Clinical OMICS

SEP-OCT 2018

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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Page 14 of 51 September/October 2018 Clinical OMICs 13 then, two other genes, LRRK2 and GBA, have been associ- ated with familial, early-onset PD. For the more common, later onset form of the disease, there are dozens of genes associated with an increase in dis- ease risk. In January of 2015, Genentech paid 23andMe $10 million (with up to $50 million more in further milestones) to access their database to find more. The resulting paper, "A meta-analysis of genome-wide association studies identifies 17 new Parkinson's disease risk loci," published in Nature in September of 2017 was the larg- est meta-analysis of PD. When asked why searching for new genes was import- ant, Graham answered that although there are known path- ways that are important for PD, the pathways need to be rounded out. The way to do that is to identify more genes within them. For example, the lysosomal pathway, with its well-known role in the degradation of protein aggregates and autoph- agy, and its role in targeting long-lived proteins and dys- functional organelles for lysosomal degradation, plays a role in PD. However, the genes that are already known in those pathways may not make good drug targets. Therefore, hunting for new genes within those key pathways to find additional targets may result in novel drug targets. In addition, the core pathways that lead to disease pro- cesses can be present a decade or longer before clinical PD symptoms, making if difficult to identify specific genes for drug targets. Graham's work aims to find pathways that could be druggable when the patients present with their ini- tial clinical symptoms. And, Graham thinks that their study made progress on all of these fronts. Some of the genes his team identified fill in gaps within the well–accepted autophagy and lysosomal pathways, providing additional support for those while helping to pinpoint which genes play a specific role in PD. They also identified new pathways. One gene that they found, inosi- tol 1,4,5-triphosphate kinase B (ITKPB), may open up new pathways to target. What is Genentech's plan with all of these new genes? Gra- ham told Clinical OMICs that the neuroscience and genetics teams at Genentech are working to try to clarify the impor- tance of the newly identified genes, determine how the net- works interact and understand what new pathways may be important. Only after that, Graham said, can we think about therapeutic approaches to best target the disease. A Multi–Omics Approach to ALS Enough people dumped buckets of ice on their heads four years ago to raise $115 million dollars for ALS research. But, with one failed drug candidate after another, researchers are taking a new approach to find a cure for the 30,000 people living with ALS. One group, "Answer ALS," which was launched in Sep- tember of 2015, has a goal to "produce the largest and most comprehensive foundation of ALS data ever amassed." And to "use that data to inves- tigate the unique pathways of each variation of ALS and begin to develop the right cure for each patient." Started by Jeffrey D. Roth- stein, M.D., Ph.D., director of the Robert Packard Center for ALS Research at Johns Hop- kins Medicine, Answer ALS is using multi-omics analysis to comprehensively assess every aspect of motor neuron function in ALS patients. This includes genomics, tran- scriptomics, epigenomics, proteomics, metabolomics, and robotic imaging. Patients are recruited at eight centers across the United (continued on next page) "We're still trying to understand the basic biology of PD." —Rob Graham, Ph.D. Genentech wildpixel / Getty Images

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