Why are ATMPs different?
Advanced therapy medicinal products (ATMPs) are treatments for humans that are based on genes, tissues or cells. They offer groundbreaking new opportunities for the treatment of diseases and injury that were not previously available.
How do ATMPs differ from traditional therapies?
It may contain recombinant nucleic acid (normally DNA or RNA) intended to have a therapeutic effect by regulating a genetic sequence or by introduction/modification of a genetic sequence – all gene therapies and gene-corrected cell therapies are ATMPs
It may contain cells that have been taken from donor/patient and modified so they are deemed to no longer be the same as the donor cells before being returned to the patient
It may contain cells that have been taken from donor/patient and returned to the patient for what is deemed to be a different function.
The 3 main types of ATMPs
These contain cells or tissues that have been modified so they can be used to repair, regenerate, or replace human tissue.
Somatic-cell therapy medicines
Cells or tissues that have been manipulated to change their biological characteristics or not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases
Gene therapy medicines
Genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting 'recombinant' genes into the body, usually to treat genetic disorders, cancer or long-term diseases.
What is CAR-T Therapy?
CAR-T cell therapy is probably the best known ATMPs treatments.
Its an individualized approach to treating certain types of cancer. This breakthrough type of cell and gene therapy harnesses the power of a patient's own immune system to destroy cancer cells.
With CAR-T cell therapy, a patient’s T cells are reprogrammed into CAR-T cells, which enhances their ability to detect and destroy cancer cells.
T cells are a part of the immune system that help defend the body.
T cells detect and destroy infected or cancerous cells by looking for certain antigens, which are markers that help the immune system identify normal cells from infected or cancerous cells. But the antigens on cancerous cells look like normal antigens, and normal T cells cannot recognize them.
T cells reprogrammed to be CAR-T cells
In CAR-T cell therapy, a patient’s T cells are modified and reprogrammed with CARs, or chimeric antigen receptors.
When a CAR is added to the patient’s own T cells, they become CAR-T cells, and they’re able to recognize specific characteristics on certain types of cancer cells. CAR-T cells specifically target certain receptors on normal and cancerous B cells, breaking them down and destroying them.
CAR-T Cell Therapy Treatment Process
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1. Collecting the T cells
A patient's white blood cells, including T cells, are taken out of the body through a specialized process called leukapheresis, where blood is removed from the patient and separated. This is done at a certified CAR-T treatment center. Depending on the specific type of CAR-T cell therapy, the T cells are frozen before they are sent to a manufacturing facility for reprogramming.
2. Reprogramming the cells
Using an inactive virus (known as a viral vector), T cells are reprogrammed by introducing DNA into them, to produce chimeric antigen receptors (CARs) on the surface of the T cells. Once these new, patient-specific CAR-T cells are armed to detect and destroy cancer cells, they are frozen and sent back to the CAR-T treatment center where the patient is being treated.
4. Infusing the cells
The reprogrammed patient-specific CAR-T cells are delivered into the patient’s blood through an IV infusion, which is done at a certified CAR-T treatment center. CAR-T cell therapies are designed to be one-time therapies and an infusion usually takes less than 30 minutes.
3. Preparing for infusion
Within 2 weeks before the CAR-T cells are infused, most patients will receive a short course of chemotherapy that helps prepare the body to receive their reprogrammed CAR-T cells. This is referred to as “lymphodepletion” and is done to create space for the new CAR-T cells to expand and grow.
CAR-T cell therapy requires both short-term monitoring and ongoing follow-up visits. A health care team will monitor patients both to make sure the CAR-T cell therapy is working, and to watch for side effects. The doctor will continue to follow-up with the patient to understand the long-term results of the treatment. It is important that any technical solutions can monitor patient over the long term and be regulatory compliant.
5. Detecting and destroying cells
Within the patient’s body, CAR-T cells may detect and destroy normal and cancerous B cells. CAR-T cell therapy may provide long-lasting remission as the patient’s immune system reproduces its own CAR-T cells.