You may have heard about stem cell therapy in the news, or perhaps you or someone you know has been given a stem cell transplant, or is considering it to treat an illness or disease.
Stem cell therapy is a very promising new medical treatment. It holds the potential to treat degenerative diseases, cancer, and repair damaged tissues for which there are no current cures or therapies.
If you or someone you know is thinking of undergoing stem cell therapy, there are many questions that must be taken into consideration before receiving treatment. It is important for the patient to have all of the information and understand the risks involved with the procedure when speaking with their doctor.
What is stem cell therapy?
To understand stem cell therapy, it is important first to know what a stem cell actually is. Stem cells are the body’s raw materials. They are the starting point from which all other cells with special functions generate.
Stem cells divide and become what are called daughter cells. Daughter cells either become new stem cells (this is how stem cells self-renew so we don’t run out of them), or they become a new, specialized cell. This could be a blood cell, brain cell, heart muscle cell, or bone cell.
Stem cells are the only cell in the body with the natural ability to generate new cell types, so they are crucial to our health.
Stem cell therapy uses stem cells to cure or reduce the severity of a disease or disorder. They are typically used in one of two ways:
1. As a transplant
In this procedure, the desired stem cells are harvested either from the patient or from a donor, and then modified or refined in some way. They are then injected or grafted into the patient.
Stem cell transplants are used to replace stem cells or bone marrow that have been damaged or destroyed either by disease, or by radiation and chemotherapy during cancer treatment.
2. As a target for a drug or other biologic
In this case, doctors use a drug or biologic (a drug produced from living organisms) to activate a desired response from the stem cells that already exist in the patients tissues or organs.
What types of therapies are available?
You have likely heard of a bone marrow transplant, the most common form of stem cell treatment available today. It is also referred to as a hematopoietic stem cell transplant, and is a common treatment for blood diseases like leukemia or lymphoma.
What is a bone marrow transplant?
Some of the larger bones in your body have a soft, spongy center. This is called the bone marrow, and it produces all of the different cells that make up your blood, including red blood cells, white blood cells, and platelets, as well as the cells that make up your immune system.
The bone marrow is very active, producing millions of cells every hour. These cells remain within the marrow until they are transformed into red blood cells and released into the bloodstream, where they perform important functions like carrying oxygen, providing protection from infection, and helping your blood to clot.
A bone marrow transplant is often required upon completion of cancer treatment. Chemotherapy and radiation are highly toxic to the bone marrow, so after a patient undergoes treatment, they must have a healthy supply of stem cells reintroduced or transplanted.
Once the new cells have been transplanted, they can begin to reestablish blood cell production in the bone marrow. There are two types of bone marrow transplants, autologous and allogeneic.
Autologous means that before undergoing chemotherapy, the patient’s own stem cells are removed from the bone marrow and frozen. Once treatment is completed, the cells are thawed and returned to the patient.
Allogeneic means that the cells were taken from a donor, usually a family member like a brother or sister, or someone with similar genetic makeup.
There are, of course, risks and side effects associated with both types of transplants. Patients may experience mouth sores, abdominal pain, diarrhea, nausea and vomiting, or hair loss. There is also the risk for infertility, and that the treatment could damage other organs, like the lungs, liver, and bones.
With an allogeneic transplant, there is the risk that the donor will be rejected if it is not a good enough match, and as with any major surgery or medical treatment, there is always the possibility of death.
Other stem cell treatments
There are also various types of stem cell treatments for bone, skin, and eye illnesses or injuries, including spinal cord injury, heart failure, retinal and macular degeneration, tendon ruptures, and type 1 diabetes.
In Canada, all stem cell therapies must be approved by Health Canada before your doctor can offer them to you. Until then, any unapproved treatment is considered experimental or unproven.
What are unapproved therapies?
The term “unapproved” means that the treatment has not gone through the proper clinical trials to ensure that it is safe and effective. Often, a patient has to pay for it out-of-pocket, but there is not enough published research, if any at all, to prove that it will work for its intended purpose.
One unapproved treatment being offered by for-profit clinics is known as “autologous cell therapies”. Patients may also hear it referred to as “bone marrow aspirate concentration (BMAC) injections,” “stromal vascular fraction (SVF),” or “adipose-derived stem cells.”
