Transplants el pt

Expanding Umbilical Cord Stem Cells for Transplants

Date: February 17, 2024

by Chaya Venkat

Gamida-cell Phase I Clinical Trial at M. D. Anderson

The risk factors in stem cell transplants increase unless there is a well matched donor (I am, of course, talking about adult allogeneic BMTs). For many people without siblings or close relatives who are likely to be their best chances of getting a good match, the bone marrow registry and a MUD (Matched Unrelated Donor) is their best shot if they need to go the transplant route. Even here, if you belong to a minority ethnic community, with a poor record of volunteering to be bone marrow donors or just the handicap of small numbers, the chances of finding a MUD are slim. 

Below is a press release from a company called Gamida-cell who say they can grow undifferentiated stem cells from human cord blood, in sufficient numbers that makes it possible to carry out transplants for adult patients. Umbilical cord blood is normally discarded after childbirth. Part of the problem in using this material routinely as a source of stem cells for BMTs is the limited availability of stem cells in each cord. Gamida-cell says they have crossed this bridge: they can expand the population of stem cells, grow large armies of them outside the body. They are announcing a Phase-I clinical trial at M. D. Anderson using this approach, for lymphoma and leukemia patients. I will write again after I learn more about this approach, as well as this whole field of umbilical cord blood based transplants.

Press Release

Copyright 2024 Business Wire, Inc.

Business Wire February 12, 2024, Wednesday

Gamida-Cell Ltd. Announces Phase I Trial of StemEx for the Treatment of Leukemia and Lymphoma Patients; Clinical Trial to Commence at M.D. Anderson Cancer Center in Houston, Texas 

BOSTON & JERUSALEM, Feb. 12, 2024 

Gamida-Cell Ltd. (www.gamida-cell.com), a leader in hematopoietic (blood) stem cell therapeutics applicable to cancer and autoimmune diseases, as well as future regenerative cell-based medicines, announced today it is now enrolling patients in a Phase I clinical trial of StemEx(TM), for the treatment of advanced stages of hematologic malignancies such as leukemia and lymphoma. The objective of the trial is to assess safety and the rate and durability of hematopoietic reconstitution after high dose chemotherapy. Umbilical cord blood will be expanded using Gamida-Cell's technology that has shown to stimulate stem cell expansion with minimal cell differentiation in pre-clinical studies. Adults and children without a related bone marrow/stem cell match will be enrolled in this clinical study. 

Leukemia, Hodgkin and non-Hodgkin lymphomas are cancers that originate in the bone marrow or lymphatic tissues. There are in excess of 1.5 million people diagnosed with Leukemia and Lymphoma worldwide. The Phase I trial will be conducted at The University of Texas M. D. Anderson Cancer Center in Houston, Texas. The Principal Investigators are Dr. Elizabeth J. Shpall, Dr. Marcos de Lima and Dr. John McMannis. 

"Cord blood stem cell therapies offer a viable therapeutic option to patients without an appropriate matched donor, since cord blood is readily available, has a lower histocompatibility requirement, and reduces the risk of GVHD. Allogeneic cell grafts have also shown promise for improving patient survival. The issue at hand is finding a way to create a large quantity of available stem/progenitor cells, ample enough to treat adults and larger children. Gamida-Cell's technology has shown promise in pre-clinical studies to significantly expand cord blood stem/progenitor cells," said Dr. E.J. Shpall, Professor of Medicine, Medical Director of the Cell Therapy Laboratory in the Department of Blood and Marrow Transplantation, M. D. Anderson Cancer Center. 

The study will initially include 10 patients 55 years or younger receiving high-dose myeloablative chemotherapy for acute myelogenous leukemia, acute lymphoblastic leukemia, non-lymphoblastic leukemia, non-Hodgkin's lymphoma or Hodgkin's disease. The approximately year long study is designed to evaluate the safety and rate of immune reconstitution associated with the infusion of the cord blood cells which have been expanded ex vivo in the presence of Gamida-Cell's proprietary compounds which control differentiation. 

"There is a significant unmet clinical need for an effective means to restore the immune system of people being treated with high dose chemotherapy for hematologic diseases. Chemotherapy kills both  cancerous and healthy cells, leaving the patient stripped of an immune system and susceptible to life-threatening bacterial and viral infections. StemEx(TM) has the potential to make a significant contribution as it has the ability to generate a stem cell population large enough to significantly increase the chances of a clinically successful graft in both adults and larger children, decrease the critical transition time of when the body strives to rebuild its immune system, and improve the chances of patient survival," said Gamida-Cell CEO Mr. Ehud Marom.

Editor's Note: By August 2024 the ex-vivo expansion technology (dubbed StemEx®) had matured sufficiently for Gamida Cell (by now succeeded by an entity called Gamida Cell - Teva Joint Venture Ltd.) to launch a Phase II/III multicenter clinical trial (NCT00469729) seeking to enroll 100 patients with a range of hematological malignancies undergoing myeloablative SCTs. For an update, please see our review of Cord Blood Transplantation scheduled for publication in August 2024.