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			Jesse Gelsinger A family’s quest for answers After their son died as part of a medical experiment, his family struggles with the honesty and ethics of medical research in America. NBC News

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			“I SAW HIM to the gate and I looked him in the eye and I told him how proud I was of him — that he was my hero,” says Paul Gelsinger.        A proud father of a brave son. Eighteen-year-old Jesse Gelsinger volunteered to help make medical history in an experiment with a world-famous scientist at America’s oldest university.        But something went terribly wrong.        For Paul Gelsinger, a father’s fight for answers and accountability would become an agonizing journey through government bureaucracies to congress and finally to court — in a case that raises fundamental questions about patient’s rights and the honesty and ethics of medical research in America.        “I’m a very slow man to anger,” says Paul. “I want to know the truth. And I want to know it all. And I want accountability from everybody.”        Ask friends about Jesse Gelsinger, and they describe a teenager full of life — always quick to smile.        “Just the fact that he was there made everything better,” said one friend.        Or to spar like the pro wrestlers he loved to watch on TV.        “We’d sit there and wrestle for hours,” says another friend. “He would never, ever give up.”
	“It was a good feeling that he could give. And that’s where he was coming from; that he was going to be able to help others. Like he said: ‘Help the babies.” — MICKIE GELSINGER		But, for Jesse, life had never been easy.        “Jesse battled for his life his whole life long and his father has brought him thru it every time,” says Pattie Gelsinger.        They’re divorced, now, but Jesse’s mother, Pattie, says his father has been at Jesse’s side ever since the first medical emergency at age two.        “Jesse went to sleep,” says Paul Gelsinger. “And we could not wake him back up.”        Jesse had lapsed into a coma and doctors at the University of Pennsylvania diagnosed a rare, but serious, genetic problem.        An article about him in the “New England Journal of Medicine” explained that: because of a defective gene, Jesse had trouble producing one of the enzymes we need to help filter out waste.        Without it, dangerous chemicals — like ammonia — could accumulate in his blood and poison his brain, causing confusion, coma, and possibly, even death.        “So this kid had a very rare disorder that was life threatening,” says Paul.        In fact, many babies born with the same genetic problem die        within weeks. Jesse had survived. But the doctors warned — to stay healthy and keep growing, he would need a special diet and a massive regimen of drugs for the rest of his life.        “It was all based on body weight, and the bigger he got, the more pills he had to take,” says Paul. “By the time he was 15, 16, he was up to 54 pills a day.        Fifty-four a day?        “Yes, that’s a lot of pills,” says Paul. “And these were not small pills.”        “He’d say it’s a meal in itself. So, he didn’t want eat after that,” says his step-mother, Mickie.        She says simple pleasures like a hamburger were off-limits for Jesse. Eating the wrong foods or skipping his pills could have disastrous consequences.        And if there was ever any doubt about that, it was erased in December 1998, mid-way thru Jesse’s senior year at this high school in Tucson, Arizona.        That Christmas, his family discovered that Jesse, frustrated with the daily mountain of pills, had stopped taking some of his medication.
	“He was excited to participate,” says Paul. “It became a focal point — the focal point — in his life.” — PAUL GELSINGER		“I found him on his couch and he looked at me and said, ‘I can’t stop vomiting, and I can’t hold anything down. And I’m really scared,’” says Paul.        They rushed Jesse to the hospital.        “And he started tremors,” says Paul. “We had never seen this before.”        “Oh, we were holding him and he had been shaking,” says Mickie. “He had been shaking. And then, all of a sudden, one of his breaths, he just stopped.”        “This kid wasn’t breathing,” says Paul.        “He stopped breathing, just like that,” says Mickie.        How scared were they?        “I thought he had died,” says Paul. “For five minutes, I thought my son was dead.”        But the episode that took him so close to death would lead to a major break-through. Doctors were able to revive Jesse And switch him to a new, more effective kind of medicine. Almost overnight, his entire life seemed to change.        “This kid had never had normal ammonia levels in his life,” says Paul. “And now they were the same as you and I.”
	Paul Gelsinger		“It was joyous,” says Mickie.        Paul says, “This kid popped out of it. He came out of the coma and wanted to eat. I said: ‘You want to eat?’”        And he did start eating?        “Oh yes,” says Mickie.        “He started eating,” says Paul. “This kid put on 40 pounds in four months.”        “You can see it in the pictures?        “Oh, yes,” says Paul.        Mickie says, “And he was eating anything and everything. And he was enjoying it.”        He was back on track, and doing as well as ever?        “Better,” says Mickie.        “Better than ever,” agrees Paul.        His friends noticed the change, too. In a matter of months, jesse went from just 97-pounds to nearly 140.
