CIRM interviews Lorenz Studer: 2017 recipient of the Ogawa-Yamanaka Stem Cell Prize [Video]

For eight long years, researchers who were trying to develop a stem cell-based therapy for Parkinson’s disease – an incurable movement disorder marked by uncontrollable shaking, body stiffness and difficulty walking – found themselves lost in the proverbial wilderness. In initial studies, rodent stem cells were successfully coaxed to specialize into dopamine-producing nerve cells, the type that are lost in Parkinson’s disease. And further animal studies showed these cells could treat Parkinson’s like symptoms when transplanted into the brain.

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Lorenz Studer, MD
Photo Credit: Sloan Kettering

But when identical recipes were used to make human stem cell-derived dopamine nerve cells the same animal experiments didn’t work. By examining the normal developmental biology of dopamine neurons much more closely, Lorenz Studer cracked the case in 2011. Now seven years later, Dr. Studer, director of the Center for Stem Cell Biology at the Memorial-Sloan Kettering Cancer Center, and his team are on the verge of beginning clinical trials to test their Parkinson’s cell therapy in patients

It’s for these bottleneck-busting contributions to the stem cell field that Dr. Studer was awarded the Gladstone Institutes’ 2017 Ogawa-Yamanaka Stem Cell Prize. Now in its third year, the prize was founded by philanthropists Hiro and Betty Ogawa along with  Shinya Yamanaka, Gladstone researcher and director of the Center for iPS Cell Research and Application at Kyoto University, and is meant to inspire and celebrate discoveries that build upon Yamanaka’s Nobel prize winning discovery of induced pluripotent stem cells (iPSCs).

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(L to R) Shinya Yamanaka, Andrew Ogawa, Deepak Srivastava present Lorenz Studer the 2017 Ogawa-Yamanaka Stem Cell Prize at Gladstone Institutes. Photo Credit: Todd Dubnicoff/CIRM

Studer was honored at the Gladstone in November and presented the Ogawa-Yamanka Stem Cell Prize Lecture. He was kind enough to sit down with me for a brief video interview (watch it below) a few minutes before he took the stage. He touched upon his Parkinson’s disease research as well as newer work related to hirschsprung disease, a dangerous intestinal disorder often diagnosed at birth that is caused by the loss of nerve cells in the gut. Using human embryonic stem cells and iPSCs derived from hirschsprung patients, Studer’s team has worked out the methods for making the gut nerve cells that are lost in the disease. This accomplishment has allowed his lab to better understand the disease and to make solid progress toward a stem cell-based therapy.

His groundbreaking work has also opened up the gates for other Parkinson’s researchers to make important insights in the field. In fact, CIRM is funding several interesting early stage projects aimed at moving therapy development forward:

We posted the 8-minute video with Dr. Studer today on our official YouTube channel, CIRM TV. You can watch the video here:

And for a more detailed description of Studer’s research, watch Gladstone’s webcast recording of his entire lecture:

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Giving thanks to Caleb and all of our stem cell pioneers [Video]

For our last blog before the Thanksgiving holiday, we give thanks to the patients and their caregivers who are forging a path toward a new era of regenerative medicine therapies through their participation in CIRM-funded clinical trials.

Some of our trials are in the early stages which means they are mainly focused on safety. Participants go into these trials knowing that the cell therapy dose they receive will probably be too low to get any benefit for themselves. And in later trials, some patients will receive a placebo, or blank therapy, for comparison purposes. Even if a patient gets an effective dose, it may not work for them. So the decision to enroll in an experimental clinical trial is often a selfless act. Yet final approval of a therapy by the U.S. Food and Drug Administration (and other regulatory agencies around the world) depends on these brave souls and for that we are truly grateful.

So, with this Thanksgiving Day spirit in mind, we leave you with our latest video featuring Caleb Sizemore, a charming young man who epitomizes the courage of our clinical trial pioneers. At just 7 years old, Caleb was diagnosed with Duchenne Muscular Dystrophy (DMD), a degenerative muscle disease which makes it difficult for him to walk and climb stairs, has led to dangerous scarring of his heart muscle and carries a shortened life expectancy with most DMD patients not living past their 20s or 30s.

