New FDA report: Science Moving Forward, A Progress Report to the FDA Science Board

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New FDA report: Science Moving Forward, A Progress Report to the FDA Science Board

Contributor: Sherry Ward, AltTox

“Modernize Toxicology to Enhance Product Safety” is the first of the FDA’s 9 Scientific Priority Areas described on page 11 of the FDA’s new report, Science Moving Forward, A Progress Report to the FDA Science Board (

Intramural research on a non-animal approach includes the example on page 28 where “a Chief Scientist Challenge grant was awarded to scientists at CDRH and CDER to study novel in vitro electrophysiological approaches to measure the effects of drugs substances on human cardiac contractility….”

External research collaborations and funding are another approach to new toxicology tools resulting in the development of non-animal models, such as the funding of the Wyss Institute’s project to extend the capability of their organ-on-a-chip models for the assessment of radiation damage as part of the medical countermeasures program (pages 33 and 67).

As outcomes of this program, several predictive in silico systems and in vitro research models are described on pages 37-38. The development of new in vivo animal-based models is also a large part of the FDA effort to modernize toxicology. The following examples of accomplishments and activities were provided to illustrate the FDA’s progress:

  • Computational approaches including research in physiologically based pharmacokinetic (PBPK) modeling to improve dosimetry correlations between nonclinical species and individuals who are difficult to study, such as pregnant women and newborns.
  • In a collaborative study between the FDA, the Hamner Institute, and others, systems pharmacology modeling approaches were used to evaluate and predict drug hepatotoxicity through development of the DILIsym model.
  • Improved and patented methods of in silico modeling were used to build new models to predict drug toxicity to inform population-based safety risks; one such approach might enable precision medicine by identifying patient-specific genetic susceptibilities to individual drugs. • Novel model systems including zebrafish and human induced pluripotent (iPS) stem cells were used to study developmental toxicity and organ-specific toxicities.
  • Bioimaging techniques were developed to allow non-invasive assessment of toxicity. Coupled with cognitive function tests, these techniques were used to demonstrate, in non-human primates, neurotoxic effects of anesthetics that are routinely used in newborns (see example).
  • Organ-specific toxicities, such as drug-induced pancreatitis, were examined at the cellular level to improve the predictive usefulness of pre-clinical animal models.
  • Genomics, metabolomics, proteomics, and epigenetics were used to identify new biomarkers of toxicity. Work to date includes the identification of potential translational biomarkers of drug-induced liver injury in animals and humans.
  • Next-generation sequencing, bioinformatics, resistomics, transcriptomics, and metagenomics were applied to monitor trends and better understand the mechanism, emergence, persistence, and spread of antibiotic resistance.
  • Bioinformatic approaches were used to develop tools (e.g., FDALabel) to assist reviewers and create knowledge bases of divergent information that can be queried to identify previously unknown associations.