The basic theory behind cell therapy was stated best by Paracelsus, a 16th-century physician who wrote: “Heart heals the heart, lung heals lung, spleen heals spleen; like cures like.” Paracelsus and many other early physicians believed that the best way to treat illness was to use living tissue to rebuild and revitalize ailing or aging tissue. Modern orthodox medicine lost sight of this method, so it now uses chemicals to interrupt or override living processes. While chemicals and drugs work only until they are broken down by the body’s metabolic processes, cell therapy has a long-term effect, because it stimulates the body’s own healing and revitalizing powers.
Regenerative medicine is an emerging field that aims to repair, replace, and/or regenerate damaged tissues and organs in vivo (in the living body) by stimulating previously irreparable organs into healing themselves. Cell therapy, meaning using healthy cells or cell components to replace diseased, damaged or dead cells, is a broad umbrella term capturing many elements of treatments derived from the human body’s cells or cell constituents. Some of the better known types of treatments in this class include cells and proteins from the blood (i.e. platelets or mononuclear cells) and bone marrow, but medical science is advancing the field quickly to include using our own immune cells and fat cells in ways we couldn’t have imagined twenty years ago. Regenerative medicine holds the potential to treat previously incurable chronic diseases and conditions including Alzheimer’s disease, Parkinson’s disease, diabetes, heart disease, renal failure, osteoporosis and spinal cord injuries, to name a few. The most obvious societal benefit of regenerative medicine is the possibility of significantly reduced healthcare costs from such treatments.
Sources of cell therapy include embryonic and adult stem cells. Embryonic stem cells are derived from human embryos while the sperm and egg are undergoing the very early phase of cell division. Embryonic stem cells are derived during the blastocyst stage of embryonic development, so their potential power for generating any new tissue is immense. Embryonic stem cells are pluripotent in nature and show significant promise for the future of medicine. Isolating the embryoblast or inner cell mass results in destruction of the fertilized human embryo which that raises ethical issues for its use in the standard practice of medicine.
Adult (Somatic) stem cells are unspecialized cells that are found in different parts of the body and, depending on the source tissue, have different properties. Adult stem cells are capable of self-renewal and give rise to daughter cells that are specialized to form the cell types found in the original body part. Adult stem cells are multipotent in nature. The sources include Peripheral Blood, bone marrow, adipose, cord blood and embryonic stem cells. Peripheral Blood Cells are the rich source of platelets and platelet derived growth factors. Using a patient’s own platelets for improving soft tissue healing has been in use for more than a decade, and new rapid, point of care technologies devices to isolate these cells have recently been developed. Bone marrow is the central reservoir and “incubator” for the body’s own immature immune cells, new blood cells, and stem cells. The use of bone marrow derived cells is widely used in various cancer treatments currently across the world, and is becoming the most consistently effective source of cells for natural biological repair. Adipose Cells or “fat” has been used for several years in cosmetic and reconstructive surgery. However, science has begun to understand that adipose houses a significant pool of stem cells as well. Many new technologies are coming to market now allowing surgeons to use the patient’s own fat to help repair or regenerate damaged or diseased areas of the body. This is extremely important in cosmetic and reconstructive surgery. Cord blood, a significant source of residual blood in the umbilical cord and placenta typically discarded after childbirth consist of young naïve and potentially potent cells for various cellular therapies. Cord blood is typically cryopreserved in liquid nitrogen and can be made available upon future use. Over the past decade, cord blood transplants have started reshaping the way in the treatment of pediatric hematological based cancers, genetic disorders and autoimmune disorders. Cord blood is in clinical trials for potential therapeutic benefits in brain injury diseases such as cerebral palsy.
Although the roots of regenerative medicine go back 30 years, the field is in its commercial infancy for several reasons (i) Cell-based biological products represent both new technology and a business model that vary from traditional drug or device development. (ii) Start-up biotech and cell therapy companies lack the financial means and the clinical, regulatory, and manufacturing capabilities necessary to establish a sustainable product portfolio and technology pipeline. The search for innovative and viable business models is a pressing challenge for established and emerging healthcare industries given long-term declines in the productivity of therapeutic pipelines. However, the cell therapy industry has been here before and successfully built viable business models around innovative healthcare products and services.
