New Cancer Research Suggests More Accessible Personalized Treatments On the Horizon

It has only been about a year and a half since the United States Food and Drug Administration approved the first CAR-T cell therapies.  These are the acute lymphoblastic leukemia treatment Kymriah from Novartis and Gilead-owned Kite Pharmaceutical’s Yescarta, which treats certain types of large B-cell lymphoma (non-Hodgkin). 

Both of these treatments are autologous therapies: they are patient-specific.  Essentially, these treatments use the patient’s own T-cells in the blood to help recognize and attack cancer cells.  The therapy actually involves collected these t-cells from the blood, preserving them, and shipping them to the manufacturer where they are genetically engineered for cancer warfare, effectively.  These modified T-cell receptors are called chimeric antigen receptor T-cells (or CAR-T cells), and are reintroduced to the patient.  

This type of personalized therapy, of course, has revolutionized cancer treatment as well as healthcare as a whole.  However, the treatment does still have its limitations, mostly in the fact that each treatment must be personalized to the patient (and cannot be used by any other patient). 

But the labor-intensive work is also an obstacle to widespread use.  Personalizing T-cells significantly increases the price of the treatment, whose median price is around $425,000. 

To overcome these obstacles, researchers are now moving to develop the next generation of CAR-T therapies for new allogenic—“off the shelf”—treatments which drugmakers can mass produce to be more accessible. 

A new study from researchers at the University of California, Los Angeles transformed pluripotent stem cells into T-cells through artificial thymic organoids, which essentially mimic the body’s natural thymus, where T-cells originate.  The study demonstrates that it could be possible to use stem cells to develop the T-cells necessary to facilitate these treatments. 

Lead study author Gay Crook explains, “What’s exciting is the fact that we start with pluripotent stem cells.” The director of the UCLA Jonsson Comprehensive Cancer Center Cancer and Stem Biology Program goes on to say, “My hope for the future of this technique is that we can combine it with the use of gene editing tools to create ‘off-the-shelf’ T-cell therapies that are more readily available for patients.”

In addition, study co-first author Amelie Montel-Hagen notes, “Once we create genetically-edited pluripotent stem cell lines that can product tumor-specific T-cells in artificial thymic organoids, we can expand those stem cell lines indefinitely.”

The results of this study have been published in the journal Cell Stem Cell.