In a sit-down interview with us and in clips from his June 2017 presentation to the CIRM governing Board, Caleb talked about the impact of DMD on his life and his experience enrolling in Capricor Therapeutics’ CIRM-funded clinical trial. The trial is testing a stem cell therapy designed to repair the heart scarring that occurs with DMD. By the end of the three-minute video, I can assure you that you’ll be as captivated as we were by Caleb’s delightful, sincere and full-of-faith personality.

Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]

During this second week of the Month of CIRM, we’ve been focusing on the people who are critical to accomplishing our mission to accelerate stem cell treatments to patients with unmet medical needs.

These folks include researchers, like Clive Svendsen and his team at Cedars-Sinai Medical Center who are working tirelessly to develop a stem cell therapy for ALS. My colleague Karen Ring, CIRM’s Social Media and Website Manager, featured Dr. Svendsen and his CIRM-funded clinical trial in Monday’s blog. And yesterday, in recognition of Stem Cell Awareness Day, Kevin McCormack, our Senior Director of Public Communications, blogged about the people within the stem cell community who have made, and continue to make, the day so special.

Today, in a new video, I highlight a brave young patient, Ronnie, and his parents who decided to participate in a CIRM-funded clinical trial run by St. Jude Children’s Research Hospital and UC San Francisco in an attempt to save Ronnie’s life from an often-fatal disease called severe combined immunodeficiency (SCID). This disorder, also known as bubble baby disease, leaves newborns without a functioning immune system which can turn a simple cold into a potentially deadly infection.

Watch this story’s happy ending in the video above.

For more details about all CIRM-funded clinical trials, visit our clinical trials page and read our clinical trials brochure which provides brief overviews of each trial.

How a funny-looking creature could unlock the secrets of limb regeneration

The axolotl, also known as the Mexican salamander

In the world of funny-looking creatures, the Axolotl would have to rank in the top ten alongside such notables as the naked mole rat and the blob fish (the official mascot for the Ugly Animal Preservation Society). But the Axolotl does have one attribute that makes it attractive to more than just another Axolotl. That’s because this Mexican salamander has the ability to regenerate entire limbs.

Now, even as you read this, many stem cell researchers are hard at work trying to figure out ways to regenerate damaged or diseased tissues and organs in humans. That’s why the Axolotl is so intriguing. If we can understand how they are able to repair their own damaged limbs, maybe we can use that knowledge to help people repair or even replace a lost finger, hand or arm.

It’s a fascinating idea and one that is explored in this video from STAT, an online publication produced by the Boston Globe, that explores science and health.

It’s only four minutes long and is definitely worth watching. It shows that there is beauty in even the strangest creatures, if only you know what to look for.

Unfolding Collaboration: New EuroStemCell video about promoting public engagement around stem cells

What does origami have to do with stem cells? Scientists at EuroStemCell, which is a partnership of more than 400 stem cell labs across Europe, are using origami and other creative activities to engage and educate the public about stem cells.

EuroStemCell’s goal is to “make sense of stem cells” by providing “expert-reviewed information and road-tested educational resources on stem cells and their impact on society.” Their educational resource page is rich with science experiments for kids, students and even adults. They also have science videos on topics ranging from what stem cells are to bioengineering body parts.

Unfolding Organogenesis

Recently EuroStemCell posted a video about how successful public engagement activities are based on strong collaborations between scientists, doctors, educators and communicators. This video was particularly powerful because it showed how good ideas can start from an individual, but great ideas happen when individuals work together to develop these initial ideas into activities that will really connect with their audience.

The video features Dr. Cathy Southworth who begins by telling the story of how she and her collaborators developed an origami activity called “Unfolding Organogenesis”. Southworth explains her rationale behind using paper to simulate how stem cells develop the tissues and organs in our body.

“I was mulling how to use a prop or activity to talk about stem cells, and it suddenly came to me that paper and origami is a bit like the process. The whole idea of starting from a blank slate. Depending on the instructions you follow, makes a different object. If you start with a stem cell, you can make any type of cell you find in the body. And that made me think it was quite a nice analogy to talk to the public about.”

Her initial idea was made a reality when Southworth began working with science and math educators Karen Jent and Tung Ken Lam. Together the team developed an interactive activity where people used paper to build 3D hearts that can actually beat.

Ken Lam making organ origami.

