BBC HomeExplore the BBC
This page has been archived and is no longer updated. Find out more about page archiving.

24 September 2014
Science & Nature: TV & Radio Follow-upScience & Nature
Science & Nature: TV and Radio Follow-up

BBC Homepage

In TV & Radio
follow-up
:


Contact Us

You are here: BBC > Science & Nature > TV & Radio Follow-up > Horizon

Life Blood
BBC Two 9.00pm Thursday 11 October 2001

Professor Hal Broxmeyer developed the technique behind cord blood transplant Click for programme summary
Back to homepage
BBC Health has more on this subject



Transcript

NARRATOR (DILLY BARLOW): Matthew Farrow was born with a rare and fatal blood disease. It seemed just a matter of time before he would die, but when he was just five years old he became the first person in the world to be given a radical new treatment that many believed would fail, yet it saved his life.

MATTHEW FARROW: It's just kind of wild that it was just me like a guinea pig and I had my second chance, like a cat has nine lives so I guess this is my second chance in life.

NARRATOR: The treatment that saved Matthew is so simple it seems almost incredible that it should work. This is the story of how that treatment was discovered and how it is now giving new hope to thousands of critically ill children. Duke University Hospital in North Carolina is the place you hope your child never has to go. Here many of the children are so ill their local hospital can do no more for them. They've come to Duke from all over the world because it's pioneering a radical new treatment for children with a range of fatal blood diseases. Even with this treatment many will still die, but without it they have almost no chance at all. Six-year-old, Max Dearborn, has just been transferred to Duke for the new treatment. It's his last hope. He's suffering from a particularly aggressive form of childhood leukaemia. It seemed to strike him out of the blue.

CHRIS DEARBORN: Since he was, he was five he started to get some bruising, some really weird bruising and we went to the paediatricians and asked quite a few times... thought it was strange and the initial response was well, you know, he's a kid, right, I mean he's five years old, he's out playing, he's, he's a boy, he'll tend to bruise, but it, it kept on so we finally got them to do a blood test.

JAYNE DEARBORN: Before that he's been a very healthy child. He, the day before he was out doing a slip and slide on the grass, you know, with water and just finished his baseball season. No fevers, nothing unusual, so it really came as a very big shock to us.

NARRATOR: Max's doctor at Duke is Joanne Kurtzberg. She's one of the world's experts on children's blood diseases and is a driving force behind the new treatment which will be used on Max. The treatment is still seen as experimental, but if offers Max his last chance.

PROF JOANNE KURTZBERG (Duke University Medical Centre): We think he has roughly a 10% chance of being cured of this disease.

INTERVIEWER: That's not great.

JOANNE KURTZBERG: No, that's not great, but I think when you're in that situation and you've been told your chances are zero and that you should take your child home to die 10% sounds better than it would if you had many options.

JAYNE DEARBORN: You want to push your button and get… Want me to push it for you? Yeah, you have to just...

NARRATOR: Max's leukaemia is a form of cancer and it's overwhelming the healthy white blood cells of his body, the cells that fight infection. Without enough healthy white blood cells to fight disease Max could die of even a minor illness and before he receives his new treatment Max must overcome his current infections.

JAYNE DEARBORN: He's just got a lot of strength and he knows that a lot of people love him and care for him and I think he's going to do just fine, so, but yeah it's, it's a bit hard on him. I mean he'd rather be out, you know, swimming in a pool and playing baseball and, and all that other good stuff - right Max? - so next summer we'll do that.

NARRATOR: Around the world there are thousands of children, like Max, whose blood has failed. The only way to treat them is to strip out all the diseased blood and plant the seeds of a completely new blood supply taken from a healthy person and the new treatment being adopted here by Dr Kurtzberg couldn't be more simple. It's nothing more and nothing less than a small bag of baby's blood. Dr Kurtzberg is one of many doctors who believe that the tiny quantity of blood taken from the umbilical cord and placenta of a newborn baby has the power to build an entire new blood system. They call it cord blood and the reason she's so sure cord blood can work is because it's full of blood stem cells, the key to life. These are stem cells dividing to make new blood cells. What makes them so special is that they have one truly unique capacity. They can create any type of blood cell that we need; the white blood cells that fight infection and the red blood cells that carry oxygen. Blood stem cells are very rare, yet they are the factory of our entire blood system.

