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A Pilot Study of Tumor-Specific Peptide Vaccination and IL-2 With or Without Autologous T Cell Transplantation in Recurrent Pediatric Sarcomas

Phase 2
30 Years
Not Enrolling
Ewing's Sarcoma, Rhabdomyosarcoma

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Trial Information

A Pilot Study of Tumor-Specific Peptide Vaccination and IL-2 With or Without Autologous T Cell Transplantation in Recurrent Pediatric Sarcomas

Nearly all patients with non-metastatic Ewing's sarcoma family of tumors (ESFT) and alveolar
rhabdomyosarcoma (AR) have a dramatic initial response to multiagent chemotherapy. However,
approximately 30 percent of patients develop recurrent disease for which conventional
chemotherapy is ineffective and treatment options are limited. Immunotherapy may represent
an effective approach for treatment of recurrent ESFT and AR. Experimental evidence has
shown that immune mediated anti-tumor effects can occur in vivo when T cells recognize and
respond to antigens present on tumor cells. In ESFT and AR, tumor-specific chromosomal
translocations resulting in the production of novel fusion proteins have been identified in
the great majority of tumors. Peptides derived from these fusion proteins have been shown
to function as tumor antigens for cytolytic T cells in animal studies. This protocol will
study the safety, feasibility and efficacy of tumor-specific peptide vaccination
administered with interleukin-2 therapy with or without autologous T cell infusions in
patients with recurrent ESFT and AR.

Inclusion Criteria


Patients with malignancies of the following histologic subtypes are eligible for
evaluation for treatment on this protocol: alveolar rhabdomyosarcoma (AR), and Ewing's
sarcoma family of tumors (ESFT) which includes classical, atypical and extraosseous
Ewing's sarcoma, primitive peripheral neuroectodermal tumors, peripheral neuroepithelioma,
primitive sarcoma of bone, and ectomesenchymoma. Eligibility will not be confirmed until
the presence of a tumor-specific fusion protein is documented by RT-PCR which corresponds
to one of the tumor-specific fusion peptides available for vaccination.

Patients must be less than or equal to 30 years at the time of initial diagnosis of
alveolar rhabdomyosarcoma or ESFT, weight must be greater than 10kg at the time of
apheresis. Patients between 10-15 kg must be approved by the apheresis unit in the DTM
prior to enrollment on protocol.

All patients or their legal guardians must give written informed consent indicating their
understanding of the investigational nature and risks of the study.

Informed consent must be repeated prior to experimental therapy

Patients may be enrolled on Arm A at the time of initial tumor diagnosis, prior to any
cytoreductive therapy. Cell harvest will take place at this time. They are then
eligible to receive immunotherapy at any time after tumor recurrence if they meet the
criteria. Alternatively, patients may be enrolled on Arm A at any time following tumor
recurrence in an apheresis specimen is available which was collected and processed
according to the guidelines. Such products will have been obtained by apheresis at the
Clinical Center, NIH with informed consent administered as per protocol 98-C-0037,
95-C-0025 or as described on standard government request form 2626 for invasive

Patients enrolled on Arm B must have had a tumor recurrence during or after receiving at
lest first line cytoreductive therapy for ESFT and AR. They are eligible for enrollment if
they have received up to two post-recurrence salvage regimens. Patients who have received
more than two post-recurrence salvage regimens are eligible if the peripheral CD4+T Cell
number is greater than 400 cells/mm(3).

Patients enrolled on Arm B must have disease which is evaluable for tumor response.
Evaluable disease is not required for patients receiving immunotherapy on Arm A since they
are eligible to receive salvage cytoreductive chemotherapy or radiation therapy following
tumor recurrence and prior to immunotherapy.

Patients must have not received cytoreductive therapy for at least 2 weeks and have
recovered from all of the acute toxicities related to any previous cytoreductive therapy.

Patients must have an ECOG performance status of 0, 1 or 2 (i.e. an activity level wherein
the patient is out of bed greater than 50% of the day or more) and a life expectancy of at
least 8 weeks.

Patients must have adequate renal function (serum Cr less than 1.5 mg/dl or Cr Cl. greater
than 60 ml/min./1.73 m(2)) and liver function (transaminases less than 3x normal,
bilirubin less than 2.0 mg/dl).

Patients will not be excluded based upon abnormal hepatic function which is related to
hepatic involvement by tumor.

Patients must have no major disorder of the pulmonary or cardiovascular system and have a
cardiac ejection fraction of greater than 40% as measured by radionuclide MUGA scanning or
a fractional shortening of greater than 27% as measured by echocardiography.

Patients must have adequate bone marrow function as measured by Hgb greater than 9.0 gm/dl
prior to large volume apheresis and Hgb greater than 8.0 gm/dl prior to immunotherapy
cycles, Plt greater than 50,000 mm(3) and ANC greater than 1.0 x 10(3) micro l.


Women who are pregnant or lactating.

Patients with human immunodeficiency virus infection due to confounding effects on immune
function. Patients with hepatitis B or hepatitis C infection.

Patients who require daily oral corticosteroid therapy for any underlying disease will be
excluded. Topical or inhaled corticosteroids are permitted.

Patients who are allergic to eggs, egg products, or thimerosal, or have a history of
Guillain-Bare syndrome may be enrolled on study but are ineligible to receive the
influenza vaccine.

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Crystal L Mackall, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Cancer Institute (NCI)


United States: Federal Government

Study ID:




Start Date:

December 1996

Completion Date:

October 2007

Related Keywords:

  • Ewing's Sarcoma
  • Rhabdomyosarcoma
  • Rhabdomyosarcoma
  • Ewing's Sarcoma
  • Immunotherapy
  • Tumor Vaccine
  • Fusion Protein
  • Rhabdomyosarcoma
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma



National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland  20892