While there is a story in everybody’s life, only a few have an extraordinary story to share with the world like the Crowleys. John and Aileen Crowley’s story is filled with pain and sorrow, like any other parents who have children suffering from terminally ill diseases. However, the element that sets their story apart is their ability to not lose hope and their determination to take on the disease. Extraordinary measures is a movie, which is based on the real life experiences of John and Aileen Crowley, and it delineates to us how, rather than accepting the inevitable, John Crowley embarked on a race against time to find a cure for the disease that was slowly killing his children.
‘Extraordinary measures’ is a 2010 film, starring Brendan Fraser, Keri Russell and Harrison Ford, which portrays the story about a couple who establish a biotechnology company to find a cure for their children suffering from Pompe disease. The disease is a rare genetic disorder that usually causes the death of the child before his/her tenth birthday. John and Ailey, parents of 3 children, try to save their 2 younger children, Megan and Patrick, from the clutches of this disease.
John learns that Dr. Robert Stonehill has done some useful research on this disease, at the University of Nebraska. He contacts Dr. Stonehill and finds that he is unable to make progress in his research due to lack of funding. So, John resigns his job and works with venture capitalists and other pharmaceutical companies to fund Dr. Stonehill’s research. The profit motives of their financial partners and Dr. Stonehill’s rather difficult personality puts up many challenges to the efforts of John.
However, in the end John is able to arrange for a sibling medical trial, so that his children can be tested with the new drug developed by Dr. Stonehill. The children are administered the drug and they began to show improvements. John’s efforts pay off and the drug invented is working. The movie ends with Megan and Patrick showing signs of recovery, and Dr. Stonehill cashing in his check to start his own research facility.
Pompe disease is also known by names such as acid maltase deficiency (AMD) or glycogen storage disease type II (GSDII). It is caused due to the deficiency of acid-α-glucosidase (GAA). The disease causes lysosomal glycogen to accumulate in tissues such as skeletal and cardiac muscles thus, leading to progressive debilitation, organ failure and death. Symptoms of this disease include progressive skeletal muscle dysfunction. As the muscle weakness worsens, patients are usually confined to wheelchair and they would require assisted ventilation. Until recently, there was no specific treatment for Pompe disease.
Recombinant human enzyme alpha-glucosidase (myozyme), an orphan drug, is now used for treatment of Pompe disease. This enzyme replaces the rhGAA and facilitates normal muscle development and functions. Myozyme, used for the treatment of Pompe disease was granted Orphan drug status by the FDA in 2006. An orphan drug is a drug used to treat diseases or conditions that affect fewer than 200,000 people in the U.S. The process from the discovery to marketing of drugs to treat rare diseases is very long and expensive, and they are not very profitable, since only a small number of patients are going to benefit out of it. Thus, the US and EU has introduced the concept of orphan drugs, to encourage companies to invest in the research and marketing of drugs for rare diseases.
The movie also brings into fore the struggles faced by John because of his inexperience in the medical world, particularly with respect to the rules of the FDA pertaining to drug approvals. The film highlights how drug discovery and marketing is a long and complex process, and how while a researcher may be strong in theory, still he cannot get an approval until he has expert guidance. FDA takes extreme caution to ensure the safety and efficacy of drugs being patented. The movie clearly outlines the hard-to- swallow fiscal issues and regulatory issues involved in a new drug development.
There is a dialogue by John in the movie, which clearly communicates the suffering of the parents whose two young children are fighting for their life. He says he could either watch his children slowly die, or he could try to fight it. Naturally, every parent would want to select the second option but few have the courage and determination to pursue it. The ever-changing nurses whose names Aileen keep forgetting, the well-meaning doctors saying that it is better that the child dies early before she suffers more, and Aileen arguing with John how they would pay the health insurance if he jeopardizes his job in search of the cure – all these scenes portray the sorrow and pain of the family realistically.
In conclusion, it may be said that Extraordinary measures is a movie which describes both the anguish of the families during their struggle with an incurable disease and also the efforts and struggle involved in developing a drug for a rare disease. It also gives an account of the day to day problems a parent has to endure, like insurance payments, arranging for nurse, and most of all understanding the nature of the disease, in their effort to give the best possible treatment for their children. Above all else, the film is a tribute to a father who fought against all odds, raced against time, denied to accept that there was no escape for his children and risked everything to find a way to buy some time for his children to live.
FDA. FDA NEWS RELEASE. 28 April 2006. Web. <http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2006/ucm108645.htm>. 20 June 2015.
McGovern, Margaret M. Genetics of Glycogen-Storage Disease Type II (Pompe Disease) Medication. 15 August 2014. Web. <http://emedicine.medscape.com/article/947870-medication#2>. 20 June 2015.
National Center for Biotechnology Information. Pompe disease diagnosis and management guideline . May 2006. 20 June 2015. <Web. <http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3110959/>>.
