Charting a new course for stem cell research

What are the latest advances in stem cell research targeting cancer? Can stem cells help people battling COVID-19 or even help develop a vaccine to stop the virus? What are researchers and the scientific community doing to help address the unmet medical needs of underserved communities? Those are just a few of the topics being discussed at the Annual CIRM Alpha Stem Cell Clinic Network Symposium on Thursday, October 8th from 9am to 1.30pm PDT.

Like pretty nearly everything these days the symposium is going to be a virtual event, so you can watch it from the comfort of your own home on a phone or laptop. And it’s free.

The CIRM Alpha Clinics are a network of leading medical centers here in California. They specialize in delivering stem cell and gene therapies to patients. So, while many conferences look at the promise of stem cell therapies, here we deal with the reality; what’s in the clinic, what’s working, what do we need to do to help get these therapies to patients in need?

It’s a relatively short meeting, with short presentations, but that doesn’t mean it will be short on content. Some of the best stem cell researchers in the U.S. are taking part so you’ll learn an awful lot in a short time.

We’ll hear what’s being done to find therapies for

  • Rare diseases that affect children
  • Type 1 diabetes
  • HIV/AIDS
  • Glioblastoma
  • Multiple myeloma

We’ll discuss how to create a patient navigation system that can address social and economic determinants that impact patient participation? And we’ll look at ways that the Alpha Clinic Network can partner with community care givers around California to increase patient access to the latest therapies.

It’s going to be a fascinating day. And did I mention it’s free!

All you have to do is go to this Eventbrite page to register.

And feel free to share this with your family, friends or anyone you think might be interested.

We look forward to seeing you there.

Meet the people who are changing the future

Kristin MacDonald

Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day.

I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial to treat retinitis pigmentosa (RP). The disease had destroyed Kristin’s vision and she was hoping the therapy, pioneered by jCyte, would help her. Kristin, being a bit of a pioneer herself, was the first person to test the therapy in the U.S.

Anyway, Kristin was doing a Zoom presentation and wanted to look her best so she asked a friend to come over and do her hair and makeup. The woman she asked, was Rosie Barrero, another patient in that RP clinical trial. Not so very long ago Rosie was legally blind. Now, here she was helping do her friend’s hair and makeup. And doing it beautifully too.

That’s when you know the treatment works. At least for Rosie.

There are many other stories to be heard – from patients and patient advocates, from researchers who develop therapies to the doctors who deliver them. – at our CIRM 2020 Grantee Meeting on next Monday September 14th Tuesday & September 15th.

It’s two full days of presentations and discussions on everything from heart disease and cancer, to COVID-19, Alzheimer’s, Parkinson’s and spina bifida. Here’s a link to the Eventbrite page where you can find out more about the event and also register to be part of it.

Like pretty much everything these days it’s a virtual event so you’ll be able to join in from the comfort of your kitchen, living room, even the backyard.

And it’s free!

You can join us for all two days or just one session on one day. The choice is yours. And feel free to tell your friends or anyone else you think might be interested.

We hope to see you there.

Stem Cell All-Stars, All For You

goldstein-larry

Dr. Larry Goldstein, UC San Diego

It’s not often you get a chance to hear some of the brightest minds around talk about their stem cell research and what it could mean for you, me and everyone else. That’s why we’re delighted to be bringing some of the sharpest tools in the stem cell shed together in one – virtual – place for our CIRM 2020 Grantee Meeting.

The event is Monday September 14th and Tuesday September 15th. It’s open to anyone who wants to attend and, of course, it’s all being held online so you can watch from the comfort of your own living room, or garden, or wherever you like. And, of course, it’s free.

BotaDaniela2261

Dr. Daniela Bota, UC Irvine

The list of speakers is a Who’s Who of researchers that CIRM has funded and who also happen to be among the leaders in the field. Not surprising as California is a global center for regenerative medicine. And you will of course be able to post questions for them to answer.

