Stem cell summer: high school students document internships via social media, Part 2

Well, just like that, summer vacation is over. Most kids in California are back in school now and probably one of the first questions they’ll ask their friends is, “what did you do this summer?”. For 58 talented high school students, their answer will be, “I became a stem cell scientist.”

Best Instagram Post Award: Mia Grossman

Those students participated in a CIRM-funded internship called the Summer Program to Accelerate Regenerative medicine Knowledge, or SPARK for short, with seven programs throughout Northern and Southern California which include Caltech, Cedars-Sinai, City of Hope, Stanford, UC Davis, UCSF and the UCSF Benioff Children’s Hospital Oakland. Over the course of about 8 eight weeks, the interns gained hands-on training in stem cell research at some of the leading research institutes in California. Last week, they all met for the annual SPARK conference, this year at the UC Davis Betty Irene Moore School of Nursing, to present their research results and to hear from expert scientists and patient advocates.

As part of their curriculum, the students were asked to write a blog and to post Instagram photos (follow #cirmsparklab) to document their internship experiences. Several CIRM team member selected their favorite entries and presented awards to the winning interns at the end of the conference. We featured two of the winners in a blog from last week.

Our two winners featured today are Cedars-Sinai SPARK student, Mia Grossman – a senior at Beverly Hills High School – one of the Instagram Award winners (see her looping video above) and UC Davis SPARK student Anna Guzman – a junior at Sheldon High School – one of the Blog Award winners. Here’s her blog:

The Lab: A Place I Never Thought I’d Be
By Anna Guzman

AnnaGuzman

Anna Guzman

My CIRM SPARK journey started long before I ever stepped foot in the Institute for Regenerative Cures at UC Davis. Instead, my journey started two years earlier, when my older sister came home from the same internship with stories of passaged cells, images of completed western blots, and a spark in her eye when she described the place she had come to love. Barely 14 years old, I listened wide-eyed as my sister told us about the place she disappeared to each morning, stories of quirky professors, lovable mentors, and above all, the brilliant flame that everyone in her lab shared for learning. But even as she told her stories around the dinner table, I imagined this cold place where my charismatic, intelligent, and inquisitive sister was welcomed. I imagined the chilling concentration of dozens of geniuses bent over their work, of tissue culture rooms where every tiny movement was a potential disaster, and above all, of a labyrinth of brilliant discoveries and official sounding words with the door securely locked to 16 year old girls – girls who had no idea what they wanted to do with their life, who couldn’t confidently rattle words like “CRISPR,” “mesenchymal” and “hematopoietic” off their tongues. In short, this wasn’t a place for me.

But somehow I found myself applying for the CIRM SPARK internship. Seconds after I arrived for my first day at the place I was sure I would not belong, I realized how incorrect my initial assumption of the lab was. Instead of the intimidating and sophisticated environment filled with eye-rolling PhDs who scoffed at the naïve questions of a teenager, I found a room filled with some of the kindest, funniest, warmest people I had ever met. I soon found that the lab was a place of laughter and jokes across bays, a place of smiles in the hallways and mentors who tirelessly explained theory after theory until the intoxicating satisfaction of a lightbulb sparked on inside my head. The lab was a place where my wonderful mentor Julie Beegle patiently guided me through tissue culture, gently reminding me again and again how to avoid contamination and never sighing when I bubbled up the hemocytometer, miscalculated transduction rates, or asked question after question after question. Despite being full of incredibly brilliant scholars with prestigious degrees and publications, the lab was a place where I was never made to feel small or uneducated, never made to feel like there was something I couldn’t understand. So for me, the lab became a place where I could unashamedly fuel my need to understand everything, to ask hundreds of questions until the light bulbs sputtered on and a spark, the same spark that had glowed in the eyes of my sister years ago, burned brightly. The lab became a place where it was always okay to ask why.

At moments towards the middle of the internship, when my nerves had dissolved into a foundation of tentative confidence, and I had started to understand the words that tumbled out of my mouth, I’d be working in the biosafety cabinet or reading a protocol to my mentor and think, Wow. That’s Me. That’s me counting colonies and loading gels without the tell-tale nervous quiver of a beginner’s hand. That’s me explaining my project to another intern without an ambiguous question mark marring the end of the sentence. That’s me, pipetting and centrifuging and talking and understanding – doing all the things that I was certain that I would never be able to do. That’s the best thing that the CIRM SPARK internship has taught me. Being an intern in this wonderful place with these amazing people has taught me to be assured in my knowledge, unashamed in my pursuit of the answer, and confident in my belief that maybe I belong here. These feelings will stay with me as I navigate the next two years of high school and the beginning of the rest of my life. I have no doubt that I will feel unsure again, that I will question whether I belong and wonder if I am enough. But then I will remember how I felt here, confident, and unashamed, and assured in the place where I never thought I’d be.

