Cell replacement therapy

Allogeneic hematopoietic stem cell transplantation (HSCT), a curative therapy for patients with life-threatening hematological disorders, is said to be the most successful cell replacement therapy developed to date, with more than 40,000 transplantations performed per year worldwide. Cord blood (CB), an ideal starting point for allogeneic HSCT, can be collected and banked without compromising the donor and is more easily matched to recipients relative to bone marrow–derived material. However, the limited number of stem cells available in a single cord unit limits their use to pediatric patients and can cause delayed engraftment.

Initially, Plasticell approached Anthony Nolan to source cord blood cells for the research and development of the stem cell product from its cord blood bank. Further discussions led to the realization that multiple synergies could be found between the two organizations. The common goal is to develop products that will ultimately improve the outcomes of stem cell transplant patients.

According to Dr. Diana Hernandez, head of immunotherapy at Anthony Nolan, “Despite our success in connecting donors and recipients of HSCT through the Anthony Nolan stem cell register, we currently fail to find a suitable match for some of the patients waiting for a stem cell transplant. We see Plasticell’s expanded cord blood product as an exciting opportunity to save the lives of these patients and to improve the treatment outcome for many more blood cancer patients.”

Dr. Yen Choo, founder and executive chairman of Plasticell, added, “The partnership with Anthony Nolan and its network of transplant centers and surgeons will accelerate clinical translation of this therapy by providing world-class clinical expertise, access to cord blood banking and experience in cord blood manipulation, and importantly will greatly facilitate patient recruitment which can be a bottleneck in clinical development of expanded cord blood products.”

Plasticell has developed a small-molecule driven, GMP-compliant method of expanding hematopoietic stem cells from cord blood, bone marrow and peripheral blood. The project is part of Plasticell’s hematopoietic cell therapy portfolio, which includes the expansion of hematopoietic stem cells and the manufacture of various blood cell types from pluripotent cell sources. In the past year Plasticell has announced two separate academic collaborations in the hematology space, one with the University of Edinburgh and one with Kings College London, as well as a larger commercial collaboration with GlaxoSmithKline.

In this agreement, Plasticell will be responsible for GMP manufacturing and clinical development of the expanded cord blood product, as well as all commercialization of the product. The company’s patented formulation for the ex-vivo expansion of human hematopoietic stem cells from cord blood is currently in late preclinical testing. The next step in the development of this product is to apply for clinical trials authorization, tapping into the expertise at Anthony Nolan for strategic advice and help with the supply of cord blood cells.

With more than 40 years of experience in hematopoietic stem cell transplants, Anthony Nolan set up the first unrelated bone marrow donor register in the United Kingdom and led the way globally in increasing the number of unrelated donors to the combined worldwide register. The company’s groundbreaking research in HLA typing and matching “has already vastly improved the outcomes for many transplant patients, and it is now the gold standard across the world,” according to Hernandez.

Anthony Nolan has a substantial network of clinical professionals who understand the clinical landscape dedicated to the care of transplant patients and expertise in the logistics of cell procurement (cord blood bank) and delivery to patients. The organization uses its stem cell register to match potential stem cell donors to patients with blood cancer and blood disorders in need of a stem cell transplant. It also carries out pioneering research to increase stem cell transplant success, and supports patients through their transplant journeys.

“We believe the partnership will allow both parties to reach the common goal of improving the outcomes of patients undergoing stem cell transplants to be reached much faster and more efficiently than it otherwise would have,” Choo remarked. “We hope it will also lead to further research and development in other cell therapy products of mutual interest.”

“HSCT can be used to treat over 70 indications, ranging from cancer to metabolic diseases. The commercial potential is to significantly increase the market for HSCT by allowing cord blood to be used as a source of HSCs,” he concluded.