Category Archives: Stem Cell Research

It’s All About the Patients

Whenever you are designing something new you always have to keep in mind who the end user is. You can make something that works perfectly fine for you, but if it doesn’t work for the people who are going to work with it day in and day out, you have been wasting your time. And their time too.

At California Institute for Regenerative Medicine (CIRM), our end users are the patients. Everything we do is about them. Starting with our mission statement: to accelerate stem cell treatments to patients with unmet medical needs. Everything we do, every decision we make, has to keep the needs of the patient in mind.

So, when we were planning our recent 2020 Grantee Meeting (with our great friends and co-hosts UC Irvine and UC San Diego) one of the things we wanted to make sure didn’t get lost in the mix was the face and the voice of the patients. Often big conferences like this are heavy on science with presentations from some of the leading researchers in the field. And we obviously wanted to make sure we had that element at the Grantee meeting. But we also wanted to make sure that the patient experience was front and center.

And we did just that. But more on that in a minute. First, let’s talk about why the voice of the patient is important.

Some years ago, Dr. David Higgins, a CIRM Board member and patient advocate for Parkinson’s Disease (PD), said that when researchers are talking about finding treatments for PD they often focus on the dyskinesia, the trembling and shaking and muscle problems. However, he said if you actually asked people with PD you’d find they were more concerned with other aspects of the disease, the insomnia, anxiety and depression among other things. The key is you have to ask.

So, we asked some of our patient advocates if they would be willing to be part of the Grantee Meeting. All of them, without hesitation, said yes. They included Frances Saldana, a mother who lost three of her children to Huntington’s disease; Kristin MacDonald, who lost her sight to a rare disorder but regained some vision thanks to a stem cell therapy and is hoping the same therapy will help restore some more; Pawash Priyank, whose son Ronnie was born with a fatal immune disorder but who, thanks to a stem cell/gene therapy treatment, is now healthy and leading a normal life.

Because of the pandemic everything was virtual, but it was no less compelling for that. We interviewed each of the patients or patient advocates beforehand and those videos kicked off each session. Hearing, and seeing, the patients and patient advocates tell their stories set the scene for what followed. It meant that the research the scientists talked about took on added significance. We now had faces and names to highlight the importance of the work the scientists were doing. We had human stories. And that gave a sense of urgency to the work the researchers were doing.

But that wasn’t all. After all the video presentations each session ended with a “live” panel discussion. And again, the patients and patient advocates were a key part of that. Because when scientists talk about taking their work into a clinical trial they need to know if the way they are setting up the trial is going to work for the patients they’re hoping to recruit. You can have the best scientists, the most promising therapy, but if you don’t design a clinical trial in a way that makes it easy for patients to be part of you won’t be able to recruit or retain the people you need to test the therapy.

Patient voices count. Patient stories count.

But more than anything, hearing and seeing the people we are trying to help reminds us why we do this work. It’s so easy to get caught up in the day to day business of our jobs, struggling to get an experiment to work, racing to get a grant application in before the deadline. Sometimes we get so caught up in the minutiae of work we lose sight of why we are doing it. Or who we are doing it for.

At CIRM we have a saying; come to work every day as if lives depend on you, because lives depend on you. Listening to the voices of patients, seeing their faces, hearing their stories, reminds us not to waste a moment. Because lives depend on all of us.

Kevin McCormack,
Senior Director Public Communications & Patient Advocate Outreach, CIRM

Browse more programs in Patient Perspective – CIRM Grantee Meeting 2020.

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3D Printing with Stem Cells

Transplants are expensive and risky, and donor organs are in short supply. Researchers at UC San Diego are working on technology to change all of that. It’s called bioprinting. In simple terms, bioprinting is 3D printing with living tissue. Researcher Shaochen Chen has been perfecting the process in his lab for years.

Bioprinting is a complex process that takes place in a matter of seconds right before your eyes. Chen’s lab builds their own printing machines, which shine light into a gel the team has developed. Any spot the light hits becomes solid. Because the process uses light, it allows the team to recreate microscopic structures like liver cells or vascular networks with incredible precision.

While the process enables researchers to accurately reproduce biological structures, it’s what’s inside the gel that makes bioprinting truly remarkable. The gel can be filled with stem cells from a potential transplant recipient. Those cells can fuse with tissue in the body as the gel disintegrates, essentially repairing damage with the patient’s own cells. Chen’s lab has shown the process can work in rats with severe spinal cord injuries. Someday, the process could be used in humans to do the same.

Bioprinting is also helpful to researchers in other fields. Chen has teamed up with Alysson Muotri and Karl Wahlin to help them study the connection between the eye and the brain. Their labs are conducting research using organoids – tiny organ-like structures grown from stem cells. They realized in order to effectively study how brain and retinal organoids interact with one another, they need to physically separate them at just the right distance, similar to how they might be separated in the womb. Chen’s lab developed a bioprinted structure to achieve that separation, taking the partnership to the next level.

Watch — 3D Printing with Stem Cells – Shaochen Chen

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Searching for a Cure for Blindness

There are a number of diseases that can lead to blindness. But, a researcher at UC San Diego thinks there might be one way to cure them all. It’s called endogenous regeneration. Think of a lizard re-growing a lost tail. Zebrafish can do something similar with retinal tissue. Researcher Karl Wahlin says there is evidence humans have the potential to do the same, if scientists can figure out how to activate the process.

Wahlin’s work isn’t limited to teaching the body to repair itself. He’s also using stem cells to study different eye diseases and search for cures. He works with what are known as retinal organoids – miniature retinal models grown in the lab. These can be made from stem cells of people with specific eye diseases so researchers can see how those diseases might develop in the womb, and which treatments might be effective against them.

Now, Wahlin is teaming up with Alysson Muotri from the UC San Diego Stem Cell program who uses brain organoids for similar research. The two have begun working together with the help of a bioengineer who builds 3D-printing machines that can incorporate stem cells. Learn how it all works in the latest piece from the Building the Brain Series.

Watch — Stem Cells and Curing Blindness – Karl Wahlin

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Building the Brain With Alysson Muotri

Inside of each brain, there is the possibility to understand how trillions of neural connections come to sense the world, record memories, create an individual, and shape who you are and who you will become. Can we ever learn how this happens?

By using cortical organoids – self organized clusters of neurons generated by stem cells, that is what Alysson Muotri’s lab at the Sanford Consortium for regenerative medicine wants to learn.

Called “brain organoids” because they exhibit many of the characteristics of a developing brain they are asking what happens to build a brain? What happens to create a human mind, and who we are? How does this process become disordered, giving rise to conditions like autism, schizophrenia, epilepsy, and degeneration? And how can they find ways to intervene and rescue the mind from disorders, and even restore lost function?

Muotri’s lab and a host of collaborators in and out of UC San Diego are using a diverse array of methods and tools on these brain organoids, from researching the details of vision to how neurons connect and form networks, to engineering ways to help the organoids become more complex, to the differences between normal brains and brains with cognitive disorders, even to growing brain organoids in space to understand causes of autism and Alzheimer’s disease.

Join Alysson Muotri, Director of the UC San Diego Stem Cell Program as he takes you on a journey to visit the labs and collaborators who are exploring how a brain is built on Building the Brain.

Watch — Building The Brain With Alysson Muotri

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