Scientific Program

Conference Series LLC Ltd invites all the participants across the globe to attend 9th International Conference on Predictive, Preventive and Personalized Medicine & Molecular Diagnostics Boston, Massachusetts, USA.

Day 1 :

Session Introduction

James Lyons-Weiler

Institute for Pure and Applied Knowledge,USA

Title: Personalized Immunity: Lose the Bias, Secure the Public Trust
Speaker
Biography:

Lyons-Weiler is the CEO of The Institute for Pure and Applied Knowledge. He has published over 50 peer-reviewed studies in well-respected journals, has play a key role in over 100 basic, clinical and translational research studies, and has written three books on biomedicine and biomedical research. Dr. Lyons-Weiler is expert in translational research, genomics, genetics, proteomics, biomarkers, systems biology, and conducts research on cellular and molecular responses to traumatic brain injury and the neuroimmunological responses to environmental toxins. Scientists at IPAK focus on reducing human pain and suffering through knowledge.

Abstract:

As translational research goes, vaccine safety science has a deplorable record that now places the artificial immunization enterprise at real risk. Examples of research practices that border on fraud and certainly fit an agenda of risk perception minimization are well-known to the public, and this knowledge translates into vaccine hesitancy and refusal. Pushes for mandates without exemptions alienate the public to both medicine and science.  Truly objective vaccine risk management via personalized immunity and respect for informed consent and choice will help practitioners build better relationships with their patients. A re-assessment of strategies to artificial immunization is needed. To that end, seven tracts, founded on thousands of research studies, are outlined that will help bring artificial immunization into the 21st Century: (1) reformulation, (2) risk indicators and biomarkers, (3) scheduling, (4) respect for laws and regulations governing informed consent, (5) regulating conflicts of interest, (6) regulatory reform, and (7) enforcement of adverse event reporting are all areas ripe for improvement.  An era of cottage-industry innovation in artificial immunization competing on the platform of safety is needed to foster competition.

Speaker
Biography:

Sergey Suchkov graduated from Astrakhan State Medical University with MD, then in 1985 maintained his PhD at I.M.Sechenov Moscow Medical Academy and in 2001, his Doctorship at the Nat Inst of Immunol, Russia. From 1987 through 1989, worked for Koltzov Inst of Dev Biology. From 1989 through 1995, a Head of the Lab of Clin Immunol, Helmholtz Eye Res Inst in Moscow. From 1995 through 2004, a Chair of Dept for Clini Imunol, Moscow Clin Res Inst (MONIKI). Trained at: NIH; Wills Eye Hospital, PA, Univ of Florida in Gainesville; UCSF; J Hopkins Univ, Baltimore, USA. He was an Exe Secretary-in-Chief of the Edit Board, Biomedical Science, a Journal published by the USSR Acad of Sci and the Royal Society of Chemistry, UK. At present, a Chair, Dept for Transl Med, MEPhI, and Director, Center for Personalized Medicine, Sechenov University, Russia. A member of: NY Acad of Sci,; American Chemical Society (ACS); American Heart Association (AHA); EPMA, Brussels, EU; ARVO, ISER, PMC, USA.

 

Abstract:

A new systems approach to diseased states and wellness result in a new branch in the healthcare services, namely, personalized medicine (PM). To achieve the implementation of PM concept into the daily practice including clinical cardiology, it is necessary to create a fundamentally new strategy based upon the subclinical recognition of bioindicators (biopredictors and biomarkers) of hidden abnormalities long before the disease clinically manifests itself.

Each decision-maker values the impact of their decision to use PM on their own budget and well-being, which may not necessarily be optimal for society as a whole. It would be extremely useful to integrate 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 whilst securing the healthy state and wellness, reduced adverse events, and more cost effective use of health care resources. One of the most advanced areas in cardiology is atherosclerosis, cardiovascular and coronary disorders as well as in myocarditis. A lack of medical guidelines has been identified by the majority of responders as the predominant barrier for adoption, indicating a need for the development of best practices and guidelines to support the implementation of PM into the daily practice of cardiologists!

