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.

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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.

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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.

The Journey of a Homegrown Stem Cell Research All-Star

Nothing makes a professional sports team prouder than its homegrown talent. Training and mentoring a promising, hard-working athlete who eventually helps carry the team to a championship can lift the spirits of an entire city.

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Brian Fury

Here at CIRM, we hold a similar sense of pride in Brian Fury, one of our own homegrown all-stars. Nearly a decade ago, Brian was accepted into the inaugural class of CIRM’s Bridges program which provides paid stem cell research internships to students at California universities and colleges that don’t have major stem cell research programs. The aim of the program, which has trained over 1200 students to date, is to build the stem cell work force here in California to accelerate stem cell treatments to patients with unmet medical needs.

A CIRM full circle
Today, Brian is doing just that as manager of manufacturing at the UC Davis Institute for Regenerative Cures (IRC) where he leads the preparation of stem cell therapy products for clinical trials in patients. It was at UC Davis that he did his CIRM Bridges internship as a Sacramento State masters student back in 2009. So, he’s really come full circle, especially considering he currently works in a CIRM-funded facility and manufactures stem cell therapy products for CIRM-funded clinical trials.

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Gerhard Bauer

“Many of the technicians we have in the [cell manufacturing] facility are actually from the Bridges program CIRM has funded, and were educated by us,” Gerhard Bauer, Brian’s boss and director of the facility, explained to me. “Brian, in particular, has made me incredibly proud. To witness that the skills and knowledge I imparted onto my student would make him such an integral part of our program and would lead to so many novel products to be administered to people, helping with so many devastating diseases is a very special experience. I treasure it every day.”

“It sustains me”
Brian’s career path wasn’t always headed toward stem cell science. In a previous life, he was an undergrad in computer management information systems. It was a required biology class at the time that first sparked his interest in the subject. He was fascinated by the course and was inspired by his professor, Cathy Bradshaw. He still recalls a conversation he had with her to better understand her enthusiasm for biology:

“I asked her, ‘what is it about biology that really made you decide this is what you wanted to do?’ And she just said, ‘It sustains me. It is air in my lungs.’ It was what she lived and breathed. That really stuck with me early on.“

Still, Brian went on to earn his computer degree and worked as a computer professional for several years after college. But when the dot com boom went bust in the early 2000’s, Brian saw it as a sign to re-invent himself. Remembering that course with Professor Bradshaw, he went back to school to pursue a biology degree at Sacramento State University.

On a path before there was a path
Not content with just his textbooks and lectures at Sac State, Brian offered to volunteer in any lab he could find, looking for opportunities to get hands-on experience:

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Brian at work during his Sacramento State days.

“I was really hungry to get involved and I really wanted to not just be in class and learning about all these amazing things in biology but I also wanted to start putting them to work. And so, I looked for any opportunity that I could to become actively involved in actually seeing how biology really works and not just the theory.”

This drive to learn led to several volunteer stints in labs on campus as well as a lab manager job. But it was an opportunity he pursued as he was finishing up his degree that really set in motion his current career path. Gerhard Bauer happened to be giving a guest lecture at Sac State about UC Davis’ efforts to develop a stem cell-based treatment for HIV. Hearing that talk was an epiphany for Brian. “That’s really what hooked me in and helped determine that this is definitely the field that I want to enter into. It was my stepping off point.”

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Brian Fury (center) flanked by mentors Gerhard Bauer (left) and Jan Nolta (right)

Inspired, Brian secured a volunteering gig on that project at UC Davis – along with all his other commitments at Sac State – working under Bauer and Dr. Jan Nolta, the director of the UC Davis Stem Cell Program.

That was 2008 and this little path Brian was creating by himself was just about to get some serious pavement. The next year, Sacramento State was one of sixteen California schools that was awarded the CIRM Bridges to Stem Cell Research grant. Their five-year, $3 million award (the total CIRM investment for all the schools was over $55 million) helped support a full-blown, stem cell research-focused master’s program which included 12-month, CIRM-funded internships. One of the host researchers for the internships was, you guessed it, Jan Nolta at UC Davis.

Good Manufacturing Practice (GMP) was a good move
Applying to this new program was a no brainer for Brian and, sure enough, he was one of ten students selected for the first-year class. His volunteer HIV project in the Nolta lab seamlessly dovetailed into his Bridges internship project. He was placed under the mentorship of Dr. Joseph Anderson, a researcher in the Nolta lab at the time, and gained many important skills in stem cell research. Brian’s project focused on a stem cell and gene therapy approach to making HIV-resistant immune cells with the long-term goal of eradicating the virus in patients. In fact, follow on studies by the Anderson lab have helped lead to a CIRM-funded clinical trial, now underway at UC Davis, that’s testing a stem cell-based treatment for HIV/AIDs patients.

