Join Us on Stem Cell Awareness Day (October 12)!

In 2004, the California Institute for Regenerative Medicine (CIRM) was created by the people of California to accelerate stem cell treatments to patients with unmet medical needs.  
 
Since then, we’ve expanded our mission to accelerate world class science for California and the world. We’ve funded and supported a pipeline of medical research from initial scientific discovery to development and testing. We also remain committed to training the next generation of regenerative medicine scientists to research cutting edge therapies for patients.  
 
We’ve achieved a lot but recognize there’s still lots more work to be done. That’s why we’re inviting everyone to join us for a virtual webinar on October 12th for Stem Cell Awareness Day, a day when we mark the progress being made in regenerative medicine, stem cell and gene therapy research. 
 
This Stem Cell Awareness Day, the CIRM team will highlight our achievements in research, clinical trials and education. We will also look ahead to explore how we can best further our mission. 
 
Speakers will include: 
Jonathan Thomas — Chair, CIRM Board  
Dr. Kelly Shepard — Associate Director, Scientific Programs 
Dr. Lisa Kadyk — Associate Director, Therapeutics Development 
Dr. Mitra Hooshmand — Sr. Science Officer, Special Projects & Strategic Initiatives 
 
The event is free and you can register here. If you have a question you would like to ask the team, please email them to info@cirm.ca.gov ahead of time. We will do our best to answer all questions during the webinar and those we can’t get to we’ll answer The Stem Cellar.  
 
We look forward to seeing you there! 

A newfound passion for stem cell research

All her life, Madison Waterlander knew that she wanted to be a part of the medical field. But soon after graduating from the University of Hawaii with her undergraduate degree, the COVID-19 pandemic hit. It was during this time that she noticed how crucial biomedical research was in the medical field and lives of patients, and when she realized she had a passion for research.  

She soon after found a master’s program in biotechnology and bioinformatics at California State University Channel Islands (CI), just a few minutes from Camarillo, the town she grew up in.  

Looking further into the program, she learned that to pursue a Stem Cell Technology and Laboratory Management emphasis for the degree, she would have to complete a one-year lab internship funded by the California Institute for Regenerative Medicine (CIRM). The internship was part of CIRM’s Bridges to Stem Cell Research and Therapy Program, which prepares California undergraduate and master’s graduate students for highly productive careers in stem cell research and therapy development.  

The opportunity to have hands-on experience in a lab through the internship solidified her decision to join the graduate program. 

Once she settled into the program at CSU Channel Islands, she began her internship, which took place at UC Santa Barbara in the Weimbs Lab. While there, she researched the underlying mechanisms and possible new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder characterized by the growth of numerous cysts in the kidneys.  

“This CIRM-funded internship was so enriching for me, and I was able to expand my knowledge and skill set immensely in the laboratory,” Madison says. “I always knew that I loved science and the medical field, but this experience truly helped me realize that my strongest passion resides in the scientific research that goes into improving the quality of patient care and treatments.” 

While Madison says the internship supported her knowledge in the lab and was an overall positive experience, she also faced some personal challenges during that time, including losing her grandma. She struggled with the loss, but Madison says her time in the lab allowed her to focus on something she loved doing and that her grandma always encouraged her to do. 

“My grandma never would have wanted me to give up, so that truly helped to push me to continue on, and to try my hardest in every day to make an impact,” Madison says. 

After a year of hard work in the lab, Madison officially graduated from CSU Channel Islands this summer with a Master of Science Degree in Biotechnology and Bioinformatics with a Stem Cell Technology and Laboratory Management emphasis. Now, Madison is pursuing a role in the biotechnology industry within translational biomedical research.

“I truly enjoyed every moment of my CIRM internship, and I feel that it truly revealed to me just how much I enjoy participating in biomedical research,” Madison says. “I’ve always felt that research feels like a treasure hunt looking for cures and treatments, so the more of us that are partaking in the treasure hunt, the quicker we can find new treatments and provide solutions for patients.” 