Implementation of PM requires a lot before the current model “physician-patient” could be gradually displaced by a new model “medical advisor-healthy person-at-risk”. This is the reason for developing global scientific, clinical, social, and educational projects in the area of PM to elicit the content of the new branch.

Speaker
Biography:

Ken Rotondo is an authority on consumer experience engineering, client/patient communication, and empirical messaging insights.  He has served on the Advisory Counsel to the Dean at the Veterinary College, Cornell University, is a Past President of the New York State Veterinary Medical Society, and represented New York to the AVMA.  Currently he is an annual guest lecturer at the Wharton Business School, RPI Lally School of Business, Villanova, Cornell University and Skidmore College. He serves on the Canadian Bio-Alliance Mentoring Board, the AVMA “Futures Commission”, and several not-for-profit boards.  As President of Mind Genomics Advisors he has presented at IBM Watson Conference, IBM THINK, Social Media Week NYC, and a variety of medical and professional conferences.

Abstract:

Statement of the Problem:  With the growth of personalized medicine, we know that a single drug will not be effective to the same degree in everyone, so why do we still believe that the same singular message would have the same effect for everyone?  Day after day, the health care community struggles to effectively deliver critical messages to patients.  In many areas of medicine, we can offer precise tailored and effective therapies, but in our attempts to communicate recommendations to patients, we lack the same specificity.  Methodology & Theoretical Orientation: Our over-arching goal is to improve health-related communications and messaging resulting in greater adherence, fewer wasted resources and improved outcomes for patients. A patient communication tool, operating at scale, has been developed to identify individual patient “mind-sets” specific to a medical condition. Findings: This presentation will describe a case study in colon cancer screening compliance. The investigation focuses on colon cancer screening because it is commonly recommended, has tremendous life-saving potential and each case of colon cancer is estimated to create a financial burden of nearly $250,000.  Conclusion & Significance: The promise of “machine learning” is to gain insights to improve prediction accuracy and increase the probability of a desired outcome. An actionable medical communication methodology will be demonstrated that promotes and encourages healthy behaviors through scientifically-based, patient centered, tailored messaging based on individual mindsets. Applying basic machine learning principles to communication attendees will learn to better understand the variability of patient motivations specific to decision-making and choice.  

Speaker
Biography:

Founder & CEO of R&D Ribitzky, Dr. Ron is a Physician executive with extensive experience in the convergent healthcare IT, life-sciences informatics, and Precision Medicine. He worked for and with global leading brands and world-class organizations, R&D, and academia in the U.S. and 23 developed and emerging economy countries; in addition to ten years in clinical practice. Previously, he co-founded SPH Analytics, and served as a Senior Healthcare Strategist at Intel, VP Advanced Research at Eclipsys, CIO at UMass Medical Center, among others. He published, presented, and led workshops around the world on technology innovation and practice in industry events, public sector, and academia; and held academic appointments at Harvard, UMass, Emory, Weizmann Institute of Science (Israel), Kigali Institute of Science & Technology and Kigali Health Institute (Rwanda). He is a member of HIMSS Blockchain Work Group, and Strategic Advisor to Blockchain start-ups ARNA Genomics, BitMed, and Cryptfunder, among others.

Abstract:

The Total Addressable Market (TAM) for Precision Medicine is on a trajectory of 10%+ CAGR to surpass $100B around 2025 as patient demand is on the rise, the cost of human genome sequencing continues to go down, AI happens deep inside the chip, blockchain is heading everywhere, and medical devices are just about to switch channel to IoT chat. Yet, it still takes 10+ years for basic science discovery to make a difference in the life of the first consumer it was intended for. In this session, we will re-examine the ever-elusive Precision Medicine Paradox, white space R&D, healthcare in disruption, and We the Consumers. 

