Cell Therapy

Donor-Derived NK Cells Safe and Efficacious in Relapsed and Refractory AML

Cell Therapy Study

Scientists have found a way to use special cells called NK cells to help treat a type of blood cancer called AML. They can now grow these cells outside the body and give them to patients in multiple doses, making the treatment safer and more effective. Read more about how researchers have used their approach for growing large numbers of NK cells to allow repeated dosing for a more effective and less toxic treatment in Pediatrics Nationwide.

Open Cell Therapy Clinical Studies

Acute Myeloid Leukemia

Researchers Studying Safety and Efficacy of Expanded, Universal Donor Natural Killer Cells for Relapsed, Refractory AML

The goal of this study is to determine the safety and recommended phase II dose of adoptive NK cell therapy using UD-NK cells.

Researchers Studying Donor NK Cell Infusions in patients with High-Risk AML Undergoing a HaploHCT

The goal of this study is to test the safety and effectiveness of a new treatment of infusing natural killer (NK) cells into the patient’s body to help fight AML.

Hematopoietic Stem Cell Transplantation

TB19DHCT: Depletion of αβT Cells and CD19+ B Cells in Alternative Donor Stem Cell Transplant for Children and Young Adults with Blood Cancers

Help researchers study the one-year overall survival of patients undergoing allogeneic HSCT using the T-Cell Receptor (TCR) αβ/CD19+ depleted platform and grafts from alternative donors (MUD, MMUD and haploidentical).

CSIDE: A Phase II Trial Comparing Low and Moderate Busulfan Doses in Infants with Severe Combined Immunodeficiency (SCID) Undergoing TCRαβ+/CD19+ Depleted Transplant, by the PIDTC and PBMTC

Help researchers study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation.

Infectious Diseases

Pilot Study of Donor Virus-Specific T-Cells to Treat Cytomegalovirus (CMV) or Adenovirus (AdV) Infections After Solid Organ or Stem Cell Transplant

Help researchers assess how safe and practical a new treatment (anti-viral cytotoxic T-lymphocytes, or CTLs) is for patients who have had a solid organ transplant (SOT) or hematopoietic cell transplant (HCT) and are dealing with an infection or reactivation of cytomegalovirus (CMV) or adenovirus (AdV).

Pilot Study in the Treatment of Refractory Adenovirus (ADV) Infection with Related Donor ADV Cytotoxic T-Lymphocytes (ADV-CTLs) in Children, Adolescents and Young Adult Recipients

The goal of this study is to find out if we can safely give the patient special immune cells called cytotoxic T-cells (CTLs). These cells are made from the patient’s stem cell donor's cells or a closely matched family donor and grown in the lab. These immune cells are designed to help fight adenovirus (ADV) infection.

A Pilot Study on Treating Resistant Epstein-Barr Virus (EBV) Infections in Children, Adolescents, and Young Adults Using Donor Immune Cells Specifically Targeting EBV

The goal of this study is to see if we can safely give the patient special immune cells called cytotoxic T-cells (CTLs). These cells are made from the patient’s stem cell donor or a closely matched family donor and grown in the lab. These immune cells are designed to help fight Epstein-Barr Virus (EBV) infection.

Neuroblastoma

Researchers Studying Universal Donor NK Cells in Combination with IV Drug Therapy in Patients With Relapsed or Refractory Neuroblastoma

Help researchers study the safety, tolerability, and efficacy of a fixed dose of UD TGFβi NK cells in combination with standard irinotecan, temozolomide, and dinutuximab IV therapy.

Neuro-Oncology

Early-Phase Study of Targeted Natural Killer Cell Therapy for Children and Young Adults With Recurrent, Progressive or Refractory Brain Tumors

The purpose of this study is to see if it is safe and feasible to treat malignant brain tumors with donor natural killer (NK) cells. The hope is to see if the NK cells are effective against the tumors and improve quality of life.

PBTC-059: Phase 1 Trial of HER2-specific CAR T Cells for Children with Hard-to-Treat or Returning Ependymoma

Help researchers evaluate the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells).

Sarcoma

Researchers Studying Donor NK Cell Infusions Combined With IV Medications in Patients With Relapsed or Refractory Pediatric Sarcomas

Help researchers determine if adding infusions of a type of immune cell called a “natural killer,” or NK cell to the sarcoma chemotherapy regimen GEM/DOX (gemcitabine and docetaxel) is safe and can improve outcomes in people with childhood sarcomas that have relapsed or not responded to prior therapies.