Stories like Madison’s are why CIRM remains committed to training the next generation of scientists to conduct research and deliver regenerative medicine and stem cell therapies to patients. To date, there are 1,663 Bridges alumni, and another 109 Bridges trainees—including Madison—who are completing their internships in 2022.   

CIRM-funded stem cell-gene therapy shows promise in ALS safety trial

Senior author of the study Clive Svendsen, PhD (center)

With funding support from the California Institute for Regenerative Medicine (CIRM), Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Lou Gehrig’s disease. 

In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans. The findings were reported in the peer-reviewed journal Nature Medicine

What causes ALS? 

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. About 6,000 people are diagnosed with ALS each year in the U.S., and the average survival time is two to five years.  

The disease results when the cells in the brain or spinal cord that instruct muscles to move—called motor neurons—die off. People with the disease lose the ability to move their muscles and, over time, the muscles atrophy and people become paralyzed and eventually die. There is no effective therapy for the disease. 

Using Stem Cells to Treat ALS 

In a news release, senior author Clive Svendsen, PhD, executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, says using stem cells shows lots of promise in treating patients with ALS.  

“We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS,” Svendsen says.  

Aimed at preserving leg function in patients with ALS, the engineered cells could pave the way to a therapeutic option for this disease that causes progressive muscle paralysis, robbing people of their ability to move, speak and breathe.   

The study used stem cells originally designed in Svendsen’s laboratory to produce a protein called glial cell line-derived neurotrophic factor (GDNF). This protein can promote the survival of motor neurons, which are the cells that pass signals from the brain or spinal cord to a muscle to enable movement.  

In patients with ALS, diseased glial cells can become less supportive of motor neurons, and these motor neurons progressively degenerate, causing paralysis.   

By transplanting the engineered protein-producing stem cells in the central nervous system, where the compromised motor neurons are located, these stem cells can turn into new supportive glial cells and release the protective protein GDNF, which together helps the motor neurons stay alive.   

Ensuring Safety in the Trial 

The primary goal of the trial was to ensure that delivering the cells releasing GDNF to the spinal cord did not have any safety issues or negative effects on leg function.   

In this trial, none of the 18 patients treated with the therapy—developed by Cedars-Sinai scientists and funded by CIRM—had serious side effects after the transplantation, according to the data. 

Because patients with ALS usually lose strength in both legs at a similar rate, investigators transplanted the stem cell-gene product into only one side of the spinal cord so that the therapeutic effect on the treated leg could be directly compared to the untreated leg.  

After the transplantation, patients were followed for a year so the team could measure the strength in the treated and untreated legs. The goal of the trial was to test for safety, which was confirmed, as there was no negative effect of the cell transplant on muscle strength in the treated leg compared to the untreated leg.    

What’s Next? 

Investigators expect to start a new study with more patients soon. They will be targeting lower in the spinal cord and enrolling patients at an earlier stage of the disease to increase the chances of seeing effects of the cells on the progression of ALS. 

“We are very grateful to all the participants in the study,” said Svendsen. “ALS is a very tough disease to treat, and this research gives us hope that we are getting closer to finding ways to slow down this disease.”   

The Cedars-Sinai team is also using the GDNF-secreting stem cells in another CIRM-funded clinical trial for ALS, transplanting the cells into a specific brain region, called the motor cortex that controls the initiation of movement in the hand. The clinical trial is also funded by CIRM. 

The California Institute for Regenerative Medicine (CIRM) remains committed to funding research and clinical trials to treat ALS. To date, CIRM has provided $93 million in funding for research to treat ALS.  

Read the original source release of the study here.  

CIRM Bridges intern researches stem cells to grow kidneys 

David Anjakos in the lab. Photo courtesy Sarah White/SDSU.

When he was younger, David Anjakos experienced kidney failure due to an autoimmune disease, leaving him without kidneys in his body. As a trainee in the California Institute for Regenerative Medicine’s Bridges to Stem Cell Research Internship Program, Anjakos is researching methods of growing organs for transplantation to help people on a transplant list, himself included. 

