Developing a 21st Century Neuroscience Workforce Workshop Summary (2015) / Chapter Skim
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5 Enhancing Training to Support Translational Research
5 Enhancing Training to Support Translational Research
Pages 57-68
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From page 57... ...
Joan Ferrini-Mundy and Story Landis described three common fallacies surrounding translation: undertranslation, overtranslation, and "pseudotranslation." Frank Yocca, vice president of Neuroscience iMed at AstraZeneca Neuroscience, discussed the pharmaceutical industry's re 57
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From page 58... ...
James Barrett, professor of pharmacology and physiology at the Drexel University College of Medicine, and Anthony Ricci, professor at the Stanford School of Medicine, described programs at their institutions dedicated to training students and postdoctoral researchers in translational science. UNDER-, OVER-, AND PSEUDO-TRANSLATION Ferrini-Mundy stated that neuroscientist trainees have to be mindful about both undertranslations -- failure to translate promising discoveries from the lab into clinical therapies -- as well as overtranslations -- misguided attempts to use neuroscience discoveries to explain or solve every human problem.
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From page 59... ...
For example, no biomarkers are known to exist for many large-market neurological diseases such as Parkinson's disease and Alzheimer's disease. With no way to quantify disease progression or to stratify patients into various disease stages, Yocca noted that it is difficult to determine the effect candidate treatments are having in patients.
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From page 60... ...
I SOURC CE: James Barrrett presentatio on, Drexel Uniiversity, Octobber 29, 2014. BOX 5-1 Neuroscien nce Presents Several Tran nslational Cha allenges entific Scie • Biologica al complexity and a nonvalida ated targets • Poor preeclinical modells • Challengge of the blood d/brain barrier • Direct ex xamination of drug d exposure e and target en ngagement nical Clin • Patient recruitment r • Patient heterogeneity h • Disease is advanced when w symptom ms appear • Capturingg therapeutic effects e on cliniccal scales with high variabilityy Low w productivity • Long cyc cle times • High cos sts • Low prob bability of succ cess SOURCE: Frank Yocca presenttation, AstraZe eneca Neurosccience, Octoberr 28, 2014.
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From page 61... ...
BOX 5-2 Opportunities for Changing the Approach to Training in Translational Neuroscience • Large internal teams working on literature targets and follow-on approaches Small internal teams collaborating with academic and biotech part ners working on genetically driven innovative targets • Limitations driven by rigid disease strategies More opportunistic approaches to find tractable targets regardless of disease state • Template approaches Smart discovery and development strategies (translational focus) • Focus on larger diseases driven by peak year sales Focus on smaller, genetic-based diseases driven by "line of sight" and return on investment SOURCE: Frank Yocca presentation, AstraZeneca Neuroscience, Oc tober 28, 2014.
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From page 62... ...
SOURC CE: Frank Yo occa presentatiion, AstraZeneeca Neurosciennce, October 228, 2014, adapted a from Cook C et al., 201 14. BOX 5-3 Novel Approaches A to t Neuroscien nce Translatiion • Large-scale unbiased app proaches to da ata collection and analysis and DNA seq quencing • Optogenetics s to focus on circuits c involve ed in diseasess • Identification of biochemic cal pathways iinvolved in dissease patho genesis • Akin to cancer, mutations within cells m may be proving g to be more important to therapy t than the t cell of orig in • The best way y to determinee convergent p pathophysiological mecha nisms lies in starting with genetic g discovveries • Multilevel analysis to eluciidate the caussal pathway frrom mutation to behaviorall disorder • Reprogramm ming skin cells from patients into functionaal neurons af fords us the opportunity o to develop cellu lar disease models
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From page 63... ...
However, Richard Tsien, professor of neuroscience at the New York University Langone Medical Center, emphasized that even though there are many trainees with the right skills and an interest in working on disease-targeted research challenges remain in matching trainees to the right company. Tsien added that similar to the decreased number of positions in academia industry might not be a secure career option for recent graduates.
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From page 64... ...
• Cell biologists with expertise in neuroscience and neurodevelopment • Cell biologists with expertise in stem cells • Neurophysiologists with system modeling expertise • Clinicians with expertise in neuroscience and neurodevelopment • Neurodevelopmental processes • Neurophysiologists/neuropsychologists • High-quality clinical studies • Novel pharmacology and repositioning tools • Genetics and patient segmentation • Objective end-points and biomarkers • High-quality diagnostics and patient segmentation • Response biomarkers • Clinical neurophysiologists and clinical psychologists • Functional imaging • High-quality clinical studies • Novel treatment strategies • Patient segmentation • Behavioral analysis, animal models in neuroscience and data cap ture and data analysis SOURCES: James Barrett presentation, Drexel University, October 29, 2014, and Frank Yocca presentation, AstraZeneca Neuroscience, October 28, 2014. 1 See http://www.jnj.com/partners/innovation-centers (accessed November 3, 2014)
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From page 65... ...
Yocca pointed out that the resulting translational research will also be enhanced, by providing training in translational science and neuroscience to experts in other fields such as cell biologists and clinicians, neurophysiologists, and clinical psychologists. BOX 5-5 Program Example: Drexel University Drexel University offers a Master of Science in Drug Discovery and Development that provides the rigorous scientific and technical training necessary to facilitate a smooth transition to a productive ca reer in the biotechnology or pharmaceutical industry.
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From page 66... ...
SOURCE: James Barrett presentation, Drexel University, October 29, 2014. Program Example: Stanford University School of Medicine Ricci discussed some of the innovative approaches that Stanford University is taking in training students in translational neuroscience.
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From page 67... ...
Finally, Ricci noted that Stanford also offers three unique professional development programs that prepare trainees for careers in translational neuroscience: Master of Medicine,2 Biodesign Program,3 SPARK Program.4 SUMMARY Neuroscience is in an era of growth and popularity. Given the scientific progress in the field, trainers seek to develop and strengthen training programs to better prepare the 21st century neuroscience workforce.
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