The CAR T-Cell Therapy Clinical Journey Guide
Glossary
Allogenic
Allo means “other”. Allogeneic means that the cells or tissue given to a patient come from someone other than the patient.
Angiography
An X-ray image of blood vessels that uses a dye so that the blood vessels can be seen.
Anticoagulant
A substance used to prevent the formation of blood clots or break up existing blood clots.
Antigen
An antigen is anything that can be “seen” by the immune system. There are many different types of antigens. In CAR T-cell therapy, antigens are a substance (or marker) on the outside of a cell that is recognised by the CAR T cell and ‘marks’ the cell for destruction. An example of an antigen (or marker) in CAR T-cell therapy is CD19, found on the outside of both normal and cancerous B cells.
Apheresis
The process of removing whole blood from a patient, processing the blood in a machine that separates out one component and then returning the remainder of the blood components to the patient. There are several kinds of apheresis procedures, each named after the component of blood that is extracted. When white blood cells are extracted, the process is called leukapheresis.
Approved
Indicates that a drug or therapy has been fully tested in clinical trials to ensure it is safe and effective and has been approved for use outside of a clinical trial by the MHRA.
Autologous
Auto means “self”. Autologous means that the cells or tissue given to a patient come from that same individual.
B cell
A type of white blood cell (or lymphocyte) that makes antibodies to fight infection. Like all other cells in the body, B cells can become cancerous.
B-cell aplasia
Low numbers of B cells. B-cell aplasia is one of the expected results of successful anti-CD19 targeted therapy.
Bone marrow biopsy
A procedure that takes a sample of bone marrow by inserting a hollow needle through the bone (usually the hip bone) and into the bone marrow.
Bridging therapy
Therapy that is meant to control cancer during the time between leukapheresis and CAR T-cell infusion.
Cannula
A small tube inserted into a vessel, cavity or duct to allow flow of substances (usually fluids).
CAR
Chimeric Antigen Receptor
CAR
See Chimeric antigen receptor (CAR).
Catheter
A thin tube used to deliver blood, fluids or medications into a vein.
CD19
An antigen, or “marker” found on the surface of B cells. See ‘antigens’.
Cell
The basic membrane-bound unit of life that composes all living things.
Chimeric antigen receptor (CAR)
A protein created in a laboratory that is designed to recognise an antigen (or marker) on cancer cells. When added to T cells, CARs give T cells (now called “CAR T cells”) the ability to identify and destroy cancer cells.
Chronic stressor
Stress resulting from repeated exposure to situations that lead to the release of stress hormones. This type of stress can cause wear and tear on your mind and body.
Clinical Research Team
A group of professionals involved in all aspects of running a clinical trial. Team members can include investigators, research nurses and coordinators, data managers, staff nurses and doctors. In some centres, team members may also serve as the healthcare providers for trial participants.
Clinical trial
Research that involves one or more human participants, carried out to learn if a treatment is safe and/or works.
CRS
Cytokine Release Syndrome
CT or CAT scan
Uses a group of X-rays taken at different angles around the body and a computer to create detailed cross-sectional images.
Cytokine Release Syndrome (CRS)
A common side effect of CAR T-cell therapy. CRS occurs when many cytokines are released by immune cells during immunotherapy. Some symptoms are nausea, fever, headache, rapid heartbeat, low blood pressure, rash and trouble breathing. CRS can be mild or moderate. CRS can feel like a very bad case of the flu, but in rare cases it can be severe or life-threatening.
Cytokine Storm
See Cytokine Release Syndrome.
Cytokines
Small proteins that regulate the immune system. Cytokines are released by certain immune system cells. They can stimulate the immune system to attack cancer and also cause the production of more cytokines.
Cytopenias
Low blood cell counts, including red blood cells, white blood cells, and platelets.
Discharge Instructions
Instructions given to a patient when discharged from the hospital, usually by a nurse. They include important information for patients and carers to manage their own care when outside of a hospital or treatment centre. These instructions can be given in written or verbal form.
Distress
Emotional, social, spiritual or physical pain or suffering that may cause a person to feel sad, afraid, depressed, anxious or lonely. People in distress may also feel that they are not able to manage or cope with changes caused by normal life activities or by having a disease, such as cancer.
Dose escalation
A study design in which a very small dose of a treatment is given to a small number of patients. If it is found to be safe, the next group receives a higher dose. This continues until all the planned doses have been tested.
ECG
Electrocardiogram, electrocardiograph. Records the electrical activity of your heart.
ECHO (echocardiogram)
An ultrasound image of the heart.
ECOG performance score
A questionnaire that measures the ability to care for oneself and perform daily activities and physical functions (like walking, working, etc.).
Effective
How well a treatment works under “real-world” conditions.
Efficacy
How well a treatment works under ideal and controlled circumstances, as in a clinical trial.
Eligibility criteria
Factors that define the population of patients that will be allowed to participate in a particular trial.
Emergency Wallet Card
A card with information about the CAR T-cell therapy you have received and contact information for your clinical research and healthcare teams.
Exclusion criteria
Factors that disqualify a patient from participating in a particular clinical trial.
Gene Therapy
A therapy that uses genes to treat or prevent disease. In CAR T-cell therapy, a gene carrying instructions to make a new protein receptor is inserted into T cells.
Gene Therapy Advisory Committee (GTAC)
The UK national REC for gene therapy clinical research.
Genes
Stretches of DNA that serve as the “instructions” or blueprints for cells to make proteins.
