Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. 

Stem cells in space. Since one of the first stories I ever wrote professionally was about how Grumman Corp. was building a beam builder that would be sent into space to build the international space station I could not resist this one. Even before John Glenn took that first orbit around the Earth, space scientists have worried about the impact of weightlessness on astronauts. Now some stem cells have taken up residence at the international space station to help answer that question.

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Stem cells need to get a grip. For some time it has become evident that the environment you place a stem cell in greatly influences what it decides to do when it grows up. Mesenchymal stem cells, for example, can become fat or bone among other tissues. Now a team at Penn has found that the choice between fat and bone depends on how well the stem cells can grip onto the media, on which they are growing. I wish a few of my fat cells would loose a grip.

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Making old blood young again. The blood-forming stem cells found in our bone marrow loose some of their vigor as we age. They just don’t make new copies of all the various components of our blood as well as they used to do. Now a team in Sweden has looked at the genetic switches that are intertwined in our DNA, and found that some of those switches have been altered in older blood stem cells. More important they found a way to change those switches back to a more youthful setting. In a few years, this could be better than the True Blood of vampire fame.

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Stem cell bank with a view. We announced a major new initiative last week to develop a stem cell bank that would house 9,000 reprogrammed stem cell lines, so called iPS cells, made from blood samples of 3,000 patients with 11 diseases that are believed to have genetic culprits. The bank is expected to provide new insight into the true causes of those disease and opportunities to find new therapies. The bank will be housed at the Buck Institute, which sits on a beautiful bluff in northern Marin county. The local paper published a story today with pictures of the empty labs waiting to be filled with bustling scientist, as well as the beautiful setting. I should note that those labs will be filled with folks hired by two companies CIRM lured to California from other states.

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15 stem cells in late-stage clinical trials. When stem cell research pushes a therapy into the clinic the cells need to pass through three phases, the first checks for safety, the second looks for early signs that the cells make a difference, and third is pivotal. That Phase 3 trial has to show definitively that the cells benefit the patient and do so better than any standard therapy. A blogger I follow is writing about the 15 stem cell trials in Phase 3. Here is his first installment.

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Questionable stem cells in Italy. The Italian minister of health riled scientists on both sides of the Atlantic last week when he allowed a clinic offering an unproven therapy to continue treating patients even though the stem cells involved are not manufactured according to Italy’s legal safety standards. It has stirred quite a debate about patients’ rights to determine the risk they will take. Here are two pieces: a short version from Reuters and a longer one from Nature. The comment field in the latter captures the two sides of the issue.

And to end with a fun note from Italy. Italian movie star and legendary sex symbol, Gina Lollobrigida is auctioning off her jewelry to fund a stem cell hospital and research center. She’s retired from acting and says that her current career as a sculptor does not require flashy jewelry. Growing up as a gay man I may not have appreciated her attributes as much as my straight male friends, but I did love her style and her bling, so I applaud her choice of where those baubles can do the most good.

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D.G.

When we first came up with the idea for the Elevator Pitch Challenge the idea was to videotape all the entries at a big meeting of researchers who get funding from us. But there’s always someone who has to be different, someone who doesn’t play by the rules. In this case it was the folks from USC. And I loved what they did.

Several researchers at USC did pre-recorded elevator pitches, videotaped on location at USC by their videographer Ryan Ball, next to a bank of elevators. But it’s how they did those pitches and livened them up that I really liked.

As someone who spent many years in TV news I loved the little edits that they threw in to help make the pitches visually more interesting. OK, so I know that you are not likely to have access to clever computer-generated graphics when you are explaining your work to someone in a grocery store or at a party, but who cares – this was a creative approach and one of the points about the Challenge was for researchers to think in creative ways how they can explain their work.

My favorite was Toshio Miki’s pitch because he began with a simple question; “Did you know stem cells can cure liver diseases? Yes they can.” Right away I wanted to know more. He then builds on that by talking about using human placenta cells that he says are “safe and clean as a newborn baby”, what a great verbal image, and then adds in a charming graphic that shows him throwing the cells at an obviously very sick cartoon liver that immediately perks up and looks well.

It’s a wonderfully simple and clear explanation of his work, supported with some clever graphics. And he does it all in 31 seconds.

In fact all the USC pieces came in almost dead on 30 seconds, or even under in a few cases.

Gage Crump was another favorite because he shot his in an elevator and clearly did it all in one single take, talking directly to the camera. Not an easy thing to do when explaining complex basic science. But Gage did it in a wonderfully engaging manner.

