Stem Cells have been repeatedly talked in public and scientific community and all the stem cell biologists and scientists agree that stem cell have the potential for treating human diseases and disorders. For the business purpose the possibilities of a transfer of these biological concepts to clinical practice continue to attract extremely high interests. Therefore, stem cells are a resource providing unprecedented potential for biological discovery, drug development & future cellular medicines. Undoubtedly, resolving the challenges of developing a new medicine based on stem cells as a therapy lies currently with the small to medium sized biotechnology industry, as larger companies if they perceive the value, view it as too distant in the future. Developing safe, effective medicines and having the ambition to make them as widely available as possible to meet the challenge of major unmet medical needs will require very large financial resources, much beyond the capacity of small biotech companies & arguably those companies best placed to compete in the race will typically have an international disposition & multiple revenue generating business streams.
It is widely believed that stem cells hold great promises and potential for both basic sciences and medicine. Stem cells are expected to improve our understanding of fundamental biological processes, help in developing model for human diseases such as birth defect or cancer, help in establishing a platform for drug screening and toxicity studies in order to shorten the process of drug development and most importantly stem cell promise created lots of hope to find way of using these cells to repair damaged tissues and curing serious diseases for which currently no treatments are available.
What is Stem Cell?
In a layman’s term stem cells are capable of self renewal and are capable of differentiating into multiple cell types of ectoderm, endoderm and mesoderm. For example stem cells can be differentiated into liver cells, brain cells or skin cells. This process is called as differentiation. These differentiated cells that have certain function and properties therefore can be used for cell replacement therapies. Thus, stem cell has a capacity to develop into any kind of cells in the human body.
Stem cells are broadly classified as embryonic stem cells (ESC) since they are derived from pre-implantation stage embryos such as blastocyst (day 5 embryo) and adult stem cells, those found in various adult tissues.
Stem cells from different sources vary in their abilities to differentiate or to produce mature cell types. Embryonic stem cells (ESC) can give rise to any cell type in the adult body, whereas adult and cord blood-derived stem cells are more restricted in their ability to differentiate into various cell lineages. Understanding the versatility of a given stem cell type is crucial to unlocking its therapeutic potential.
Adult Stem Cells
Adult stem cells are most widely used stem cells for human therapy and clinical trial. Global stem cell market is currently dominated by the adult stem cells. More than 58% of the companies are currently using adult stem cells for research and therapies. Among the adult stem cells, bone marrow derived stem cells are most preferred stem cells that are currently being used for therapy. Other adult stems cells are adipose tissue derived stem cells, keratinocytes, cord blood and neural stem cells. Recently bone marrow derived Mesenchymal stem cells (MSCs) showed promising tools in newly emerging avenues of regenerative medicine. MSCs have multipotential properties and currently being used for several clinical applications. Several scientific reports demonstrated that the administration of MSC can home to the site of injury, can circumvent problem of immune incompatibility and they are non-tumorigenic and hence can develop affordable off the self therapeutic product for multiple patients.
To a certain degree, tissue regeneration takes place in the human body throughout life. For example, blood and skin are continuously restored, and bone, muscle, liver, and blood vessels have a limited capacity for self-renewal. However, disease or trauma can cause a significant loss of functional cells that the regenerative power of the body cannot repair.
Although the general public is keenly aware of the stem cell promise for curing many diseases, many people do not realize that stem cell therapy is already a reality in clinics today. In fact, hematopoietic stem cell (HSC) transplantation for the treatment blood diseases, such as leukemia, lymphomas, and anemia, has been around for more than 40 years. Originally such transplants were performed using bone marrow, but more recently, they are performed using mobilized stem cells from peripheral blood, as well as cord blood stem cells. From a business perspective, current HSC transplants are not considered to be a commercial product. The procedures are carried out by medical transplant centers; therefore, the financial benefits are relegated to the health care providers and affiliated institutions.
Clearly not every single patient suffering from a degenerative condition will require a cell-based treatment. Nevertheless, experts believe that if indeed stem cell treatments are truly disease modifying and produce a significant clinical benefit to a patient, some of them could achieve a blockbuster status. By some predictions, cell-based therapies could be a US$ 20 billion market by 2010.
Market assessment indicates that India and other Asia –Pacific countries like Singapore, Taiwan, Malaysia and Thailand are the immediate market for entry. As per the estimation India as a region with maximum market annual potential approximate US$ 540 million followed by Taiwan US$ 320 million, Thailand US$ 230 million, Malaysia US$ 157 million and Singapore US$ 100 million.
