Investing in a stem cell treatment for Hurler syndrome

The California Institute for Regenerative Medicine (CIRM) awarded $5,444,353 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This is an inherited condition caused by a faulty gene.

Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. As a result, undigested sugar molecules build up in the body, causing progressive damage to the brain, heart, and other organs.

There are no signs or symptoms of the condition at birth, although some have a soft out-pouching around the belly-button or lower abdomen. Those with severe MPS I generally begin to show other signs and symptoms of the disorder within the first year of life. There is no effective treatment and life expectancy for many of these children is only around ten years.

Dr. Gomez-Ospina will use the patient’s own blood stem cells that have been genetically edited to restore the missing enzyme. The goal of this preclinical program is to show the team can manufacture the needed cells, to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.

“The funding will pave the way for trials in people to realize a more effective therapy for this devastating disease,” Gomez-Ospina said. “We will also generate safety and toxicity data that could facilitate the application of our genome editing platform to other genetic disorders for which a significant unmet need still exists.”

Making transplants easier for kids, and charting a new approach to fighting solid tumors.

Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but they also increase the risk of infection, gastrointestinal disease, pancreatitis and cancer.

Dr. Alice Bertaina and her team at Stanford University were awarded $11,998,188 to test an approach that uses combined blood stem cell (HSC) and kidney transplantation with the goal to improve outcomes with kidney transplantation in children. This approach seeks to improve on the blood stem cell preparation through an immune-based purification process.

In this approach, the donor HSC are transplanted into the patient in order to prepare for the acceptance of the donor kidney once transplanted. Donor HSC give rise to cells and conditions that re-train the immune system to accept the kidney. This creates a “tolerance” to the transplanted kidney providing the opportunity to avoid long-term need for medications that suppress the immune system.

Pre-clinical data support the idea that this approach could enable the patient to stop taking any immunosuppression medications within 90 days of the surgery.

Dr. Maria T. Millan, President and CEO of CIRM, a former pediatric transplant surgeon and tolerance researcher states that “developing a way to ensure long-term success of organ transplantation by averting immune rejection while avoiding the side-effects of life-long immunosuppression medications would greatly benefit these children.”

The CIRM Board also awarded $7,141,843 to Dr. Ivan King and Tachyon Therapeutics, Inc to test a drug showing promise in blocking the proliferation of cancer stem cells in solid tumors such as colorectal and gastrointestinal cancer.

Patients with late-stage colorectal cancer are typically given chemotherapy to help stop or slow down the progression of the disease. However, even with this intervention survival rates are low, usually not more than two years.

Tachyon’s medication, called TACH101, is intended to target colorectal cancer (CRC) stem cells as well as the bulk tumor by blocking an enzyme called KDM4, which cancer stem cells need to grow and proliferate.

In the first phase of this trial Dr. King and his team will recruit patients with advanced or metastatic solid tumors to assess the safety of TACH101, and determine what is the safest maximum dose. In the second phase of the trial, patients with gastrointestinal tumors and colorectal cancer will be treated using the dose determined in the first phase, to determine how well the tumors respond to treatment.  

The CIRM Board also awarded $5,999,919 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This is an inherited condition caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. As a result, undigested sugar molecules build up in the body, causing progressive damage to the brain, heart, and other organs. There is no effective treatment and life expectancy for many of these children is only around ten years.

Dr. Gomez-Ospina will use the patient’s own blood stem cells that have been genetically edited to restore the missing enzyme. The goal of this preclinical program is to show the team can manufacture the needed cells, to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.

Finally the Board awarded $20,401,260 to five programs as part of its Translational program. The goal of the Translational program is to support promising stem cell-based or gene projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Those can include therapeutic candidates, diagnostic methods  or devices and novel tools that address critical bottlenecks in research.