By now, Anjakos thought he’d have his own kidney and that he would be off the transplant list and dialysis. That’s not the case, so he realized he wanted to try and do something about it.  

“Fifteen years later, we haven’t really gotten there. It just shows how complex the problem is and how even with thousands of hours and scientists working on this, we still haven’t quite got there,” he says. “What that showed me is that I needed to step in. We need more people on these problems.” 

David Anjakos in the lab. Photo courtesy Sarah White/SDSU.

That’s what inspired him to join the CIRM Bridges Program at San Diego State University. Specifically, he wanted to get into stem cells to try to control them to do what he wanted them to do. He’s completing his internship at the Sanford Consortium for Regenerative Medicine, where he is working toward developing a protein that will be able to activate stem cells to turn into different organs. 

If successful, this will be important for drug discovery, growing organs and vascularization, the process of growing blood vessels into a tissue to improve oxygen and nutrient supply. 

“CIRM’s Bridges to Stem Cell Research program has really been a huge opportunity for me to get into science, to practice science, to practice the skills that I’ll need,” said Anjakos. “It has really helped me in my confidence in my ability to do science.” 

After finishing his Bridges internship at the Sanford Consortium, Anjakos plans to start a PhD program so he can apply all he has learned from creating approximations of the Wnt protein that is essential for turning stem cells into organs with functioning vessels.  

To date, there are 1,663 Bridges alumni, and another 109 Bridges trainees are completing their internships in 2022. 

Started in 2009, the Bridges program provides paid stem cell research internships to students at universities and colleges that don’t have major stem cell research programs. Each Bridges internship includes thorough hands-on training and education in regenerative medicine and stem cell research, and direct patient engagement and outreach activities that engage California’s diverse communities. Click here to learn more about CIRM’s educational programs.  


This story was first covered by Sarah White and Susanne Clara Bard. Read the original release on the San Diego State University website.  

 

How this scientist uses Legos to explain the power of stem cells 

Explaining science is hard. Explaining stem cells, which have their very own unique complexities, can be even more of a challenge, especially when communicating with a non-scientific audience.  

That’s why when we received this blog submission from a CIRM SPARK Program intern through UCSF’s High School Intern Program (HIP) explaining stem cells in a simple, straightforward way using Legos, we knew we had to share it with our readers.

Before we share the intern’s brilliant explanation of stem cells, here’s how the California Institute for Regenerative Medicine (CIRM) defines stem cells. These and other key terms can be found on our website

The first thing to know about stem cells is that there is not just one kind. In fact, there are many different types of stem cells, each with very different potential to treat disease. There are various types of stem cells, including pluripotent, embryonic, adult, and iPSC (induced pluripotent stem cell).  

Stem cells also have the potential to become other kinds of cells in the body. For example, embryonic stem cells can become many other kinds of cells, whereas adult stem cells, such as in fat, can only become bone or cartilage. 

Now, the fun part! Here’s what the student shared in their prize-winning SPARK Program blog submission.


If someone were to ask me what stem cells are in a simple and perhaps figurative way now, I would say that stem cells are just like Legos. Legos are special building-blocks that are in a blank or default-like state, but can be something greater and unique on its own later on.  

Similarly, stem cells are called “unspecialized cells” because they are yet to be “specialized” or become a certain type of cell. They can be a blood, brain, heart, and basically all types of cells respectively, with little to no exceptions. Moreover, not all Legos are built the same. Some can be regular block-shaped, while some can be circular or even triangular. Therefore, this limits Legos’ abilities to a certain degree. Similarly, not all stem cells are necessarily the same. 

With just the right amount and type of Legos, you can easily assemble and build a house, a car, or whatever you could possibly think about. Similarly, the possibilities are endless with stem cells as well, which is why it’s truly a promising and key aspect in regenerative medicine today. 


Bravo! In addition to creating a unique way of explaining stem cells during their internship, the student also learned how to differentiate the different types and sources of stem cells from one another through hands-on experience at a world-renowned institution.  

