CIRM weekly stem cell roundup: stomach bacteria & cancer; vitamin C may block leukemia; stem cells bring down a 6’2″ 246lb football player

gastric

This is what your stomach glands looks like from the inside:  Credit: MPI for Infection Biology”

Stomach bacteria crank up stem cell renewal, may be link to gastric cancer.

The Centers for Disease Control and Prevention estimate that two-thirds of the world’s population is infected with H. pylori, a type of bacteria that thrives in the harsh acidic conditions of the stomach. Data accumulated over the past few decades shows strong evidence that H. pylori infection increases the risk of stomach cancers. The underlying mechanisms of this link have remained unclear. But research published this week in Nature suggests that the bacteria cause stem cells located in the stomach lining to divide more frequently leading to an increased potential for cancerous growth.

Tumors need to make an initial foothold in a tissue in order to grow and spread. But the cells of our stomach lining are replaced every four days. So, how would H. pylori bacterial infection have time to induce a cancer? The research team – a collaboration between scientists at the Max Planck Institute in Berlin and Stanford University – asked that question and found that the bacteria are also able to penetrate down into the stomach glands and infect stem cells whose job it is to continually replenish the stomach lining.

Further analysis in mice revealed that two groups of stem cells exist in the stomach glands – one slowly dividing and one rapidly dividing population. Both stem cell populations respond similarly to an important signaling protein, called Wnt, that sustains stem cell renewal. But the team also discovered a second key stem cell signaling protein called R-spondin that is released by connective tissue underneath the stomach glands. H. pylori infection of these cells causes an increase in R-spondin which shuts down the slowly dividing stem cell population but cranks up the cell division of the rapidly dividing stem cells. First author, Dr. Michal Sigal, summed up in a press release how these results may point to stem cells as the link between bacterial infection and increased risk of stomach cancer:

“Since H. pylori causes life-long infections, the constant increase in stem cell divisions may be enough to explain the increased risk of carcinogenesis observed.”

vitamin-c-1200x630

Vitamin C may have anti-blood cancer properties

Vitamin C is known to have a number of health benefits, from preventing scurvy to limiting the buildup of fatty plaque in your arteries. Now a new study says we might soon be able to add another benefit: it may be able to block the progression of leukemia and other blood cancers.

Researchers at the NYU School of Medicine focused their work on an enzyme called TET2. This is found in hematopoietic stem cells (HSCs), the kind of stem cell typically found in bone marrow. The absence of TET2 is known to keep these HSCs in a pre-leukemic state; in effect priming the body to develop leukemia. The researchers showed that high doses of vitamin C can prevent, or even reverse that, by increasing the activity level of TET2.

In the study, in the journal Cell, they showed how they developed mice that could have their levels of TET2 increased or decreased. They then transplanted bone marrow with low levels of TET2 from those mice into healthy, normal mice. The healthy mice started to develop leukemia-like symptoms. However, when the researchers used high doses of vitamin C to restore the activity levels of TET2, they were able to halt the progression of the leukemia.

Now this doesn’t mean you should run out and get as much vitamin C as you can to help protect you against leukemia. In an article in The Scientist, Benjamin Neel, senior author of the study, says while vitamin C does have health benefits,  consuming large doses won’t do you much good:

“They’re unlikely to be a general anti-cancer therapy, and they really should be understood based on the molecular understanding of the many actions vitamin C has in cells.”

However, Neel says these findings do give scientists a new tool to help them target cells before they become leukemic.

Jordan reed

Bad toe forces Jordan Reed to take a knee: Photo courtesy FanRag Sports

Toeing the line: how unapproved stem cell treatment made matters worse for an NFL player  

American football players are tough. They have to be to withstand pounding tackles by 300lb men wearing pads and a helmet. But it wasn’t a crunching hit that took Washington Redskins player Jordan Reed out of the game; all it took to put the 6’2” 246 lb player on the PUP (Physically Unable to Perform) list was a little stem cell injection.

Reed has had a lingering injury problem with the big toe on his left foot. So, during the off-season, he thought he would take care of the issue, and got a stem cell injection in the toe. It didn’t quite work the way he hoped.

In an interview with the Richmond Times Dispatch he said:

“That kind of flared it up a bit on me. Now I’m just letting it calm down before I get out there. I’ve just gotta take my time, let it heal and strengthen up, then get back out there.”

It’s not clear what kind of stem cells Reed got, if they were his own or from a donor. What is clear is that he is just the latest in a long line of athletes who have turned to stem cells to help repair or speed up recovery from an injury. These are treatments that have not been approved by the Food and Drug Administration (FDA) and that have not been tested in a clinical trial to make sure they are both safe and effective.

In Reed’s case the problem seems to be a relatively minor one; his toe is expected to heal and he should be back in action before too long.

Stem cell researcher and avid blogger Dr. Paul Knoepfler wrote he is lucky, others who take a similar approach may not be:

“Fortunately, it sounds like Reed will be fine, but some people have much worse reactions to unproven stem cells than a sore toe, including blindness and tumors. Be careful out there!”

