Thursday, October 27, 2011

Regenerative Medicine and Cell Therapy Briefing: The Road to Commercialization

The following blog was originally posted on Prize4Life's ALS Forum. For more articles on the latest research and drug development news, along with a wide variety of ALS research tools and resources, visit

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&J, 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

Tuesday, October 25, 2011

Dr. Ed Kaye on ALS and Incentive Prizes: The Extended Interview

As part of our continuing series featuring interviews with some of the leading players in the field of ALS research, drug development, and treatment, Prize4Life spoke with Dr. Ed Kaye, currently the Chief Medical Officer at AVI BioPharma and the former Group Vice President and Therapeutic Head for Lysosomal Storage Disorders and Neurodegenerative Diseases at Genzyme Corporation. Dr. Kaye serves on Prize4Life's Scientific Advisory Board. In this video, he shares his thoughts on the recent $1M ALS Biomarker Prize award, the importance of private funding to complement public funding of ALS research, and the potential of the incentive prize model in biomedical research.

You can watch all of the extended interviews by subscribing to our YouTube channel here. You can also view the original video these interviews were collected for, 'Driving Breakthroughs in ALS Research: Prize4Life and the $1M ALS Biomarker Prize,' below.

Friday, October 14, 2011

Dr. Robert Horvitz on ALS Research: The Extended Interview

Earlier this year, Prize4Life interviewed some of the top minds in the ALS research, clinical, and industry communities asking for their thoughts on ALS, the drug development landscape, and the potential of the incentive prize model in spurring biomedical innovation. These interviews were compiled into a short video that was screened at a gala in New York City celebrating the awarding of the $1M ALS Biomarker Prize to Dr. Seward Rutkove.

We are sharing the extended versions of these interviews on our website. The first video featured Dr. Vicki Sato, a professor at Harvard Business School and former president of Vertex Pharmaceuticals. You can view that video here.

The following is an interview with Dr. Robert Horvitz, a Professor of Biology at the Massachusetts Institute of Technology, an investigator with the Howard Hughes Medical Institute, and winner of the 2002 Nobel Prize in Physiology for discovering and characterizing the genes controlling cell death in the nematode worm Caenorhabditis elegans. Dr. Horvitz discusses his personal connection with ALS, the need for increased funding in the field, and how his work advances ALS research.

You can also view the original video, 'Driving Breakthroughs in ALS Research: Prize4Life and the $1M ALS Biomarker Prize,' below.

Tuesday, October 11, 2011

5K4Life Is Fast Approaching!

The Third Annual 5K4Life, benefiting Prize4Life, is almost upon us!

Our founder and CEO, Avi Kremer, will be in attendance for the race this year. Avi was diagnosed with ALS in 2006 and launched Prize4Life to rapidly accelerate the discovery of a cure for the disease.

When: November 6th
8:00 - 10:45 AM Race Registration and Race Packet Pick-Up
11:00 AM Race Start
Where: Kendall Square / 15 Cambridge Center

Members of Team Ashley helped make last year's 5K4Life a tremendous success...

ALS is a devastating neurodegenerative disease that afflicts more than 600,000 people around the world, robbing them of the ability to move, speak, and ultimately to breathe. Most patients will die within 2 to 5 years.

But you can be a part of the cure! Support Prize4Life's efforts to drive breakthroughs in research at the 5K4Life!

Here's how you can help:

1. Register today for the 5K at
2. Volunteer for the race by emailing
3. Donate to Prize4Life here
4. Fundraise for the 5K4Life to help bring us closer to a world free of ALS

Fundraising for Prize4Life is a way to honor someone you know living with ALS, to remember someone who has passed away from the disease, or to deliver hope to the thousands of ALS patients and their families battling it. Join Prize4Life's efforts, and the next ALS breakthrough could be yours...

Create your own fundraising page here.

Thank you for your support! Please share word of the 5K4Life with your friends and networks, and RSVP for the race on Facebook.