Stem Cell Agency Board Approves Roadmap for Next Five Years

Dr. Maria Millan, CIRM’s President & CEO

It’s hard to get somewhere if you don’t know where you are going. Without a map you can’t plan a route to your destination. That’s why the CIRM Board approved a new Strategic Plan laying out a roadmap for the Stem Cell Agency for the next five years.

The plan builds on the achievements of Proposition 71, the voter approved ballot initiative that created the Agency in 2004, including:

  • Supporting 76 clinical trials.
  • Helping cure more than 40 children born with a rare, fatal immune disorder.
  • Creating the Alpha Clinics Network that specializes in the delivery of stem cell therapies to patients.
  • Training over 3000 students and scholars to become the future workforce of regenerative medicine.
  • Stimulating California’s economy with $10.7 Billion in additional sales revenue and the creation of 56,000 new jobs (between 2004-2018)

The passage of Proposition 14 in 2020 has positioned CIRM to continue to accelerate research from discovery to clinical; to drive innovative, real-world solutions resulting in transformative treatments for patients; and to ensure the affordability and accessibility of those treatments to a diverse community of patients in an equitable manner, including those often overlooked or underrepresented in the past.

“We achieved a lot in the last 15 years and this provides a solid foundation for our strategy to bring us to the new era of CIRM and to deliver the full potential of regenerative medicine, says Dr. Maria T. Millan, the President and CEO of CIRM. “This plan lays out a roadmap for us to overcome the challenges in developing transformative therapies and making them accessible and affordable in an equitable fashion to a diverse California. The plan will guide us in that work through the development of novel scientific endeavors, effective healthcare delivery models, and expanded education and training programs.”

The Strategic Plan is organized into three main themes:

  • Advance World Class Science – Foster a culture of collaborative science by creating knowledge networks and shared research tools and technologies that encourage and facilitate data and resource sharing.
  • Deliver Real World Solutions – Accelerate approval of therapies by optimizing our support models for CIRM-funded clinical trials with attention to including underserved communities; build the California Manufacturing Network to overcome manufacturing hurdles; and expand the Alpha Clinics network and create the Community Care Centers of Excellence to deliver therapies to a diverse patient population often in underserved communities.
  • Provide Opportunity for All – Build a racially, ethnically and experientially diverse and highly skilled workforce to support the growing regenerative medicine economy in California; deliver a roadmap for access and affordability of regenerative medicine for all California patients.

Reflecting these goals, CIRM’s new mission statement is: Accelerating world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.

“We realize that these are ambitious goals but they are achievable,” says Dr. Millan, “If CIRM is going to continue to be a global leader in the field of regenerative medicine, and to live up to the faith shown in us by the people of California, we believe we have to aim high. We have a terrific team, a clear vision and a determination to fulfill our mission. And that’s what we intend to do.”

Creating a New Model for Diversity in Scientific and Medical Research

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Nature Cell Biology cover

The global pandemic has highlighted many of the inequities in our health care system, with the virus hitting communities of color the hardest. That has led to calls for greater diversity, equity and inclusion at every level of scientific research and, ultimately, of medical care. A recently released article in the journal Nature Cell Biology, calls for “new models for basic and disease research that reflect diverse ancestral backgrounds and sex and ensure that diverse populations are included among donors and research participants.”

The authors of the article are Dr. Maria T. Millan, CIRM’s President & CEO; Rick Horwitz Senior Advisor and Executive Director, Emeritus, Allen Institute for Cell Science; Dr. Ekemini Riley, President, Coalition for Aligning Science; and Dr. Ruwanthi N. Gunawardane, Executive Director of the Allen Institute for Cell Science.

Dr. Maria Millan, CIRM’s President & CEO, says we need to make these issues a part of everything we do. “At CIRM we have incorporated the principles of promoting diversity, equity and inclusion in our research funding programs, education programs and future programs. We believe this is essential to ensure that the therapies our support helps advance will reach all patients in need and in particular communities that are disproportionately affected and/or under-served.”

