Stem Cell Stories That Caught Our Eye: Halting Brain Cancer, Parkinson’s disease and Stem Cell Awareness Day

Stopping brain cancer in its tracks.

Experiments by a team of NIH-funded scientists suggests a potential method for halting the expansion of certain brain tumors.Michelle Monje, M.D., Ph.D., Stanford University.

Scientists at Stanford Medicine discovered that you can halt aggressive brain cancers called high-grade gliomas by cutting off their supply of a signaling protein called neuroligin-3. Their research, which was funded by CIRM and the NIH, was published this week in the journal Nature. 

The Stanford team, led by senior author Michelle Monje, had previously discovered that neuroligin-3 dramatically spurred the growth of glioma cells in the brains of mice. In their new study, the team found that removing neuroligin-3 from the brains of mice that were transplanted with human glioma cells prevented the cancer cells from spreading.

Monje explained in a Stanford news release,

“We thought that when we put glioma cells into a mouse brain that was neuroligin-3 deficient, that might decrease tumor growth to some measurable extent. What we found was really startling to us: For several months, these brain tumors simply didn’t grow.”

The team is now exploring whether targeting neuroligin-3 will be an effective therapeutic treatment for gliomas. They tested two inhibitors of neuroligin-3 secretion and saw that both were effective in stunting glioma growth in mice.

Because blocking neuroligin-3 doesn’t kill glioma cells and gliomas eventually find ways to grow even in the absence of neuroligin-3, Monje is now hoping to develop a combination therapy with neuroligin-3 inhibitors that will cure patients of high-grade gliomas.

“We have a really clear path forward for therapy; we are in the process of working with the company that owns the clinically characterized compound in an effort to bring it to a clinical trial for brain tumor patients. We will have to attack these tumors from many different angles to cure them. Any measurable extension of life and improvement of quality of life is a real win for these patients.”

Parkinson’s Institute CIRM Research Featured on KTVU News.

The Bay Area Parkinson’s Institute and Clinical Center located in Sunnyvale, California, was recently featured on the local KTVU news station. The five-minute video below features patients who attend the clinic at the Parkinson’s Institute as well as scientists who are doing cutting edge research into Parkinson’s disease (PD).

Parkinson’s disease in a dish. Dopaminergic neurons made from PD induced pluripotent stem cells. (Image courtesy of Birgitt Schuele).

One of these scientists is Dr. Birgitt Schuele, who recently was awarded a discovery research grant from CIRM to study a new potential therapy for Parkinson’s using human induced pluripotent stem cells (iPSCs) derived from PD patients. Schuele explains that the goal of her team’s research is to “generate a model for Parkinson’s disease in a dish, or making a brain in a dish.”

It’s worth watching the video in its entirety to learn how this unique institute is attempting to find new ways to help the growing number of patients being diagnosed with this degenerative brain disease.

Click on photo to view video.

Mark your calendars for Stem Cell Awareness Day!

Every year on the second Wednesday of October is Stem Cell Awareness Day (SCAD). This is a day that our agency started back in 2009, with a proclamation by former California Mayor Gavin Newsom, to honor the important accomplishments made in the field of stem cell research by scientists, doctors and institutes around the world.

This year, SCAD is on October 11th. Our Agency will be celebrating this day with a special patient advocate event on Tuesday October 10th at the UC Davis MIND Institute in Sacramento California. CIRM grantees Dr. Jan Nolta, the Director of UC Davis Institute for Regenerative Cures, and Dr. Diana Farmer, Chair of the UC Davis Department of Surgery, will be talking about their CIRM-funded research developing stem cell models and potential therapies for Huntington’s disease and spina bifida (a birth defect where the spinal cord fails to fully develop). You’ll also hear an update on  CIRM’s progress from our President and CEO (Interim), Maria Millan, MD, and Chairman of the Board, Jonathan Thomas, PhD, JD. If you’re interested in attending this event, you can RSVP on our Eventbrite Page.

Be sure to check out a list of other Stem Cell Awareness Day events during the month of October on our website. You can also follow the hashtag #StemCellAwarenessDay on Twitter to join in on the celebration!

One last thing. October is an especially fun month because we also get to celebrate Pluripotency Day on October 4th. OCT4 is an important gene that maintains stem cell pluripotency – the ability of a stem cell to become any cell type in the body – in embryonic and induced pluripotent stem cells. Because not all stem cells are pluripotent (there are adult stem cells in your tissues and organs) it makes sense to celebrate these days separately. And who doesn’t love having more reasons to celebrate science?

