Axicabtagene ciloleucel, sold under the brand name Yescarta, is a
medication
A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy ( pharmacotherapy) is an important part of the medical field and ...
used for the treatment for
large B-cell lymphoma
The large-cell lymphomas have large cells. One classification system for lymphomas divides the diseases according to the size of the white blood cells that have turned cancerous. A large cell, in this context, has a diameter of 17 to 20 μm. ...
that has failed conventional treatment.
T cells
A T cell is a type of lymphocyte. T cells are one of the important white blood cells of the immune system and play a central role in the adaptive immune response. T cells can be distinguished from other lymphocytes by the presence of a T-cell re ...
are removed from a person with lymphoma and
genetically engineered
Genetic engineering, also called genetic modification or genetic manipulation, is the modification and manipulation of an organism's genes using technology. It is a set of technologies used to change the genetic makeup of cells, including t ...
to produce a specific
T-cell receptor
The T-cell receptor (TCR) is a protein complex found on the surface of T cells, or T lymphocytes, that is responsible for recognizing fragments of antigen as peptides bound to major histocompatibility complex (MHC) molecules. The binding b ...
. The resulting
chimeric antigen receptor T cell
In biology, chimeric antigen receptors (CARs)—also known as chimeric immunoreceptors, chimeric T cell receptors or artificial T cell receptors—are receptor proteins that have been engineered to give T cells the new ability to target a specifi ...
s (CAR-Ts) that react to the cancer are then given back to the person to populate the bone marrow.
[ ] Axicabtagene treatment carries a risk for
cytokine release syndrome (CRS) and neurological toxicities.
[
Due to CD19 being a pan-B cell marker, the T-cells that are engineered to target CD19 receptors on the cancerous ]B cells
B cells, also known as B lymphocytes, are a type of white blood cell of the lymphocyte subtype. They function in the humoral immunity component of the adaptive immune system. B cells produce antibody molecules which may be either secreted o ...
[ also influence normal ]B cells
B cells, also known as B lymphocytes, are a type of white blood cell of the lymphocyte subtype. They function in the humoral immunity component of the adaptive immune system. B cells produce antibody molecules which may be either secreted o ...
, except some plasma cells
Plasma cells, also called plasma B cells or effector B cells, are white blood cells that originate in the lymphoid organs as B lymphocytes and secrete large quantities of proteins called antibodies in response to being presented specific substan ...
.
Side effects
Because treatment with axicabtagene carries a risk of cytokine release syndrome and neurological toxicities, the FDA has mandated that hospitals be certified for its use prior to treatment of any patients.[
]
History
It was developed by California-based Kite Pharma
Kite Pharma is an American biotechnology company that develops cancer immunotherapy products, with a primary focus on genetically engineered autologous CAR T cell therapy, a cell-based therapy which relies on chimeric antigen receptors and T ce ...
.
Axicabtagene ciloleucel was awarded U.S. Food and Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...
(FDA) breakthrough therapy
Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "br ...
designation on 18 October 2017, for diffuse large B-cell lymphoma
Diffuse large B-cell lymphoma (DLBCL) is a cancer of B cells, a type of lymphocyte that is responsible for producing antibodies. It is the most common form of non-Hodgkin lymphoma among adults, with an annual incidence of 7–8 cases per 100,00 ...
, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma
Primary mediastinal B-cell lymphoma, abbreviated PMBL, is a rare type of lymphoma that forms in the mediastinum (the space in between the lungs) and predominantly affects young adults.
It is a subtype of diffuse large B-cell lymphoma; however, it ...
. It also received priority review
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...
and orphan drug designation
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.
The assignment of ...
.[
Based on the ZUMA-1 trial, Kite submitted a ]biologics license application
A biologics license application (BLA) is defined by the U.S. Food and Drug Administration (FDA) as follows:
The biologics license application is a request for permission to introduce, or deliver for introduction, a biologic product into inters ...
for axicabtagene in March 2017, for the treatment of non-Hodgkin lymphoma
Non-Hodgkin lymphoma (NHL), also known as non-Hodgkin's lymphoma, is a group of blood cancers that includes all types of lymphomas except Hodgkin lymphomas. Symptoms include enlarged lymph nodes, fever, night sweats, weight loss, and tirednes ...
.
The FDA granted approval on 18 October 2017, for the second-line treatment of diffuse large B-cell lymphoma.[''F.D.A. Approves Second Gene-Altering Treatment for Cancer'' 2017]
/ref>[ ] It is not indicated for the treatment of patients with primary central nervous system lymphoma.
References
External links
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{{DEFAULTSORT:Axicabtagene Ciloleucel
Breakthrough therapy
Cancer treatments
Gene therapy
Gilead Sciences
Orphan drugs
Antineoplastic drugs