Dr. Sampol: "Pharmacological Immunotherapy and CAR-T Therapy Achieve Long Survival in Blood Diseases"
Dr. Antonia Sampol is a hematology specialist at the Juaneda Miramar Hospital, with a career that began at this center, a pioneer in private medicine for bone marrow transplantation in oncohematological diseases in the mid-1990s. She currently combines her private practice with her role as the Head of Hematology and the Bone Marrow and CAR-T Cell Transplant Unit at Son Espases University Hospital. In this interview, Dr. Sampol discusses the advancements in gene therapies for blood cancers, highlighting the significant leap brought by immunotherapy and CAR-T cells, which have achieved long survival rates and even cures in patients who previously faced more challenging prognoses.
At Juaneda Hospitals, particularly in this field of medicine, genetic and personalized diagnoses are conducted to ensure the highest efficacy and safety for patients.
—What is immunotherapy, and what advancements does it represent in hematological diseases?
Immunotherapy originates from bone marrow transplantation. The first immunotherapy, over 30 years ago, was allogeneic bone marrow transplantation, where healthy donor cells, specifically lymphocytes, attack the patient’s tumor. Based on this concept, numerous options have been developed against both solid and hematological (blood-related) tumors, which are now highly significant in cancer treatment.
Immunotherapeutic drugs are effective if the tumor expresses a protein that the natural immune system of the patient can target. This is also the principle behind CAR-T cell therapy, where we extract lymphocytes from the patient, modify them to specifically attack a tumor with specific markers. The most common therapies target the CD19 antigen in lymphomas, leukemias, and other lymphoid-lineage blood disorders. Additionally, new drugs and CAR-T therapies have been developed for multiple myeloma, targeting other tumor cell antigens. Other immunotherapy drugs disinhibit the immune system, restoring its anti-tumor surveillance function.
—What did traditional treatment for hematological tumors entail, and how has it evolved?
Traditional treatments always relied on chemotherapy, which, while effective, affected many other organs and caused severe side effects. Immunotherapy, whether with monoclonal or bispecific antibodies or genetically modified CAR-T cells, is a targeted therapy that specifically treats tumor cells, minimizing adverse effects on other body tissues.
This allows it to be used in older patients or those with conditions that would make bone marrow transplants or chemotherapy unfeasible. Unlike CAR-T cells, pharmacological immunotherapy also targets a tumor surface protein but stimulates the immune system to attack the tumor without using patient lymphocytes. These targeted therapies are much more specific and have shown spectacular efficacy, even in chemoresistant tumors where other therapies have failed.
—Are these new therapies suitable for all types of patients?
Understanding the tumor's biology is crucial. Depending on the type of tumor, different immunotherapies are used. Therapy must be personalized for each patient based on the markers expressed by their tumor. Accurate diagnosis is essential to optimize these treatments.
Significant progress has been made in understanding tumor biology. We now know that many factors surrounding the tumor enable it to grow and lead to cancer. By specifically targeting these mutations, we can stop tumor growth. Many new targeted treatments are being researched for both solid and hematological tumors.
—In which hematological diseases have these new therapies shown the most progress?
There are many examples. The first diseases treated with CAR-T therapy targeting the CD19 antigen were B-cell acute lymphoblastic leukemia in children, achieving cures that were previously unthinkable. These were followed by B-cell lymphomas. However, the first example of a cure with a targeted therapy occurred over two decades ago with chronic myeloid leukemia (CML).
In CML, which previously could only be cured through allogeneic bone marrow transplantation, identifying the driver mutation that causes the disease led to the development of drugs that inhibit this mutation, resulting in cures. Recently, CAR-T therapy for multiple myeloma, a disease with a median survival of three years with standard chemotherapy, has been approved in Spain. Thanks to these treatments, including CAR-T therapy and immunotherapy, we now see extended survival rates, with some patients potentially considered cured.
—Returning to the root of the problem, why doesn’t the immune system detect these tumors?
One of the proposed causes of cancer development is that the immune system lowers its defenses, allowing tumors to develop. Under normal and ideal circumstances, a healthy immune system attacks abnormal cells and prevents them from proliferating.
When the immune system fails and allows some cells to deviate from their programmed path, tumors can develop. This failure can be influenced by environmental factors such as smoking or pollution. Genetic factors, including DNA mutations that accumulate over a lifetime—especially with aging—also facilitate the development of certain diseases.
Unrepaired mutations, particularly with age, are strongly linked to hematological tumors. However, the analysis is much more complex, and the origins of neoplasms require a more personalized approach.
—This explains why personalized, "tailored" medicine is increasingly emphasized.
Exactly. Medicine is becoming more personalized, based on a comprehensive study of the biological and genetic characteristics of tumors, as well as the patient’s physical condition and characteristics.
In my practice, it’s impossible to decide on treatment without performing an accurate diagnosis, including precise histological and immunohistochemical studies, a panel of specific genetic mutation markers, and a thorough evaluation of the patient’s physical condition and comorbidities.
A good diagnosis is increasingly crucial for adapting treatment and making it more effective. Detecting genetic mutations is fundamental in diagnosis.
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