Concurrent Workshop Sessions

• Wednesday, September 16, 2015
• Thursday, September 17, 2015

Wednesday, September 16, 2015

Chair: Crystal Mackall, M.D. (NCI/CCR)

Cell-based therapeutics has been described as the next pillar of medicine, tapping into the potential of stem cell differentiation and tissue regeneration. This workshop will focus on how the NIH IRP can usher a new wave of cell-based therapies by combining established and maturing scientific disciplines such as immunobiology, genome engineering, and cell engineering. The IRP plans to expand its infrastructure for cell- and vector-production laboratories and enhance its GMP facilities, develop point-of-care stem cell harvesting technologies; and enhance stem cell differentiation and tissue regeneration with modified scaffolding. The focus will be on approaches and disease types perceived as not being commercially attractive given scientific challenges or limited market potential.

• Developing autologous cell therapy for macular degeneration using iPS cell derived RPE patch
  Kapil Bharti, Ph.D. (NEI)
• Evaluation of novel pharmacologic and genetic approaches to enhance NK cell cytotoxicity against cancer
  Richard Childs, M.D. (NHLBI)
• Chimeric antigen receptor therapies for cancer: progress and challenges
  Crystal Mackall, M.D. (NCI/CCR)
• KLF4 dependent perivascular cell plasticity promotes metastasis (FARE Award Winner)
  Meera Mergai, Ph.D. (NCI/CCR)
• Gene therapy for older children and young adults with SCID-X1
  Harry Malech, M.D. (NIAID)

Chair: Karen Frank, M.D., Ph.D. (CC)

Microbes outnumber human cells in the human body by a ratio of about 10 to 1. Most of these microbes are harmless, and many are beneficial in regulating digestion and the immune system; yet a poor mix of microbes in the gut might contribute to chronic diseases such as diabetes and rheumatoid arthritis. Moreover, drug resistance, in which our arsenal of drugs are no longer effective against infections, is among the greatest threats to public health facing us today. This workshop will explore how the IRP can advance the study of these interconnected realms of microbiome and drug resistance by building on the program’s complementary strengths across immunology, immunotherapy, microbial and human genomics, cohort studies, animal model systems, and access to well-defined patient populations in the Clinical Center.

• Genomic methods for tracking drug resistant organisms and infectious diseases diagnostics
  Karen Frank, M.D., Ph.D. (CC)
• Investigating the human microbiome in health and disease
  Heidi Kong, M.D., M.H. Sc. (NCI/CCR)
• Lung cancer metabolomics identifies metabolites as robust risk biomarkers (FARE Award Winner)
  Majda Haznadar, Ph.D. (NCI/CCR)
• Community-associated Staphylococcus aureus–a global problem
  Michael Otto, Ph.D., M.S. (NIAID)
• High throughput screening to identify drug combination regimens for drug resistant bacterial infections
  Wei Sun, Ph.D. (NCATS)

Chair: Natasha Caplen, Ph.D. (NCI-CCR)

The diversity of functions ascribed to ribonucleic acid – RNA – seems to grow daily. Once thought to be just a “messenger” molecule, in the last few years we’ve seen the discovery of whole new classes of RNA, the identification of different species of RNA as critical regulators of gene expression via a diverse range of mechanisms, and we have seen RNA-based technologies revolutionize how we study gene function. In this workshop we will explore the structures and functions of RNA, the different ways in which this versatile molecule regulates gene expression, and we will discuss how RNA-based technologies can reveal potentially new treatment strategies and how RNA could eventually be exploited as a direct therapeutic target. The goal of this workshop will be to highlight how the IRP can take a leadership role in the development of a comprehensive program for the investigation and therapeutic exploitation of RNA.

