In a significant breakthrough for leukaemia treatment, the U.S. Food and Drug Administration (FDA) has recently approved the use of a CAR-T cell therapy derived from umbilical cord blood for clinical trials. This approval marks a major milestone as it is the first-ever therapy of its kind to gain FDA approval, offering a new avenue for patients battling acute lymphoblastic leukaemia (ALL).
What Is Acute Lymphoblastic Leukaemia?
Acute lymphoblastic leukaemia is a fast-growing type of blood cancer that primarily affects white blood cells called lymphocytes. In ALL, the bone marrow produces an excessive number of immature cells known as lymphoblasts, which multiply rapidly but fail to mature into healthy lymphocytes. These immature cells crowd out healthy blood cells, leading to issues such as a weakened immune system, anaemia, and bleeding problems due to reduced platelet production.
ALL is most commonly diagnosed in children but can also affect adults. The cancer can spread to other parts of the body, including the brain, spinal cord, liver, and spleen, which makes treatment particularly urgent.
What Is CAR-T Cell Therapy?
Chimeric Antigen Receptor T-cell (CAR-T) therapy is an innovative treatment that uses a patient’s own T cells to fight cancer. These T cells are genetically modified in a lab to express a chimeric antigen receptor, which enables them to recognize and target cancer cells.
In traditional CAR-T therapy, the patient’s own T cells are extracted, modified, and then reinfused into the patient. However, this process can take several weeks, which is a major challenge for fast-progressing diseases like ALL. By the time the modified T cells are ready, the cancer may have advanced too far.
The Game-Changing Therapy: Cord Blood-Derived T Cells
The newly approved therapy revolutionizes CAR-T treatment by using T cells derived from allogeneic (donor) umbilical cord blood. These cells are in a more naïve state compared to adult T cells, which allows them to self-renew more effectively and have a lower risk of causing an immune response in the recipient. The therapy is designed to be “off-the-shelf,” meaning it can be prepared in advance and used immediately when needed, eliminating the waiting time associated with traditional CAR-T treatments.
Following promising results from initial trials, the therapy has now been cleared by the FDA for clinical trials in the United States. Researchers are also testing this therapy in other countries, with clinical trials underway in China for the treatment of lupus.
Global Advancements in Cord Blood-Derived Treatments
The use of cord blood in CAR-T therapies is not limited to one particular research center. At the Fondazione Tettamanti research centre in Monza, Italy, scientists are working on a similar treatment that uses T cells derived from cord blood. They modify the T cells using a special DNA sequence called a transposon to create CAR-enabled cytokine-induced killer cells (CARCIK). Initial trials have shown positive results, and research continues for the treatment of both ALL and non-Hodgkin lymphoma.
The Future of Cord Blood in Medical Treatments
The ability to use cord blood for CAR-T therapy opens up exciting possibilities for more targeted, personalized treatments. As the cells derived from cord blood are less likely to trigger an immune response, they hold significant potential for broader use, even for distant relatives who might not be an exact match. The availability of cord blood from public and private banks further enhances the accessibility of these treatments.
Public cord blood banks, while valuable, may not always offer a perfect match when needed because they rely on donations from a limited number of hospitals. Storing cord blood privately, on the other hand, ensures that a family has access to their own cells in the event of medical need. In fact, stored cord blood has a 25% chance of being a perfect match for a sibling, with a 50% chance of being a partial match.
With the FDA’s approval of this groundbreaking CAR-T cell therapy derived from umbilical cord blood, there is new hope for those battling acute lymphoblastic leukaemia. This development not only marks an exciting step forward in cancer treatment but also underscores the potential of cord blood in providing life-saving therapies. As research in this area continues, we may see even more innovative treatments that rely on cord blood to help save lives.
To learn more about how cord blood can be preserved for future medical use and its potential in treatments, request a free guide today.
References
- LemanBio (2023). Leman Biotech and Ucello Therapeutics enter into Comprehensive Strategic Collaboration to Advance Universal CAR-T Development. Leman Bio News
- BusinessWire. (2025). VectorBuilder Powers FDA IND Approval of World’s First Umbilical Cord Blood-Derived Allogeneic CAR-T Product. BusinessWire
- Cancer Research UK (2021). What Is Acute Lymphoblastic Leukaemia (ALL)? Cancer Research UK
- Cancer Research UK (2021). About childhood acute lymphoblastic leukaemia (ALL). Cancer Research UK
- Cancer Research UK (2021). CAR T-cell therapy. Cancer Research UK
- Leukemia & Lymphoma Society. Chimeric Antigen Receptor (CAR) T-Cell Therapy. Leukemia & Lymphoma Society
- Cerati, F. (2025). Dal cordone ombelicale le cellule modificate per trattare la leucemia. Il Sole 24 Ore. Il Sole 24 Ore
- NHS Blood and Transplant. Cord Blood Bank – Where can you donate? NHS Blood and Transplant