PROF FRANK SMITH (Cincinnati Children's Hospital): The normal blood formation is an incredible process. It's making tens of millions of new blood cells every hour. All of those cells are coming from a fairly small number of rare stem cells which are going to continue this process over weeks, months, years and decades of an individual's life.

NARRATOR: But when the blood system becomes seriously diseased the only thing to do is rebuild a new blood system from scratch. This means replacing the patient's faulty stem cells with healthy ones from someone else. For years science knew of only one source of these special stem cells: the bone marrow, a jelly-like substance inside our pelvis and other large bones. It's the home of stem cells. Until recently the only treatment for critically ill children was a bone marrow transplant. Bone marrow, including the all-important stem cells, is extracted from a donor's pelvis and infused into a patient. The new stem cells start growing a healthy blood system, a process called engraftment. The new blood has all the characteristics of the donor's blood including their immune system and even their blood group, but many of the children at Duke Hospital can't get a bone marrow transplant. That's because they can't find a compatible donor. Doctors have to locate a donor with blood as similar as possible to the patient's. Compatibility has nothing to do with blood groups but with genes. For a bone marrow transplant to work scientists must look for twelve key genes in the white blood cells that need to be the same in both donor and patient. It's what's called the perfect match.

JOANNE KURTZBERG: The closer the match the less likely that the bone marrow would be rejected or not grow at all.

NARRATOR: Rejection can be fatal, and it's why a perfect match is so essential.

JAYNE DEARBORN: How long does that usually take?

MAN: Has he got out of bed yet?

JAYNE DEARBORN: He did stand up today.

MAN: Great. (Yeah) Tremendous.

JAYNE DEARBORN: Sorry, he's just squeezing your hand too tight. Sorry Matthew.

MAN: Max, can I do anything for you? Alright.

NARRATOR: The best match is most likely to come from a close relative, but there's no guarantee and in Max's case none of his family were compatible.

CHRIS DEARBORN: Well the, the first step obviously right after he was diagnosed they, they test the family so siblings again are the gold standard. Parents come next and then you go to the Registry.

NARRATOR: The Bone Marrow Registry is a list of volunteers willing to donate some of their bone marrow to anyone who needs it. Worldwide there are now over seven million people on the list. The Registry has saved many lives by finding a match between patient and donor, but it still can't help in about a third of all cases.

JAYNE DEARBORN: The closest we got was an eleven out of twelve match, perfect being twelve out of twelve, and I think with bone marrow they try to get it as close as possible, so she's still continuing to search, but we haven't found a perfect bone marrow match for Max.

CANDY PRESSEY: There's a good boy.

DR PAUL MARTIN: Every day we come in and listen to you and check your belly. You're going to get pretty bored with that routine. How about sitting up, how about sitting up for us?

NARRATOR: The chances of finding a perfect bone marrow match for seven year old Lydell Pressey are even more remote. He also has leukaemia and has recently come to Duke with his mother Candy.

CANDY PRESSEY: First week obviously I cried every day, I cried every day and I was like why, you know, why is this happening to my child. He, he would look at me and smile and I just, we have to smile back and just be thankful that he just smile through this.

NARRATOR: The reason why it's so difficult to find a match for Lydell is that, because he's of mixed race, his blood has a very unusual genetic make-up. Finding a donor of the same complex background with similar blood is almost impossible.

JOANNE KURTZBERG: There was no match for him in the large registries in the United States or worldwide so at that point he didn't have a traditional match donor. It's hard to tell a family they can't have a transplant because it's usually their last hope.

WOMAN: You can close it.

WOMAN: Closed.

WOMAN: OK.

CANDY PRESSEY: You always want to take the pain, you don't want to see him go through the hurt, you know. It's draining looking at your child in the hospital and him not being able to go do normal things, but I pray because I believe that there's going to be a day where he can play normal, grow up to be a normal adult and do things you know.

