DURHAM, NC (Ivanhoe Newswire) - More than 760,000 children and adults are living with cerebral palsy. Now, for the first time, doctors are studying a treatment that has the potential to stop the disorder in its tracks.
There's nothing Jenny and JD Stephenson wouldn't do for their son, Weston, and that included banking his umbilical cord blood when he was born.
"We actually felt a little silly saving it because it was so expensive, and we weren't guaranteed to need it for anything," Jenny Stephenson, told Ivanhoe.
But they would need it. When Weston was 10 months old, doctors diagnosed him with cerebral palsy.
"Of course, you want the best for him, and you never want anything to be wrong," Jenny said.
Weston's developmentally delayed and has trouble using the left side of his body. There's no cure for his CP, but there's hope in his cord blood. As part of a clinical trial at Duke University, Weston receives his first infusion. Hisparents are thrilled.
"This was just the most exciting thing we had heard," Jenny said.
"If this is beneficial, it could really change the lives of those children tremendously for the better," Joanne Kurtzberg, M.D., chief scientific officer medical director at Robertson Cell and Translational Therapy Program at Duke University Medical Center, said.
Doctor Joanne Kurtzberg said the theory is cord blood cells can decrease inflammation, coax normal cells into fixing damaged tissues, and grow into new cells.
"Cord blood cells can graft and grow into some types of brain cells in the brain," Dr. Kurtzberg said.
Patients in a phase one trial reported improved speech, mobility and movement, but doctor Kurtzberg said that study did not compare the cord blood to a placebo. The new study, that Weston is a part of, does. If Weston got the placebo on his first try, he'll get the cord blood later in the trial.
"It's why you get up every day and go to work," JD Stephenson said.
"My hope is that we see a miracle, really," Jenny said.
Doctor Kurtzberg's trial is still enrolling patients. To be eligible, your child must have a CP diagnosis, be between one and six years old, and their cord blood must be available.
BACKGROUND: Cerebral palsy (CP) is a group of disorders that can impair brain and nervous system functions. Patients may have issues with movement, learning, hearing, seeing, and thinking. CP is caused by injuries or abnormalities in the brain. Most of these problems happen as the baby grows in the womb, but they can also happen at any time during the first two years of life when the baby's brain is still developing. In some patients, parts of the brain are injured because of low levels of oxygen. Premature infants have a slightly higher risk of developing CP. Other conditions like bleeding in the brain, brain infections, head injuries, infections during pregnancy and severe jaundice can cause CP. (SOURCE: U.S. National Library of Medicine - The World's Largest Medical Library)
SYMPTOMS OF CP: Symptoms of CP may vary, depending on the severity of the disorder, but they usually are present before a child is 2 years old. Some of these symptoms include: muscle tightness, abnormal gait, joint tightness, muscle weakness or loss of muscle movement, abnormal movements, tremors, unsteady gait, loss of coordination, speech problems, hearing or vision problems, seizures, pain, problems with swallowing, and floppy muscles. (SOURCE: U.S. National Library of Medicine - The World's Largest Medical Library)
STANDARD TREATMENTS: There is no cure for CP. Currently, doctors may subscribe medications to lessen muscle tightness and improve functional abilities. Drugs can also relieve pain and manage complications associated with spasticity. Injections of Botox may also help isolated spasticity. Physical therapy, occupational therapy, and speech therapy may also be used to enhance functional abilities. Surgery may be needed to lessen muscle tightness or correct bone abnormalities caused by spasticity. (SOURCE: Mayo Clinic)
NEW HOPE: Researchers from Duke University Medical Center are studying whether infusions of a child's own cord blood could help improve the symptoms of CP. Children with the spastic form of CP who are between ages 1 and 6 and have their own cord blood available for use may enroll in the clinical trial. The cord blood is infused intravenously. Because it is the child's own cord blood, there is no risk of rejection. "There's a huge need to be able to come up with a treatment for cerebral palsy for children who have lifelong problems," Joanne Kurtzberg, M.D., from Duke University Medical Center, told Ivanhoe. Dr. Kurtzberg says if her research proves to be a success, the next step is to try to use cord blood to prevent CP. She is currently conducting a study where she is treating newborns that have low oxygen at birth with their own cord blood. (SOURCE: Ivanhoe interview with Dr. Kurtzberg)
Dr. Joanne Kurtzberg, Director of Pediatric Bone Marrow Transplant Program and Director of The Carolina's Cord Blood Bank & Chief Scientific Officer of the Sullen Translational Therapy Program, talks about reversing cerebral palsy.