Southworth said that as a science communicator, educating the public is the focus of her work. But she also believes that educating scientists on how to communicate with the public effectively is equally important.

“Part of my job is to make sure that the scientists feel confident in the activities that they are going to deliver, and also that they are having a good time as part of the engagement work.”

The video also touches on important science communications tips like teaching scientists the art of storytelling. Southworth emphasized that having scientists talk about their personal story of why they are pursuing their research adds a human component that is key to connecting with their audience. Karen Jent also added that it’s important to understand your audience and their needs,

“You always have to think about what kind of audience you’re addressing and bear in mind that people aren’t all the same kinds of learners.”

Where are my stem cells?

CIRM is also dedicated to educating the public about stem cells and the importance of stem cell research. We have our own educational resources on our website, but we love to use materials from other organizations like EuroStemCell in our public engagement activities.

One of our favorite public engagement events is the Bay Area Science Festival Discovery Day held at AT&T park. This event attracts over 50,000 people, mainly young kids and their parents who are excited to learn about science and technology. At our booth, we’ve done a few different activities to teach kids about stem cells. One activity, which is great for young kids, is using Play-Doh to model embryonic development.

Teaching kids about embryonic development with Play-Doh! Photo: Todd Dubnicoff/CIRM

Another fun activity, this one developed by EuroStemCell, that we added last year was called “Where are my stem cells?”. It’s a game that teaches people that stem cells aren’t just found in the developing embryo. You’re given laminated cutouts of human organs and tissues, which you’re asked to place on a white board that has an outline of your body. While you are doing this, you learn that there are different types of adult stem cells that live in these tissues and organs and are responsible for creating the cells that make up those structures.

Where are your stem cells? A fun activity designed by EuroStemCell. Photo: Todd Dubnicoff/CIRM

If you’re interested in doing public engagement activities around stem cell education, the resources mentioned in this blog are a great start. I’d also recommend checking out the Super Cells, Power of Stem Cells exhibit, which is touring Europe, USA and Canada. It’s a wonderful interactive exhibit that explains the concept of stem cells and how they can be used to understand and treat disease. It’s also a great example of a collaboration between stem cell organizations including CIRM, CCRM, EuroStemCell, Catapult Cell Therapy and the Stem Cell Network.

We got a chance to check out the Super Cells exhibit last year when it visited the Lawrence Hall of Science in Berkeley. You can read more about it and see pictures in our blog.

Super Cells Exhibit. Photo: Todd Dubnicoff/CIRM

 

4 things to know about stem cell clinical trials [Video]

Every day, we receive phone calls and emails from people who are desperately seeking our help. Sometimes they reach out on their own behalf, though often it’s for a family member or close friend. In every case, someone is suffering or dying from a disorder that has no available cure or effective treatment and they look to stem cell treatments as their only hope.

It’s heartbreaking to hear these personal stories that are unfolding in real time. Though they contact us from a wide range of places about a wide range of disorders, their initial set of questions are often similar and go something like this:

  • “Where can I find stem cell clinical trial for my condition?”
  • “What are my chances of being cured?”
  • “How much does it cost to be in a clinical trial?”
  • “How can I be sure it’s safe?”

We think anyone thinking about taking part in a clinical trial should consider these important questions. So, in addition to providing answers as we receive them through phone calls and emails, we wanted to find a way to reach out to as many people as possible. The result? The four-minute animation video you can watch below:

As mentioned in the video, the answers to these questions are only the tip of the iceberg for finding out if a particular clinical trial is right for you. The website, A Closer Look at Stem Cells, produced by the International Society for Stem Cell Research (ISSCR), is an excellent source for more advice on what things you should know before participating in a stem cell clinical trial or any experimental stem cell treatment.

And visit the Patient Resources section of our website for even more practical information including our growing list of CIRM-funded clinical trials as well as trials supported by our Alpha Stem Cell Clinic Network.

Here’s a Fun Lab Tour Video Contest for Scientists

Scientists are often stereotyped as serious, focused individuals who spend most of their time pursuing their science with little time for anything else. Their research often is complex and hard for non-scientists to wrap their minds around. I’ve often heard my friends describe to me what they thought I did every day when I was in the lab. It was like a science fantasy story involving beakers full of brightly colored chemicals, explosions, and at the end, a cure for Parkinson’s disease…

But I am going to tell you a little known secret: scientists are normal people like everyone else. They aren’t magicians with special powers, and they know how to have fun while doing their research. The problem is that the public doesn’t know this because they don’t have the opportunity to visit a research laboratory and see scientists in action.