In the APAC region, Singapore has the highest penetration rate with CAGR of almost 66 per cent for market penetration over the next 5 years. Malaysia leads second with CAGR of 33 per cent whereas Taiwan and Thailand leads on the basis of shear numbers but would have comparatively lower demand trajectory due to restrictive healthcare policies.
Adult stem cell therapy currently dominates the global stem cells market with share of almost 58 per cent and bone marrow derived stem cell is predominantly in use for therapies. Therapies for osteochondral disorders are the forefront of development with two successfully launched products already in the market. Cardiovascular therapies for advanced cardiac failure and neo-vascularization in acute myocardial infarction are the second most developed therapeutic areas. Spinal Cord injuries & Parkinson’s rank third in development and followed by development in the renal and hepatic disorders.
Today, stem cell therapy is a reality. Results showed that stem cell therapy for myocardial infarction can improve ejection fraction significantly and reduce the infarct size. Similarly, stem cell therapy for leg ischemia, spinal cord injury, stroke, multiple sclerosis and several other diseases has shown significant improvement during phase-I and II clinical trials. Diabetes cardiovascular disorder and spinal cord injury are the therapeutic area that will benefit from stem cell treatment because in these categories there is large patients pool and offer no permanent cures, and that will allow patient rapid product acceptance by the medical community. It has already shown that stem cells can be used successfully in 132 diseases and there are more than 1500 clinical trials currently in progress around the globe.
Despite the blockbuster promises of stem cells, investors are still shy away from investing money in this initiative. Reasons are multiple and may take years to understand. First and foremost, stem cell science is still in infancy. Despite significant progress and breakthrough discoveries, fundamental knowledge of stem cell differentiation is seriously lacking. We have yet to solve the mystery of several questions. For example we do not know what causes stem cell to maintain them in an undifferentiated state? What signals do the stem cells take from the organs where they were injected so as to differentiate into appropriate lineage? What genetic or environmental signals affect differentiation? Technique for safe and efficient delivery system of stem cells to the tissues has to be identified.
Second major challenge is funding for stem cell research. There is growing consensus that countries like India, China, Singapore, Malaysia, Taiwan and Thailand are likely to play a key role in the scientific, clinical and commercial development of stem cells research. Unfortunately, research funding for stem cells in these countries are meager. India has spent US$ 2 million annually on stem cell research in comparison to that of US$ 550 million in US.
Third challenge is politically charged area. Stem cell science is one of the most controversial sciences because of its ethical reason especially in human embryonic stem cell. Unfortunately, due to such high profile political interference, scientific facts have been lost in the media and religious debate and every other country drag themselves into this controversy. However, more than 200 genetically diverse stem cell lines were developed and established after the discovery and ban on human embryonic stem cell derivation in US and most of them were derived in US and Asia. It was thought that the US ban had opened up a “new pot of gold” for Asian science and business and opportunity to march ahead in this field. Several Asian countries have taken this opportunity and made significant progress in stem cell research for example, China, Korea, Taiwan and Singapore. Unfortunately progress of stem cell research in India and Malaysia is very slow and insignificant in comparison with other Asian countries.
Urgency of laying down rules for this new branch of medical research is supported by the governments of all nations encouraging stem cell research as there are chances of misuse of this technology. Already exaggerated claims of curing certain diseases using stem cells are being made by some institutions and private clinics around the world. It would be appropriate to develop clear cut guideline for stem cell research and therapy so that such misuse can be avoided.
Fourth challenge is autologous or allogenic therapy. We all know that bone marrow transplantation could not translate into commercial product. Turning cell based therapies into commercial product require development of scalable manufacturing processes where cells are produced under current good manufacturing practices. Autologous Vs Allogenic could greatly influence the manufacturing cost and thus profit margin. Moreover, having universal allogenic stem cell therapy will make much more compelling product from business perspective. Critical cases such as acute myocardial infarction or cerebral stroke or in any genetic diseases alllogenic stem cell therapy should be a choice.
Stem cell holds enormous potential for both science and medicine. Stem cell based therapies offer a promise for curing serious debilitating diseases for which currently no treatment s are available. In our opinion, within next two years stem cell field is poised to reach an inflection point which would position it for a substantial expansion. However, power of this knowledge need to be used carefully without putting a patient to a risk.