After his Bridges internship came to a close, Brian worked on a few short-term research projects at UC Davis but then found himself in a similar spot: needing to strike out on a career path that wasn’t necessarily clearly paved. He reached out to Nolta and Bauer and basically cut to the chase in an email asking, “do you know anybody?”. Bauer reply immediately, “yeah, me!”. It was late 2011 and UC Davis had built a Good Manufacturing Practice (GMP) facility with the help of a CIRM Major Facility grant. Bauer only had one technician at the time and work was starting to pick up.

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The Good Manufacturing Practice (GMP) facility in UC Davis’ Institute for Regenerative Cures.

A GMP facility is a specialized laboratory where clinical-grade cell products are prepared for use in people. To ensure the cells are not contaminated, the entire lab is sealed off from the outside environment and researchers must don full-body lab suits. We produced the video below about the GMP facility just before it opened.

Bauer knew Brian would be perfect at their GMP facility:

“Brian was a student in the first cohort of CIRM Bridges trainees and took my class Bio225 – stem cell biology and manufacturing practices. He excelled in this class, and I also could observe his lab skills in the GMP training part incorporated in this class. I was very lucky to be able to hire Brian then, since I knew what excellent abilities he had in GMP manufacturing.”

CIRM-supported student now supporting CIRM-funded clinical trials

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Brian Fury suited up in GMP facility

Since then, Brian has worked his way up to managing the entire GMP facility and its production of cell therapy products. At last count, he and the five people he supervises are juggling sixteen cell manufacturing projects. One of his current clients is Angiocrine which has a CIRM-funded clinical trial testing a cell therapy aimed to improve the availability and engraftment of blood stem cell transplants. This treatment is geared for cancer patients who have had their cancerous bone marrow removed by chemotherapy.

When a company like Angiocrine approaches Brian at the GMP facility, they already have a well-defined method for generating their cell product. Brian’s challenge is figuring out how to scale up that process to make enough cells for all the patients participating in the clinical trial. And on top of that, he must design the procedures for the clean room environment of the GMP facility, where every element of making the cells must be written down and tracked to demonstrate safety to the Food and Drug Administration (FDA).

The right time, the right place…and a whole bunch of determination and passion
It’s extremely precise and challenging work but that’s what makes it so exciting for Brian. He tells me he’s never bored and always wakes up looking forward to what each day’s challenges will bring and figuring out how he and his team are going get these products into the clinic. It’s a responsibility he takes very seriously because he realizes what it means for his clients:

“I invest as much energy and passion and commitment into these projects as I would my own family. This is extremely important to me and I feel so incredibly fortunate to have the opportunity to work on things like this. The reality is, in the GMP, people are bringing their life’s work to us in the hopes we can help people on the other end. They share all their years of development, knowledge and experience and put it in our hands and hope we can scale this up to make it meaningful for patients in need of these treatments.”

Despite all his impressive accomplishments, Brian is a very modest guy using phrases like “I was just in the right place at the right time,” during our conversation. But I was glad to hear him add “and I was the right candidate”. Because it’s clear to me that his determination and passion are the reasons for his success and is the epitome of the type of researcher CIRM had hoped its investment in the Bridges program and our SPARK high school internship program would produce for the stem cell research field.

That’s why we’ll be brimming over with an extra dose of pride on the day that one of Brian’s CIRM-funded stem cell therapy products reaches the goal line with an FDA approval.

Alpha clinics and a new framework for accelerating stem cell treatments

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Last week, at the World Stem Cell Summit in Miami, CIRM took part in a panel discussion about the role and importance of Alpha Clinics in not just delivering stem cell therapies, but in helping create a new, more collaborative approach to medicine. The Alpha Clinic concept is to create  a network of top medical centers that specialize in delivering stem cell clinical trials to patients.

The panel was moderated by Dr. Tony Atala, Director of the Wake Forest Institute for Regenerative Medicine. He said the term Alpha Clinic came from CIRM and the Alpha Stem Cell Clinic Network that we helped create. That network now has five specialist health care centers that deliver stem cell therapies to patients: UC San Diego, UCLA/UC Irvine, City of Hope, UC Davis, and  UCSF/Children’s Hospital Oakland.

This is a snapshot of that conversation.

Alpha Clinics Advancing Stem Cell Trials

Dr. Maria Millan, CIRM’s President & CEO:

“The idea behind the Alpha Stem Cell Clinic Network is that CIRM is in the business of accelerating treatments to patients with unmet medical needs. We fund research from the earliest discovery stage to clinical trials. What was anticipated is that, if the goal is to get these discoveries into the clinics then we’ll need a specific set of expertise and talents to deliver those treatments safely and effectively, to gather data from those trials and move the field forward. So, we set out to create a learning network, a sharing network and a network that is more than the sum of its parts.”

