Making connections: For-Profit Companies Exhibiting at the CIRM Grantee Meeting

Cynthia Schaffer supports CIRM’s Business Development and Industry Engagement and Commercialization activities. 

CIRM is excited about engaging with the For-Profit sector of cell-therapy companies during our upcoming Annual Grantee Meeting March 6-8th. In 2011, we had a pilot program in which a few vendors from For-Profit support/tool companies exhibited at the CIRM Grantee Meeting in order to engage our grantees and discuss their research needs. CIRM holds a meeting with all of its grantees (500 plus attendees) once every 18 months or so. The purpose of meeting is to bring together investigators funded by CIRM, to highlight their research, and encourage scientific exchange and collaboration.

In 2012, we had a pilot program involving For-Profit companies attending our Bridges Grantee Meeting. The CIRM Bridges Grants fund comprehensive lecture and laboratory courses and facilitate internship placement efforts in stem cell research for undergraduate and master’s level students. The pilot program involved holding a career fair at the Bridges Grantee Meeting and inviting a handful of companies to attend in order to educate the students on the types of career opportunities available within industry (comparable to informational interviews on a group basis).

Our 2013 CIRM Grantee Meeting is fast approaching and we have extended the exhibit pilot program to include a selection of both large and small vendors. We look forward to their participation and anticipate exploring the opportunity for a Pre-Competitive Consortium to develop technology which will assist in overcoming key hurdles in advancing stem cell therapies.

How do these types of opportunities arise? In many ways. CIRM actively welcomes participation from For-Profit companies. For example, we encourage you to become known to us via your inclusion in the Univercell Market list of companies in California.

Sometimes we ask companies for help with our education outreach efforts – including speaking at our webinars and Roundtables. Often we meet with companies at industry events such as the Stem Cell Meeting on the Mesa- Investor & Partnering Forum sponsored by the Alliance for Regenerative Medicine.

In order to expand our industry outreach efforts, CIRM has hired Neil Littman as the Business Development Officer, to work with Elona Baum, VP of Business Development. Elona and Neil are seeking to secure follow-on funding and co-funding for existing CIRM programs as well as identify new funding opportunities within the For Profit sector. Elona recently presented an Industry Engagement and Commercialization Plan to CIRM’s Board.

Your ideas for industry engagement are important to us. Please feel free to contact me at cschaffer@cirm.ca.gov. Please also consider attending the CIRM Public Meeting on March 7 and helping with Stem Cell Awareness Day by hosting or attending events in your area.

C.S.

Sequestration: "Not only are we going to lose dollars, we are going to lose more human lives."

Our colleagues at the Salk Institute for Biologic Studies in La Jolla recently held a press conference with Congressman Scott Peters to discuss the impact of the Sequestration on science and research. At that event, breast cancer advocate Bianca Kennedy spoke about the relationship between science funding and the development of new therapies.

“We cannot afford to lose research funding because if we do that not only are we going to lose dollars, research advances, we are going to lose more human lives.”

Salk scientists have received $30 million from CIRM to develop therapies for neurological conditions and other disease (here’s a complete list).  

A.A.

Stem cell research in a lowly worm could disarm an infectious parasite

Planaria Image by Carolina Biological Supply Company 

OK, so this research wasn’t done in California and it involves stem cells in a lowly worm, but it’s a great example of how basic stem cell research in animals can address human disease.

The work, which was published in the February 20 issue of Nature, shows that a long-lived worm called a schistosome has a pool of stem cells that help re-grow organs and might contribute to it’s longevity. The World Health Organization considers infection by these worms to be the second most socioeconomically devastating parasitic disease, next only to malaria, with hundreds of millions infected worldwide (here’s more about schistosomes from Wikipedia).

Schistosomes aren’t well studied in the lab, so the researchers might have had a hard time finding the pool of stem cells if it weren’t for a relative called the planaria, which has long been a fixture in biology labs. These are the flat worms that some people might remember slicing in half during a high school biology lab, only to watch the worms regrow body parts.

Those body parts regrow thanks to a pool of flexible stem cells that can form any of the different organs in the planaria. This is in contrast to our own tissue-specific stem cells, which only form cells associated with that tissue—blood-forming stem cells only form cells of the blood and brain stem cells only form cells of the brain.

