Sarcoma Cancer is a rare disease, often misdiagnosed, often afflicting children and young adults.
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Allogeneic, Haploidentical NK cell Infusions For High Sarcoma Patients

Project Title: Allogeneic, Haploidentical NK cell Infusions For High Sarcoma Patients

Principal Investigator Name: Michael R. Verneris, Rank: Associate Professor Academic Dept: Pediatrics

Total funding requested: $96,600 – RIS 2011 Funding: $48,300 (additional match available)

Project Summary:

Patients with either Ewing sarcoma and rhabdomyosarcoma that have metastatic disease at diagnosis or who have relapsed after front-line therapy have a grim prognosis, with most dy ing from progressive disease. Our prior studies show that high dose chemotherapy and stem cell rescue can prolong survival (and even cure) some sarcoma patients. Recent studies also show that natural killer (NK) cells can recognize and kill sarcoma cells i n the laboratory and in animal models. Here we will test a novel combination of high dose chemotherapy, followed by NK cell infusion from a sibling or parent, followed by the subsequent "rescue" with the patient's own bone marrow.

The goals of this trial, which will be the first of its kind, are to test the safety and feasibility of using parental or sibling NK cells after high dose chemotherapy. We will also determine whether donor NK cells expand in the blood of patients (which is correlated with NK­ cell mediated antiluekemia activity) and the function of these NK cells, once infused into the patient. Lastly, comparisons of the NK cell treated patients will be made to identically treated patients who did not receive NK cells. The purpose of this grant is to obtain "seed" funding to prove feasibility, so that we can apply to larger funding agencies (i.e., NIH and NCI).

Name(s) of project collaborator(s), academic departments/schools, and role in the project:

  • Jeffrey Miller, MD: Holder ofNK cell therapy IND, Principal investigator of multiple NK cell protocols and collaborator.
  • Brenda Weigel, MD: Oncology and specialist in pediatric young adult sarcoma.