Glossary

A type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient’s blood. Then the gene for a special receptor that binds to a certain protein on the patient’s cancer cells is added to the T cells in the laboratory. The special receptor is called a chimeric antigen receptor (CAR). Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion. CAR T-cell therapy is used to treat certain blood cancers, and it is being studied in the treatment of other types of cancer. Also called chimeric antigen receptor T-cell therapy.

Cell therapy is a type of medical treatment where living cells are introduced into a patient's body to replace or repair damaged tissue, treat diseases, or enhance the body's immune response. These cells can come from the patient (autologous) or a donor (allogeneic) and are used to treat various conditions, including cancer, blood disorders, and autoimmune diseases. The goal of cell therapy is to use the unique properties of cells to restore normal function and health.

Chronic lymphocytic leukemia is a type of blood cancer that affects the immune system by causing a buildup of dysfunctional white blood cells called lymphocytes. These cells usually originate from a single source and gradually accumulate.

A clinical trial is a research study in people (healthy volunteers or patients) to answer specific health questions. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health.

• Interventional clinical trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments
• Observational clinical trials watch health issues in large groups of people or populations in natural settings

This is the person responsible for managing the day-to-day activities of a clinical trial and who would be, alongside your doctor, the point of contact for you while on the clinical trial.

The clinical trial site is a controlled environment / location, such as doctor’s offices, medical centers, community hospitals and clinics, where clinical trial testing can take place safely with appropriate oversight. If participating in a clinical trial, you will normally go to the clinical trial site closest to you. Each location has a research team, which includes doctors, nurses and other healthcare professionals.

A controlled clinical trial compares the experimental drug / treatment with the current standard of treatment, placebo, or no treatment. A control is the standard by which experimental observations are evaluated. In many clinical trials, 1 group of people taking part will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness (standard of care) or something called a placebo.

Dermatomyositis is a rare autoimmune disorder causing mainly inflammation of muscle and skin, and sometimes other organs. The medical condition can have a significant impact on day-to-day life, and as symptoms can come and go over time, it can be difficult to manage. There is a need for more treatments that work well and are well tolerated.

A healthy volunteer is a person with no known significant health problems participating in a clinical trial. Healthy volunteers often participate in Phase 1 clinical trials.

The factors that allow someone to participate in a clinical trial are called “inclusion criteria” and those that disallow someone from participating (the “exclusion criteria”). These criteria are based on factors such as lab values, the type and stage of a medical condition, previous treatment history and other medical conditions. Before joining a clinical trial, a participant must qualify for the clinical trial. Some clinical trials seek participants with medical conditions (patients), while others need healthy volunteers. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Informed consent is the process of learning the key facts about a clinical trial before deciding whether to participate. It is also a continuing process throughout the clinical trial to provide information to participants. To help someone decide whether to participate, the doctors and nurses involved in the clinical trial explain the details of the clinical trial. If necessary translation assistance can be provided. The research team then provides an informed consent form, which includes details about the clinical trial, such as its purpose, duration, required procedures and key contacts. Any risks and potential benefits are explained in the informed consent form. The participant then decides whether to sign the document. An informed consent form is not a contract, and the participant may withdraw from the clinical trial at any time.

Multiple myeloma is an incurable type of cancer that originates from plasma cells, which are white blood cells that produce antibodies and play a crucial role in our immune system. Multiple myeloma is a complex cancer that arises from the clonal expansion of cancerous / malignant plasma cells in the bone marrow. Treatment plans are tailored to the individual patient's condition and may also involve supportive care measures to manage symptoms and improve quality of life.

Non-Hodgkin’s lymphoma is a cancer originating from the lymphocytes, a type of white blood cell which is part of the body’s immune system.

These clinical trials occur in only a small number (often 20 to 80 people) of people, and the main focus is to watch the safety of a treatment. The researchers will test for side effects, determine how much of the treatment is safe to give and learn how the body copes with the treatment. Phase 1 clinical trials may take place with healthy volunteers or in patients. Oncology Phase 1 clinical trials for instance are with patients.

Phase 2 clinical trials are tested in a larger group of people (100 to 300 patients) and only includes patients who have the medical condition the treatment is looking to treat. During these clinical trials, it is possible to begin to get an idea as to whether the drug is likely to work and researchers will look to see what dose may work best.

If the results from the clinical trials in Phases 1 and 2 are positive (they have passed safety checks and have shown that the treatment may work), then Phase 3 clinical trials will take place. Here, the treatment is tested in a much larger number of patients (1000 to 3000 people) and the treatment is often compared with an existing treatment (standard-of-care) or placebo to see how well it works.

Phase 4 clinical trials occur after the treatment has been shown to be effective and is available to patients. During these clinical trials, the long-term safety of the treatment will be reviewed. How well the treatment continues to work in patients will also be monitored.

A placebo is an inactive pill, liquid or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment’s effectiveness. In some clinical trials, the participants in the control group will receive a placebo instead of an active drug or treatment. We do not use a placebo group in our oncology trials.

A protocol is a clinical trial plan. It is carefully designed to safeguard the health of the participants as well as to answer specific research questions. A protocol describes what types of people may participate in the clinical trial (the “inclusion criteria”), the schedule of tests, procedures, medications and dosages, and the length of the clinical trial. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

If a clinical trial is randomized, then the decision about which treatment each participant receives is made by chance, rather than decided by the doctor or participant. This process, which usually involves a computerized system, is called randomization.

Side effects are any undesired actions or effects of a drug or treatment. Examples are headache, nausea, hair loss, skin irritation or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

Standard of care is a treatment that is accepted by medical experts as a proper treatment for a certain type of condition and that is widely prescribed by health care professionals. Also called best practice, standard medical care, and standard therapy.

A chronic, inflammatory medical condition affecting nearly every organ. The immune system of the body makes antibodies (proteins) that attack its own tissues and cells. The medical condition can be life-threatening and could cause permanent organ damage. Systemic Lupus Erythematosus occurs in women more frequently. It has periods of a relatively stable medical condition phase, followed by periods of flares. The medical condition can heavily affect the quality of life. There is no cure and there is a high unmet medical need.