Because scientists knew so much about the planaria’s stem cells, they were able to quickly find similar cells in the schistosome. And, as it happens, schistosomes were also blessed in a supply of stem cells that can constantly repair the worm’s organs. They think these cells account for why the worms can live as a parasite for as long as decades.

Having found the key to the worm’s longevity, the goal is now to find a way of shutting them down, killing the worm and eliminating the infection.

In a press release from the Howard Hughes Medical Institute, the lead author on the paper Phillip Newmark, of the University of Illinois at Urbana-Champagne, says,

“People often wonder why we study the ‘lowly’ planarian, but this work provides an example of how basic biology can lead you. . . to findings that are directly relevant to important public health problems.”

A.A.
 

ResearchBlogging.orgCollins Iii JJ, Wang B, Lambrus BG, Tharp ME, Iyer H, & Newmark PA (2013). Adult somatic stem cells in the human parasite Schistosoma mansoni. Nature PMID: 23426263

Wow your audience: join CIRM’s science elevator pitch challenge

“So, what do you do?”

If you are a researcher and don’t have a compelling answer to this question, you risk more than just boring your listener. You risk losing the attention of politicians, local leaders, potential donors and reporters. You could also lose public support for science funding if you can’t explain why your research is important.

How, you ask, can you improve your descriptive powers? Join our elevator pitch challenge, described here by our own Kevin McCormack and some cooperative CIRM staff members:

On March 7-9 many of our grantees will be in San Francisco for our grantee meeting. At that event, we’ll have a camera set up to shoot our grantees’ 30 second description of their stem cell agency-funded research. We’ll post those to our website and YouTube channel. The winner—as selected by our crack communications team—will earn fame, the admiration of their colleagues, and a small prize. (Only CIRM grantees are eligible to win.)

There’s more about this challenge on our website, including information for people who want to participate but who won’t be attending the meeting.

We’d love to hear from members of the public—the people who fund most research in the United States through either federal funding like the NIH or through CIRM in California—about which research descriptions they like best. We’ll be posting to this blog when the entries are up. Feel free to comment here or on the YouTube videos.

A.A.

Diabetes Beware! JDRF and CIRM Add $3 Million to its Support for ViaCyte’s Promising Stem Cell Therapy

Cross-sectional schematic illustration of ViaCyte device

 It’s not often a promising new approach to one of the biggest health problems in the US today gets called “the holy grail” of treatments, but that’s what one independent expert called the type 1 diabetes therapy that ViaCyte is developing.

So, it’s no wonder that ViaCyte’s therapy is not only generating interest, it’s also generating capital. The Juvenile Diabetes Research Foundation has just announced that it is providing an additional $3 million in funding to help ViaCyte move the treatment out of the lab and into clinical trials in people.

This matches the $3m in supplementary funding that our Governing Board approved for the company back in December of last year. Like JDRF we have been funding ViaCyte’s product for several years, through several stages of development. The reason why we are both willing to continue to fund what is, for now at least, called VC-01 is because it’s such a promising therapy.

VC-01 is a small implantable device that contains cells, derived from a human embryonic stem cell line, that can generate insulin. The genius of the device is that it allows the cells to monitor the body’s blood glucose levels and so make insulin when it’s needed, but because it is in a capsule it is isolated from the body’s immune system, which would otherwise try to destroy it.

In a news release announcing their additional funding Julia Greenstein, PhD, JDRF’s vice president of cure therapies called the research “very promising.”

The ability to encapsulate and thereby protect implanted insulin-producing cells has been a focus for JDRF because of its potential to solve multiple problems at once. ViaCyte is currently at the forefront of developing this technology, making this a very attractive research opportunity for us.

It’s not just patients with type 1 diabetes who could benefit from this therapy. Patients with insulin-dependent type 2 diabetes could as well. Diabetes costs California a whopping $24 billion every year, so being able to cut that even by a small amount would be hugely important.

Here’s our video about this CIRM and JDRF supported Viacyte project:

K.M.

America’s Scientist Idol: conveying science to the public

How do you get researchers who are more used to talking about their work in dense scientific terms sprinkled with jargon, to translate that into every day English, something that is not only understandable to someone without a science background, but also fascinating and engaging?