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Stories that caught our eye: stem cell transplants help put MS in remission; unlocking the cause of autism; and a day to discover what stem cells are all about

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Motor neurons

Stem cell transplants help put MS in remission: A combination of high dose immunosuppressive therapy and transplant of a person’s own blood stem cells seems to be a powerful tool in helping people with relapsing-remitting multiple sclerosis (RRMS) go into sustained remission.

Multiple sclerosis (MS) is an autoimmune disorder where the body’s own immune system attacks the brain and spinal cord, causing a wide variety of symptoms including overwhelming fatigue, blurred vision and mobility problems. RRMS is the most common form of MS, affecting up to 85 percent of people, and is characterized by attacks followed by periods of remission.

The HALT-MS trial, which was sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), took the patient’s own blood stem cells, gave the individual chemotherapy to deplete their immune system, then returned the blood stem cells to the patient. The stem cells created a new blood supply and seemed to help repair the immune system.

Five years after the treatment, most of the patients were still in remission, despite not taking any medications for MS. Some people even recovered some mobility or other capabilities that they had lost due to the disease.

In a news release, Dr. Anthony Fauci, Director of NIAID, said anything that holds the disease at bay and helps people avoid taking medications is important:

“These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS. These encouraging results support the development of a large, randomized trial to directly compare HDIT/HCT to standard of care for this often-debilitating disease.”

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Scripps Research Institute

Using stem cells to model brain development disorders. (Karen Ring) CIRM-funded scientists from the Scripps Research Institute are interested in understanding how the brain develops and what goes wrong to cause intellectual disabilities like Fragile X syndrome, a genetic disease that is a common cause of autism spectrum disorder.

Because studying developmental disorders in humans is very difficult, the Scripps team turned to stem cell models for answers. This week, in the journal Brain, they published a breakthrough in our understanding of the early stages of brain development. They took induced pluripotent stem cells (iPSCs), made from cells from Fragile X syndrome patients, and turned these cells into brain cells called neurons in a cell culture dish.

They noticed an obvious difference between Fragile X patient iPSCs and healthy iPSCs: the patient stem cells took longer to develop into neurons, a result that suggests a similar delay in fetal brain development. The neurons from Fragile X patients also had difficulty forming synaptic connections, which are bridges that allow for information to pass from one neuron to another.

Scripps Research professor Jeanne Loring said that their findings could help to identify new drug therapies to treat Fragile X syndrome. She explained in a press release;

“We’re the first to see that these changes happen very early in brain development. This may be the only way we’ll be able to identify possible drug treatments to minimize the effects of the disorder.”

Looking ahead, Loring and her team will apply their stem cell model to other developmental diseases. She said, “Now we have the tools to ask the questions to advance people’s health.”

A Day to Discover What Stem Cells Are All about.  (Karen Ring) Everyone is familiar with the word stem cells, but do they really know what these cells are and what they are capable of? Scientists are finding creative ways to educate the public and students about the power of stem cells and stem cell research. A great example is the University of Southern California (USC), which is hosting a Stem Cell Day of Discovery to educate middle and high school students and their families about stem cell research.

The event is this Saturday at the USC Health Sciences Campus and will feature science talks, lab tours, hands-on experiments, stem cell lab video games, and a resource fair. It’s a wonderful opportunity for families to engage in science and also to expose young students to science in a fun and engaging way.

Interest in Stem Cell Day has been so high that the event has already sold out. But don’t worry, there will be another stem cell day next year. And for those of you who don’t live in Southern California, mark your calendars for the 2017 Stem Cell Awareness Day on Wednesday, October 11th. There will be stem cell education events all over California and in other parts of the country during that week in honor of this important day.

 

 

Seeing is Believing: New Video on the Power of Stem Cells

skepticThe world is full of skeptics. Remember when you first heard about self-driving cars? I’m sure that information was met with comments like, “When pigs fly!” or “I’ll believe it when I see it!” Well, it turns out that the best way to get people to believe something is possible, is to show them.

And that’s our mission at CIRM. To show people that stem cell research is important and funding it is essential for the development of future therapies that can help patients with all sorts of diseases be they rare, acute, or chronic.

We’re doing this in multiple ways through our Stem Cellar blog and social media channels where we post about the latest advances in regenerative medicine research towards the clinic, through disease walks and support groups where we educate patients about stem cells, and through fun and engaging videos about the cutting-edge research that our agency is funding.

Last month, the world celebrated Stem Cell Awareness Day on October 12th. One of the ways we celebrated at CIRM was to give talks at local institutes about the power of stem cells for research and therapeutic development. One of these talks was at the Buck Institute for Research on Aging in Novato as part of their special public event on “Turning Promise of Regenerative Medicine into Reality” supported by the STEAM ENGINE, the teacher outreach program at the Buck Institute.