The successful applicants are:

APPLICATIONTITLEPRINCIPAL INVESTIGATOR – INSTITUTIONAMOUNT  
TRAN4-14124Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug DiscoveryNikesh Kotecha — Greenstone Biosciences  $1,350,000
TRAN1-14003Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1αJianhua Yu — Beckman Research Institute of City of Hope  $6,036,002  
TRAN1-13983CRISPR/Cas9-mediated gene editing of Hematopoietic
stem and progenitor cells for Friedreich’s ataxia
Stephanie Cherqui — University of California, San Diego  $4,846,579
TRAN1-13997Development of a Gene Therapy for the Treatment of
Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting
Allyson Berent — Mahzi Therapeutics  $4,000,000
TRAN1-13996Overcoming resistance to standard CD19-targeted CAR
T using a novel triple antigen targeted vector
William J Murphy — University of California, Davis  $4,168,679

Study could pave the way in reducing decline in muscle strength as people age 

A study by Stanford Medicine researchers in older mice may lead to treatments that help seniors regain muscle strength lost to aging.

Muscle stem cells—which are activated in response to muscle injury to regenerate damaged muscle tissue—lose their potency with age. A study from the National Health and Nutrition Examination Survey showed that five percent of adults aged 60 and over had weak muscle strength, and thirteen percent had intermediate muscle strength. 

Now, researchers at Stanford Medicine are seeing that old mice regain the leg muscle strength of younger animals after receiving an antibody treatment that targets a pathway mediated by a molecule called CD47.  

The study was published in Cell Stem Cell and is co-funded by the California Institute for Regenerative Medicine (CIRM).  

A Closer Look at CD47 

CD47 is a protein found on the surface of many cells in the body. Billed as the “don’t eat me” molecule, it is better known as a target for cancer immunotherapy. It’s common on the surface of many cancer cells and protects them from immune cells that patrol the body looking for dysfunctional or abnormal cells.  

Stanford researchers are finding that old muscle stem cells may use a similar approach to avoid being targeted by the immune system. 

It’s been difficult to determine why muscle stem cells lose their ability to divide rapidly in response to injury or exercise as they age. Dr. Ermelinda Porpiglia, the lead author of the study, used a technique called “single-cell mass cytometry” to study mouse muscle stem cells.  

Using the technique, Porpiglia focused on CD47, and found that the molecule was found at high levels on the surface of some muscle stem cells in older mice, but at lower levels in younger animals. Porpiglia also found that high levels of CD47 on the surface of muscle stem cells correlate with a decrease in their function.   

“This finding was unexpected because we primarily think of CD47 as an immune regulator,” Porpiglia said. “But it makes sense that, much like cancer cells, aged stem cells might be using CD47 to escape the immune system.” 

Testing an Antibody 

Further investigation revealed that a molecule called thrombospondin, which binds to CD47 on the surface of the muscle stem cells, suppresses the muscle stem cells’ activity.  

Porpiglia showed that an antibody that recognizes thrombospondin and blocks its ability to bind to CD47 dramatically affected the function of muscle stem cells. Cells from older animals divided more robustly when growing in a laboratory dish in the presence of the antibody, and when the antibody was injected into the leg muscles of old mice the animals developed bigger and stronger leg muscles than control animals.  

When given prior to injury, the antibody helped the aged animals recover in ways similar to younger mice. 

Porpiglia said, “We are hopeful that it might one day be possible to inject an antibody to thrombospondin at specific sites in the body to regenerate muscle in older people or to counteract functional problems due to disease or surgery.” 

These results are significant because they could one day make it possible to boost muscle recovery in humans after surgery and reduce the decline in muscle strength as people age, but researchers say more work is needed.  

“Rejuvenating the muscle stem cell population in older mice led to a significant increase in strength,” said Dr. Helen Blau, a senior author of the study. “This is a localized treatment that could be useful in many clinical settings, although more work needs to be done to determine whether this approach will be safe and effective in humans.” 

CIRM has previously funded work with researchers using CD47 that led to clinical trials targeting cancer. You can read about that work here and here. That work led to the creation of a company, Forty Seven Inc, which was eventually bought by Gilead for $4.9 billion.  

Read the original release by Krista Conger on the Stanford Medicine website. 

Sweet 16 and counting for stem cell clinical trial

Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics

Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children.