It was not until the end of my internship, as I stood up to present a journal article to a collection of the very people who had once terrified me, that I realized the biggest thing I was wrong about two years ago. I was wrong when I assumed that this was a place where I would never belong. Instead, as I stood in front of this community of amazingly brilliant and kind people, my mouth forming words that I couldn’t have dreamed of understanding a month ago, I realized that this was precisely where I belonged. This was the place for me.

Stem cell summer: high school students document internships via social media, Part 1

My fellow CIRM team members and I just got back from two days in Sacramento where we attended one of our favorite annual events: the CIRM SPARK Student Conference. SPARK, which is short for Summer Program to Accelerate Regenerative medicine Knowledge, is a CIRM-funded education program that offers California High School students an invaluable opportunity to gain hands-on training in stem cell research at some of the leading research institutes in California.

This meeting represents the culmination of the students’ internships in the lab this summer and gives each student the chance to present their project results and to hear from stem cell research experts and patient advocates. Every summer, without fail, I’m blown away by how much the students accomplish in such a short period of time and by the poise and clarity with which they describe their work. This year was no exception.

Best Instagram Post Award: Skyler Wong

To document the students’ internship experiences, we include a social media curriculum to the program. Each student posts Instagram photos and writes a blog essay describing their time in the lab. Members of the CIRM team reviewed and judged the Instagram posts and blogs. It was a very difficult job selecting only three Instagrams out of over 400 (follow them at #cirmsparklab) that were posted over the past eight weeks. Equally hard was choosing three blogs from the 58 student essays which seem to get better in quality each year.

Over the next week or so, we’re going to feature the three Instagram posts and three blogs that were ultimately awarded. Our two winners featured today are UC Davis SPARK student, Skyler Wong, a rising senior at Sheldon High School was one of the Instagram Award winners (see his photo above) and Stanford SPARK student Angelina Quint, a rising senior at Redondo Union High School, was one of the Blog Award winners. Here’s her blog:

Best Blog Award:
My SPARK 2018 summer stem cell research internship experience
By Angelina Quint

Angelina2

Angelina Quint

Being from Los Angeles, I began the SIMR program as a foreigner to the Bay Area. As my first research experience, I was even more so a foreigner to a laboratory setting and the high-tech equipment that seemingly occupied every edge and surface of Stanford’s Lorry I. Lokey Stem Cell building. Upon first stepping foot into my lab at the beginning of the summer, an endless loop of questions ran through my brain as I ventured deeper into this new, unfamiliar realm of science. Although excited, I felt miniscule in the face of my surroundings—small compared to the complexity of work that laid before me. Nonetheless, I was ready to delve deep into the unknown, to explore this new world of discovery that I had unlocked.

Participating in the CIRM research program, I was given the extraordinary opportunity to pursue my quest for knowledge and understanding. With every individual I met and every research project that I learned about, I became more invigorated to investigate and discover answers to the questions that filled my mind. I was in awe of the energy in the atmosphere around me—one that buzzed with the drive and dedication to discover new avenues of thought and complexity. And as I learned more about stem cell biology, I only grew more and more fascinated by the phenomenon. Through various classes taught by experts in their fields on topics spanning from lab techniques to bone marrow transplants, I learned the seemingly limitless potential of stem cell research. With that, I couldn’t help but correlate this potential to my own research; anything seemed possible.

However, the journey proved to be painstakingly arduous. I soon discovered that a groundbreaking cure or scientific discovery would not come quickly nor easily. I faced roadblocks daily, whether it be in the form of failed gel experiments or the time pressures that came with counting colonies. But to each I learned, and to each I adapted and persevered. I spent countless hours reading papers and searching for online articles. My curiosity only grew deeper with every paper I read—as did my understanding. And after bombarding my incredibly patient mentors with an infinite number of questions and thoughts and ideas, I finally began to understand the scope and purpose of my research. I learned that the reward of research is not the prestige of discovering the next groundbreaking cure, but rather the knowledge that perseverance in the face of obstacles could one day transform peoples’ lives for the better.

