Clinical OMICS

JUL-AUG 2019

Healthcare magazine for research scientists, labs, pathologists, hospitals, cancer centers, physicians and biopharma companies providing news articles, expert interviews and videos about molecular diagnostics in precision medicine

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4 Clinical OMICs July/August 2019 www.clinicalomics.com News CRISPR-Cas9 Used to Create Primate Model of Autism Researchers headed by teams at the Chi- nese Academy of Sciences and Massa- chusetts Institute of Technology (MIT) have used CRISPR-Cas9 gene editing to engineer macaque monkeys with germ- line-transmissible mutations in Shank3, a gene linked with a form of autism and oth- er human neurodevelopmental disorders. The engineered animals demonstrated altered brain connectivity patterns, and similar behavioral, motor, and social ab- normalities that are seen in people with mutations in the same gene. The researchers say the primate mod- el is more relevant than existing rodent models for studying human neurodevel- opmental disorders, and could potentially help scientists identify therapeutic strate- gies and drug candidates. "We urgently need new treatment op- tions for autism spectrum disorder, and treatments developed in mice have so far been disappointing," noted co-author Robert Desimone, Ph.D., director of MIT's McGovern Institute for Brain Research. "While the mouse research remains very important, we believe that primate genet- ic models will help us to develop better medicines and possibly even gene thera- pies for some severe forms of autism." n Robert Desimone, Ph.D., MIT GRAIL's Multi-Cancer Early Detection Blood Test Delivers Positive Results GRAIL is a step closer to developing a single blood test to find cancer early. The company's candidate blood test detected a strong signal for 12 cancer types at Stages I–III, according to results presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. Further, the test performed with a specificity of at least 99% (or a false positive rate of 1% or less). The test also predicted each cancer's site of origin with high accuracy. These are early data from GRAIL's Circulating Cell-free Genome Atlas (CCGA) study, which comprises samples from about 15,000 patients. "These exciting results suggest we can achieve what we believe are the requirements for a cancer screening blood test, including detection of multiple deadly cancer types at early stages in a single test, high accuracy in determining where the cancer originat- ed, and a very low false positive rate," said then GRAIL CEO Jennifer Cook, who has since stepped down from her post. "Our improved methylation-based technology has the po- tential to address gaps that exist with today's screening options, which are limited to a few cancer types and only screen for one cancer type at a time." GRAIL's sequencing data- base of cancer and non-cancer methylations signatures is believed to be the largest of its kind and covers approximately 30 million methylation sites across the genome. With $1.6 billion in venture capital, GRAIL is a favorite to launch the first risk-determin- ing liquid biopsy test. The company is privately owned and backers include Jeff Bezos and Bill Gates. The entire market for liquid biopsies is estimated to reach $2 billion by 2020. This includes tests for monitoring, prognosis, theranostics, and screening. The CCGA is a key to GRAIL's future. It is a prospective, multi-center, observational study that has enrolled participants, both with and without cancer, from clinical networks in the United States and Canada. Clinical information, demographics, and medical data relevant to cancer status are collected from all participants and their medical record at baseline (time of biospecimen collection), and then from their medical record at intermittent fu- ture time points, at least annually for up to five years. n Emilija Manevska / Moment / Getty Images

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