

(Top)
Grey mass at left in CT scan is Samantha Emerson's tumor. (Bottom) Following
chemotherapy, the tumor has disappeared. |
Samantha's
symptoms began in the spring of 1999. "She was sick for months, with vomiting
and stomach pains that grew worse over time," Kris recalls. "She couldn't keep
any food down." The family consulted their pediatrician, who at first thought
Samantha was suffering from the flu. After a few weeks, when she did not improve,
she was referred to a pediatric gastroenterologist who diagnosed an infection
of the intestinal tract. Samantha
was treated with antibiotics, but the symptoms did not go away. During the family's
August vacation in Hilton Head, SC, they grew worse. "She was literally melting
away before our eyes," says Kris. "One morning she threw up right on the beach.
At that moment it sank in - this was really serious. We left for home that very
afternoon." Kris,
now convinced her daughter had cancer or another life-threatening illness, took
her back to the pediatric gastroenterologist. On August 25, Samantha underwent
a colonoscopy. It showed a tumor the size of a man's fist in her small intestine,
an indicator of Burkitt's lymphoma. The disease, rare in the U.S. but more common
in Africa, tends to involve the abdomen in 80 percent of U.S. patients, but more
often the jaw in Africa. Without therapy, it is rapidly progressive. Samantha
was admitted to Robert Wood Johnson University Hospital for treatment under the
care of Barton Kamen, MD, PhD. He is professor of pediatrics at UMDNJ-Robert Wood
Johnson Medical School (RWJMS) and director of Pediatric Oncology at CINJ. Kris
Emerson's worst fears had been realized. "But at least now we knew what we were
dealing with," she says. Cancer
is the leading disease-related cause of death in children between the ages of
1 and 14. Only accidents are responsible for more fatalities. While children are
spared the typical "adult" malignancies of the colon, lung, breast and prostate,
other devastating cancers take their toll. Lymphomas
account for some 12 to 14 percent of pediatric cancers. While Burkitt's lymphoma
may affect all age groups, it is most common in children and young adults. Most
patients with Burkitt's lymphoma develop abdominal tumors and severe anemia caused
by internal bleeding. Like Samantha, they are diagnosed while seeking treatment
for gastrointestinal problems. According
to Kamen, who is continued also chief of the Division of Pediatric Hematology/
Oncology in the De- partment of Pediatrics at RWJMS, the overall cure rate for
pediatric cancer is 60 percent. "We have come a long way in treating pediatric
cancer, but a lot of work still needs to be done in finding successful treatments
for that other 40 percent," he says. Kamen
has been known to begin lectures by holding up a blowtorch and announcing, "There's
no tumor I can't kill." It's his way of explaining what may not be obvious: that
curing children of cancer using the best therapies available is not without its
down side. The long-term effects of chemotherapy and radiation can be hazardous,
particularly to a growing child. Some therapies harm the kidneys or heart. Others
are neurotoxic and can severely hinder learning. "I treat kids, not cancer," he
says. "And treating kids can be very difficult. Chemotherapy can take care of
tumors, but what's the price of the cure? If there is a loss of IQ, or a chance
of developing another cancer later in life, we need to weigh those factors too."

Left
to right: Patient Katie Andryca, Barton Kamen, MD, PhD, and counselor Christine
Callsternberg |
Kamen's
bedside manner includes a host of magic tricks designed to put his young patients
at ease. Parents see his magic in a different way. "Dr. Kamen told Samantha he
was going to melt the tumor, and we believed him," says Kris. Through an intravenous
line inserted by surgeons, Samantha was given two rounds of chemotherapy over
a five-day period, and then sent home. She would receive additional treatments
in 21-day cycles on an outpatient basis. The therapy, which included methotrexate,
ARA-C, prednisone, cyclophosphamide, and other drugs, was designed to kill the
rapidly growing cells that produced the tumor mass. Throughout
the fall, Samantha remained upbeat, even though the treatments exhausted her.
She was hospitalized several times for high fevers, a side effect of chemotherapy.
Though she was not able to attend school, she kept up with her class work with
the help of tutors. All pediatric patients at CINJ receive help from counselors
and child life specialists. When a child is diagnosed with cancer, counselors
visit the school and explain the situation to classmates, teachers and administrators.