The article highlights how, in addition to cultural, environmental, and socioeconomic factors, genetic factors also appear to play a role in the way disease affects different people. For example, 50 percent of people in South Asia have genetic traits that increases their risk for severe COVID-19, in contrast only 16 percent of Europeans have those traits.

But while some studies have shown how African American men are at greater risk for prostate cancer than white men, most of the research in this and other areas has been done on white populations of European ancestry. Efforts are already underway to change these disparities. For example, the National Institutes of Health (NIH) has sponsored the All of Us Research Program, which is inviting one million people across the U.S. to help build one of the most diverse health databases in history.

The article in Nature Cell Biology stresses the need to account for diversity at the individual molecular, cellular and tissue level. The authors make the point that diversity in those taking part in clinical trials is essential, but equally essential is that diverse biology is accounted for in the scientific work that leads to the development of potential therapies in order to increase the likelihood of success.

That’s why the authors of the article say: “If we are to truly understand human biology, address health disparities, and personalize our treatments, we need to go beyond our important, ongoing efforts in addressing diversity and inclusion in the workforce and the delivery of healthcare. We need to improve the data we generate by including diverse populations among donors and research participants. This will require new models and tools for basic and disease research that more closely reflect the diversity of human tissues, across diverse donor backgrounds.”

“Greater diversity in biological studies is not only the right thing to do, it is crucial to helping researchers make new discoveries that benefit everyone,” said Ru Gunawardane, Executive Director of the Allen Institute for Cell Science.

To do this they propose creating “a suite” of research cells, such as human induced pluripotent stem cell (hiPSC) lines from a diverse group of individuals to reflect the racial, ethnic and gender composition of the population. Human iPSCs are cells taken from any tissue (usually skin or blood) from a child or adult that have been genetically modified to behave like an embryonic stem cell. As the name implies, these cells are pluripotent, which means that they can become any type of adult cell.

CIRM has already created one version of what this suite would look like, through its iPSC Repository, a collection of more than 2,600 hiPSCs from individuals of diverse ancestries, including African, Hispanic, Native American, East and South Asian, and European. The Allen Institute for Cell Science also has a collection that could serve as a model for this kind of repository. Its collection of over 50 hiPSC

lines have been thoroughly analyzed on both a genomic and biological level and could also be broken down to include diversity in donor ethnicity and sex.

Currently researchers use cells from different lines and often follow very different procedures in using them, making it hard to compare results from one study to another. Having a diverse and well defined collection of research cells and cell models that are created by standardized procedures, could make it easier to compare results from different studies and share knowledge within the scientific community. By incorporating diversity in the very early stages of scientific research, the scientists and therapy developers gain a more complete picture of the biology disease and potential treatments.  

Bridges Scholar Spotlight: Samira Alwahabi

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For more than a decade, CIRM has funded a number of educational and research training programs to give students the opportunity to explore stem cell science. One such project, the Bridges to Stem Cell Research program, helps train future generation of scientists by preparing undergraduate and master’s students from several California universities for careers in stem cell research.

Last summer, the Pacific Division of AAAS organized a ‘Moving on from COVID-19’ virtual forum specifically focused on students of science presenting their future career and research plans through 3-5 minute descriptive videos. 

Samira Alwahabi, a Bridges scholar and undergraduate student majoring in Biological Sciences at California State University, Fullerton was one of the many participants who submitted a video detailing their current work and future aspirations. Alwahabi is a CIRM intern conducting research in the Kuo lab at the Stanford University School of Medicine where she focuses on the identification and characterization of human distal lung stem cells as well as the effects of the novel SARS-CoV-2 virus on the human distal lung through the use of organoids. Her video, which you can watch below, was recognized for “Best Video Submission by an Undergraduate Student.” 

We reached out to Samira to congratulate her and she shared a few words with us about her experience with the Bridges program:

I am very grateful to the CSUF Bridges to Stem Cell Research program for giving me the opportunity to pursue research in the Kuo Lab at Stanford University. The past 11 months have been nothing less than exceptional! I have learned more than I could have even imagined and have been able to really solidify my future career goals through hands-on practice and interactions with professionals at all levels in the field of medical research. The CIRM Bridges program has allowed me to better understand how medical advancements are made and helped to further strengthen my interest in medicine. My future career goals include a career in medicine as a physician, where I will be able to use my research experience to better understand medical innovations that translate into improved quality of care for my patients. 

