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NCI Visuals Online.
Human lymphoma tumor cells.
Nelarabine Active in T-Cell
Leukemia and Lymphoma
In late 2005, FDA approved nelarabine
(Arranon®) to treat adults and children
with T-cell acute lymphoblastic leukemia
(T-ALL) and T-cell lymphoblastic
lymphoma (T-LBL), and whose disease
is refractory to or has relapsed following
at least two chemotherapy regimens.
Nelarabine, which was approved under
an accelerated approval mechanism and
also granted orphan drug status, is the
first drug cleared for these rare indications;
an estimated 500 patients per year have
relapsed or refractory T-cell malignancies.
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FDA based the accelerated approval—
which requires the drug’s manufacturer,
GlaxoSmithKline, to conduct additional
studies to verify clinical benefit—on two
CTEP-sponsored phase II clinical trials.
The nelarabine phase II trial in children
was conducted by the Children’s
Oncology Group (COG), whereas the
trial in adults was led by the Cancer and
Leukemia Group B, in conjunction with
the Southwest Oncology Group. In both
trials, complete responses were observed
in approximately 20 percent of patients.
Median OS was 21 weeks in adults and
13 weeks in children. The post-approval
study will be a CTEP-funded phase III trial
conducted by COG and will include eventfree
survival at four years as an endpoint.
A COG-conducted pilot study is testing
nelarabine upfront in patients with
T-ALL or T-LBL who are at increased
risk for relapse. This trial recently closed
to accrual.
Berg SL, Blaney SM, Devidas M, Lampkin TA,
Murgo A, Bernstein M, Billett A, Kurtzberg J,
Reaman G, Gaynon P, Whitlock J, Krailo M, Harris
MB; Children’s Oncology Group. Phase II study of
nelarabine (compound 506U78) in children and
young adults with refractory T-cell malignancies:
a report from the Children’s Oncology Group.
J Clin Oncol 2005:23;3376–82.
Adoptive Immunotherapy Makes
Mismatched Hematopoietic Stem
Cell Transplantation Possible
Blood or marrow stem cell transplants
(BMT) from a donor can cure patients with
hematologic malignancies who are not
cured by chemotherapy alone. The