Now there’s more encouraging news from a CIRM-funded clinical trial with Jasper Therapeutics. They have announced that they have tested their approach in 16 patients, with encouraging results and no serious adverse events.

Let’s back up a little. Children born with SCID have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. Several of the approaches CIRM has funded use the child’s own blood stem cells to help fix the problem. But at Jasper Therapeutics they are using another approach. They use a bone marrow or hematopoietic stem cell transplant (HCT).   This replaces the child’s own blood supply with one that is free of the SCID mutation, which helps restore their immune system.

However, there’s a problem. Most bone marrow transplants use chemotherapy or radiation to destroy the patient’s own unhealthy blood stem cells and make room for the new, healthy ones. It can be effective, but it is also toxic and complex and can only be performed by specialized teams in major medical centers, making access particularly difficult for poor and underserved communities.

To get around that problem Jasper Therapeutics is using an antibody called JSP191 – developed with CIRM funding – that directs the patient’s own immune cells to kill diseased blood stem cells, creating room to transplant new, healthy cells. To date the therapy has already been tested in 16 SCID patients.

In addition to treating 16 patients treated without any apparent problems,  Jasper has also been granted Fast Track Designation by the US Food and Drug Administration. This can help speed up the review of treatments that target serious unmet conditions. They’ve also been granted both Orphan and Rare Pediatric Disease designations. Orphan drug designation qualifies sponsors for incentives such as tax credits for clinical trials. Rare Pediatric Disease designation means that if the FDA does eventually approve JSP191, then Jasper can apply to receive a priority review of an application to use the product for a different disease, such as someone who is getting a bone marrow transplant for sickle cell disease or severe auto immune diseases.

In a news release, Ronald Martell, President and CEO of Jasper Therapeutics said:

“The FDA’s Fast Track designation granted for JSP191 in Severe Combined Immunodeficiency (SCID) reinforces the large unmet medical need for patients with this serious disease. Along with its previous designations of Orphan and Rare Pediatric Disease for JSP191, the FDA’s Fast Track recognizes JSP191’s potential role in improving clinical outcomes for SCID patients, many of whom are too fragile to tolerate the toxic chemotherapy doses typically used in a transplant.”

CIRM Board Approves Funding for New Clinical Trial Targeting Brain Tumors

The governing Board of the California Institute for Regenerative Medicine (CIRM) has awarded almost $12 million to carry out a clinical trial targeting brain tumors.

This brings the total number of CIRM funded clinical trials to 83.  

$11,999,984 was awarded to Dr. Jana Portnow at the Beckman Research Institute of City of Hope. They are using Neural stem cells (NSCs) as a form of delivery vehicle to carry a cancer-killing virus that specifically targets brain tumor cells.

Glioblastoma is the most common malignant primary brain tumor in adults and each year about 12,000 Americans are diagnosed. The 5-year survival rate is only about 10%.

The current standard of care involves surgically removing the tumor followed by radiation, chemotherapy, and alternating electric field therapy. Despite these treatments, survival remains low.

The award to Dr. Portnow will fund a clinical trial to assess the safety and effectiveness of this stem cell-based treatment for Glioblastoma.

The Board also awarded $3,111,467 to Dr. Boris Minev of Calidi Biotherapeutics. This award is in the form of a CLIN1 grant, with the goal of completing the testing needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial in people.

This project uses donor fat-derived mesenchymal stem cells that have been loaded with oncolytic virus to target metastatic melanoma, triple negative breast cancer, and advanced head & neck squamous cell carcinoma.

“There are few options for patients with advanced solid tumor cancers such as glioblastoma, melanoma, breast cancer, and head & neck cancer,” says Maria T. Millan, M.D., President and CEO of CIRM. “Surgical resection, chemotherapy and radiation are largely  ineffective in advanced cases and survival typically is measured in months. These new awards will support novel approaches to address the unmet medical needs of patients with these devastating cancers.”

The CIRM Board also voted to approve awarding $71,949,539 to expand the CIRM Alpha Clinics Network. The current network consists of six sites and the Board approved continued funding for those and added an additional three sites. The funding is to last five years.