As I look back on my journey, I am filled with gratitude for the lessons that I have learned and for the unforgettable memories that I have created. I am eternally grateful to my mentors, Yohei and Esmond, for their guidance and support along the way. Inevitably, the future of science is uncertain. But one thing is always guaranteed: the constant, unhindered exchange of knowledge, ideas, and discovery between colleagues passionate about making a positive difference in the lives of others. Like a stem cell, I now feel limitless in my ability to expand my horizons and contribute to something greater and beyond myself. Armed with the knowledge and experiences that I have gained through my research, I aspire to share with others in my hometown the beauty of scientific discovery, just as my mentors have shared with me. But most of all, I hope that through my continued research, I can persist in fighting for new ways to help people overcome the health-related challenges at the forefront of our society.

 

Starving stem cells of oxygen can help build stronger bones

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J. Kent Leach: Photo courtesy UC Davis

We usually think that starving something of oxygen is going to make it weaker and maybe even kill it. But a new study by J. Kent Leach at UC Davis shows that instead of weakening bone defects, depriving them of oxygen might help boost their ability to create new bone or repair existing bone.

Leach says in the past the use of stem cells to repair damaged or defective bone had limited success because the stem cells often didn’t engraft in the bone or survive long if they did. That was because the cells were being placed in an environment that lacked oxygen (concentration levels in bone range from 3% to 8%) so the cells found it hard to survive.

However, studies in the lab had shown that if you preconditioned mesenchymal stem cells (MSCs), by exposing them to low oxygen levels before you placed them on the injury site, you helped prolong their viability. That was further enhanced by forming the MSCs into three dimensional clumps called spheroids.

Lightbulb goes off

In the  current study, published in Stem Cells, Leach says the earlier spheroid results  gave him an idea:

“We hypothesized that preconditioning MSCs in hypoxic (low oxygen) culture before spheroid formation would increase cell viability, proangiogenic potential (ability to create new blood vessels), and resultant bone repair compared with that of individual MSCs.”

So, the researchers placed one group of human MSCs, taken from bone marrow, in a dish with just 1% oxygen, and another identical group of MSCs in a dish with normal oxygen levels. After three days both groups were formed into spheroids and placed in an alginate hydrogel, a biopolymer derived from brown seaweed that is often used to build cellular cultures.

Seaweed

Brown seaweed

The team found that the oxygen-starved cells lasted longer than the ones left in normal oxygen, and the longer those cells were deprived of oxygen the better they did.

Theory is great, how does it work in practice?

Next was to see how those two groups did in actually repairing bones in rats. Leach says the results were encouraging:

“Once again, the oxygen-deprived, spheroid-containing gels induced significantly more bone healing than did gels containing either preconditioned individual MSCs or acellular gels.”

The team say this shows the use of these oxygen-starved cells could be an effective approach to repairing hard-to-heal bone injuries in people.

“Short‐term exposure to low oxygen primes MSCs for survival and initiates angiogenesis (the development of new blood vessels). Furthermore, these pathways are sustained through cell‐cell signaling following spheroid formation. Hypoxic (low oxygen) preconditioning of MSCs, in synergy with transplantation of cells as spheroids, should be considered for cell‐based therapies to promote cell survival, angiogenesis, and bone formation.”

CIRM & Dr. Leach

While CIRM did not fund this study we have invested more than $1.8 million in another study Dr. Leach is doing to develop a new kind of imaging technology that will help us see more clearly what is happening in bone and cartilage-targeted therapies.

In addition, back in March of 2012, Dr. Leach spoke to the CIRM Board about his work developing new approaches to growing bone.

 

Stem cell roundup: summer scientists, fat-blocking cells & recent human evolution

Stem cell photo of the week: high schooler becoming a stem cell pro this summer

InstagramAnnaJSPARK

High school student Anna Guzman learning important lab skills at UC Davis

This summer’s CIRM SPARK Programs, stem cell research internships for high school students, are in full swing. Along with research assignments in top-notch stem cell labs, we’ve asked the students to chronicle their internship experiences through Instagram. And today’s stem cell photo of the week is one of those student-submitted posts. The smiling intern in this photo set is Anna Guzman, a rising junior from Sheldon High School who is in the UC Davis SPARK Program. In her post, she describes the lab procedure she is doing:

“The last step in our process to harvest stem cells from a sample of umbilical cord blood! We used a magnet to isolate the CD34 marked stem cells [blood stem cells] from the rest of the solution.”