Congratulations Samira!

Creating a diverse group of future scientists

Students in CIRM’s Bridges program showing posters of their work

If you have read the headlines lately, you’ll know that the COVID-19 pandemic is having a huge impact on the shipping industry. Container vessels are forced to sit out at anchor for a week or more because there just aren’t enough dock workers to unload the boats. It’s a simple rule of economics, you can have all the demand you want but if you don’t have the people to help deliver on the supply side, you are in trouble.

The same is true in regenerative medicine. The field is expanding rapidly and that’s creating a rising demand for skilled workers to help keep up. That doesn’t just mean scientists, but also technicians and other skilled individuals who can ensure that our ability to manufacture and deliver these new therapies is not slowed down.

That’s one of the reasons why CIRM has been a big supporter of training programs ever since we were created by the voters of California when they approved Proposition 71. And now we are kick-starting those programs again to ensure the field has all the talented workers it needs.

Last week the CIRM Board approved 18 programs, investing more than $86 million, as part of the Agency’s Research Training Grants program. The goal of the program is to create a diverse group of scientists with the knowledge and skill to lead effective stem cell research programs.

The awards provide up to $5 million per institution, for a maximum of 20 institutions, over five years, to support the training of predoctoral graduate students, postdoctoral trainees, and/or clinical trainees.

This is a revival of an earlier Research Training program that ran from 2006-2016 and trained 940 “CIRM Scholars” including:

• 321 PhD students
• 453 Postdocs
• 166 MDs

These grants went to academic institutions from UC Davis in Sacramento to UC San Diego down south and everywhere in-between. A 2013 survey of the students found that most went on to careers in the industry.

  • 56% continued to further training
  • 14% advanced to an academic research faculty position
  • 10.5% advanced to a biotech/industry position
  • 12% advanced to a non-research position such as teaching, medical practice, or foundation/government work

The Research Training Grants go to:

AWARDINSTITUTIONTITLEAMOUNT
EDUC4-12751Cedars-SinaiCIRM Training Program in Translational Regenerative Medicine    $4,999,333
EDUC4-12752UC RiversideTRANSCEND – Training Program to Advance Interdisciplinary Stem Cell Research, Education, and Workforce Diversity    $4,993,115
EDUC4-12753UC Los AngelesUCLA Training Program in Stem Cell Biology    $5 million
EDUC4-12756University of Southern CaliforniaTraining Program Bridging Stem Cell Research with Clinical Applications in Regenerative Medicine    $5 million
EDUC4-12759UC Santa CruzCIRM Training Program in Systems Biology of Stem Cells    $4,913,271
EDUC4-12766Gladstone Inst.CIRM Regenerative Medicine Research Training Program    $5 million
EDUC4-12772City of HopeResearch Training Program in Stem Cell Biology and Regenerative Medicine    $4,860,989
EDUC4-12782StanfordCIRM Scholar Training Program    $4,974,073
EDUC4-12790UC BerkeleyTraining the Next Generation of Biologists and Engineers for Regenerative Medicine    $4,954,238
EDUC4-12792UC DavisCIRM Cell and Gene Therapy Training Program 2.0    $4,966,300
EDUC4-12802Children’s Hospital of Los AngelesCIRM Training Program for Stem Cell and Regenerative Medicine Research    $4,999,500
EDUC4-12804UC San DiegoInterdisciplinary Stem Cell Training Grant at UCSD III    $4,992,446
EDUC4-12811ScrippsTraining Scholars in Regenerative Medicine and Stem Cell Research    $4,931,353
EDUC4-12812UC San FranciscoScholars Research Training Program in Regenerative Medicine, Gene Therapy, and Stem Cell Research    $5 million
EDUC4-12813Sanford BurnhamA Multidisciplinary Stem Cell Training Program at Sanford Burnham Prebys Institute, A Critical Component of the La Jolla Mesa Educational Network    $4,915,671  
EDUC4-12821UC Santa BarbaraCIRM Training Program in Stem Cell Biology and Engineering    $1,924,497
EDUC4-12822UC IrvineCIRM Scholars Comprehensive Research Training Program  $5 million
EDUC4-12837Lundquist Institute for Biomedical InnovationStem Cell Training Program at the Lundquist Institute    $4,999,999