Says counselor Christine Call-Sternberg, MTC, MAT, "We work with kids and their
families on life issues, like staying in school. The last thing a sick child needs
is to worry about being left back or failing." There is also a full-time, hospital-
based teacher to help inpatients keep up with school. By
the end of December, Samantha had completed her chemotherapy. Still in remission,
she's back in school, in dance class...in life. "Unlike more slowly growing tumors,
if a Burkitt's lymphoma does not recur in six to 12 months it is unlikely to do
so at all," says Kamen. "We all breathe a sigh of relief with each day off therapy
after six months." 
Eighteen-year-old
Keith Pasichow following treatment for osteogenic sarcoma |
Statistically,
only 3 or 4 percent of adults with cancer in the U.S. participate in clinical
trials. In contrast, approximately 85 percent of children with cancer enter studies.
CINJ, in addition to being the only National Cancer Institute-designated clinical
center in the state, is also part of a national consortium, the Children's Oncology
Group. "This gives us the latest information on clinical trials across the country,"
says Kamen. CINJ's
primary goal of applying basic research to patient care is one of the reasons
Kamen came to RWJMS in the spring of 1999. Drawn to research all his life, he
is one of only nine American Cancer Society clinical research professors nationwide
- and the only pediatrician. His laboratory at CINJ is directly above the treatment
areas, giving him quick access to his patients. He is there to bring them the
newest therapies. Kamen
is currently using aminopterin in clinical trials to treat patients with acute
leukemia in relapse. Leukemia acounts for approximately 30 percent of all newly
diagnosed cases of cancer in children each year. Acute lymphoblastic leukemia
(ALL) is the most common cancer in pediatrics, typically striking children between
1 and 9 years of age. Acute myeloid leukemia (AML) occurs in all age groups. Aminopterin
came into use 50 years ago as a chemotherapeutic agent. It is the predecessor
of methotrexate, a mainstay of modern chemotherapy. Powerful and effective, aminopterin
also has serious side effects. Because it mimics folic acid, just like methotrexate,
it can cause a deficiency in this nutrient, resulting in increased levels of plasma
homocysteine. This is associated with a risk of vascular disease and cognitive
disorders. In children, methotrexate can cause long-term learning disabilities,
seizures and an unpleasant, "zoned out" feeling. The
physician's interest in pharmacology has led him in some interesting directions.
He has found that dextromethorphan, the "DM" in the popular cough suppressant
Robitussin, blocks some of the toxic effects of increased homocysteine levels.
By giving dextromethorphan to children who are also receiving methotrexate or
aminopterin, he hopes to reduce learning and neurological problems. Seventeen-year-old
Katie Andryca of Colts Neck was diagnosed with acute myeloid leukemia (AML) on
December 31 - New Year's Eve. "It wasn't exactly the way I wanted to start the
new millennium," she jokes. Katie's symptoms, which began in September, included
stomach pains, fatigue, weakness and headaches. "I couldn't play gym. I couldn't
even walk fast," says Katie. Katie
is a vegetarian, and her physician at first blamed her symptoms on her diet, saying
she wasn't eating enough protein. After taking a series of blood tests, he referred
her to a hematologist. Puzzled by Katie's abnormally low white count, the hematologist
recommended a bone marrow biopsy. "Katie postponed it for a month because she
was so frightened," says her mother Beth. "In December, we finally convinced her
to go ahead with it." A
few days later the hematologist called and advised the family to see Roger Strair,
MD, PhD, associate professor of medicine at RWJMS, without delay. In fact he'd
already made them an appointment. When Strair informed Katie and her parents that
she had AML, the three cried. "He recommended that Katie see Dr. Kamen," says
Beth. "I agreed, even though Katie hadn't seen a pediatrician in years. She may
be 17, but she's fully grown, a young woman." Beth
Andryca's initial reaction is not surprising, says Kamen. "We aren't reaching
as many teenagers as we should," the physician states. "Many are referred to adult
oncologists when they should be coming to us. They may have adult bodies, but
they're still kids, with kids' needs." Treated as children rather than adults,
teens have a statistically higher chance of entering a clinical trial, which may
offer greater hope for a cure than the standard regimens. Just
after her first chemotherapy treatment, Katie cut her long blond hair and shaved
her head. "I didn't want to deal with losing my hair, so I cut it off myself,"
she says. In a gesture of solidarity her friend cut her hair too. The girls donated
their hair to Locks for Love, a nonprofit organization that makes wigs continued
available for cancer patients who can't afford them. Katie
has just finished her fourth cycle of chemotherapy, which included davnomycin
and cytosine arabinoside, with one more cycle remaining. Though she has been in
and out of the hospital with fevers and infections, she says she is starting to
feel stronger. 