• Riboswitch recognition of the bacterial alarmone ZMP through long-distance association of two RNA sub-domains
  Christopher Jones, Ph.D. (NHLBI)
• Single-molecule imaging reveals a switch between spurious and functional ncRNA transcription (FARE Award Winner)
  Tineke Lenstra, Ph.D. (NCI-CCR)
• Regulatory RNAs in aging
  Myriam Gorospe, Ph.D. (NIA)
• Defusing an oncogenic fusion transcript
  Natasha Caplen, Ph.D. (NCI/CCR)
• Nanoparticle platforms for siRNA delivery
  Xiaoyuan (Shawn) Chen, Ph.D. (NIBIB)

Thursday, September 17, 2015

Chair: Kanta Subbarao, M.B.B.S., M.P.H. (NIAID)

The IRP has contributed to about half of all FDA-approved vaccines currently in general use, possessing expertise across the broad spectrum of the vaccine development process, including basic immunology, molecular and structural biology, immunopathogenesis, bioinformatics, genomics, preclinical testing, vaccine production, and conduct of clinical trials. This workshop will address the development of an effective vaccine or other immune modulator for prevention and treatment of diseases such as HIV/AIDS and other high-burden diseases, such as respiratory syncytial virus, dengue, malaria, and tuberculosis, as well as biodefense threats and emerging and re-emerging infectious diseases including influenza, filoviruses (Marburg and Ebola), chikungunya, and MERS coronavirus.

• Malaria vaccines: fit to purpose
  Patrick Duffy, M.D. (NIAID)
• Structure-guided vaccine development for respiratory syncytial virus
  Barney Graham, M.D., Ph.D. (NIAID/VRC)
• Pandemic influenza vaccines
  Kanta Subbarao, M.B.B.S., M.P.H. (NIAID)
• Antibody binding to influenza nanoparticle immunogens as studied by Cryo-EM (FARE Award Winner)
  John Gallagher, Ph.D. (NIAID)
• Ebola vaccines
  Nancy Sullivan, Ph.D. (NIAID/VRC)
• A single-dose vaccine for dengue: How close are we?
  Stephen Whitehead, Ph.D. (NIAID)

Chair: Veronica Alvarez, Ph.D. (NIAAA)

One aspect of neuroscience under study at the NIH is the mechanisms and pathways that lead to obsessive behaviors such as drug and alcohol addiction, obsessive-compulsive disorder, and eating disorders. The IRP’s goal is to develop a detailed understanding of the pathway and molecular participants in these behaviors, and target therapies to exploit this improved understanding of behaviors with substantial morbidity and mortality. Neuroscience tools including advanced PET and MRI imaging, big data analysis, animal model systems, and neurochemistry and neuroanatomy will be discussed at this workshop.

• A synaptic mechanism associated with resilience to compulsive cocaine use
  Veronica Alvarez, Ph.D. (NIAAA)
• The neural circuitry of context-induced relapse to alcohol seeking after punishment-imposed abstinence (FARE Award Winner)
  Nathan Marchant, Ph.D. (NIDA)
• What can movement circuitry tell us about obesity?
  Alexxai (Lex) Kravitz, Ph.D. (NIDDK)
• Genetics and genomics of attention deficit hyperactivity disorder and substance use disorders
  Maximilian Muenke, M.D. (NHGRI)
• Neuronal ensembles in addiction
  Bruce Hope, Ph.D. (NIDA)

Chair: Carole Bewley, Ph.D. (NIDDK)

Natural products are produced by bacteria, fungi, marine organisms and plants, as opposed to synthetic chemicals assembled by humans. Many have therapeutic properties, and indeed many successful medicines (e.g., aspirin, Taxol™) derive from natural products. This workshop will cover themes ranging from the historical value of natural products and the central role that NIH researchers have played in moving natural products to the clinic, and how natural molecules inspire discovery of drugs and diagnostics. Looking to the future, speakers will discuss how the IRP can contribute to a national program facilitating natural products discovery for new molecules that target biological processes central to human disease. The IRPs vision includes a comprehensive Natural Products Library with pre-fractionated compounds for modern high-throughput targeted screening technology, and the creation of public databases and bioinformatics platforms to integrate source organisms, biological activities, structural, and genomic data.

• The evolution and use of the NCI’s Natural Products Repository, 1991–2014
  David Newman, D.Phil. (NCI)
• A potent anti-malarial from titration-based screening of marine natural product extracts
  James Inglese, Ph.D. (NCATS)
• Proteomic predictors of IgG responses to natural Plasmodium falciparum infection (FARE Award Winner)
  Eugene Liu, Ph.D. (NIAID)
• Natural products breaking bad–drug design for addiction
  Amy Newman, Ph.D. (NIDA)
• The NCI program for natural product discovery: building on strength
  Barry O’Keefe, Ph.D. (NCI)

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