Dr. Joshua Hare,  Interdisciplinary Stem Cell Institute, University of Miami, said that idea of collaboration is critical to advancing the field:

 

“What we learned is that having the Alpha Stem Cell Clinic concept helps investigators in other areas learn from what earlier researchers have done, helping accelerate their work.

For example, we have had a lot of experience in working with rare diseases and we can use the experience we have in treating one disease area in working in others. This shared experience can help us develop deeper understanding in terms of delivering therapies and dosing.”

Susan Solomon, CEO New York Stem Cell Foundation Research Institute. NYSCF has several clinical trials underway. She says in the beginning it was hard finding reputable clinics that could deliver these potentially ground breaking but still experimental therapies:

 

“My motivation was born out of my own frustration at the poor choices we had in dealing with some devastating diseases, so in order to move things ahead we had to have an alpha clinic that is not just doing clinical trials but is working to overcome obstacles in the field.”

Greg Simon represented the, Biden Cancer Initiative, whose  mission is to develop and drive implementation of solutions to accelerate progress in cancer prevention, detection, diagnosis, research, and care, and to reduce disparities in cancer outcomes. He says part of the problem is that people think there are systems already in place that promote collaboration and cooperation, but that’s not really the case.  

 

“In the Cancer Moonshot and the Biden Cancer Initiative we are trying to create the cancer research initiative that people think we already have. People think doctors share knowledge. They don’t. People think they can just sign up for clinical trials. They can’t. People think there are standards for describing a cancer. There aren’t. So, all the things you think you know about the science behind cancer are wrong. We don’t have the system people think is in place. But we want to create that.

If we are going to have a unified system we need common standards through cancer research, shared knowledge, and clinical trial reforms. All my professional career it was considered unethical to refer to a clinical trial as a treatment, it was research. That’s no longer the case. Many people are now told this is your last best hope for treatment and it’s changed the way people think about clinical trials.”

The Process

Maria Millan says we are seeing these kinds of change – more collaboration, more transparency –  taking place across the board:

“We see the research in academic institutions that then moved into small companies that are now being approved by the FDA. Academic centers, in conjunction with industry partners, are helping create networks and connections that advance therapies.

This gives us the opportunity to have clinical programs and dialogues about how we can get better, how we can create a more uniform, standard approach that helps us learn from each trial and develop common standards that investigators know have to be in place.

Within the CIRM Alpha Stem Cell Clinic Network the teams coming in can access what we have pulled together already – a database of 20 million patients, a single IRB approval, so that if a cliinical trial is approved for one Alpha Clinic it can also be offered at another.”

Greg Simon says to see the changes really take hold we need to ensure this idea of collaboration starts at the very beginning of the chain:

“If we don’t have a system of basic research where people share data, where people are rewarded for sharing data, journals that don’t lock up the data behind a paywall. If we don’t have that system, we don’t have the ability to move therapies along as quickly as we could.

“Nobody wants to be the last person to die from a cancer that someone figured out a treatment for a year earlier. It’s not that the science is so hard, or the diseases are so hard, it the way we approach them that’s so hard. How do we create the right system?”

More may not necessarily be better

Susan Solomon:

“There are tremendous number of advances moving to the clinic, but I am concerned about the need for more sharing and the sheer number of clinical trials. We have to be smart about how we do our work. There is some low hanging fruit for some clinical trials in the cancer area, but you have to be really careful.”

Greg Simon

“We have too many bad trials, we don’t need more, we need better quality trials.

We have made a lot of progress in cancer. I’m a CLL survivor and had zero problems with the treatment and everything went well.

We have pediatric cancer therapies that turned survival from 10 % to 80%. But the question is why doesn’t more progress happen. We tend to get stuck in a way of thinking and don’t question why it has to be that way. We think of funding because that’s the way funding cycles work, the NIH issues grants every year, so we think about research on a yearly basis. We need to change the cycle.”

Maria Millan says CIRM takes a two pronged approach to improving things, renovating and creating:

“We renovate when we know there are things already in place that can be improved and made better; and we create if there’s nothing there and it needs to be created. We want to be as efficient as we can and not waste time and resources.”

She ended by saying one of the most exciting things today is that the discussion now has moved to how we are going to cover this for patients. Greg Simon couldn’t agree more.

“The biggest predictor of survivability of cancer is health insurance. We need to do more than just develop treatments. We need to have a system that enables people to get access to these therapies.”

Throwback Thursday: Progress towards a cure for HIV/AIDS

Welcome to our “Throwback Thursday” series on the Stem Cellar. Over the years, we’ve accumulated an arsenal of exciting stem cell stories about advances towards stem cell-based cures for serious diseases. Today we’re featuring stories about the progress of CIRM-funded research and clinical trials that are aimed at developing stem cell-based treatments for HIV/AIDS.