			Clinical trials •  For basic information about clinical trials and common questions volunteers need to ask, visit ClinicalTrials.gov, a government-sponsored Web site. •  Center Watch: A privately-funded site also tracks clinical trials, lists thousands of them which are currently recruiting volunteers, and has an "informed consent" section for volunteers.          “He looked like he should have looked,” says on of Jesse’s friends. “He didn’t look like a scrawny little kid any more.”        Jesse became more active, building sets for the high school drama club and looking forward to something he’d been dreaming about since he was a little boy — getting his own motorcycle.        But, most of all, his family says the crisis that nearly claimed his life, seemed to fill it with new purpose.        “It changed his whole life,” says Mickie.        “This was Jesse’s wake-up call,” says Paul. “And he took it.”        Having benefited from the life-saving power of modern medicine, Jesse Gelsinger discovered he might be able to make his own contribution to medical science by helping in an experiment aimed at saving the babies dying of the same genetic problem he had.        It was happening at the University of Pennsylvania, where he’d first been diagnosed.        So, after his high school graduation, the Gelsingers went to Philadelphia to find out if Jesse could volunteer.        “He had a chance to help other people,” says Paul.        Later that day, he was running up the stairs in front of the Rocky statue in Philadelphia with his arms raised over his head.        “That’s how ecstatic he was,” says Paul.        “It was a good feeling that he could give,” says Mickie. “And that’s where he was coming from; that he was going to be able to help others. Like he said: ‘Help the babies.’”         EAGER TO HELP OTHERS        A teenager, healthier than he’d ever been, eager, now, to help others in an experiment that would make medical history. But in a way the Gelsingers never imagined.        “I said: ‘Jesse, why are you signing yourself up for a medical experiment?’ That’s when he told me about wanting to help the kids. He said that’s why he wanted to do it.”        His friends in Arizona say Jesse Gelsinger, the teenager saved by modern medicine, was excited when he learned he’d been accepted in a medical experiment that might help save others — even babies. It was being supervised by one of the world’s leading scientists.
	“This kid was getting on a plane, going across the country for the first time by himself. He was going to hail a cab at the airport when he got to Philly to take him to the hospital. It took guts. And he was going of get sick, you know. I was very proud of him.” — PAUL GELSINGER		Dr. James Wilson of the University of Pennsylvania was on the front lines of medical science — a field called “gene therapy”.        For years, scientists have believed that harnessing the power of genes is the golden ring of medicine. That’s because defective genes may be the cause of all kinds of illnesses. If doctors can find a way to replace faulty genes with healthy ones, it could bring cures for everything from heart disease to cancer and add years to our lives.        And no one has been more hopeful than Dr. James Wilson.        “There’s no doubt in my mind that gene therapy will have a tremendous impact,” Dr. Wilson said when he spoke to NBC News in an interview in 1993.        Back then, Dr. Wilson’s team was making headlines by giving a woman copies of a gene her body was missing. In effect, re-writing part of her genetic code — an achievement so remarkable, a fellow scientist called it: “The Kitty Hawk of gene therapy.”        Dr. Wilson went on to be elected President of the American Society of Gene Therapy — the leader in his field. Dr. Wilson’s break-through was based on a fascinating concept. He wondered: Could you take a common cold virus called an adenovirus and modify it by inserting a human gene, then use the virus to deliver a cure rather than a cold?        If the idea worked, Dr. Wilson and his team thought they could save the babies who were dying from the same genetic problem Jesse had and eventually cure dozens of other illnesses, as well.        They needed volunteers and they weren’t easy to find. But his family says Jesse jumped at the opportunity.        “He was excited to participate,” says Paul. “It became a focal point, the focal point, in his life.”        But no parent would want their child to volunteer for a medical experiment without understanding the risks. That’s why the Gelsingers had traveled to Philadelphia to meet face-to-face with the doctors before Jesse signed up.        Dr. Wilson, the scientist in charge, didn’t deal directly with patients, so the Gelsingers talked with other doctors on his team.        What did they say about the possible risks involved with receiving the adenovirus?        “There was no great risk there,” says Paul. “That they hadn’t seen any really bad side effects. That there was just flu-like symptoms is all Jesse would experience with this. The way they described it, this thing looked so safe. Jesse was going to get the flu.”        And Paul Gelsinger says the doctors were already talking about the encouraging results they were seeing.        “It was indicated to me that a patient prior to Jesse had shown a 50 percent increase in her ability to excrete ammonia following gene therapy,” he says.        Fifty percent?        “A fifty percent increase,” says Paul.         WHAT WERE THE RISKS?        The doctors made it clear those remarkable results would not last. A cure was still years away. And they warned that there were some risks:        - a one-day surgery to administer the virus.        - a stay in the hospital to monitor those flu-like symptoms.        - and, later, a biopsy to confirm the gene had been delivered.        But for Jesse, the teenager who never seemed to give up in those playful wrestling matches, another trip to the hospital seemed like a small sacrifice to help in what could be an enormous medical break-through.        And there’s one other reason the Gelsingers felt confident when Jesse signed up: Dr. Wilson’s plan had been approved by the federal government which has rules to protect patients and ensure safety in medical experiments.