The goal of the Alpha Clinics award is to expand existing capacities for delivering stem cell, gene therapies and other advanced treatment to patients. They also serve as a competency hub for regenerative medicine training, clinical research, and the delivery of approved treatments.

Each applicant was required to submit a plan for Diversity, Equity and Inclusion to support and facilitate outreach and study participation by underserved and disproportionately affected populations in the clinical trials they serve.

The successful applicants are:

ApplicationProgram TitleInstitution/Principal InvestigatorAmount awarded
INFR4-13579The Stanford Alpha Stem Cell ClinicStanford University – Matthew Porteus  $7,997,246  
INFR4-13581UCSF Alpha Stem Cell ClinicU.C. San Francisco – Mark Walters  $7,994,347  
INFR4-13586A comprehensive stem cell and gene therapy clinic to
advance new therapies for a diverse patient
population in California  
Cedars-Sinai Medical Center – Michael Lewis  $7,957,966    
INFR4-13587The City of Hope Alpha Clinic: A roadmap for equitable and inclusive access to regenerative medicine therapies for all Californians  City of Hope – Leo Wang  $8,000,000
INFR4-13596Alpha Stem Cell Clinic for Northern and Central California  U.C. Davis – Mehrdad Abedi  $7,999,997  
INFR4-13685Expansion of the Alpha Stem Cell and Gene Therapy Clinic at UCLA  U.C. Los Angeles – Noah Federman  $8,000,000
INFR4-13878Alpha Clinic Network Expansion for Cell and Gene Therapies  University of Southern California – Thomas Buchanan  $7,999,983  
INFR4-13952A hub and spoke community model to equitably deliver regenerative medicine therapies to diverse populations across four California counties  U.C. Irvine – Daniela Bota  $8,000,000
INFR4-13597UC San Diego Health CIRM Alpha Stem Cell Clinic  U.C. San Diego – Catriona Jamieson  $8,000,000

The Board also unanimously, and enthusiastically, approved the election of Maria Gonzalez Bonneville to be the next Vice Chair of the Board. Ms. Bonneville, the current Vice President of Public Outreach and Board Governance at CIRM, was nominated by all four constitutional officers: the Governor, the Lieutenant Governor, the Treasurer and the Controller.

In supporting the nomination, Board member Ysabel Duron said: “I don’t think we could do better than taking on Maria Gonzalez Bonneville as the Vice Chair. She is well educated as far as CIRM goes. She has a great track record; she is empathetic and caring and will be a good steward for the taxpayers to ensure the work we do serves them well.”

In her letter to the Board applying for the position, Ms. Bonneville said: “CIRM is a unique agency with a large board and a long history. With my institutional knowledge and my understanding of CIRM’s internal workings and processes, I can serve as a resource for the new Chair. I have worked hand-in-hand with both the Chair and Vice Chair in setting agendas, prioritizing work, driving policy, and advising accordingly.  I have worked hard to build trusted relationships with all of you so that I could learn and understand what areas were of the most interest and where I could help shed light on those particular programs or initiatives. I have also worked closely with Maria Millan for the last decade, and greatly enjoy our working relationship. In short, I believe I provide a level of continuity and expertise that benefits the board and helps in times of transition.”

In accepting the position Ms. Bonneville said: “I am truly honored to be elected as the Vice Chair for the CIRM Board. I have been a part of CIRM for 11 years and am deeply committed to the mission and this new role gives me an opportunity to help support and advance that work at an exciting time in the Agency’s life. There are many challenges ahead of us but knowing the Board and the CIRM team I feel confident we will be able to meet them, and I look forward to helping us reach our goals.”

Ms. Bonneville will officially take office in January 2023.

The vote for the new Chair of CIRM will take place at the Board meeting on December 15th.

Chemistry Nobel Prize winner Carolyn Bertozzi had a hand in early stem cell research

Carolyn Bertozzi. Image credit: Andrew Brodhead

The 2022 Nobel Prize in Chemistry has been awarded to Carolyn R. Bertozzi of Stanford University, Morten Meldal of the University of Copenhagen, and K. Barry Sharpless of Scripps Research. The three scientists are recognized for their independent development and contributions to the field known as click chemistry and bioorthogonal chemistry. 

