Researchers conduct first-in-man clinical trial for new stem cell technology

Reviewed by Emily Henderson, B.Sc.May 12 2021

Institutes and centres under the SingHealth Duke-NUS Academic Medical Centre (AMC) are joining hands together with Singapore's first private cord blood bank, Cordlife Group Limited (Cordlife), to test a novel technology that expands the number of blood-forming stem cells from stored umbilical cord blood (UCB) in a first-in-man study in Singapore. This is the first time a home-grown UCB cell therapy is being tested on humans. The technology has the potential to increase treatment options for patients suffering from blood cancers or blood-related conditions.

One of the most effective treatments for patients with blood cancers, such as leukaemia, lymphomas and hereditary blood-related conditions, such as thalassaemia is to transplant haematopoietic stem and progenitor cells (HSPCs) harvested from UCB. However, the current applications of HSPCs are limited as the number of HSPCs that can be harvested from a UCB is typically low, yielding few useable units for adult transplant patients. Administering a UCB with low cell count often results in slower recovery and greater susceptibility to fatal infections.

The technology to be tested in this trial uses a laboratory-synthesised compound called C7 for ex-vivo expansion of HSPCs, which, in turn, enables specific cell therapy products to be manufactured for use in patients.

"Based on pre-clinical studies, C7 appears to be able to expand banked cord blood stem cells to generate sufficient quantity for adult transplant patients while maintaining their quality," said Professor William Hwang from Duke-NUS' Cancer and Stem Cell Biology Programme. Prof Hwang, who heads the SingHealth Duke-NUS Cell Therapy Centre, is also medical director of the National Cancer Centre Singapore (NCCS) and a senior consultant with the Department of Haematology at Singapore General Hospital (SGH).

As a haematologist, he has dedicated his career caring for patients with blood cancers, one of the most common causes of cancer deaths, accounting for approximately 720,000 deaths globally every year. This accounts for 7 per cent of all cancer deaths, according to GLOBOCAN 2018 statistics.

Prof Hwang and his laboratory team, together with researchers from NUS, discovered C7's ability to increase the numbers of blood-forming stem cells from UCB. "The effect of C7 on blood stem cells also seems to be better than anything else we have used in other clinical trials for cord blood expansion."

Leading this clinical trial is Dr Francesca Lim, a consultant at SGH's Department of Haematology and SingHealth Duke-NUS Blood Cancer Centre, who said, "The ability to expand UCB HSPCs for clinical use provides an opportunity to overcome cord blood transplants' short-term disadvantages of low total cell dose.

This is an important step forward to improve treatment outcomes for transplant patients, especially those who rely on umbilical cord blood as the only source of grafts due to the lack of a fully matched bone marrow or peripheral blood stem cells."

Duke-NUS and SingHealth manage the patent for the application of C7 in expanding UCB HSPC through their Joint Centre for Technology and Development (JointCTeD), which sealed the industry partnership with Cordlife to enable this clinical trial.

Associate Professor Chris Laing, Senior Associate Dean for Innovation and Entrepreneurship at Duke-NUS, said:

This technology has the potential to significantly improve patients' treatment outcome. We are privileged to work with the inventors and industry partners to ensure this promising technology continues to advance. This landmark clinical trial - the first clinical trial of a Singapore stem cell expansion technology from UCB - is an example of our commitment in translating cutting-edge innovations into better patient care."

In preclinical studies, UCB treatment has been shown to be immunologically superior compared with other cell and gene therapies given its greater tolerance for human leucocyte antigen mismatch, reduced graft versus host disease and lower incidence of relapse.

"We are very excited to be a part of this revolutionary clinical trial in Singapore. This project is significant to us because different players across the ecosystem are coming together to create a paradigm shift in cellular therapies. Once the HSPC expansion technology is proven safe and effective, more patients can rely on cord blood for treatment," said Ms Tan Poh Lan, Group CEO and Executive Director of Cordlife.