John Crowley (Brendan Fraser), an executive at Bristol-Meyers, is trying to leave his office. He has a balloon bouquet that says “Happy Birthday” in pink, and a pink remote-controlled car. He hurries, but misses his train. He scrambles for a taxi, and tries to get to his destination: His daughter’s 8th birthday party.
At home, his wife Aileen (Keri Russell) is reading a book to young son, Patrick (Diego Velasquez), while daughter Megan (Meredith Droeger) is tormenting her other brother, John Jr. (Sam Hall). Megan is in an electric wheelchair, and has a respirator attached to her neck, yet zooms around the house chasing John. Mom tells them to stop and that if Megan kills her brother, then she won’t have her birthday party. They drive to the bowling alley, where Megan bowls with the aid of a metal launcher. Patrick is shown, also in a wheelchair and respirator. Brother John is healthy, and also at the party with lots of other children. Megan asks her mom where her dad is, and she tells her he is on hi way. While opening presents, Dad arrives, and gets to enjoy the last part of the party with his family. Megan loves the remote-controlled car.
Late at night, John is reading his notes on Pompe Disease, which 2 of his children have. It is a genetic disease, which is characterized by the child not having a key enzyme, and this leads to enlarged organs, and eventually death by age 9. There is no treatment or cure, and John refers to promising research being done at the University of Nebraska by Dr. Robert Stonehill. His notes show that he has called and left many messages, but never spoken to Dr. Stonehill.
In the morning, Megan’s alarm on her respirator is bleeping; she has a cold and is having difficulties breathing. She tells John to go to work, and she will call with information later. He gets a call, and they are in the hospital. The doctors are concerned about her breathing, and want to keep her for observation. He hurries to the hospital, and is told that she has been moved to Intensive Care. Her doctor talk to both Aileen and John and says that her organs are so enlarged, and failing, and that she doesn’t have long to live. He also says that it just might be a blessing if she dies so that she wouldn’t suffer. John and Aileen spend the night in her hospital room. In the morning, the doctor comes to tell them that Megan is some kind of fighter, and is going to be ok. John tells the doctor that her recovering is the real blessing.
When John is in a meeting at work, he suddenly announces that he has to leave, right in the middle of his presentation. He flies to Nebraska to speak with Dr. Stonehill (Harrison Ford), without even telling his wife.
John waits all day in the lobby of Dr. Stonehill’s office, and hasn’t gotten to see him; he is busy doing his research. John dozes, and Dr. Stonehill leaves. John is woken up by the secretary, and he rushes out to catch him. He follows Stonehill in his car, and finds him at a bar. He introduces himself, and says two of his children have Pompe disease. Dr. Stonehill says he’s sorry because it is a terrible disease. John asks him to explain his research, and Dr. Stonehill says that he is at a standstill since he doesn’t have adequate funding. He says he’d need $500,000 to get a working study and marketable drug ready. John tells him that he is the leader of the Children’s Pompe Foundation, and will get him the money before they meet at a medical conference in one month. They shake hands and part.
At home, John is met by an angry Aileen, who can’t believe he’d leave his presentation in the middle and jeopardize his job, which they desperately need for the health insurance. He says he just snapped, and had to go. He tells her about his “new” foundation, and how they need to raise $500k. They start fundraising. When Dr. Stonehill arrives, they give him a check for $91,000. He is amazed that they got that much, but knows it isn’t enough. Dr. Stonehill proposes that the two go into business together, with John as CEO and the business/fundraising end, while he’ll manage the testing and research. They’ll need to go to Nebraska. They shake and agree.
In Nebraska, John sits down with Bob and wants to formulate a business plan to present to venture capitalists to invest. Bob wants no part of it, and says he is too busy, then leaves to go fishing. When they meet with the potential investors, who are scientists at biomed research firms, they are impresses with Bob’s research, and then ask questions about his plan to test and get FDA approval. Bob has never done this, and balks at their suggestions. They tell him that he has no experience at developing, testing and marketing a new drug, and he is furious, yells at them and storms out.
John stays up late, and formulates n business offer to take to the head, Ken. He flies to Chicago and catches him at his house, presents the offer. Ken reads it and is surprised at the terms: he asks if Bob Stonehill has approved the deal and John lies and says yes. Ken signs it, and they are in business. When he later shows Bob the deal, and that they have funding, Bob is furious and vents at John. John explains to him that he just discovers treatments on paper, and that all his research has never helped one person. After a long pause, Dr. Stonehill signs the agreement.
With funding, they build and set up their new company, Prio-zine. Bob has hired a bunch of grad students to work for them. While working one evening, the power goes out from a storm. Dr. Stonehill says not to worry because the back-up generator will turn on. John tells him that there is no back-up generator, since he didn’t mention it in the building specs. Bob rants that they have 45 minutes to get the refrigeration back running or the experiments will all be ruined. They hurry to Home Depot and neither has cash or credit enough to get it, so the grad student offers his credit card, and just asks that they pay him before month end so he doesn’t get evicted. Power is restored in time, and all is saved.
