Joanne Kurtzberg, M.D., director of the Pediatric Blood and Marrow Transplant Program at Duke University Medical Center, talks about the future of treatment with umbilical cord blood.

Where do you see the future of cord blood going?

Dr. Kurtzberg: Right now, cord blood is used for most of the same conditions that a bone marrow transplant for. That includes treating patients who have cancer, especially leukemia, sickle cell anemia or thalassemia, those who have failure of their immune system or their bone marrow to make blood cells, and occasionally children with metabolic diseases. In all those cases a donor's cells are required to correct the condition and to rescue the bone marrow from chemotherapy and or radiation.

I think in the future we're going to also see cord blood emerge as a source of cells for what is being called "cell therapy" to treat patients with heart disease, strokes, and with vascular ulcers that have trouble healing. It may also help patients with diabetes. Now when I say all this, I don't mean it's just going to work alone – it's going to work in combination with therapies that address those diseases. Here at Duke, we're piloting some studies in children with cerebral palsy and other kinds of newborn brain injuries to see if their own cord blood may help them recover from the injury, so I think the emerging field of cell therapy is going to be the next exciting use of cord blood.

Can you tell us a little bit about Max – the patient we just saw?

Dr. Kurtzberg: Max is a little boy who has cerebral palsy which he developed after he had in utero strokes, but it wasn't really discovered when he was born. As he got older, as an infant, he started to have some neurologic problems and had a scan which showed the strokes. We actually gave Max his own cord blood cells because we know from our work with children who have genetic diseases of the brain that cells go to the brain after we infuse them in the blood and that they can help repair damage in the brain. We're hoping in children with cerebral palsy that that will happen as well. There are a lot of things we don't know about it. We don't know how many cells we have to give, we really don't know if giving them in the blood is the best way to do it, and we also don't know if the age of the tot matters. It might work better if he was two months old than if he's six years old, but we are doing a study to figure all those things out.

How long will he need infusions?

Dr. Kurtzberg: He only has enough cells to have one infusion, and it will take about a year to know if it's made a difference.

What other research on neurological conditions are you working on?

Dr. Kurtzberg: This fall, we'll be embarking on a study we call a randomized trial where we'll be treating half the children enrolled with their own cells at the time we first meet them and the other half a year later, and we won't know which children got their cells at which timeframe. We're then going to evaluate them neurologically and with scans to determine whether the cells made a difference, and that will be the definitive way to be able to, we hope, prove that cells are
helpful.

When you say obviously make a difference, are you talking about children improving neurologically or physically?

Dr. Kurtzberg: In children with cerebral palsy, most of them have something called spasticity, which is stiffness or inability to use an extremity or one side of their body, so we're going to look to see if their function improves after the cells are infused.

In general, do you consider cord blood a miracle liquid?

Dr. Kurtzberg: It saves lives – that part of it is miraculous.

Are you still very cautious?

Dr. Kurtzberg: Yes, and we have to really demonstrate that it helps with this kind of condition, as well as some of the others I mentioned. We do know in animals and in some of the work we've done in donor cells that cord blood cells have the potential to help various organs of the body, but we still have to learn how to do it