April 13, 2004
Narrator: Joy Veron is a schoolteacher from Texas who paid a terrible price for a mother's instinct. She was paralyzed from the waist down after a tragic accident that almost claimed the lives of her children. It happened on a family vacation in October of 1999, during a roadside stop near a canyon in the Colorado Rockies.
Joy Veron: My children jumped in the front seat before my mother and I made it to the car. And they were all three standing in the front seat. As the car lurched and lunged forward, I remember them all looking at me with these huge eyes, and we immediately all took off running. It was headed for the canyon. I could see the kids going over in the car in my mind as I was running.
And so I got in front of the car and tried to stop it with my hands, which, of course, I couldn't. And I remember feeling it begin to run over me. I fell backwards, and the front of the car caught me by the heels. I did like a somersault under the car and the third time it hit I knew that it broke my back. I was 30-years-old and the whole life I knew was gone.
Narrator: But now there is new hope for Joy. Her injuries are being treated with an experimental new therapy called regenerative medicine, which endeavors to heal injuries and cure diseases by harnessing the body's own ability to heal itself.
Around the world, patients like Joy are taking part in experimental trials aimed at such elusive goals as treating spinal cord injuries, curing heart attacks and saving lives.
John Martin: I was taught as a medical student that the only way the heart could go is downhill once you're an adult. We've now discovered that's not so. And in fact, it can repair itself and reverse damage done to it. And that has overturned the medical wisdom I was taught.
Narrator: The driving force behind this medical revolution is the stem cell. Before we are born, embryonic stem cells manufacture the 200 types of other cells that every human is made of. After birth, adult stem cells repair damaged cells in the body. The aim of regenerative medicine is to harness the work these cells naturally do, to treat other conditions they were not necessarily designed to heal.
Tom Okarma: We see a day in the not too distant future, when we can change the very meaning of medical therapy, from a focus on treating symptoms, to a focus on fundamentally correcting the basics of the illness.
Narrator: In labs around the world, scientists are studying the genetic and chemical signals that allow stem cells to differentiate and create new cells. Progress is being made on many fronts, and in some labs, doctors have already managed to produce neural cells that could one day treat brain disorders such as Parkinson's disease.
Some of this research is controversial, because it probes the ethics of using embryonic stem cells that are derived from fertilized human eggs. But embryonic stem cells aren't the only ones being studied. Adult stem cells obtained after birth can be harvested from a patient's own body when they are needed. Their full capabilities are still being deciphered, but in one study, doctors believe they have gotten bone-marrow stem cells to create new heart muscle cells just like those produced as an embryo develops.
Tom Okarma: Cells will be pills. The notion here is to actually replace the damaged cells with new ones. That's the promise and that's a sea change for clinical medicine.
Narrator: Regenerative medicine cannot come too soon for patients like 41-year-old Kim Gould. In May of 1998, a brutal riding accident left her paralyzed from the waist down. Kim's doctors told her not to expect any further recovery, but she is determined to regain something of the life she once knew.
Kim Gould: Horses, they've been my life, and I used to keep my horses here as well, so I was up -- up here everyday, and it was like my second home, you know, it was my life here. We were doing just a friendly cross-country course, and as I approached the last jump, the horse clipped the fence and stumbled and I was catapulted over the jump. And it was the actual way I landed, just snapped my spine.
My break's at T7, which is bra line, sort of between the shoulder blades. When they just come up to you and say you're never going to walk again, you just think, oh, once I get out of hospital I'll be fine. You know, it doesn't really sink in. In the first couple of years of my accident I just felt I wanted to sit indoors and mope. And to be stuck in and restricted, you know, it was awful. My whole way of life changed just completely.
Narrator: But now, spinal injury patients like Kim may have another option. Medical researchers believe they have found a way of using adult stem cells to regenerate nerve fibers. The source of the cells was staring them in the face.
Geoffrey Raisman: What was found is that the nerve cells in the nose are continually replaced throughout life, and that's quite important, because we lose them all if we have a cold, or if there's pollution. But we don't lose the sense of smell. That comes back. So that means that those nerve cells are capable of being replaced from a stem cell. This is one part of the nervous system, where there is a continuous replacement and formation of new nerve cells throughout life. And this is there as a result of the evolution of the nose.
Narrator: The idea that stem cells from one's own nose could offer a safe and effective therapy for spinal injuries, prompted immediate research.