The USC team also employed a clever use of words printed on the screen to support the scientists in what they were saying. Done poorly that could have been distracting but instead this increased the effectiveness of the pitches – and I’m thinking in particular of the one done by Songtao Shi.

These pitches could easily stand alone without graphics. They are all good, clear explanations for the work being done. Adding the visual elements makes them even stronger and perfect for use in social media and online. And that’s ultimately the test of a message, how well does it connect with the audience. These will be able to connect with audiences anywhere and that’s why they are among my favorites.

K.M.

I have spent much of my career working with faculty at schools on both coasts, Stanford and Harvard, convincing them to rethink the way they talk about their science, insisting they make it more understandable to the public.

For many, I had my work cut out just convincing them this was worth the effort. But others seemed to immediately understand that if the public is funding their work through tax dollars, they have an obligation to explain why it matters in easily understandable language. Others were convinced of the value when, after a donor event, I could point to the one faculty member who accepted personal coaching and note that he was the only one to have a donor follow him back to his lab.

At CIRM, we seem to be lucky in having a large number of lead faculty researchers who get it. We had 28 agree to take us up on our challenge to describe their work on a lay level in just 30 seconds, which we dubbed The Elevator Pitch Challenge.

These brave faculty provided too many good examples for me to highlight them all here, but I do want to point out a few that cover some of my pet issues.

Gage Crump (faculty at University of Southern California)- Gage tackles something I have long fought to get the public to understand, the value of animal models. He briskly tells about zebra fish and their ability to regrow parts of their bodies. Then he transitions to the hope that from these little swimmers we could learn how to better repair our bodies. Here’s Gage’s pitch.

Darryl D’Lima (faculty at the Scripps Institute)- Darryl steps in immediately to deliver something I pound into faculty, perspective. He reminds viewers that arthritis impacts more people than heart disease or cancer. He talks about the potential to generate new cartilage and closes with more perspective: that this would be the first time that a treatment would change the progression of this chronic disease. Here’s Darryl’s pitch.

Deepak Srivastava (researcher at the Gladstone institutes, faculty at UC San Francisco)- Deepak gracefully delivers one of my favorite messages, that sometimes the best way to understand how to fix something is to understand how a normal version was created. After noting that adult hearts have very little ability to repair themselves, he talks about using stem cells to unlock the secrets of how hearts develop in a fetus and ends with the hope that this knowledge will guide us in repairing adult hearts. Here’s Deepak’s pitch.

There are many more examples that could be studied by those wanting to perfect the art of clarifying science, like those from Marius Wernig from Stanford University and Edward Hsiao from UCSF. The full roster of pitches can be found here.

D.G.

A year and two days ago, our grantees at Stanford University led by Irv Weissman described their work with a protein that’s found on the outside of cancer cells. They had devised an antibody that latches on to this protein and, in the process, brings those cancer cells to the attention of the immune system, which destroys them.

The work got a lot of attention at the time, and it got more attention yesterday when a reporter apparently misread the 2012 date on the Science paper that first reported the work. Yesterday’s New York Post carried the story, a year and a day after the paper was published rather than the usual one day later for news stories.

Despite that minor news goof, it was a good story explaining work we’re really excited about, hailing the potential therapy as both a “Holy Grail” and “miracle drug”.

Given that the work has yet to be tested in people we’re reluctant to call it a miracle drug, but the work is certainly promising. Weissman has a $20 million award from CIRM to develop the therapy.

The New York Post described the work like this:

The drug works by blocking a protein called CD47 that is essentially a “do not eat” signal to the body’s immune system, according to Science Magazine.

This protein is produced in healthy blood cells but researchers at Stanford University found that cancer cells produced an inordinate amount of the protein thus tricking the immune system into not destroying the harmful cells.

With this observation in mind, the researchers built an antibody that blocked cancer’s CD47 so that the body’s immune system attacked the dangerous cells.

As it so happens, Weissman recently described this research to us in an Elevator Pitch:

A.A.

If we build it, will they come?

That’s the question Amy, Kevin, and I (aka the CIRM film crew) were asking a few Thursday mornings ago in the lobby outside the CIRM Grantee Meeting. All systems were go with our makeshift film studio: camera on the tripod – check; microphones live – check; lighting in position – check; mural hung up against wall to prettify the background – check.

Waiting patiently for elevator pitch volunteers

Then, only crickets. Would not even one of the several hundred CIRM grantees in attendance participate in our Elevator Pitch Challenge? Hours seemed to go by, in reality probably many minutes. Suddenly a figure emerged from the throngs of CIRM scientists hanging out in the lobby during an intermission. It was a UCLA graduate student, William Kim. “Is this where I do the video pitch contest?” he asked.

