Pluristem Therapeutics Inc., a leading developer of placenta-based cell therapy products, announced it has advanced its phase I trial of PLX-R18 cells to treat insufficient hematopoietic recovery following hematopoietic cell transplantation (HCT) by contracting with a leading global clinical research organization (CRO).

The trial received clearance from the US Food and Drug Administration earlier this year and enrollment is planned to begin in the coming months. The multi-center, open-label, dose-escalating phase I trial will evaluate the safety of intramuscular injections of PLX-R18 cells in 30 patients with incomplete hematopoietic recovery persistent for 6 months or more after HCT.

“Data from this trial will inform the potential of PLX-R18 to treat a wide range of indications including blood cancers and radiation therapy-related blood diseases. The CRO we chose has extensive experience working with leading pharmaceutical and biotech companies to successfully manage clinical trials. We are excited to move forward to bring clinical sites online and begin enrolling patients,” said Pluristem chairman and CEO Zami Aberman.

Data from multiple preclinical studies conducted by world-class research institutions, including the US National Institutes of Health and Hadassah Medical Center, have shown that PLX-R18 cells secrete a range of specific proteins that support the regeneration of bone marrow and the recovery of its ability to produce normal amounts of all three blood cell types. PLX-R18 is designed to be administered without matching, and using a standard syringe to inject the cells intramuscularly.

Hematopoietic cell transplantation is a standard treatment for a range of conditions, including malignant diseases such as multiple myeloma, non-Hodgkin’s lymphoma, Hodgkin’s disease, and acute myeloid leukemia, as well as non-malignant diseases and autoimmune disorders such as aplastic anemia and thalassemia. The hematopoietic cells for HCT can come from a donor (allogeneic) or from the patient (autologous), and can be harvested from peripheral blood, bone marrow or umbilical cord blood.

In a number of cases, complete hematopoietic recovery following HCT is not reached, and patients are at increased risk of bleeding, infection, poor general function and death for months afterwards. Current treatments include administration of factors stimulating growth of specific blood cell types, such as granulocyte-colony stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), and erythropoietin. However, a significant number of patients do not respond to growth factors and may require frequent transfusions, which expose them to transfusion-related risks such as allo-sensitization and infections, without providing a curative solution. These are also associated with significant costs.