Kim Gould: There's always a risk if you have cells from somebody else, or from a fetus, that they'd be rejected. But with regenerative therapy it's encouraging your own cells to regenerate and to repair yourself. I could get on with my life as I am, but you want the best quality of life. So I'm going to go for whatever's out there.
Narrator: Experiments on rats have yielded promising results. In one trial in the UK, scientists cut the nerves that lead to the animal's left forepaw. With such an injury, the rat could no longer use the paw to climb effectively. Nor could it use that paw to reach for its food. Stem cells were then harvested from the animal's nose and transplanted around the damaged nerve. Within weeks, the treatment produced noticeable results.
Geoffrey Raisman: We've been able to restore the ability to climb. We've been able to restore complex reaching and control movements of the use of the forepaw. The sort of functions that a patient would want to recover if they didn't have the use of their hand. When we transplant the cells into that area of damage, the function comes back. You're seeing a glimpse through a doorway that has never been opened before.
This is what will get people out of wheelchairs. This is what will make stroke patients get better. This is what will restore the optic nerve in blindness, and the auditory nerve in deafness. If we can push that door open, there's an immense amount behind it. This will be revolutionary if we're successful.
Narrator: This is welcome news for actor, director and quadriplegic Christopher Reeve. Injured when he fell from a horse in 1995, he is paralyzed from the neck down. But Reeve has vowed that he will walk again. He has made remarkable gains with intense physiotherapy, but he is now past his fiftieth birthday, and fighting the natural downturn in his body's ability to heal.
He is a vocal supporter of stem cell research, and the Christopher Reeve Paralysis Foundation keeps a close eye on all the latest advances. As the research progresses, scientists learn more about the mechanism that allows stem cells to repair the body.
Geoffrey Raisman: Here in this electron microscopic image, we're looking at a mass of stem cells harvested from the nose and have been transplanted into a rat's spinal cord. You can see this pale, this grayish area, with the two blobs. These are the transplanted stem cells. So here you can see the degree of intimacy in the relationship between the nerve fibers, which are growing, and the transplanted cells, which are making them grow.
So there's a nerve fiber, it's a nerve fiber which has been repaired, it's regenerated. And this is the nucleus of a transplanted stem cell, and you can see how the stem cell is wrapping round that nerve fiber. And here's another stem cell, and here it is wrapping around another nerve fiber. What you're seeing here is an act of creation by these cells.
Narator: The stem cells surround and support the rat's damaged nerve cells, giving them the ability to heal and regain function.
Geoffrey Raisman: We get consistent reconnection and restoration of function.
Kim Gould: When I keep hearing all the scientists saying. Well, its -- ye ah, we can prove it on rats and mice, and it's, yeah, well, you know, forget about the rats and mice get on to us. It's so frustrating. It's got to be done now, and if we are prepared to take the risk, try it on us. I am prepared to be the human animal experiment. Why not?
Narrator: But getting permission for human trials is a slow process, as regulators must evaluate the safety of every proposed treatment. Such trials are at least two years away in the US and the UK. Here in Portugal however, the first human tests of this experimental new therapy are already underway. At this public hospital in Lisbon, neurologist Carlos Lima has treated more than 20 carefully-selected spinal injury patients with their own nasal stem cells. Kim Gould has met Dr. Lima's stringent requirements and been approved for the trial. She is eager to know all the details before she goes under the knife.
Kim Gould: So where is my break on the MRI scan?
Carlos Lima: Well, here you can see your vertebrae, and here, you can see your spinal canal with the spinal cord in black and you can see when we come to this part, where you've broken your vertebra. You see there's a gap here.
Kim Gould: Where the cord is actually severed?
Carlos Lima: Yes, it is. We think it's about three centimeters in length.
Kim Gould: And you'll fill this gap with the stem cells from the nose yeah?
Carlos Lima: Yes.
Kim Gould: And where are they on the scan?
Carlos Lima: Well, they are in the upper part of the nose.
Kim Gould: So sort of eye level?
Carlos Lima: Yes, a very high level.
Kim Gould: Right. Okay.
Carlos Lima: You can see the nasal septum, and you also can see your eyeballs here.
Kim Gould: Oh, yeah.
Carlos Lima: We know that the cells start repairs, start to make connections. But nature will do most of the job, not we.
Narrator: Roughly half of Dr Lima's patients come from abroad. Joy Veron is the sixth American spinal injury patient to be treated. Two days after her operation, she is already seeing results. It is still too early for the stem cells to have taken effect, so the improvement is most likely from the removal of scar tissue and the increase in blood flow.