These treatments use the patient’s own cells to treat their health condition, which is why the practitioner will argue that it is safe. Without being subjected to the proper testing, however, there is no way to be certain that there won’t be dangerous or potentially life-threatening consequences for the patient.
Autologous cell therapy is considered to be a drug under the Food and Drugs Act, and therefore must comply with the rules laid out within it. Health Canada has published five statements to warn Canadians about the potential health risks associated with an unapproved stem cell treatment. Including the following:
“Health Canada considers that there is the potential for life threatening or life altering risks from treatments with autologous cell therapies that have not been developed and studied in accordance with the Food and Drugs Act and the Food and Drug Regulations.”
Patients who wish to have access to an unapproved therapy can do so by participating in clinical trials offered by Health Canada that are investigating new products and treatments, of which little is known about their safety or efficacy.
Patients can verify whether or not a treatment is authorized by referring to Health Canada’s Drug Product Database and Clinical Trials Database to confirm cell therapies that are authorized, and cell therapies that are undergoing clinical trials, respectively.
What questions should I ask my doctor?
If you or someone you know is considering stem cell therapy, it is important to be well-informed during every step of the process. Here are some questions to ask your doctor before receiving treatment:
What type of stem cell transplant are you recommending for me?
There are different types of stem cell transplants, and the one you receive will depend on multiple factors including your disease or illness, your age, and your health status, among others.
The treatment you will receive will dictate what is needed from you before the procedure, as well as what to expect in terms of recovery.
How will you find a donor?
If you are undergoing an allogeneic transplant, you will require a donor who is a good genetic match. Your doctor will first look through your immediate family, such as a sibling, parent, or child.
You should have detailed knowledge about their medical histories, including a history of cancer, heart problems, heavy smoking, or anything that is considered an alternative lifestyle.
Will I need other types of treatment?
In the case of cancer or other type of disease, stem cell therapy is usually part of a treatment plan, which often involves chemotherapy.
In the case of lymphoma or other type of cancer, you will receive chemotherapy first to kill the cancer, or in some cases the defective bone marrow. Once that is complete, you will then receive a bone marrow transplant.
In some cases, patients will receive follow-up therapy such as low-dose chemotherapy or radiation after the stem cell transplant is complete. Depending on the disease for which they are being treated, a patient could be put on some type of maintenance plan for years following the procedure.
What are the risks associated with a stem cell transplant?
Stem cell transplants as part of a cancer treatment plan are usually very successful, and have put many cancer patients into remission. There are, however, some risks associated with the procedure that should be discussed with the patient’s doctor.
Some patients may experience side effects from pre-transplant chemotherapy, graft-versus-host disease, or general infections. Patients should speak with their doctors about possible fatal complications from the procedure, and what is going to be done to lower their risk for treatment-related death.
What is graft-versus-host disease?
This is a possible problem that could arise when undergoing an allogeneic transplant, in which cells are donated from another person. Sometimes, a patient’s cells will recognize the donor cells and foreign and attack them.
This could cause mild symptoms, like a rash or loss of appetite, or could cause more severe issues like diarrhea or jaundice. The condition can be life-threatening, however early detection can prevent long-term complications.
Before undergoing treatment, patients should talk with their doctor so they understand the symptoms of graft-versus-host disease, which will allow them to seek care earlier rather than later.
As a preventative measure, patients who are given an allogeneic stem cell transplant are often given immuno-suupressive medications.
How long will I have to stay in the hospital during treatment?
The typical hospital stay for a stem cell transplant lasts between four and six weeks. After that period, it is recommended that patients stay close to the hospital, if possible, to allow for close monitoring, and quicker response should there be any complications.
What will my life be like after treatment?
Provided everything goes well, you will most likely feel relatively normal after you have recovered from treatment. Despite this, it is important to understand that your body will remain in “recovery mode” for quite some time afterwards.
Your immune system will be suppressed for anywhere from 6 months to one year, and so you will have to be immunized like an infant, to reset your entire immune system. You will have to be much more cautious about your daily activities, and avoid scenarios that could expose you to germs.
Questions for unapproved therapies
If you’re considering an unapproved stem cell therapy, you should be even more cautious about the type of treatment you are seeking. There are several important questions you should ask the clinician offering the procedure before you agree to undergo treatment.