 

Stem Cell Roundup: Battle of the Biotech Bands, “Cells I See” Art Contest and Teaching Baseball Fans the Power of Stem Cells

This Friday’s stem cell roundup is dedicated to the playful side of stem cell science. Scientists are often stereotyped as lab recluses who honorably forgo social lives in the quest to make game-changing discoveries and advance cutting-edge research. But as a former bench scientist, I can attest that scientists are normal people too. They might have a nerdy, slightly neurotic side around their field of research, but they know how to enjoy life and have fun. So here are a few stories that caught our eye this week about scientists having a good time with science.

Rockin’ researchers battle for glory (Kevin McCormack)

Did you know that Bruce Springsteen got his big break after winning the Biotech Battle of the Bands (BBOB)? Probably not, I just made that up. But just because Bruce didn’t hit it big because of BBOB doesn’t mean you can’t.

BBOB is a fun chance for you and your labmates, or research partners, to cast off your lab coats, pick up a guitar, form a band, show off your musical chops, play before a live audience and raise money for charity.  This is the fourth year the event is being held. It’s part of Biotech Week Boston, on Wednesday, September 27th at the Royale Nightclub, Boston.

Biotech Week is a celebration of science and, duh, biotech; bringing together what the event organizers call “the most inventive scientific minds and business leaders in Boston and around the world.” And they wouldn’t lie would they, after all, they’re scientists.

If you want to check out the competition here’s some video from a previous year – see if you can spot the man with the cowbell!

“Cells I See” Stem Cell Art Contest

It’s that time again! The “Cells I See” art contest hosted by Canada’s Centre for Commercialization for Regenerative Medicine (CCRM) and The Stem Cell Network is now open for business. This is a super fun event that celebrates the beauty of stem cells and biomaterials that support regenerative medicine.

Not only is “Cells I See” a great way for scientists to share their research with the public, it’s also a way for them to tap into their artistic, creative side. Last year’s ­contestants submitted breathtaking microscope images, paintings and graphic designs of stem cells in action. The titles for these art submissions were playful. “Nucleic Shower” “The Quest for Innervation” and “Flat, Fluorescent & Fabulous” were some of my favorite title entries.

There are two prizes for this contest. The grand prize of $750 will be awarded to the submission with the highest number of votes from scientists attending the Till and McCulloch Stem Cell Meeting in November. There is also a “People’s Choice” prize of $500 given to the contestant who has the most numbers of likes on the CCRM Facebook page.

The deadline for “Cell I See” submissions is September 8th so you have plenty of time to get your creative juices flowing!

Iris

The 2016 Grand Prize and People’s Choice Winner, Sabiha Hacibekiroglu, won for her photo titled “Iris”.

Scientists Teach Baseball Fans the Power of Stem Cells

San Francisco Giants fans who attended Tuesday’s ball game were in for a special treat – a science treat that is. Researchers from the Gladstone Institutes partnered with the SF Giants to raise awareness about the power of stem cells for advancing research and developing cures for various diseases.

Gladstone PhD student Jessica Butts explains the Stem Cell Plinko game to a Giants fan.

The Gladstone team had a snazzy stem cell booth at the Giant’s Community Clubhouse with fun science swag and educational stem cell activities for fans of all ages. One of the activities was a game called “Stem Cell Plinko” where you drop a ball representing a pluripotent stem cell down a plinko board. The path the ball travels represents how that stem cell differentiates or matures into adult cells like those in the heart.

Gladstone also debuted their new animated stem cell video, which explains how “stem cell research has opened up promising avenues for personalized and regenerative medicine.”

Finally, Gladstone scientists challenged fans to participate in a social media contest about their newfound stem cell knowledge cells on Twitter. The winner of the contest, a woman named Nicole, will get an exclusive, behind-the-scenes lab tour at the Gladstone and “see firsthand how Gladstone is using stem cells to overcome disease.”

The Gladstone “Power of Stem Cells” event is a great example of how scientists are trying to make research and science more accessible to the public. It not only benefits people by educating them about the current state of stem cell research, but also is a fun way for scientists to engage with the local community.

“Participating in the SF Giants game was very fun,” said Megan McDevitt, vice president of communications at the Gladstone Institutes. “Our booth experienced heavy traffic all evening, giving us a wonderful opportunity to engage with the San Francisco community about science and, more specifically, stem cell research. We were delighted to see how interested fans were to learn more on the topic.”

And as if all that wasn’t enough, the Giants won, something that hasn’t been happening very much this season.

Go Giants. Go Gladstone.

Gladstone scientist dropping stem cell knowledge to Giants fans.

Stories that caught our eye: An antibody that could make stem cell research safer; scientists prepare for clinical trial for Parkinson’s disease; and the stem cell scientist running for Congress

Antibody to make stem cells safer:

There is an old Chinese proverb that states: ‘What seems like a blessing could be a curse’. In some ways that proverb could apply to stem cells. For example, pluripotent stem cells have the extraordinary ability to turn into many other kinds of cells, giving researchers a tool to repair damaged organs and tissues. But that same ability to turn into other kinds of cells means that a pluripotent stem cell could also turn into a cancerous one, endangering someone’s life.

A*STAR

Researchers at the A*STAR Bioprocessing Technology Institute: Photo courtesy A*STAR

Now researchers at the Agency for Science, Technology and Research (A*STAR) in Singapore may have found a way to stop that happening.

