Medical Minute 6-3: Saving Quentin: Little Sister to the Rescue

By: Vanessa Welch Email
By: Vanessa Welch Email

Quentin Murray is a funny, energetic eight-year-old.

Hard to believe that just four years ago, he was living with excruciating pain every day.

"My legs would hurt every time I was standing up. I'd be saying, 'Oww!' said Quentin.

"He was totally healthy and then, all of a sudden, these mysterious aches and pains," said Mary Webb Murray.

Weeks later, when Mary was three months pregnant, Quentin was diagnosed with a high-risk, potentially deadly leukemia. His best chance of survival: An experimental stem cell transplant.

"I just kind of decided I was going to believe everything was going to work out at that point because I wanted Quentin to be saved. I wanted Quentin to still be with us."

Against all odds, baby sister Jory was a perfect match. That allowed Quentin to have what may be the first-ever human transplant of placenta-derived stem cells, which have a powerful anti-leukemic effect.

"Once the stem cells start growing and producing, it will make him have normal cells, and hopefully, get rid of his leukemic cells," said Lolie Yu, MD Professor of Pediatrics LSU Health Sciences Center.

Thanks to his baby sister, Quentin's cancer-free.

"I'm glad she saved my life because I would've died."

"He's completely healthy now. It's like he's never been sick."

Now, with a healthy future ahead of him, Quentin's his old self again.

For more information on other series produced by Ivanhoe Broadcast News contact John Cherry at (407) 691-1500, jcherry@ivanhoe.com.

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MEDICAL BREAKTHROUGHS - RESEARCH SUMMARY:

BACKGROUND: Leukemia is a cancer of the body's blood-forming tissues including the bone marrow and the lymphatic system. It usually starts in the white blood cells. White blood cells are potent infection fighters. They normally grow and divide in an orderly way as the body needs them. However, in patients with leukemia, the bone marrow produces a large number of abnormal white blood cells that do not function properly. Many types of leukemia exist. Some forms are more common in children while other forms occur mostly in adults.
(SOURCE: Mayo Clinic)

ALL: Acute lymphoblastic leukemia (ALL) usually progresses rapidly without treatment. It is the most common type of cancer in children ages 1 to 7 years old. ALL is the most common type of leukemia in children from infancy up to age 19. ALL affects the blood cells and immune system.
(SOURCE: Leukemia and Lymphoma Society)

TREATMENT: Treatment for leukemia depends on many factors. Doctors may recommend a treatment plan based on the patient's age, overall health, type of leukemia, and stage of the cancer. Common treatment strategies include chemotherapy, biological therapy, targeted therapy, radiation therapy, and stem cell transplant.
(SOURCE: Mayo Clinic)

PLACENTA STEM CELLS: Now, doctors are looking at placenta-derived stem cells as an experimental option for patients with leukemia. In one case, doctors at LSU successfully transplanted stem cells from a baby's afterbirth to treat a child with ALL. They used human placenta-derived stem cells and umbilical cord blood to treat the patient. The patient, who was just 4 years old when he was diagnosed with ALL, received the cells from his baby sister who was a perfect match. The likelihood of a sibling matching the patient is between 25 percent and 35 percent. The placenta-derived stem cells have a powerful, anti-leukemic effect. Now, the patient is cancer-free. "We followed him closely in the first 100 days after the transplant, and after that, on a monthly and three-month basis. When they are two years out after the transplant, basically, we say they’re cured of their leukemia, and he is," Lolie Yu, M.D., professor of pediatrics at the LSU Health Sciences Center, told Ivanhoe. ( Source: Interview with Dr. Yu)

FOR MORE INFORMATION, PLEASE CONTACT:
Leslie Capo, Media Relations
LSU Health Sciences Center
(504) 568-4806
LCapo@lsuhsc.edu

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Lolie Yu, M.D., Professor of Pediatrics at the LSU Health Sciences Ctr., discusses how a stem cell transplant could help some patients with leukemia.

So, talking about Quentin, what was his specific problem and how dangerous was it?