What is cerebral palsy?
Dr. Joanne Kurtzberg: Cerebral palsy describes children who have problems with spasticity and sometimes abnormal movements that affect their motor development.
What are some of the standard treatments?
Dr. Joanne Kurtzberg: There is no cure for cerebral palsy. Cerebral palsy can result from different things that damage the young child's brain, so sometimes that means a baby might have a stroke during gestation or a baby might have low oxygen around the time of birth and have damage to the brain as a result of the low oxygen. In premature babies there can be bleeding in the brain during the period of prematurity that leads to damage to the brain that causes cerebral palsy, but bottom line, regardless of the reason a child might have cerebral palsy there's no cure or therapy, and currently children receive supportive care in the form of physical therapy and medications that help relax their muscles. Sometimes they receive injections of Botox into the muscle to help it relax and then just developmental support of care and trying to find ways to adapt their function with tools and devices.
Tell me about your study.
Dr. Joanne Kurtzberg: We're studying whether an infusion of a child's own cord blood cells, that would have been collected and stored when they were born, can lessen symptoms of cerebral palsy. The reason we had the idea to do that is because we had been treating children with genetic diseases that affect the brain for years with unrelated donor cord blood transplantation. In those diseases we're using cord blood cells to replace missing enzymes or factors in the blood and in the brain, but in doing those transplants we learned that the cells do go to the brain even though we give them in the blood and that they help the brain repair damage that's been caused by the disease. From that we thought well maybe in the case of a child with cerebral palsy who's damage isn't due to a genetic disease, it's due to something that happened generally in pregnancy or birth, if we gave them their own cord blood cells if could help their body repair the damage.
What have been the results of the study?
Dr. Joanne Kurtzberg: We have formal safety results knowing that it's safe and feasible. We made some observations in those studies that look very promising, but to really know if this is working we felt we had to do what's called a randomized placebo control trial and we're in the middle of that trial right now.
How many patients are currently enrolled in that trial?
Dr. Joanne Kurtzberg: I can't give you the exact number, but it's between 35 and 40. We will do our first evaluation after 60 patients have been evaluated one year after treatment.
Is it a blind trial?
Dr. Joanne Kurtzberg: It's totally blinded to everyone caring for and evaluating the children, but we do see some children improve and we don't know yet if those are the children who got their cells or if those children would have improved anyway. We really need to wait until the study is done to be able to say for sure.
Will the parents have to save or donated the cord blood?
Dr. Joanne Kurtzberg: Some parents have elected to store their babies cord blood for their own family's use in something called private banking, not because their child will need it, but in case the cord blood has value, they're saving it as kind of an insurance policy for future use. Those are the families that have brought their children who also ended up having cerebral palsy to our study, but if our study were to show benefits, then I think we'd have to rethink when and why parents might want to save cord blood. There's also a public cord blood system where parents donate cord blood for use. We'd have to figure out a way somehow every child's cord blood could be saved but it could be used for them if they needed it or used in the public registry if they didn't.
Who would be a candidate for this study?
Dr. Joanne Kurtzberg: A child with cerebral palsy that predominately is what we call the spastic form, which is the form where children have stiffness of their usually legs or arms, they have to be between the ages of 1 and 6 years and they have to have their cord blood available. The cord blood itself has to have been stored in a way that would keep the cells alive and in a way that we would know the cells are safe. That means they have to be cultured, we have to know that they were sterile, we'd have to get a sample to test to make sure it matches that child.
And how many infusions are there we saw one the other day
Dr. Joanne Kurtzberg: Um we're in general doing 3 infusions a week um that's not because we don't have more patients that want to come but it's what we can handle in terms of getting all the testing and scanning and things that are required for the study.
How many infusions will each patient need during the course of the study?
Dr. Joanne Kurtzberg: The amount of cord blood that is collected when a baby is born can vary tremendously based on the size of the baby, the condition and health of the baby at delivery and who's collecting it. If the obstetrician is collecting the cord blood and responsible for the care of the mother and the baby, and the baby is also sick, frequently less cord blood is collected than if everything is going very smoothly. On average anywhere from 1 to 10 ounces can be collected and if more is collected, there may be enough up to 3 doses, but if just a little bit is collected there might only be enough for one dose.
Where is it infused?
Dr. Joanne Kurtzberg: It's infused intravenously. A regular peripheral, what we call an IV, is started in the arm, foot or hand, just wherever the child has a good vein and then it's infused just like IV fluid.
