Research & Clinical Trials

The Hematology Cell Bank of BC is a bank that stores samples of blood and cancer cells. After obtaining written informed consent, specimens are collected when a routine medical procedure is being performed. We call the bank a ‘Cell Bank’ because it is the cells within the blood and bone marrow that are stored and used by scientists for research purposes. Specimens donated to the bank are not used as blood transfusions or for transplant purposes. These donated specimens are used solely for research purposes.

Scientists who wish to use specimens stored in the Cell Bank must meet the requirements of the BC Cancer Agency Research Ethics Board (an independent ethics board that reviews all scientific research projects that use human specimens). The researcher must also demonstrate to a scientific review committee that their work has scientific merit.

Participation in the Cell Bank is completely voluntary.

Everyday the body normally produces billions of new white blood cells, red blood cells and other cells. All of these are produced from a small number of very specialized cells called blood stem cells (also called hematopoietic stem cells). Occasionally, something goes wrong with the growth and behavior of the blood cells and a blood disease or cancer develops. Currently, not much is known about how normal blood cells or blood stem cells become diseased or turn into cancers so this is an important area of research. In addition, scientists are also trying to develop safer and more effective treatments for blood diseases, as well as, improved tests and ways of preventing blood cancers and other diseases.

You may hear your physician or researchers in the media talk about ‘translational research’ or sometimes this may be referred to as ‘bench to bedside’ research. What does this mean? Translational research is the promotion of knowledge and scientific development by improving communication between the clinic and the laboratory. To improve human health, scientific discoveries must be translated into practical applications. Such discoveries typically begin at the ‘bench’ with basic research in the laboratory and then progresses to the clinical level, or the patient’s ‘bedside’ with new treatments. Scientists provide clinicians with new tools to help treat patients and clinicians provide scientist’s access to specimens and clinical data to help further research.

The specimens collected may include:

• Blood samples
• Bone marrow material (from bone marrow biopsies or harvested bone marrow)
• Peripheral blood stem cells
• Leukapheresis material (Leukapheresis is a procedure for reducing the white blood count in persons with leukemia with a very elevated white blood cell count).

Scientists also use specimens donated from healthy individuals to help them understand how healthy blood cells compare to diseased blood cells. Specimens may be donated by your family member or an unrelated donor at the time they are donating for your transplant. With their consent a specimen will be collected and stored at the Hematology Cell Bank for scientific research. The specimens collected may include:

• Blood samples
• Peripheral blood stem cells
• Harvested stem cells from bone marrow

With your consent specimens of blood and bone marrow material will be collected at the time you are scheduled for blood collection, a bone marrow biopsy or bone marrow harvest. You may be asked to have one additional blood sample collected, but you will not be asked to have additional bone marrow biopsies or stem cell collection procedures for research purposes. Donating these specimens is entirely voluntary. If you are donating peripheral blood stem cells for transplant or having leukapheresis to reduce a high white blood cell count, specimens will be collected at that time.

You will not be able to decide the specific research your specimens will be used for, just as when you donate blood to a blood bank, you cannot decide which patient will receive that blood. The use of your specimens will be reviewed by a panel of research scientists and clinicians. They will review the scientist’s research proposal to determine the use of the specimens. All research proposals are approved by the BC Cancer Agency Research Ethics Board (an independent ethics board that reviews all scientific research projects that use human specimens).

Research using human specimens can take a long time. The results of the research may not be determined for many years. You will not be informed of the results of that research, but may hear of important discoveries through the media or from your physician.

Human specimen research aims to provide better treatments for patients – it may lead to research involving the trial of new medications to treat your disease. The scientists using these specimens work closely with the physicians of the Leukemia/BMT Program of BC to bring new and more promising treatments to patients. Information about the type of research being done using donated specimens will be posted on this web site as well.

Research using human specimens can take a long time. The results of the research may not be determined for many years. You will not be informed of the results of that research, but may hear of important discoveries through the media or from your physician.