Thank goodness for the enthusiasm of students. By taking that first plunge in front of the camera, I think William helped set off the avalanche of 43 pitches we would end up filming over two days (remarkably, 14 others also submitted their own videos). I particularly liked how William made a connection between his work to generate an unlimited supply of embryonic stem-cell derived red blood cells with the promise of one day helping soldiers in the field where blood is in short supply. (You can watch his video here.)

Time and time again, I’ve been so impressed and inspired by the undergraduates, graduates, postdocs, and even high school students that I’ve had the privilege to film over the past few years. The enthusiasm they show while explaining their research is contagious. Our elevator pitch interviews were no different. Of the 57 total pitches, 29 were in our non-lead scientist category (those just starting out or in the early days of their career). And though we only announced a few winners for the purposes of our contest I think there are several other non-lead scientists, like William, that deserve a shout-out:

Stefano DaSacco (Postdoctoral Fellow at Children’s Hospital, Los Angeles)– It was great to see Stefano’s friendly face again and to see that his excitement for stem cell research still thrives. Amy and I had filmed a short interview with him at the 2011 grantee meeting. At that time, I wasn’t able to use any of the footage in the final edit. While filming his video pitch this year, it was clear to Amy and me that he had improved his communication skills and he nailed his project aim of identifying an amniotic fluid-derived stem cell to generate kidney cells for development of a kidney failure therapy. He received several top votes from our judges. Here’s Stefano’s pitch.

Joseph Hargan Calvopiña (Graduate Student at UCLA)- I have to give Joseph props for taking on the challenge of explaining genetic imprinting in 30 seconds. Many of the other pitches were more straight-forward: “in disease x, cell type A is destroyed , our approach is to grow stem cell derived cell type A and put it back in the body to cure disease X.” Joseph didn’t have that luxury but clearly did a lot of preparation and was very open to our suggestions. In the end, his pitch was a bit over 30 seconds but he gave an easy to understand explanation of how genes from your mother vs. your father are distinguished through imprinting and the implications for disease when the imprinting goes awry. Here’s Joseph’s pitch.

Lina Nih (Postdoctoral Fellow at UCLA). Lina dropped by our film booth on the first day to watch her lab mate Jonathan Lam give what became the first place pitch in the non-lead scientist category. Lina seemed interested to give her own pitch but then we lost sight of her that day. I’m really glad she came back the next day because she delivered a very compassionate pitch about her research on stroke. Her opening was very compelling and made me want to hear more and I thought her conclusion was one of the most unique in that she spoke on a personal level to the public by saying: “and in the California Institute for Regenerative Medicine we believe in a better future where these [stem cell based stroke] treatments would be true and so we need you to believe in this too.” Here’s Lina’s pitch.

I also really enjoyed watching Youngtae Jeong from Stanford University give his pitch. He had clearly worked hard to come up with an analogy that would explain his research to a person who doesn’t understand the relationship between stem cells and cancer. That kind of creativity will serve him well when trying to communicate complex science.

I didn’t get to meet Victoria Bendersky from Scripps Research Institute because she recorded her pitch and sent it to us, but she also shows incredible passion for the kids who might benefit from her research. It’s nice to know that the people who will be leading stem cell labs in the future have such compassion for the patients they might end up helping.

I could tell you behind the scenes stories about all of the scientists who pitched at the meeting and I actually feel bad for leaving so many people out of this blog. I really do think everyone was a winner and we hope all of the pitchees gained a little more experience for their next elevator ride.

T.D.

Last week Bradley Fikes of the San Diego Union Tribune wrote about work by our grantee Jeanne Loring, who is helping to develop a therapy for Parkinson’s disease using funds raised by mountain climbing.

Many of her projects are funded by us (here’s a list of her awards), but this one is different. A group of 8 patients who wanted to work with Loring on this therapy are raising money through a project called Summit4StemCell, in which patients climb a mountain to raise funds. They’ve climbed Mount Kilimanjaro and in October plan to climb to base camp at Mount Everest.

Conveniently, Loring and another scientist on the project Suzanne Peterson both recently explained their work to us as part of our elevator pitch challenge, in which grantees give short descriptions of their work. (Peterson’s explanation came in second in the non-lead scientist competition.) Here’s the project in their own words:

This project is unusual in that patients are directly funding the development of therapies that they hope to benefit from. The more normal funding strategy is for patients to donate to their disease organization, like the Parkinson’s Association of San Diego, and that organization funds promising research.