Only a few days in and Anna already looks like a pro! It’s important lab skills like this one that could land Anna a future job in the stem cell field. Check out #cirmsparklab on Instagram to view the ever-growing number of posts.

Swiss team identifies a cell type that block formation of fat cells

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(Left) Mature human fat cells grown in a Petri dish (green, lipid droplets). (Right) A section of mouse fat tissue showing, in the middle, a blood vessel (red circle) surrounded by fat cell blocking cells called Aregs (arrows). [Bart Deplancke/EPFL]

Liposuction surgery helps slim and reshape areas of a person’s body through the removal of excess fat tissue. While the patient is certainly happy to get rid of those extra pounds, that waste product is sought after by researchers because it’s a rich source of regenerative cells including fat stem cells.

The exact populations of cells in this liposuction tissue has been unclear, so a collaboration of Swiss researchers – at Ecole Polytechnique Fédérale de Lausanne (EPFL) and Eidgenössische Technische Hochschule Zürich (ETHZ) – used a cutting-edge technique allowing them to examine the gene activity within single cells.

The analysis was successful in identifying several newly defined subpopulations of cells in the fat tissue. To their surprise, one of those cell types did not specialize into fat cells but instead did the opposite: they inhibited other fat stem cells from giving rise to fat cells. The initial experiments were carried out in mice, but the team went on to show similar fat-blocking cells in human tissue. Further experiments will explore the tantalizing prospect of applying these cells to control obesity and the many diseases, like diabetes, that result from it.

The study was published June 20st in Nature.

Connection identified between recent human evolution & risk for premature birth
Evidence of recent evolution in a human gene that’s critical for maintaining pregnancy may help explain why some populations have a higher risk for giving birth prematurely than others. That’s according to a recent report by researchers at the University of Stanford School of Medicine.

The study, funded in part by CIRM’s Genomics Initiative, compared DNA from people with East Asian, European and African ancestry. They specifically examined the gene encoding the progesterone hormone receptor which helps keep a pregnant woman from going into labor too soon. The gene is also associated with preterm births, the leading cause of infant death in the U.S.

The team was very surprise to find that people with East Asian ancestry had an evolutionarily new version of the gene while the European and African populations had mixtures of new and ancient versions. These differences may explain why the risk for premature birth among East Asian populations is lower than among pregnant women of European and African descent, though environment clearly plays a role as well.

Pediatrics professor Gary Shaw, PhD, one of the team leaders, put the results in perspective:

“Preterm birth has probably been with us since the origin of the human species,” said Shaw in a press release, “and being able to track its evolutionary history in a way that sheds new light on current discoveries about prematurity is really exciting.”

The study was published June 21st in The American Journal of Human Genetics.

School’s Out! Stem cells are in! High school students start CIRM-funded summer research internships.

Robotic engineering, coding, video game design, filmmaking, soccer and swimming: these are just a few of the many activities that are vying for the attention of high school students once school lets out for the summer.

But a group of about 50 high schoolers in California have chosen a different path: they will be diving into the world of stem cell biology. Each student earned a spot in one of seven CIRM-funded SPARK Programs across California. That’s short for Summer Program to Accelerate Regenerative Medicine Knowledge (yes, technically it should be SPARMK but we like SPARK better).

The SPARK students will gain hands-on training in stem cell research at some of the leading research institutes in California by conducting a six-week research internship in a stem cell lab. Maybe I’m bias, as the Program Director at CIRM who oversees the SPARK programs, but I think they’ve made a great decision. Stem cell research is one of, if not the most exciting and cutting-edge fields of research science out there today.

The pace of progress is so rapid in the field that a large workforce over the next century is critical to sustain CIRM’s mission to accelerate stem cell treatments to patients with unmet medical needs. That’s why the Agency has invested over $4 million to support over 400 SPARK interns since 2012.

Yesterday, I had the pleasure to be in Sacramento to welcome the UC Davis SPARK interns on their first day of their program which is led by Gerhard Bauer, director of the Good Manufacturing Practice (GMP) laboratory at the UC Davis Institute for Regenerative Cures. The other programs, like the one at Cedars-Sinai in Los Angeles (see photo below), are also starting this week or next.

CedarSinaiSPARK2018

Because everything we do at CIRM is focused on the patient, the SPARK programs are required to include patient engagement as part of the students’ internships. Here are some Instagram posts from last year that highlight those patient-centered activities.