Sharpless and Meldal are credited with laying the foundation for click chemistry, a functional form of chemistry in which molecular building blocks snap together quickly and efficiently. But it’s Bertozzi—a Stanford professor, chemist, mentor and early CIRM grantee—who is being recognized for taking click chemistry to a new dimension and utilizing it in living organisms.

A press release from The Royal Swedish Academy of Sciences describes Bertozzi’s accomplishments as follows:  

“To map important but elusive biomolecules on the surface of cells – glycans – Bertozzi developed click reactions that work inside living organisms. Her bioorthogonal reactions take place without disrupting the normal chemistry of the cell.  

These reactions are now used globally to explore cells and track biological processes. Using bioorthogonal reactions, researchers have improved the targeting of cancer pharmaceuticals, which are now being tested in clinical trials.” 

Click chemistry and bioorthogonal reactions, the press release notes, have taken chemistry into the era of functionalism and brings the greatest benefit to humankind. 

Bertozzi celebrates her Nobel Prize win. Image courtesy Kurt Hickman and Harry Gregory for Stanford.

A Hand in Early Stem Cell Research 

Bertozzi also had a hand in early stem cell research funded by the California Institute for Regenerative Medicine (CIRM), California’s stem cell agency.  

As a recipient of a SEED Grant from the agency in 2007, Bertozzi helped jump-start human embryonic stem cell (hESC) research in California. Through that funding, Bertozzi’s lab at UC Berkeley studied the roles of cell surface sugars in the transformation of hESCs into cell types useful for the treatment of human diseases.  

“This work will contribute to a better understanding of how stem cells interact with other cells in their environment and how they mature into different cell types,” Bertozzi said. 

A Prolific Mentor

Bertozzi is also recognized as a prolific mentor, having advised more than 250 undergraduates, graduate students, and postdoctoral fellows, including CIRM Bridges alumni Ian Blong, whose experience working in Bertozzi’s lab was profiled in The Stem Cellar.  

Bertozzi founded and continues to lead the Sarafan ChEM-H Chemistry-Biology Interface Predoctoral Training Program, which helps train graduate students to bridge the gap between chemistry, biology, and medical research.  

She also helped launched a program to prepare recent college graduates from diverse and historically underrepresented backgrounds to apply for doctorate programs in the sciences. In 2022, Bertozzi was recognized with a Lifetime Mentor Award from the American Association for the Advancement of Science for her commitment to mentorship and increasing diversity in science.


CIRM congratulates Bertozzi, Meldal and Sharpless on their Nobel Prize award and for their impressive accomplishments. Read an in-depth profile of Bertozzi and her work on the Stanford Magazine website. Read more about all three scientists and their work here. Read the news release from Stanford here.

Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder

Bone marrow transplant

For children born with severe combined immunodeficiency (SCID) life can be very challenging. SCID means they have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life.

There are stem cell/gene therapies funded by the California Institute for Regenerative Medicine (CIRM), such as ones at UCLA and UCSF/St. Judes, but an alternative method of treating, and even curing the condition, is a bone marrow or hematopoietic stem cell transplant (HCT). This replaces the child’s blood supply with one that is free of the SCID mutation, which helps restore their immune system.

However, current HCT methods involve the use of chemotherapy or radiation to destroy the patient’s own unhealthy blood stem cells and make room for the new, healthy ones. This approach is toxic and complex and can only be performed by specialized teams in major medical centers, making access particularly difficult for poor and underserved communities.

To change that, Dr. Judy Shizuru at Stanford University, with CIRM funding, developed an antibody that can direct the patient’s own immune cells to kill diseased blood stem cells, creating the room needed to transplant new, healthy cells. The goal was to make stem cell transplants safer and more effective for the treatment of many life-threatening blood disorders.

