TOPICS > Health

Test Treatments Provide Hope for Pediatric Cancer Patients

December 28, 2006 at 6:25 PM EDT
LISTEN SEE PODCASTS

TRANSCRIPT

SUSAN DENTZER, NewsHour Health Correspondent: About 10,000 children in the U.S. under age 15 are diagnosed with cancer each year. Thanks to medical advances, most are cured.

But Tara Shirkey, age 4, is typical of the more than one in five kids who are not. Standard treatment failed to halt her aggressive case of a normally treatable form of leukemia.

DR. ALAN WAYNE, National Cancer Institute: So, while you play, we’re just going to take a quick check-up all right?

SUSAN DENTZER: Tara is now on an experimental drug dubbed CAT-3888. It was developed here at the National Institutes of Health in Bethesda, Maryland.

DR. ALAN WAYNE: OK, young lady. Listen, you have a great day. We’ll come and see you later, OK?

SUSAN DENTZER: Having just completed her second of six cycles of treatment, Tara is so far responding well, says Dr. Alan Wayne. Shown here with Tara’s parents, he heads the pediatric cancer branch at NIH’s National Cancer Institute.

DR. ALAN WAYNE: We’re very pleased with how well she’s tolerated this drug. Watching her, clinically, she looks better today than when she started the therapy, so we’re cautiously optimistic, if you will.

SUSAN DENTZER: Tara’s mother, 28 year-old Martha Koon, knows her daughter still faces long odds.

MARTHA KOON, Mother of Cancer-Stricken Child: Her life is depending on this drug. If this doesn’t work, there’s nothing else for Tara. You know, I don’t know what’s going to happen if this doesn’t work. I’m not ready to let go of Tara, and I don’t think Tara’s ready to let go, either.

Common childhood cancers

Martha Koon
Mother of cancer patient
Children's Hospital tried everything they knew of, you know. So I just got on the telephone and started calling different places, because the doctor there was -- they started mentioning like there wasn't much more that they could do for her.

SUSAN DENTZER: From her experimental drug to the support extended to her family here at the Children's Inn at NIH, Tara Shirkey is getting top pediatric care. But her case also points up the need to make more progress in the cancers that commonly affect children, such as leukemias, lymphomas, and brain cancers.

Tara, shown here with her fraternal twin sister, Tasha, fell ill last year when she was just 3.

MARTHA KOON: She started out with a fever. It seemed like the flu symptoms at first. But what really alarmed me is she kept getting nosebleeds, and she would wake up late at night crying. And she would tell me that she was hurting, but she couldn't tell me exactly where she was hurting at.

SUSAN DENTZER: Koon, who also has two other children, told us she took Tara to the family pediatrician near their home in Perry County, Ohio.

MARTHA KOON: I showed her some bruising that Tara had on the palms of her hands. And she told me right then, "I don't want to scare you, but I think she's got leukemia."

DR. ALAN WAYNE: Tara has the most common form of childhood cancer known as acute lymphoblastic leukemia, or ALL. It's a cancer originating in the bone marrow and then percolating out into the bloodstream, so it's a cancer of a blood-forming cell that basically fills up the bone marrow, choking out all the other normal blood-producing cells.

SUSAN DENTZER: Wayne told us children like Tara with ALL can present with everything from infections and anemia to uncontrolled bleeding. That's because their bodies experience shortages of white or red blood cells or clot-producing blood platelets.

If untreated, the cancerous leukemia cells spread quickly to other organs, and ultimately cause death.

DR. ALAN WAYNE: So children with ALL are treated with a complex program, usually lasting approximately two years, with a wide variety of chemotherapy drugs, with or without radiation therapy, highly complex, very effective.

SUSAN DENTZER: That's what Tara got during her initial course of treatment at Columbus Children's Hospital in Ohio.

DR. ALAN WAYNE: Unfortunately, her disease relapsed very early on during therapy. She was on chemotherapy for less than one year when her disease came back.

MARTHA KOON: Children's Hospital tried everything they knew of, you know. So I just got on the telephone and started calling different places, because the doctor there was -- they started mentioning like there wasn't much more that they could do for her.

Turning to experimental treatments

Dr. Alan Wayne
National Cancer Institute
We know that there are differences between cancer cells and normal cells. And frequently, those can be seen on the surface of the cell, for example, a different protein.

SUSAN DENTZER: One cancer organization she contacted referred her to an information line at NCI.

MARTHA KOON: So I called it, you know, kind of left a brief message, you know, that Tara had been diagnosed with ALL, and that she relapsed, and none of the standard chemotherapy treatments were working. And I asked them if they had anything available for her. And the very next morning, Dr. Wayne called.

SUSAN DENTZER: His message was that Tara Shirkey might be eligible for the trial of CAT-3888. It's one of a new generation of cancer drugs dubbed "targeted" therapies, designed specifically to attack cancer cells. That's different from standard chemotherapy drugs, which can kill healthy cells and cancer cells alike.