CedarSinaiSPARK2017Patients

And speaking of Instagram, we have also included a social media component to the program. We believe it’s critical for scientists to connect with the public about the important work they do. During the UC Davis orientation, Jan Nolta, PhD, the director of the Stem Cell Program at UC Davis School of Medicine, pointed out to the students that making the science accessible and understandable to the public, makes stem cell research less scary and, as a result, it’s more likely to gain public support.

So, as part of their curriculum, the interns will share a few Instagrams per week that capture their summer in the lab. You can follow their posts at #CIRMSPARKLab. In addition to communicating through photos, the students will describe their internship experiences by writing a blog. We’ll post the most outstanding blogs later this summer. In the meantime, you can read last summer’s winning blogs.

At the end of their program, the students get to show off their hard work by presenting their research at the SPARK annual conference which will be held this year at UC Davis. It’s going to be an exciting summer!

UC Davis Stem Cell Director Jan Nolta Shares Her Thoughts on the Importance of Mentoring Young Scientists

Dr. Jan Nolta (UC Davis Health)

Jan Nolta is a scientific rockstar. She is a Professor at UC Davis and the Director of the Stem Cell Program at the UC Davis School of Medicine. Her lab’s research is dedicated to developing stem cell-based treatments for Huntington’s disease (HD). Jan is a tireless advocate for both stem cell and HD research and you’ll often see her tweeting away about the latest discoveries in the field to her followers.

What I admire most about Dr. Nolta is her dedication to educating and mentoring young students. Dr. Nolta helped write the grant that funded the CIRM Bridges master’s program at Sacramento State in 2009. Over the years, she has mentored many Bridges students (we blogged about one student earlier this year) and also high school students participating in CIRM’s SPARK high school internship program. Many of her young trainees have been accepted to prestigious colleges and universities and gone on to pursue exciting careers in STEM.

I reached out to Dr. Nolta and asked her to share her thoughts on the importance of mentoring young scientists and supporting their career ambitions. Below is a summary of our conversation. I hope her passion and devotion will inspire you to think about how you can get involved with student mentorship in your own career.


Describe your career path from student to professor.

I was an undergraduate student at Sacramento State University. I was a nerdy student and did research on sharks. I was planning to pursue a medical degree, but my mentor, Dr. Laurel Heffernan, encouraged me to consider science. I was flabbergasted at the suggestion and asked, “people pay you to do this stuff??” I didn’t know that you could be paid to do lab research. My life changed that day.

I got my PhD at the University of Southern California. I studied stem cell gene therapy under Don Kohn, who was a fabulous mentor. After that, I worked in LA for 15 years and then went back home to UC Davis in 2007 to direct their Stem Cell Program.

It was shortly after I got to Davis that I reconnected with my first mentor, Dr. Heffernan, and we wrote the CIRM Bridges grant. Davis has a large shared translational lab with seven principle investigators including myself and many of the Bridges students work there. Being a scientist can be stressful with grant deadlines and securing funding. Mentoring students is the best part of the job for me.

Why is it important to fund educational programs like Bridges and SPARK?

There is a serious shortage of well-trained specialists in regenerative medicine in all areas of the workforce. The field of regenerative medicine is still relatively new and there aren’t enough people with the required skills to develop and manufacture stem cell treatments. The CIRM Bridges program is critical because it trains students who will fill those key manufacturing and lab manager jobs. Our Bridges program at Sacramento State is a two-year master’s program in stem cell research and lab management. They are trained at the UC Davis Good Manufacturing Practice (GMP) training facility and learn how to make induced pluripotent stem cells (iPSCs) and other stem cell products. There aren’t that many programs like ours in the country and all of our students get competitive job offers after they complete our program.

We are equally passionate about our high school SPARK program. It’s important to capture students’ interests early whether they want to be a scientist or not. It’s important they get exposed to science as early as possible and even if they aren’t going to be a scientist or healthcare professional, it’s important that they know what it’s about. It’s inspiring how many of these students stay in STEM (Science, Technology, Engineering and Math) because of this unique SPARK experience.

Jan Nolta with the 2016 UC Davis SPARK students.

Can you share a student success story?

I’m so proud of Ranya Odeh. She was a student in our 2016 SPARK program who worked in my lab. Ranya received a prestigious scholarship to Stanford largely due to her participation in the CIRM SPARK program. I got to watch her open the letter on Instagram, and it was a really incredible experience to share that part of her life.