Keith
hopes to improve his range of motion enough to allow him to run with his dog. |
A
primary goal of the new therapies, says Kamen, is a maximum cure rate with minimal
toxicity. "We're trying to eliminate 'no pain, no gain' from our vocabulary,"
he says. Another goal is decreasing time spent in the hospital. For leukemia patients,
hospital admission is usually required only for the first chemotherapy treatment.
Later treatments are generally given on an outpatient basis. "We
are also coordinating our efforts with other specialties to develop more organized
treatments," he continues. "In treating bone tumors, for example, the highest
cure rates are achieved through the use of intensive chemotherapy, surgery and
sometimes radiation." The chemotherapy aims to shrink the tumor, in many cases
allowing for limb-salvaging procedures rather than outright amputation. Bone
cancer strikes more frequently in childhood and adolescence than adulthood. Some
50 percent of these tumors affect the area of the knee. This type of cancer -
osteogenic sarcoma - accounts for some 250 pediatric cases annually in the U.S.
Eighteen-year-old
Keith Pasichow of East Brunswick was one of them. In 1996 the teenager noticed
pain and swelling in his left knee while away at summer camp. He assumed it was
caused by playing too much tennis, but saw the camp doctor anyway. "He had no
idea what it was, but didn't think it was serious," Keith recalls. By
the end of the summer, there was a grapefruit-sized lump in Keith's lower thigh,
just above the knee. He was referred to a pediatric orthopedist, who made a tentative
diagnosis of osteogenic sarcoma and referred him to Joseph Benevenia, MD, associate
professor and vice chair of the Department of Orthopedics
at UMDNJ-New Jersey Medical School and a specialist in musculoskeletal oncology.
A biopsy confirmed the diagnosis. "When
I learned I had cancer, my mind went completely blank, and then I started to cry,"
says Keith. "I was so afraid I was going to die." Keith
began a year of chemotherapy, entering a clinical trial using methotrexate as
well as other drugs. He received his diagnosis just before the beginning of his
senior year of high school, and only attended one day. After that, he was home-tutored.
In December, 1996 Keith came to UMDNJ-University Hospital in Newark for surgery.
Benevenia removed a 25-centimeter section of Keith's tumor and replaced it with
a cadaver allograph. Following the surgery, Keith returned home to recuperate
and continue his chemotherapy. "In
three months I lost 20 pounds," says Keith. "The combination of nausea and depression
completely took away my appetite." Eventually he received anti-depressants and
was taught self-hypnosis and visualization techniques by counselor Christine Call-Sternberg.
"She taught me to relax and concentrate on the food going down into my stomach.
It sounds new-age, I know, but it really helped." Kamen
says depression is not uncommon among teens with cancer. "Three- and four-year-olds
perceive their cancer as something that causes them short-term pain," he notes.
"They cope with the pain, and then forget about it. On the other hand, teenagers
know about mortality, as well as morbidity. Death scares them."
Keith has made a good recovery. Now in remission, he's a freshman at Muhlenberg
College, majoring in theater and business. However, he has developed a strong
interest in medicine and is considering switching majors to pre-med. He plans
to work in Kamen's lab in the summer. Despite
extensive physiotherapy, Keith has not regained full range of motion in the knee.
This spring, he had additional surgery to replace his knee with a prosthesis.
Since
Kamen has been at CINJ, the number of pediatric patients has doubled. Plans include
recruitment of three additional faculty members for the hematology/oncology division.
However, he says his mission is more than providing the best possible care. "With
certain diseases, the best available therapies are just not good enough. So what
we are trying to do here is write the book, rather than just following the recipe,"
he says.
Among Kamen's goals is to develop strategies for identifying patients who relapse.
"Why do some patients with leukemia survive for the long-term while others do
not? We're trying to find differences in cells and patients so we can make accurate
predictions." Another goal is to improve treatment for more resistant cancers.
"We've greatly improved the cure rate for ALL, but we have a long way to go in
treating brain tumors," he says.
With more patients surviving, an important initiative is the program's Long Term
Survivors Clinic, which provides care and support to children and adolescents
who have been successfully treated for malignancies. "Once you're off therapy
for four or five years, the cancer isn't likely to come back, but other issues
arise," says Kamen. "Are you insurable? Can you join ROTC in high school or college
if you want to? Has your renal function been affected by chemotherapy? Are you
at increased risk for early breast cancer? We can help answer those questions."
It
just takes a little magic. |