Hypogammaglobulinaemia
Low levels of antibodies, or immunoglobulins.
ICANS
Immune Effector Cell-Associated Neurotoxicity Syndrome
ICF
Informed Consent Form
Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS, or neurotoxicity)
A severe side effect of CAR T-cell therapy in which the immune response can produce a toxic effect on the nervous system. Patients may suffer headaches, confusion, seizures, loss of speech and loss of motor skills. ICANS can range from very mild to quite severe and life-threatening.
Immunotherapy
A type of treatment that uses the immune system to treat disease (including cancer).
Inclusion criteria
Factors that a patient must have in order to participate in a particular clinical trial.
Informed consent
See Participant Information Sheet.
Informed consent process
A process that makes sure clinical trial participants are fully informed about and understand all the details of a clinical trial before agreeing to participate.
Intravenous immunoglobulin (IVIG)
A treatment often given to people who have received CAR T-cell therapy. IVIG provides extra antibodies and helps to prevent frequent infections until B cells return to more normal levels.
Investigator
A person responsible for the conduct of a clinical trial at the clinical research site. If there is more than one investigator at a site, the leader is called the “Principal Investigator”.
IVIG
Intravenous Immunoglobulin
Leukapheresis
A procedure that involves removing blood from the body, delivering it to a machine that separates and collects white blood cells and then returning the remaining blood components to the body.
Long-term follow-up study (LTFU)
A study in which patients in a clinical trial are monitored for an extended period of time to learn about possible delayed side effects.
LTFU
Long-term Follow-Up (study)
Lumbar puncture (spinal tap)
A procedure that takes a sample of cerebrospinal fluid (liquid that surrounds the brain and spinal cord). This is done by inserting a needle between two vertebrae (bones in the spine).
Lymphocytes
White blood cells that play an important role in the immune system. There are three types: B cells, T cells and Natural Killer (NK) cells.
Lymphodepleting chemotherapy
Chemotherapy given before CAR-T cell infusion in order to decrease the number of white blood cells (including T cells) in the body. CAR T cells grow and expand better if there are not as many of the patient’s own T-cells present.
Marker
See antigens.
MBSR
Mindfulness-Based Stress Reduction
Medicines and Healthcare Products Regulatory Agency (MHRA)
The UK agency responsible for ensuring medicines and healthcare product safety, quality and effectiveness.
MHRA
Medicines and Healthcare products Regulatory Agency
MRI (magnetic resonance imaging)
A procedure that uses a strong magnetic field, radio waves, and a computer to make detailed pictures of the body.
MUGA (multiple gated acquisition)
A type of heart scan that uses a small amount of a radioactive tracer and a special video camera to see how well the heart is pumping.
Mutation
A change that occurs in the DNA sequence. Certain mutations may lead to cancer or other diseases.
Neurological
Related to the brain, spine, or nerves.
Neurotoxicity
Producing a toxic effect on the nervous system. See Immune Effector Cell-Associated Neurotoxicity Syndrome.
Participant
An individual who participates in a clinical trial.
Participant Information Sheet (PIS)
A document that provides potential clinical trial participants with enough information about the clinical trial to allow them to make an informed decision about whether to participate. This is sometimes called an Informed Consent document.
PET-CT scan (Positron Emission Tomography/Computed Tomography)
A type of scan that uses a small amount of a radioactive tracer to light up active cells. Cancer cells show up as bright spots on a PET scan.
PFT (pulmonary function tests)
Breathing tests to see how well the lungs are working.
PIS
Participant Information Sheet
Placebo
An inactive treatment, used in some studies to compare to an experimental treatment.
Platelets
Blood cells that help blood to clot to stop bleeding.
Post-Traumatic Growth
Positive life changes that develop through a stressful, frightening experience.
Principal Investigator
The person in charge of all aspects of a clinical trial.
Psychosocial Oncologist
A person who specialises in the psychological, behavioural, emotional and social issues that arise for cancer patients and their loved ones.
Randomised
A clinical study design that randomly assigns participants to different groups to compare different treatments.
REC
Research Ethics Committee
Receptor
A protein on the surface of a cell that can recognise and attach to an antigen.
Research Ethics Committee (REC)
A group of doctors, nurses, scientists, social workers, chaplains and community members that protect the rights and welfare of anyone participating in a clinical trial. The REC will review and monitor any research at their institution that has human participants.
Safe
There are not unacceptable levels of unwanted effects of the treatment.
Side effect
Any unwanted effects of a treatment.
Sign
Something that can be seen or measured.
Skeletal survey
A series of X-rays to look for areas of bone that may be damaged.
Sponsor
The person, company, group or organisation that pays for and oversees a clinical trial. The sponsor collects and analyses data from the trial.
Standard-of-Care Treatment
The best (or generally accepted) treatment for a specific disease.
Stress responses
Normal reactions to situations that make us feel frustrated, angry or nervous—like a cancer diagnosis and its treatment.
Supportive Care Services
Services aimed at preventing or treating symptoms of cancer as early as possible. This includes helping with side effects caused by treatment and psychological, social and spiritual problems related to a cancer diagnosis or its treatment.
Symptom
Something the patient feels or experiences.
T cell
A type of white blood cell that travels throughout the body and destroys damaged or infected cells.
Tumour biopsy
A sample of tumour tissue.
Tumour Lysis Syndrome
Condition that can occur when a large number of cancer cells die within a short period of time, releasing their contents into the blood.
Ultrasonography
An imaging procedure that uses sound waves to create pictures of structures deep within the body.