Human specimen research aims to provide better treatments for patients – it may lead to research involving the trial of new medications to treat your disease. The scientists using these specimens work closely with the physicians of the Leukemia/BMT Program of BC to bring new and more promising treatments to patients. Information about the type of research being done using donated specimens will be posted on this web site as well.

There may be times when a scientist may need to know more about you. This information helps the scientist learn more about the disease and how it has affected you. The information a scientist may need may include:

• The date of your diagnosis
• Your age
• Your gender
• Your general health history
• The treatment you have received
• Your family health history

Every effort will be made to protect your privacy. Each specimen donated by you will be given a coded number. All information that identifies you will be removed. All scientists who require further health information about you sign a confidentiality agreement. No information identifying you will be released in publications describing the research work. All information that identifies you will be kept behind locked doors or in secure computer files.

Research records and medical records identifying you may be inspected in the presence of representatives of Health Canada and the BCCA Research Ethics Board for the purpose of monitoring the research.

Your participation is entirely voluntary. At the time of your admission to hospital or when you visit the medical clinic for an appointment with your physician you will have an opportunity to read the consent form and decide if you would like to participate in this research. A clinical research nurse will review the consent and answer any questions you may have.

Your decision not to participate in this research will not affect the treatment you receive as a patient of the Leukemia Bone Marrow Transplant Program. You may withdraw at any time. If you withdraw, no further specimens will be collected from you. You can withdraw by notifying the Principal Investigator or the coordinator for the research project. The contact information is provided in the consent form.

Research is a process that moves forward in small and carefully planned steps. Usually research begins as basic science in the lab and after years of testing in cells and tissues, testing is done on animal models of human cells. After treatments prove successful in animals, these treatments may be tested on people through clinical trials. These trials are carefully designed and undergo rigorous review by nurses, doctors, research coordinators, as well as an ethics board before they are implemented.

A clinical trial is a research study designed to answer questions about safety and effectiveness of a drug or treatment. These trials follow a rigorous scientific process with built-in safeguards for participants who volunteer and are selected for research. There are two main types of clinical trials: interventional clinical studies and observational clinical studies. In the latter, the patients are strictly observed by the investigator and his team as they receive a therapy that is standard of care. On the other hand, in interventional trials, the participants will be followed as they receive a specific intervention – such as a new therapy or medication put forth in the study protocol. Many clinical trials will aim to either compare novel treatments to standard ones, to placebos that contain no active medication, or to no specific regimen.

Most clinical research involves the testing of a new drug in an orderly series of steps called phases. The process starts with small trials testing the safety of a treatment and then moves towards progressively larger trials. The larger trials compare the safety and effectiveness of the new therapy to the currently accepted standard of care.
It is important to remember that in a clinical trial, researchers do not know if the new treatment will be more effective than the current standard of care treatment.

Note that to participate in a clinical trial, patients must first meet screening criteria.

Phase I Trials

Summary

• Earliest trials in the life of a new drug or treatment
• Usually smaller trials; recruitment is low from 15 to 50 patients
• Since Phase I trials are usually the first time a drug is given to a patient, the first few patients, or cohorts, are given a small dose of the drug.
• The dose is then gradually increased with each group. The effect is monitored until the best dose is determined.
• Phase I trials require significant time commitment from participants because they involve frequent blood tests and side effects monitoring.
• Often patients with a cancer that lacks or does not respond to standard treatment are participants in Phase I trials.

