Actress, screenwriter and stem cell agency Board member talks about her commitment to Alzheimer’s

Lauren Miller being sworn in as a member of the stem cell agency Board

The members of our governing Board do an incredible job helping fund the best science and ensuring that we do everything we can to fulfill our mission. But even though they work hard most people know very little about them, how they ended up on the Board or what their goals and aspirations for their work with the stem cell agency are.

So it was a pleasant surprise to read the latest blog from U.C. Davis stem cell scientist and CIRM grantee, Paul Knoepfler, Ph.D. It’s an interview with one of the newest members of our Board, Lauren Miller.

Along with her advocacy credits Lauren is also an accomplished actress, screen writer and movie producer. In the interview she talks about the family history that pushed her to become a Patient Advocate for Alzheimer’s research, and how she hopes to raise awareness about the disease among younger people.

Here’s Paul’s interview.

In the interview Lauren talks about her husband, Seth Rogen’s testimony to Congress on Alzheimer’s. We wrote a blog about that and included a link to video of the event.

kevin mccormack

The 2013 CIRM Annual Report: a concise, informative and yawn-free update on the state stem cell agency’s progress


2013 CIRM Annual Report cover page

At last week’s governing Board meeting, communications director Kevin McCormack unveiled CIRM’s 2013 annual report. Was that a yawn I just heard, dear reader? Are you about to click away from this blog? It’s true that annual reports can be bulky tomes that make better dust collectors than reading material. But for the past few years, here at CIRM, our approach to annual reports has been keep it concise, get to the point, make it accessible, and provide links to our website for further reading. In a mere four pages, here’s what to expect:

  • Our Accomplishments – read the agency’s achievements in supporting clinical trials, training young scientists and pumping tax revenue into the state economy
  • Stories of Hope – read poignant vignettes and view striking photography of two people, one with pediatric heart disease; the other multiple sclerosis, who help represent the hope that stem cell therapies will treat the millions living with chronic disease
  • Progress Toward Therapies – view a chart of the 81 disease-focused projects (out of 607 total awards) that are on the path toward clinic trials in people. One clear sign of progress since last year: an extra column in the chart to make room for Phase II clinical trials, the stage when the research team begins testing if the therapy works.
  • CIRM Research Funding – get the 2013 and lifetime stats on the number of grants and funding awarded by our agency
  • Letters from the Chairman and President – read President Trounson’s last annual CIRM letter as he is stepping down this year

You can also go online to view past annual reports and stories of hope. If you’d like a hard copy of the 2013 report, send us your name and address to info@cirm.ca.gov.

Todd Dubnicoff

Stem cell stories that caught our eye: tendon repair, disc disease and cancer drugs’ impact on normal stem cells

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-cell embedded sutures for tendon repair. I love it when a simple idea seems to work. Repairing a torn tendon surgically often results in a joint less strong and durable than the original, so some teams have tried injecting stem cells at the site of injury hoping they would strengthen the tendon. But as is often the case, just injecting stem cells offers no guarantee the cells will stay where you put them and aid in the healing. So a team from the MedStar Union Memorial Hospital in Baltimore performed the surgery—in rats—using sutures that had been embedded with stem cells. Those animals healed better than others treated with just surgery and surgery plus injected stem cells. They published their work in Foot and Ankle International and it was written about on the Science Codex web site.

Analysis of stem cells for disc disease. A Mayo clinic team has done a much-needed analysis of six of the better animal studies that looked at the impact of stem cell therapy on degenerative disc disease. They found that the therapy increased the height of the damaged spinal cord discs by an average of 24 percent. The therapy seemed to benefit the discs by restoring the nucleus pulposus structure, the jelly-like substance that gives the disc its cushioning effect. They presented the work at a meeting of the American Academy of Pain Medicine and it was featured in ScienceDaily. CIRM funds a team at Cedars-Sinai that is looking for ways to use stem cells for vertebral compression fractures.

Some cancer drugs activate cancer stem cells. A large and growing camp of the research community lays the blame for cancer recurrence on cancer stem cells. Now a team at Harvard and the University of Massachusetts suggests part of the blame should go to the interaction between ordinary stem cells and chemotherapy agents. They found that several different chemotherapeutics that halt rapidly growing tumors have the opposite effect on stem cells causing them to proliferate too rapidly, which they suggested could lead to new tumors. They published their research in the Proceedings of the National Academy of Sciences and it was written up by Fierce Biotech Research.