That’s the goal of a couple of different workshops at the Annual Meeting of the AAAS (American Association for the Advancement of Science) in Boston.

The first workshop was called America’s Scientist Idol and it used the American Idol model to show people how to do a great presentation. Six scientists had three minutes each to give a talk about their work and why it matters to people outside the world of academia. At the end a panel of three judges – scientists and communicators all – gave each presenter a critique of their performance and then chose a winner.

It was a fun and incredibly varied group with one researcher giving a wonderfully illustrated discussion of sex. Well, insect sex actually but she peppered her talk with enough illustrations of Ryan Gosling and cheerleaders and Charles Darwin (surprisingly unsexy) to show how her work can also apply to people.

She faced tough competition with one presenter explaining his work in a rap – you try finding something that rhymes with epigenetic – and another who used complex mathematical formula and pictures of clouds with smiley faces to explain why weather forecasting isn’t always accurate.

The presentations were fun and fast and quite fascinating, but they also had a serious point; namely that if scientists want the public to understand their work then they have to be able to explain it to them in ways they’ll understand. And the more engaging and entertaining the presentation, then the greater the likelihood you’ll find an audience. At a time when funding for science is getting harder to come by, being able to make a strong case about the importance of your work and the need to support it is vital.

Without public support, and the public funding that springs from that support, most of the most important science of the last 50 years would not have been possible. For instance NIH research led to the development of 15 of the top 21 drugs introduced between 1965 and 1992.

But not all scientists are natural communicators and most don’t get any training in communication skills as they study for their PhD. So what are they to do? That’s where the second workshop came in. Titled Bad Presenter Bingo 2.0: Be a Loser of this Science Communication Game, the goal here was to give researchers some tips and basic strategies on how to talk about their work.

They handed out bingo cards to everyone with each square of the card containing something to avoid in a presentation, such as “Introduction of Introducers’ (boy I hate that), ‘Facing Screen Not Audience’, Reads a Written Talk’, and everyone’s favorite “Talking at Slides with the Pointer.’

Now, avoiding those pitfalls won’t necessarily make you a great and dynamic speaker, but it will certainly make you a better one.

Monica Metzier, from the Illinois Science Council says her game sprang out of bitter disappointment after going to a science conference where there were lots of subjects she was interested in, but the presenters were awful so she left feeling “geeked out”. She was determined to do something about it, and came up with “Bad Presentation Bingo’.

Her goal is to drive home to researchers the enormous importance of scientific outreach and doing it well. She says if researchers can do a better job explaining their work it can better serve the public, removing confusion about topics as varied as climate change, the importance of childhood vaccinations and the importance of stem cell research. OK, I added that last bit.

K.M.

Image from Mikey Angels

Keeping up with stem cell research: it takes a village

Cynthia Schaffer supports CIRM’s Business Development and Industry Engagement and Commercialization activities.

Keeping up with the news in the Stem Cell world is a bit like riding a hurricane. We could not do it without the dedicated efforts of our friends who devote their precious free time to giving back to the community via their blogs, forums and newsletters.

I would like to highlight a few of the dedicated professionals whose newsletters help me with my job tracking industry news, clinical trials and regulatory advances.

Paul Knoepfler publishes the Knoepfler Lab Stem Cell Blog. Paul regularly uses his humor and insight to spark insightful discussion on the positive and negative news of the day. One of my favorite pieces is Paul’s cartoon depicting one possible future scenario for the stem cell industry. From my perspective, Paul’s blog is great at translating scientific developments. Paul is rare in that he is also an active stem cell researcher at the UC Davis school of Medicine.

Lee Buckler is the genius behind both the Cell Therapy blog as well as the Cell Therapy Industry Group on LinkedIn which he started in 2008 and which now has over 4,500 members. I treasure Lee for his valuable analysis on the business side of the regenerative medicine industry including the way he tracks a constructed portfolio of 34 public cell therapy companies. One of favorite recent posts from Lee is his 2012 Financing Recap. I like others believe in the maxim, that once the profits start to flow in a new industry, investment money will pour in.

Alexey Bersenev produces the excellent Stem Cell Assays Blog. Alexey continually amazes me with the quality of the data he compiles on everything from cell culture to lists of cell therapy clinical trials. One of my recent favorite posts includes Alexey’s prognostications for 2013.

