Understanding how stem cells become new bone could make the process more efficient, cheaper

We frequently write about using synthetic scaffolds and various biomaterials to coax stem cells to become specific tissues in specific shapes, such as for creating a new windpipe. On the surface, these materials seem like steel beams that provide support for a building but don’t really impact the make-up of the walls they are holding up. It turns out the scaffolds can have a bigger role.

A team at the University of California San Diego, led by CIRM-funded Shyni Varghese, published an enlightening example this week in the Proceedings of the National Academy of Sciences. They found that a compound in the scaffold had a direct impact on the metabolism of the stem cells that drove them to become bone.

That compound, calcium phosphate, has been known to help drive stem cells to become bone, but no one knew why or how. So, getting the desired impact has been an inefficient and expensive trial and error effort. Now, researchers have a pathway they can try to manipulate directly to impact hard-too-heal bone fractures and diseases like osteoporosis. A press release from UCSD has a quote from Varghese explaining the importance of knowing this path:

“We knew for years that calcium phosphate-based materials promote osteogenic differentiation of stem cells, but none of us knew why. As engineers, we want to build something that is reproducible and consistent so we need to know how building factors contribute to this end.”

This work echoes a theme I heard yesterday at a talk at the neighboring Gladstone Institutes delivered by Todd McDevitt of Georgia Tech. His work also focuses on the factors needed to get stem cells to become a desired tissue. He developed a system of loading microscopic particles with those factors and embedding them in the clumps of stem cells he wanted to become a specific tissue. It turned out that delivering those factors locally where they could do their job allowed him to use one-tenth the amount of the normal process—dissolving them in the lab culture broth. Since those factors are often the most expensive part of developing a needed tissue, this system could dramatically bring down the cost of research and eventually the cost of developing cell-based therapies.

The current UCSD work only applies to bone, but the team hopes to use this understanding to develop material that can more efficiently drive stem cells to become muscle, blood vessels, or other tissues.

CIRM funding: Shyni Varghese (RN2-00945)

Don Gibbons

New Year’s Resolution – Follow the Money

Following the money
There is a scene, possibly apocryphal, in the movie “All the President’s Men” where a character called “Deep Throat” is advising reporter Bob Woodward from the Washington Post on how to untangle the Watergate scandal: “Follow the money” he says. And they do and next thing you know President Nixon is resigning.

Now that approach may not always produce such dramatic results but it’s still true that whenever you are trying to understand and analyze a situation, one of the best ways is to simply follow the money- who is spending it, who is making it. So, if you are wondering about the future of the Regenerative Medicine (RM), then join the stem cell agency at the January 13 Regenerative Medicine State of the Industry Briefing where the focus will be on tracking the capital in RM. It could give you a fascinating glimpse into where the industry is heading in the next few years.

The free event is sponsored by CIRM’s friends at the Alliance for Regenerative Medicine (ARM). This is the fourth year for the briefing and each year the event grows larger and more ambitious. In addition to a review of the sector’s major financing deals, speakers will offer some predictions and thoughts on the Phase 2 and 3 clinical trials slated to be completed in 2014.

This is one time when the success of your rivals is good for you; if they can get money, then it is more likely that you will also be able to find financing. If they can get through the regulatory hurdles, then you are more likely to successfully navigate the Biologics License Application (BLA) process – the application process required to get Food and Drug Administration (FDA) approval to market a biological product such as a drug. That alone is surely an excellent reason to attend.

The briefing is being held at the start of the Biotech Showcase, which is an amazing event in itself, bringing together Regenerative Medicine (RM) companies and investors with technology that fosters the formation of meetings and deals.

Biotech and JP MorganWhile all this is going on nearby, Union Square will be overrun with executives from the broader biotechnology industries who are attending the JP Morgan conference (who knows who you will run into at the Starbucks – think of the possible deals over a grande macchiato!).

New Year’s Resolution: Spend Wisely One of the most popular New Year’s Resolution is to do a better job of controlling finances and CIRM has been focusing on it’s future spending plans to make sure we are using our money wisely.
At our recent December Board meeting our governing Board said its priorities are to:

  1. Focus on putting selected projects on an accelerated pathway towards FDA approval
  2. Partner with the broader RM community to bring new funds and support for moving CIRM’s existing early translational awards through to the development stage 
  3. Lose Weight (oops, sorry – wrong resolution list) 

CIRM is almost 60 employees and 29 board members and hundreds of thousands of supporters with a vision for a healthier future for Californians. May 2014 bring all of us that reality.

Cynthia Schaffer supports CIRM’s Business Development Team and organizes CIRM’s educational outreach with the FDA. She can be reached a cschaffer@cirm.ca.gov