	“My last words were ‘I love you son,’” says Paul. “And he gave it right back to me,‘’I love you, too, Dad.’” — PAUL GELSINGER		What’s more, this one was being conducted at the University of Pennsylvania’s prestigious “Institute for Human Gene Therapy.        “They’re experts,” says Mickie. “These are the experts.”        And surely they were telling the Gelsingers everything they needed to know?        “I thought so,” says Paul and Mickie.        Paul Gelisinger says he thought the riskiest part of the experiment sounded like the biopsy surgery at the end.        That’s when he planned to take time off from his job as a Tucson handyman to be with Jesse.        So, early one September morning, a proud father drove his 18-year-old son to the airport.        “This kid was getting on a plane, going across the country for the first time by himself,” says Paul. “He was going to hail a cab at the airport when he got to Philly to take him to the hospital. It took guts. And he was going of get sick, you know. I was very proud of him.”        The experiment started on a Monday morning at a hospital at the University of Pennsylvania.         COMPLICATIONS        Jesse had surgery to insert the genetically-engineered virus. By Monday night, he was on the phone, telling his family back in Arizona that things seemed to be going as planned.        “It was a brief phone call,” says Paul. “Very brief, five minutes, max.”        So when the Gelsingers went to bed that night how did they feel things were going?        “Under control,” says Mickie.        “My last words were ‘I love you son,’” says Paul. “And he gave it right back to me, ‘I love you, too, Dad.’”        The next day, a phone call from one of the Penn doctors, saying: There was an unexpected complication involving Jesse’s all-important blood ammonia level.
	“He told me that Jesse’s ammonia had doubled. That they were very, very seriously concerned he was going into a coma. And I said, ‘Oh, man, I’m on an airplane, I’ll be there as soon as I can.’” — PAUL GELSINGER		“He told me that Jesse’s ammonia level was elevating,” says Paul. “That he was going in and out of coherency.”        It was something the doctors thought they could control. But Jesse wasn’t responding as well as they’d hoped.        And within hours, things were getting much worse.        “He told me that Jesse’s ammonia had doubled,” says Paul. “That they were very, very seriously concerned he was going into a coma. And I said, ‘Oh, man, I’m on an airplane, I’ll be there as soon as I can.’”        Paul Gelsinger would arrive the next morning and hear a warning about his son he could hardly believe.        Three months after graduating from high school, Jesse Gelsinger had volunteered for a medical experiment that could lead to a revolutionary breakthrough in gene therapy.        He knew it wouldn’t cure him, but it might help save other children who suffered from the same genetic illness he’d been fighting his entire life.        “It’s about as pure as it gets,” says Paul. “This kid was doing the right thing.”        But something was going terribly wrong. Instead of the flu-like symptoms, he’d been warned about, Jesse had suddenly lapsed into coma. Doctors said it was totally unexpected in an experiment that had been going well.        Now, this father was flying across the country to be at his son’s side.        “I was awake all night on that airline,” he says.         SERIOUS QUESTIONS        But there was something Paul Gelsinger says he didn’t know and wasn’t told. Years earlier, a scientist just a few miles away from the Gelsinger’s home in Tucson had raised serious questions about whether the experiment was safe.
			Documents •  Feb. 8, 2002: FDA letter, attempt to ban Dr. Wilson from other experiments. •  March 3, 2000: FDA Warning Letter, original findings about the gene therapy experiment.          “I came in initially quite negative,” says Dr. Robert Erickson, a prominent gene researcher at the University of Arizona.        He had no direct involvement in Jesse’s case, but back in 1995, he had been a member of a government panel that reviewed Dr. Wilson’s experiment when it was first proposed.        In this strongly worded critique, Erickson wrote: “I find this gene therapy protocol unacceptable.”        Why?        Erickson wrote: “An animal dying or being so sick that it’s put down to be studied is obviously a major concern.”        What troubled Erickson was a report from Dr. Wilson’s team that an early version of his genetically engineered viruses had triggered severe reactions, even death, in some animals. Apparently because so much of it — trillions of particles — had been injected.        “Such huge amounts of virus are being given,” says Erickson. “I mean fantastically more than would ever be produced in our body from an infection, triggering all sorts of inflammatory responses, etc., have a potential toxicity.”        Risking that kind of immune system reaction, where the body, in effect, turns on itself, might be justified, Erickson thought, on terminally ill patients with no other hope.        But not on someone like Jesse Gelsinger, whose condition was under control.        The bottom line is: he saw risk with relatively little benefit?