John meets with Bob and tells him that financial reasons make it necessary that they sell their company to their competitor, Zymagen. With their backing, they will each make $6 mil in the sale, plus be funded for development and FDA testing, and eventual marketing. Again, Bob is angry and doesn’t want to give up control, but grudgingly realizes that John is right. Zymagen execs fly to their lab in a helicopter, and listen to the presentation from John, and then the scientific data from Bob. They tell Bob his procedures and testing methods won’t work with the FDA and he gets angry (again). He calms down, and realizes as much as he hates it, they have the money and expertise he doesn’t. They make a deal, and move to Seattle, where Zymagen is located. First day there, Stonehill tacks his check up on a bulletin board, and tells John he won’t cash it until they have a useable enzyme. John is told by the head executive that everyone on the board is a doctor but John, and others are resentful that he has such a high position. He tells him that he’d better not make trouble and just keep his head down, if he wants to keep his job.
John and Aileen move to Portland, in a large house with a view of the water. The kids see the doctor there, and they are told that at best, Megan will live a year or less, and Patrick has less time than that.
John is now highly motivated to get things going. At Zymagen, there are 3 other researcher projects with Pompe disease. But Zymagen policy keeps them all separate with no sharing of knowledge or ideas. So, John plans a breakfast meeting and invites his friend to fly in and speak. Aileen and the kids are there and talk about life with Pompe disease, and the researchers see the children with the disease. His friend tells that he, too, has two daughters that both have Pompe. The oldest is Lauren, and she was too ill to come. The baby, Megan, isn’t showing symptoms yet, but she also has it. He weeps, and thanks them for doing what they can for his children. The executives at Zymagen and angry that John planned this meeting behind their backs and that he is too emotionally involved in it.
The executives eventually agree to allow a leadership team with representatives from the 4 different Pompe research projects…but not if one if Dr. Stonehill. His abrasive personality has alienated him from almost everyone there, so John says he’ll take his place on the team. When Bob hears this, he goes ballistic, and doesn’t want to have anything to do with John anymore. The team decides that they will each work independently for now, and then all 4 lines of research will get a color-code: it will be a blind evaluation of each one, to see which is most promising. This one will be “the mother of all enzymes”. Each will just be known by its color, except to the top people. When the best one is identifies, it will be the one to be tested in clinical trials. John brings Bob the data, but he angrily kicks John out of his office. Later, he brings the data to John and tells him the green line is the best, and it isn’t his work. He thinks his is better, but not ready, and the green one is adequate and ready to go.
John is thrilled that testing is about to begin, and hopes to have his children be first, but they decide that from a clinical standpoint, the first trials will be done only with infants. Infants should respond better and require far less drug. John goes to his office, and breaks down when he hears this. Later that evening, he sneaks into Bob’s briefcase and steals is id badge. This scans him into the lab where he grabs a few vials of the drug, to use on his own kids. The security guard catches him, and wants to know that he’s doing as he doesn’t have access to this area. Just then, Bob comes in and says he sent him to get the vials, and it’s OK. The guard leaves, and the two have a talk. Bob realizes how devastated John is by being excluded from the drug trials, and has an idea: a sibling study. If they write up a protocol for testing on 2 siblings who both have the disease, this has great medical value. He will try to find hospital that is willing to perform the trial, and if he finds one, then present the sibling trial idea to the executives. John hugs him and thanks him.
When he gets a letter from a hospital in Portland that agrees to the trial, he is excited and heads to the exec’s office. Before he gets there, he is yelled at by the executive, as he has just heard about the trial. They are furious at John and Bob for doing this behind their back and without permission. John goes to his office, and begins typing his letter of resignation. The executive comes in with a security guard. He tells him not to finish, because they are firing him. He goes on to explain that they are firing him so that there won’t be any conflict of interest, and that they will do the sibling study, with his 2 children. He leans over to shake John’s hand and says that he never liked him, with a smile on his face, John answers back, “likewise” and they shake.
He tells Aileen at home, and they are all happy. At the hospital, Patrick and Megan are in the same room, in beds next to each other. John pushes the button to start the drug drip into the IV, and Megan comments that the drug isn’t pink, like she requested. A nurse tells them that “Uncle Bobby” is here for a visit. They look confused…and in walks Dr. Stonehill. HE brought presents for the kids, and wants to see the trial in action. They all wait for hours….and then they hear laughing. Crazy wild laughter from their two kids. John and Aileen can’t figure out what would make them laugh like that, and then Dr. Stonehill says “It’s a sugar high”. The drug enzyme is working! They all rush in to see the kids, and even Dr. Stonehill smiles and gets choked up.
Later, we see that Megan is able now to sit up in a car, which she hasn’t been able to do since she was a toddler. Baby Megan, their friend’s baby, took her first steps and can walk, since she was one of the infants in the trials. Megan still wants the drug to be pink. John is working for further research and development for Pompe treatment, and Dr Bob Stonehill finally cashed his check…and started his own company, Stonehill Bio.
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