Of course we are thrilled with the work of the Alliance for Regenerative Medicine (ARM) who collaborates with the Genetics Policy Institute to produce the Regenerative Medicine Forum Newsletter. Their bi-weekly newsletter includes important business announcements, a listing of clinical trial updates, pressing policy and advocacy developments and several of the research community’s major scientific advancements

Remarkably, none the four resources I have mentioned above tend to overlap and provide the same story. Each of them comes from a unique perspective and there always seems to be plenty of news for them to analyze and aggregate.

I cannot conclude my list of favorite information sources without mentioning the amazing efforts of CIRM’s Communications Staff. Amy Adams has written 545 blogs while maintaining and building the CIRM website and managing our LinkedIn and Facebook forums and generally doing handstands with one hand tied behind her back [Editor's note: Amy Adams cannot to handstands]. Todd Dubnicoff has produced 194 videos for the CIRM website and is very skilled at making the complex seem accessible and the dry seem engaging.

CIRM made an investment in information sharing, specifically focusing on sharing the results of clinical trials, via the journal Stem Cells Translational Medicine, which CIRM supports. We were very pleased by the recent inaugural online edition of Stem Cells eSource from the publishers of Stem Cells Translational Medicine. (subscription information at http://www.stemcellsportal.com/stem-cells-esource.html)

It takes a village to make all of us as knowledgeable as we are. Luckily the resources above are free and available to everyone. Happy reading.

C.S.

Hope for broken hearts this Valentine’s day

A stem cell heartbeat for Valentine’s day:

These heart muscle cells are created from embryonic stem cells. Although on most days they don’t beat in time to music, they do hold promise for mending broken hearts.

Read about these and other stem cells that could be used to treat heart damage after heart attacks, or repair damage in kids born with heart defects on our heart disease fact sheet.

A.A.

(Video magic by Todd Dubnicoff)

California’s biotech industry ranked number one in new therapy development

The 2013 California Biotechnology Industry report has some good things to say about the state’s biotech industry. The state ranks number one in jobs, development of new therapies, venture capitol investment, and federal funding. Jobs in the industry also held steady through the recent recession, providing much-needed employment and tax revenue for the state.

The report came from surveys sent to 175 biotech companies in the state. A press release put out by BayBio, PwC, and California Healthcare Institute, which conducted the survey, reported that respondents said that there were improvements in the FDA regulatory process over the past year, which is a good sign for the development of new therapies. They also reported that lack of funding is one factor that could slow the pace of new therapies.

The full report is available here. They also included an overview graphic showing biotech employment over the past year.

A.A.

Positive signs for spinal cord injury trial

The company StemCells Inc has released data about the first group of patients participating in their stem cell clinical trial for spinal cord injury.

But first, the full disclosure. We didn’t fund this work, but we did award this company a Disease Team award to continue the trial. The trial is based on work by our grantees Aileen Anderson and Brian Cummings at the University of California, Irvine. Anderson is also a collaborator on the disease team award.

Having said that, here’s what StemCells Inc is reporting about the trial. They were initially testing their stem cells in three people who had received spinal cord injuries in the mid-chest region four to nine months before the transplants. None of the three had any sensation below the injury.

At six months after getting the transplant, two people started to be able to feel touch below the point of the injury. Now, at one year after the transplant, the two patients continue to have slight improvements in the ability to feel beneath the point of the injury and the third patient is stable.

Keep in mind that for this type of early stage clinical trial, the most important factor scientists are monitoring is safety. This is the first time the cells have been tested in people rather than in animals or in a lab dish, and people are very different than animals. That’s why the company used a very low dose of the cells and only tested them in a small number of people.

A press release from the company quotes Martin McGlynn, President and CEO of StemCells Inc.

“While we need to be cautious when interpreting data from a small, uncontrolled trial, to our knowledge, this is the first time a patient with a complete spinal cord injury has been converted to a patient with an incomplete injury following transplantation of neural stem cells. We are encouraged that the cells appear to convey clinical benefit in such severely injured patients.”

The company is going to be tested the cells in more people, this time in some people who don’t have complete injuries.

For more information about stem cell research for spinal cord injury and a complete list of the awards we fund, see our spinal cord injury fact sheet.

A.A.

Image by Ed Yourdon