CAR (Chimeric Antigen Receptor) T-cell therapy is to hit the market with huge expectations
Cancer has always been treated with chemotherapy, radiation, and surgery. The medical community has made great advances in these areas. But, as medical science advances at a remarkable rate, we can see the introduction of targeted therapies and, more recently, immunotherapy therapies. These strengthen the power of a patient’s immune system to tackle tumors. Immunotherapy is also known as the ”fifth pillar” of cancer treatment.
Recently, there has been an exciting advancement in immunotherapy and gene therapy known as Adoptive Cell Transfer (ACT), which collects and uses a patient’s immune cells to treat their own cancer. One type of ACT attracting a lot of attention is the CAR T-cell therapy. Notably, the Food and Drug Administration (FDA) has recently approved CAR T-cell therapy to treat children and young adults with B-cell acute lymphoblastic leukemia. CAR T-cell therapy will provide a safe and affordable cancer therapy in the years to come, which is promising in terms of market access and patient care.
CAR (Chimeric Antigen Receptor) T-cell therapy is a form of immunotherapy in which T cells are collected from a patient via apheresis. One or more blood components like plasma or white blood cells are removed from the blood. The remaining blood is then returned to the patient’s body.
These T cells are then reprogrammed in a laboratory to produce genetically coded cells which target the cancer cells and specific antigen expressing B cells.
The engineered T cells consist of a Chimeric Antigen Receptor (CAR). The receptor has an external target-binding domain and an internal activation domain. The external domain recognizes a specific tumor antigen whereas the internal domain activates the cell when it binds to the target.
The significance of CART therapy is explained with their treatment success rates in the following clinical trials:
|Name of Company and Study||Molecule||Patient Characteristics in the Trial||Clinical Significance|
|Novartis – ELIANAstudy||Tisagenlecleucel(Kymriah brand)||Relapsed or refractory B-cell lymphoblastic
|Complete remission in 82% andrelapse free in 60% patients after
|Kite Pharma – ZUMA – 1study||Axicabtageneciloleucel||Chemo refractoryaggressive B-cell Non-
Hodgkin Lymphoma (NHL)
|Achieved complete response rateor lymphoma clear in 36%|
On August 30, 2017, Kymriah (tisagenlecleucel) suspension for intravenous infusion received FDA approval for the treatment of patients up to the age of 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory, or who have relapsed at least twice. Kymriah is going to be introduced into the market at a price of $475,000 for a single infusion.
CAR T-cell therapy is promising for patient care though there are certain side effects like Cytokine Release Syndrome (CRS), B cell aplasia or loss of B cells. Other less common side effects include cerebral edema and neurotoxicity.
Several companies are working to develop safer CAR T-cell therapies. The safer therapies work by:
The following table illustrates the technologies used by companies to resolve problems associated with CAR T-cell therapy.
|Switch control system||Only engineered cells are activated in the presence of rapamycin.||Servier and Pfizer|
|GoCAR-T Technology||CAR T-cells are activated in the presence of rimiducid andthe targeted antigen expressed on the surface of the cancer cells.||Bellicum Pharmaceuticals|
|NKR-T Cell Platform||CAR T-cells do not act on normal lymphocytes, thereby avoiding the side effects. T cells producedfrom CAR T-cell therapy binds to eight naturally-occurring ligands which are overexpressed in 80% tumors||Celyad|
There are huge expectations from CAR T-cell therapy as it is a far more effective and less intrusive therapy when compared to other treatments. It is expected to have a huge market potential and very high growth rate.
The global market for CAR T-cell therapy is estimated at $70 million in 2017. The market is expected to grow at a compound annual growth rate (CAGR) of 45% from 2019 to 2028.
This is an exciting time and the brink of a new era for cancer treatment. According to Timothy Cripe of Nationwide Children’s Hospital, the CAR T-cell therapy is “the most exciting thing I’ve seen in my lifetime“. As professionals in pharmaceutical consulting, we are excited to witness an age where such an innovative new treatment stands to improve patient care and market access issues around the world.
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