What types of cells will be used for the procedure, and why?
This is a basic question that should be asked to understand why and how the treatment will be effective. The practitioner should have a scientific basis from which the procedure was developed, and be able to provide some research-based evidence to support the efficacy of the treatment.
Does the clinic offer stem cell treatments for a variety of conditions?
If the answer to this question is yes, this is a warning sign that the treatment is not legitimate, will not work, and may not be safe. This is because stem cells are specific to certain tissues, and cannot be used for other parts of the body unless they are manipulated in a lab.
For this reason, it is very unlikely that the same stem cell treatment will be effective for multiple different tissues in the body. You should be wary of clinics that claim their one treatment will be effective for multiple health issues.
What are the promised benefits of the procedure, and can you provide evidence that it will work?
The staff should be able to provide some peer-reviewed, scientific research that demonstrates their stem cell treatment has proven benefits. If they don’t have this information, it is most likely not worth the money or the potential risk of undergoing treatment.
Is the treatment part of a formal clinical trial?
First and foremost, the trial should be registered. This does not guarantee its credibility, but unregistered trials should be avoided altogether. The trial should meet national regulations and there should be some patient protection measures in place.
The trial should also have independent oversight, meaning it should be evaluated by experts who are not a part of the trial, and you should be aware if any of the researchers have a conflict of interest, such as the potential for financial gain, and what their qualifications are.
Finally, it is important to find out whether undergoing this treatment could prevent you from participating in future clinical trials.
What benefits should I expect?
The researchers should clearly lay out for you what to expect from the procedure, how they will measure its efficacy, and how long it will take before you begin to notice the benefits.
They should also inform you of any other medications or special care you may require before or after the treatment.
How is the procedure done?
The practitioner should be able to clearly answer all of the following questions:
- What is the source of the stem cells?
- How are the stem cells identified, isolated, and grown?
- Are the cells differentiated into specialized cells before therapy?
- How are the cells delivered to the right part of my body?
- If the cells are not my own, how will my immune system be prevented from reacting to the transplanted cells?
What are the risks?
You should find out all of the potential health risks to the procedure, as well as what will be done in the event that you develop an adverse reaction to it. You should be aware of who the contact person is in case of emergency, and who will provide emergency medical care.
What is the success rate of the procedure?
If you can find out how many other people have been treated in the same way for your condition, and what the success rate for those treatments has been, it will give you a better idea as to whether or not your treatment will be successful, too.
You should ask if and where those findings have been published, so you can read and verify them yourself.
What are my rights as a patient?
It is important to have a clear understanding of your rights as a participant, including confidentiality, the right to be informed of any new information, and the right to withdraw from the trial.
You should also ask about what type of compensation you are entitled to should you be injured as a result of the study.
How much does the treatment cost?
This includes not only the cost of the initial treatment, but also what other costs might come up as a result of the procedure, including follow-up care, as well as costs associated with emergency treatment if something goes wrong.
Be an active participant in your own care
Any time you are seeking medical care, it is your responsibility as a patient to ask questions, and ensure you have full and complete understanding of the the goals of the procedure, the potential risks, the efficacy of treatment, and what your rights are as a patient.
Always be wary of unapproved treatments, and if you do decide to pursue an unapproved therapy, you should be even more vigilant to ensure that you are not wasting your time and money on an ineffective treatment, and even more importantly, that the treatment is safe.
The future of stem cell therapy
There is currently a lot of promising research in the field of stem cell therapy for many conditions, and we will begin to see new therapies in the near future. It is important that all new therapies meet the requirements laid out by Health Canada to ensure that they are both safe and effective.
In the meantime, always be cautious when considering unapproved therapies, and never agree to a treatment that has little to know scientific evidence to prove that it will work.
- Risk factors in the development of stem cell therapy
- Stem cells, what they are and what they do
- About stem cell therapies
- Biologics: definition, side effects, uses, and drug list
- Stem cell transplant
- Stem cells: past, present, and future
- Health Canada is advising Canadians about the potential health risks associated with unauthorized cell therapy treatments such as stem cell therapy
- Health Canada policy position paper- autologous cell therapy products
- Patient education: Hematopoietic cell transplantation (bone marrow transplantation) (Beyond the Basics)