When you change, or differentiate, stem cells into other kinds of cells there will always be some of the original material that didn’t make the transformation. Those cells could turn into tumors called teratomas. Scientists have long sought for a way to identify pluripotent cells that haven’t differentiated, without harming the ones that have.

The team at A*STAR injected mice with embryonic stem cells to generate antibodies. They then tested the ability of the different antibodies to destroy pluripotent stem cells. They found one, they called A1, that did just that; killing pluripotent cells but leaving other cells unharmed.

Further study showed that A1 worked by attaching itself to specific molecules that are only found on the surface of pluripotent cells.

In an article on Phys.Org Andre Choo, the leader of the team, says this gives them a tool to get rid of the undifferentiated cells that could potentially cause problems:

“That was quite exciting because it now gives us a view of the mechanism that is responsible for the cell-killing effect.”

Reviving hope for Parkinson’s patients:

In the 1980’s and 1990’s scientists transplanted fetal tissue into the brains of people with Parkinson’s disease. They hoped the cells in the tissue would replace the dopamine-producing cells destroyed by Parkinson’s, and stop the progression of the disease.

For some patients the transplants worked well. For some they produced unwanted side effects. But for most they had little discernible effect. The disappointing results pretty much brought the field to a halt for more than a decade.

But now researchers are getting ready to try again, and a news story on NPR explained why they think things could turn out differently this time.

tabar-viviane

Viviane Tabar, MD; Photo courtesy Memorial Sloan Kettering Cancer Center

Viviane Tabar, a stem cell researcher at Memorial Sloan Kettering Cancer Center in New York, says in the past the transplanted tissue contained a mixture of cells:

“What you were placing in the patient was just a soup of brain. It did not have only the dopamine neurons, which exist in the tissue, but also several different types of cells.”

This time Tabar and her husband, Lorenz Studer, are using only cells that have been turned into the kind of cell destroyed by the disease. She says that will, hopefully, make all the difference:

“So you are confident that everything you are putting in the patient’s brain will consist of  the right type of cell.”

Tabar and Studer are now ready to apply to the Food and Drug Administration (FDA) for permission to try their approach out in a clinical trial. They hope that could start as early as next year.

Hans runs for Congress:

Keirstead

Hans Keirstead: Photo courtesy Orange County Register

Hans Keirstead is a name familiar to many in the stem cell field. Now it could become familiar to a lot of people in the political arena too, because Keirstead has announced he’s planning to run for Congress.

Keirstead is considered by some to be a pioneer in stem cell research. A CIRM grant helped him develop a treatment for spinal cord injury.  That work is now in a clinical trial being run by Asterias. We reported on encouraging results from that trial earlier this week.

Over the years the companies he has founded – focused on ovarian, skin and brain cancer – have made him millions of dollars.

Now he says it’s time to turn his sights to a different stage, Congress. Keirstead has announced he is going to challenge 18-term Orange County Republican Dana Rohrabacher.

In an article in the Los Angeles Times, Keirstead says his science and business acumen will prove important assets in his bid for the seat:

“I’ve come to realize more acutely than ever before the deficits in Congress and how my profile can actually benefit Congress. I’d like to do what I’m doing but on a larger stage — and I think Congress provides that, provides a forum for doing the greater good.”

A look back at the last year – but with our eyes firmly on the future

Randy

CIRM President & CEO Randy Mills doesn’t want “good”, he wants “better”

Better.

With that single word Randy Mills, our President and CEO, starts and ends his letter in our 2015 Annual Report and lays out the simple principle that guides the way we work at CIRM.

Better.

But better what?

“Better infrastructure to translate early stage ideas into groundbreaking clinical trials. Better regulatory practices to advance promising stem cell treatments more efficiently. Better treatments for patients in need.”

“Better” is also the standard everyone at CIRM holds themselves to. Getting better at what we do so we can fulfill our mission of accelerating stem cell treatments to patients with unmet medical needs.

The 2015 Annual Report highlights the achievements of the last year, detailing how we invested $135 million in 47 different projects at all levels of research. How our Board unanimously passed our new Strategic Plan, laying out an ambitious series of goals for the next five years from funding 50 new clinical trials, to creating a new regulatory process for stem cell therapies.

Snapshot of CIRM's 2015 Funding

The report offers a snapshot of where our money has gone this year, and how much we have left. It breaks down what percentage of our funding has gone to different diseases and how much we have spent on administration.

Jonathan Thomas, the Chair of our Board, takes a look back at where we started, 10 years ago, comparing what we did then (16 awards for a total of $12.5 million) to what we are doing today. His conclusion; we’re doing better.

But we still have a long way to go. And we are determined to get even better.

P.S. By the way we are changing the way we do our Annual Report. Our next one will come out on January 1, 2017. We figured it just made sense to take a look back at the last year as soon as the new year begins. It gives you a better (that word again) sense of what we did and where we  are heading. So look out for that, coming sooner than you think.

Stem cell stories that caught our eye: turning on T cells; fixing our brains; progress and trends in stem cells; and one young man’s journey to recover from a devastating injury

Healthy_Human_T_Cell

A healthy T cell

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.

Directing the creation of T cells. To paraphrase the GOP Presidential nominee, any sane person LOVES, LOVES LOVES their T cells, in a HUGE way, so HUGE. They scamper around the body getting rid of viruses and the tiny cancers we all have in us all the time. A CIRM-funded team at CalTech has worked out the steps our genetic machinery must take to make more of them, a first step in letting physicians turn up the action of our immune systems.