Joy Veron: You know I have constant nerve pain at all times. At all times It's never gone the nerve pain. So for me -- Oh my gosh -- not to have that. It's the first relief I've had in three years.
Narrator: Dr. Lima carefully selects patients who have access to top rehab centers, because he believes physiotherapy is crucial to the healing process. Less than a week after the operation, Joy returns to Texas. Picking up where she left off before the surgery, she undergoes intensive daily workouts using a special exercise bike that electrically stimulates her paralyzed leg muscles.
Joy Veron: I try to exercise as many hours a day as I can, because I feel like that's going to, in combination with the transplant of the cells, that both of them together, I'll get the maximum benefit.
Narrator: Joy is very lucky to be in a position to rehabilitate at all. Her accident happened near her family's vacation home in the Rockies. The runaway SUV with her children aboard came very close to killing her, and the injuries caused by the tires were horrific.
Joy Veron: The back tire basically went this way, and just crushed everything, my lungs, my ribs, my clavicle. And I had my head tilted, and they said that's what saved my life as the tire went this way. My father put his hand on the brake before it went over the canyon and said, "Joy, Joy, I got it!" And then I remember hearing my mother scream, and then they ran to the back of the car and found me.
And I couldn't see because the blood vessels had broken in my eyes from the pressure of being run over, and so I told my dad. I said, I don't want to live, I said I'm blind and I'm paralyzed, I just want to die. And he said, no, no, no, don't say that, you don't want to die, don't say that. And so the next thing I remember is in a helicopter being flown. My family, they were a huge support. They're the reason I basically survived. They gave me the desire to keep living and to go on.
Narrator: In Portugal, Kim Gould hopes she will soon be on the same recovery path as Joy. Her parents are with her as she prepares for surgery.
Ann Bassett: Ever since she had the accident she's been looking into everything, hasn't she? Tried everything, you know. And was convinced that one day there would be something that would help her and ...
Tony Bassett: It's here.
Ann Bassett: Yeah, she's waited a long time for this day.
Tony Bassett: We're still, a little bit apprehensive for her, but should all go well and we're, hoping it will.
Dr. Carlos Lima: Something is changing to give not just hope, but to give function to patients with spinal cord injury. All of our patients have some kind of recovery. We have no doubts about sensory recovery, and some voluntary motor recovery. And even some bladder control recovery.
Narrator: Dr Raisman is not yet ready for his own human trials, but is following Dr. Lima's results closely.
Geoffrey Raisman: I don't think we're ever going to make the person what they were before they had the injury. But if a person can't even move their arms, to be able to throw a switch, to be able to manipulate a piece of machinery, to be able to drive a car, it's going to make an enormous difference to their way of life.
Narrator: Three hours into the nine-hour operation, Kim's spine is ready for the transplant, and doctors can begin harvesting stem cells from her nose. The procedures are complicated, but for Kim, the potential results are worth the risk.
Geoffrey Raisman: This is repair. This is regeneration. This is not treating symptoms. This is reversing part of the damage that has been done, permanently we hope.
Narrator: As regenerative medicine develops, doctors may be able to make the stem cells more potent by manipulating them in the lab prior to reinsertion. But in these early stages, Dr. Lima's aim is simply to harvest as much stem cell-bearing tissue as possible, and transplant it directly back into the patient.
Dr. Carlos Lima: I am asking the patient to treat himself, because your own body will know which are the best cells to multiply, how much and in what direction they should go. And the stem cells that we have, our own body has, that's their job. What we are exploiting is the natural role, their instinct to replace and to repair, because nature made stem cells for that.
Narrator: Six hours into the operation, the stem cells from Kim's nose are set to be packed around her spinal cord. The doctors hope this pioneering step, filmed here for the first time, will trigger a uniquely-safe and long-lasting repair.
Dr. Carlos Lima: When you put the cells in your spinal cord, they're yours, it's natural, you've got an environment which is a very friendly one, and so the cells will grow. We ask the cells to do their job, with the cues that come from the environment and from the damaged area. We know that the stem cells will stay active for months, even years. So we hope to get more function even years after the surgery. So what we say to patients is that if you keep improving, even little by little, who knows where you can go.
Narrator: Kim is finally reunited with her family the next day. But only time will tell if the risks she's taken, will provide the benefits she craves.
Kim Gould: You've got to take that chance. If there's a chance there that you could possibly be better than being stuck in a wheelchair paralyzed, I think you've got to take it. It's that bit of hope that just keeps you going.