Satish P. RamachandraRao

The University of Trans-Disciplinary Health Sciences and Technology

Title: Exosomes and Precision Delivery of Personalized, Preventive Medicine
Speaker
Biography:

Rao received his Ph. D. in Neurochemistry from the Christian Medical College, Vellore, India. He has performed postdoctoral studies in Indian Institute of Science, Bangalore, India. He was recruited to Department of Medicine/Nephrology, Thomas Jefferson University, Philadelphia, USA to work on kidney organ function dysfunction biomarkers specifically in subjects with diabetes. In 2007 he moved to University of California at San Diego School of Medicine, California, USA, as an Assistant Professor of Medicine, where he continued his Kidney disease related work in Nephrology. After setting up 2 independently functional laboratories completely from ground up in these two universities in USA, he changed his focus to Clinical Biomarkers of Acute Kidney Injury in the NIH-funded George O’Brien Center for AKI Research at UC San Diego, as the Director of the Biomarkers Laboratory. He moved to Kolar Medical College in 2016 as Professor and Head of the department of Cell Biology & Molecular Genetics, Director of the Center for Exosome Research and the Research Officer of the University, along with multiple translational research collaborations in about a dozen institutions across the country. He continues his association with his previous institution as a Visiting Professor at University of California, San Diego. Recently he has assumed the leadership of a Bangalore-based consortium to develop programs for preventing dengue (ICMR-INFOSYS Initiative for Dengue Vaccine Trial Site Development), and biomarkers of health and wellness in Indian Population at the interface of Western Medicine and Integrative Medicine. His work has been continuously funded by USA-based public health agencies such as National Institutes of Health, Bethesda, Maryland, Juvenile Diabetes Research Foundation, New York. One of his research interests is developing non-invasive technology platforms, such as urine exosome biomarkers of kidney physiology in health and disease.

Abstract:

Multiple clinical and para-clinical research projects carried out at our Biomarkers Laboratory of the O’Brien Center for Acute Kidney Injury Research, UC San Diego School of Medicine demonstrate that the urinary exosome protein content of an individual is rather specific to the health or disease etiology of the subject. For the purpose of this personalized medicine conference, we have focused on one of these etiologies, namely Coronary Artery Bypass Grafting (CABG). Between 4 to 15 days after cardiac surgery insult, 4 out of 10 CABG surgeries result in Acute Kidney Injury (AKI). AKI is determined by elevation in serum creatinine (Scr). Scr elevation can only indicate kidney injury that has already occurred. Therefore, using only Scr elevation is of limited utility in either prediction or prevention of AKI episodes. Presently, AKI prevention in the clinic is thus an unmet clinical necessity.

Our data show that urinary exosome protein content can potentially discriminate between who among the cardiac surgery patients will develop AKI versus who will not, even before Scr elevation has taken place. We reason that this provides the clinician with an opportunity to help the patient prevent AKI phenotype precipitation. In this talk / meeting, the following will be highlighted: (a) how to utilize and develop this clinical opportunity into personalized preventive medicine strategy, (b) the constituent elements of this strategy, (c) the role of personalized omic integration analysis, (d) how to couple it with preventive therapy in principle guided by specific exosome biomarker analysis, and finally (e) the role of big data in precision delivery of personalized and preventive medicine. Further in addition, how exosome biomarker specificity can be expanded and utilized to achieve this precision delivery in other diseases or in healthy individuals in general, will be discussed.

Speaker
Biography:

Hornshaw is the Director of Scientific Marketing at Metabolon. Before joining Metabolon he worked for Thermo Fisher Scientific and Applied Biosystems-MDS Sciex in roles ranging from a scientist working at the bench through to leading a large team of scientists supporting customers in the field using mass spectrometry. At Metabolon Dr Hornshaw’s goal is to educate the public in general as well as scientists specifically as to the power of mass spectrometry based metabolomics to identify underlying mechanisms in biology affecting health and disease such as how the microbiome communicates with its host, in biomarker discovery and validation, as a tool to enable both precision and personalized medicine and more. Dr Hornshaw has many years of experience as a scientist and of managing scientists working with the ‘mass spec OMICS’ of metabolomics, lipidomics and proteomics as well as clinical and diagnostic applications of mass spectrometry. 