I’m also very proud of our former Bridges student Jasmine Carter. She was a mentor to one of our SPARK students Yasmine this past summer. She was an excellent role model and her passion for teaching and research was an inspiration to all of us. Jasmine was hoping to get into graduate school at UC Davis this fall. She not only was accepted into the Neuroscience Graduate Program, but she also received a prestigious first year program fellowship!

UC Davis Professors Jan Nolta and Kyle Fink with CIRM Bridges student Jasmine Carter

[Side note: We’ve featured Ranya and Jasmine previously on the Stem Cellar and you can read about their experiences here and here.]

Why is mentoring important for young students?

I can definitely relate to the importance of having a mentor. I was raised by a single mom, and without scholarships and great mentors, there’s no way I would be where I am today. I’m always happy to help other students who think maybe they can’t do science because of money, or because they think that other people know more than they do or are better trained. Everybody who wants to work hard and has a passion for science deserves a chance to shine. I think these CIRM educational programs really help the students see that they can be what they dream they can be.

What are your favorite things about being a mentor?

Everyday our lab is full of students, science, laughter and fun. I love coming in to the lab. Our young people bring new ideas, energy and great spirit to our team. I think every team should have young trainees and high school kids working with them because they see things in a different way.

Do you have advice for mentoring young scientists?

You can sum it up in one word: Listen. Ask them right away what their dreams are, where do they imagine themselves in the future, and how can you help them get there. Encourage them to always ask questions and let them know that they aren’t bothering you when they do. I also let my students know that I’m happy to be helping them and that the experience is rewarding for me as well.

So many students are shy when they first start in the lab and don’t get all that they can out of the experience. I always tell my students of any age: what you really want to do is try in life. Follow your tennis ball. Like when a golden retriever sees a tennis ball going by, everything else becomes secondary and they follow that ball. You need to find what that tennis ball is for you and then just try to follow it.

What advice can you give to students who want to be scientific professors or researchers?

Find somebody who is a good mentor and cares about you. Don’t go into a lab where the Principle Investigator (PI) is not there most of the time. You will get a lot more out of the experience if you can get input from the PI.

A good mentor is more present in the lab and will take you to meetings and introduce you to people. I find that often students read papers from well-established scientists, and they think that their positions are unattainable. But if they can meet them in person at a conference or a lecture, they will realize that all of the established scientists are people too. I want young students to know that they can do it too and these careers are attainable for anybody.

New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease

UC Davis Institute for Regenerative Cures

For people battling Duchenne Muscular Dystrophy (DMD), a rare and fatal genetic disorder that slowly destroys muscles, hope has often been in short supply. There is no cure and treatments are limited. But now a new clinical trial at the site of one of the newest CIRM Alpha Stem Cell Clinic Network members could change that.

The HOPE-2 clinical trial has treated its first patient at UC Davis Medical Center, inaugurating the institution’s Alpha Stem Cell Clinic. The clinic is part of a CIRM-created network of top California medical centers that specialize in delivering stem cell clinical trials to patients. The key to the Network’s success is the ability to accelerate the delivery of treatments to patients through partnerships with patients, medical providers and clinical trial sponsors.

UC Davis is one of five medical centers that now make up the network (the others are UC San Francisco, UCLA/UC Irvine, UC San Diego and City of Hope).

Jan NoltaIn a news release, Jan Nolta, the director of the UC Davis Institute for Regenerative Cures, says the UC Davis Alpha Clinic is well equipped to move promising therapies out of the lab and into clinical trials and people.

“We have the full range of resource experts in regenerative medicine, from the cellular to the clinical trials level. We’re also excited about the prospect of being able to link with other Alpha Stem Cell Clinics around the state to help speed the process of testing and refining treatments so we can get therapies to patients in need.”

The news of this first patient is a cause for double celebration at CIRM. The trial is run by Capricor and CIRM funded the first phase of this work. You can read the story of Caleb Sizemore, who took part in that trial or watch this video of him talking about his fight.

When the CIRM Board approved funding for the UC Davis Alpha Clinic in October of 2017, Abla Creasey, CIRM’s Vice President for Therapeutics and Strategic Infrastructure, said:

“The Alpha Clinics are a one-of-a-kind network that gives patients access to the highest quality stem cell trials for a breadth of diseases including cancer, diabetes, heart disease and spinal cord injury. Expanding our network will allow more patients to participate in stem cell trials and will advance the development of stem cell treatments that could help or possibly cure patients.”