The student added, “My newly-found interest in regenerative medicine and stem cells is definitely something that I’m looking forward to with great passion and knowledge moving forward.” 

To learn more about CIRM’s internship programs, visit our website. To read another prize-winning blog submission from a SPARK intern, click here.

How one student overcame uncertainty in CIRM’s high school internship program

This summer—as well as year round—the California Institute for Regenerative Medicine (CIRM) will highlight blog submissions, photos, and other fun content from our SPARK Program.

SPARK—also known as the Summer Program to Accelerate Regenerative Medicine Knowledge—gives high school students a chance to spend their summer working in a world class stem cell research facility here in California. At the end of their internship, they write about their experiences and what they learned. 

As always, we received many wonderful submissions from the students, so choosing a winner was particularly tough. In the end we chose two winners. The first blog—which you can read below—was submitted by Saranya Anandakumar, who interned at Sanford Burnham Prebys in La Jolla.  

The second winning submission was authored by John Casilao, who interned at UCSF. The blog will also be shared on The Stem Cellar this week.  

Check out Saranya’s winning blog submission below and be sure to follow the blog for more updates from CIRM’s SPARK Program.


Saranya in the lab at Sanford Burnham Prebys

Certainty?

Submitted by Saranya Anandakumar 
2022 SPARK Program Intern at Sanford Burnham Prebys 

My whole life I’ve been indecisive, which I think is fair. I mean, think about it. If the multiverse theory is coupled with the butterfly effect then the difference between a vanilla and strawberry ice cream could mean the difference between my continued life or my sudden death.  

So if I can’t even choose which cereal to eat in the morning, what in the world makes my counselor think I can decide what career I want to pursue in the future? What’s worse is while an ice cream cone only lasts about ten or fifteen minutes in the beaming hot summer Sun before wasting away, my occupational choice, my major, and my degree, they’ll stay with me forever if not for at least ten or so years. So why not give a six-week free trial a shot? That way of thinking, dear reader, is precisely how I ended up in CIRM’s SPARK Program.  

Hi, my name is Saranya Anandakumar. I love oceanography and glaciology as much as I love law and psychiatry, but the medical field has always had a special place in my heart. That’s how I took the first step of applying for this internship.  

Saranya poses with her research poster at the SPARK conference in Oakland

To give a brief summary, my grandma had a stroke and pneumonia and has been bedridden since because a doctor gave her the incorrect dose of medication (according to medical staff she should have been dead). My mom has struggled with chronic pain, migraines, memory issues, and high blood pressure for years, and my entire family struggles with severe anxiety and depression (plus the occasional eating disorder).  

Of course, I have a breathing disorder and struggle with migraines. I also have an extremely weak immune system to the point where I’m ill eight months of the year. Needless to say, emergency room and ICU visits have become the norm, so much that the nurses and I are on a first-name basis. 

But, enough about my glamorous history, and more about my experience with the internship. As I established earlier, I am certain about nothing in my life, which is why it should speak volumes to you when I say I now know that I want to major in immunology and minor in neuroscience.  

Saranya (right) at the campus Intern Celebration. Photo courtesy of Sanford Burnham Prebys.

Still, a little voice inside my head cries, “Oh, what if this is the best lab you’ll ever be in?! What if you think the field is better than it really will be?!” A valid concern. I’m sure Dr. Blaho is the only former Trombone player and drum major I will ever work under, and she’s certainly the funniest. I doubt anyone will have the dad jokes Josh does or the endearing laugh and interesting input Yosiris does.  

It felt like falling in love and the best part was it didn’t matter what it was as long as I was learning something new.  

Saranya Anandakumar

However, my time at Sanford Burnham Prebys still left me with more than that. I loved walking in every day. Every lecture on oligodendrocytes and microglia had me entranced. Every western blot or stem cell experiment left me all giddy inside. Don’t even get me started on looking at human blood under a microscope! It felt like falling in love and the best part was it didn’t matter what it was as long as I was learning something new.  

