Deriving Drug Discovery Value from Large-Scale Genetic Bioresources Proceedings of a Workshop (2016) / Chapter Skim
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5 Potential Next Steps in Using Genomics to Advance Drug Discovery
5 Potential Next Steps in Using Genomics to Advance Drug Discovery
Pages 49-58
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From page 49... ...
; it is an in vitro platform that uses human tissues to assess the safety, efficacy, and toxicity of potential drugs. Following a brief presentation on the Tissue Chip, individual workshop participants revisited Sarwar's question and focused on two main topics: potential ways to foster a broader view of biology that fuses genetics with research on many other facets of health and disease; and ideas for sharing information, including data from genetic cohort studies, to enhance progress in developing new disease treatments.
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From page 50... ...
(Matrisian) Broadly communicate to patients and the public the value that is provided by the new tools and resources associated with genomics-driven drug discovery efforts.
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From page 51... ...
In 2016, NCATS plans to use the tissue chip technology to model rare diseases, and it will further expand the program to more common diseases in 2017. There are also plans to harness gene-editing technology to introduce various polymorphisms into induced pluripotent stem cells to look at individual drug responses.
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From page 52... ...
Russ Altman of Stanford University said that genetics research is an integral component of any data portfolio and can help drive drug discovery, but he cautioned that genetic data on their own are not sufficient to get the job done. "When we understand the biology, we can make enormous inroads in drug discovery," said Tim Rolph of Pfizer.
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From page 53... ...
For example, one workshop participant commented that while the concept of 23andMe is exciting, the population that the company is studying may not accurately represent the general population. And while disease registries tend to have relatively good phenotypic datasets, electronic health records generally do not contain research-grade phenotypic data, the participant noted, saying "phenotypic data has not yet reached a level that we've achieved for genotypic data." While phenotype measurements can be subjective, some fields are beginning to rethink the boundaries of phenotypic descriptions so that they are more biologically meaningful, said Mark Daly of the Broad Institute of the Massachusetts Institute of Technology and Harvard University.
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From page 54... ...
PROMOTING INFORMATION SHARING AND COLLABORATION The potential for collaboration and data sharing to enhance drug discovery has been demonstrated in the past, Mark Daly said. Over the past 7 years, more than 100 genetic associations have been discovered for schizophrenia, he said, which happened because more than 50 independent groups around the world were willing to deposit their genetic data on a single server and engage in a collaborative analysis activity
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From page 55... ...
Data Sharing as a Way to Reduce Research Duplication The sharing of genetic data is a powerful opportunity because it engages an entire community of data analysts, each of whom might have been previously working in individual data silos, Daly said. Geneticists need to do a better job of reaching out to the broader molecular biology community with well-annotated genetic results in order to make these data more accessible to the entire research community, he continued.
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From page 56... ...
To increase data sharing, Altman suggested establishing a website, potentially named geneticcohorts.gov that would function like ClinicalTrials.gov, where all applicable clinical trials in the United States must be preregistered before beginning participant enrollment. Such a website could announce the existence of a genetics-based cohort study, its size, the types of metadata associated with it, and information on whom to contact to initiate a collaboration.
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From page 57... ...
The realization that genetic support for drug targets increases the odds of success has underlined the importance of genomic research. This new perspective has contributed to substantial advances in the ability to treat and potentially cure diseases, and hope for continued advances is a strong motivator and enabler.
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From page 58... ...
58 USING GENETIC BIORESOURCES FOR DRUG DISCOVERY Each sector involved in drug discovery and development is now in a position to help realize the potential behind that privilege.
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