Kevin, CIRM’s communicators director, and I did a joint presentation on the different ways that scientists are using stem cells to model disease and to develop new treatments for patients. We also shared a few particularly exciting stories about new stem cell advancements that are being tested in clinical trials. One of them was a heartbreaking turned heartwarming story of Evangelina, a baby born with severe combined immunodeficiency (SCID), a disease that leaves children without a functioning immune system and often kills babies within a year of birth. Evangelina was part of a CIRM-funded clinical trial run by UC Los Angeles that transplanted the patient’s own genetically corrected blood stem cells. Evangelina is one of 30 children the UCLA team has cured and CIRM is now funding a Phase 2 clinical trial for this work.

Our talk was followed by exciting stories of stem cell research in the lab. Three talented postdoctoral fellows, who spoke about new developments in stem cell therapies for HIV, degenerative eye disease and neurodegenerative diseases. The talks were well received by the audience, who were actively speaking up to ask questions during the panel discussion with the speakers.

Panel on stem cells.

Stem cell panel: Kevin McCormack, Imilce Rodriguez-Fernandez, Joana Neves, Karen Ring.

It was a truly inspiring day full of learning and excitement about the future of stem cell research and regenerative medicine. But for the skeptics out there, don’t take my word for it, you can see for yourself by can watching the video recording here:


Related Links:

Celebrating Stem Cell Awareness Day with SUPER CELLS!

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To all you stem cell lovers out there, today is your day! The second Wednesday of October is Stem Cell Awareness Day (SCAD), which brings together organizations and individuals that are working to ensure the general public realizes the benefits of stem cell research.

For patients in desperate need of treatments for diseases without cures, this is also a day to recognize their struggles and the scientific advances in the stem cell field that are bringing us closer to helping these patients.

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Induced pluripotent stem cells.

How are people celebrating SCAD?

This year, a number of institutes in California are hosting events in honor of Stem Cell Awareness Day. Members of the CIRM team will be speaking on Saturday about “The Power of Stem Cells” at the Buck Institute for Research on Aging in Novato (RSVP on Facebook) and at the Berkeley Student Society for Stem Cell Research Conference in Berkeley (RSVP on Eventbrite). There are also a few SCAD events going on this week in Southern California. You can learn more about these all events on our website.

You can also find out about other SCAD celebrations and events on social media by following the hashtag #StemCellAwarenessDay and #StemCellDay on Twitter.

Super Cells: The Power of Stem Cells

Super Cells exhibit at the Lawrence Hall of Science

Super Cells exhibit at the Lawrence Hall of Science

Today, the CIRM Stem Cellar is celebrating SCAD by sharing our recent visit to the Lawrence Hall of Science, which is currently hosting an exhibit called “Super Cells: The Power of Stem Cells”.

This is a REALLY COOL interactive exhibit that explains what stem cells are, what they do, and how we can harness their power to treat disease and injury. CIRM was one of the partners that helped create this exhibit, so we were especially excited to see it in person.

Super Cells has four “high-tech interactive zones and a comprehensive educational guide for school children ages 6-14”. You can read more details about the exhibit in this promotional handout. Based on my visit to the exhibit, I can easily say­­ that Super Cells will be interesting and informative to any age group.

The exhibit was unveiled on September 28th, and the Hall told us that they have already heard positive reviews from their visitors. We had the opportunity to talk further with Susan Gregory, the Deputy Director of the Hall, and Adam Frost, a marketing specialist, about the Super Cells exhibit. We asked them a few questions and will share their interview below followed by a few fun pictures we took of the exhibit.


Q: Why did the Lawrence Hall of Science decide to host the Super Cells exhibit?

The Lawrence Hall of Science has a history of bringing in exciting and engaging traveling exhibitions, and we were looking for something new to excite our visitors in the Fall season. When the opportunity presented itself to host Super Cells, we thought it would be a good fit for our audience. Additionally, the Hall is increasing its programming and exhibits in the fields of biology, chemistry and bioengineering.

Q: What aspects of the Super Cells exhibit do you think are valuable to younger kids?

We strive to make our exhibit experiences hands-on and interactive. The Hall believes that the best way for kids to learn science is for them to be active in their learning. Super Cells offers a variety of elements that speak to our philosophy of learning and make learning science more fun.

Q: How is exhibit similar or unique to other exhibits you’ve hosted previously?

 The Hall hosts and develops exhibits across a broad range of scientific, engineering, technology and mathematical topics. We are always looking for exhibits that address recent scientific advances, and also try to showcase cutting edge research.