 Tomorrow, December 1st, is World AIDS Day. In honor of the 34 million people worldwide who are currently living with HIV, we’re dedicating our latest #ThrowbackThursday blog to the stem cell research and clinical trials our Agency is funding for HIV/AIDS.

world_logo3To jog your memory, HIV is a virus that hijacks your immune cells. If left untreated, HIV can lead to AIDS – a condition where your immune system is compromised and cannot defend your body against infection and diseases like cancer. If you want to read more background about HIV/AIDs, check out our disease fact sheet.

Stem Cell Advancements in HIV/AIDS
While patients can now manage HIV/AIDS by taking antiretroviral therapies (called HAART), these treatments only slow the progression of the disease. There is no effective cure for HIV/AIDS, making it a significant unmet medical need in the patient community.

CIRM is funding early stage research and clinical stage research projects that are developing cell based therapies to treat and hopefully one day cure people of HIV. So far, our Agency has awarded 17 grants totalling $72.9 million in funding to HIV/AIDS research. Below is a brief description of four of these exciting projects:

Discovery Stage Research
Dr. David Baltimore at the California Institute of Technology is developing an innovative stem cell-based immunotherapy that would prevent HIV infection in specific patient populations. He recently received a CIRM Quest award, (a funding initiative in our Discovery Stage Research Program) to pursue this research.

CIRM science officer, Dr. Ross Okamura, oversees Baltimore’s CIRM grant. He explained how the Baltimore team is genetically modifying the blood stem cells of patients so that they develop into immune cells (called T cells) that specifically recognize and target the HIV virus.

Ross_IDCard

Ross Okamura, PhD

“The approach Dr. Baltimore is taking in his CIRM Discovery Quest award is to engineer human immune stem cells to suppress HIV infection.  He is providing his engineered cells with T cell protein receptors that specifically target HIV and then exploring if he can reduce the viral load of HIV (the amount of virus in a specific volume) in an animal model of the human immune system. If successful, the approach could provide life-long protection from HIV infection.”

While Baltimore’s team is currently testing this strategy in mice, if all goes well, their goal is to translate this strategy into a preventative HIV therapy for people.

Clinical Trials
CIRM is currently funding three clinical trials focused on HIV/AIDS led by teams at Calimmune, City of Hope/Sangamo Biosciences and UC Davis. Rather than spelling out the details of each trial, I’ll refer you to our new Clinical Trial Dashboard (a screenshot of the dashboard is below) and to our new Blood & Immune Disorders clinical trial infographic we released in October.

dashboardblooddisorders

MonthofCIRM_BloodDisordersJustHIV.png

As you can see from these projects, CIRM is committed to funding cutting edge research in HIV/AIDS. We hope that in the next few years, some of these projects will bear fruit and help advance stem cell-based therapies to patients suffering from this disease.

I’ll leave you with a few links to other #WorldAIDSDay relevant blogs from our Stem Cellar archive and our videos that are worth checking out.

 

It’s time to vote for the Stem Cell Person of the Year

KnoepflerPaul14263

Paul Knoepfler

Oh well, it’s going to be another year of disappointment for me. Not only did I fail to get any Nobel Prize (I figured my blogs might give me a shot at Literature after they gave it to Bob Dylan last year), but I didn’t get a MacArthur Genius Award. Now I find out I haven’t even made the short list for the Stem Cell Person of the Year.

The Stem Cell Person of the Year award is given by UC Davis researcher, avid blogger and CIRM Grantee Paul Knoepfler. (You can vote for the Stem Cell Person of the Year here). In his blog, The Niche, Paul lists the qualities he looks for:

“The Stem Cell Person of the Year Award is an honor I give out to the person in any given year who in my view has had the most positive impact in outside-the-box ways in the stem cell and regenerative medicine field. I’m looking for creative risk-takers.”

“It’s not about who you know, but what you do to help science, medicine, and other people.”

Paul invites people to nominate worthy individuals – this year there are 20 nominees – people vote on which one of the nominees they think should win, and then Paul makes the final decision. Well, it is his blog and he is putting up the $2,000 prize money himself.

This year’s nominees are nothing if not diverse, including

  • Anthony Atala, a pioneering researcher at Wake Forest Institute for Regenerative Medicine in North Carolina
  • Bao-Ngoc Nguyen, who helped create California’s groundbreaking new law targeting clinics which offer unproven stem cell therapies
  • Judy Roberson, a tireless patient advocate, and supporter of stem cell research for Huntington’s disease

Whoever wins will be following in some big footsteps including patient advocates Ted Harada and Roman Reed, as well as scientists like Jeanne Loring, Masayo Takahashi,  and Elena Cattaneo.

So vote early, vote often.

LINK: Vote for the 2017 Stem Cell Person of the Year