Lymphoma : gene therapy revolutionary

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Published the 19.10.2017 at 11: 38 am

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Keywords :

lymphomethérapie gene

The axicabtagene ciloleucel is the first treatment of the type “BECAUSE T-cell” approved by the FDA for certain types of non-hodgkin’s lymphoma. It was approved following a priority procedure because of the designation “revolutionary treatment” and ” orphan drugs “.

In August, the FDA had approved another CAR-T cell, the tisagenlecleucel, for the treatment of lymphoblastic leukemia, acute precursor B-cell, recurrent, or refractory, in children and young adults.

T cells genetically reprogrammed

T lymphocytes of the patient, the immune system cells are collected, reprogrammed genetically to attack a specific antigen on the surface of cells lymphomateuses, and then are cultured before being re-injected to the patient.

The antigen target is a protein, CD19, which is expressed at the surface of these cells lymphomateuses. Once re-injected into the patient’s body, the T cells modified to replicate quickly and directly attack the malignant cells with a remission quick.

Certain lymphomas (B-resistant

The axicabtagene ciloleucel has been specifically approved for use in adults with B cell lymphoma with large cells that have resisted at least two other types of treatment. The lymphomas are affected by this treatment are the lymphoma diffuse large B-cell (DLBCL) lymphoma mediastinal primary large B-cell, B-cell lymphoma at high risk and the DLBCL secondary to follicular lymphoma.
This CAR-T cell, however, is not indicated for the treatment of patients with primary lymphoma of the central nervous system.

A multicentre trial.

The approval was based on a multicenter clinical trial involving more than 100 adult patients with a B cell lymphoma that is refractory or relapsed. The rate of complete remission after treatment with immunotherapy gene is 51 %.

The main complications of this type of treatment are the occurrence of a cytokine release syndrome and neurological toxicities, both of which can be fatal or potentially fatal.
Other side effects include serious infections, anemia and immune suppression. The side effects usually appear within the first 2 weeks.

A framework for requirements

The axicabtagene ciloleucel will be made available to hematologists with a warning on the box, because of the possibility of the occurrence of a cytokine release syndrome and toxicities neurological. There will be a framework of requirements for an “assessment strategy and risk mitigation” (REMS). Under this REMS, the hospitals must be certified to dispense the drug, with the staff required to be trained in the identification and management of these toxicities.
The FDA also requires a study observational post-marketing.

A new era

We are entering a new era of medical innovation, in which we are now able to reprogram the cells of a patient so that they attack themselves, a deadly cancer. These new immunotherapies gene have the potential to transform medicine and create a real break in the ability of hematologists to treat, and even cure, many diseases hitherto incurable.

A press release from the company indicates that the list price of this molecule in the United States will be from 373 000 dollars.