NARRATOR: Lydell's plight is a stark illustration of why doctors like Joanne Kurtzberg have been so desperate to find a new source of stem cells as an alternative to bone marrow. For years the very thing that might save Lydell and Max's life, the blood from the placenta and umbilical cord of a newborn baby, was simply thrown away. Nobody realised it was of any value until scientists discovered something quite unexpected about the blood system of an unborn baby. A baby in the womb has a very different blood system from children and adults. The stem cells are not in the bone marrow, but for some unknown reason they are in the liver. Close to the moment of birth they begin to leave the liver and travel to the bone marrow. At the moment of birth there's a window of opportunity. For just a few days the stem cells are still migrating through the baby's whole bloodstream towards the bone marrow. The umbilical cord and placenta are an extension of the baby's blood system and so at this precise moment they too are full of these rare stem cells, but the cord only contains a tiny amount of blood, far less than is used in a typical bone marrow transplant. For a long time scientists assumed that this small volume of cord blood couldn't contain enough stem cells for a successful transplant, but then in the 1980s a small team of scientists decided to challenge that assumption. Back in the 80s the problem of bone marrow rejection was becoming increasingly clear, so a few scientists decided to look at cord blood as a new source of stem cells. Among them was Hal Broxmeyer. His task was clear.

PROF HAL BROXMEYER (Indiana University School of Medicine): We had to prove that there were enough stem cells there that could be used for transplantation and that was not a foregone conclusion by any stretch of the imagination.

NARRATOR: Hal Broxmeyer knew that for a successful bone marrow transplant it took at least a litre of bone marrow to provide enough of these rare stem cells.

HAL BROXMEYER: If you think about a litre of bone marrow and, and, and what's needed really for transplantation, why would anybody have thought there were enough cells in cord blood because at the, at the best you're only dealing with maybe a half a cup.

JOANNE KURTZBERG: The scientific community in general was very sceptical and didn't believe that there would be sufficient stem cells in umbilical cord blood to reconstitute bone marrow, so there was really a lot of distrust and, and very low expectations that this would work.

NARRATOR: But nonetheless Broxmeyer and his team set about the painstaking work of counting the number of stem cells in cord blood. Because stem cells are rare they're hard to count and it took months of analysis, but in the end their results amazed them.

HAL BROXMEYER: There were many more cells of the stem cell nature in cord blood than there was in a comparable amount of bone marrow, so you didn't have to have as much volume of cord blood because in a smaller volume there were a lot more cells.

NARRATOR: It was an important discovery. Though there were probably not enough stem cells for an adult transplant, that half cupful of cord blood might contain just enough stem cells to save a child, but to prove cord blood really could work they had to cure a patient. For this, Broxmeyer's team needed to find a doctor and family who were willing to try out cord blood for the very first time. For months his team hit the phones calling hospitals right across America. No one seemed prepared to let a sick child be part of an experiment and use anything other than the conventional bone marrow treatment. While Broxmeyer was still searching for his first patient, Joanne Kurtzberg began treating a new patient at Duke Hospital. Matthew Farrow was born with a rare condition called Fanconi's anaemia, a fatal genetic blood disease.

JOANNE KURTZBERG: We met Matthew when he was roughly a couple of years old and we diagnosed Fanconi anaemia. It's a disease that causes lots of different problems but most seriously the blood system, or the bone marrow will fail or develop leukaemia usually within the first decade of life.

SHIRLEY FARROW: He wouldn't live to be very old and if he lived to be maybe a teenager they did say the Fanconi's would turn into leukaemia.

NARRATOR: Dr Kurtzberg knew that Matthew's only hope was to have a bone marrow transplant, but he was running out of time.

JOANNE KURTZBERG: In Matthew's case because he had abnormalities of his blood counts when he was only two that meant he had a severe form of the disease that would progress rapidly and it was very unlikely he would live to the age of eight or ten years. You know without a transplant at that point his life expectancy was just going to be a few years. We looked for a bone marrow donor in Matthew's family. He had an older sister and we also looked at his parents, but they didn't match.