Video shows how to grow a beating heart. This brief YouTube video does a nice job explaining the steps of how you could use a cadaver heart, remove all the soft tissue and then seed the remaining scaffold of the old heart with a patient’s own stem cells. This would theoretically lead to a new heart that was immunologically compatible with the patient so the patient could avoid a lifetime of immune suppressant drugs. The video features Doris Taylor of the University of Texas Southwestern Medical Center in Dallas. You can read about CIRM-funded efforts to mend damaged hearts on our heart failure fact sheet.

Don Gibbons

A mother’s plea – find a stem cell therapy to help my son escape autism

When a mother says “I’ve never heard my son speak, never heard him tell me he loves me” it’s hard not to feel deeply moved.

Those are the words that Jennifer Bidlovski used to describe her son Milo, who has a severe form of autism spectrum disorder, in a video presentation she made to our Board at it’s meeting last week.

Jennifer made the video as part of our Spotlight on Disease presentation at the meeting. These Spotlights are important opportunities for us to update the Board on the research that is being done in specific disease areas, but it’s also an important way of connecting them with the people who are waiting for stem cell therapies, who are looking to us to help them or their loved ones.

We all need to be reminded from time to time why we do what we do. It’s all too easy to get caught up in the day-to-day business around the office and lose sight of our ultimate goal – finding treatments and cures for currently incurable diseases.

Jennifer helped remind us why we are here. To help parents like her. To help children like Milo. Jennifer says she feels like her son has been stolen from her. Through the work we fund we hope to be able to help her find him.

The other segment of last week’s Spotlight presentation featured Alysson Muotri, a scientist from the University of California at San Diego who discussed his research using stem cells to create laboratory models of various types of autism spectrum disorder. We will post that video shortly.

kevin mccormack

Finding 72 million new ways to hit the same target

Why is this man smiling? Jonathan Thomas, Chair of the CIRM Board

Finding new ways to do the same thing is never easy, particularly when the goal you are aiming for is to try and get the most promising therapies out of the lab and into clinical trials and patients. But yesterday our governing Board found some new ways to try and reach that goal.

The Board, more formally known as the Independent Citizens Oversight Committee, approved a new $40 million initiative called the Preclinical Development Award. The award is designed to support projects that the agency is already funding, to help them with all the development activities needed to get a therapy ready for approval by the Food and Drug Administration (FDA). This includes developing plans to manufacture the stem cell-based products needed for the therapy and doing the studies to show that these cells function as expected. The award also allows for researchers not yet funded by the agency to apply for the award as well, as long as they meet certain conditions.

In a news release the stem cell agency’s President, Alan Trounson, Ph.D., talked about the importance of this new award:

“If a researcher or company has already identified a therapy or approach that shows potential in battling disease, we want to help them in every way we can. By working with them to develop plans on manufacturing the therapy and how to ensure the quality of the product and what the best dosage will be we can really give them the tools they need to get this to the FDA and ultimately to patients in as short a time as possible.”

The Board also approved the fourth round of our Strategic Partnership initiative, approving another $32 million to increase our engagement with industry and help them move their most promising projects into patients as fast as possible.

But perhaps the easiest vote of the day was over the issue that just one year ago was a source of real contention, namely changes to the way we operate in the wake of the Institute of Medicine (IOM) report.

As a brief refresher, the IOM report praised us as a “bold social initiative” but also raised questions about some of our procedures saying they raised the potential for a perception of a conflict of interest. They didn’t say there had been any conflicts only that there was a potential for that perception. Even so, we took their recommendations seriously and introduced some fairly sweeping changes to the way our Board votes on funding issues including:

• The 13 Board members appointed from institutions eligible for funding from the stem cell agency, such as those in the University of California system, no longer vote on any grants brought before the Board but would instead abstain
• All members of the Board are able to participate in discussions on applications but only patient advocates and independent members of the Board can vote on funding issues (members will continue to refrain from any discussion of specific applications from their institutions)

