Cellular Technologies

From Patients to Neurons and Back Again

Monday, February 5
3:30 PM - 4:00 PM
Location: 6D

The foundational idea for the use of patient-specific induced pluripotent stem cells (iPSCs) in disease modeling was that this approach would result in a significant improvement in the efficiency of drug discovery and, eventually, in more success in the clinic. Whether this is true depends heavily on the predictive power of this human cell-centric approach, compared to that using cell lines of the types more widely employed in industry. This is a reasonable time to assess the amount of progress that has been made thus far.

Most of the work in my laboratory has focused on studying neurodegenerative and neuropsychiatric diseases using human neurons and other cells derived from iPSCs. Our work, as well as that from numerous other labs, has shown that some, but not all, aspects of these disorders can be reproduced in rather simple tissue culture environments. Moreover, recent studies have demonstrated that iPSC-based research can provide new information concerning previously unrecognized disease features, as well as generate data supporting the use of specific therapeutics on defined patient populations. On the other hand, it seems increasingly likely that more complex in vitro systems, particularly those in which neurons differentiate in 3-dimensional conditions, will be required to model other disease-associated features. These include changes that either require significant amounts of time to occur or are associated with modified neural circuit behavior. I will present several examples of work showing the advantages and limitations of iPSC-derived neuronal work.

Lee L. Rubin

Professor; Director of Translational Medicine
Dept of Stem Cell and Regenerative Biology, Harvard University; Harvard Stem Cell Institute

Dr. Rubin has a broad experience in both academia and industry, particularly in the realms of cell-based assays and drug discovery. Prior to coming to Harvard, he was Chief Scientific Officer of Curis, Inc., a Cambridge-based biotechnology company, where his group identified the first small molecule antagonist of the hedgehog signaling pathway that was developed by Genentech and is now approved as oral treatment for basal cell carcinoma. 
 
At Harvard, much of his work is focused on finding key molecular mediators of different neurodegenerative diseases. For example, his group has identified new targets for the treatment of the motor neuron disorders Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis.  Recently, his group discovered that a circulating protein, GDF11, has the ability to reverse some of the changes in the CNS associated with aging. They are actively exploring the therapeutic implications of these observations as well.

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