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Dr. Deepak Srivastava, Gladstone Institutes

The key speakers include:

Larry Goldstein: the founder and director of the UCSD Stem Cell Program talking about Alzheimer’s research

Irv Weissman: Stanford University talking about anti-cancer therapies

Daniela Bota: UC Irvine talking about COVID-19 research

Deepak Srivastava: Gladsone Institutes, talking about heart stem cells

Other topics include the latest stem cell approaches to COVID-19, spinal cord injury, blindness, Parkinson’s disease, immune disorders, spina bifida and other pediatric disorders.

You can choose one topic or come both days for all the sessions. To see the agenda for each day click here. Just one side note, this is still a work in progress so some of the sessions have not been finalized yet.

And when you are ready to register go to our Eventbrite page. It’s simple, it’s fast and it will guarantee you’ll be able to be part of this event.

We look forward to seeing you there.

Perseverance: from theory to therapy. Our story over the last year – and a half

Some of the stars of our Annual Report

It’s been a long time coming. Eighteen months to be precise. Which is a peculiarly long time for an Annual Report. The world is certainly a very different place today than when we started, and yet our core mission hasn’t changed at all, except to spring into action to make our own contribution to fighting the coronavirus.

This latest CIRM Annual Reportcovers 2019 through June 30, 2020. Why? Well, as you probably know we are running out of money and could be funding our last new awards by the end of this year. So, we wanted to produce as complete a picture of our achievements as we could – keeping in mind that we might not be around to produce a report next year.

Dr. Catriona Jamieson, UC San Diego physician and researcher

It’s a pretty jam-packed report. It covers everything from the 14 new clinical trials we have funded this year, including three specifically focused on COVID-19. It looks at the extraordinary researchers that we fund and the progress they have made, and the billions of additional dollars our funding has helped leverage for California. But at the heart of it, and at the heart of everything we do, are the patients. They’re the reason we are here. They are the reason we do what we do.

Byron Jenkins, former Naval fighter pilot who battled back from his own fight with multiple myeloma

There are stories of people like Byron Jenkins who almost died from multiple myeloma but is now back leading a full, active life with his family thanks to a CIRM-funded therapy with Poseida. There is Jordan Janz, a young man who once depended on taking 56 pills a day to keep his rare disease, cystinosis, under control but is now hoping a stem cell therapy developed by Dr. Stephanie Cherqui and her team at UC San Diego will make that something of the past.

Jordan Janz and Dr. Stephanie Cherqui

These individuals are remarkable on so many levels, not the least because they were willing to be among the first people ever to try these therapies. They are pioneers in every sense of the word.

Sneha Santosh, former CIRM Bridges student and now a researcher with Novo Nordisk

There is a lot of information in the report, charting the work we have done over the last 18 months. But it’s also a celebration of everyone who made it possible, and our way of saying thank you to the people of California who gave us this incredible honor and opportunity to do this work.

We hope you enjoy it.

Stem Cells for Parkinson’s Disease

While the world has been turned upside down by the coronavirus pandemic, the virus poses an increased threat to people with Parkinson’s disease (PD). Having a compromised immune system, particularly involving the lungs, means people with PD are at higher risk of some of the more dangerous complications of COVID-19. So, this seems like an appropriate time for CIRM to hold a special Facebook Live “Ask the Stem Cell Team” About Parkinson’s disease.

We are holding the event on Tuesday, May 5th at noon PDT.

The initial reason for the Facebook Live was the CIRM Board approving almost $8 million for Dr. Krystof Bankiewicz at Brain Neurotherapy Bio, Inc. to run a Phase 1 clinical trial targeting PD. Dr. Bankiewicz is using a gene therapy approach to promote the production of a protein called GDNF, which is best known for its ability to protect dopaminergic neurons, the kind of cell damaged by Parkinson’s. The approach seeks to increase dopamine production in the brain, alleviating PD symptoms and potentially slowing down the disease progress.

Dr. Bankiewicz will be joined by two of CIRM’s fine Science Officers, Dr. Lila Collins and Dr. Kent Fitzgerald. They’ll talk about the research targeting Parkinson’s that CIRM is funding plus other promising research taking place.

And we are delighted to have a late addition to the team. Our CIRM Board member and patient advocate for Parkinson’s disease, Dr. David Higgins. David has a long history of advocacy for PD and adds the invaluable perspective of someone living with PD.