The Board voted to introduce the changes and come back in one year to assess how they are working. Yesterday they clearly thought they were working well and, without any debate or argument, voted unanimously to make them permanent. Chairman Jonathan Thomas was clearly pleased at the result:

“The vote today shows the changes we introduced one year ago, though difficult, have succeeded in protecting the integrity of the Board and the integrity of the work we do and the way we do it,”

For those of you who just can’t get enough in-depth information about policy and process you can find all the details of those changes on the Board page on our website.

kevin mccormack

Great chance to learn more about modeling diseases using stem cells

A webinar later this morning will address a topic we frequently write about: the power of using induced pluripotent stem cells (iPS) to create laboratory models of diseases. Those models help us understand the cause of the disease and look for ways to treat it. This relatively simple process takes tissue—usually a skin sample or bit of blood—from a patient with a disease and reprograms those cells to be iPS cells. You can then mature those cells into the tissue that reflects the disease, say nerves for degenerative brain diseases, and in essence, have the disease in a lab dish.

The International Society for Stem Cell Research (ISSCR) has started a series of webinars designed to provide the public with more information about where our field is making progress. The second half hour program in the series called “Stem Cells in Focus” is today beginning at 11:00 a.m. Pacific time and will address iPS disease models. It will feature a former colleague of mine from the Harvard Stem Cell Institute, Kevin Eggan, and they have titled his session “Disease Modeling with iPS Cells: Diseases in a Dish Explained.”

There is a link to log into the webinar on the Stem Cells in Focus home page and if you miss the live stream, the video will be available at that same web site. Last month’s webinar on using your own stem cells to repair your heart is posted there already. It features CIRM grantee Deepak Srivastava of the Gladstone Institutes.

We feature Deepak in a video on this same work looking at stem cells for pediatric heart repair. And if you want to know more about how you create the iPS cells that become the disease-in-the-dish, we have a four-minute video primer on the topic.

Don Gibbons

Stem cells may help ease depression, anxiety and other symptoms of Alzheimer’s disease

Ask anyone what the symptoms of Alzheimer’s disease are and the first thing they’ll almost certainly say is loss of memory. It’s not surprising, it’s that fear of losing our memories, losing our sense of self, that makes it such a terrifying disease. But Alzheimer’s has many other symptoms – such as anger, depression, anxiety and delusions – and now a new study says stem cells may be able to help reduce those, possibly even reverse those symptoms that are often the bane of patients and caregivers.

The study, from researchers in Israel, is published in the latest issue of the journal Behavioural Brain Research.

The researchers, working with mice that had Alzheimer’s disease-like symptoms. introduced stem cells into the part of the brain that controls behaviors like fear and anxiety. Mice that didn’t get stem cells ran around without checking new areas or habitats for potential threats; but the mice given the stem cells were much more cautious in assessing new surroundings, the same as healthy mice do.

In a story in Science Daily Prof. Daniel Offen, the lead researcher, says the new stem cells seem to increase the number of neurons in the treated mice:

 “Normal mice will recognize the danger and avoid it. Mice with the disease, just like human patients, lose their sense of space and reality. We first succeeded in showing that new neuronal cells were produced in the areas injected with the gene. Then we succeeded in showing diminished symptoms as a result of this neuron repopulation.” 

Offen says tackling these symptoms is no small matter because they can cause a great deal of embarrassment for patients, their families and caregivers by fueling impulsive behavior, such as undressing in public.

The researchers hope their findings might point the way towards potential therapies to reduce some of the secondary symptoms of Alzheimer’s. In the meantime they are now working in the parts of the brain that control memory, hoping to see a similar improvement there.

We are currently funding 16 projects looking into different aspects of Alzheimer’s, including an almost $20 million Disease Team award to explore the use of stem cells to help restore memory.

kevin mccormack

Nine reasons why the biotech world loves L.A.

Los Angeles – biopharma business hub

Magazines love to use numbers in titles to attract attention. You know the kind, “Top Ten Tips for Trimmer Hips”, “Six Ways to Slim for Swimsuits”, “Five Steps to Perfect Abs”. But here’s a headline you probably won’t see on a magazine cover in the supermarket checkout line – Top 10 U.S. Biopharma Clusters. But it certainly caught our attention, particularly as three of the top ten are here in California.

