Neurology Central

Neuroregeneration: pipelines, trials and challenges – an interview with the California Institute for Regenerative Medicine

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As part of our Spotlight on neuroregeneration, we spoke to Helen Budworth, Kent Fitzgerald (both Science Officers) and Kevin McCormack (Senior Director Public Communications & Patient Advocate Outreach) from the California Institute for Regenerative Medicine (CIRM; CA, USA). They tell us more about CIRM’s current clinical portfolio and discovery pipeline, as well as the challenges involved in bringing neuroregenerative therapies to the clinic, and where we could be 10 years from now.

First, please could you tell us about your backgrounds? How did you both become interested in regenerative medicine?

HB: I completed my studies in the UK with a BSc in Genetics and Microbiology from the University of Liverpool and a DPhil in Biochemistry from the University of Oxford (both UK). My academic background is in the genetics of rare diseases and I studied these using a variety of model organisms including those with remarkable regenerative capabilities such as Planaria. I spent a number of years at Lawrence Berkeley National Laboratory (CA, USA) conducting cellular studies of diseases including Huntington’s disease, various ataxias, DNA repair deficiencies and cancers.

KF: Much of my academic and professional career has centered around neurodegenerative diseases. Working in this space in the context of drug discovery and development one becomes painfully aware of the limited therapeutic interventions currently available to those suffering from degenerative diseases. Regenerative medicine is at a critical inflection point where knowledge generated over the past decade-plus is at a point where it can be applied to clinical evaluation. CIRM has been at the forefront of these developments and my previous experience in translational science gives me a chance to help push our funded projects toward clinical application.

Can you tell us a little about CIRM? What do your roles there entail?

HB: At CIRM our mission is to accelerate stem cell treatments to patients with unmet medical needs and this mission guides all our activities. I am a Senior Science Officer in the Therapeutics Group at CIRM. My role includes recruiting high quality regenerative medicine programs at the clinical and late-stage preclinical stages to apply to CIRM for funding, and enabling CIRM-funded investigators to drive successful programs to next stages of development.

KF: As Helen mentioned, all of our job roles point to enabling the CIRM mission of accelerating therapies to patients. In my role as a Senior Science Officer / Program Officer in the Discovery and Translation group, I manage a diverse portfolio of CIRM-funded projects at this stage of development. Management from the Science Officer end involves acting as an external resource for project PIs where I can use my own expertise as well as other CIRM resources to increase the likelihood scientific success and advancement to the next stage of development. I also work with prospective applicants (for funding) to align their projects with the appropriate CIRM program.

What are CIRM’s focuses at the Discovery/Translational stage of neuroregeneration research?

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