Dr Alan Trounson, President, California Institute for Regenerative Medicine
A year after his last conversation with International Innovation, Alan Trounson returns to discuss the latest developments at CIRM, and cites a new era of biological medicine that will improve quality of life for the next generation
Since we last interviewed you at BioVision 2011, how has CIRM developed its strategy?
We have completed a nine-month strategic planning process and the resulting revised strategic plan was approved by our board on May 24. The current plan builds on our first one approved late in 2006 and an update we completed in 2009/2010. This time we have more clearly outlined our strategies for working closely with industry to move the fruits of our earlier projects into the clinic for patients. We have also adjusted the previously set goals to reflect the scientific advances in the field and reaffirmed our commitment to funding the continuum of research from basic to early phase clinical trials, adding an emphasis on basic research that has the potential to be transformative, ie. akin to the discovery of induced pluripotent stem (iPS) cells.
What areas within regenerative medicine does CIRM particularly focus on and why?
Early on we had a very strong preference for funding human embryonic stem cell work because that was the area in this field that had been starved for funding and needed jump-starting, both with direct research awards and training grants to bring new talent to the field. In the past couple of years we have pushed to fund more translational science, but kept an emphasis on projects that could not readily secure funding elsewhere. For example, our Disease Team awards, which have a goal of getting to a clinical trial in four years, include several adult stem cell projects that are more complex than most others in the field. They involve adult stem cells plus gene therapy to correct a defect, prevent infection (HIV/AIDS) or use adult cells as drug carriers. Going forward, we will be following the advances in the field and funding any cell types and projects that have the most promise of evolving into patient therapeutics.
Although your projects focusing on specific disease therapies have the end goal of finding a candidate drug or cell type to be developed into a therapy, what percentage of your research actually achieves this? Could you offer examples of how CIRM is supporting healthcare research right through to its clinical application?
Our translational projects are too recent to have transferred into clinical application. That is traditionally a very long process, often taking 10-20 years. We have 13 Disease teams that had a goal of getting to a clinical trial within four years of their 2010 launch. We had hoped they could accelerate the process because our awards required them to assemble a bench-to-bedside team at the outset and provided up to US $20 million to keep that team focused on the research, rather than raising money for various milestones along the way. Using expert external reviewers, we have completed extensive progress reviews on each of those teams and it looks like a good number of them should succeed in getting into clinical trials within that timeframe. Recently, on 26 July, we awarded another $151 million to eight more disease teams.
With increasing competition to secure funding, how will CIRM ensure that the agency attracts the best possible support?
CIRM’s funding derives from bonds issued by the state of California and the agency has the authority to expend up to $3 billion in this effort. We are working towards partnerships with major biotechnology, pharmaceutical and venture capital industries that will enhance our capacity to deliver therapies to patients. We also have collaborative funding arrangements with the National Institutes of Health (NIH) and 22 other international organisations and foundations. This creates an incredibly powerful leveraged network to drive therapeutic opportunity.
Each month you personally sift through stem cell research advances and publish a list of those you think move the field toward new therapies. In the last year, how has this field advanced and what are you particularly excited about?
There are many exciting advances, but let me cite just three types of advances that I think can really move the field forward; two are milestones the field knew it needed to reach, and one is a group of those potentially transformative discoveries I mentioned above that were not completely expected:
• Advance 1: A few groups have reported the ability to coax stem cells into forming complex, multilayer tissue; something that is key to many even simple repair scenarios
• Advance 2: Other researchers have made some very fundamental advances in scaling up stem cell and progenitor cell production, which is critical to commercialising most cell-based therapies, in particular, allogeneic ones
• Advance 3: During the past year, several teams have greatly expanded on a discovery initially made with muscle cells that you can directly reprogramme one adult cell to the function of a different adult cell without first taking it back to a pluripotent state such as an iPS cell. This opens up whole new avenues to therapy that had not been expected
In 2011, CIRM’s $271 million investment in 12 research facilities leveraged $884 million from private donors and institutions. In addition to creating 13,000 job-years and $100 million in tax revenue, the buildings will speed research toward cures by providing needed resources and encouraging collaboration. How have these research facilities evolved with the help of this generous funding?
This investment has enabled the scientists to collocate and to attract many new scientists to the centres. When I travel around the state, researchers working in these facilities frequently pull me aside to tell me how great the facilities are for accelerating their work, in providing ready access both to specialised equipment and valuable collaborators. Furthermore, we surveyed our grantees this winter and found that they had cited our facilities to attract follow-on funding of $190 million in new grants from NIH and other non-CIRM sources. That represents more real leverage.
How fundamental is collaboration and knowledge sharing to the advancement of healthcare research, and in what ways has it contributed to the success of CIRM’s work to date?
As I mentioned previously, one of the strengths of our new dedicated stem cell facilities is they make it easier to find and work with collaborators at all ends of the spectrum, from basic to translational and clinical research. Collaboration across that spectrum, including industry and academia, is the hallmark of our disease teams and why we think we may have been able to accelerate the normal pace of migration of discoveries from bench to bedside.
Do you have any involvement with international projects in developing countries and what value does CIRM place on such programmes?
We do not have relationships with developing countries but we do have formal collaboration agreements with 13 countries, ranging from Brazil and Argentina in South America, to Spain and Germany in Europe, and to China, Japan and India in Asia. These partners have committed more than $60 million to collaborative projects involving scientists in their country and in California. I have personal concerns that the developments we are making should be shared with all communities of the world and I shall work towards this outcome.
Do you see the public dissemination of your work as an important part of your purpose, and in what ways do you implement this?
We have recently put forward regulations that would place the same requirements for public access to our research results as the NIH puts on work it funds. That requires all research papers to be made publicly available within one year. We are also providing start up funding for a new journal called Stem Cells Translational Medicine, which had a first issue in January and is fully open access in its online edition. That was a requirement of our bidding process when we sought a publisher.
For the lay public, we have a robust web site and social media presence. On our website, all our grants are searchable by disease and we have 15 brief ‘chapters’ of Stem Cells 101 that walk people through a basic understanding of the field. We also have an active following on Facebook and Twitter and more than 160 videos on CIRMTV on YouTube. Our blog posts several items every week on advances in the field.
What are CIRM’s goals for the future? How do you see the role of the Institute changing in the next 10 years and beyond?
Our new strategic plan has set revised five-year goals that seek to increase our partnerships with industry and actively move discoveries into clinical trials. We expect to be funding several clinical trials within that window, and that plan is available on our website. With the last of our current bond financing set to be awarded in 2016 or 2017, any future activity we conduct would depend on the outcome of various strategies now under consideration to work towards the sustainability of the agency. It is premature to go into those options now.
Where do your personal research interests lie?
I gave up active research when I moved from Australia to California to lead CIRM. Prior to that my research was focused on lung repair and regeneration using stem cells, introducing gene expression markers into embryonic stem cells to enrich for cell lineage commitment in differentiation and the induction of tolerance for allogeneic cell transplantation.
How would you sum up the significance of regenerative medicine?
I am confident that we are entering a new era of biological developments that will enable us to utilise stem cells for biomedicine across an extraordinary breadth of disease, injury and infection. I see major advances in personalised medicine, increased understanding of disease and infection and unparalleled opportunity to improve quality of life for many people of the world.