	“Such huge amounts of virus are being given. I mean fantastically more than would ever be produced in our body from an infection, triggering all sorts of inflammatory responses, etc., have a potential toxicity.” — DR. ROBERT ERICKSON Gene researcher, University of Arizona		“That’s right, yes,” says Erickson. “I came in very concerned.”        On the other hand, Erickson says Dr. Wilson’s team assured the government reviewers: the experiment would be closely monitored for safety, the doses for people would be much lower than the monkeys got, and volunteers would be fully informed of the risks.        So despite his reservations, Dr. Erickson ultimately joined others on the government panel in approving the experiment — confident, above all, that it was being supervised by a scientist with an impeccable reputation — Dr. James Wilson.        Careful?        “Careful,” says Erickson.        Methodical?        “Methodical,” says Erickson. “Really good basic science.”        He’s one of the stars in this field?        “Yes,” says Erickson. “On the physician side of things, I would say Jim is the best.”
			More from Dateline • Schedule • Homepage • Newsletter        The trouble is, Paul Gelsinger says, Dr. Wilson’s team had never told him that anyone had ever raised questions about safety.        “No, I had no idea that that had occurred,” says Paul.        He says he didn’t know when Jesse had signed up, and he still didn’t know as he rushed to be with his son.        By the time Paul Gelsinger’s red-eye flight to Philadelphia arrived Wednesday morning, the doctors said the poisonous ammonia in Jesse’s blood had soared even higher. He was sicker now than he had ever been.        “He was in a coma,” says Paul. “He was hyperventilating. I called Mickie, I was crying. I told her this kid was in big trouble.”         IMMUNE SYSTEM IN TROUBLE        It had been just 48-hours since Jesse Gelsinger had been given the experimental virus, and, now, it seemed as if his entire body was reacting to the invasion. Jesse’s immune system was in over-drive — the kind of reaction Dr. Erickson had feared, only much worse.        One by one, key organs were failing, and, then, from one of the doctors — a warning Paul Gelsinger could hardly believe: His boy, who had spent so many years successfully battling his illness, might not survive the experiment.        “I said, ‘Whoa, Whoa. What are you telling me?’” says Paul. “He’s saying 10 percent chance of survival. I said, ‘What? Don’t you have an artificial lung or something you can use to help this kid, you know, to save him.’ And he said, ‘You know, there’s a unit. We’ll need to get a specialist here.’ And I said, well, let’s do it.”        As doctors struggled to keep Jesse alive, a hurricane was bearing down on the east coast. Mickie Gelsinger was racing across the country, her plane landing just minutes before the hurricane closed the airport. She was praying this was just another crisis they would pull through.        “In my heart, it was just another trip, another big scare. Another ‘damn it, we did it again, you did it again, now let’s go home,’” she says.        By the time she joined Paul at the hospital Thursday morning, Jesse was on a dialysis machine to filter his blood and an external lung to breathe for him. But no machine could stop the chain reaction the virus had triggered.        “This immune reaction was going on that was unbelievable,” says Paul. “When we finally got to see Jesse in the early afternoon, he was unrecognizable. He had swollen so much, we couldn’t recognize our own son.”        The bloating was that extreme?        “That bad,” says Paul. “His ears had swelled shut, extruded the wax out. His eyes were swelled shut. There was no movement. Not a twitch. Nothing. It was pretty overwhelming to see him like that.”        And to think that he didn’t even have to do this.        “Of course not, no,” says Paul.        “He was looking forward to it,” says Mickie.        “He wanted to help,” says Paul.        The next day, when doctors said there was no sign of brain activity, the Gelsingers prayed together at Jesse’s bedside and made the decision no parent wants to face.        “It was a beautiful ceremony, just very brief, five minutes,” says Paul. “And when the chaplain was done, I indicated to the doctors to go ahead and came in, turned off the ECMO unit, clamped off the blood supply. Shut off the ventilator. About 60 seconds later, Dr. Raper came in with a stethoscope, put it on Jesse’s chest. And he said: good-bye Jess, we’ll figure this out. My boy, doing the right thing.”        Through his pain, this father was about to begin a search for the truth. Was Jesse’s death really as unexpected as the doctors said?        What Paul Gelsinger was about to discover would raise questions about honesty and integrity in medical research, about cover-ups and conflicts-of-interest and, in the end, whether Jesse was betrayed by the doctors he and his family thought they could trust.         Read Part Two: How a possible medical miracle, became a terrible medical mistake

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