We have known for some time the identity of the genetic switch that is the last, critical step in turning blood stem cells into T cells, but nothing in our body is as simple as a single on-off event. The Caltech team isolated four genetic factors in the path leading to that main switch and, somewhat unsuspected, they found out those four steps had to be activated sequentially, not all at the same time. They discovered the path by engineering mouse cells so that the main T cell switch, Bcl11b, glows under a microscope when it is turned on.

“We identify the contributions of four regulators of Bcl11b, which are all needed for its activation but carry out surprisingly different functions in enabling the gene to be turned on,” said Ellen Rothenberg, the senior author in a university press release picked up by Innovations Report. “It’s interesting–the gene still needs the full quorum of transcription factors, but we now find that it also needs them to work in the right order.”

Video primer on stem cells in the brain.  In conjunction with an article in its August issue, Scientific American posted a video from the Brain Forum in Switzerland of Elena Cattaneo of the University of Milan explaining the basics of adult versus pluripotent stem cells, and in particular how we are thinking about using them to repair diseases in the brain.

The 20-minute talk gives a brief review of pioneers who “stood alone in unmarked territory.” She asks how can stem cells be so powerful; and answers by saying they have lots of secrets and those secrets are what stem cell scientist like her are working to unravel.  She notes stem cells have never seen a brain, but if you show them a few factors they can become specialized nerves. After discussing collaborations in Europe to grow replacement dopamine neurons for Parkinson’s disease, she went on to describe her own effort to do the same thing in Huntington’s disease, but in this case create the striatal nerves lost in that disease.

The video closes with a discussion of how basic stem cell research can answer evolutionary questions, in particular how genetic changes allowed higher organisms to develop more complex nervous systems.

kelley and kent

CIRM Science Officers Kelly Shepard and Kent Fitzgerald

A stem cell review that hits close to home.  IEEE Pulse, a publication for scientists who mix engineering and medicine and biology, had one of their reporters interview two of our colleagues on CIRM’s science team. They asked senior science officers Kelly Shepard and Kent Fitzgerald to reflect on how the stem cell field has progressed based on their experience working to attract top researchers to apply for our grants and watching our panel of outside reviewers select the top 20 to 30 percent of each set of applicants.

One of the biggest changes has been a move from animal stem cell models to work with human stem cells, and because of CIRM’s dedicated and sustained funding through the voter initiative Proposition 71, California scientists have led the way in this change. Kelly described examples of how mouse and human systems are different and having data on human cells has been critical to moving toward therapies.

Kelly and Kent address several technology trends. They note how quickly stem cell scientists have wrapped their arms around the new trendy gene editing technology CRISPR and discuss ways it is being used in the field. They also discuss the important role of our recently developed ability to perform single cell analysis and other technologies like using vessels called exosomes that carry some of the same factors as stem cells without having to go through all the issues around transplanting whole cells.

“We’re really looking to move things from discovery to the clinic. CIRM has laid the foundation by establishing a good understanding of mechanistic biology and how stem cells work and is now taking the knowledge and applying it for the benefit of patients,” Kent said toward the end of the interview.

jake and family

Jake Javier and his family

Jake’s story: one young man’s journey to and through a stem cell transplant; As a former TV writer and producer I tend to be quite critical about the way TV news typically covers medical stories. But a recent story on KTVU, the Fox News affiliate here in the San Francisco Bay Area, showed how these stories can be done in a way that balances hope, and accuracy.

Reporter Julie Haener followed the story of Jake Javier – we have blogged about Jake before – a young man who broke his spine and was then given a stem cell transplant as part of the Asterias Biotherapeutics clinical trial that CIRM is funding.

It’s a touching story that highlights the difficulty treating these injuries, but also the hope that stem cell therapies holds out for people like Jake, and of course for his family too.

If you want to see how a TV story can be done well, this is a great example.

Women in Bio on The Influential Paths of Great Visionary Leaders

Powerful women made powerful statements last week at the Women in Bio (WIB) Plenary Event during the 2016 BIO International Convention. A panel of influential women leaders discussed difficult yet critical topics, such as how to brand yourself as a woman in a male-dominated industry, the importance of side hustles, and how to close the gender gap. It was a dynamic and inspiring event that engaged both men and women in the audience in productive conversation about how we can all work together to support women in the life sciences industry.

The panel was moderated by Nicole Fisher, the Founder and CEO of HHR Strategies and Forbes Contributer, and the speakers included Renee Compton Ryan, VP of Venture Investments at Johnson & Johnson and Frances Colón, Deputy Science and Technology Adviser to Secretary of State John Kerry.

Frances Colon, Renee Ryan, Nicole Fisher.

Frances Colon, Renee Ryan, Nicole Fisher.

The panel was more of a fire-side chat with the three woman talking intimately at a small coffee table, first sharing stories about their career paths and the road blocks along the way, and then delving into the controversial topics that women in the life sciences face.

Career Paths of Influential Women

Nicole told her story about how she got into the healthcare space. She started by ghostwriting about healthcare, innovation, and politics for the Congressional Budget Office director. Her passion turned into an opportunity with Forbes where she now runs the Health Innovation and Policy page and eventually into her company HHR Strategies which focuses on healthcare and human rights.

