UCLA starts Phase I stem cell trial for cancers with NY-ESO-1 tumour marker


The University of California, Los Angeles (UCLA) has started a Phase I clinical trial to evaluate new genetically engineered blood-forming stem cells to treat cancers such as melanoma and sarcoma with a specific tumour marker called NY-ESO-1.

Led by Dr Antoni Ribas, UCLA researchers at the Eli and Edythe Broad Centre of Regenerative Medicine and Stem Cell Research will investigate the ability of the stem cells to produce T-cells.

Dr Ribas is a professor of the medicine, surgery, and molecular and medical pharmacology departments in the UCLA David Geffen School of Medicine.

With a $20m grant from the California Institute for Regenerative Medicine (CIRM), the trial will enrol patients suffering from metastasised tumours and with HLA-A2.1 type of immune system.

To provide short and long-term immune response, the trial will use a dual approach, where initially mature T-cells and blood-forming stem cells will be collected from the blood of each subject.

The cells will then be inserted with a gene for a receptor to NY-ESO-1 for generating a continual supply of T-cells programmed to specifically identify and destroy the cancer with NY-ESO-1.

As these cells take time to mature, the researchers also intend to provide immediately available cancer-fighting cells by genetically modifying a patient’s own mature T-cells to detect the NY-ESO-1 marker.

The genetically modified blood-forming stem cells and mature T-cells will be administered in a single transplant.

"Engineering a patient’s own blood-forming stem cells to produce a continual supply of the T-cells needed to attack cancer for a prolonged period could help us overcome the challenges and limitations with the current method."

UCLA David Geffen School of Medicine medicine assistant professor Dr Hu-Lieskovan said: “Clinical trials using adoptive T-cell immunotherapy have shown remarkable results in some patients.

“Engineering a patient’s own blood-forming stem cells to produce a continual supply of the T-cells needed to attack cancer for a prolonged period could help us overcome the challenges and limitations with the current method.”

To ensure safety during the trial, the researchers intend to carry out regular scans using positron emission tomography (PET) to monitor the functioning of organs and tissues in the body.

In case of any abnormality, a drug will be administered to specifically destroy the engineered cells.


Image: Dr Antoni Ribas in the lab. Photo: courtesy of UCLA Jonsson Comprehensive Cancer Centre.