Dr. Lolie Yu: He was diagnosed at an early age with acute lymphoblastic leukemia, which is the most common form of childhood leukemia. However, in addition to just figuring out what type of leukemia it was we also do testing to find out some biological factors that may affect their outcome as well as their response to treatment. In Quentin’s case he had what we call a very, very hyper diploid type of leukemia, which puts him in a very high-risk category. Without getting a transplant and just chemotherapy the disease two year survival is only in the range of 20 -25 %.

So, is there an alternative? Before these stem cells came along what was the alternative for treating children that have this particular type of leukemia?

Dr. Lolie Yu: Without transplant, they are treated with conventional chemotherapy and eventually the patient relapses and then you try to put them back into remission. Historically, when we look at all those patents they’re the patients who don’t go into remission, or if they do then it’s very short-lived. The leukemia comes back again and eventually they die from the disease.

So, what did you do in Quentin’s case?

Dr. Lolie Yu: When he was diagnosed, his mom was pregnant and at that time. I was also conducting a study with the use of human placental derived stem cell and cord stem cells. So, we approached the mom to ask if she would be interested in having us collect her cord blood once the baby is delivered and she said yes. At that point we went ahead and initially treated Quentin with conventional chemotherapy to put him in remission and when the baby was delivered we collected the cord blood and with the method that Celgene has developed they also collected the human placenta-derived stem cells. After the collection, we did some studies to figure is there were enough stem cells to ensure the success of the transplant and sure enough the collection was a very good collection. So, we knew we had adequate stem cells to proceed with the transplant.

And how is the transplant done? How do you transplant stem cells in a little boy like Quentin?

Dr. Lolie Yu: He was initially given what we call, a conditional regimen with high doses of chemotherapy and total body radiation. Then we rescue him with the infusion of the cord blood stem cells initially and it was immediately followed by the human placental-derived stem cells. He tolerated the infusion quite well and recovered very promptly, as a matter of fact earlier than we expected.

What do the stem cells do? Why do they work? What do they do inside the body?

Dr. Lolie Yu: With the condition regimen, basically, it’s called an ablative form of conditional regimen, which means that we try to get rid of all his leukemic cells and in so doing unfortunately we also affect the normal cells that are in his marrow. Without stem cells, the normal cells that are infused would not recover from that. The stem cells, basically, need to be closely matched stem cells that he won’t reject and you have to have a good environment for the cells to grow. Once the cells start growing and producing then it will make him have normal cells and hopefully get rid of his leukemic cells.

And the cells are infused directly into his blood stream?

Dr. Lolie Yu: Yes, it’s just like a blood transfusion/

And what was the outcome for Quentin? Is he cancer free? How quickly did that happen?

Dr. Lolie Yu: The first thing that needs to happen is to know that he has not rejected the graph and this is what we call engraphment. For cord-engraphed stem cells we expect evidence of recovery in at least 14 days and it can be as long as 42 days. In his case he recovered before 14 days. As a matter of fact, we were able to discharge him from the hospital by 17 days after the transplant.

And is he cancer free now? Where is he now?

Dr. Lolie Yu: We followed him closely in the first 100 days after the transplant and after that on a monthly and 3 month basis. When they are two years out after the transplant basically, we say they’re cured of their leukemia and he is. He’s two years old.

How much of a breakthrough is this whole stem cell thing for these kids with this particular leukemia?

Dr. Lolie Yu: I want to make it very clear it doesn’t apply only for this type of leukemia, but for any form of patients who need a transplant to cure them of their leukemia. So, it could be ALL, which he has, or it could be AML. It appears at this time, based on laboratory work as well as this first clinical transplant, that the human placenta stem cells do augment cord stem cells to facilitate the engraphment. Secondly, I think there are some unique characteristics and properties of the human placenta stem cells that can help also with what we call a graph vs. leukemia effect and that I think is important for patients as well as for this study to proceed.

Is this like every transplant, not every sibling is going to be a match. There’s a certain amount of luck involved here too, right?

Dr. Lolie Yu: That’s correct. The likelihood of a sibling matching the patient is between 25 – 35% chance and this is his only sister. Before she was born we figured out she was a match. This was very good for him and for the family.

How happy are you when you see him now?

Dr. Lolie Yu: Oh! Tremendously encouraged. I think seeing him and our first patient undergoing this transplant and he is doing so well really tells us that we should continue with the research and hopefully it can be applied to every patient who needs it.


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