Abstract:

We have heard much about genomic medicine but genotype does not equate to phenotype. A potential future of effective precision medicine requires not only estimates of lifetime risk of disease but also accurate measurement of phenotype: molecular phenotype. This will enable assessment of disease risk in the near term, disease progress, effect of intervention on disease progression and so on. Metabolomics enables measurement of molecular phenotype in the form of the assessment of an individual’s overall metabolism (approx 1000 metabolites in plasma or serum) and changes to metabolism, not just individual metabolite measurements, in samples such as plasma, urine and tissue. When utilized in a ‘Systems Medicine’ approach metabolomics becomes a powerful indicator of health and disease. A pragmatic systems medicine approach would be to merge genomics data with large scale phenotypic measurements such as metabolomics, proteomics and imaging. A global metabolomics platform and its use in recent years in population health studies and applying knowledge of metabolism to precision medicine research will be discussed. These areas include diagnosis of inherited metabolic disease, undiagnosed illness, assessment of penetrance, drug toxicity and ‘scientific wellness’. 

Speaker
Biography:

Ana Sabater studied IT at the University of Barcerlona, Spain in 1997. She has a Maser degree in Marketing from the Universitat of Barcelona. She has been for the past 14 years involved in the IT business related to the health care. She co-founded EUGENOMIC in 2008, where her role has been to develop and put to the market g-Nomic® pharmacogenetics interpretation software to correctly apply pharmacogenetics. She teaches practical applications of pharmacogenetics at the University of Barcelona. Has given more than 30 international keynote presentations about pharmacogenetics and has designed the training program that has been running now for more than 5 years.
 

 

Abstract:

A single word Pharmacogenetics is the answer for ‘why certain drugs produce toxic effects to some people & why in certain individuals, a particular drug does not help?’

Knowing the genetic polymorphisms of a patient shows whether a drug will make the intended effect according to the dosage from clinical trials, will require more or less dose, or it should be avoided and seek a therapeutic alternative.In general, patients are polymedicated, however the effect of various drugs administered together may be different (produce ineffectiveness or toxicity), different to what would happen when administered alone. Sometimes, even if a person could take the drugs individually according to the genetics, the drugs themselves could inhibit or induce the other.To successfully apply pharmacogenetics and make a prescription safely and accurately, many parameters should be taken into account, such as drug-drug interactions, drug-lifestyle, inhibitions and inductions and dose variation according to patient studied genes. All this information can only be interpreted together, using a pharmacogenetics interpretation software like g-Nomic®. g-Nomic crosses information concerning prescribed drugs, patient’s genetic variations to give a personalized report with all necessary information: drug interactions, drug with lifestyle, inhibitions, inductions and dose variation according to the patient's genes. Applied pharmacogenetics can indeed avoid many emergency cases, safe lives and money. But to correctly apply pharmacogenetics, an interpretation software is needed since there are too many variables to consider.

Speaker
Biography:

Brahma D. Sharma, PhD is a retired chemist from R&D and manufacturing of pharmaceutical and medical device industry. He is an author, inventor, scientist, entrepreneur, coach, and speaker. His current book is ‘Cracking the Genetic Code for Prescription Drugs’. His mission to reduce Adverse Drug Reactions (ADR). It’s the fourth leading cause of death in the US after heart attacks, strokes, and cancer. One person dies every 4 minutes. Doctors have been relying on pharmaceutical manufacturers far too long. Drug companies often recommend one dose without considering patients’ genetic variations which impact heavily their drug’s metabolism. One drug/one dose does not fit all. He is educating everyone, patients, doctors, healthcare providers etc. how to reduce ADR by incorporating Pharmacogenetics (PGx) testing in the practice and personalizing prescription drugs that match patient’s hepatic gene variations. ADR is estimated to cost over $136 Billion in the US not counting priceless suffering of patients and their loved ones. He believes that the addition of opioids often begins by the drugs prescribed by a doctor and taken as directed. His goal is for doctors to start with “Let’s start with swabbing your cheek. This will tell me which drugs will do good for you and which drugs may harm you.”