Joy Veron: My hope, and I went in to it telling Doctor Lima this -- any little thing to me was going to make the surgery worth it -- the smallest thing. Of course my goal from the surgery would be to walk again, and be as I was before, but I told him anything I didn't have before -- any little bit was gravy for me.
Joy Veron: OK we're going to race. We're going to go that way. Chloe you ready to race? No using your legs. 1, 2, 3 ...
Narrator: Joy Veron is convinced she is already seeing results.
Joy Veron: I have noticed some improvement on my right side. I was always numb really high up. And now sensation level's probably gone down two levels, and I'm completely ... It's a completely straight line now, and I never had that before.
Joy Veron: Stay in Sequence.
Chloe: I'm doing it.
Joy Veron: So I have noticed some improvement and I'm only two months out, so my anticipation is that I will continue to improve.
Narrator: Joy's treatment has brought her together with another patient from Texas who received the same therapy. In November of 2001, 26-year-old Susan Fajt was in a car accident that left her paralyzed from the waist down. Joy and Susan do the crucial physiotherapy together, and have become close friends -- forming a unique bond of common experience.
Joy Veron: Susan is a huge support for me. She's the one that I talk to because I know that she truly knows what I'm going through and I know her struggles every day.
Susan Fajt: Basically my philosophy is kill me or cure me. This isn't my life. I don't like not being able to go on an airplane without assistance. I don't like not being able to do my own laundry. I don't like not being able to reach up and get a plate. I don't like a curb that knocks me over and breaks my nose. I would like to have had children -- normally, you know, and I've been robbed of that at a very young age and would like somebody to return it back to me because this is the most awful thing that could happen to a human being.
Today is my three-month anniversary, being out of surgery. And, number one, I've got my bladder sensation back, to a certain degree, and, you know, there's nothing better than the feeling of knowing when you need to pee. It's very important. And then, number two, about three weeks ago, I can wake up in the night and my legs are moving, and I can feel them moving, and I can actually suggest one to go to the right and the other to the left, and it's working. And it's kind of in a spasm type movement, but, nevertheless, it is movement.
Narrator: Both Susan and Joy attended the spinal injury unit at the Detroit Medical Center, where doctors are monitoring half of the American patients who have been treated in Portugal. Nineteen-year-old Laura Dominguez was one of the first to receive a transplant. Laura broke her neck in a car accident in 2001. After some initial recovery, she was left paralyzed from the chest down, with little control or sensation in her arms.
Laura Dominguez: I was on my way home from summer school, and there was an oil spill on the road, and we lost control of the car and hit the concrete embankment. My spinal cord was injured right above the bone that sticks out in your neck, the C7, it's right above that one. So it's pretty high, but it's not as high as it could be, thank god.
Narrator: Neurologist Stephen Hinderer is seeking FDA approval to bring Dr. Lima's therapy to the US. While it is still early days, he believes the procedure is triggering recovery not normally seen in spinal injury patients.
Dr. Steven Hinderer: The first thing to know is that Laura's injury was a few years old at the point in time that she had the surgery, and usually after about one year we're telling folks you've got most of the recovery you can expect. At two years, certainly, we're not really expecting much change. In the last six months since Laura's surgery, we've seen clear change in her neurological function during a timeframe when little or no change would be anticipated ordinarily.
Dr. Steven Hinderer: Good, alright! Oh! The finger flexors -- excellent! So they're coming along. The thumb's there too. That's a change again. So it's coming.
Laura Dominguez: Everything is so slow, but I've gotten some feeling back, and a lot more sensation in my trunk. And I know it's going to work. There's stuff going on all over the world, and there's a cure out there for spinal cord injuries. From now on, I'll just continue getting better, and then get up and walk.
Dr. Steven Hinderer: We've done some testing today that shows that her sensation is down in to her belly button level. Before it was up around the collarbone level. Trunk muscles in that same area, have recovered some. We now see more arm function around the wrist. The thumb muscle and index finger muscles in the hands are beginning to function as well. Her therapy and physical activity regimen was virtually identical before surgery to afterward, so it's reasonable to begin to assume that surgery is -- is a factor here.
This has the incredible potential for them, and it's the first thing in the 25 years of clinical practice that I have seen something that really has that possibility. The difference is, I will be able to just say to somebody with a spinal cord injury, yes, you will walk again, as opposed to telling them life is good from a wheelchair.
