‘The Cure’ is centered around a father’s refusal to give up hope in his children. John and Aileen Crowley are hit with the worst news of their lives after two of their three children are diagnosed with Pompe disease, a rare, untreatable, and fatal genetic disease which attacks the muscles. The condition gradually degenerates muscles until the victim cannot sit or breathe. The condition also enlarges the heart to a dangerous degree, which eventually causes death. With sheer determination to save his children, John Crawley starts up a biotech company to develop a cure for the disease, to protect his children. The disease strikes about 10,000 people annually, and since there is no known cure, pharmaceutical companies lack the financial incentive to develop a treatment. However, John refuses to accept the situation and sets his mind to push through with his plans.
When John discovers that no drug company is willing to commit to finding the cure, he immerses himself in the quest to save his children, using everything he had. As his finances shrink, John approaches venture companies to fund his research. Despite the rejection of these venture capitalists, John refuses to give up. At last, a venture capitalist called Catalyst invests 3 million in it. After John’s company proves to have great potential, Genzyme, a Cambridge based capitalist firm, acquires John’s company, pays $ 137.5 million, and makes John a senior vice president at Genzyme. In his position, John continues to push hard for Pompe’s research. He also ensures that his two children are included in clinical trials of developed drugs. Executives within Genzyme were under the impression that John has an apparent conflict of interest since he bends corporate strategy based on his desire to save his children, this leads to his children being rejected more than once, for the clinical study. However, the children finally receive the treatment that their father helped to create and live beyond their fifth birthday (Anand, 2006).
Genetic and clinical aspects of Pompe Disease
Pompe Disease is a rare genetic disorder inherited as a recessive trait. It is caused by a mutation in a gene that makes an enzyme GAA (acid alpha-glucosidase) (Kohler et al., 2018). This mutation restricts the body’s ability to produce enough protein to break down glycogen for energy. Sugar builds up in the body, causing fatal damage to the organs (liver, heart, and lungs) and muscles. The disease also causes trouble breathing and weakness due to degenerated muscles.Pompe disorder is estimated to affect one child in every 40,000 births, according to Byrne et al. (2019).
Impacts of Pompe disorder on the family structure
Generally, the Pompe disease puts a significant strain on a family’s wellbeing in several aspects. The affected member spends a substantial amount of time in the hospital, which requires support from other family members. Thus, couples spend most of their time providing care, whether at home or in the hospital, and less time on their marriage. Since all the attention is focused on the patient, parents may spend less time with other children, which may affect them psychologically. The Pompe disease also puts a strain on the family’s finances, since most of the money is directed towards providing health care for the affected member.
An orphan drug is a medicinal product intended for the prevention, diagnosis, and treatment of rare, life-threatening disorders. This drug is termed ‘orphan’ because, under normal market conditions, pharmaceutical companies have little interest or commitment in developing and marketing medicines intended for the treatment of a minimal number of patients (Sharma et al., 2010). Thus, pharmaceutical companies avoid developing orphan drugs, as expected sales of the medicine cannot recover the high cost of establishing the product in the market. Consequently, the potential market for an orphan drug is tiny, and drug companies would incur an enormous financial loss. In the book, John had to start his own drug company, since no pharmaceutical firm was willing to develop an orphan drug for Pompe Disease.
Moreover, most venture capitalists turned John away, since they saw no profitability in investing in his company
Roles of pharmaceutical companies and FDA in drug development
The primary function of pharmaceutical companies is to discover and develop drugs that are marketed for use as a medication to prevent, cure, or alleviate the symptoms of a disease. Pharmaceutical corporations, therefore, create value that improves the wellbeing of patients. On the other hand, the FDA (Food and Drug Association)is a regulating organization that protects public health. The FDA accomplishes its function by ensuring that pharmaceutical companies prove the efficacy and safety of a developed drug (Darrow et al., 2020). The FDA also makes sure that drug companies manufacture and market medicinal products appropriately. Thus, the FDA is a critical consumer watchdog for pharmaceutical corporations, which guarantees consumers that developed drugs are effective and safe for their intended use.
Phases of drug development
Several mandatory processes grope the pharmaceutical industry before the sale of a drug can commence on the market. The FDA outlines five comprehensive stages of drug development (Dugger et al., 2020). The first is discovery and development. Here, drug companies conduct substantial research and scientific studies on a particular disease while spending millions of dollars. They obtain funds from grants, own revenues, and even governments. The second phase is preclinical research, which is a preliminary stage that involves testing drugs on animals. Safety testing is also carried out to determine the effects of the drug. The third stage is clinical research, which is one of the most critical phases of drug development. After the drug is cleared from preclinical trials, it goes on to be tested on humans in the clinical trial. The FDA regulates specific standards for clinical trials such as scientific testing, selection criteria of humans being tested as well as the environment in which the test is being done. The fourth phase is the FDA review, where pharmaceuticals submit fully documented findings and conclusions of clinical research to the FDA for review. If the FDA accepts the submission, a response is provided within 6 to 10 months. The last phase is the PDA post-market safety monitoring. Here, the FDA closely monitors the advertisement of the drug for accuracy. The FDA also monitors problems and complaints associated with the drug. The FDA, therefore, has the power to offer warnings and restrict drug sales. Overall, the FDA conducts routine manufacturing inspections to ensure safety standards are upheld (Darrow et al., 2020).
Once a reader gets hold of the boo, it is hard to put it down. However, one can get frustrated and even disappointed in some of the impulsive and unwise approaches that John takes in the quest to save his children. Nonetheless, his determination to find a cure and the unwillingness to accept the fate brought by the genetic disease is genuinely admirable. The book points out the fierce competition and tension between pharmaceutical companies, physicians, research scientists, healthcare providers, and parents. John refuses to sit back and wait for the system to work in favor of his children. He disregards the wait on research processes and approval of the FDA. The reader also gets to comprehend that only someone with John’s connections and position could have made the moves to raise finances for research possible, which is unlikely for most people.
Anand, G. (2006). The Cure: How a Father Raised $100 Million–and Bucked the Medical Establishment–in a Quest to Save His Children. Harper Collins.
Kohler, L., Puertollano, R., & Raben, N. (2018). Pompe disease: from basic science to therapy. Neurotherapeutics, 15(4), 928-942.
Byrne, B. J., Fuller, D. D., Smith, B. K., Clement, N., Coleman, K., Cleaver, B., … & Corti, M. (2019). Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy. Annals of Translational Medicine, 7(13).
Sharma, A., Jacob, A., Tandon, M., & Kumar, D. (2010). Orphan drug: Development trends and strategies. Journal of Pharmacy and Bioallied Sciences, 2(4), 290.
Darrow, J. J., Avorn, J., & Kesselheim, A. S. (2020). FDA Approval and Regulation of Pharmaceuticals, 1983-2018. JAMA, 323(2), 164-176.
Dugger, S. A., Platt, A., & Goldstein, D. B. (2018). Drug Development in the era of precision medicine. Nature reviews Drug discovery, 17(3), 183.
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