Fikes goes on to write about this unusual situation:

For the patients, the project represents new hope. Loring’s researchers say they also benefit from actually getting to know the people their research is devoted to. It makes the science less abstract and more human.

“The patients and the researchers feed off each other,” said TSRI researcher Suzanne Peterson. “The patients get more hopeful, and the researchers get more motivated. And that’s such an unusual situation. We feel this responsibility to make this work for them. I think it’s nice to have that interaction between the patients and the researchers.”

The team still has a lot of hurdles ahead, as with all potential therapies involving transplanting stem cells into patients—whether they are embryonic or reprogrammed cells as in this case. The team needs to show the FDA that the approach could work and is safe, which can be time consuming and costly, and they have to carry out clinical trials to see if the cells actually treat the disease symptoms.

A.A.

Kevin McCormack explains the concept of the elevator pitch in our promo video

My colleague Amy Adams has created a monster. I don’t think she meant to, it just happened by accident. But truth be told, we’re rather happy she did.

It began innocently enough with an article in Nature about scientists trying to put together an “Elevator Speech” where they explain their work, what they do and why it’s important in two minutes or less. “Hey, we should do that with the researchers we fund,” Amy helpfully suggested.

One month, 2 days of shooting, one week of editing and 57 videos later the idea has taken on a life of it’s own, with stories in both the San Francisco Chronicle and ABC.

You can find out most of the details here in a news release about the event but basically our Elevator Pitch Challenge asked researchers who got stem cell agency funding to do a video in which they explained their work in plain English in as close to 30 seconds as they could. Some were really good at sticking to the time limit. Some were really good at speaking in everyday English. But only a very few were really good at both. And they emerged as the winners.

We divided the winners into two categories, Lead Scientists (those who have been doing research for some time) and non-Lead Scientists (those just starting out or in the early days of their career)

Here they are:

Non-Lead Scientist
1st. Jonathan Lam  (UCLA, stroke)
2nd. Suzanne Peterson (Scripps Research Institute, Parkinson’s)
3rd. Michael Rothenberg (Stanford, intestinal cancer)
3rd. Anica Sayoc (City of Hope/CSU Long Beach, leukemia)

Lead Scientist
1st. Amy Sprowles (Humboldt State U., cancer/Bridges Program)
2nd. John Zaia (City of Hope, HIV/AIDs)
3rd. Paul Knoepfler (UC Davis, cancer stem cells/epigenetics)
The prizes for winning aren’t quite on par with our funding awards, but the winners in both categories do at least get to choose between a $50 gift certificate from Amazon or Starbucks.

One pitch also deserved a special award category unto itself:

Most creative and original
Asaf Presente (UCSD, lung disease)

We have to point that Asaf is a seasoned comedian who has dabbled in improv. How lucky are we that he chose stem cells over showbiz?

Because there were so many good videos we are going to post some follow-up blogs in the coming days with staff here at the agency identifying their particular favorites. You can also get more information about the challenge, the winners and some of our favorite runners up on our website.

Amy’s monster just won’t die.

K.M.

Frozen iPS cells, Photo courtesy of William Collins in the lab of Deepak Srivastava at the Gladstone Institutes

When the discovery of reprogrammed stem cells, so-called iPS cells, won the Nobel Prize last October, these versatile cells were generally portrayed as offering the promise of changing medicine in the distant future. But in reality they are having an impact today. This is not through cells being given to patients but rather as research tools helping to find new drugs and to sort out toxic from non-toxic drug candidates.

My colleagues and I have written about using iPS cells as disease-in-a-dish models before (see blog entries here). Now, a CIRM-funded team at Stanford has proven that heart cells grown from iPS cells can be used to detect drug toxicity.

Experts have assumed this would be possible, and most major drug companies are investing in iPS research assuming it is possible. Here we have a great example of a known drug toxicity being verified through a disease-in-a-dish.

The team lead by Joseph Wu took skin samples from three family members who had inherited a faulty heart rhythm and one who did not inherit the defect. They made iPS cells from those skin samples and turned those into beating heart tissue in a dish. There they saw differences in function between those cells with the defect and the ones without it.

They then tested three drugs on these cells, one that has been shown to be safe, one that has some toxicity in certain patients, and one that was pulled from the market in 2000 because it was causing deaths. In each case the cells in the dish verified what had been seen in patients. But with the sometimes-lethal drug, they were able to show that the effect depended on dose. This information could have given the manufacturer the chance to alter the way the drug was used and prevent the deaths.