Renee discussed how she started as an investment banker in healthcare and made an investment in a company that benefitted patients. This experience made her want to be a part of the solution for patients, which she described as “a calling we are all fortunate to have,” and ultimately brought her to her current position at J&J.

After completing a Ph.D. in developmental neurobiology, Frances switched gears and found her strengths and assets in science policy and communications. She wanted to bring science into international affairs and shared that her mission now is to “make science cool to political scientists and diplomats to the point where my job becomes irrelevant.”

Other Panel Highlights

Branding

Renee’s advice on branding was, “challenge yourself to know your brand, and revisit your brand”. Everyone builds a resume chronologically, but she forces herself to revisit her resume every two years. Her trick is to flip the resume over to the blank side and list all her skills but do it through a different lens so you can have perspective. This process helps her decide where she wants to grow and learn.

Having Side Hustles

Frances mentioned the importance of having “side hustles”. These are things that you are really passionate about that will also build on your strengths, raise your visibility and help you take your brand to the next level. She mentioned two side hustles in particular, a non-profit she founded that supports the Puerto Rican Diaspora Network and a group she organized called the Science Technology Table, which brings together government and the private sector to discuss trending topics in science, tech and innovation. Nicole chimed in and said that all three of her side hustles have turned into companies or big opportunities that have significantly advanced her career.

Closing the Gender Gap, No More Manels!

The panelists had much to say about closing the gender gap. Renee encouraged women in high-up positions to mentor other women that show promise and to be a hands-on mentor. She also said that everyone in the biotech and pharma industries should be studying the data to see why there are less women in the life sciences and what can be done about it.

Frances said that the gender policies at companies need to change, and that people at companies have to hold each other accountable and have the conversations that can create change. One of her key points that got a laugh from the crowd was getting rid of “manels”, or all men panels, which are prevalent at major conferences in the biotech and healthcare space. She also spoke about how we need to strive for 50/50 representation on boards and executive management.

What the audience had to say

The panel was a hit with the Women in Bio audience. Dr. Leah Makley, a WIB member and Founder and CSO of ViewPoint Therapeutics, had this to say about the event,

Leah Makley

Leah Makley

“The panelists shared candid wisdom from their own career trajectories, passions, and ‘side hustles’ that far surpassed the typical depth of career panels.  Moreover, I thought Nicole Fisher did an exceptional job of framing the conversation and asking provocative questions.”

She also spoke about the importance of the WIB community and the resources they offer:

“WIB is a supportive community of powerful, inspiring women. Both the members and the events tend to be action- and solution-oriented, and I’ve walked away from each event I’ve attended with new insights, perspectives, and energy. I’m so grateful that this resource exists.”

Marco Chacon

Marco Chacon

A moment that really stood out in my mind was a moving speech by Marco Chacon, Founder of Paragon Bioservices, and a WIB sponsor. Marco shared that he recently attended a meeting in Boston and listened in on a few diversity forums. He was appalled to hear the statistics on gender diversity in the executive suite and boards of directors in biotech and pharma. Passionately he said, “This has got to change, and to the degree that I can affect this in some way, I can assure you I will do so.”

Final Thoughts

Influential leaders like Nicole, Renee, Frances, and Marco and organizations like Women in Bio, are laying the groundwork for the career advancement of women in science. This event was a great reminder that the issues facing women in the life sciences industry can be addressed in the immediate future if we continue the conversation and challenge one another to create change.

BIO 2016: IMAGINE Curing Disease and Saving Lives Part 2

As promised, here is Part 2 of our blog coverage on the BIO International Convention currently ongoing in San Francisco. Here are a few more insights on the talks we attended and highlights of other coverage from top biotech journalists and media outlets.

Keynote with Dr. Bennet Omalu and Will Smith on “Concussion”

If you haven’t seen the movie Concussion, add it to your watch list right now. It’s certainly at the top of mine after listening to Nigerian-American doctor Bennet Omalu share his story about how he single-handedly changed the way the National Football League (NFL) and the world views concussions and brain science.

Will Smith and Dr. Bennet Omalu at #BIO2016

Will Smith and Dr. Bennet Omalu at #BIO2016

In this keynote address, Dr. Omalu sat down with actor Will Smith, who portrays Dr. Omalu in the movie, to discuss how knowledge and truth precipitates evolution. Because of his passion for seeking the truth, Omalu’s autopsy of former NFL player Mike Webster led to the first diagnosis of chronic traumatic encephalopathy (CTE). Omalu’s main message was that faith and science go hand in hand. “Faith searches for truth and science searches for truth. There is no end to truth.” He also emphasized that while the truth can be inconvenient, it’s worth pursuing because truth is empowering.

For Will Smith, portraying Dr. Omalu in Concussion, was both an honor and a duty. As a parent of a son who plays football, he was compelled to tell this story and share this knowledge with parents around the world. Smith was so motivated to take on Omalu’s character that he even watched Omalu conduct four autopsies so he could really understand both the man and the science behind CTE.

This dynamic conversation was the highlight of BIO, and you can read more details about it in this article by Eleena Korban of BIOtechNOW. 