DR. ALAN WAYNE: We know that there are differences between cancer cells and normal cells. And frequently, those can be seen on the surface of the cell, for example, a different protein.

This designer drug goes after a protein known as CD22. It's almost like the cell has an I.D. badge that says, "I'm a cancer cell," or, "I'm an ALL cell wearing CD-22 on my surface."

SUSAN DENTZER: One component of the drug detects the protein and homes in on the cancerous cell. Another component, a powerful bacterial toxin, then kills the cancer cells. In an earlier trial testing the drug on adults with a different form of leukemia, half those treated went into a complete remission.

DR. ALAN WAYNE: So it's almost like you have a bomber with a very sophisticated guidance system that can direct the bomb, the poison payload, to the cell of interest, the cancer cell, avoiding the healthy, surrounding normal cells.

SUSAN DENTZER: When blood tests confirmed Tara was eligible for the trial, she and her mother traveled to the NIH just before Thanksgiving. Tara became the 19th child to receive the drug.

Dr. Wayne says widespread use of clinical trials such as these is one reason so many kids with cancer are now cured.

DR. ALAN WAYNE: Approximately 70 percent to 80 percent of children with cancer are cured. And more than half -- up to 65 percent of children -- are treated on clinical trials, and that's in stark contrast to adults with cancer, where only a very small minority are treated on controlled clinical trials. That's allowed us to very systematically improve the therapies.

SUSAN DENTZER: Now, by studying the safety and efficacy of newer targeted drugs like CAT-3888, researchers hope to develop treatments that will be more effective against aggressive cancers like Tara's.

Since these drugs also target just cancerous cells, it's also hoped that they'll leave kids with fewer long-lasting side effects.

Psychological side-effects

Martha Koon
Mother of cancer patient
It's been like a tornado, kind of. It kind of like picks you up, and all of your family and your whole life, and spins you around. And where you're going to end, you don't know.

SUSAN DENTZER: Then, there are all the other side-effects, the ones felt by families of kids with cancer, who can be torn apart financially and emotionally by their child's illness. No one knows that better than Valerie Sobel. Her 18 year-old son, Andre, died of an inoperable brain tumor in 1995.

VALERIE SOBEL, Andre Sobel River of Life Foundation: I don't think that all the instruments in an orchestra could really describe the impact on my husband, myself, my parents, my daughter, the unbelievable loss of my husband.

SUSAN DENTZER: And in fact, exactly one year after Andre's death, Sobel's depressed husband, a successful trial lawyer, took his own life.

Sobel has since created the Andre Sobel River of Life Foundation in her son's memory. It provides financial assistance to needy families with very sick children and is now helping Martha Koon and Steve Shirkey, Tara's parents.

Koon has had to quit her minimum-wage job to care for Tara in the hospital, and the family has moved in with Shirkey's mother. The parents struggle to hold the family together, as each alternates between being in Bethesda with Tara or staying back in Ohio with the other children.

MARTHA KOON: It's been like a tornado, kind of. It kind of like picks you up, and all of your family and your whole life, and spins you around. And where you're going to end, you don't know.

SUSAN DENTZER: Koon told us that, as much as she wants to be with Tara, the long separations from her other children are hard to bear.

MARTHA KOON: I haven't been there mothering them. Somebody cannot mother your kids. Only you can mother your kids. And I feel like I'm doing a terrible job of it.

SUSAN DENTZER: It's a typical predicament, says Kathy Russell, chief executive officer of the Children's Inn at the NIH. An independent nonprofit organization on the NIH campus, it offers lodging and psychological and social support services to families of NIH pediatric patients.

KATHY RUSSELL, The Children's Inn at NIH: Often the mother or the father will come with the child while they're here, separating the balance of the family, children and the other spouse. That's very divisive and very difficult for these families.

And you couple that with economic disadvantages or loss of employment, and life becomes very, very difficult.

SUSAN DENTZER: Recently, Koon spoke with Amy Lewis, the spiritual wellness coordinator at Children's Inn, about her fears of spending Christmas in Ohio without Tara.

MARTHA KOON: Every day I've got with Tara, I'm just thankful. And I don't really see having Christmas without Tara. It's not really going to be a big day for us, probably, especially if Tara is not back.

AMY LEWIS, Spiritual Wellness Coordinator: It sounds like the biggest gift would be to be together.

MARTHA KOON: Yes.

SUSAN DENTZER: And a few days later, that's exactly what happened. The Andre Sobel Foundation paid to have Tara's entire family come to NIH late last week so they could be together over Christmas.

Reunited, the family joined Tara in her hospital room. She wore a mask since, with her immune system so seriously compromised, she was battling two potentially deadly infections.

As she continues on the experimental drug, Tara's physicians say she's now in uncharted territory. The outlook for her survival is guarded. The miracle is that, with her family now gathered around her, she's lived to see another Christmas.