Super Cells presents both basic cell biology and information about recent medical and scientific advances, so it fits. Also, as mentioned in our behind the scenes story about the exhibit install, in the past many of our traveling exhibits were very large experiences that tended to take up a lot of space on the museum floor. One thing that is great about Super Cells is that it packs a lot of information into a relatively small space, allowing us to keep a number of experiences and activities that our audience has come to love on the floor, instead of removing them to make room.

Q: Will there be any special events at the Hall featuring this exhibit?

On November 11, the Hall will host a fun day of activities centered around DNA and the exhibit. Younger visitors will make DNA bracelets based on the unique traits in their genome, while older kids will isolate their own DNA using a swab from inside their cheek. We are still finalizing the details of this event, but it will definitely happen.

Q:  Why do you think it’s important for younger students and the general public to learn about stem cells and stem cell research?

As UC Berkeley’s public science center, the Hall is committed to providing a window into cutting edge research and the latest scientific information. We think it’s really important for people and kids to learn about the skills and science behind current research so they can be prepared for a future of incredible scientific challenges and opportunities that we can’t foresee.


Super Cells will be open at the Lawrence Hall of Science until November 27th, so be sure to check it out before then. If you don’t live in California, don’t worry, Super Cells will be traveling around the U.S., Europe and Canada. You can find out where Super Cells is touring next on their website.

We hope you enjoy our photos of the Super Cells exhibit!

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Happy Stem Cell Awareness Day!

SCAD_Logo_2015I woke up today extra early this morning feeling like a kid at Christmas time because it’s Stem Cell Awareness day!

This exciting day brings together organizations and people around the world working to ensure that we realize the benefits of one of the most promising fields of science in our time. The day is a unique global opportunity to foster greater understanding about stem cell research and the range of potential applications for disease and injury.

For the millions of people around the world who suffer from incurable diseases and injury, Stem Cell Awareness Day is a day to celebrate the scientific advances made to-date and be hopeful of what is yet to come.

Institutions and scientists around the world will be participating in talks and activities that celebrate and also educate the community about stem cell research. For a list of events, check out our Stem Cell Awareness Day webpage. You can also follow other events on twitter by following the hashtags #stemcellday and #astemcellscientistbecause.

In celebration of this exciting day, the Stem Cellar team would like to highlight a few videos and webpages dedicated to stem cell awareness. Enjoy!

Videos:

“A Stem Cell Story” from our friends at EuroStemCell

#AStemCellScientistBecause videos via Cell Stem Cell on twitter

 

Stem Cell Awareness Webpages:

Stem Cell Awareness Day and the Stem Cell Person of the Year

Today is Stem Cell Awareness Day, an event that seeks to bring together individuals and organizations around the world working to celebrate and raise awareness about the tremendous progress being made in stem cell research, and to ensure we remain focused on keeping that progress going until we have cures or treatments for people in need.

Paul Knoepfler

Paul Knoepfler

Today is also the day that our colleague, U.C. Davis stem cell researcher and avid blogger, Paul Knoepfler reveals the nominations for his annual Stem Cell Person of the Year award. It’s perfect timing, because many of those nominated have been pivotal in helping move the research forward, and in helping raise awareness about the field in general.

The rules for the competition are pretty simple. In Paul’s own words: “Who has been the single most important, influential person in the world of stem cells this year? Who has made the biggest positive contribution in 2014?”

This is the third year of the competition and Paul is not just a tireless champion of stem cell research, he’s also someone who puts his money where his mouth is. The prize for this year’s winner is $2,000, double that of the previous two years, and all that cash comes out of Paul’s own pocket. It’s a generous gesture from a tireless advocate. In fact those two qualities alone would suggest Paul qualifies to win his own competition. But he has too much integrity to ever even consider that.

So without further ado here is the list of nominations with quotes from some of the people who nominated them in green.

*Spoiler alert: a couple of those nominated are colleagues of mine here at the stem cell agency. I didn’t nominate them, but I can certainly testify to the fact that they deserve it.

Bernie Siegel – a long-time stem cell advocate who runs the yearly World Stem Cell Summit. “Has any one single person done more for the stem cell field?”

Chris Fasano: A principal investigator at the Neural Stem Cell Institute where he uses stem cells to study early nervous system development. Chris stands out for his energy, enthusiasm, dedication to the field, creativity and accomplishments.”

Diana DeGette, the Democratic Congresswoman from Colorado: a politician who has been working to pass important stem cell legislation. “Long time supporter of legislation to support stem cell research and regenerative medicine.”

Don Reed: long-time stem cell research advocate who played a key role in the success of Prop 71 and the creation of CIRM. “A tireless stem cell advocate always there to make a positive difference.”