The UC Davis Alpha Clinic provides a one-stop shop for delivering stem cell therapies, gene therapies and immunotherapies, as well as conducting follow-up visits. It’s this type of CIRM-funded infrastructure that helps steer potential clinical trial participants away from illegitimate, unproven and potentially harmful fee-for-service stem cell treatments.

The DMD trial is the first of what we are confident will be many high-quality trials at the Clinic, bringing promising stem cell therapies to patients with unmet medical needs.

 

Patients at the heart of Alpha Stem Cell Clinics Symposium

I have been to a lot of stem cell conferences over the years and there’s one recent trend I really like: the growing importance and frequency of the role played by patient advocates.

There was a time, not so long ago, when having a patient advocate speak at a scientific conference was almost considered a novelty. But more and more it’s being seen for what it is, an essential item on the agenda. After all, they are the reason everyone at that conference is working. It’s all about the patients.

That message was front and center at the 3rd Annual CIRM Alpha Stem Cell Clinics Network Symposium at UCLA last week. The theme of the symposium was the Delivery of Stem Cell Therapeutics to Patients. There were several fascinating scientific presentations, highlighting the progress being made in stem cell research, but it was the voices of the patient advocates that were loudest and most powerful.

First a little background. The CIRM Alpha Stem Cell Clinics Network consists of six major medical centers – UCLA/UC Irvine (joint hosts of this conference), UC San Diego, City of Hope, UC San Francisco and UC Davis. The Network was established with the goal of accelerating the development and delivery of high-quality stem cell clinical trials to patients. This meeting brought together clinical investigators, scientists, patients, patient advocates, and the public in a thoughtful discussion on how novel stem cell therapies are now a reality.

It was definitely thoughtful. Gianna McMillan, the Co-Founder and Executive Director of “We Can, Pediatric Brain Tumor Network” set the tone with her talk titled, “Tell Me What I Need to Know”. At age 5 her son was diagnosed with a brain tumor, sending her life into a tailspin. The lessons she learned from that experience – happily her son is now a healthy young man – drive her determination to help others cope with similar situations.

Calling herself an “in the trenches patient advocate champion” she says:

“In the old days doctors made decisions on behalf of the patients who meekly and gratefully did what they were told. It’s very different today. Patients are better informed and want to be partners in the treatment they get. But yet this is not an equal partnership, because subjects (patients) are always at a disadvantage.”

She said patients often don’t speak the language of the disease or understand the scientific jargon doctors use when they talk about it. At the same time patients are wrestling with overwhelming emotions such as fear and anxiety because their lives have been completely overturned.

Yet she says a meaningful partnership is possible as long as doctors keep three basic questions in mind when dealing with people who are getting a new diagnosis of a life-threatening or life-changing condition:

  • Tell me what I need to know
  • Tell me in language I can understand
  • Tell me again and again

It’s a simple formula, but one that is so important that it needs to be stated over and over again. “Tell me again. And again. And again.”

David Mitchell, the President and Founder of Patients for Affordable Drugs, tackled another aspect of the patient experience: the price of therapies. He posed the question “What good is a therapy if no one can afford it?”

David’s organization focuses on changing policy at the state and federal level to lower the price of prescription drugs. He pointed out that many other countries charge lower prices for drugs than the US, in part because those countries’ governments negotiate directly with drug companies on pricing.

He says if we want to make changes in this country that benefit patients then patient have to become actively involved in lobbying their government, at both the state and local level, for more balanced prices, and in supporting candidates for public office who support real change in drug-pricing policy.

It’s encouraging to see that just as the field of stem cell research is advancing so too is the prominence of the patient’s voice. The CIRM Alpha Stem Cell Clinics Network is pushing the field forward in exciting ways, and the patients are becoming an increasingly important, and vital part of that. And that is as it should be.

UC Davis researchers make stem cell-derived mini-brains that contain blood vessels

Growing neurons on a flat petri dish is a great way to study the inner workings of nerve signals in the brain. But I think it’s safe to argue that a two-dimensional lawn of cells doesn’t capture all the complexity of our intricate, cauliflower-shaped brains. Then again, cracking open the skulls of living patients is also not a viable path for fully understanding the molecular basis of brain disorders.

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Brain organoids (two white balls) growing in petri dish.
Image: Pasca Lab, Stanford University.

The recent emergence of stem cell-derived mini-brains, or brain organoids, as a research tool is bridging this impasse. With induced pluripotent stem cells (iPSCs) derived from a readily-accessible skin sample from patients, it’s possible to generate three-dimensional balls of cells that mimic particular parts of the brain’s anatomy. These mini-brains have the expected type of neurons, as well as other cells that support neuron function. We’ve written many blogs, most recently in January, on the applications of this cutting-edge tool.