Fireside chat with US FDA Commissioner Robert Califf

Robert Califf and Steve Usdin

Robert Califf and Steve Usdin

Robert Califf, the Commissioner of the US Food and Drug Administration, sat down with Steve Usdin, the Senior Editor with BioCentury, to discuss the most important topics facing the FDA right now. Here are some of his main points:

  • FDA will focus more on patient engagement. Califf said that patients should be involved from the beginning and not just be the recipients of the end product. He also touched on risk tolerance for patients and that it can vary based on disease. The FDA wants to engage patients, advocacy groups, and industry on this topic so that patients can make more educated decisions about their treatment options.
  • The cost of clinical trials is going up 3-4 times the consumer price index which is not sustainable. Califf suggested that we can use integrated health systems and already available data from electronic medical records and patient registries to reduce the costs of large clinical trials. He commented, “The question is, can you create a different playing field that would radically reduce the cost of clinical trials while actually getting us better data about what people really care about and solve their problems related to the use of our products. I think we are close to that point now.”
  • Califf mentioned the FDA’s role in President Obama’s Precision Medicine Initiative as a step towards radically accelerating the rate of drug development. The FDA is partnering with the NIH to create a cloud-based workspace where genetic information on disease can be stored, shared, and studied.
  • Lastly, Califf mentioned how the FDA is creating a virtual center of excellence for cancer research as part of the Cancer Moonshot Initiative. He said that the FDA needs to do a better job of collaborating across its different product centers and that drug devices and biologics will be brought together starting first in the oncology space, and then eventually rolled out to other disease areas. On the clinical side, they will focus on patient involvement and the needs of cancer patients.

More coverage on the FDA fireside chat from BIOtechNOW

 Final Thoughts

While BIO ends today, the partnerships, conversations, and innovation certainly will not. In just four short days, the vibrant and eager atmosphere of BIO has transformed this year’s theme of Imagination into one of hopeful reality. Curing disease and saving lives might not be in the immediate future, but after what I’ve seen at BIO, I’m confident that the groundwork has been laid out to accelerate us down this path.


Other #BIO2016 coverage

IMAGINE Curing Disease and Saving Lives: BIO 2016 Part 1

Did you hear that? It’s the sound of more than 15,000 people taking a collective breath. That’s because we are now at the halfway point of the 2016 BIO International Convention, the world’s largest biotechnology gathering with over 900 speakers, 180 company presentations, 19 education tracks, 6 super sessions, and 35,000 partnering meetings. Now that’s a lot of stuff!

While many at BIO are focused on partnering – establishing new and exciting relationships with other biotech and pharmaceutical companies to push their products forward – others come to BIO to learn about the latest in research, innovation, and healthcare in the biotechnology space.

With so much going on at once, it’s hard to choose where to spend your time. If you follow BIO on twitter using the hashtag #BIO2016, you’ll get a condensed version of the who, what, and how of BIO.

For those of you who are more partial to blogs, here’s a brief recap of the talks that we’ve attended so far:

Mitochondrial Disease Education Session

A panel of scientific experts and patient advocates gave an overview of mitochondrial diseases and the latest research efforts to develop therapies for mitochondrial disease patients. Phil Yeske of the United Mitochondrial Disease Foundation described his foundation as the largest funder of mitochondrial research next to the government. Their focus is on patient-centered therapeutic development and they’ve established a community registry of patients that makes patients the central stewards for research and clinical development.

The most moving part of this session was an impromptu speech by Liz Kennerley, a mitochondrial disease patient and advocate. She bravely spoke about the roller coaster of symptoms affecting all of the organs in her body and aptly described her daily experience by quoting Forest Gump, “Life is like a box of chocolates, you never know what you’re gonna get.” She ended with the powerful statement that patients are at the core of everything scientists do, and encouraged the panel to engage patients more often because they will tell you everything if you ask them the right questions.

Mitochondrial Disease Patient Liz Kennerley.

Mitochondrial Disease Patient Liz Kennerley speaks at BIO 2016.

Moving out of Stealth Mode: Biotech journalists offer real-world advice on working with media to tell your story

One of my favorite panels of the conference so far featured three biotech journalists, Christina Farr of Fast Company, Jeff Cranmer of BioCentury, and Alex Lash of Xconomy. It was a dynamic conversation about how biotech companies coming out of stealth mode can best pitch their story to the media. Take home points include:

  • When pitching to a journalist, make sure that you are honest about what you can and can’t say. Have a “BS committee” that can address the validity of your work and your research claims.
  • When pitching, journalists want to know what the problem is you’re trying to solve and how you are trying to solve it better than anyone else.
  • On press releases: Unless it’s a press release from a big name, journalists won’t read it. The panel said they would prefer a personalized email detailing a company’s background and stage of work. They would also consider reading a press release that included a short personalized email from the company CEO.
  • Most hated words used to describe research: “Revolutionary” “Game-changing” “Disruptive”.

    Biotech journalist panel with.

    Moderator Carin Canale-Theakston with biotech journalists Jeff Cranmer, Alex Lash, and Christina Farr

Fireside Chat with University of California President Janet Napolitano

In an intimate Fireside chat, Janet Napolitano described her passion for higher education and making a difference in students’ lives. In her new role as the President of the UC system, her main focus is on aligning the policies and initiatives between the UC campuses and promoting research and innovation that can be commercialized around the world.