That approach, JSP191, is now being championed by Jasper Therapeutics and they just got some very good news from the Food and Drug Administration (FDA). The FDA has granted JSP191 Fast Track Designation, which can speed up the review of therapies designed to treat serious conditions and fill unmet medical needs.

In a news release, Ronald Martell, President and CEO of Jasper Therapeutics, said this is good news for the company and patients: “This new Fast Track designation recognizes the potential role of JSP191 in improving clinical outcomes for these patients and will allow us to more closely work with the FDA in the upcoming months to determine a path toward a Biologics License Application (BLA) submission.”

Getting a BLA means Jasper will be able to market the antibody in the US and make it available to all those who need it.

This is the third boost from the FDA for Jasper. Previously the agency granted JSP191 both Orphan and Rare Pediatric Disease designations. Orphan drug designation qualifies sponsors for incentives such as tax credits for clinical trials. Rare Pediatric Disease designation means that if the FDA does eventually approve JSP191, then Jasper can apply to receive a priority review of an application to use the product for a different disease, such as someone who is getting a bone marrow transplant for sickle cell disease or severe auto immune diseases.

The researcher who is following her bliss, and tackling diseases of aging at the same time

Dr. Jill Helms, and associate! Photo courtesy Stanford University

Jill Helms is not your average Stanford University faculty member. Yes, she is a professor in the Department of Surgery. Yes, she has published lots of scientific studies. Yes, she is a stem cell scientist (funded by CIRM). And yes, she is playing a leading role in Ankasa Regenerative Therapeutics, a company focused on tissue repair and regeneration. But she is so much more than all that.  

She is a brilliant public speaker, a fashionista, and has ridden her horse to work (well, Stanford is referred to as The Farm, so why not!) and she lives on a farm of her own called “Follow Your Bliss.” The name comes from philosopher Joseph Campbell who wrote, “If you follow your bliss, you put yourself on a kind of path that has been there all the while, waiting for you. And the life you ought to be living is the one you are living.”  

Dr. Helms says that pretty much sums up her life. She says she feels enormously blessed.  

Well, we felt enormously blessed when she agreed to sit down with us and chat about her work, her life and her love of fashion for the California Institute for Regenerative Medicine podcast, Talking ‘Bout (re)Generation.  

We hope you enjoy the latest episode! 

Life lessons learned in the CIRM summer intern program

SPARK poster session; Photo by Esteban Cortez

When I was in high school I spent my summers working in a shoe shop and playing soccer with my mates. It never occurred to me that I could do something really  worthwhile with that time. So, when I meet the high school students who took part in the California Institute for Regenerative Medicine’s SPARK program I realized I had wasted a lot of time.

For those not familiar with SPARK, it stands for Summer Program to Accelerate Regenerative Medicine Knowledge. It’s a summer program offering high school students a chance to work in a world-class stem cell and gene therapy research facility. The quality of the work they do is truly remarkable. By the end of the summer they are doing projects that many full-time researchers would be proud of.

As part of that program the students also must write blogs and post photos and videos to Instagram to chart their progress. The quality of that work is equally impressive. Last week we posted items about the two best blogs from the students. But there were so many other fine entries that we thought it would be worthwhile to highlight elements of those.

For instance, Ricardo Rodriguez at Charles R. Drew University had some interesting observations on life, even when it’s not always working out the way you planned:

Ricardo Rodriguez: Photo by Esteban Cortez

“Cancer is not life going wrong so much as it is life changing. If mutation is random, then so is life. That beautiful randomness that drives evolution and extinction, change and stagnation, life and death, and for you to think that that part of your body could be simple in any way, whether it be simply evil, simply inconvenient, simply structured, is simply hilarious. There is beauty in your body’s complexity, adaptability, and resilience, and these attributes are not barred from any part of your life.”

Mindy Rodriguez at Beckman City of Hope says she learned valuable lessons from working with mice, creatures she previously considered scary, dirty and vicious, but later came to like:

“The CIRM SPARK program reinforced the value of facing my fears by exploring the unknown and most importantly taught me to be comfortable with the uncomfortable. In both cases, I found that it is our response to fear that shapes who we are. We can either run away from the thing that scares us or take each moment as a learning opportunity, embracing change over comfort.”