How does it work in the body?
Dr. Joanne Kurtzberg: The theory behind how it might work in the body is complicated because it probably works in more than one way. We know that cord blood has the property of some of the cells in decreasing inflammation. We know that when there's an injury in the brain, one of the first ways the body reacts to that injury is with inflammation and inflammation causes damage. We also know that the cord blood cells will home or go to the areas that are damaged and tissues that are damaged and then in those tissues, give out signals that cause the normal cells that are there to start repairing damage. They're recruiting normal cells to fix pathways or reconnect nerve that go to muscles or calm down nerves that are excited. It has an influence on the cells that are normally in the body in that area that's called a paraquin or an atrophic effect. Thirdly, we know in the genetic disease patients, that cord blood cells can graft and grow into some types of brain cells in the brain. We don't know if that would be happening in the patients with cerebral palsy because this is their own cells and we don't have any way to tell the difference between what we put in and what's already there.
What's the next step for this research after this part of the trial is done?
Dr. Joanne Kurtzberg: We expect that if we can treat children at a younger age, we could maybe prevent cerebral palsy instead of lessen effects of cerebral palsy. We actually have a study ongoing now where we're treating newborns who have low oxygen at birth or identified at birth with a problem with their own cord blood. If we find that the current study is beneficial we're going to have to figure out a way to treat the children who don't have their own cord blood. We can't really make this a study that's restricted just to people who happen to save their own cord blood, we have to know how to use the donor banked cord blood safely in a child with CP. Right now we use donor cord blood in children with cancer, but when those children get the cord blood they also get high doses of chemotherapy and sometimes radiation, which both treats their cancer and also makes their body to be able to accept and not reject donor cells. Now, in a child with cerebral palsy, we don't want to give high dose chemotherapy, so they accept a donor cell. We have to figure out a way that will help the donor cells stay in their body for a while and help it repair the brain but not expose them to the high risks of chemotherapy.
What is the benefit of using the patient's own cord blood opposed to a donor cord blood?
Dr. Joanne Kurtzberg: When we use a child's own cord blood in the case of cerebral palsy or other brain injuries, their body will accept their cord blood because it was theirs and it's not anything foreign to them. If we were to give a child with cerebral palsy donor cord blood without any preparation, their body would reject those cells and those cells would not be able to work or have an effect. Your own cells are much safer to administer and will be accepted by your body without any other kind of preparation, whereas donor cells will not be accepted unless the immune system is suppressed so that they're not rejected. When you use your own cord blood there's no risk that the cord blood cells will react against you, when you use donor cord blood there is a risk that the donor cells will react against the patient, that's called graft versus host disease and that's a major limitation and complication of a transplant with a donor cell. Cord blood causes less of that then says bone marrow, but it still causes some and when you use your own cells then you don't have to worry about that. There are different arguments on both sides of the fence if you will, so if you have leukemia you can't use your own cord blood because your own cord blood may have leukemia cells in it, and also can't properly fight off any residual leukemia. So for cancer or a genetic disease where we don't know how to fix genes yet your own cord blood would be effective, you have to use a donor, but when you're thinking of regenerative medicine and you're thinking of repairing tissue, then having your own cells available has much more benefit and it's much less risky.
Can you talk about the public cord bank?
Dr. Joanne Kurtzberg: We run a public bank here and public banks have to support all the costs associated with donation, so no bank can afford to open sites to collect units at every hospital in the country. Our bank actually has been a little bit innovative in that we developed a kit donation program where if a mom lives in an area where there's nothing set up at a local hospital for her to donate, she can contact us and we can send her a kit. As long as her obstetrician is willing to do the collection and meet our specifications, then the cord blood can be sent back to Duke and be banked here.
What happens if they decide they want to use the cord blood in the future?
Dr. Joanne Kurtzberg: If you donated your baby's cord blood to a public bank and your own baby needs it, you can request to have it back, but it has to be there for you to be able to have it. If it was used for another patient by the time you asked for it, then it wouldn't be available for you. Also, not all units donated to public banks actually are stored because they have to meet pretty rich strict criterion in order to be put into the bank. It might not be there because it didn't meet that criterion, so there's not a guarantee that it will be there.
How about private banks?
Dr. Joanne Kurtzberg: The private banks charge a fee to the family both for the initial banking and then a storage fee every year. When you donate to a public bank, there's no fee to the person donating but the bank actually is responsible for all the costs associated with collecting, testing and storing the cord blood needed.
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