The ranking comes from the magazine GEN, which reports on genetic engineering and biotechnology news – not a big seller at Safeway or Piggly Wiggly though you can spot one in just about any lab you visit. They based their ranking on the number of patents awarded in a region, the amounts of NIH grant funding and venture capital funding it attracted, the total lab space in the region, and the number of jobs in the field.

There were the usual suspects in the top three with San Francisco heading the list (again – hurrah), followed by Boston/Cambridge and San Diego. But there was a new entry from California with Los Angeles coming in at number 9. L.A. broke into the top ten based on a number of factors according to the magazine:

“the Los Angeles region has some notable employers, from Amgen in Thousand Oaks, to California Institute of Technology (Caltech) in Pasadena, and UCLA in, where else? Together they employed 23,054 people in “biomedical” positions (California Biomedical Industry Report 2013), giving the region one of the smallest workforces among the top-10. LA placed ninth in NIH funding ($65.4 million) but climbed to the middle of the pack in patents granted (550).”

Just as the stem cell agency has played a key role in making San Francisco and San Diego major players in the field, we also been instrumental in helping make L.A. a player with hundreds of millions of dollars in stem cell research funding for UCLA ($213m), USC ($106m), Cedars-Sinai Medical Center ($41m), Children’s Hospital of Los Angeles ($17.5m) among others.

If you are judged by the company you keep, we are keeping very good company indeed.

kevin mccormack

Deep brain stimulation may help advanced Parkinson’s patients by producing new stem cells

The fact that the symptoms of advanced Parkinson’s disease patients improve after implantation of electrodes has always felt a bit like voodoo to me. But this “deep brain stimulation” works. The dyskinesias, the involuntary muscle tics, that plague these patients improve after the implants.

Now, a team of scientists from the University of Florida and the University of Auckland in New Zealand has published research that offers part of the explanation for why it works. These patients produce more nerve stem cells.

The researchers published their work in PLOSone March 3 and The New Zealand Herald ran an interview with lead New Zealand researcher yesterday. It quoted Maurice Curtis on the gap in understanding about the effect of deep brain stimulation:

We always knew that when people had these electrodes implanted in their brains that their symptoms would improve, but we’ve never really known why that should make a difference. What these electrodes seem to be doing is to actually increase the amount of stem cells that are present in the key areas of the brain that normally just have a small number of stem cells. So the hope is that those stem cells are actually doing something beneficial.

However, Curtis admits that the study leaves a lot of unanswered questions.

We know that stem cells mount a major regenerative response, but is that what really brings about changes in the brain that improves the symptoms? Even though this paper is definitely a step in the right direction it leaves lots of unanswered questions about why deep brain stimulation works the way it does.

CIRM funds several projects, both fundamental research and projects moving toward the clinic, to help unravel part of those mysteries. You can read about those projects on the CIRM Parkinson’s Disease Fact Sheet.

One of those projects is using electrical currents to guide stem cells to the site of brain injury. That work is described by Min Zhao of the University of California at Davis in this video.

http://www.cirm.ca.gov/our-progress/video/min-zhao-uc-davis-cirm-stem-cell-sciencepitch

Don Gibbons

Best of the blog: Progress in liver disease

Liver disease has many causes ranging from various types of hepatitis to genetic mutations and alcohol damage. But when any of these causes lead to severe disease, the patients often find themselves on waiting lists for liver transplants. There are currently 16,000 people in the U.S. on that list and many of them will die before they get the needed organ.

Clearly, we need a way to repair, regenerate or replace livers without waiting for donors. On this blog, we’ve written about how our grantees and others around the world are progressing to stem cell-based treatments for liver disease. Here we have pulled together some of the most interesting of those posts. We start with the recent work of our grantees at the Gladstone Institutes and the University of California, San Francisco, and move on to show what a global effort this is with projects on the U.S. East Coast, Japan and the United Kingdom. We close with a post for a video about a young girl whose life was turned around by a new liver.

1. Turning skin into mature liver cells

2. Miniature liver grown from stem cells

3. Scientists grow human liver cells in mice

4. Researchers create functional liver tissue

5. $1 million offered for first team to create fully functional liver

6. Liver transplant saves girl’s life

A complete list of CIRM grants for research in liver disease is on our web site.

Don Gibbons