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Paul Knoepfler

UC Davis Professor Paul Knoepfler is addressing this issue with his new lab tour video contest that he recently announced on his blog, The Niche. He’s asking scientists to make short videos of their daily lives in the lab and post them on Twitter with the hashtag #labvideocontest. The winner will receive a cash prize and “free PR for their lab”. The videos can be serious or funny, but Paul asks contestants to use their imagination and think out of the box.

This contest will not only be a fun way for scientists to talk about their research and what they do every day, but it will also benefit the public who will get an inside view of what it’s like to be a scientist. The goal of science communications is to make science relatable to everyone, and this video contest on social media is a great example of new ways that scientists can connect with the public and make science more approachable.

You scientists out there can learn more about Paul’s contest and how to participate on his blog. The deadline to submit lab videos is March 15th, so you better get to work!

And if you need a place to start, watch our recent video featuring the McDevitt lab, a stem cell bioengineering lab at the Gladstone Institutes.

What’s Your 2017 Stem Cell Resolution?

January marks the beginning of a new year and is typically a time when people make resolutions to better themselves. This year at CIRM, we’re shaking things up and making stem cell resolutions.

What’s your #StemCellResolution?

Our goal is to raise awareness about the importance of funding stem cell research and accelerating the development of safe and effective stem cell treatments for patients. We want to promote this goal not only within the scientific and patient communities but also within the general public.

That’s why we are challenging you (yes you the reader) to come up with your own stem cell resolution for 2017 and share it with us on social media during the month of January.

It’s easy and fun to participate. All you need to do is think of a resolution about stem cell research. If you’re a scientist, it could be making a resolution to apply for funding for your newest stem cell project. Don’t know anything about stem cells? How about making a resolution to learn about stem cell research for a specific disease? The options are endless!

After you decide on your resolution, you can post a selfie, video, or stem cell resolution graphic that we’ve designed (available on our website  https://www.cirm.ca.gov/stemcellresolution) on Instagram, Facebook, or Twitter. Make sure to write your resolution in your post, include the hashtag #stemcellresolution, and tag CIRM’s social media accounts.

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Have more than one resolution? No problem! Feel free to post as many stem cell resolutions during January as you want. We also encourage you to share this campaign with your friends and challenge them to participate.

Check out our video for more details on how to participate:

There be prizes!

At the end of January, we will pick the most inspiring stem cell resolutions and blog about them on the Stem Cellar. We’ll also send the people who wrote those resolutions CIRM Stem Cell Champions t-shirts.

So, what are you waiting for? We want to hear from you!

Easier, Cheaper Stem Cell-Based Heart Muscle Sets Stage for Large-Scale Drug Development

The great inventions – like the automobile, the Internet or aviation – are marked as important turning points in human history. But it’s usually the additional tinkering that goes on in the ensuing years after the initial invention that makes the technology feasible in terms of cost, reproducibility and mass production.

The same holds true for the Nobel prize winning induced pluripotent stem cell (iPS) technique. The sight of human iPS-derived heart muscle cells, or cardiomyocytes, beating in a petri dish brought a lot of early excitement in the late 2000’s (and still does today) about the potential of using human cells rather than animal models to screen for novel heart disease therapies and to test for drug toxicity.

And since iPS cells can be created directly from the skin or blood of a heart disease patient, it opened new opportunities to better understand the cellular basis of heart disease.

The Earth isn’t flat and neither is the heart
But the human heart is more than a two dimensional layer of cardiomyocytes in a petri dish. As a result, more complex three dimensional miniature heart structures that better mimic cardiac function have been developed over time. Now a CIRM-funded research team at the Gladstone Institutes has gone a step further and devised a “Micro-Heart Muscle” (µHM) technique that is easy to make and uses much fewer cells. The method detailed in Scientific Reports yesterday is poised to make large-scale, high-throughput drug and toxicology testing for heart disease therapies a reality.

