The moment of truth. A video about the stem cell therapy that could help millions of people going blind.

“No matter how much one prepares, the first patient is always something very special.” That’s how Dr. Mark Humayun describes his feelings as he prepared to deliver a CIRM-funded stem cell therapy to help someone going blind from dry age-related macular degeneration (AMD).

Humayun, an ophthalmologist and stem cell researcher at USC, spent years developing this therapy and so it’s understandable that he might be a little nervous finally getting a chance to see if it works in people.

It’s quite a complicated procedure, involving turning embryonic stem cells into the kind of cells that are destroyed by AMD, placing those cells onto a specially developed synthetic scaffold and then surgically implanting the cells and scaffold onto the back of the eye.

There’s a real need for a treatment for AMD, the leading cause of vision loss in the US. Right now, there is no effective therapy for AMD and some three million Americans are facing the prospect of losing their eyesight.

The first, preliminary, results of this trial were released last week and they were encouraging. You can read about them on our blog.

Thanks to USC you can also see the team that developed and executed this promising approach. They created a video capturing the moment the team were finally taking all that hard work and delivering it where it matters, to the patient.

Watching the video it’s hard not to think you are watching a piece of history, something that has the potential to do more than just offer hope to people losing their vision, it has the potential to stop and even reverse that process.

The video is a salute to the researchers who developed the therapy, and the doctors, nurses and Operating Room team who delivered it. It’s also a salute to the person lying down, the patient who volunteered to be the first to try this. Everyone in that room is a pioneer.

Video illustrates potential path to stem cell repair for multiple sclerosis

“Can you imagine slowly losing the ability to live life as you know it? To slowly lose the ability to see, to walk, to grab an object, all the while experiencing pain, fatigue and depression?”

These sobering questions are posed at the beginning of a recent video produced by Youreka Science and Americans for Cures about multiple sclerosis (MS), a debilitating neurodegenerative disorder in which a person’s own immune system attacks cells that are critical for sending nerve signals from the brain and spinal cord to our limbs and the rest of our body.

In recognition of Multiple Sclerosis Awareness Week, today’s blog features this video. Using an easy to understand narrative and engaging hand-drawn illustrations, this whiteboard “explainer” video does a terrific job of describing the biological basis of multiple sclerosis. It also highlights promising research out of UC Irvine showing that stem cell-based therapies may one day help repair the damage caused by multiple sclerosis.

But don’t take my word for it, check out the five-minute video below:

Related Links:

Just a Mom: The Journey of a Sickle Cell Disease Patient Advocate [video]

Adrienne Shapiro will tell you that she’s just a mom.

And it’s true. She is just a mom. Just a mom who is the fourth generation of mothers in her family to have children born with sickle cell disease. Just a mom who was an early advocate of innovative stem cell and gene therapy research by UCLA scientist Dr. Don Kohn which has led to an on-going, CIRM-funded clinical trial for sickle cell disease. Just a mom who is the patient advocate representative on a Clinical Advisory Panel (CAP) that CIRM is creating to help guide this clinical trial.

She’s just a mom who has become a vocal stem cell activist, speaking to various groups about the importance of CIRM’s investments in both early stage research and clinical trials. She’s just a mom who was awarded a Stem Cell and Regenerative Medicine Action Award at last month’s World Stem Cell Summit. She’s just a mom who, in her own words, “sees a new world not just for her children but for so many other children”, through the promise of stem cell therapies.

Yep, she’s just a mom. And it’s the tireless advocacy of moms like Adrienne that will play a critical role in accelerating stem cell therapies to patients with unmet medical needs. We can use all the moms we can get.

Adrienne Shapiro speaks to the CIRM governing Board about her journey as a patient advocate

Patient’s Stage IV Cancer Held in Check by CIRM-Funded Clinical Trial [Video]

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Tom Howing

“In the last three scans, which I have every six weeks, they’re showing that there is no mestastasis (invasive cancer) anywhere in my body. [The doctors] I guess were quite blown away because they didn’t expect [the treatment] to be so quick or to be that complete.”

 

Today we’re sharing the story of Tom Howing, who took part in Forty Seven, Inc.’s CIRM-funded clinical trial that’s testing an innovative treatment for cancer.

The two-minute video below sums up Tom’s address to CIRM’s governing Board back in December. During his talk, he gave a personal perspective on his cancer diagnosis, the promising but ultimately disappointing results of standard anti-cancer treatments and the remarkable results he’s experienced from Forty Seven’s clinical trial.

Tom’s story is featured in our 2017 Annual Report (page 18), now available on our website.

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:

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.