June 21, 2015
“We’ve learned more about life and love from our two kids than we’ve ever taught them.”These were the words uttered by John Crowley, while talking about his astonishing quest to find a cure for the Pompe disease that threatened to cut short the life of his children. Based on his life, CBS Films produced a movie titled ‘Extraordinary measures’ with Brendan Fraser and Harrison Ford in the lead. The film explicates, among other things, the astounding resolve of the Crowleys and how the love of these parents had to compete with two other powerful forces – a scientist’s quest for knowledge and recognition and a corporation’s drive for profits.
‘Extraordinary measures’ narrates the story of a father’s struggle to find a cure for Pompe disease that was threatening the life of his children. The movie starts by showing the viewers how Megan and Patrick, children of John Crowley, are suffering from a disease which causes their internal organs to enlarge. The disease would eventually cause the death of the children before they reach age 9 and there is no existing cure for the disease. John, an advertising executive, finds out that Dr. Robert Stonehill has done some path-breaking research in this area and approaches him to seek his help.
However, he finds out that Dr. Stonehill is struggling because of lack of funding for his research. John volunteers to raise fund for the research and later meets with many potential investors. However, the road is not smooth as Dr. Stonehill has a thorny personality and they meet with many financial and operational difficulties. After facing many losses, they sell off the company to the competitor, Zymagen, whose profit motive sometimes frustrates John. John takes the initiative to combine with Dr. Stonehill’s research 3 other work done by other researchers.
When the research reaches the trial stage, the company prefers to test the drug on infant, much to the disappointment of John who wanted to use the drug on his children. After much hardship and verbal and emotional battles, John convinces the executives to administer the trial drug to Megan and Patrick. The drug has a positive effect on them and the film ends on a positive note.
The disease that has been extensively dealt with in the film is a rare genetical disorder called Pompe disease. According to the National Institute of Health, “Pompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells.” The excessive amassing of glycogen severely impairs normal body functions. Pompe disease is commonly classified into three types - classic infantile-onset, non-classic infantile-onset, and late-onset. About 1 in 40,000 people in the US are affected by Pompe disease. Mutations in the GAA gene is the cause behind this disease, and the symptoms include weakening of muscle tissues and organ damages.
The treatment for Pompe disease is ERT or Enzyme replacement therapy. Under this treatment, the deficient Lysosomal enzyme acid alpha-glucosidase is replaced by a genetically engineered form of the GAA enzyme. The drug alglucosidase alfa in the form of Myozyme was recognized as an orphan drug by the FDA in 2006.Orphan drug is the terminology given to those drugs that are used to treat rare diseases. The pharmaceutical companies will be reluctant to invest in the development of such medicines because they are rarely profitable as they benefit only a small group of patients. So, to encourage the development of drugs which might otherwise lack sufficient profit motive FDA has assigned the orphan status on such drugs, which would ensure that the company has tax incentives and better patent protection and marketing rights.
According to Parvis, currently the process of drug development and approval takes an average of 14.2 years. And this delay is mostly due to the extreme caution exercised by FDA to ensure the safety of newly developed drugs. Though both researchers and FDA know that no medicine is risk free, FDA does not grant approval to any medicine without first clearly establishing that the benefit of the drug outweigh its risks. The film clearly explains this aspect involved in a drug development, particularly through Dr.Stonehill’s character.
Besides talking about the disease and its cure, the movie also heart wrenchingly portrays a family’s suffering and anguish. Instead of sadly highlighting the suffering of the family, the movie presents it using day to day realities and humor. Megan chasing her brother John in her wheelchair, wishing the medicine was in pink color and other such light hearted scenes subtly conveys the suffering of the patients without over dramatizing it. The movie also touches subjects like health insurance, medical care, profit motives of pharmaceutical companies, researcher’s need for recognition etc.
In conclusion, the movie accurately reproduces the plight of people inflicted with rare diseases and the amount of effort put in developing a new drug. Besides delineating the suffering endured by the Crowley family and their determination and resolve, the film also touches upon some uncomfortable truths about drug development and marketing. Drug companies mostly are interested in marketing what is commonly known as ‘blockbuster drugs,’ which are used by large number of people and hence yield high profits, like diabetic medicines. Drug development for rare diseases is an often neglected area, and the film is a timely reminder that more efforts should be taken in this direction by the concerned authorities, to encourage pharmaceutical companies to invest in treatment of rare diseases.
Davies, Kevin. John Crowley Relives His Extraordinary Measures to Combat Pompe Disease. 3 February 2010. Web. <http://www.bio-itworld.com/news/02/03/10/john-crowley-extraordinary-measures-pompe-disease.html>. 20 June 2015.
Duke University Health System . What is Pompe? . 2013. Web. <http://www.dukechildrens.org/services/medical_genetics/pompe>. 20 June 2015.
National Institute of Health. Pompe disease. February 2010. 20 June 2015. <Web. <http://ghr.nlm.nih.gov/condition/pompe-disease>>.
Parvis, Ethan N. The Pharmaceutical Industry: Access and Outlook. New York: Nova Publishers, 2002. Print.