Emory University has been highly successful in discovering small molecules, which became invaluable in the clinic for treating human diseases such as HIV/AIDS, HBV and HCV. These successes in drug discovery were possible due to the innovative work of outstanding academic scientists, as well as strong support from the NIH, CFAR and the University. Using these successes as a stepping stone, the Children’s Center for Drug Discovery serves as an academic hub of the Emory School of Medicine and Children’s Clinics for executing two highly coordinated and cooperative missions: 1) performing leading-edge basic science research for identifying new disease targets and 2) translating the newly identified targets to drug design, synthesis, evaluation and selection as potential clinical candidates. The ultimate goals of the Center are the development of extramurally funded basic research programs and the identification of small molecule candidates that will contribute to the discovery of new drugs. The clinical potential of the drugs will be evaluated up to Phase 2 clinical trials.
The initial disease targets of the Children’s Center for Drug Discovery are:
- Anti-HIV/AIDS drugs targeting replication and various viral reservoirs
- Anti-HCV drugs targeting viral replication
- Anti-HBV drugs targeting viral replication
- Anti-Ebola virus drugs
- Anti-Zika virus drugs
- Anti-Influenza virus drugs
- virus drugs targeting viral replication
- Anti-Norovirus drugs targeting viral replication
- Anti-Dengue virus drugs targeting viral replication
- Anti-respiratory syncytial virus (RSV) drugs targeting viral replication
- Anti-cancer drugs
- Treatment for opportunistic infections such as cryptosporidium, cytomegalovirus, trachoma, chlamydia, and tuberculosis
- Neglected diseases in children
CDD is pleased to have provided funding for proof-of-concept pilot projects for the last three years.
2018 Proof-of-Concept Pilots were awarded to the following investigators:
- Rabindra Tirouvanziam, PhD: "Cell-based Screen for Enhancers of Bacterial Killing by CF Airway Neutrophils"
- Roy L. Sutliff, PhD and Bum-Yong Kang, PhD: "Targeting Hemin-Induced Endothelial Cell Proliferation to Reduce Pulmonary Hypertension in Sickle Cell Disease"
2017 Proof-of-Concept Pilots were awarded to the following investigators:
- Paul Dawson, PhD: "Targeting Bile Acid Transporters for Pediatric Cholestatic Diseases"
- Christina Gavegnano, PhD: "Screening for Agents That Stimulate HIV-1 Replication and Expression in Macrophages for HIV Cure"
2016 Proof-of-Concept Pilots were awarded to the following investigators:
- Greg Melikian, PhD: "Screen a Small Molecule Library Provided by CDD for Inhibitors of Ebola Virus Fusion"
- Changwon Park, PhD and Young-sup Yoon, MD, PhD: "Cell-based Screening for Identifying Drugs for Diabetic Neuropathy"
- Chunhui Xu, PhD: "Screening Small Molecules to Promote the Proliferation of Cardiomyocytes Derived from Human Pluripotent Stem Cells"
Children’s Center for Drug Discovery includes faculty from the Department of Pediatrics, as well as other departments within the School of Medicine, Emory-Children’s Center, and other interested schools within the university.
Dr. Baek Kim - Director; Professor, Department of Pediatrics
Dr. Kim joined Emory’s faculty as a Professor within the Laboratory of Biochemical Pharmacology in 2013, joining us from the University of Rochester. He guides, oversees, promotes, and develops the Center and facilitates collaborations across the University. Dr. Kim’s 20 years of experience in biochemical and virological research, which has been fully supported by NIH, has been focused on the replication process and cell tropism of HIV/AIDS and influenza virus. Recently, Dr. Kim has recently initiated enzymological and mechanistic research on WNV and Dengue RNA polymerases, which will be incorporated into the drug discovery programs of the center.
Dr. Raymond Schinazi - Professor, Department of Pediatrics
Dr. Schinazi serves as the Frances Winship Walters Professor of Pediatrics and Director of the Laboratory of Biochemical Pharmacology, which houses over 40 scientists specializing in chemistry, virology, molecular biology, biochemistry, pharmacokinetics, and molecular modeling related to the development of drugs to treat HIV/AIDS, hepatitis B, hepatitis C, herpesviruses, Norovirus, and Dengue. Dr. Schinazi’s proven track record of success in drug discovery and development, combined with the diversity of scientific expertise within his group, will contribute toward the launch of the Center. Dr, Schinazi serves as the Director of the Scientific Working Group on Viral Eradication within Emory’s Center for AIDS Research.
Dr. Stefan Sarafianos - Acting Professor, Department of Pediatrics
Dr. Jan Mead - Professor, Department of Pediatrics
Dr. Mead focuses on opportunistic infections related to HIV infection, specifically Cryptosporidium parvum. Her expertise in immunology and SCID mouse models is frequently sought through grant collaborations and interdisciplinary projects.
Dr. James Kohler – Assistant Professor, Department of Pediatrics
Dr. Kohler serves as co-Director of the Scientific Working Group on Viral Eradication within Emory’s Center for AIDS Research. His experience in immunology and animal models is actively engaged within the Laboratory of Biochemical Pharmacology for drug development studies and grant proposals involving viral eradication for HIV, hepatitis B, and Dengue.
Dr. Selwyn Hurwitz – Assistant Professor, Department of Pediatrics
Dr. Hurwitz’s expertise in pharmacokinetic (PK) and pharmacodynamic (PD) modeling contributes to antiviral studies conducted in the Laboratory of Biochemical Pharmacology. He regularly collaborates with the Yerkes Primate Research Center and the Winship Cancer Institute on studies involving drug development and dosing for HIV, hepatitis, and cancer.
Dr. Franck Amblard – Assistant Professor, Department of Pediatrics
Dr. Amblard serves as the senior chemist in the Laboratory of Biochemical Pharmacology, focusing on organic and medicinal chemistry for the synthesis of anti-HIV and anti-HCV derivatives.
Dr. Leda Bassit – Assistant Professor, Department of Pediatrics
Dr. Bassit specializes in virology research focused on hepatitis B, hepatitis C, and Dengue. In the Laboratory of Biochemical Pharmacology, she has developed a high-throughput assay to test drug candidates for activity against hepatitis B and is currently receiving training through the University in translational research methods.
Dr. Christina Gavegnano – Assistant Professor, Department of Pediatrics
Dr. Gavegnano is involved in macrophage specific antiviral efforts for HIV and other viruses. Her work focuses on immunological mechanisms as drug targets. She oversees immunological, pharmacological, and toxicity based assays to identify and characterize novel cellular factor based inhibitors to treat viral diseases.
Children’s Center for Drug Discovery leverages the members’ existing collaborations with interested stakeholders including the Laboratory of Biochemical Pharmacology, Yerkes Primate Research Center, Emory’s Center for AIDS Research, and Atlanta VA Medical Center to promote interdisciplinary work in pursuit of the Center’s goals. The director also promotes the Center across the university to recruit additional members with specialties that can contribute to the Center’s mission of developing drugs to eradicate viral diseases affecting children.