Pilot Study of Zoledronic Acid and Interleukin-2 for Refractory Pediatric Neuroblastoma
Status:
Terminated
Trial end date:
2014-08-01
Target enrollment:
Participant gender:
Summary
Neuroblastoma (NB) is the most common extracranial solid tumor in children, with an annual
incidence of 10.5 per million children less than 15 years of age. NB accounts for 15% of
childhood cancer deaths. High risk (HR) patients carry a poor prognosis despite treatment
with intensive chemotherapy, surgery and/or radiation, autologous bone marrow transplant, and
treatment with cis-retinoic acid. New therapies are desperately needed for such patients.
Recently, it has been demonstrated that HR NB patients benefit from anti-GD2 antibody therapy
which directs the immune system against NB cells. To further explore means of harnessing the
immune system to attack NB, the investigators are studying the combination of zoledronic acid
(ZOL) and interleukin-2 (IL-2). ZOL has been demonstrated to have direct anti-neuroblastoma
effects in laboratory studies. ZOL also augments the production of tumor killing white blood
cells called gamma-delta T cells. When used in combination with IL-2, ZOL is capable of
eliciting potent anti-cancer effects in patients, in part, via the expansion of gamma-delta T
cells. In this present trial the investigators aim to study the tolerability of the
combination of ZOL and IL-2 in pediatric NB patients. Patients will also be monitored
radiologically for tumor response to therapy. Correlative biological studies will study the
ability of this drug combination to elicit the production of NB killing gamma-delta T cells
in children.