Narrator: Early experimental trials like Dr. Lima's seem to indicate that adult stem cells hold immense promise for the future of medicine. But there are still many questions about the long-term risks and benefits of such procedures. However some adult stem cell therapies have a long and successful record of treating devastating human illnesses. For over twenty years, bone marrow and blood-derived stem cells have been curing dozens of fatal cancers and diseases of the blood and the immune system.
For Julie and John Henderson, these treatments provided much-needed hope, when they discovered a lump on the neck of their son, Nicolas. He was 2 years old at the time.
Julie Henderson: When the surgeon said, Nicolas has T-cell lymphoma, I said, that's cancer of the lymph system, isn't it? And it was like the world stopped moving, and time froze, and I was petrified.
John Henderson: He told us that Nick had cancer. It felt like a trap door open up beneath us, and it just -- he went on talking, I don't think I heard anything else he said. I thought we would lose him. It was very traumatic and the drive home was surreal.
Julie Henderson: Before Nick was diagnosed, I found out I was pregnant. It was just horrible, you know, because we wanted to be happy, you know, we're going to have another baby, but the baby we have now could be dying. I couldn't understand why -- why am I pregnant now, God, why now? Why now?
Narrator: But the timing turned out to be perfect. It allowed doctors to develop a unique treatment plan for Nicolas -- hinged on the one-in-four chance that the siblings would be a genetic match. If they were, blood-derived stem cells from the baby's umbilical cord blood would be harvested right after birth. These cells could then be used to rebuild Nicolas's immune system, which was being destroyed by the chemotherapy that was fighting his cancer. The stem cells in his sibling's cord blood became Nicolas's only chance of survival.
Gerald Maass: It's a simple procedure that creates no harm to the mother, no harm to the child, it's really taking a waste product -- what would normally be a waste product -- isolating and cryo-preserving the stem cells, knowing that at some point in the future they could be useful.
Narrator: Blood from the umbilical cord is a rich source of blood-derived stem cells, and storing them is a simple procedure that may one day become routine for all newborns.But since Nicolas did not have his cord blood stored when he was born, the doctors are hoping to use the baby's instead. After an agonizing wait, the tests reveal the baby's cord blood is a perfect match. His parents name him Nathaniel.
Julie Henderson: His name, Nathaniel, it means "given by God". And he came in to this world as a gift to us to save his brother's life. I knew he was a match, I just knew it. I can't explain that, I just did. And he's brought with him life, not just his own. So it's amazing.
Narrator: In April of 2002 -- two months after Nathaniel was born -- Nicolas began an intense course of radiation and chemotherapy to destroy his cancerous cells and pave the way for the transplant.
John Henderson: I remember the day they brought the cord blood. The lady in a long white lab coat came walking down the hall with a little cooler type thing. I was amazed when I saw it. There's my son's life. That's a central line goes in to his heart, there's the stem cells. This is where I was so nervous, when she's pushing the air out, and I was just, you know.
Julie Henderson: One false move, and there it goes. Don't loose a drop not one drop.
John Henderson: And that's it. Nathaniel's stem cells going in to Nicolas. In the hospital, when they do this with a patient, they do a birthday celebration for them -- like a new birthday. It was an awesome day. I always told Nick that, you know, we got you twice, when you were born, and the day you got your brother's cord blood.
Narrator: Within two weeks, the stem cells from Nathaniel's cord blood began rebuilding Nicolas's immune system. His white blood cell count began to climb.
John Henderson: It's hard to think, sometimes, when you look back at that little thing of stem cells that that's going to do what it did, and there were dark days, and we feel like we were pulled out of an abyss. Stem cells saved our son's life.
Narrator: Today Nicolas is cancer free. He is off all medication and has recently started school.
Julie Henderson: I can't take my eyes off of Nicolas. I just want to see every move he makes on the playground, you know, I want to see everything he does. You know, he's like a normal kid. For so long he wasn't, and now it's life. You know, and they both have life now, and it's just amazing.
Class (sings): Tell us what the weather's like ...
Nic: It's a sunny day!
Teacher: Nicolas says it's a sunny day.
Class (sings): Monday is a sunny, sunny day ...
Narrator: Treating lymphoma with bone marrow stem cells is an established therapy, but doctors are now trying to put these cells to other uses. Here in Germany, they are being used to treat the number one cause of early death in the western world -- heart attacks.
At Frankfurt's leading teaching hospital, cardiology professor Andreas Zeiher is running an experimental trial for severe heart-attack victims. He hopes to repair patients' hearts by using their own stem cells -- stopping their slide into disability and avoiding the need for high-cost medical care.
