Frequently Asked Questions

Clinical trials are experiments that use human subjects to answer a scientific question. Uses of clinical trials include testing if a new therapy is safe to use or if it is more effective than the standard treatment for a disease. It is important to note that there are risks associated with all new medical treatments, so clinical trials are carefully designed and are very carefully regulated by government agencies to ensure that the health and welfare of patients are protected. In Canada, this regulation is overseen by Health Canada; in the United States, the regulating body is the Food and Drug Administration (FDA).

Clinical trials that are used in the development of new treatments are generally divided into stages or phases:

• Phase I – Usually involve a small number of patients to test the feasibility and safety of a new *clinical intervention.
• Phase II – Continue to test a therapy’s safety, but also begin testing the effectiveness of the therapy. Phase II trials are usually only slightly larger than Phase I trials.
• Phase III – At this stage the number of patients in the trial is increased and the main focus is understanding how effective a new therapy is as a treatment for the disease.
• Phase IV – Once a therapy has been approved by the health regulator and is available through commercial means, this phase can be used to perform a longer-term study of the treatment risks and benefits in a much larger group of patients.

*Clinical intervention definition: An intervention carried out to improve, maintain or assess the health of a person, in a clinical situation

Many potential new treatments do not advance beyond Phase I or II for a variety of reasons, including identified safety concerns, lack of efficacy compared with currently approved options, or costs associated with the trial and/or treatment itself.

SCN cannot provide medical advice on any health-related issues. SCN encourages individuals to consult with their licensed General Practitioner (GP) or specialist and seek specific medical advice from them on treatment options, including the suitability of any new stem cell-related therapies or whether they qualify for an ongoing clinical trial.

If you are interested in participating in a clinical trial, please contact your GP or specialist with the specific trial details and request their advice and assistance to understand if you are eligible to participate in the trial.

Health Canada hosts a Clinical Trials Database that lists all trials in Canada that have met the regulatory requirements in Canada.

ClinicalTrials.gov and the ISRCTN are searchable registries that contain information on current and upcoming registered clinical trials. Most of these trials are occurring outside of Canada, and it is important to note that just because a trial is listed on one of these registries does not mean that it has passed the regulatory requirements in that country.

Participating in a Phase I, II or III clinical trial is not a way to receive treatment as it is uncertain whether the intervention being tested in the trial is effective. Only about 1 in 10 interventions brought to a phase I trial become a commercial product. Clinical trial participation offers patients a more active role in research and permits patients to make a valuable contribution to the development of potential new therapies. Patients participating in clinical trials receive close monitoring of their response to the new therapy that is being tested during, and sometimes after, their enrollment in a clinical trial.

There is no guarantee that a new intervention will work, and even if it does help some patients enrolled in the trial, it may not work for you. New treatments may have serious side effects. Participation in a clinical trial can take much more time and require more appointments, medical interventions and tests than a routine treatment that is the standard of care.

Unapproved stem cell products/therapies are marketed as effective treatments for a condition despite not having been proven by rigorous clinical testing to have a therapeutic benefit in patients. These products may include surgical and non-surgical procedures, injections, treatments, drugs and topical ointments/creams. An unapproved stem cell product has not passed through the regulated clinical trial process and therefore has not been approved for regular use by Health Canada (or similar agency in other countries). However, loopholes in the regulation have created an opportunity for some clinics to offer these unapproved products/therapies as a fee for service. These clinics operate around the globe, including in Canada. There are many factors to consider if you choose to purchase an unapproved stem cell therapy:

• There may be serious health risks associated with the unapproved therapy;
• There is no published evidence that the unapproved therapy works;
• By choosing an unapproved therapy you may become ineligible to participate in an approved clinical trial or receive approved therapies in the future;
• The quality, identity or safe production of the product is not guaranteed.

If you are uncertain about whether a therapy you are considering is approved or safe, there are a number of questions you can ask. SCN recommends these questions, published by the International Society for Stem Cell Research.

No clinical trial for a new therapy is without risk to the patient. However, ensuring that the trial is regulated by a government agency, such as Health Canada or the Food and Drug Administration in the USA, provides reassurance that the safety and interests of the patient has been a primary factor in the design and implementation of the trial. SCN only funds clinical trials that meet Health Canada regulatory requirements.

Additional information from the International Society for Stem Cell Research:

• Nine Things to Know About Stem Cell Treatments
• Patient Handbook on Stem Cell Therapies