Text of Statement by Amander Clark of UCLA on Future Direction of California's Stem Cell Program

Clark's Talk was Aimed at Needs in Basic Research

Here is the full text of Amander Clark’s remarks to directors of the California stem cell agency. Clark provided them at the request of the California Stem Cell Report.

Thank you to Dr. Millan and CIRM Leadership for inviting me to frame the discussion on the strategic direction of basic science stem cell research by CIRM.

I am going to frame my discussion today in three areas: 

1. CIRM’s critical role in supporting basic science research and collaboration.

2. The importance of CIRM in taking a leadership role in regulation and policy of new stem cell science.

3. The role of CIRM in orienting basic stem cell research towards improving the health of Children and Young Adults

1. Basic Science Research and Collaboration

One thing we can all agree upon is that basic science discoveries have a profound and long-lasting impact on biomedical research and translation;  and these discoveries can come from the unlikeliest of places.  

Given this, leveraging and supporting excellence in basic science research in California, irrespective of discipline, should be the very foundation of the CIRM scientific pipeline.  California is home to a wide variety of Tier 1 research institutions containing laboratories and faculty who dedicate their careers to fundamental scientific discovery.  CIRM is in the unique position to harness this talent through research grants that support scientific excellence above and beyond what can be achieved with funds from the NIH and NSF.  For example, CIRM could focus on bold collaborative multi-PI stem cell grants that would be considered high-risk compared to those traditionally funded through the NIH system.  

Consistent with building this pipeline, CIRM is in a key position to provide resources to attract the best and the brightest to the State,  through a recruitment program that could involve matching funds with the recruiting Institution.  CIRM is also in a position to stabilize funding in critical areas of stem cell research that tend to be caught in shifting political winds.  This includes; human embryonic stem cells, fetal tissue research, stem cell-based embryo models and human embryo and gamete research, which is the fundamental science behind healthy families.

Today, it is rare for transformative stem cell science to occur in a single laboratory. Instead, it demands collaboration;  and in many cases the need to work with scientists and clinicians outside the State. Therefore, CIRM should leverage tax-payer dollars to develop a framework that enables California stem cell scientists to freely work with colleagues around the nation and world, while protecting IP developed with CIRM funding.  This could include evaluating the previous MOU agreements, establishing new agreements or creating a Fellows Program to fund outstanding  early career stem cell researchers inside and outside  the State. 

A major shift in stem cell science is the need for increased funding towards quantitative data science and machine learning.  CIRM should take a leadership role in establishing the infrastructure so that data-sets created with CIRM funding are centralized and accessible for scientists with a range of skill sets.  

In addition, and where possible, the collection of large data sets that utilize human samples should be inclusive of the diversity within the State of California, so that treatments and cures can be developed for everyone.  Therefore, CIRM should consider a diversity requirement when supporting basic science studies that involve human subjects research.   This is because promoting inclusion of diverse samples at the beginning of the basic science pipeline could be strong step towards eliminating health disparities as the pipeline builds towards treatments.

2. Taking a leadership role in regulation and policy

As CIRM continues to build on the stem cell-based screening and therapeutic pipeline created under proposition 71, it is imperative that CIRM continues to take a leadership role and also a global view in coordinating National Academies, Societies Governments and Funding Agencies world-wide to ensure that new forms of stem cell science move forward under a coordinated scientific and ethical framework.  Specific examples are the new stem cell based embryo models.  This will be essential to ensure that stem cell therapies move safely and swiftly from bench to bedside in California and around the world.    

3. The future

With few notable exceptions, SCID being one,  the last 14 years of CIRM funding under proposition 71 has placed significant emphasis on the use of stem cells to improve health and develop cures for adults including a focus on cancer as well as degenerative disease.  It is important that the next phase of CIRM funding intentionally focus on the health of future Californians.  Including understanding the developmental origins of disease, with a focus on developmental biology, child health and the health of young adults.  This is particularly critical as CIRM turns its focus towards the use of stem cells to understand and treat diseases of the brain and central nervous system. CIRM should be applauded for taking a leadership role in this area.   Today we will hear a number of talks about the use of stem cells to treat degenerative brain disorders, Here, I would also like to advocate for other diseases of the brain, including psychiatric or mental disorders which disproportionately affect children and young adults, and in many cases disproportionately affect minority populations.  To make an impact in this area will require an investment in basic science.  

For example, due to the large and diverse population in California, CIRM is uniquely positioned to develop the framework for identifying genes in children who are at high risk for autism, bipolar disorder and other psychiatric diseases, and utilizing ips cell technology to understand role of these genes in brain cell development.  

Furthermore, given there is a developmental etiology to many severe psychiatric conditions, it is essential that CIRM invests in basic research on prenatal and postnatal brain development including non-invasive studies in children in order to understand circuits and wiring.   

Finally, a focus on how the environment affects brain development, and how the environment could be associated with the emergence of psychiatric disorders and neurological disease will be important areas of future investigation.  Once fundamental discoveries are made in these areas, the utility of stem cells to understand and treat diseases of the brain and CNS will begin to emerge.

In summary, CIRM is in a unique position to make a significant impact in stem cell science in the state of California the nation and around the world.  This begins with investment in the basic science, in collaboration, regulation and policy and an intentional focus on the health of current and future generations of Californians.