A press release from Stanford quoted Wu about the research that was published in Circulation:

“This study shows that the use of patient-specific stem cells to detect cardiotoxic properties of pharmaceutical compounds may be more accurate than the current drug-safety assays mandated by the FDA. We are also able to demonstrate disease-specific responses to cardiotoxic drugs. We believe that, in the future, this may become a standard way to test drug safety and efficacy.”

The Stanford team speculated that this use of iPS cells could save millions of dollars in developing new drugs. This is one reason why CIRM invested $32 million last week to develop an iPS cell bank that will have some 9,000 cells lines from 3,000 patients with 11 different diseases (we wrote about that initiative here). Wu, who led the research, received one of the awards to develop cell lines that we’ll be storing in the bank.

D.G.

New research from our grantees at Stanford harkens back to my own days in graduate school. I was toiling long hours in a lab trying to understand what makes cells become different from each other.

I mean think about it. One day, an animal is just a fertilized egg. Then it divides into two cells, both with the same DNA, and you’d think with all the same cellular juices from that original egg. And those divide. And those divide. And somewhere along the way, one of those cells up and becomes different. And the cells around it become different. And before you know it the cells are organized into a front and back, left and right, and specialized cells like skin, neurons, and muscle. How crazy is that?

The question isn’t just navel-gazing. Answering it could also help us treat diseases that occur when the cells don’t quite get around to being different.

For my tastes, the question was more interesting than the lab work, which is why I turned to writing. But thankfully there are others still at it, including our grantee Roel Nusse at Stanford University. He turned his (and his lab’s) attention to understanding how a single embryonic stem cell in a lab dish divides and eventually produces cells that are different from itself. This is a critical step for all those researchers who are working to grow stem cells into different cell types for use in treating or studying disease.

A press release from Stanford quotes Nusse talking about his work, which was just published in Science:

“In the body, it is likely that every cell grows and differentiates in some kind of orientation,” said Roeland Nusse, PhD, professor of developmental biology. “Without this guidance, specialized cells would end up in the wrong place. Now, we can study the division of single mammalian cells in real time and see them dividing and differentiating in an oriented way.”

Stanford’s release includes a nice description of exactly how Nusse and his team carried out the painstaking work. It involved coating tiny beads with a protein that’s known to prod stem cells to divide, then attaching the bead to a specific spot on the cell. They could then watch, in real time, how the cell divided in relation to the bead.

It’s meticulous work, and will likely be important for future stem cell therapies. But it makes me glad I wasn’t the one carefully placing that bead on the cell, or sitting there watching it divide (paint dries about as quickly).

A.A.

 CIRM funding: Roel Nusse (RB4-05825, TR1-01249)

Habib, S., Chen, B., Tsai, F., Anastassiadis, K., Meyer, T., Betzig, E., & Nusse, R. (2013). A Localized Wnt Signal Orients Asymmetric Stem Cell Division in Vitro Science, 339 (6126), 1445-1448 DOI: 10.1126/science.1231077

A few weeks ago we announced our CIRM Elevator Pitch Challenge. In the video above you can see our own Kevin McCormack explaining the challenge–each scientist has to explain their research in 30 seconds or less in a way that a lay person would understand.

We ended up with 58 pitches, many recorded at our grantee meeting and several sent in from people who weren’t going to be attending. You can see all of the videos here.

Entries ranged widely in both length and content. Some managed to be short and to the point, others were longer, or used creative approaches to communicating the science (and here I’m thinking specifically of Asad Presente of UCSD). Several people from University of Southern California pre-recorded their pitches with the help of USC videographer Ryan Ball, who added some clever graphics to a few videos. I admit to a soft spot for an ailing liver in an entry by Toshio Miki.

Our elite panel of judges met at CIRM headquarters yesterday. Erin Allday of the San Francisco Chronicle attended the judging and wrote this story about it.

After this experience, I have more sympathy for Academy Award judges. How do you rank such vastly different entries? Paul Knoepfler of UC Davis wrote about his top picks in a blog entry today. He very politely left out his own rather creative entry in his top picks.

We’ll be announcing the winners next week. In the mean time, I’d be interested in hearing other people’s top picks. For those on Twitter, you can also follow the conversation about these videos at #sciencepitch.

(3/22/13) After posting this piece yesterday, a story by an ABC reporter who also attended the judging came out. The story gives a nice sense of the mood of the room during the judging. I think we all had fun watching our grantees struggle to condense years of painstaking work into a few simple sentences.

A.A.