Dr. Andreas Zeiher: What happens during a heart attack is that the cells die within an hour, hour and a half of being deprived from oxygen. And this finally results in scar formation of the heart. That means those dead cells are like a scar on your skin, they do not contract, they do not contribute to the pump function of the heart. And if your heart attack compromises more than 40% of your left heart, then you will go into severe congestive heart failure.
And it simply eats up your body. It does not allow you to exercise. You have difficulty climbing stairs, and you feel that you cannot breathe at all -- you are almost going to die. And their two year survival rate is less than 60%. So it also has a very, very bad prognosis, comparable to the most aggressive cancer.
Narrator: Dr. Zeiher believes that a low-cost, outpatient bone-marrow self-transplant may be the solution. 41-year-old Thomas Goetz had a major heart attack in January, 2003. Six months later, he underwent the transplant, and his heart soon returned to two thirds its normal pumping capacity. Now, Goetz is back for a second transplant -- hoping for even more improvement.
Dr. Andreas Zeiher: Two months ago, he received almost two hundred million bone marrow derived cells. I would assume that less than 3% of these are actually stem cells. And as long as we don't know which specific cell type is the most potent stem cell for cardiac regeneration, we use all the bone marrow cells we can get.
Narrator: The operation begins with a tiny incision on the inside of the thigh -- to access the femoral artery. This enables the doctors to feed a balloon-catheter into the left side of the patient's heart. Once in place, the balloon is inflated, to gently widen the blocked artery that caused the heart attack. The catheter can then be used to deliver the bone marrow stem cells into the damaged heart. Widening the blocked artery is standard therapy for heart attack victims, but the stem cell transplant is new.
John Martin: We still don't know why the heart can't repair itself, but I think its part of that ageing process that may mean that the stem cells are not as active as they should be, and we're trying to concentrate them where they're needed to increase their activity. Patients understand this instantly. It's not complicated. They like to use the natural processes of their body to heal themselves, and this is more appealing than having some foreign chemical coming into their body. When you're taking your own bone marrow, there's absolutely no danger to you as the patient, so it's a great advance. Self therapy is safe therapy.
Dr. Andreas Zeiher (Subtitle): Everything ok?
Dr. Andreas Zeiher: Ok, here we go. We have now the balloon right where we want to have it. And here we have 36 million stem cells. And now we give the cells.
Narrator: As the cells are injected, blood flow to the damaged portion of the heart is temporarily stopped. Unfortunately, for the patient, this feels like the heart attack has returned.
Dr. Andreas Zeiher: Now blood flow is entirely blocked. This is actually an artificial heart attack. We do have some ECG changes, and I think he starts to feel something now.
Dr. Andreas Zeiher (Subtitle): Do you feel something?
Thomas Goetz (Subtitle): Yes.
Dr. Andreas Zeiher: So the feeling comes again. It's a typical heartburn right in to the middle of the chest, goes in to the left arm, left side of the face. This period of infusion normally causes pain.
Dr. Andreas Zeiher (Subtitle): It hurts doesn't it?
Thomas Goethz (Subtitle): Yes.
Dr. Andreas Zeiher: The bone marrow derived stem cells accelerate and enhance the healing capacity of the heart, simply by releasing substances -- vascular growth factors -- which attract other stem cells which circulate in the blood, so it's an on-going cascade of repair. We try to keep it for two, two-and-a-half minutes -- to give the cells time to adhere -- before they are washed away by the blood. But if patients cannot stand it, we stop. It's not a very pleasant feeling. It's really that you feel that you are in danger of your life.
Dr. Andreas Zeiher (Subtitle): That's right it feels really uncomfortable doesn't it?
Thomas Goetz (Subtitle): Yes.
Dr. Andreas Zeiher: So we stop after 2:30, so the pain quickly disappears after we deflate the balloon and let the blood flow restore oxygen supply to the heart. And now the ECG is already normal.
Dr. Andreas Zeiher (Subtitle): Does that feel better now?
Thomas Goetz (Subtitle): Yes.
Dr. Andreas Zeiher (Subtitle): Has the pain gone completely?
Thomas Goetz (Subtitle): Not completely.
Dr. Andreas Zeiher (Subtitle): Can you still feel some pressure?
Thomas Goetz: Yes.
Narrator: The pilot study for this therapy, published in December, 2002, involved 20 patients. They all showed an improvement in heart pumping capacity three times greater than that from standard treatments.