Abstract:

Time is up for continuing to prescribe drugs by trial and error. Eliminate the guess work. Most drugs work when prescribed to a ‘right’ patient.On the other hand, a ‘right’ drug prescribed to a ‘wrong’ patient could be and often is deadly.

The dilemma is: how do you differentiate between ‘right’ and ‘wrong’ patient before prescribing a drug? Even siblings often metabolize drugs differently due to inherited mutations (variations) among genes in their livers. Pharmacogenetics (PGx) is here to guide you. PGx test involves a simple cheek swab, yet provides powerful clinically actionable data for the life of a patient. It predicts before a drug is prescribed, if it is a ‘right’ drug for the ‘right’ patient or it has a high risk of potentially creating Adverse Drug Reaction (ADR) due to a slow acting gene, representing a ‘wrong’ patient, and should be replaced by another therapeutically equivalent drug which is metabolized by another gene which is functioning normally in the patient’s liver. Unmetabolized drug starts accumulating in the body and can lead to ADR due to increased toxicity and side effects, in millions of patients in the US alone. In fact, ADR is now the 4th leading cause of death here, one patient dies every 4 minutes. Insurance companies including Medicare often pay for the test.

Who needs PGx test urgently

It’s too late to order the test during surgeries in ERs. You need to have patient’s PGx report through EMR, EHR or cloud to prescribe personalized medicine to control pain, bleeding etc. I recommend every newborn should have the PGx test to insure even first prescription the child will ever get be personalized to reduce potential of an ADR. The risk of ADR increases exponentially by the number of prescription the patient is on. Coumadin/Warfarin, a commonly prescribed blood thinner to control thrombosis is number one drug requiring ER visits among senior patients.

PGx reducing healthcare cost

University of Illinois Health’s Personalized Medicine Program saved $600K annually by reducing hospital readmissions when they used PGx test to guide dosing of only one drug, Coumadin/Warfarin.

FDA requiring PGx test

FDA along with EMA, PDMA and HCSC is recommending, in fact requiring a PGx test before prescribing 54 of the drugs carrying Black Box Warnings. Alas, healthcare professionals keep ignoring the requirement. I appeal to all healthcare professionals to be progressive and adopt PGx test and change their practices to personalized medicine.

Speaker
Biography:

Gil Blander is internationally recognized for his research in the basic biology of aging and translating research discoveries into new ways of detecting and preventing age-related conditions. He leads a team of biology, nutrition & exercise physiology experts, and computer scientists at InsideTracker. He received a Ph.D. in biology from the Weizmann Institute of Science and completed his Post Doctoral fellowship at MIT, before going on to found InsideTracker. The InsideTracker platform analyzes key biochemical and physiological markers and applies algorithms and large scientific databases to determine optimal zones for each marker. The system then provides nutrition, exercise, supplements and lifestyle interventions that empower people to optimize their markers, increasing vitality, improving overall health, as well as athletic performance and extending life.

Abstract:

The trend toward personalized approaches to health and medicine has resulted in a need to collect high-dimensional datasets on individuals from a wide variety of populations, in order to generate customized intervention strategies. However, it is not always clear whether insights derived from studies in patient populations or in controlled trial settings are transferable to individuals in the general population. To address this issue, an observational analysis was conducted on blood biomarker data from 1033 generally healthy individuals who used an automated, web-based personalized nutrition and lifestyle platform. Using the resulting dataset, a correlation network was constructed to generate biological hypotheses that are relevant to researchers and, potentially, to users of personalized wellness tools. The correlation network revealed expected patterns, such as the established relationships between blood lipid levels, and novel insights, such as a connection between neutrophil and triglyceride concentrations that has been suggested as a relevant indicator of cardiovascular risk. Biomarker changes were assessed from baseline to follow-up, relative to platform use. Preliminary associations were found between the selection of specific nutrition and lifestyle interventions and biomarker outcomes. Across many biomarkers measured, there was a significant trend toward normalcy in participants whose biomarker values were out-of-range at baseline.