Prior to this current publication, the Engineered Heart Muscle (EHM) has been the gold standard for petri dish models of human heart function. To better reflect the cellular environment of heart tissue than a simple layer of heart cells, EHM is composed of a mixture of iPS-derived cardiomyocytes, fibroblasts, and extracellular matrix, a natural scaffold that supports the heart’s cellular structure. These components are grown into 3D molds in a lab dish and embedded into posts that give the muscle cells something to contract against. This setup provides a means to do detailed analysis of the impact of drugs on heart muscle function. While these miniature EHM structures successfully produce physiologically relevant heart tissue for testing in the lab, they nonetheless carry some practical limitations. The complexity of the molds and the need for millions of cells for each EHM tissue makes the cost of large-scale drug development experiments too high. And the use of extracellular matrix would muck up the miniaturized instrumentation that is used for these therapy development efforts.

Dog bones: the key to easier, cheaper muscle tissue
The Gladstone team, led by Bruce Conklin, has overcome these challenges with their Micro-Heart Muscle product. With some educated trial and error, they zeroed in on a simple dog bone shaped mold to grow the mixture of cells in. The shape of the mold encouraged the muscle cells to self organize and grow into contracting tissue without the need of extracellular matrix. And better yet, less than 10,000 cells were needed to form each tissue. Here’s a cool video, recorded by first author Nathaniel Huebsch, of the cells in action:

These technical improvements of the Micro-Heart Muscle could help make large-scale, systematic approaches to study heart disease and toxicology a reality. As you read Conklin’s summary of the results in a Gladstone press release, you can hear his excitement about the future applications of this method:

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Bruce Conklin, Gladstone Institutes Senior Investigator

“The beauty of this technique is that it is very easy and robust, but it still allows you to create three-dimensional miniature tissues that function like normal tissues. Our research shows that you can create these complex tissues with a simple template that exploits the inherent properties of these cells to self-organize. We think that the micro heart muscle will provide a superior resource for conducting research and developing therapies for heart disease.”

 

Micro-Heart Muscle: the Model T of iPS innovations?
Karl Benz is generally credited as the inventor of the automobile, but a few years later it was Henry Ford’s efficient assembly line and manufacturing process that helped make mass production and affordability of the car possible. In a somewhat similar way, Shinya Yamanaka’s iPS technique will no doubt go down as one of the greatest inventions in the 21st century and maybe Conklin’s team’s Micro-Heart Muscle will also feature prominently in the history books as a follow up innovation that made the development of heart disease therapies possible.

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Senior author Bruce Conklin and first author Nathaniel Huebsch [Photo: Chris Goodfellow]

Growing Stem Cell Research in California (Video)

How a Gladstone scientist is using bioengineering to push the pace of stem cell research

At CIRM, we strive to fund the most promising stem cell research and speed the advancement of stem cell treatments to patients who need them. Because we are a state agency, we generally focus on funding scientists, universities, and companies located in California. But we recognize that high quality stem cell research is ongoing throughout the country. That’s why CIRM has programs that fund research originating outside California and that recruit talented stem cell scientists to join our state’s vibrant stem cell community.

Today we want to share a video we produced titled, “Growing Stem Cell Research in California” that provides an example of how CIRM has catalyzed the growth of stem cell research by helping recruit Dr. Todd McDevitt, a leading biomedical engineer in stem cell research, to the Gladstone Institutes in San Francisco.

Todd started his lab at the Georgia Institute of Technology in Atlanta and moved to the Gladstone a year ago to conduct research using human pluripotent stem cells to engineer 3D micro-tissues for use in drug development and disease modeling. His move was made possible by a CIRM Research Leadership Award, which allowed the Gladstone to recruit Todd and is now his lab’s major source of funding.

Todd McDevitt, Gladstone Institutes

Todd McDevitt, Gladstone Institutes

With an expertise in tissue engineering, Todd and his team are collaborating with other researchers at the Gladstone on projects that use human stem cells to create organ-like tissues to advance research and therapeutic development for a wide range of areas including brain disease, heart disease and spinal cord injury.

Todd is a young and talented scientist who is using his expertise in bioengineering to push the pace of stem cell research ultimately, we hope, to improve human health. You can read more about Todd’s first year anniversary at the Gladstone in their latest news release.