SHIRLEY FARROW: Those were trying years and it was hard on both of us. We took him everywhere or we tried to that we could afford just because we didn't want him to miss out on, on going to Disneyland or going to other amusement parks before he died.

NARRATOR: Without a suitable bone marrow donor there seemed only one hope for Matthew. If the Farrows had another baby there was a chance it would be a perfect match. They decided to try.

SHIRLEY FARROW: When you have a sick kid it's like you have to open all the doors, you have to open all the windows so that when your child dies you can say I did the best I could.

NARRATOR: Shirley became pregnant and tests showed the baby was a match for Matthew, but there was still a serious problem. They'd have to wait nearly a year before the baby would be large enough to have its bone marrow extracted. Nobody knew whether Matthew would survive this long.

JOANNE KURTZBERG: He could have evolved to leukaemia quickly at any, any time and no one knows if that's going to happen or when it might happen, so when you have a child with Fanconi in the clinical condition he was in it's very nerve-wracking and you're worried all the time that something might happen that's life-threatening and preclude the ability to even try a transplant.

NARRATOR: It was at that moment, as Matthew's condition was deteriorating, that Dr Kurtzberg was contacted by Hal Broxmeyer's team.

JOANNE KURTZBERG: They said what do you think about approaching this family to see if they would be willing to be the first cord blood transplant family in the world. I thought it was a good idea to get Matthew to transplant earlier if we used the cord blood than if we waited for his sister to be big enough to be a bone marrow donor.

SHIRLEY FARROW: We decided since it was Matthew's body that we'd let him decide which way and Matthew said he wanted to help other kids that were sick like him.

NARRATOR: On 25 February 1988 Matthew's baby sister Alison was born. Within minutes of her birth the umbilical cord and placenta were drained of blood and it was frozen to preserve the precious stem cells. The stage was set for the first ever cord blood transplant. They decided to take Matthew Farrow to the hospital with the best record in the world for treating Fanconi's anaemia, in Paris. the idea of using umbilical cord blood as a treatment was then so novel that their top doctor had never even heard of it.

PROF ELIANE GLUCKMAN (Saint-Louis Hospital): Hal called me and told me that he had done some studies on cord blood cells, that he had found that they were enriched in cells which are necessary for engraftment and he asked me if I wanted to do it and I said, "Oh! it's a very great idea," and I said yes immediately.

JOANNE KURTZBERG: Yeah we chose her because of her success and wanting to kind of position this so that if it didn't work we can understand why and you know if it did work to give it the best shot.

NARRATOR: The Farrows had never left the US before and recorded everything that happened in France on their home video camera. Just as with a conventional bone marrow transplant the process began with Matthew's diseased blood stem cells being eradicated by chemotherapy. Then his baby sister Alison's cord blood was infused directly into his body.

RICHARD FARROW: Oh right, how does it feel getting somebody else's blood that's going to do you some good?

NARRATOR: All they could do now was wait to see if the new stem cells would engraft. All this time Matthew had to be kept in sterile conditions for fear of infection.

SHIRLEY FARROW: No you couldn't touch him. He had to have gloves on, no physical contact. I used to put a blanket or a sheet over me and have him sit on my lap but I couldn't actually touch him and then I had a mask on so you wouldn't breathe on him.

I've been in here, you've been sleeping.

RICHARD FARROW: Come in.

HAL BROXMEYER: Hello Matthew. You're feeling better?

SHIRLEY FARROW: Yeah, he feels a little better.

NARRATOR: As with bone marrow transplants the only way to measure success was to count the number of new white cells in Matthew's blood. If the blood count rose the cord blood was probably working. If it didn't increase, the whole experiment had failed.

HAL BROXMEYER: The longer it takes for the white cells to come back which are necessary to fight infection the greater it is the possibility that you could get infected.

JOANNE KURTZBERG: And they can die of an infection before the bone marrow has a chance to regrow and so it's very nerve-racking from day to day in that time period just because you know that that something had changed very quickly and a child could get sick very quickly.

RICHARD FARROW: Even the biggest, strongest guy getting sick, OK, so it's alright if you get sick.