When asked about how she values basic research compared to applied research, Napolitano responded,

UC President Janet Napolitano

UC President Janet Napolitano

“We want an atmosphere where basic research is supported and one where innovation and entrepreneurship is fostered through incubators and public/private partnerships. We need to make these a tangible reality.”

 

Napolitano also mentioned that the UC system needs support from the private sector and gave PrimeUC – a collaboration with Johnson & Johnson Innovation that is part of her innovation and entrepreneurship initiative – as an example of a step in the right direction. You can read more about PrimeUC in this Event Recap.

From Ebola to Zika, how can we go faster in a global emergency?

I was only able to sit in on part of this expert panel, but here is the gist of their conversation. The global number of human infectious diseases is rapidly increasing every year due to hard-to-control factors like overpopulation, deforestation, and global climate change.  As a result, we’ve had two global health emergencies in the past two years: Ebola and Zika. We were more prepared to deal with the Ebola epidemic because more treatments were already in development. Unfortunately, we weren’t as prepared for Zika as it wasn’t on the world’s radar as a serious disease until 2015.

Martin Friede of the World Health Organization (WHO) said we should take what we learned from the recent Ebola outbreak and apply it to the Zika threat. He said the WHO wants to plan ahead for future outbreaks and remove bottlenecks to health benefits. They want to predict what diseases might surface in the future and have products ready for approval by the time those diseases manifest.


That’s all for now, but be sure to read Part 2 of our BIO2016 coverage tomorrow on the Stem Cellar. We will give highlights from an entertaining and fascinating Keynote address with Dr. Bennet Omalu (the doctor who blew the whistle on concussion in the NFL) and Oscar-nominated actor Will Smith (who played Dr. Omalu in the movie “Concussion”) on “Knowledge precipitates Evolution”. I’ll also tell you about an eye-opening Fireside chat with the US Food and Drug Administration Commissioner Robert Califf, and much more!

Get your BIO on: Sneak Peak of the June 2016 BIO Convention in SF

Screen Shot 2016-06-01 at 8.43.36 AM

Summer is almost here and for scientists around the world, that means it’s time to flock to one of the world’s biggest biotech meetings, the BIO International Convention.

This year, BIO is hosted in the lovely city of San Francisco. From June 6-9th, over 15,000 biotechnology and pharma leaders, as well as other professionals, academics, and patients will congregate to learn, educate, and network.

There’s something for everyone at this convention. If you check out the BIO agenda, you’ll find a plethora of talks, events, education sessions, and fire side chats on almost any topic related to science and biotechnology that you can imagine. The hard part will be deciding what to attend in only four short days.

For those going to BIO this year, make sure to check out the myBIO event planning tool that’s free for attendees and allows you to browse events and create a personalized agenda. You can also set up a professional profile that will share your background and networking interests with others at BIO. With this nifty tool, you can search for scientists, companies, and speakers you might want to connect with during the convention. Think of all the potential networking opportunities right at your fingertips!

Will Smith (source)

Will Smith (source)

For those who can’t make it to BIO, don’t worry, we have you covered. CIRM will be at the convention blogging and live tweeting. Because our mission is to bring stem cell treatments to patients with unmet medical needs, the majority of our coverage will be on talks and sessions related to regenerative medicine and patient advocacy. However, there are definitely some sessions outside these areas that we won’t want to miss such as the Tuesday Keynote talk by Dr. Bennet Omalu – who helped reveal the extent of brain damage in the NFL – and actor Will Smith – who plays Dr. Omalu in the movie ‘Concussion’. Their join talk is called “Knowledge Precipitates Evolution.”

Here’s a sneak peak of some of the other talks and events that we think will be especially interesting:


Monday June 6th

Education Sessions on Brain Health and Mitochondrial Disease

Moving Out of Stealth Mode: Biotech Journalists Offer Real-World Advice on Working with Media to Tell Your Story

“In this interactive panel discussion, well-known biotech reporters from print and online outlets will share their insights on how to successfully work with the media. Session attendees will learn critical needs of the media from what makes a story newsworthy to how to “pitch” a reporter to strategies for translating complicated science into a story for a broad audience.”

The Bioethics of Drug Development: You Decide

A discussion of the critical bioethical issues innovative manufacturers face in today’s healthcare ecosystem. Panelists will provide insights from a diverse set of perspectives, including investors, the patient advocacy community, bioethicists and federal regulators.”


Tuesday June 7th

Fireside Chat with Robert Califf, Commissioner of the US Food and Drug Administration (FDA)

Fireside Chat with Janet Napolitano, President of the University of California

Casting a Wider Net in Alzheimer’s Research: The Diversity of Today’s Approaches and Signs of Progress

Hear clinical researchers, biotech CEOs, and patient advocates explain how the field is pivoting from the failures of past approaches to make use of the latest generation of beta-amyloid research results as well as pursue alternative therapeutic angles to improve brain health.”

From Ebola to Zika: How Can We Go Faster in a Global Emergency?

This interactive panel of public health and industry leaders will discuss what has been learned through our global response to Ebola and what is and is not applicable to Zika or other pathogens of pandemic potential.”


Wednesday June 8th

Curative Therapies: Aligning Policy with Science to Ensure Patient Access

“The promise of curative treatments creates an urgent need to ensure access for patients, promote an environment conducive to developing new treatments, and manage the concentration of healthcare expenses in a sustainable manner.  A diverse set of panelists will tackle the tough questions around curative therapies and discern what changes are necessary for our health care delivery system to meet the challenges they pose.”

