Experimental treatment of multiple myeloma shows 70% efficacy

THE Hospital Clínic de Barcelona-IDIBAPS developed a new treatment to treat patients with multiple myeloma resistant to standard treatments. It’s a CAR-T (Chimeric antigen receptor T cell), a type of cell therapy and a gene in which the patient becomes his own donor and which consists of modifying the patient’s T lymphocytes so that they have the capacity to attack tumor cells. It is the first developed in Europe for the disease.

The data obtained are encouraging, as all patients improve with the procedure, and 75% of them maintain their response within 12 months. The results of the treatment, called ARI-0002h, were presented on December 12 at the American Congress of Hematology in Atlanta (USA).

This treatment is very specific and effective against multiple myeloma malignant cells.

“This is the second CAR-T that we have developed at the hospital. On this occasion, unlike ARI-0001, it is directed against another target, BCMA, the most widespread antigen in immunotherapy in myeloma”, he explains. manel juan, head of the Immunology Service of the Biomedical Diagnostics Center of the Clinical Hospital.

Through apheresis –a technique that allows the separation of blood components– T lymphocytes are obtained –a type of white blood cell in charge of the immune response–, which are genetically reprogrammed so that, when transfused from new to the patient, they can recognize specifically at tumor cells and attack them.

This CAR-T is directed against the BCMA antigen, which is found on the surface of tumor cells in a type of plasma cell cancer: multiple myeloma. The treatment demonstrated experimentally in the laboratory that the ARI-0002h was very specific Y effective against malignant cells of this type of cancer, which led to the Spanish Agency for Medicines and Health Products (AEMPS) to approve the clinical trial ARI-0002h in myeloma patients in whom conventional treatments have failed.

At 12 months, 75% maintain the response and have no disease progression and 60% have complete remission and no residual disease

“In relation to the previous CAR-Ts that we developed, this one presents something new and it is humanized”, explains Manel Juan.

“Mouse antibodies are often used for the development of the CAR-T, and in this case we have humanized it so that it has greater durability in the patient and less probability of rejection”, adds the researcher.

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Best response without great toxicity

THEmultiple myelomais a type of blood cancer located in the bone marrow, where plasma cells (a type of white blood cell) are responsible for producing the antibodies needed to fight infections. In it, plasma cells carry out an abnormal growth process and form tumors in areas of the bones. It is responsible for 10% of cancers in the bone marrow and it is the second most common blood cancer, after lymphoma.

Myeloma accounts for 10% of bone marrow cancers and is the second most common blood cancer after lymphoma.

Although there are several treatments for multiple myeloma, from chemotherapy to bone marrow transplantation, there are people who do not respond and therefore have a very limited life expectancy. This is where the CAR-T ARI-0002h developed by the Clínic team comes into play.

In addition to the changes in the molecule to target it against multiple myeloma, “this CAR-T has an innovative aspect in terms of administration, which is divided into two doses. The first is divided into three small doses to limit toxicity and, after four months, a second dose is administered to patients whose disease has not progressed”, he highlights. Carlos Fernandez de Larrea. “This allows us to improve the response without relevant toxicity”, he adds.

The results of the trial in which they participated 30 patients resistant to treatments, show that at 12 months 75% maintain the response and they have no progression of the disease and that 60% have complete remission and no residual disease.

These results are comparable in efficacy to the commercial CAR-T that exists against multiple myeloma and with less toxicity.

Álvaro Urbano-Ispizua (Clinic)

“These results are comparable in efficacy to the commercial CAR-T that exists against multiple myeloma and with less toxicity”, he emphasizes. Álvaro Urbano-Ispizua, director of Clinical Institute of Hemato-Oncological Diseases . “Now we are preparing all the documentation according to the results to request the AEMPSits use as a handcrafted advanced therapy drug”, concludes Urbano-Ispizúa.

Source: SYNC

Rights: Creative Commons.

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