NARRATOR: For Matthew it took longer for the cord blood to work than a bone marrow transplant probably would have done because in only a cupful of cord blood there are fewer stem cells than in a litre of bone marrow, but finally, after 18 days, the cord blood engrafted. Six months later Matthew Farrow was allowed home. Ever since the transplant, twelve years ago, Matthew has been free of the disease. His baby sister's umbilical cord blood had saved him.

ALISON FARROW: It just feels weird to know that you know you saved your brother's life.

MATTHEW FARROW: I'm glad that she did. She was sent here 'cos if it wasn't for her I wouldn't be here.

RICHARD FARROW: He's got your blood type now which he had mine before, B positive. Now he's got O positive which is your blood type. He's actually got your blood system growing in him.

NARRATOR: The experiment with Matthew had proved that cord blood might be a real alternative to bone marrow, but it would be a long time before the rest of the scientific community would accept it. Despite the success of Matthew's transplant in 1988, most of the medical community remained sceptical about the use of cord blood on a broader range of diseases. In the meantime, converts such as Joanne Kurtzberg began to use it to treat more and more children for a wider and wider variety of diseases, not just the very rare Fanconi's anaemia, but more common blood disorders and crucially blood cancers like leukaemia. And as they examined the properties of their new discovery further the more remarkable it turned out to be and in one crucial respect they thought that cord blood might not just be an alternative to bone marrow, it might even be better because cord blood doesn't need a perfect match. It's all because of where cord blood comes from - babies in the womb. Protected inside the womb, a baby doesn't have a strong immune system, because the mother will fight off any infection for it.

FRANK SMITH: A baby actually is in a sterile, protected environment where their need to have a powerful functional immune system is much less than a child or an adult who's outside of the womb, so in a sense the baby's immune system is unprimed, it hasn't been exposed to a lot of these different infectious agents that we face living in the world.

NARRATOR: Because it is unprimed a baby's immune system is less aggressive, so the cord blood taken from a newborn is less likely to attack the patient's body after a transplant. Cord blood is, therefore, less likely than bone marrow to cause rejection. For doctors working with cord blood this has been the big breakthrough.

JOANNE KURTZBERG: We began to think that maybe we wouldn't have to match cord blood as closely as we do bone marrow for a transplant and for the, you know, many people who couldn't find a closely matched or fully matched bone marrow donor the hope was that cord blood could mismatch and still be a useful donor and if that was true then with several thousand cord blood units we would be able to find donors for 90% of the patients who couldn't find bone marrow donors.

NARRATOR: Because it's so easy to obtain cord blood from discarded placentas more and more hospitals began to collect and freeze it. In this vat alone there are over 2,000 units of cord blood quickly available for any patient. There are now seven public cord blood banks in America and they've also started to appear in France, Germany and now Britain. With this many banks and no need for a perfect match it's now possible instantly to find cord blood for children like Lydell and Max who couldn't find a bone marrow match.

NURSE: See if I can get into it first. This is it guys.

JAYNE DEARBORN: That's it.

NURSE: So these are the stem cells that Max will be receiving the new cord blood.

CHRIS DEARBORN: Alright.

NURSE: OK (TALKING TOGETHER)

JAYNE: We want to say a prayer.

NURSE: If you want to, OK.

JAYNE: We can say it to ourselves. Do you want to?

CHRIS: Sure.

NURSE: OK.

NARRATOR: Max is now strong enough for the cord blood transplant, but he is still very ill.

JAYNE DEARBORN: There is a little bit of uncertainty just because it hasn't been around as long as bone marrow. But I know that they've seen really good results here, and this is the place to be for cord blood, and other kids have done really well with it and we're just thinking, you know, and praying that that's going to be the best solution for Max and at this point it's really, I think, our only option, and so we need to go with, with that, so.

(ACTUALITY CHAT)

NURSE: This is it pal.

JAYNE DEARBORN: This is it, this is what we came here for.

NURSE: Can you lift up your leg, just for your arm for a second. OK and you put that down. (INAUDIBLE REMARK) That's fine.

CHRIS DEARBORN: There was some tight times there for a while and so we're very thankful that, that we're here. It's, it's just been a long road.