An Evolving Paradigm: Advancing the Science of Patient Input in the Drug Development and Regulatory Processes

This panel will explore advances in the field of assessing patient views and perspectives, and highlight how the patient voice is being incorporated into development programs and informing FDA review and approval decisions.”

A Media Perspective

“Any press is good press or so they say. You want your story known at the right time and in the right light, but how do you get industry journalist to notice you? What peaks their interest and how do they go about story discovery? What will they be looking to write about in the next 3 to 12 months? Three top journalists will discuss their approaches to keeping current and what makes a story newsworthy.”
Patient Advocacy Meetup

Over 40 patient advocacy organizations will be discussing their latest partnerships and developments in the areas of advancing disease research and drug development.


Thursday June 9th

Novel Advances in Cancer R&D: Meeting the Needs of the Patient

This panel will feature the views of patients and advocates, regulators, and companies who are working to change the way in which we diagnose and evaluate patients with cancer by better understanding the underlying biology of their disease.”


 To follow our coverage of BIO, visit our Stem Cellar Blog or follow us on Twitter at @CIRMNews.

Helping stem cells sleep can boost their power to heal

Mouse muscle

Mighty mouse muscle cells

We are often told that sleep is one of the most important elements of a healthy lifestyle, that it helps in the healing and repair of our heart and blood vessels – among other things.

It turns out that sleep, or something very similar, is equally important for stem cells, helping them retain their power or potency, which is a measure of their effectiveness and efficiency in generating the mature adult cells that are needed to repair damage. Now researchers from Stanford, with a little help from CIRM, have found a way to help stem cells get the necessary rest before kicking in to action. This could pave the way for a whole new approach to treating a variety of genetic disorders such as muscular dystrophy.

Inside out

One problem that has slowed down the development of stem cell therapies has been the inability to manipulate stem cells outside of the body, without reducing their potency. In the body these cells can remain quiescent or dormant for years until called in to action to repair an injury. That’s because they are found in a specialized environment or niche, one that has very particular physical, chemical and biological properties. However, once the stem cells are removed from that niche and placed in a dish in the lab they become active and start proliferating and changing into other kinds of cells.

You might think that’s good, because we want those stem cells to change and mature, but in this case we don’t, at least not yet. We want them to wait till we return them to the body to do their magic. Changing too soon means they have less power to do that.

Researchers at Stanford may have found a way to stop that happening, by creating an environment in the lab that more closely resembles that in the body, so the stem cells remain dormant longer.

As senior author, Thomas Rando, said in a Stanford news release, they have found a way to keep the stem cells dormant longer:

Dr. Thomas Rando, Stanford

Dr. Thomas Rando, Stanford

“Normally these stem cells like to cuddle right up against their native muscle fibers. When we disrupt that interaction, the cells are activated and begin to divide and become less stemlike. But now we’ve designed an artificial substrate that, to the cells, looks, smells and feels like a real muscle fiber. When we also bathe these fibers in the appropriate factors, we find that the stem cells maintain high-potency and regenerative capacity.”

Creating an artificial home

When mouse muscle stem cells (MuSCs) are removed from the mouse they lose their potency after just two days. So the Stanford team set out to identify what elements in the mouse niche helped the cells remain dormant. They identified the molecular signature of the quiescent MuSCs and used that to help screen different compounds to see which ones could help keep those cells dormant, even after they were removed from the mouse and collected in a lab dish.

They whittled down the number of potential compounds involved in this process from 50 to 10, and then tested these in different combinations until they found a formulation that kept the stem cells quiescent for at least 2 days outside of the mouse.

But that was just the start. Next they experimented with different kinds of engineered muscle fibers, to simulate the physical environment inside the mouse niche. After testing various materials, they found that the one with the greatest elasticity was the most effective and used that to create a kind of scaffold for the stem cells.

The big test

The artificial niche they created clearly worked in helping keep the MuSCs in a dormant state outside of the mouse. But would they work when transplanted back into the mouse? To answer this question they tested these stem cells to see if they retained their ability to self-renew and to change into other kinds of cells in the mouse. The good news is they did, and were far more effective at both than MuSCs that had not been stored in the artificial niche.

So, great news for mice but what about people, would this same approach work with human muscle stem cells (hMuSCs)? They next tested this approach using hMuSCs and found that the hMuSCs cultured on the artificial niche were more effective at both self-renewal and retaining their potency than hMuSCs kept in more conventional conditions, at least in the lab.

In the study, published in the journal Nature Biotechnology, the researchers say this finding could help overcome some of the challenges that have slowed down the development of effective therapies:

“Research on MuSCs, hematopoietic stem cells and neural stem cells has shown that very small numbers of quiescent stem cells, even single cells, can replace vast amounts of tissue; culture systems that that maintain stem cell quiescence may allow these findings to be translated to clinical practice. In addition, the possibility of culturing hMuSCs for longer time periods without loss of potency in order to correct mutations associated with genetic disorders, such as muscular dystrophy, followed by transplantation of the corrected cells to replace the pathogenic tissue may enable improved stem cell therapeutics for muscle disorders.”