After 30 years of research, many regenerative medicine technologies are in development and approaching commercialization. Clinical programs are underway worldwide and patients can look forward to numerous innovative solutions to pressing global health issues in the years ahead. The current market for regenerative medicine is estimated at between $2-5 billion USD. It is projected that the stem cell market including therapeutics, cord blood banking and drug development tools will achieve annual growth of 29.2% resulting in sales of more than $11 billion by 2020. Venture capital investors also see near-term revenue opportunities for companies making tools for the industry or using stem cells for drug discovery and development. The 60% share of the global stem cell market is currently held by US; and forms an especially lucrative market for areas such as bone marrow transplantation through stem cells. This research report will help stakeholders gain a better understanding of product pricing and regulatory framework governing the dynamics of the Cell therapy market. The key players in the market include Osiris Therapeutics, Stem Tronix Inc., Stemcell Technologies, Genzyme Corp., Cytori, and Geron.
173 companies currently have products in clinical trials, comprised mainly for therapeutic treatments. These companies already have 68 products on the market compared with only 25 in 2012. There has been significant progress to bring regenerative medicine products to market with 25% products in Phase I or late stage clinical development, 45% products in Phase II, 15% products in Phase III trials and 15% products are commercially distributed respectively. Over one third of global regenerative medicine companies are now involved in biomaterials development. Biomaterials refer to any natural or synthetic material that interfaces with living tissue and/or biological fluids. Polymers, metals (titanium, stainless steel, cobalt-chromium alloys), ceramics (alumina, zirconia, silicon nitride), and composite materials which can be developed into biocompatible materials are presently used in hip and dental implants and cardiac stents. These biomaterials can be modified to incorporate biological activity, such as growth factors and cells, aid in transplantation or expedite the healing process. Applications for tissue engineering and biomaterials were originally limited to prosthetic devices and surgical manipulation of tissues but now include Bone, Cardiac, Liver, Corneal tissue engineering,ƒƒ Development of biomaterial scaffolds and wound healing.
Regenerative medicine is expected to begin bringing complex skin, cartilage, and bone substitutes to market within two years. As stakeholder familiarity with new technologies is established, new business models will also be identified for cellular therapies. The leading multinational pharmaceutical companies have made a commitment to the field as evidenced through internal discovery programs and by establishing major research collaborations with leading academic groups worldwide. The maturation of the industry is also reflected by a high number of clinical development activities. The National Institutes of Health, US is tracking over 900 clinical trials involving adult stem cells for therapeutic applications such as heart disease, diabetes, severe burns, bone regrowth, and vision impairments. Moreover, several FDA approved cell therapies for immune disorders and repopulation of bone marrow following chemotherapy are already on the market.
There is a vast opportunity for new entrants in the field of cellular therapy to use global strengths in fundamental scientific research and commercialization strategy to lay a foundation for global leadership in this emerging industry. The fact is that one should understand that cell therapies are not cheaper or faster to develop as compared to predecessor biotherapeutics, and have yet to produce profound clinical efficacy over standard medical care. The Indian stem cell industry is growing and there is potential for fast growth and global players will enter the market through mergers and acquisitions. With the benefits of stem cell therapy growing, it is attracting several organizations and industry to invest, create and commercialize promising technologies. The market for stem cells will include specific products, services and technologies to aid in diagnosis to cure. Most of the Indian companies are focused on stem cell banking and research, the study released recently stated. Indian market forecasts an annual growth rate of around 15 per cent, mainly driven by investments from the Government and private players. The Indian Government has been quite proactive in promoting work in stem cells, especially in clinical trials, basic research and creating applications. The Department of Biotechnology has invested $60 million into stem cell research during 2005-2010. The National Centre for Biological Sciences, Bangalore, is a major player. Similarly, the Hyderabad-based Centre for Cellular and Molecular Biology has set up a clinical research facility for stem cell technologies. Other important centres for stem cell work are National Centre for Cell Sciences at Pune, LV Prasad Eye Institute, Hyderabad, Centre for Stem Cell Research at CMC, Vellore, National Brain Research Centre, New Delhi and so on, the report identified.
Not only the new and successful business models appear over the next fifteen years, the prediction is that there will also be a range of robust scientific, bio-manufacturing and regulatory best practices. Further, as more cellular therapies navigate the regulatory pathway, and the expectations of regulators become clear, clinical trial best practices are likely to emerge, reducing trial costs, durations and attrition rates. Therefore, a degree of long-term flexibility in business models and industry best practices will be essential if the industry is to successfully manage the poorly understood competitive threat posed by novel big data collection and analysis tools. Cell therapy industry need to unite and build a foundation for a prosperous cell therapy industry in the coming years that could offer sustainable solutions to the most pressing challenges in global healthcare.