Manvi Ketireddy at work at UC Davis

Manvi Ketireddy at UC Davis had a similar experience, learning to accept things not working out.

“A researcher must be persistent and have the ability to endure lots of failures. I think that is what I love about research: the slight possibility of discovery and answers amid constant defeat is one of the greatest challenges to exist. And boy, do I love challenges.”

Ameera Ali in the lab (fish not included)

Ameera Ali at Sanford Burnham Prebys says she had struggled for years to decide on a career direction, but the internship gave her a fresh perspective on it all.

“Growing up, I never really knew what I wanted to do for a living, and I think that’s because I wanted to do everything. In kindergarten I wanted to be a paleontologist. In 5th grade I wanted to be the CEO of The San Diego Union Tribune, and in 9th grade I wanted to be a physicist at NASA. By 10th grade I was having an existential crisis about what to do with my life, and so began the search for my purpose at the ripe old age of 15.

So now, writing this blog, I never thought I’d end up spending so much of my time in a room filled floor to ceiling with fish tanks. You might be wondering, how does one end up going from physicist to fish farmer? Well, I’m not completely sure to be honest, but it’s been a very fun and interesting experience nonetheless.”

She says by the end she says what initially felt like mundane chores were actually moments worth celebrating.

“These aquatic friends have taught me a lot of valuable life lessons, like being appreciative of the little things in life, caring for others and see things from a different perspective, and realizing that

working in a biology lab allows me to explore my passions, be creative, and be a mother to hundreds of fish children on the side.”

SPARK attracts students from all over California, and it’s that diversity that makes it so important.

Alexa Gastelum

My name is Alexa Gastelum and I am from a small border town called Calexico. It is located in the Imperial Valley around two hours away from San Diego. I found out about this Internship from my Math teacher and Mesa Coordinator. They discussed what it was about, and I immediately knew that I wanted to apply. I have always been interested in doing labs and researching so I knew that it would be the perfect opportunity for me. It is not normal to be presented with an opportunity like this from where I’m from because it is a small and low-income town. When I told my family about this internship they were very supportive. They agreed that I needed to apply for it since it was an extremely good opportunity. Even though I would need to spend my summer away from my hometown, they were okay with it because they knew that I could not miss out on the opportunity. I decided to write my personal statement on a disease that hit close to home with my family which was Alzheimer’s. It is a disease that runs in my family and my uncle passed from it. I believe that this is what sparked my interest because I wanted to understand how it worked and how it affects the brain.

At the SPARK event Alexa told me her grandmother was so proud of her for being accepted at the program that she was going around town telling everyone about it. Her grandmother, and all the other grandmothers and mothers and fathers, had every reason to be proud of these students. They are remarkable young people and we look forward to following their careers in the years to come.

Fighting for his life and the lives of other stroke survivors

Sean Entin, stroke survivor and founder of Stroke Hacker

The word “miraculous” gets tossed around a lot in the world of medicine, mostly by people who have made an unexpected recovery from a deadly or life-threatening condition. In Sean Entin’s case calling his recovery from an almost-fatal stroke could be called miraculous, but I think you would also have to say it’s due to hard work, determination, and an attitude that never even considered giving up.

Sean had a stroke in 2011. Doctors didn’t think he’d survive. He was put into a coma and underwent surgery to create an opening in his skull to give his brain time and space to heal. When he woke he couldn’t walk or talk, couldn’t count. Doctors told him he would never walk again.

They didn’t know Sean. Fast forward to today. Sean is active, has completed two 5k races – that’s two more than me – and has created Stroke Hacker, a program designed to help others going through what he did.

Sean is a remarkable man, which is why I sat down to chat with him for the latest episode of the California Institutes for Regenerative Medicine’s podcast, ‘Talking ‘Bout (re)Generation’.

He is a fascinating man, and he makes for fascinating company. Enjoy the podcast.

The California Institute for Regenerative Medicine (CIRM) has invested more than $80 million in stroke research, including one clinical trial currently underway.