I was always scared of life after college, but I thought it was because I was scared of committing to one career for the rest of my life. And though that was certainly part of it, I think I was afraid of living my life without learning anymore. So bio research is perfect for me because it’s impossible to not learn even if you try. 


To see photos of this year’s SPARK conference in Oakland, click here. To learn more about CIRM’s education programs, visit our website.  

[PHOTOS] California high school students celebrate regenerative medicine science at CIRM’s annual SPARK conference

We had a wonderful time meeting so many energetic and enthusiastic high school students at the 2022 SPARK Program annual conference hosted by UCSF at the MLK Research Building. The SPARK program is one of the California Institute for Regenerative Medicine’s (CIRM) many programs dedicated to building a diverse and highly-skilled workforce to support the growing regenerative medicine economy right here in California. 

Held in-person for the first time since 2019, the event hosted students and program directors from all over California, allowing them the opportunity to share their research through oral and poster presentations. This year, students also attended talks about new approaches to sickle cell disease curative therapies, anti-racism in STEM, and patient advocacy.    

The SPARK Program—also known as the Summer Program to Accelerate Regenerative Medicine Knowledge—provides California high school students with summer research internships at leading stem cell institutes in California. To date, there have been 530 SPARK alumni, and another 110 high school interns are completing their training this summer.  

The SPARK program specifically selects students who represent the diversity of California’s population, particularly those who might not otherwise have opportunities to take part in research internships due to socioeconomic constraints.  

“I really enjoyed being a part of this program, and I feel like I understand so much better what it’s like to be a researcher,” said Brighton C., a student in the SPARK program at Charles R. Drew University of Medicine and Science (pictured below). “I also feel more confident in the subject of stem cells and I might want to dedicate my future to it.” 

We’ll be sharing more stories from CIRM’s SPARK Program throughout the year, including blog submissions from students that summarize their summer experiences. Stay tuned for more and be sure to follow CIRM on Instagram, where we will share more photos and fun content created by the students. 

There are currently 11 active SPARK programs throughout California, each with its own eligibility criteria and application process. If you are interested in learning more, please visit this web page for more details about each program. If you have questions about CIRM’s education programs, please email Dr. Kelly Shepard at education@cirm.ca.gov. 

Thank you to UCSF for hosting the event, and to all the SPARK program directors for supporting this year’s bright interns!

Check out some of the photos from this year’s SPARK conference below. 

[PHOTOS] CIRM’s Bridges to Stem Cell Research Program hosts annual conference in sunny San Diego

For more than a decade, the California Institute for Regenerative Medicine (CIRM) has funded educational and research training programs to give students the opportunity to explore regenerative medicine and stem cell science right here in California.   

This summer, the CIRM team was thrilled to meet the bright scientists taking part in this year’s Bridges to Stem Cell Research Program, which culminated at the 2022 Bridges Trainee Meeting in sunny San Diego.  

Started in 2009, the Bridges program provides paid stem cell research internships to students at universities and colleges that don’t have major stem cell research programs. Each Bridges internship includes thorough hands-on training and education in regenerative medicine and stem cell research, and direct patient engagement and outreach activities that engage California’s diverse communities. 

To date, there are 1,663 Bridges alumni, and another 109 Bridges trainees are completing their internships in 2022. 

In addition to networking with other scientists across the state, the annual Bridges Trainee Meeting provides students the opportunity to share their research in poster presentations and to learn about careers in the regenerative medicine field. This year, students also attended talks about cutting edge science research, anti-racism in STEM, science communication through social media, and patient advocacy.  

“As the field advances, we must also meet the demand for promising young scientists,” says Maria T. Millan, M.D., President and CEO of CIRM (pictured below). “The CIRM Bridges programs across the state of California will provide students with the tools and resources to begin their careers in regenerative medicine.” 

There are currently 15 active Bridges programs throughout California, each with its own eligibility criteria and application process. If you are interested in applying, please visit this web page for more details about each program. If you have questions about the Bridges program, please email the CIRM Bridges director, Dr. Kelly Shepard at education@cirm.ca.gov. 

