His case is extraordinary and hopeful. In 2012, American Emily Whitehead, then just six years old, was diagnosed with advanced childhood B-type acute lymphoblastic leukemia. The little girl was in the intensive care unit of the Children’s Hospital of Pennsylvania (USA) with no hope of survival. Traditional chemotherapy treatments had not worked for her. It was then that Dr. Carl June, an immunologist and oncologist, decided to apply an experimental therapy he was investigating: an anti-CD19 CAR-T immunotherapy.
In 2012, American doctor Carl June pioneered this experimental therapy on a six-year-old girl with leukemia who had no chance of survival. Today, 16-year-old Emily’s cancer has been in remission for a decade
Today, Emily’s leukemia has been in remission for a decade. This 16-year-old girl is the prime clinical example of how the immune system can be turned into a powerful weapon against cancer. Leading experts in the development of CAR-T immunotherapies, such as Dr. Javier Briones and Manel Juan, whose research projects are supported by the “La Caixa” Foundation, will talk about their progress in a new cycle of Caixa Research Debates. Sign up here to follow this online meeting next Tuesday, January 31st at 7:00PM ET.
Three types of therapy in one treatment
CAR-T (Chimeric Antigen Receptor T-cell or Chimeric Antigen Receptor in T lymphocytes) is a type of therapy in which the patient becomes the donor. This involves modifying the patient’s T lymphocytes so that they have the ability to attack tumor cells. T lymphocytes are a type of white blood cells responsible for the immune response, especially antiviral and antitumor, that is, they are the cells that protect us from threats such as infections and tumors.
Emily and other patients who were treated in the same way had their blood extracted for these cells using a technique that separates blood components. The T lymphocytes were then genetically reprogrammed so that, when reintroduced into the patient, they could recognize, attack, and destroy cancer cells. According to data provided by Dr. Carol June, director of the Center for Cellular Immunotherapies at the University of Pennsylvania, more than 10,000 people in the world have received the therapy.
cART-T therapies have achieved complete remissions of blood tumors—leukemias, lymphomas, and myelomas—while it is hoped that they can be successfully applied to other solid tumors as well.
CAR-T combines three types of therapy: cell, immunotherapy and gene. It is considered a cell therapy because it is a living drug that is administered immediately into the patient’s bloodstream. It is also an immunotherapy because the cells of the patient’s own immune system will fight against the cancer cells. And, third, it is a gene therapy—one of the first approved by the US Food and Drug Administration, an FDA agency—because the lymphocytes are genetically modified to combat the disease.
Innovative Research by Kaixa Research Experts
Dr. Briones is Head of the Clinical Hematology Unit and Director of the Cellular Immunotherapy and Gene Therapy Research Group of the Saint Pau Hospital Research Institute (IIB-Sant Pau). His group has led the creation of the first CAR-T in Europe for patients with classical Hodgkin lymphoma and T-cell non-Hodgkin lymphoma. According to the Spanish Society of Medical Oncology, Hodgkin lymphoma affects 3 out of every 100,000 people. The group also developed the new CAR-T 19 for patients with various types of B-cell non-Hodgkin lymphoma.
The doctor works as Head of the Immunology Service at the Biomedical Diagnostic Center (CDB) of the Juan Hospital Clinic (HCB)-IDIBAPS and is responsible for the platform for advanced therapies between the Hospital Sant Joan de Deu and the Hospital Clinic. His research group has promoted the first CAR-T in Europe for the treatment of acute lymphoblastic leukemia, particularly in adults, and other B-lineage lymphoproliferative syndromes, and more recently another therapy for the treatment of multiple myeloma. Myeloma accounts for 10% of bone marrow cancers and is the second most common cancer of the blood.
expand its use to other tumors and reduce its high cost
The results achieved by CAR-T therapy are very promising and have been confirmed in blood tumors such as leukemia or lymphoma, where it has shown efficacy in people who have already exhausted other options. The success of these early years has prompted efforts to expand its use against other types of tumors. Indeed, it is beginning to be tested in solid tumors such as glioblastoma, sarcoma, or breast, ovarian, testicular, and gastric cancers, and even in other diseases such as autoimmune pathology and transplant rejection.
Like other novel treatments or drugs, one of its biggest drawbacks is its high price. In recent years, groups of hematologists, immunologists, and oncologists have created academic CAR-Ts.
Other challenges include reducing the cost of treatment. Most CAR-T treatments are supplied through a pharmaceutical company and are, therefore, the result of private clinical trials, which lead to very high costs. In recent years, some groups of hematologists and immunologists have begun to create so-called academic CAR-Ts. They are self-promoted by CAR-T hospitals and as a result, with more affordable costs. Currently, commercial and academic CAR-T clinical trials co-exist in Spain.
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