New Way to Engineer CAR T-Cells Could Make it More Accessible

Recently, an article in Nature shed light on the possibility of making CAR T-cell therapy quicker, more affordable, and more widely available for people with blood cancer by engineering the cells directly inside the body instead of in a lab.

What is CAR T-cell therapy? 

Chimeric antigen receptor (CAR) T-cell therapy is a type of blood cancer treatment. It modifies a person’s own immune cells (T-cells) to better recognize and eliminate cancer cells. 

Currently, the cells are engineered in a lab. This process can take weeks. It can also be expensive, costing up to $500,000 per treatment before insurance. First, the patient’s blood is collected, and T-cells are separated out. The T-cells are then sent to a lab to be modified with the CAR protein. After the modified cells have been grown and multiplied in large numbers, they are frozen and shipped back to the hospital. Then the patient can undergo an infusion of their own cells.

Factors like engineering time and cost represent barriers for many patients who are eligible for this treatment that could extend their lives. 

Engineering T-cells inside the body to make CAR-T more accessible

To address these issues, scientists and biotechnology companies are exploring “in vivo” CAR T-cell therapy. This method delivers the CAR gene directly into the patient’s immune cells while they’re still inside the body using engineered viruses or nanoparticles to carry the CAR instructions straight to the T-cells. 

If successful, this approach could make treatment quicker and eliminate the need for pre-treatment chemotherapy. Pre-treatment chemotherapy can be a barrier to receiving CAR-T because of the side effects and impact on the immune system. In vivo CAR-T could result in a more affordable, readily available therapy.

One of the biggest challenges with in vivo therapy is making sure only T-cells are modified. In the lab, T-cells have been separated from other cells. In the body, it is not as easy to avoid modifying other cells. Companies are developing different strategies to do this. For example:

• Interius BioTherapeutics uses a delivery method that targets a protein found only on T-cells and similar immune cells. This is still in early development. 
• Umoja Biopharma uses a lentiviral vector (a modified virus) that recognizes three specific receptors on T-cells, improving targeting accuracy. It is still in early phases. 

Potential safety improvements with RNA-based therapies

Another emerging approach uses RNA, instead of permanently altering the DNA of T-cells. Companies like Capstan Therapeutics and Orna Therapeutics are using nanoparticles to deliver RNA that temporarily instructs cells to produce the CAR protein.

This method may be safer because it doesn’t make permanent genetic changes. If a patient experiences severe side effects, the therapy can be stopped, and the CAR protein will quickly disappear from the body. This contrasts with DNA-based approaches, which may remain active for years.

What to expect

Human trials of some in vivo CAR T-cell therapies are already underway. Interius has begun testing its therapy for people with non-Hodgkin lymphoma. While the first two patients didn’t respond to lower doses, a third patient who received a higher dose showed a significant drop in cancer cells within 6 days.

Umoja and EsoBiotec are also conducting early trials in blood cancers, with one patient reportedly having no detectable cancer a month after treatment. These early results are being closely watched, and more data is expected by the end of 2025.

Waiting for promising results

If these newer approaches succeed, in vivo CAR T-cell therapy could become more accessible, safer, and faster for patients. This would be especially important for patients who can’t undergo the current CAR-T process.

While this new type of therapy still needs further research in clinical trials, the fact that many companies and researchers are investing in it shows that simplifying and improving CAR T-cell therapy is a major goal in cancer treatment development.

Stay tuned for more articles on treatment advances with the HealthTree News site! 

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• Cancer-fighting immune cells could soon be engineered inside our bodies