CAR-T Therapy in Pediatrics: Treating Childhood Cancers in Barcelona

In recent years, CAR-T therapy has become a major breakthrough in modern oncology, especially in pediatrics. This approach has opened new possibilities for treating children with severe forms of blood cancer who do not respond to standard therapies. Today, CAR-T cell therapy is actively used in leading hospitals around the world, including in Barcelona, showing promising results.

One of the hospitals within the Barcelona International Hospitals group is actively engaged in both research and clinical application of this therapy — SJD Barcelona Children’s Hospital.

This hospital is one of the top pediatric centers in Europe and offers a comprehensive, multidisciplinary approach to treating complex and rare diseases in children (from birth to age 18) and pregnant women. Hundreds of research projects are conducted here, including those focused on CAR-T therapy, with the goal of developing new treatments, improving quality of life, and offering a chance of recovery even in the most challenging clinical cases.

What Is CAR-T Therapy?

CAR-T therapy is a form of immunotherapy that uses a patient’s own T-lymphocytes, which are genetically modified to recognize, attack, and destroy cancer cells in a targeted way. The main idea is to boost the body’s natural defenses and “teach” the immune system to detect cancer cells.

The process begins with apheresis — a procedure to collect T-cells from the patient’s blood. In the laboratory, these cells are genetically engineered to express a special receptor (CAR – chimeric antigen receptor) on their surface. This receptor enables the T-cells to identify and destroy cancer cells, especially those associated with leukemia and lymphoma.

Stages of CAR-T Cell Therapy

The treatment process typically involves several key stages:

1. T-cell collection: Through leukapheresis, T-lymphocytes are separated from the patient’s blood.
2. Genetic modification: In the lab, the cells are modified to carry the gene encoding the CAR receptor.
3. Cell cultivation: The modified T-cells are multiplied under controlled conditions.
4. Infusion: The prepared cells are infused back into the patient intravenously.
5. Monitoring: The child is closely monitored for response and potential side effects.

Depending on the clinic and the child’s condition, the entire process may take anywhere from a few weeks to a couple of months.

Side Effects of CAR-T Therapy

Although CAR-T therapy is highly effective, it can be associated with several side effects, such as:

• Cytokine Release Syndrome (CRS): An acute inflammatory response that may involve fever, low blood pressure, and organ dysfunction.
• Neurological complications: Confusion, seizures, headaches.
• Immunosuppression and increased vulnerability to infections.

Despite these risks, most side effects can be managed using modern medical protocols.

CAR-T Therapy for Myeloma and Other Pediatric Conditions

Currently, the primary indication for CAR-T therapy in children is acute lymphoblastic leukemia (ALL). However, ongoing research is exploring its effectiveness in multiple myeloma, lymphoma, and neuroblastoma.

While conclusive results have yet to be achieved for these conditions, early outcomes are encouraging. The main objective of these studies is to make CAR-T therapy accessible to a broader range of young patients. There is hope that in the near future, CAR-T will no longer be seen as a “last resort” but rather a standard option within pediatric oncology.

The Future of CAR-T Therapy in Pediatrics

The development of CAR-T therapies for childhood cancers offers great promise. New generations of CARs are being developed, delivery methods are improving, and toxicity is being reduced. In the future, this therapy could be used not only for leukemia, but also for brain tumors, sarcomas, lymphomas, and even certain autoimmune diseases in children.

CAR-T therapy in pediatrics represents an innovative approach to cancer treatment. Despite its high cost and some risks, its effectiveness makes it one of the most promising directions in pediatric hematology and oncology. In the coming years, we can expect broader indications, increased accessibility, and reduced costs — all of which could help save the lives of thousands of children worldwide.

CAR-T Therapy at SJD Barcelona Children’s Hospital

In pediatric practice, CAR-T therapy is primarily used to treat acute lymphoblastic leukemia (ALL) — the most common form of blood cancer in children. The method has proven especially effective in cases of relapse or resistance to standard chemotherapy.

For example, a clinical trial of CAR-T ARI therapy was conducted at SJD Barcelona Children’s Hospital, within its Pediatric Cancer Center. One of the first patients was a 7-year-old girl diagnosed with B-cell lymphoblastic leukemia.

The first study began in 2023, and to date, around 30 patients from across Spain have participated. Numerous reports from doctors and parents have shown that over 80% of children treated with CAR-T therapy achieved remission, highlighting not only a better quality of life but also a significant chance of full recovery.

CAR-T Research at barnaclínic+ / Grupo Hospital Clínic

In addition to SJD Barcelona Children’s Hospital, barnaclínic+ / Grupo Hospital Clínic has played a pioneering role in the development of academic CAR-T therapies in Spain and Europe. Two original treatments have been developed here:

• ARI-0001 – for acute lymphoblastic leukemia
• ARI-0002h – for multiple myeloma

Both therapies have been approved by the Spanish Agency of Medicines and Medical Devices (AEMPS) and have shown high efficacy in patients who did not respond to conventional treatments.

For more information about Barcelona International Hospitals and our services, please contact us!