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:


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Meeting the scientists who are turning their daughter’s cells into a research tool – one that could change her life forever

There’s nothing like a face-to-face meeting to really get to know someone. And when the life of someone you love is in the hands of that person, then it’s a meeting that comes packed with emotion and importance.

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Lilly Grossman

Last week Gay and Steve Grossman got to meet the people who are working with their daughter Lilly’s stem cells. Lilly was born with a rare, debilitating condition called ADCY5-related dyskinesia. It’s an abnormal involuntary movement disorder caused by a genetic mutation that results in muscle weakness and severe pain. Because it is so rare, little research has been done on developing a deeper understanding of it, and even less on developing treatments.

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The Grossmans and Chris Waters meet the Buck team

 

That’s about to change. CIRM’s Induced Pluripotent Stem Cell  iPSC Bank – at the Buck Institute for Research on Aging – is now home to some of Lilly’s cells, and these are being turned into iPS cells for researchers to study the disease, and to hopefully develop and test new drugs or other therapies.

Gay said that meeting the people who are turning Lilly’s tissue sample into a research tool was wonderful:

“I think meeting the people who are doing the actual work at the lab is so imperative, and so important. I want them to see where their work is going and how they are not only affecting our lives and our daughter’s life but also the lives of the other kids who are affected by this rare disease and all rare diseases.”

Joining them for the trip to the Buck was Chris Waters, the driving force behind getting the Bank to accept new cell lines. Chris runs Rare Science a non-profit organization that focuses on children with rare diseases by partnering with patient family communities and foundations.

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Steve and Gay Grossman and Chris Waters

In a news release, Chris says there are currently 7,000 identified rare diseases and 50 percent of those affect children; tragically 30 percent of those children die before their 5th birthday:

“The biggest gap in drug development is that we are not addressing the specific needs of children, especially those with rare diseases.  We need to focus on kids. They are our future. If it takes 14 years and $2 billion to get FDA approval for a new drug, how is that going to address the urgent need for a solution for the millions of children across the world with a rare disease? That’s why we created Rare Science. How do we help kids right now, how do we help the families? How do we make change?”

Jonathan Thomas, the Chair of the CIRM Board, said one way to help these families and drive change is by adding samples of stem cells from rare diseases like ADCY5 to the iPSC Bank:

“Just knowing the gene that causes a particular problem is only the beginning. By having the iPSCs of individuals, we can start to investigate the diseases of these kids in the labs. Deciphering the biology of why there are similarities and dissimilarities between these children could the open the door for life changing therapies.”

When CIRM launched the iPSC Initiative – working with CDI, Coriell, the Buck Institute and researchers around California – the goal was to build the largest iPSC Bank in the world.  Adding new lines, such as the cells from people with ADCY5, means the collection will be even more diverse than originally planned.

Chris hopes this action will serve as a model for other rare diseases, creating stem cell lines from them to help close the gap between discovery research and clinical impact. And she says seeing the people who are turning her idea into reality is just amazing:

“Oh my gosh. It’s just great to be here, to see all these people who are making this happen, they’re great. And I think they benefit too, by being able to put a human face on the diseases they are working on. I think you learn so much by meeting the patients and their families because they are the ones who are living with this every day. And by understanding it through their eyes, you can improve your research exponentially. It just makes so much more sense.”

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RARE Bears for RARE Science

To help raise funds for this work Rare Science is holding a special auction, starting tomorrow, of RARE Bears. These are bears that have been hand made by, and this is a real thing, “celebrity quilters”, so you know the quality is going to be amazing. All proceeds from the auction go to help RARE Science accelerate the search for treatments for the 200 million kids around the world who are undiagnosed or who have a rare disease.