Neuroblastoma, a rare cancer in

A cancer of the sympathetic nervous
system, neuroblastoma most
commonly occurs as a solid tumor
arising from the adrenal gland in the
abdomen. Neuroblastoma remains one
of the most puzzling of childhood
cancers—ranging from cases of
widespread but benign tumors in
infants that spontaneously and
completely disappear, to high-risk
subtypes in older children that are
relentlessly aggressive.

Keryx Biopharmaceuticals Inc. said  
that its experimental cancer drug
perifosine received orphan drug status
as a treatment for neuroblastoma, a
cancer that affects the nervous system
and mostly strikes young children.

The Food and Drug Administration
grants orphan drug incentives to
potential drugs that treat disease that
affect less than 200,000 people in the
The company recently reported results
from an early stage clinical trial of
perifosine as a treatment for
neuroblastoma. The drug is also being
tested against other cancers.

3F8 is the name of a substance called
a monoclonal antibody. It attaches to
GD2, which is a marker on the surface
of neuroblastoma cells. 3F8 was
produced by white blood cells of mice,
and it must be carefully prepared for
human use.
A person's immune system makes
antibodies to attack germs such as
bacteria or viruses, but it will not attack
neuroblastoma because the tumor is
part of our own bodies. An antibody
that attaches to neuroblastoma can be
made in a laboratory and then given
intravenously to a patient. This
antibody will circulate in the
bloodstream until it finds and attaches
to a neuroblastoma cell. And then the
patient's own immune system will
attack and kill that neuroblastoma cell.

For the past 20 years, pediatric
patients battling neuroblastoma — a
malignant tumor originating in the
nervous system or adrenal gland —
could undergo 3F8 Monoclonal
Antibody Therapy only at Manhattan’s
Memorial Sloan-Kettering Cancer
Center. But thanks to a multicenter
study that includes 14 hospitals
nationwide, more patients will have
access to the unique treatment for the
childhood cancer.

To study the effects of the drug, a
clinical trial in collaboration with United
Therapeutics, the company that
produces 3F8, has begun. As the most
common cancer in infants less than 1
year old, neuroblastoma can be
detected by ultrasound in utero,
according to the American Cancer

Although not yet approved by the U.S.
Food and Drug Administration, the
treatment boasts a five-year survival
rate of 80%. To administer the drug,
MSKCC developed a curriculum for
nurses from the other institutions
involved in the study.

The 3F8 treatment is provided in one-
or two-week cycles. Patients usually
stay in the clinic for three hours. This
includes time for giving medicines to
prevent possible side effects, the 30-
minute 3F8 treatment, and one to two
hours to monitor the patient for side
effects, such as pain, rash, rapid heart
rate, high blood pressure, fever,
vomiting and diarrhea. Most patients’
side effects can be treated on an
outpatient basis, but some patients are
admitted to the hospital overnight for

A trial of an ALK inhibitor for children
who have relapsed after
neuroblastoma treatments.
A national immunotherapy trial  using
monoclonal antibodies and cytokines
to selectively target neuroblastoma
Another experimental treatment  uses
MIBG, a radioactive isotope that
zeroes in on neuroblastoma cells.