Pharmaceutical consulting - the impact of car t-cell therapy

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How CAR T-Cell Therapy Will Impact the Market

September 26th, 2017 - 0 Comments

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.

What is CAR T-cell therapy?

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.

Significance of CART therapy

The significance of CART therapy is explained with their treatment success rates in the following clinical trials:

Name of Company and StudyMoleculePatient Characteristics in the TrialClinical Significance
Novartis – ELIANAstudyTisagenlecleucel(Kymriah brand)Relapsed or refractory B-cell lymphoblastic


Complete remission in 82% andrelapse free in 60% patients after

six months

Kite Pharma – ZUMA – 1studyAxicabtageneciloleucelChemo 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.


Complications and side effects

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.

  • CRS releases cytokines in massive amounts and at a rapid rate into the bloodstream. This leads to high fevers and severe drops in blood pressure. CRS is life threatening, but reversible.
  • The infused CAR T-cells often kill normal B cells with B cell Aplasia or loss of B cells. This side effect is due to the expression of CD19 by normal B cells. Patients lack the antibodies to kill pathogens.This side effect can be mitigated by the infusion of gamma globulin.


Companies are working to make CAR T-cell therapy safer

Several companies are working to develop safer CAR T-cell therapies. The safer therapies work by:

  • Regulated activation of engineered T cells by external small molecules
  • Avoiding killing the normal B cells by engineered T cells

The following table illustrates the technologies used by companies to resolve problems associated  with CAR T-cell therapy.

Switch control systemOnly engineered cells are activated in the presence of rapamycin.Servier and Pfizer
GoCAR-T TechnologyCAR 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 PlatformCAR 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% tumorsCelyad


Market size of CAR T-cell therapy

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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 phamax is a Swiss healthcare consulting firm. Our team is enthusiastic and determined to simplify and resolve various healthcare market accesscomplexities in the pharmaceutical industry while making sure that everyone who needs to access to a particular drug or treatment receives the attention they deserve. To find out more about how we can help you with your market access concerns, email us at

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