Kate Simon

Biologics Consulting Group,USA

Title: Bringing New Biomarkers to Market
Speaker
Biography:

Simon came to Biologics Consulting with over eleven years of experience at the FDA. During her time at FDA she served as a Master Scientific Reviewer in the Office of In Vitro Diagnostics and Radiological Health (OIR). Dr. Simon has been a lead reviewer of many pre-market applications for IVD devices including PMA, 510(k), Pre-IDE, IDE and Pre-submissions. She has served as an FDA speaker in public forums on a diverse array of topics, including the de novo process, pharmacogenomics and personalized medicine, and premarket IVD submissions. Dr. Simon holds a Ph.D. in Microbiology from the University of Virginia.

Abstract:

This presentation by a former FDA reviewer will provide an overview of FDA regulation of in vitro diagnostic devices (IVDs). This presentation will help audience members make the leap from discovering a new biomarker to marketing an FDA approved or cleared diagnostic test. Design of appropriate analytical and clinical validation studies to support subsequent IVD data submissions to FDA will be covered, as well as best practices for preparing an FDA premarket submission. Seasoned professionals as well as those new to in vitro diagnostics will benefit from hearing about IVD device regulation from a former FDA reviewer. This will better enable them to prepare for IVD device clinical trials and subsequent data submission to FDA.

K. K. Jain

Jain PharmaBiotech, Switzerland

Title: Personalized Neurology
Speaker
Biography:

Jain is a neurologist/neurosurgeon with career in North America. He holds fellowships in neurosurgery from the Royal Colleges of Surgeons in Canada and Australia. After retirement from neurosurgery, he started a second career in pharmaceutical medicine and biotechnology in Switzerland. Currently he is a Fellow of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians of UK. In addition to work as a consultant in neurology, he is also involved in biotechnology. His company, Jain PharmaBiotech is involved in Research and publications on biotechnology and applications in pharmaceuticals and healthcare. He is integrating advances in biotechnology and other areas to advance personalized medicine. He has given several invited lectures and conducted workshops on personalized medicine since 2000 in Asia, Europe, and USA. He served as a visiting professor personalized medicine at the University of Kazakhstan at Almaty in April 2018. Jain is developing personalized medicine since 1998, and wrote the first monograph on this topic, which evolved into “Textbook of Personalized Medicine”, 2nd edition (Springer 2015). His 465 publications, besides several papers on personalized medicine, include 30 books (6 as editor and 25 as the author). Important authored books include “Applications of Biotechnology in Neurology” (Springer, 2013), “Applications of Biotechnology in Oncology” (Springer, 2014) “Handbook of Biomarkers”, 2nd ed (Springer 2017), and “Handbook of Neuroprotection”, 2n ed (Springer 2018, in preparation). He has edited “Applied Neurogenomics” (Springer 2015). Editorial board memberships of journals include “Technology in Cancer Research & Treatment and “Nanomedicine” (London).

Abstract:

Personalized neurology is the application of principles of personalized medicine, i.e., the prescription of specific therapeutics best suited for an individual taking into consideration both genetic, epigenetic, and environmental factors that influence response to therapy. The aim is to improve efficacy and reduce adverse effects of therapy. Personalization of therapies for neurological disorders is based on a better understanding of the disease at the molecular level. Molecular diagnostics, molecular neuroimaging, sequencing and monitoring of gene expression by microarrays are important technologies for this purpose. Besides omics, e.g., neurogenomics and neuroproteomics, nongenomic technologies such as nanobiotechnology are also used. Biological therapies for neurological disorders, such as cell therapy, gene therapy, gene editing, RNAi, vaccines and monoclonal antibodies can also be personalized. Biomarkers and integration of diagnostics with therapeutics are important for the selection and monitoring of treatments. Biomarkers enable presymptomatic diagnosis, selection of appropriate treatment, assessment of disease progression, and evaluation of patient response to therapy. Nanobiotechnology has refined molecular diagnosis, improved drug formulation and targeted delivery through the blood-brain barrier to the lesion in the brain and spare the normal tissues to reduce systemic toxicity. Integration of multiple factors into personalized approach requires use of bioinformatics. A personalized approach can be incorporated in algorithms for the management of various neurologic disorders. Examples of neurological disorders will include Alzheimer disease, Parkinson disease, epilepsy, and stroke. Advantages and future of personalized neurology are:

  • The availability of low-cost genomic sequencing will expand the use of genomic information in the practice of neurology.
  • Sequencing of the genome is enabling genetic redefinition of several neurologic disorders as well as better insight into their pathomechanisms to improve our understanding and facilitate early detection by molecular methods.
  • An increase in the ability to anticipate diseases rather than just reacting to them after onset may enable the institution of preventive measures.
  • The precision and effectiveness of drugs is increased.
  • Drugs can be better targeted to diseases in some patients based on genotype information.
  • Development of more effective personalized medicines may obviate the need for surgery in some chronic neurological disorders.

Larina Vera

Russian national research medical university

Title: Problems and perspectives of personalized medicine
Speaker
Biography:

Larina Vera, PhD, MD, professor of the department of outpatient medicine of Russian national research medical university named after  N. I. Pirogov. She has published more than 100 papers in Russian journals and is a member of the editorial board.

Abstract:

In Russia, as in many countries around the world, there was an individualized and paternalistic approach to the treatment of the patient. In the past, it was based on the personal doctor’s experience, the doctor-patient relationship and often the patient's socioeconomic status. The priority of individualized treatment began to give way to clinical guidelines based on evidence-based medicine in relation to the introduction of the principles of "industrialized" and evidence-based medicine into practice. At the same time, over the past decade it has become clear that such approaches cannot resolve the complex of problems associated with the patient's "burden of disease".

Awareness of the need to minimize the burden of treatment for the patient, his family and the health care system has emerged in relation to the development of new technologies, increased longevity, increase patients number with multimorbidity and fragility, emergence of research results indicating that the same method of treatment (drug) can affect the patient in different ways. More and more specialists are talking about the need for individualized approach in the choice of tactics for managing the patient, taking into account the biological characteristics, the possibilities of medicine and many other non-medical factors (ethical, social, financial).

A lack of knowledge is a significant problem for the introduction into the clinical practice of personalized medicine despite of their geometric growth at the present time. In fact, there are no randomized studies, that all the way of treatment and diagnostic methods with an infinite variability of multimorbidity are evaluated, there is no possibility to use in clinical work all the accumulated knowledge in the world (this the limited capabilities of a doctor and even a multidisciplinary team; lack of a common language of communication, a database of such information, rapid access to it, taking into account the individual needs of the patient), only the beginning of the selection of treatment taking into account genetic and epigenetic factors.

Speaker
Biography:

Tori Strong has completed his PhD from the University of Texas Medical Branch at Galveston. He is the director of Intellectual Property and Technology at Vyripharm Biopharmaceuticals, a premier biotech organization. He has served as a Patent Examiner for the USPTO and is now directly involved in the technology development to commercialization which includes strategies of building intellectual propery.        