NARRATOR: The fact that Lydell Pressey has even a chance of life is remarkable. Because of his mixed race background his likelihood of finding a perfect bone marrow match was very small, but Dr Kurtzberg found suitable cord blood within a day. It was nowhere near a perfect match, but because it was cord blood it was close enough.

WOMAN: This is what's going to transplant, this is your new life. It came from a little baby girl. It's got a bear on it, a lion, mouse, a monkey. He might sleep through the whole thing.

(GRANDFATHER COMFORTS CANDY PRESSEY)

JOANNE KURTZBERG: Many of the patients that come to our programme are patients at the end stages of their disease who've been searching for other options and haven't been able to find them. In Lydell's case though I would say his odds are in the 60-70% range, that he'll do well. But even if I said his odds were 99%, if he's the 1% who doesn't do well... that's it's an all or nothing kind of thing for him.

NURSE: We're not going to waste even a drop of that blood. We're going, once it gets down in here we're going to rinse the bag out with saline, put that in the tube.

NARRATOR: The next few weeks are crucial for both Lydell and Max. First the cord blood must engraft. The stem cells have to travel into the bone marrow and start producing all the new blood cells, but even if this happens their lives are still in danger. Until their blood systems are fully rebuilt, which can take up to ten weeks, Max and Lydell will have virtually no immune system. It means both children remain extremely vulnerable to infection.

CHRIS DEARBORN: Max has come a long way, he's a tough kid.

NARRATOR: Even though cord blood usually takes longer to engraft than bone marrow, Dr Kurtzberg now prefers it as a treatment for most of her patients.

JOANNE KURTZBERG: I believe for children that cord blood is better than unrelated bone marrow. I do, because it causes less complications for some of the aggressive leukaemias that babies can get. I think that it's the best donor source. It's available rightaway and for many of those diseases timing is, is very important, but that's my personal opinion and not everyone would agree with me.

NARRATOR: Those who disagree with Dr Kurtzberg point to the biggest problem with cord blood. A half cupful of cord blood still contains far fewer stem cells than the normal larger bone marrow transplant. For some children the sheer quantity of stem cells they receive can mean the difference between life and death. The bigger the child, the more stem cells they need and this problem is magnified many times for adults. Heide Hauschild is 32 years old and has leukaemia. It's a disease that affects ten times as many adults as it does children. Her doctor's first response was to look for a bone marrow match in the Registry. Heidi didn't think it would be a problem.

HEIDE HAUSCHILD: After searching and searching and searching: nothing, which was really kind of like a heartbreaker 'cos they'd been through four... four million people to find a bone marrow donor match for me. They can't find one. For whatever reason I'm just totally rare.

NARRATOR: Without a bone marrow match, Heide's outlook is bleak. Being an adult, the usual cord blood treatment is ruled out because this small amount just doesn't contain enough stem cells. Attempts to mix different cord blood samples together to increase the number of stem cells have so far proved ineffective because the different samples react against each other. Heidi now has only one option left. Scientists have started experimenting with cord blood to find a way to grow more stem cells artificially.

HAL BROXMEYER: Many people feel that the only way this is ever going to take on really general applicability for adults is if we can expand the number of stem cells in cord blood outside the body and then give it to people.

NARRATOR: Hal Broxmeyer is one of many research scientists now working to solve this crucial problem. One of his projects involves adding special chemicals called growth factors to each bag of cord blood with the aim of multiplying the number of stem cells it contains. These growth factors are meant to mimic those found naturally in the body which stimulate stem cells to divide, but it remains highly experimental. Nevertheless Heidi's only option now is to try this experimental expanded cord blood. In October last year she volunteered to take part in a study.