John Martin: What we can do is we can measure how the heart squeezes the blood. Every single patient in the pilot study had an improvement in how that pumping occurred, which is unusual for a trial. Because biology's so complicated, some patients get better, some patients get worse. In this pilot study, everybody got better on the quantitative analysis.
This rates as a very non-invasive therapy. And it would require very little expense, very little organization to get it in to mainstream clinical practice and it will have a major effect on millions and millions of people around the world.
Narrator: Three hundred heart attack patients have now been treated with their own bone marrow stem cells. The oldest was 84. Wolf Machowitsch is a 40-year-old patient from the original pilot study. He had a massive heart attack in September 2002 and was briefly on the heart-transplant waiting list. Instead, he received the stem cell transplant, and is now at the hospital to find out how his heart has healed.
Dr. Andreas Zeiher: And how do you feel now?
Wolf Machowitsch: Quite well. I have no problems whatsoever.
Dr. Andreas Zeiher: I can just show you what this therapy actually does. This angiogram simply shows the pumping function of the heart. This was before treatment, and I think it's very obvious. In this area here, the heart is perfectly contracting, as we say, so it's moving inward, and therefore blood is propelled outward. But in this area over here, there's almost no movement in whatsoever, no pump function at all.
And this is the scar damage of the heart attack, which does not pump any more, and thereby does not contribute to the expulsion of blood into the body. And this is what we find after four months. And now you see that the scar area is almost completely gone. You see a perfect inward movement of the heart over here, and therefore the expulsion of blood is much better. This we would regard as a completely normal function of the heart. So there is no residual state after the heart attack.
John Martin: This is the beginning of a sea-change in medicine, whereby we understand these cells, we find more types of them, and we understand their importance, not only in repairing damage, but also in the aging of the body, stopping the aging of the body generally.
Wolf Machowitsch: I now have a completely normal life, not like other heart attack victims and for the first time in my life I'm exercising -- walking and jogging. I feel like I can do anything again, and this is really a great experience. I didn't expect that to happen.
Dr. Andreas Zeiher: There's no doubt that this is going to be a mainstream therapy, definitely. So we have, for the first time in our lives, some kind of regenerative therapy for in quote, "a broken heart."
Narrator: While still experimental, the lifesaving potential of this therapy was demonstrated last year. Paramedics received a call that a 16-year-old Michigan boy, had been shot through the heart with a nail gun. The victim later described the shooting at a press conference.
Dimitri Bonnville: My boss, he has a 20-year-old son. He was, I guess, kidding around and there was a nail gun in the room. We were just working, and he picked up the nail gun, and asked to shoot me in the foot, and I said, "Shoot yourself in the foot." And he kind of like pointed at me, you know, was running around the room, and he popped me in the chest, real quick, and then I just fell down. It was just like someone had knocked the wind out of me.
Dr. William O'Neill: It was quite fortunate that the nail was there in his heart, because if the nail had been removed, he probably would have died. The nail was actually preventing him from bleeding to death. There was a chest X-ray that was done when the patient came in to the emergency room, and you can see that the nail is actually sitting right here. It's going through the chest. It's three inches long.
Narrator: Dimitri's heart was so badly bruised, he suffered a series of major heart attacks. The damage to his heart tissue was tremendous.
Dr. William O'Neill: The whole front part of his heart is not working. You can see that this part is normal. But all of the front part and the tip of the heart is not moving at all, there's really no function. In addition, we did an MRI study, and that showed that the entire front portion of Dimitri's heart was entirely dead. Two-thirds of the front and tip had no blood flow going to it. And that all would have ended up as severe scarring that if he would have survived would have left him crippled for life.
Narrator: It looked like Dimitri's only hope was a heart transplant. But even with a new heart, his life expectancy would only be about 10 years, because of the effects of the anti-rejection drugs. Dimitri needed a better option.
Dr. William O'Neill: We were aware of the regenerative therapy that was going on in Europe, Doctor Zeiher's work. We tried not to oversell it but we really had nothing else to offer him and we were honestly trying to prepare them for the worst.
Craig Bonnville: He just had really limited options besides maybe potentially having a transplant at the very worst scenario or even dying. We felt that it was a very low risk, high reward scenario for Dimitri.
Narrator: Dimitri's young age helped with the decision, since stem cells are most vigorous early in life. The doctors filtered the cells from his blood, then transplanted them into his heart using the same procedure as in the German trials.
Dr. Steven Timmis: Yeah, looks fantastic. Really had a great result.