Emmanuel (Ed) Baetge: Head of Nestlé Institute of Health Sciences at Nestlé Health Science and formerly CSO of Novocell/ViaCyte. “Ed was the driving force behind the development of Novocell (Viacyte’s) diabetes program using hESCs to develop a cell therapy for T1/2 patients…the phase 1 trial is enrolling this year. It has huge potential and Ed deserves the credit for his leadership and development of the technology as well as the company.”

Ellen Feigal; Vice President, Research and Development at the California Institute for Regenerative Medicine (CIRM). “At CIRM Dr. Feigal makes the Development Program happen.”

Ian mcNiece: Professor, Department of Stem Cell Transplantation,
Division of Cancer Medicine, The University of Texas MD Anderson
Cancer Center.

Janet Rossant: Senior Scientist in the Developmental & Stem Cell Biology Program and Chief of Research at The Hospital for Sick Children, Toronto

Jeanne Loring: Stem cell researcher and scholar, leading iPS cell clinical work in the pipeline for Parkinson’s Disease, and patient advocate. “Supportive to the advocacy community. Courage in supporting the challenge to WARF patents. Excellent scientist engaged in policy discussions.”

John Sinden: The co-founder of ReNeuron, a biotech company based in UK. “Thanks to his determination, and drive ReNeuron has a clinical approved stem cell product currently tested in two clinical trials.”

Judy Roberson: Long-time Huntington’s Disease patient advocate. “She makes concrete positive developments happen such as millions of dollars in research funding for HD.”

Leigh Turner: Outspoken advocate of evidence-based medicine in the stem cell field. “Gave an inspiringly frank talk about for-profit stem cell clinics flouting FDA regulations at ISSCR 2014.”

Malin Parmer: Associate Professor, Developmental and Regenerative Neurobiology, Lund University. Top neural regeneration scientist. “Young, hard worker who is doing very well”.

Masayo Takahashi: Stem cell researcher leading the team that is doing the first ever clinical study based on human iPS cells. “Creative and courageous clinical stem cell researcher.”

Mike West: He founded Geron and was CSO of Advanced Cell Technology before his current position as head of BioTime. “He has been a leader in our regen field for many years but he made a bold decision to resurrect Geron’s cell therapy for spinal cord injury this year and hopefully confirm the promise of hESC technology clinically.”

Patricia Olson: Executive Director of Scientific Activities at CIRM and active in CIRM scientific leadership from day 1. “A driving force in the stem cell field.”

Peter Zandstra: Professor at University of Toronto. Stem cell researcher. Canada Research Chair of Stem Cell Bioengineering. “Peter is a recognized pioneer and respected world leader at bringing concepts of scale-up, regulation, and industrialization to stem cell-derived cell therapy technologies.”

Pope Francis: Leader of Worldwide Catholic Church. “Strong supporter of adult stem cell biotechs and research”.

Richard Cohen: An MS patient in a clinical trial who has chronicled his experiences. An author and Meredith Vieira’s husband, father of three children. “Mr. Cohen is an inspiration to me and others because he’s very honest and doesn’t sugar-coat his struggles, losses, frustrations, anger, embarrassments, and he also shares his gains.”

Richard Garr: CEO of Neuralstem, conducting work on using stem cells for ALS and advocate of Right To Try laws. “Richard does two excellent things at once: making a treatment for ALS and connecting with ALS patients.”

Robert Lanza: CEO of Advanced Cell Technology, which has multiple ES cell-based clinical trials ongoing. “Visionary and practical so makes the impossible possible”.

Shoukhrat Mitalipov: Stem cell researcher who first successfully made nuclear transfer human ES cells by therapeutic cloning and developing oocyte transfer-based therapies for mitochondrial disorders. “Gutsy pioneer of new, game changing technologies.”

Susan Solomon: Co-Founder and CEO of The New York Stem Cell Foundation (NYSCF). Remarkably effective advocate for stem cell research. “not many leaders have created their own research laboratories and raised $100 million plus. Seriously, what an accomplishment!”

Takaho Endo: Senior Researcher at the Riken Center for Integrative Medical Sciences in Yokohama who published key genetic work on the possible origin of STAP cells. “Courage to publish this being from RIKEN.”

Ted Harada: Leading stem cell research advocate and very effective ALS patient advocate. “An Energizer Bunny for the ALS community and stem cell advocate”

Tory Williams: Stem cell advocate and author of the 2014 book, Inevitable Collision. Co-Founder and Executive Director of the Alabama Institute of Medicine (AIM). “A true hero who inspires and makes concrete things happen like AIM”.