These are not the only awards we make to support training the next generation of scientists. We also have our SPARK and Bridges to Stem Cell Research programs. The SPARK awards are for high school students, and the Bridges program for graduate or Master’s level students.

Using a stem cell’s journey to teach kids science

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As far as Aldo Pourchet is concerned you are never too young to learn about stem cells. Aldo should know. He’s a molecular and cellular biologist and the co-founder and CEO of Omios Bio, which develops immunotherapies for cancer, infectious and inflammatory diseases.

Aldo Pourchet

And now Aldo is the author of a children’s book about stem cells. The book is “Nano’s Journey! A Little Stem Cell Visits the Heart and Lungs.” It’s the story of Nano, a stem cell who doesn’t know what kind of cell she wants to be when she grows up, so she goes on a journey through the body, exploring all the different kinds of cell she could be.

It’s a really sweet book, beautifully illustrated, and written in a charming way to engage children between the ages of 5 and 8. I asked Aldo what made him want to write a book like this.

“I was interested in providing very general knowledge such as the principle of life, the basic logics of nature and at the same time to entertain. It was very important for it not to be a textbook.

“Why Stem cells? Because it is the most fascinating biology and they are at the origin of an organism and throughout its life play an essential role. They evolve and transform, so they have a story that unfolds. An analogy with children maybe. It’s easy to imagine children are like stem cells, trying to decide who they are, while adults are like differentiated cells because they have already decided.

“For the kids to appropriate the story, I thought that humanizing cells was important.  I wanted children to identify themselves with the cells and especially Nano, the little girl main character. It’s a book written for the children, in the first place. We tell the story at their level. Not try to bring them up to the level of life science.

Aldo says right from the start he had a clear idea of who he wanted the lead character to be.

“I think the world needs more female leaders, more female voices and influence in general and in every domain. So quite early it became natural for me that Nano would be a girl and also would have a strong character, curious and adventurous.

“Blasto came later because I was looking for a companion to share the adventure with Nano. Blasto is a fibroblast so he is not supposed to leave the Bone Marrow but fibroblasts are everywhere in our organism so I thought it was an acceptable stretch.

The drawings in the book are delightful, colorful and fun. Aldo says he had some ideas, rounded shapes for the cells for example and a simple design that reflected the fact that there are no lines in nature. Illustrator Jen Yoon took it from there:

“Based on Aldo’s direction and imagination, I envisioned the style like drawings on a chalkboard. Soft curves with rough textures. After that everything went smoothly. Following Nano’s journey with my iPad pencil, it felt like a boat ride at an amusement park.”

The books are written to be read aloud by parents, adults and teachers to kids. But, spoiler alert, we don’t find out what cell Nano decides to be in this book. She’s going to have more adventures in other books before she makes up her mind.

Board Funds Fifteen Bridges to Stem Cell Research and Therapy Programs Across California and New Sickle Cell Disease Trial

Yesterday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $8.39 million to the University of California, San Francisco (UCSF) to fund a clinical trial for sickle cell disease (SCD).  An additional $51.08 million was awarded to fifteen community colleges and universities across California to fund undergraduate and master’s level programs that will help train the next generation of stem cell researchers. 

SCD is an inherited blood disorder caused by a single gene mutation that changes a single base in the B globin gene leading to the production of defective hemoglobin that polymerizes and damages red blood cells thus the “sickle” shaped red blood cells.  The damaged cells cause blood vessels to occlude/close up and that can lead to multiple organ damage as well as reduced quality of life and life expectancy. 