Dr. Steven Timmis: I've been taking care of Dimitri ever since, and I see him every few months. I've never had a patient like Dimitri, both in terms of his youth, in terms of his otherwise good health, and I think that we were really pessimistic about his outlook at the beginning. But he's been doing very well. He's been recovering. Just recently I allowed him to go back to school. I gave him unrestricted activity, which I think is a great victory. This allows him to engage in sports and, basically, carry on life in a normal fashion. Dimitri's a snapshot of the future. I think that the potential for stem cell therapy, not just for heart patients, but for all patients is mind-boggling.
Narrator: If such therapies are successful, they could require less follow-up, and be cheaper than traditional treatments.
Dr. William O'Neill: Dimitri's never had any of the usual complications of heart attacks that we would expect. He didn't need heart drugs, he didn't need water pills, he didn't need anything, really, to support his system and he never had any severe rhythm abnormalities of the heart. I've treated thousands of heart attack patients, and I've never seen that kind of level of improvement in any patient before.
Narrator: Dimitri's recovery provides hope for cardiac patients worldwide, because his own cells restored the pumping capacity of his heart. Doctors measure this capacity using a ratio called the ejection fraction.
Dr. William O'Neill: Normal ejection fraction is 50%, so when he started he had an ejection fraction at 15%, after therapy it went up to 45%, and we're cautiously optimistic that when we repeat studies at a year's time that he may actually return to normal function. The underlying cause of the vast majority of the symptoms that heart patients have, are related to problems with the heart muscle pumping function. If we can fix that pumping function, then people will really basically go largely back to normal.
Narrator: The main pumping chamber of Dimitri's heart was two-thirds destroyed by his injury. His doctors believe the stem cells healed it in a revolutionary way.
Dr. William O'Neill: What we suspect is that the stem cells have floated to that area, and have grown new cardiac cells. And that concept has just completely flipped cardiology on its head. It just has revolutionized the way that we think about the heart and treatments for heart problems. Worldwide, millions of patients could benefit from this. Certainly for the Western industrial countries this has dramatic promise.
Narrator: The spinal injury patients are also optimistic. Six months after her surgery, Kim Gould is back at home and fully recovered from her stem cell transplant. Her legs are still paralyzed, but she is working hard at her rehab, and seeing some early signs of improvement.
Kim Gould: It's still very early days and I am not stupid. I don't think "wow" it's just going to come back just like that. But I have noticed a difference in my lower back muscles and my lower abdominal muscles. Just the way I move and hold myself. I do feel it is a bit stronger down there. And that just gives you hope that it is all knitting together and it's just waiting to see what you are going to get back out of it now.
Doctor: I want to tighten up your muscles and help me raise, alright? Ready ...
Narrator: A year after her transplant, and still doing intensive physical therapy, Laura Dominguez is continuing to amaze her doctors. After two years of paralysis from the chest down, she is regaining sensation and muscle control in ways that shatter normal expectations. Like a baby learning to walk, she is flexing her foot, and pushing out her toes. Her continued recovery is an inspiration for Dr. Lima's other patients, like Joy Veron and Susan Fajt.
Laura Dominguez: I was just trying to get my foot to move in any way that it could even if it was just a little flicker. And I did it. It happened and I was really surprised and then I started thinking well, if that can happen then I can definitely get some more movement back.
Doctor: Push it, push it. Come on, push, push. Good ...
Laura Dominguez: I was excited and I wanted to keep doing it -- make it stronger. The first time it took, like, three or four people to help me stand up, just to make sure I wouldn't fall. I was pretty wobbly. A month ago I could only stand up a few times and I was really tired, I was beat. And today I stand up thirty times, ten seconds each time. And now when I stand I stand on my toes.
Narrator: Laura's results herald a stem cell future that may revolutionize medicine as we know it. But there is still much to be learned about these miracle cells, and only time will tell if they are all we hope them to be.
Doctor: Ready, come on Laura. Push. Good. Ok, come on back down.
Laura Dominguez: Since I got the push in my ankles back I know that if I can do then I can do a lot more. I'm going to be the way I was walking and running and all that. This is just the beginning.
Written, Produced and Directed by
Series Open and Additional Graphics
JULIE SCHAPIRO THORMAN
Executive Producer, Carlton
Executive in Charge
WILLIAM R. GRANT
A Carlton production for Thirteen/WNET New York in association with Carlton International
© 2004 Educational Broadcasting Corporation and Carlton International
INNOVATION was produced by Thirteen/WNET New York, which is solely responsible for its content.