Objectives

• To determine safe dose range
• To determine side effects
• To determine how the drug works in the body (pharmacologic behaviour) and how the body copes with and excretes the drug (pharmacokinetic studies)
• To determine how well the drug works (efficacy)

Benefits

• If the new drug or therapy has an effect on the cancer, participants may be the first to benefit

Risks

• Unpredictable side effects can occur

Phase II Trials

Summary

• To evaluate the effectiveness in a larger population, usually less than 100 patients
• Often focus on cancers for which no effective treatment exists or that are most likely to show a response to therapy
• All participants receive the same dose

Objectives

• To assess effectiveness of drug or therapy
• To assess for additional safety information

Benefits

• May benefit if treatment has an effect on the cancer

Risks

• Unpredictable side effects may occur
• Trials are often too short to determine long term benefits

Phase III Trials

Summary

• Large trials with 100+ participants
• Multi-centre, often global
• Survival time and quality of life measured
• Subjects are ‘randomized’ to a group: the Study Group — receiving study therapy, or the
• Control Group — receiving standard of care

Objectives

• To determine whether a new therapy or drug is more effective or less debilitating than standard of care

Benefits

• May be among the first to benefit if drug or treatment is shown to work

Risks

• New treatments are not always better than or even as good as standard of care
• Side effects may be worse than standard of care
• Unexpected side effects may occur despite preceding phase I and II trials
• Subjects in the Control Group who are receiving standard of care may receive less benefits than those in the Study Group who are receiving the study regimen.

A committee of nurses and doctors at the Leukemia/BMT Program reviews all clinical trials proposed by the Leukemia/BMT Program. The purpose of the Review Committee is to ensure that the trials are clinically and scientifically sound.

Once the protocol has been accepted by the Leukemia/BMT Program Review Committee, the study protocol must be approved by an Ethics Board. Before a trial can go ahead, the Ethics Board must be satisfied that the trial addresses an important scientific question, is well planned, is as safe as possible for the patients, and is ethically sound.

Prior to participating in any clinical trial, patients must sign a consent form which reviews details such as study goals, study background, description of medications and protocols that will be conducted. This ensures that patients are fully informed and thoroughly understand the study protocol and the risks and benefits of their participation.

Your medical information is confidential, but individuals involved in monitoring the conduct of the study and recording information on the treatments may need to access your records in the presence of a study investigator. These individuals may be representatives of the study sponsors, such as the Canadian Cancer Trials Group or the drug company supplying the trial drug. They may also be representatives from regulatory bodies such as Health Canada’s Health Protection Branch.

Any forms or paperwork recording your information and results will identify you by code number only, and your name will never appear in any publication resulting from the study.

You do not waive, or sign away, your legal rights by signing consent to a study. Taking part in a study is voluntary. This means you may choose not to take part or may withdraw your consent and leave the study at any time. If you withdraw from or decline a study, your doctor will discuss further treatments with you and continue to treat your cancer with the best means available. If there are other clinical trial opportunities relevant to you later in your treatment, these will be offered to you. Please note that withdrawing from a study will not hinder the quality of treatment you receive.

In addition to you deciding whether the trial is right for you, your hematologist will have to make sure you are right for the trial. All participants must meet the eligibility criteria as written in the study protocol. After the consent form is signed, tests to determine the status of your disease and other baseline tests, such as blood, urine, or heart tests may be necessary to determine your eligibility.

• What treatment options are available to me?
• Why is this study being done? What other studies with this medication have been done?
• Will I get a placebo?
• What inconveniences might I face, as part of participating in the trial? 
• How much additional time is required to participate in this trial? How long is the study?
• What are the main side effects of the study medication? What are the possible risks to me, compared to standard treatment?
• What are the pros and cons of participating in this clinical trial?
• Will there be reasonable reimbursement of the cost of my travel for study visits?
• What if I change my mind? What happens when the study is over?
• Can I take my regular medications while participating on the study? 

• Do I feel comfortable receiving a treatment whose risks and benefits are somewhat unknown? 
• Can I dedicate the time needed to participate in this trial? 
• Will I be able to manage if taking part in the trial means I might miss more work? 
• Am I satisfied that my decision is right for me, and not a decision that is being made to please someone else? 
• Do I have supportive family, friends or caregivers who can help me if I experience special problems such as nausea, tiredness, headaches, and diarrhea?

The information in this section is cited with permission from Dana-Farber Cancer Institute and the BC Cancer Agency web sites.