Cell Therapy in Regenerative Medicine

Frost & Sullivan forecasts the regenerative medicine market to reach $67.5 billion by 2020, clocking a compound annual growth rate of 22.5% over the next three years. Read how cell therapy is expected to contribute more than $10 billion to that market by 2020.

The term “regenerative medicine” was coined by Leland Kaiser, a multifaceted personality renowned in academic circles for his authority on the health care system, and beyond as a motivational speaker and a futurist. In an article titled “The Future of Multihospital Systems” (published in 1992), Kaiser predicted that a field would emerge that would focus on regenerating “tired and failing organ systems.” Writing in the Top Health Care Finance journal, he predicted that this new branch of medicine would change the course of chronic disease treatment and impact the future of hospitals. William Haseltine, an academician and researcher famed for his work related to cancer and HIV/AIDS, further elucidated this concept. In a seminal conference in 1999, Haseltine described regenerative medicine the way it is understood today: as an approach that brings together tools and technologies, such as genes, cells and factors supporting cell growth, to replace or regenerate tissues affected by disease or trauma.

Regenerative Medicine: A Growing Market

The vast majority of the treatments available for chronic diseases are palliative. The lack of proven therapies and the long gestation period before they get the necessary regulatory clearance further add to patient woes. By 2030, more people in the world will be aged 60 or older than aged 10 or younger. Already, there are more adults over 60 than children under 5. The aging population confronts a near-barren landscape of therapies for chronic diseases. After several years of basic research, regenerative medicine is beginning to translate into a clinical option.

The desired end result of organ or tissue regeneration is achieved along four lines: cell therapy, gene therapy, tissue engineering, and small molecules and biologics. Frost & Sullivan forecasts the market to reach $67.5 billion by 2020, clocking a compound annual growth rate of 22.5% over the next three years. Cell therapy is expected to contribute more than $10 billion to that market by 2020.

As the name indicates, cell therapy uses living cells to replace or augment diseased tissues. The largest application for cell-based products is chronic wounds and other dermatological conditions. This is due, in part, to the fact that these products were the first to make it through the U.S. Food and Drug Administration (FDA) clinical trial pipeline. The next wave of new products will be in the areas of cancer, cardiovascular and ophthalmic therapies. A major component of cell therapy involves the use of stem cells. Despite periodic protests about the ethics of harvesting and using stem cells, this has become a popular therapy option.

Cell Therapy: Leading Commercial Products

A snapshot of leading cell therapy products is presented here.

Apligraf by Organogenesis, Inc. (Canton, Mass.)

Apligraf was the first bioengineered, cell-based product to receive FDA approval, and is the only FDA-approved product to treat both venous leg ulcers and diabetic foot ulcers. Apligraf is supplied as a living, bi-layered skin substitute composed of neonatal foreskin keratinocytes and fibroblasts with bovine type I collagen.

ReCell Spray from Avita Medical (Cambridge, United Kingdom)

ReCell is used on patients who require non-conventional treatment for chronic wounds, burn wounds, skin abnormalities and cosmetic procedures. A physician excises a small piece of skin tissue (a 1- to 2-square-centimeter biopsy), which is broken down into live, healthy cells through a proprietary cell sorting mechanism. These cells are collected; however, the cell breakdown simulates cells to secrete an effusion of growth factors, cytokines, extracellular matrix proteins and a cocktail of other biomolecules that are beneficial for tissue regeneration. This cell suspension, known as Regenerative Epithelial Suspension (RES™), can simply be sprayed onto the patient’s injury site or skin condition.

Trinity Evolution by Orthofix, Inc. (Lewisville, Texas)

Trinity Evolution is a bone allograft consisting of osteogenic cells and mesechymal stem cells (progenitors of bone cells) in the natural bone matrix, ideally suited to address musculoskeletal defects. Trinity Evolution walks a middle ground between autografting and seeding the patient with embryonic stem cells. The product uses bone grafts harvested from donors, and the multipotential mesenchymal stem cells have the ability to develop into any kind of cell as needed and depending upon the growth environment. Trinity Evolution has been approved for clinical use in the United States and in Europe, and has been used to treat more than 90,000 patients so far.

Cell Therapy: Innovations on the Horizon

Cell-gene therapies include the use of stem cells or non-stem cells to deliver new, lifesaving DNA treatments to patients. Therapies often involve the use of gene editing tools in combination with vector delivery systems.

CTL019 from Novartis (Basel, Switzerland)

Still under development, the CTL019 therapy uses chimeric antigen receptor T-cells (CAR-T cells) engineered to specifically target B cells (a type of white blood cell) that become cancerous in certain lymphomas, such as childhood acute lymphoblastic leukemia and adult chronic lymphocytic leukemia, as well as types of blood-related cancers. The progress on clinical trials has been slow because patient enrollment has been limited.

JCAR017 from Juno Therapeutics (Seattle, Wash.)

This is a promising CAR-T cell therapy that has been developed to treat relapsed/refractory diffuse large B-cell lymphoma. The JCAR017 therapy platform comprises a specific ratio of CD4+ and CD8+ T-lymphocytes, setting it apart from other candidates designed for the same indication. Another Novartis candidate (JCAR015—until recently considered a promising therapy) has been halted in its development following adverse results in a clinical trial. JCAR015 notably had a different formulation of CD3+ lymphocytes. In March 2017, Juno Therapeutics said that it had fast-tracked the development of the JCAR017 therapy and has announced encouraging early results from Phase I studies.

The Road Ahead: Tip of the Iceberg

The $10 billion potential of cell therapy does not present the entire story; a far greater future may be in store. One of the indicators of this is that, of the more than 700 regenerative companies in the world, more than 500 specialize in cell therapy. These companies—more than half of which are in the United States—have thousands of therapy offerings in development that will soon cause an explosion of lucrative revenue. Frost & Sullivan estimates that nearly 85% of the more than 500 cell-based products undergoing clinical trials are in their early stages, implying a long and well-stocked pipeline for sustained market growth.

Large pharmaceutical companies such as Pfizer, Novartis and Juno Therapeutics are entering the market because there is growing evidence of the safety and efficacy of numerous products in the pipeline. The curative potential of some of the cell-gene therapies is driving the creation of new partnerships, mergers, and acquisitions, which would augur well for the numerous small companies dotting the landscape.

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