BioPharm America 2011: Regenerative Medicine and Cell Therapy Briefing: The Road to Commercialization

Amidst the buzz of partnering activity throughout the three day BioPharm America 2011 meeting held in Boston, MA from September 7-9, 2011, there were ample opportunities to hear industry leaders’ perspectives on industry trends through workshops and panel discussions.  Of particular interest was a session focused on stem cells in regenerative medicine and featured here, given the powerful allure of stem cells to captivate ALS researchers, drug developers, and patients alike.  With the generation of ALS patient-derived iPS cell lines and the ground-breaking safety trial of Neuralstem’s stem cell therapy in ALS patients, there are high expectations for stem cells to revolutionize ALS drug discovery and therapeutics.  Let’s hear what industry leaders had to say …

The session opened with a panel moderated by Brock Reeve, Executive Director of the Harvard Stem Cell Institute.  In his introductory comments, Reeve reminded the audience that the first iPS publication came out just five years ago and since then the technology has become almost commonplace in labs across the world.  Moreover, the technology has already been improved upon: a subsequent technique was developed to directly induce differentiated fibroblasts into a motor neuron identity, skipping the reprogramming step to the undifferentiated state.  These so called induced motor neurons (or iMN) could potentially make it even easier and faster to generate patient specific models of motor neuron disease. [See also related news on recent advances to purify and differentiate stem cells as well as remaining potential pitfalls.]

The “big opportunity” that lay in stem cells was clearly appreciated by the pharma and biotech panel experts who acknowledged the growing trend to incorporate adult, embryonic or induced pluripotent stem cell technologies into a variety of efforts, ranging from drug discovery to development of cell-based therapies.  The exciting ferment in the stem cell space has also added to industry’s growing interest in and enthusiasm for fostering academic collaborations.  While innovation in the stem cell space can come from anywhere, Gary Neil, Corporate VP at J&J, expects most to come from the academic sector.

Both panelists and audience members commented on the more frequent appearances of pharma representative of the likes of Pfizer, J&aJ, and GSK turning up at regenerative medicine conferences and partnering meetings such as this one as illustrative of pharma’s growing interest in regenerative medicine.  One explicit example of this interest, Shire’s recent acquisition of AdvBioHealing, was taken as a signal that the specialty pharma’s intends to build a regenerative medicine franchise around its newly acquired iPS technology.

Reeve asked panelist to offer their perspectives on potential breakthroughs within the proximate five-year horizon for regenerative medicine.  Near term success was expected for macular degeneration and other eye diseases as well as for diabetes.  Simple models with easy to measure clinical endpoints were perceived as more likely to succeed, whereas diseases with more complex biology (i.e.: stroke and ALS) were
perceived as more difficult.

The second panel moderated by John McNeish, Founder of Boston Stem Cell and ex-Pfizer Executive Director of Regenerative Medicine, explored the use of stem cells for drug discovery and translation in greater depth.  iPS cells in particular hold great excitement for use in disease modeling, target validation, and drug and biomarker discovery programs.  iPS cells also offer a unique opportunity to identify which patients are more likely to respond to a drug by first testing the drug on the patient’s own stem cells.  In contrast to the relatively young iPS field, panelists noted that the more mature areas of adult and embryonic stem cell biology have yielded several cellular therapies that have moved steadily ahead into a clinical setting, including Advanced Cell Technology’s hESC-based Retinal Pigment Epithelial (RPE) therapy for degenerative retinal diseases, Geron’s hESC-derived oligodendrocyte progenitor cell therapy for spinal cord injury, Neuralstem’s neural stem cell therapy for ALS and major depression, and over 37 ongoing clinical trials using adult stem cell therapeutics.

How close are we to remediating disease in a dish? Very close, according to Leonard Zon, stem cell researcher at HHMI/Children’s Hospital Boston and co-founder of Fate Therapeutics, who projects that in three to five years researchers will be able to successfully treat a disease pathology exhibited by the affected cell type derived from patient iPS cells.  This is an area of intense investigation, as many groups are actively working to establish disease models in a dish from patient-derived iPS cells and to develop functional assays to enable drug screening, including those studying ALS at The New York Stem Cell Foundation, the California Institute for Regenerative Medicine and elsewhere.

For ALS, progress has been slowed by an unanticipated finding.  Unlike motor neurons derived from ALS mouse ESCs, which exhibit a characteristic disease pathology, motor neurons generated from both familial and sporadic ALS patient iPS cell lines lack an obvious disease phenotype even after extended periods in culture.  The apparently healthy nature of these cells may bode well for transplantation back into the patient for use as a cellular therapy.  However, the difficulty in detecting a disease phenotype necessitates the development of sensitive assays to identify a disease signature, a necessary step before these cells can be used to screen drugs.

While progress is being made on several fronts to advance stem cell-based drug discovery and therapies, the field continues to wrestle with the hype surrounding stem cells – a double edged sword for the field. While a certain amount of hype attracts investment money, it also preys on the vulnerabilities of patient communities, desperate for treatment.  As ACT’s Business Development Director Matthew Vincent noted, regenerative medicine’s snake oil practitioners will hamper legitimate scientific efforts and slow the entire field.  He cautioned against getting caught up in the hype and hubris over the revolutionary nature of stem cells when it comes to managing the associated risks, which range from regulatory to manufacturing to reimbursement.  Goeff MacKay, President and CEO of Organogenesis, further emphasized the importance of assessing reimbursement very early in the process, calling it an early go/no-go decision point.

To watch the videos of the regenerative medicine panels presented here, click the links: panel 1 and panel 2.  Additional meeting coverage discussing pharma trends can be found here: The future of drug development, Angus Russell: Pharma must change to compete in new world market and Creativity, productivity and the future of pharma. – Sheila Menzies, PhD, Scientific Program Officer at Prize4Life

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