Title : Personalized and Precision Medicine (PPM) as a unique healthcare model to secure the human healthcare, wellness and biosafety: Through the view of cell-based therapy and rehabilitation
Abstract:
A principally new and upgraded systems approach to subclinical and/or diseased states and wellness resulted in a new trend in the healthcare services, is called as Personalized and Precision Medicine (PPM). But despite breakthroughs in translational research that have led to an increased understanding of PPM-based human disease, the translation of discoveries into therapies for patients has not kept pace with medical need.
Genomics and Genomics editing have raised great expectations concerning the impact on PPM-related customizing the individualized treatment based on the use of genomic products and development of targeted drugs. So, PPM has progressed into fields such as gene therapy and surgical treatment/design. And thus the targeted gene-based therapies today represent an important stake for the clinical community, patients, persons-at-risk and biopharma, in terms of market access, of return on investment and of image among the prescribers.
Meanwhile, most disease, trauma, cancer and regeneration-related pathologies are consequences of cellular damage at differing levels. And the key to the mitigating cell and/or tissue damage repair needs cell therapy which, in turn, is becoming a promising treatment that can be tailored not only to an illness but also to an individual patient and even for a person-at-risk. So, simultaneously, regenerative medicine and cellular therapy use cell-rooted products in order to develop PPM-based treatments being based on different sources of Stem Cells (SCs) which are being used in targeted therapies whilst providing a personalized approach and could give many advantages as well, including a possibility for SC-based individualized therapy be adjusted according to the patient-specific profile.
For instance, intracerebral SC therapy has provided promising results for treatment of Parkinson’s Disease (PD). But! The method is very invasive and is often associated with unacceptable side effects which would hamper the implementation of peripheral administration of SC-derived neural precursor cells for the recovery of impaired motor function and amelioration in PD cases. So, cellular reprogramming and PPM-based approach would allow for previously unattainable cell therapies and patient-specific modeling of PD with no risks of immune rejection, using induced Pluripotent Stem Cells (iPSCs) which, in turn, is considered to be the foundation of SC-based regenerative medicine, including PD and Muscular Dystrophies (MDs). Moreover, in combination with genome editing, hiPSC technology is utilized in functional genomic screening for identification of the roles essential genes play in specific cellular process, whilst securing the availability of less severe phenotypes compared with patient-specific hiPSCs carrying the same mutations, and thus providing the exclusively important functional implications for PPM and PPM-based cardiac treatment.
A key role in bone tissue engineering may play patient-derived Mesenchymal Stromal Cells (MSCs) whose pooling would help to compensate donor-dependent variability and does not negatively influence MSC vitality, proliferation and osteogenic differentiation.
Ex vivo expansion of Hematopoietic Stem Cells (HSCs) can help to overcome material shortage for transplantation purposes and genetic modification protocols. But translating new findings from basic research into clinical protocols is still crucial to eventually boost SC gene therapy.
The establishment of 2D- and 3D-based intestinal SC cultures of patient-derived epithelial tissues is becoming a promising breakthrough for treating some cases of Colorectal Cancer (CRC).
Of particular interest to be stressed on, are SC-based therapies to strengthen the heart muscle and treat cardiovascular diseases (including ischemic attacks and post-infarction conditions). For instance, human hiPSCs have revolutionized the field of disease modeling, with an enormous potential to serve as paradigm shifting platforms for preclinical trials, personalized clinical diagnosis, and drug treatment, whilst bridging the gap between basic and clinical research via translational bridge to bring the best science to every patient. The phenomenal achievement in this area is the identification of resident Cardiac SCs (CSCs), supposed to be a crucial source to initiate and prompt myocardial self-renewal and regeneration but can be developed inside immature cardiac cells by formation of “Cell-In-Cell Structures” (CICSs), which, in turn, being encapsulated are involved into cardiac myogenesis and thus opening up a green light to secure the targeted management of post-infarction regeneration.
The above-mentioned areas being an integral part of PPM is really an interdisciplinary field that results from the application of the innovative tools and approaches to medicine and has the potential to significantly improve some canonical treatments, prevention, prophylaxis and rehabilitation. The SCs would confirm a high subclinical and predictive value as tools for PPM-based monitoring protocols. SCs can be programmed and re-programmed to suit the needs of the body metabolism or could be designed for the development of principally new combinatorial (genomics/cellomics-rooted) drugs with no natural counterparts. So, PPM through SC therapy has many benefits that are essential for the future of personal health and wellness.
The SC market itself is predicted to grow to around $12.1 billion by 2024, whilst the true potential of regenerative medicine has yet to be demonstrated fully. For instance, the allogeneic or ‘off-the-shelf’ business model for SC-based therapies is far more akin to current biopharma-related products. So, partnering between SC-related researchers, cell bio-designers and bioengineers, cardiac clinicians and surgeons, business and regulatory bodies and government can help ensure an optimal development program that leverages the Academia and industry experience and FDA’s new and evolving toolkit to speed our way to getting new tools into the innovative SC markets.
The worldwide SC therapy market is still in an early stage. And the developing translational pipelines for rising applications in the above-mentioned areas will build the competition among merchants amid the conjecture time frame.
Meanwhile, the prospective research in the above-mentioned area should focus on the identification of prognostic SC- and cardiac damage-related biomarkers to identify patient cohorts who benefit most from SC-based treatments. Thereby, a higher standardization of study designs and the establishment of a global open-access database for the registration and publication of pre-clinical and clinical trials would greatly improve the comparability and access of obtained data. And now would be extremely useful to integrate those data harvesting from different databanks for applications such as prediction and personalization of further treatment to thus provide more tailored measures for the patients and persons-at-risk resulting in improved outcomes and more cost effective use of the latest health care resources including preventive, prophylactic, therapeutic and rehabilitative manipulations as the new and upgraded Restorative ENTITY!
The latter would provide a unique platform for dialogue and collaboration among thought leaders and stakeholders in government, academia, bioindustry, foundations, and disease and patient advocacy with an interest in improving the system of healthcare delivery on one hand and drug discovery, development, and translation, on the other one, whilst educating the policy community about issues where biomedical science and policy intersect.