Mark Walters, M.D., and his team at UCSF Benioff Children’s Hospital Oakland will be conducting a clinical trial that uses CRISPR-Cas9 gene editing technology to correct the genetic mutation in the blood stem cells of patients with severe SCD.  The corrected blood stem cells will then be reintroduced back into patients with the goal of correcting the defective hemoglobin and thus producing functional, normal shaped red blood cells.

This clinical trial will be eligible for co-funding under the landmark agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI) of the NIH.  The CIRM-NHLBI agreement is intended to co-fund cell and gene therapy programs under the NHLBI’s “Cure Sickle Cell” initiative.  The goal is to markedly accelerate the development of cell and gene therapies for SCD. CIRM has previously funded the preclinical development of this therapy through a Translational award as well as its IND-enabling studies through a Late Stage Preclinical award in partnership with NHLBI.

The CIRM Bridges to Stem Cell Research and Therapy program provides undergraduate and master’s students with the opportunity to take stem cell related courses and receive hands on experience and training in a stem cell research related laboratory at a university or biotechnology company.  Fifteen institutions received a total of $51.08 million to carry out these programs to train the next generation of scientists.

The awards are summarized in the table below.

ApplicationTitleInstitutionAward Amount
  EDUC2-12607Bridges to Stem Cell Research and Therapy at Pasadena City College  Pasadena City College$3,605,500
  EDUC2-12611CIRM Bridges to Stem Cell Research and Therapy Training Grant  CSU San Marcos$3,606,500
  EDUC2-12617Bridges to Stem Cell Research Internship Program  San Diego State University$3,606,500
EDUC2-12620CIRM Bridges 3.0  Humboldt State$3,605,495
  EDUC2-12638CIRM Regenerative Medicine and Stem Cell Research Biotechnology Training Program  CSU Long Beach$3,276,500
    EDUC2-12677Stem Cell Internships in Laboratory-based Learning (SCILL) continue to expand the scientific workforce for stem cells research and therapies.  San Jose State University$3,606,500
  EDUC2-12691Strengthening the Pipeline of Master’s-level Scientific and Laboratory Personnel in Stem Cell Research  CSU Sacramento$2,946,500
EDUC2-12693CIRM Bridges Science Master’s Program  San Francisco State University$3,606,500
      EDUC2-12695CIRM Graduate Student Training in Stem Cell Sciences in the Stem Cell Technology and Lab Management Emphasis of the MS Biotechnology Program  CSU Channel Islands$3,606,500
  EDUC2-12718CSUN CIRM Bridges 3.0 Stem Cell Research & Therapy Training Program  CSU Northridge$3,606,500
      EDUC2-12720Stem Cell Scholars: a workforce development pipeline, educating, training and engaging students from basic research to clinical translation.  CSU San Bernardino$3,606,500
  EDUC2-12726Training Master’s Students to Advance the Regenerative Medicine Field  Cal Poly San Luis Obispo$3,276,500
  EDUC2-12730Building Career Pathways into Stem Cell Research and Therapy Development  City College of San Francisco$2,706,200
      EDUC2-12734Bridges to Stem Cell Research and Therapy: A Talent Development Program for Training Diverse Undergraduates for Careers in Regenerative Medicine  CSU Fullerton$3,606,500
  EDUC2-12738CIRM Bridges to Stem Cell Research and Therapy  Berkeley City College  $2,806,896

“We are pleased to fund a promising trial for sickle cell disease that uses the Nobel Prize winning gene editing technology CRISPR-Cas9,” says Maria T. Millan, M.D., President and CEO of CIRM.  “This clinical trial is a testament to how the CIRM model supports promising early-stage research, accelerates it through translational development, and advances it into the clinics. As the field advances, we must also meet the demand for promising young scientists.  The CIRM Bridges programs across the state of California will provide students with the tools and resources to begin their careers in regenerative medicine.”

We’ve got cash, here’s how you can get some

When the voters of California approved Proposition 14 last November (thanks folks) they gave us $5.5 billion to continue the work we started way back in 2014. It’s a great honor, and a great responsibility.

