2nd World Congress on Rare Diseases and Orphan Drugs June 29-30, 2017 London, UK

Biography:

Dan Tagle is associate director for special initiatives at NCATS. He also recently served as acting director of the NCATS Office of Grants Management and Scientific Review and currently serves as executive secretary to the NCATS Advisory Council and Cures Acceleration Network Review Board. Prior to joining NCATS, Tagle was a program director for neurogenetics at the National Institute of Neurological Disorders and Stroke (NINDS), where he was involved in developing programs concerning genomics-based approaches for basic and translational research in inherited brain disorders.

Abstract:

There are fewer than 400 approved treatments for approximately 7000 rare diseases affecting more than 30 million Americans.  The NIH have More than 30% of promising medications have failed in human clinical trials because they are determined to be toxic despite promising pre-clinical studies in animal models, and another 60% fail due to lack of efficacy. The challenge of accurately predicting drug toxicities and efficacies is in part due to inherent species differences in drug metabolizing enzyme activities and cell-type specific sensitivities to toxicants. These challenges are particularly acute for rare diseases where adequate tools and resources are severely lacking. To address this challenge in drug development and regulatory science, the Tissue Chips program aims to develop alternative approaches that would enable early indications and potentially more reliable readouts of toxicity or efficacy. The goal this program is to develop bio-engineered microdevices that mimic functional units of the 10 major human organ systems: circulatory, respiratory, integumentary, reproductive, endocrine, gastrointestinal, nervous, urinary, musculoskeletal, and immune. The opportunities for significant advancements in the prediction of human drug toxicities through the development of microphysiological systems, requires a multi-disciplinary approach that relies on an understanding of human physiology, stem cell biology, material sciences and bioengineering. This unique and novel in vitro platform could help ensure that safe and effective therapeutics are identified sooner, and ineffective or toxic ones are rejected early in the drug development process. These microfabricated devices are also useful for modeling human diseases, especially for studies in rare diseases, as well as precision medicine, environment exposures, reproduction and development, infectious diseases, microbiome and countermeasures agents.

Biography:

Deputy Chairman, Intensive Care Department, Consultant, Pulmonary and Critical Care Medicine, Professor, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Kingdom of Saudi Arabia, Dr.AlDawood graduate from College of Medicine in Kingdom of Saudi Arabia. Then he completed the Residency in Internal Medicine Program in McMaster University in Canada He obtained Critical Care and Respirology Fellowship Program in McMaster University, Canada (Jul 97-Jun 00).  In July 2000, he joined King Abdulaziz Medical City, Riyadh, Saudi Arabia as a Consultant in Critical Care and Pulmonary Medicine up to the present. He is currently the Deputy Chairman of the Intensive Care Department and Professor in King Saud Bin Abdulaziz University for Health Sciences. In addition, he has more than 40 publications including articles in the New England Journal of Medicine (NEJM), JAMA, American Journal of Respiratory Critical Care Medicine, BMC Anesthesiology, and Critical Care Medicine.  

Abstract:

Background: Since September 2012, 170 confirmed infections with Middle East respiratory syndrome coronavirus (MERS-CoV) havebeen reported to the World Health Organization, including 72 deaths. Data on critically ill patients with MERS-CoV infection are limited.

Objective: To describe the critical illness associated with MERS-CoV.

Design: Case series.

Setting: 3 intensive care units (ICUs) at 2 tertiary care hospitals in

Saudi Arabia.

Patients: 12 patients with confirmed or probable MERS-CoV infection.

Measurements: Presenting symptoms, comorbid conditions, pulmonary and extrapulmonary manifestations, measures of severity of illness and organ failure, ICU course, and outcome are described, as are the results of surveillance of health care workers (HCWs) and patients with potential exposure.

Results: Between December 2012 and August 2013, 114 patients were tested for suspected MERS-CoV; of these, 11 ICU patients (10%) met the definition of confirmed or probable cases. Three of these patients were part of a health care–associated cluster that also included 3 HCWs. One HCW became critically ill and was the 12^th patient in this case series. Median Acute Physiology and Chronic Health Evaluation II score was 28 (range, 16 to 36). All 12 patients had underlying comorbid conditions and presented with acute severe hypoxemic respiratory failure. Most patients (92%) had extrapulmonary manifestations, including shock, acute kidney injury, and thrombocytopenia. Five (42%) were alive at day 90. Of the 520 exposed HCWs, only 4 (1%) were positive.

Limitation: The sample size was small.

Conclusion: MERS-CoV causes severe acute hypoxemic respiratory failure and considerable extra pulmonary organ dysfunction and is associated with high mortality. Community-acquired and health care–associated MERS-CoV infection occurs in patients with chronic comorbid conditions. The health care–associated cluster suggests that human-to-human transmission does occur with unprotected exposure.