Personalized medicine

Personalized medicine is the largest achievement and a key scientific trend of healthcare of our century It aims at individually tailored therapy of grave and incurable cancer patients, as well as cases of hereditary diseases of nervous system and organic diseases of the central nervous system (CNS). The main principle of the personalized approach relies on the personal data of molecular-biological (genomic, transcriptomic, proteomic, metabolomic and secretomic) markers on the basis of which an individual cellular and/or gene-therapeutic preparation is developed to provide the etiopathogenetic therapeutic effect on the signs of the main disease or its fatal complications. The personalized molecular-biological diagnostics is based on the whole or partial sequencing of the genome, transcriptomic and proteomic mapping and profiling of its cell systems and production of the individually tailored molecularly targeted and/or gene-therapeutic preparations and biomedical cell products (BMCPs). It is an absolutely new scientific, methodological and biotechnological platform for diagnostic and therapy. The personalized approach to the treatment of obstinate and orphan (rare) diseases is regulated by the Order of the Minister of Healthcare of the Russian Federation №186 dated April 24, 2018 On approval of the Concept of predictive, preventive and personalized medicine that approves this approach for clinical practice. We used the recommendations of this Order as the basis for the development of the strategy of the personalized medicine in our hospital and chose the following fields of predictive, preventive and personalized medicine: oncology, neurology, autoimmune diseases, orphan hereditary diseases of the nervous system as well as the issues of longevity and life span extension. Our choice is conditioned by our long-term scientific research that are protected by the patents (30 patents of the Russian Federation and 1 patent of the USA). The following has been defined at the main competence of this work:

1. Development of the science driven methodical recommendations to use the biotechnologies of predictive, preventive and personalized medicine in oncology, neurology and geriatrics;
2. Gathering and processing of the molecular-biological (genetic, transcriptomic, proteomic, metabolomic) data of the biomaterial of the patients with neurological and oncological diseases using neural networks and artificial intelligence;
3. Use of the genome and post-genome biomarkers to monitor the efficiency of the diagnostics and treatment of the non-infectious oncological and neural diseases;
4. Development, preclinical and clinical trials and registration of the non-manipulated and low manipulated cell preparations, BMCPs and gene-therapeutic preparations for personalize regenerative and post-genome medicine;
5. Development and use of the genome-, proteome- and transcriptome-restored hematopoietic stem and precursor cells for gene therapy in the treatment of some of the incurable hereditary, oncological, autoimmune, neurodegenerative and orphan diseases of nervous system;
6. Development, registration and use of the autologous cellular vaccines loaded with virus-specific or pathology-specific protein antigens in neurooncological patients;
7. Virus-lithic therapy for the personalized treatment of the fatal oncological and neurological diseases;
8. Development of the standards of the effectiveness and safety of the administration of the cell preparations and BMCPs in neurology, oncology and geriatrics.

When the main genetic and protein markers of the process and additional somatic mutations of the HSCs genome of the specific patient have been detected, the production of the personalized cell preparation takes from two to six months up to a year of labor of the team of immunologists, geneticists and molecular biologists. Moreover, in some cases we appear unable to restore the damaged HSCs by 100% and choose the ones without genetic defects and neotransformations. We do not use the technologies of gene editing, vectors, plasmids and viruses for the delivery of the genetically engineered constructions to the cell in our production of the personalized drugs, cell preparations and BMCPs. We employ the nature-like mechanism of sanogenesis (natural instrument of self-restoration of the HSCs) of frequently dividing cells, which is known as homologous replacement of the parts of the DNA by two-chain DNA and RNA of a healthy donor. We let the autologous cell choose the appropriate biomaterial from the provided genetic resource of the DNA of the same organism from different tissues or of the approved therapeutic substance Panagen that contains the DNA of a healthy donor. The main achievement of our molecular biologists is the opportunity to manage the recombination in the patient’s own HSC and to provide the favorable conditions under which this mechanism will be effective. Our task is to secure the quality of the self-reconstruction of these stem cells and to check for the absence of neoplastic transformation and additional mutations that lead to the genome instability.