It’s also a great opportunity to look at what we do and how we do it and try to come up with even better ways of funding groundbreaking research and helping create a new generation of researchers.

In addition to improving on what we already do, Prop 14 introduced some new elements, some new goals for us to add to the mix, and we are in the process of fleshing out how we can best do that.

Because of all these changes we decided it would be a good idea to hold a “Town Hall” meeting and let everyone know what these changes are and how they may impact applications for funding.

The Town Hall, on Tuesday June 29, was a great success with almost 200 participants. But we know that not everyone who wanted to attend could, so here’s the video of the event, and below that are the questions that were posed by people during the meeting, and the answers to those questions.

Having seen the video we would be eternally grateful if you could respond to a short online survey, to help us get a better idea of your research and education needs and to be better able to serve you and identify potential areas of opportunity for CIRM. Here’s a link to that survey: https://www.surveymonkey.com/r/VQMYPDL

We know that there may be issues or questions that are not answered here, so feel free to send those to us at info@cirm.ca.gov and we will make sure you get an answer.

Are there any DISC funding opportunities specific to early-stage investigators?

DISC funding opportunities are open to all investigators.  There aren’t any that are specific to junior investigators.

Are DISC funding opportunities available for early-mid career researchers based out of USA such as Australia?

Sorry, you have to be in California for us to fund your work.

Does tumor immunology/ cancer immunotherapy fall within the scope of the CIRM discovery grants?

Yes, they do.  Here is a link to various CIRM DISC Awards that fall within the cancer category.  https://www.cirm.ca.gov/grants?disease_focus%5B%5D=1427&program_type%5B%5D=1230

Will Disc1 (Inception awards) and/or seed funding mechanisms become available again?

CIRM is anticipating launching a program to meet this need toward the end of this year.

For DISC award is possible to contact a grant advisor for advice before applying?

Please email discovery@cirm.ca.gov to discuss Discovery stage applications before applying

Is co-funding requirement a MUST for clinical trials?

Co-funding requirements vary.  Please refer to the following link for more information: https://www.cirm.ca.gov/sites/default/files/files/about_cirm/CLIN2_Mini_Brochure2.pdf

Hi, when will reviews for DISC 2: CIRM Quest – Discovery Stage Research Projects (deadline March 2021) be available? Thanks!

Review summaries for the March 2021 Discovery submitted applications will be available by mid-August, with final board funding decisions at the August 24th Application Review Subcommittee Meeting

Has CIRM project made it to Phase III or product launch with FDA approval? What is CIRM strategy for start-up biotech companies?

CIRM has funded several late-stage Phase III/potentially pivotal clinical trials. You can view them here: https://www.cirm.ca.gov/our-impact/funding-clinical-trials

CIRM funding supports non-profit academic grantees as well as companies of all sizes.

I am studying stem cells using mouse. Is my research eligible for the CIRM grants?

Yes it is.

Your programs more specifically into stem cell research would be willing to take patients that are not from California?

Yes, we have treated patients who are not in California. Some have come to California for treatment and others have been treated in other states in the US by companies that are based here in California.

Can you elaborate how the preview of the proposals works? Who reviews them and what are the criteria for full review?

The same GWG panel both previews and conducts the full review. The panel first looks through all the applications to identify what each reviewer believes represents the most likely to be impactful and meet the goals of the CIRM Discovery program. Those that are selected by any reviewer moves forward to the next full review step.

If you meet your milestones-How likely is it that a DISC recipient gets a TRAN award?

The milestones are geared toward preparation of the TRAN stage.  However, this is a different application and review that is not guaranteed to result in funding.

Regarding Manufacturing Public Private partnerships – What specific activities is CIRM thinking about enabling these partnerships? For example, are out of state for profit commercial entities able to conduct manufacturing at CA based manufacturing centers even though the clinical program may be primarily based out of CA? If so, what percent of the total program budget must be expended in CA? How will CIRM enable GMP manufacturing centers interact with commercial entities?

