What are the only stem cell therapy conditions currently approved in Canada?

As of 2020, Canada has approved a total of 3 cellular therapeutic products! These include the use of stem cells for the treatment of acute lymphoblastic leukemia (ALL), Adult B-cell Lymphoma, and Graft V Host disease.

Most approved therapies use hematopoietic stem cells (HSC). Bone marrow, blood, and the umbilical cord all contain HSCs. HSCs mature into cells that make up the blood and immune system. This makes them perfect for the treatment of conditions such as leukemia, lymphoma, and blood disorders. We can now treat over 80 medical conditions with the HSCs collected from cord blood.

Three cellular therapeutic products do not sound like enough. To understand why there are a few approved stem cell therapies on the market we must first ask the following questions:

How do stem cell therapies get approved in Canada?

In Canada, all cell therapies are considered drugs under the Food and Drugs Act. They must be authorized by Health Canada to ensure they are safe and effective before they are offered to Canadians. All other treatments are experimental or unproven. Canadians should refer to Health Canada’s Drug Product Database [2]and Clinical Trials Database [3].

How do stem cell therapies work?

Stem cell therapies can work in one of two ways to reduce the severity of a disease or disorder. The first is a stem cell transplant, and the second one is to act as a target for a drug or other biologic. A stem cell transplant is when existing stem cells in your body, which have been damaged or destroyed by disease, are replaced. The stem cell transplant can either be allogeneic or autologous.

  • An allogeneic transplant involves a person (donor) giving his/her stem cells to another person (recipient).
  • An autologous transplant involves a person giving his/her stem cells, which are then manipulated or processed and reintroduced to the same person.

Stem cells can also act as a target for a drug or other biologic. In this type of treatment, the drug activates a desired response from the stem cells that already exist in the patient’s tissues or organs.

The future of stem cell treatments is dependent on Induced Pluripotent Stem Cell (IPSC) technology. This technology involves reprogramming skin or blood cells into an embryonic-like pluripotent state. Cells in a pluripotent state can generate any other type of cell in the human body.

The promise of stem cells lies in the clinical trials

Unapproved therapies are those that have gone through the safety and efficacy process through clinical trials. Clinical trials are research studies performed in people to find out if a new treatment is safe and effective. There are approximately 1300 trails underway that use HSCs to address conditions such as[5]:

  • Cerebral Palsy
  • Diabetes
  • Multiple Sclerosis (MS)

Mesenchymal stem cells (MSCs), found in cord tissue and placental tissue, can differentiate into many types of cells and tissues. These include cartilage, skin, bone, and fat cells. There are more than 300 major MSC clinical trials underway worldwide for conditions such as:

  • Spinal Cord Injuries
  • Lung Cancer
  • Type 1 diabetes
  • Stroke

Finally, IPSC based clinical trials have been at the forefront of stem cell therapeutics. In 2019, heart surgeons used IPSC-derived cardiomyocytes to treat patients with heart disease. There is hope to treat patients with Parkinson’s Disease by 2022[6].

With so many ongoing trials, those who choose to invest in cord blood, placental tissue banking or live cell tissue banking are not just investing in the medicine of today, but recognize the future of stem cell and regenerative medicine.

Cell Therapy in Canada: Striking the Right Balance Between Regulation and Innovation

When it comes to the development of new health technologies and drug treatments, we have to be patient. In fact, the stakes couldn’t be higher because not taking the correct approach, with proper oversight at each step, could have dire consequences, including injury — or even death. That’s why it can take years, even a decade, for new forms of medicine to be tested for safety and efficacy before they’re approved.

This is exactly why we need to take a science-based approach when it comes to regulating cell therapy treatment, or regenerative medicine, which will play a major role in the future of healthcare. According to the Mayo Clinic, patients who might benefit from these forms of treatment are extremely diverse, including those suffering from Type-1 diabetes, Parkinson’s, Alzheimer’s, burns, cancer and strokes, just to name a few. There is no shortage of excitement from physicians, scientists and patients.

While the benefits of cell therapy are promising, the regulatory and oversight framework in Canada will need to keep pace with advancements made in the sector on a global scale. According to the Alliance for Regenerative Medicine, there are 906 gene and cell therapy companies worldwide, with just over 1,000 clinical trials currently taking place. The industry is also growing rapidly. In 2018, the field raised $13.8 billion internationally, an increase of 73 per cent from the year before.

Recognizing this, Health Canada recently launched consultationswith stakeholders to “modernize its regulatory framework to meet the new demands of today’s increasingly complex health technology while still protecting the health and safety of Canadians.” The government should be applauded for reaching out and engaging with industry leaders, since taking a collaborative approach backed by informed expert opinion will ensure we foster innovation while upholding patient safety, which is the top priority.

As the founder of a company that does not administer cell therapy treatment but collects live cells and maintains them through cryopreservation for possible future therapeutic use, we fully support and encourage the development of new clinical trial regulations that can establish Canada as a world leader in regenerative medicine.

But this country isn’t alone when it comes to foreseeing the large-scale impact cell therapy treatment will have and the need to play regulatory catch-up with rapidly advancing technology.

Earlier this year, the Food and Drug Administration (FDA) in the United States announced plans to accelerate cell therapy approvals.The FDA expects it will receive some 200 investigational New Drug Applications, which are requests for authorization to administer an investigational drug to humans.

This increased focus on regenerative medicine has prompted the FDA to hire an additional 50 staffers to expedite the approval process. The fast-track process is not to be confused with final approval for use with the general public. Rather, it’s an “accelerated approval pathway may offer a faster route to approval for new treatments, including potentially curative benefits in significant, unmet medical needs.”

As with any new treatment in medicine, there will always be a debate on its future impact on patients. While there are differences of opinion regarding the timelines for regenerative medicine treatments, one thing is clear: The industry is growing on a global scale and the federal government has taken notice. Canadians should feel confident that Health Canada is taking the right steps in consulting with industry to ensure their safety is protected and that our nation remains a world leader in cell therapy innovation.

I believe that our own cells are the next currency in healthcare, opening up regenerative medicine treatments and next-generation analytics, and I am excited about the role Canada is taking in this bright future.