Abstract:

Novel Formulation of Cannabinoid Analogues Treating DLBCL and MCL: Diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL) represent the most common and most aggressive forms of Non-Hodgkin lymphoma (NHL) respectively. With CB1 antagonists as potential therapeutics for both DLBCL and MCL, we formulated VYR-206, developed from existing obesity treatment Rimonabant by the addition of our tetraazacyclic (N4) conjugate derivative. This allows the potential for image guided theranostic application for diagnosis, precision and assessment of therapeutic response through radiotracer chelation.  Our study is aimed at demonstrating VYR-206 activity in DLBCL and MCL for sensitivity or resistance. Cells from representative DLBCL and MCL cell lines were plated at 5,000 cells per well. The cells were incubated for 72 hours in 20 µL medium with 10% FBS and varied concentrations of experimental cannabinoid antagonist VYR-206, Rimonabant, or dimethylsulfoxide (DMSO). Viability assays were conducted using Celltiter-Glo Luminescent Cell Viability Assay. Experiments were performed 2-3 times independently, with concentration tested in triplicate. Most DLBCL cell lines treated with VYR-206 had a reduction in viability at concentrations of 50μM or greater with few cells line displaying limited response even at concentrations of 100μM. Increased variability is seen among MCL cell lines treated with VYR-206, most having a reduction of viability at concentrations of 25μM or greater, with few cell lines at concentrations of 50μM and 2 cell lines showing no response even at concentrations of 100μM. The discrepancy in response in both DLBCL and MCL may be due to genetic variability among cell lines.

Speaker
Biography:

Romina Ortiz completed her Bachelor’s and Master’s degrees at Johns Hopkins University in Neuroscience and Molecular Microbiology and Immunology, respectively. Her thesis focused on the interplay between the microbiome and hormones in autoimmune disease. She is a co-founder of Rare Genomics Institute (RG), COO and VP of Patient Advocacy at RG. In 2016 she was awarded the Patient Advocacy Leadership award by Sanofi Genzyme. She trains volunteers on the topics of Genetics and Sequencing, directs a Patient Advocacy Program in next generation sequencing and research services to undiagnosed and rare disease patients and currently directs a philanthropic program with Illumina called iHope, offering free whole genome sequencing to children in need of a diagnosis. Her goal is to integrate phenotypic medical information with genomic data to identify the genetic cause to disease and accelerate the path to treatment and a cure.

Abstract:

From the Orphan Drug Act of 1983, a rare disease is a condition that affects fewer than 200,000 people in the United States (1). In the European Union, the condition must only affect fewer than 1 in 2,000 people (2). While the numbers seem small, there is an estimated 350 million people that suffer from rare diseases, with 25-30 million belonging to the US alone and so far there have been over 7,000 different rare diseases identified (1). To put this in perspective, there are more Americans affected by rare disease than for HIV, Heart Disease or Stroke combined (3). It is important to understand that by nature rare diseases are difficult to diagnose, and consequently are not tracked. Thus, it is hard to accurately determine the number of rare diseases and their impact on a population. The average length of time from onset of symptoms to an accurate rare disease diagnosis is nearly 5 years, and patients see an average of over 7 different physicians before a diagnosis is made (5).This delay in diagnosis results in chronic physical, emotional and socioeconomic burden to both the patient and their family. A European Cost of Illness Study interrogating published literature on the cost of 10 selected rare diseases found that overall, the availability of data on economic burden for rare diseases was correlated with the availability of therapies, not the severity of the disease. Also, most rare diseases reviewed were found to have significant economic burden and indirect costs ( many associated with loss of productivity) exceeded the level of direct costs (5). Rare Genomics has served over 500 undiagnosed patients since 2011, helping them access next generation sequencing to accelerate their pathway to a cure. We have seen the same patterns reported for rare diseases in our own patients including heterogeneous disease marked by a range of severity across a variety of biological systems. The most common systems affected are Neurologic, Respiratory, Gastrointestinal, Muscular and Cardiovascular. The average RG patient has also seen a range of physicians, the top three specialties consulted are: Neurologist, Clinical Geneticist, Opthamologist and Gastroenterologist.Lastly, undiagnosed/rare disease patients typically have already undergone a gamete of testing, the most common tests are: MRI, DNA Microarray and Single/Panel Sequencing. Because 80% of rare disease are genetic in origin, we hope that by providing support and access to next generation sequencing, we can help reduce the time and burden these families must undergo before identifying appropriate treatment for their disease.