We are in the early stages of developing this concept with continued input from various stakeholders. The preliminary vision is to build a network of academic GMP manufacturing centers and industry partners to support the manufacturing needs of CIRM-funded projects in California.

We are in the process of widely distributing a summary of the manufacturing workshop. Here’s a link to it:

If a center is interested in being a sharing lab or competency hub with CIRM, how would they go about it?

CIRM will be soliciting applications for Shared Labs/Competency hubs in potential future RFAs. The survey asks several questions asking for feedback on these concepts so it would really help us if you could complete the survey.

Would preclinical development of stem cell secretome-derived protein therapies for rare neuromuscular diseases and ultimately, age-related muscle wasting be eligible for CIRM TRAN1 funding? The goal is to complete IND-enabling studies for a protein-based therapy that enhances tissue regeneration to treat a rare degenerative disease. the screening to identify the stem-cell secreted proteins to develop as therapeutics is done by in vitro screening with aged/diseased primary human progenitor cells to identify candidates that enhance their differentiation . In vivo the protein therapeutic signals to several cell types , including precursor cells to improve tissue homeostasis.

I would suggest reaching out to our Translation team to discuss the details as it will depend on several factors. You can email the team at translational@cirm.ca.gov

Here are the slides used in the presentations.

Heads or tails? Stem cells help guide the decision

Two cell embryo

There are many unknown elements for what triggers the cells in an embryo to start dividing and multiplying and becoming every single cell in the body. Now researchers at the Gladstone Institutes in San Francisco have uncovered one of those elements, how embryos determine which cells become the head and which the tail.

In this CIRM-funded study the Gladstone team, led by Dr. Todd McDevitt, discovered almost by chance how the cells align in a heads-to-tail arrangement.

Todd McDevitt

They had created an organoid made from brain cells when they noticed that some of the cells were beginning to gather in an elongated fashion, in the same way that spinal cords do in a developing fetus.

In a news article, Nick Elder, a graduate student at Gladstone and the co-author of the study, published in the journal Development, says this was not what they had anticipated would happen: “Organoids don’t typically have head-tail directionality, and we didn’t originally set out to create an elongating organoid, so the fact that we saw this at all was very surprising.”

Further study enabled the team to identify which molecules were involved in signaling specific genes to switch on and off. These were similar to the process previously identified in developing mouse embryos.

“This is such a critical point in the early development of any organism, so having a new model to observe it and study it in the lab is very exciting,” says McDevitt.

This is not just of academic interest either, it could have real world implications in helping understand what causes miscarriages or birth defects.

“We can use this organoid to get at unresolved human developmental questions in a way that doesn’t involve human embryos,” says Dr. Ashley Libby, another member of the team. “For instance, you could add chemicals or toxins that a pregnant woman might be exposed to, and see how they affect the development of the spinal cord.”

Physicians and patient advocates on the front lines of the fight for a more equitable health system

Over the last year there has been increasing awareness of the inequalities in the American healthcare system. At every level there is evidence of bias, discrimination and unequal access to the best care. Sometimes unequal access to any care. That is, hopefully, changing but only if the new awareness is matched with action.

At the recent World Stem Cell Summit CIRM helped pull together a panel of physicians and patient advocates who have been leading the charge for change for years. The panel was called ‘Addressing Disparities, Promoting Equity and Inclusion in Clinical Research.’

The panelists include:

This image has an empty alt attribute; its file name is ysabel-duron.jpeg
Ysabel Duron – Founder of The Latino Cancer Institute & CIRM Board member
Adrienne Shapiro – sickle cell disease patient advocate, Founder of Axis Advocacy – Sickle Cell Disease support and advocacy group
Dr. Leah Ke‘ala‘aumoe Dowsett – Clinical geneticist, serves on hospital DEI committee, Board member Association of Native Hawaiian Physicians
Dr. Nathan Chomilo – Co-Founder, Minnesota Doctors for Health Equity and head of the Minnesota COVID Vaccine Equity Program

The conversation they had was informative, illuminating and fascinating. But it didn’t sugar coat where we are, and the hard work ahead of us to get to where we need to be.