HEIDI HAUSCHILD: They're expanding the cells and it's a thick juicy cord - that's his words - and so I'm just hoping that there's enough cells. It's only like 40ccs which isn't a lot, you know, well I'm skinny so that, that helps out. But we know pretty much that this is totally experimental, totally, but it's a chance we have to take at this point so I'll take it, even if it buys me two months, you know, it's two months with my daughter, so... I've got two pictures up there that she drew for me in school. The top one is a picture of God so I wouldn't be afraid if I died. The second one says, "Let us do good to all people," and then says, "I was holding Mommy's hand when she had to get her blood taken." But I have, I have you know to do it for my little girl, I have to do it because I look her in the face and say I did everything I could, but I've got my burial plot all set, you know it's kind of scary when you're 32 years old, you know, I know where I'm going to be buried, so...

WOMAN: Day zero is the day… I bet Heidi could tell you..

MAN: Sure.

HEIDI HAUSCHILD: Day zero's easy, it's the day you get your cells.

WOMAN: So that's kind of the beginning.

HEIDI: Yeah.

WOMAN: Right, got a present for you. It's like you've just delivered a baby.

WOMAN: That's right. Ready?

HEIDI: Ready. Ready as I'll ever be.

(ACTUALITY CHAT)

HEIDI: Yes. Right, here we go. You know I wish I could see into the future. I wish I could see what's going to happen. I just wish I could see.

NARRATOR: After Heidi's transplant they closely monitor the progress of the cord blood stem cells and initially everything seemed to be going well. Her blood system began to rebuild, but even though the stem cells had engrafted, Heidi's immune system was already so weakened that when she caught an infection she could not fight it off. Just a few weeks after the transplant Heidi Hauschild died. There were 20 adult patients in the same study as Heidi. All received expanded cord blood and several of them engrafted. However, only two patients are still alive. Cord blood is a significant medical breakthrough, but even its advocates admit it will never take over from bone marrow, especially in adults. That's why much work is now also being done with bone marrow to get around some of the problems of rejection.

FRANK SMITH: Cord blood certainly has helped us in many, many ways and there's a lot of people alive that got cord blood transplants who would not have been without it, but is it the answer? No. Is it going to replace bone marrow? Probably not. It's complementary to bone marrow.

NARRATOR: Two weeks after his cord blood transplant, Lydell's new stem cells have engrafted and begun to rebuild his blood and immune system completely. The cord blood worked for him and just 21 days later he was ready to leave the hospital.

JOANNE KURTZBERG: When we cure a child who otherwise was going to die from their disease it's terrific and it's wonderful and everyone is happy and, and grateful and it's, it's almost like performing a miracle, but you have to have a healthy respect for the fact that a number of the children that we treat really don't survive and still die either from their disease or a complication of the procedure, so I, I don't think you can just take the positive and, and ignore the negative.

NARRATOR: Max Dearborn was one of those who did not make it. Like Lydell, his cord blood had engrafted successfully, but his immune system took too long to recover. He was hit by a series of infections and - unable to fight them off - he died. The main wish of Max's parents now is that as many people as possible put their names down on the Bone Marrow Registry. This and cord blood are the best way of fighting diseases like leukaemia. Cord blood is still considered experimental and its use is not yet widespread, but there's a growing number of doctors like Joanne Kurtzberg who are convinced that it makes an important difference. In Britain, doctors have so far carried out only 19 cord blood transplants on children with leukaemia and of those just over half have been successful. In total around the world there are over a thousand children alive today who were treated with cord blood, who might otherwise have died.

JOANNE KURTZBERG: We had our ten year reunion this weekend and brought back just a fraction of our patients who we've transplanted, who are now out several years from transplant. And just to see all those kids running around, and some of them now young adults, who are here because they had a chance at having a cord blood transplant, was very meaningful and powerful to all of us.

MAN: (INAUDIBLE REMARK)

JOANNE KURTZBERG: I knew you when you were a baby.

MAN: Tell her, tell her thank you.

CHILD: Thank you.

JOANNE: You're welcome, but it's nice to see you so big.

MAN: Give her a hug for me.

WOMAN: Nice Mother's Day present.

JOANNE: It sure is.

MAN: Sure is.

JOANNE: Sure is.


The BBC Actionline has info for anyone affected by this issue

Actionline is a free, confidential advice service provided by the BBC. If you want to find out about treatments for blood disorders, you can read our printer-friendly Actionline Factsheet.


Click for programme summary Back to homepage BC Health has more on this subject