With any new technology, there is always room for improvement. One thing that most mini-brains lack is their own system of blood vessels, or vasculature. That’s where Dr. Ben Waldau, a vascular neurosurgeon at UC Davis Medical Center, and his lab come into the picture. Last week, their published work in NeuroReport showed that incorporating blood vessels into a brain organoid is possible.

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A stained cross-section of a brain organoid showing that blood vessels (in red) have penetrated both the outer, more organized layers and the inner core. Image: UC Davis Institute for Regenerative Cures

Using iPSCs from one patient, the Waldau team separately generated brain organoids and blood vessels cells, also called endothelial cells. After growing each for about a month, the organoids were embedded in a gelatin containing the endothelial cells. In an excellent Wired article, writer Megan Molteni explains what happened next:

“After incubating for three weeks, they took a single organoid and transplanted it into a tiny cavity carefully carved into a mouse’s brain. Two weeks later the organoid was alive, well—and, critically, had grown capillaries that penetrated all the way to its inner layers.”

Every tissue relies on nutrients and oxygen from the blood. As Molteni suggests, being able to incorporate blood vessels and brain organoids from the same patient’s cells may make it possible to grow and study even more complex brain structures without the need of a mouse using fluidic pumps.

As Waldau explains in the Wired article, this vascularized brain organoid system also adds promise to the ultimate goal of repairing damaged brain tissue:

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Ben Waldau

“The whole idea with these organoids is to one day be able to develop a brain structure the patient has lost made with the patient’s own cells. We see the injuries still there on the CT scans, but there’s nothing we can do. So many of them are left behind with permanent neural deficits—paralysis, numbness, weakness—even after surgery and physical therapy.”

 

 

Stem Cell Roundup: New infertility tools, helping the 3 blind mice hear and cow ESCs

Cool Stem Cell Image of the Week

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Human egg grown from immature cells in ovarian tissue. (credit: David Albertini)

This week’s Cool Stem Cell Image of the Week comes to us from the lab of reproductive biologist Evelyn Telfer at the University of Edinburgh. Telfer and her team successfully grew human eggs cells from immature ovarian tissue.

This technology could revolutionize the way doctors approach infertility. For instance, when girls and young women undergo chemotherapy for cancer, their eggs are often damaged. By preserving a small piece of ovarian tissue before the cancer treatments, this method could be used to generate eggs later in life for in vitro fertilization. Much more work is necessary to figure out if these eggs are healthy and safe to use to help infertile women.

The study was recently published in Molecular Human Reproduction and was picked up this Science writer Kelly Servick.

Forget 3 blind mice, iPS cells could help 3 deaf mice hear again (Kevin McCormack)
For years scientists have been trying to use stem cells to restore hearing to people who are deaf or hearing impaired. Now a group of researchers in Japan may have found a way.

The team used human iPS cells to create inner ear cells, the kind damaged in one of the most common forms of hereditary deafness. They then transplanted them into the inner ears of mice developing in the womb that are suffering from a congenital form of hearing loss. The cells appeared to engraft and produce a protein, Connexin 30, known to be critical in hearing development.

The research, published in the journal Scientific Reports, could be an important step towards developing a therapy for congenital hearing loss in people.

UC Davis team isolates cow embryonic stem cells for the first time

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An early stage cow embryo. Inner cell mass (red) is source of embryonic stem cells. (Credit: Pablo Ross/UC Davis) 

Although human embryonic stem cells (ESCs) were isolated way back in ’98, researchers haven’t had similar luck with embryonic stem cells from cows. Until this week, that is.  A UC Davis team just published a report in PNAS showing that they not only can isolate cow ESCs but their method works almost 100% of the time.

 

Genetic engineering of these cow stem cells could have huge implications for the cattle industry. Senior author Pablo Ross mentioned in a press release how this breakthrough could help speed up the process of generating superior cows that produce more milk, release less methane and are more resistant to disease:

“In two and a half years, you could have a cow that would have taken you about 25 years to achieve. It will be like the cow of the future. It’s why we’re so excited about this.”

These cow ESCs may also lead to better models of human disease. Because of their small size, rat and mouse models are not always a good representation of how potential therapies or drugs will affect humans. Creating stem cell models from larger animals may provide a better representation.