Enjoy the event, with apologies for the inept cameo appearance by me at the beginning of the video. Technology clearly isn’t my forte.

Women who have changed, and are changing, the world

The problem with trying to write about something like Women’s History Month is where do you start? Even if you narrow it down to women in science the list is vast.

Marie Curie

I suppose you could always start with Maria Salomea Skłodowska who is better known as Marie Curie. She not only discovered radium and polonium, but she was also the first woman to win a Nobel Prize (in Physics). When she later won another Nobel (in Chemistry) she became the first person ever to win two Nobels and is still the only person ever to win in two different fields. Not a bad place to start.

Agnes Pockels

Or how about Agnes Pockels (1862–1935). Even as a child Agnes was fascinated by science but, in Germany at the time, women were not allowed to attend university. So, she depended on her younger brother to send her his physics textbooks when he was finished with them. Agnes studied at home while taking care of her elderly parents. Doing the dishes  Agnes noticed how oils and soaps could impact the surface tension of water. So, she invented a method of measuring that surface tension. She wrote a paper about her findings that was published in Nature, and went on to become a highly respected and honored pioneer in the field.

Jennifer Doudna (left) and Emmanuelle Charpentier: Photo courtesy Nature

Fast forward to today we could certainly do worse than profile the two women who won the 2020 Nobel Prize in Chemistry for their work with the gene-editing tool CRISPR-Cas9; Jennifer Doudna at the University of California, Berkeley, and Emmanuelle Charpentier at the Max Planck Unit for the Science of Pathogens in Berlin. Their pioneering work showed how you could use CRISPR  to make precise edits in genes, creating the possibility of using it to edit human genes to eliminate or cure diseases. In fact, some CIRM-funded research is already using this approach to try and cure sickle cell disease.

In awarding the Nobel to Charpentier and Doudna, Pernilla Wittung Stafshede, a biophysical chemist and member of the Nobel chemistry committee, said: “The ability to cut DNA where you want has revolutionized the life sciences. The ‘genetic scissors’ were discovered just eight years ago but have already benefited humankind greatly.”

Barbara McClintock: Photo courtesy Brittanica

Appropriately enough none of that work would have been possible without the pioneering work of another woman, Barbara McClintock. She dedicated her career to studying the genetics of corn and developed a technique that enabled her to identify individual chromosomes in different strains of corn.

At the time it was thought that genes were stable and were arranged in a linear fashion on chromosomes, like beads on a string. McClintock’s work showed that genes could be mobile, changing position and altering the work of other genes. It took a long time before the scientific world caught up with her and realized she was right. But in 1983 she was awarded the Nobel Prize in Medicine for her work.

Katherine Johnson at her desk at Langley Research Center: Photo courtesy NASA /AFP

Katherine Johnson is another brilliant mind whose recognition came later in life. But when it did, it made her a movie star. Kind of. Johnson was a mathematician, a “computer” in the parlance of the time. She did calculations by hand, enabling NASA to safely launch and recover astronauts in the early years of the space race.

Johnson and the other Black “computers” were segregated from their white colleagues until the last 1950’s, when signs dictating which restrooms and drinking fountains they could use were removed. She was so highly regarded that when John Glenn was preparing for the flight that would make him the first American to orbit the earth he asked for her to manually check the calculations a computer had made. He trusted her far more than any machine.

Johnson and her co-workers were overlooked until the 2016 movie “Hidden Figures” brought their story to life. She was also awarded the Presidential Medal of Freedom, America’s highest civilian honor, by President Obama.

There are so many extraordinary women scientists we could talk about who have made history. But we should also remind ourselves that we are surrounded by remarkable women right now, women who are making history in their own way, even if we don’t recognized it at the moment. Researchers that CIRM funds, Dr. Catriona Jamieson at UC San Diego, Dr. Jan Nolta at UC Davis, Dr. Jane Lebkowski with Regenerative Patch technologies and so many others. They’re all helping to change the world. We just don’t know it yet.

If you would like to learn about other women who have made extraordinary contributions to science you can read about them here and here and here.