scientists must do years of preclinical testing on animals to make sure a new medicine works sufficiently well before even considering administering it to humans. In the United States, possible deadly consequences and possible future litigation motivate the scientists to act with integrity by performing tightly controlled experiments, so as not to have to fudge research results.
Another aspect of drug development, is avoiding a conflict of interest such as that in Crowley’s situation. He played a pivotal role in the race to bring a medicine to market; it appeared he was doing it to get the medicine for his own children.
The estimated annual expense of the enzyme for each child was $200,000, and $1 million for all future annual medical expenses, including the enzyme, plus wheelchairs, nurses, ventilators, catheters, etc.
Read the book to learn of the Crowley family’s experiences with American biotechnology.