Finally, a sincere thank you goes to the Bridges Program from California State University, San Marcos for hosting this year’s CIRM Bridges Trainee Meeting! 

Check out some of the photos from this year’s conference below.

Study reveals new evidence of key mechanism in Alzheimer’s

In California, 690,000 people aged 65 and older are living with Alzheimer’s, a degenerative brain disease and the most common form of dementia. In the United States, 5.8 million people aged 65 and older live with Alzheimer’s disease. Alzheimer’s affects memory, thinking and behavior and symptoms eventually grow in severity to interfere with daily tasks.  

There is no cure for Alzheimer’s, which is why Rutgers scientists are examining human brain cells in mice to identify a pivotal mechanism that could result in a potential therapy for the disease. In a recent study, the Rutgers team found more clear-cut evidence of how the destructive proteins linked to Alzheimer’s disease attack human brain cells and destroy surrounding tissue. 

The researchers studied human brain immune cells injected into the brains of specially bred immunodeficient mice, creating what they called a human-mouse chimera. The researchers detailed what happened to specialized immune brain cells known as microglia after those cells were exposed to tau proteins—destructive substances believed to be involved in Alzheimer’s and other severe human brain diseases. 

“This provided an unprecedented opportunity to investigate the role of human microglia in brains as well as the cognitive impairment seen in Alzheimer’s Disease and Down syndrome, a genetic disorder with a high risk of developing Alzheimer’s disease,” said Peng Jiang, an associate professor in the Department of Cell Biology and Neuroscience at the Rutgers School of Arts and Sciences. 

By studying the process in the newly-developed brain—which allowed human cells to grow, develop and mature with appropriate functions—the scientists were able to witness and analyze a cellular brain attack that has been largely elusive up to this point. 

In autopsies, scientists have been able to study the brains of people who died from Alzheimer’s and have seen residues of tau proteins and cellular changes. The human-mouse brain chimera has allowed the Rutgers team to extract and see human cells in the actual process of deterioration. 

The mice in the study were specially bred to be immunodeficient so that they could receive implanted human cells without rejecting them due to normal immune defenses.  The immunodeficient mice were injected with human microglial cells and, later, with tau proteins, which are linked to the development of the brain disease. 

“Since microglial cells are one of the first cell responders when something goes wrong in the brain, we believe the changes we saw to be significant,” said Mengmeng Jin, a postdoctoral researcher in the Department of Cell Biology and Neuroscience at Rutgers and first author on the study. 

The California Institute for Regenerative Medicine (CIRM) is committed to investing at least $1.5 billion—more than double what CIRM funded between 2006 and 2020—in treatments that target conditions affecting the brain and central nervous system (CNS), including Alzheimer’s. 

Read the source release about the study here.  

Reminder! Apply now for discovery stage stem cell and gene therapy research funding

The California Institute for Regenerative Medicine (CIRM) is seeking applications for its next round of Quest Awards (DISC2) for discovery stage research.

Applications are due August 2nd, 2022, at 2:00 PM PDT. Please visit the CIRM website for full details.  

The purpose of the Quest Awards is to promote the discovery of promising new stem cell-based or gene therapy technologies that could be translated to enable broad use and ultimately, improve patient care.

Applications should propose technology that is uniquely enabled by human stem/progenitor cells or directly reprogrammed cells, or that is uniquely enabling for the advancement of stem cell-based therapies or aimed at developing a genetic therapy approach.

The expected outcome, at the end of the award, is a candidate therapeutic or technology that can immediately progress to translational stage activities. For projects that culminate in a candidate that is a diagnostic, medical device or tool, the proposed project period must not exceed 2 years and direct project costs can be up to $500,000 per award. For projects that culminate in a candidate that is a therapeutic, an applicant may request up to $1,500,000 in direct project costs for up to 3 years duration.

Important Update: Please note that the DISC2 Program Announcement has been updated since the last round of applications. Please read the new program announcement on the CIRM funding website before submitting your application.

To receive updates about future funding opportunities through CIRM, please visit our e-mail newsletter page to sign up.