Stem Cell Therapy, What Is It and How Does It Work?

Stem cell therapy is a cutting-edge field in medicine that leverages the unique regenerative abilities of stem cells to treat various diseases and injuries. These therapies use stem cells—undifferentiated cells that can develop into different types of cells in the body—to repair or replace damaged tissues or organs. While some applications are in the experimental stage, several therapies have gained approval from regulatory bodies like the U.S. Food and Drug Administration (FDA), reflecting their efficacy and safety.

How Stem Cell Therapy Works

Stem cells can be sourced from different origins:

• Embryonic Stem Cells (ESCs): Derived from early-stage embryos, these cells can differentiate into any cell type but raise ethical concerns. For this reason, it is extremely uncommon for stem cell companies to use ESCs.
• Perinatal Stem Cells: Perinatal stem cells are multipotent cells found in tissues associated with childbirth, including cord blood, cord tissue, and the placenta. These cells are often leverage their regenerative properties due to their ability to differentiate into multiple cell types and their immunomodulatory functions.
• Adult Stem Cells: Found in tissues like bone marrow, adipose tissue (fat), peripheral blood, and dental pulp, these cells are more limited in the types of cells they can become. Currently, this is by far the most common type of stem cell used in treatments worldwide.
• Induced Pluripotent Stem Cells (iPSCs): These are adult cells that are genetically reprogrammed to behave like embryonic stem cells. They have the capacity to become any of the nearly 200 cell types that compose a human being. While there is not yet an iPSC-derived cellular product that has been approved by any regulatory body worldwide, progress with this cell type is rapidly advancing.

Once collected, stem cells are processed, sometimes genetically modified, and then administered to the patient through injections, infusions, or surgical implantation. Their goal is to promote the repair of damaged tissues, mitigate disease progression, or restore normal function.

Though many clinics market unapproved stem cell treatments, the U.S. FDA has approved 43 cell and gene therapies that have undergone rigorous testing.

Global Innovations in Stem Cell Therapy

Outside the U.S., other countries are also advancing the development and regulation of stem cell therapies:

• Japan: Known for its leadership in iPSC technology, Japanese researchers and clinicians have been pioneering treatments for Parkinson’s disease, several types of macular degeneration, spinal cord injury, and limbal stem cell deficiency since iPSCs were first discovered in 2006.
• South Korea: South Korea is leading the way in global MSC-based cell therapy approvals. At present, five of the 12 globally approved MSC-base cell therapeutics (42%) have been approved in the country. These MSC-based cell therapy product approvals include:
  • Queencell from Anterogen
  • Cellgram AMI from Pharmicell
  • Cupistem from Anterogen
  • Cartistem from Medipost
  • NeuroNataR from Corestem.
• Europe: Therapies like Holoclar highlight the continent’s advancements in niche treatments​. Holoclar is a stem-cell-based therapy designed to restore damaged epithelial cells on the surface of the cornea, aiding in the repair and regeneration of the eye’s outermost layer. Previously, Alofisel—a stem cell therapy used to treat perianal fistulas from Takeda/TiGenix—was approved by the EMA as well, but it was later withdrawn.

Despite its promise, stem cell therapy still faces quite a few regulatory and strategic challenges, including:

• Safety Concerns: Unregulated clinics offering unapproved treatments pose risks, including infections, immune reactions, and tumor formation​.
• Regulatory Scrutiny: The complexity of stem cell production and use necessitates stringent oversight to ensure patient safety and therapeutic efficacy.
• Lack of Approvals: Currently, there are no FDA-approved therapies using embryonic stem cells (ESCs). While ESCs hold significant promise due to their ability to differentiate into any cell type, their use in clinical applications has been limited by ethical concerns, technical challenges, and the availability of alternative stem cell sources, such as iPSCs and adult stem cells​. At this time, there are also no iPSC-derived cellular therapeutics in any major healthcare market worldwide. However, this could change in the near future because there are at least 72 different companies that are exploring the therapeutic potential of iPSC-derived therapies for use in human patients.

The Future of Stem Cell Therapy

Stem cell therapy holds immense potential to revolutionize medicine, particularly for conditions previously considered untreatable, such as neurodegenerative diseases and certain genetic disorders. Continuous advancements in stem cell research, supported by robust regulatory frameworks, are crucial to translating this potential into broader clinical applications.

Stem cell therapies have already transformed lives, offering hope where traditional treatments fall short. Companies like Gamida Cell, the New York Blood Center, Mesoblast, Cynata Therapeutics and others lead the charge, representing the strides made in this transformative field.

Ready to Learn More?

As the world’s largest publisher of stem cell industry news, BioInformant is committed to providing timely industry updates. However, as a publisher, we understandably cannot offer medical advice or treatments. To support you, we’ve developed a 7-page guide to help educate and answer your questions:

Stem Cell Fact Sheet – Types of Stem Cells and Their Use in Medicine