As always, we want this to be as interactive as possible, so we want to get your questions. You can do this on the day, posting them alongside the live feed, or you can send them to us ahead of time at info@cirm.ca.gov. We’ll do our best to answer as many as we can on the day, and those we don’t get to during the broadcast we’ll answer in a later blog.

We look forward to seeing you there.

Ask the Stem Cell Team About Autism

On March 19th we held a special Facebook Live “Ask the Stem Cell Team About Autism” event. We were fortunate enough to have two great experts – Dr. Alysson Muotri from UC San Diego, and CIRM’s own Dr. Kelly Shepard. As always there is a lot of ground to cover in under one hour and there are inevitably questions we didn’t get a chance to respond to. So, Dr. Shepard has kindly agreed to provide answers to all the key questions we got on the day.

If you didn’t get a chance to see the event you can watch the video here. And feel free to share the link, and this blog, with anyone you think might be interested in the material.

Dr. Kelly Shepard

Can umbilical cord blood stem cells help reduce some of the symptoms?

This question was addressed by Dr. Muotri in the live presentation. To recap, a couple of clinical studies have been reported from scientists at Duke University and Sutter Health, but the results are not universally viewed as conclusive.  The Duke study, which focused on very young children, reported some improvements in behavior for some of the children after treatment, but it is important to note that this trial had no placebo control, so it is not clear that those patients would not have improved on their own. The Duke team has moved forward with larger trial and placebo control.

Does it have to be the child’s own cord blood or could donated blood work too?

In theory, a donated cord product could be used for similar purposes as a child’s own cord, but there is a caveat- the donated cord tissues must have some level of immune matching with the host in order to not be rejected or lead to other complications, which under certain circumstances, could be serious.

Some clinics claim that the use of fetal stem cells can help stimulate improved blood and oxygen flow to the brain. Could that help children with autism?

Fetal stem cells have been tested in FDA approved/sanctioned clinical trials for certain brain conditions such as stroke and Parkinson Disease, where there is clearer understanding of how and which parts of the brains are affected, which nerve cells have been lost or damaged, and where there is a compelling biological rationale for how certain properties the transplanted cells, such as their anti-inflammatory properties, could provide benefit.

Alysson Muotri in his lab and office at Sanford Consortium in La Jolla, California; Photograph by David Ahntholz http://www.twopointpictures.com http://www.davidahntholz.com

In his presentation, Dr. Muotri noted that neurons are not lost in autistic brains, so there is nothing that would be “replaced” by such a treatment. And although some forms of autism might include inflammation that could potentially be mitigated, it is unlikely that  the degree of benefit that might come from reducing inflammation would be worth the risks of the treatment, which includes intracranial injection of donated material.  Unfortunately, we still do not know enough about the specific causes and features of autism to determine if and to what extent stem cell treatments could prove helpful. But we are learning more every day, especially with some of the new technologies and discoveries that have been enabled by stem cell technology. 

Some therapies even use tissue from sheep claiming that a pill containing sheep pancreas can migrate to and cure a human pancreas, pills containing sheep brains can help heal human brains. What are your thoughts on those?

For some conditions, there may be a scientific rationale for how a specific drug or treatment could be delivered orally, but this really depends on the underlying biology of the condition, the means by which the drug exerts its effect, and how quickly that drug or substance will be digested, metabolized, or cleared from the body’s circulation. Many drugs that are delivered orally do not reach the brain because of the blood-brain barrier, which serves to isolate and protect the brain from potentially harmful substances in the blood circulation. For such a drug to be effective, it would have to be stable within the body for a period of time, and be something that could exert its effects on the brain either directly or indirectly.

Sheep brain or pancreas (or any other animal tissue consumed) in a pill form would be broken down into basic components immediately by digestion, i.e. amino acids, sugars, much like any other meat or food. Often complex treatments designed to be specifically targeted to the brain are delivered by intra-cranial/intrathecal injection, or by developing special strategies to evade the blood brain barrier, a challenge that is easier said than done. For autism, there is still a lot to be learned regarding how a therapeutic intervention might work to help people, so for now, I would caution against the use of dietary supplements or pills that are not prescribed or recommended by your doctor. 

What are the questions parents should ask before signing up for any stem cell therapy

There is some very good advice about this on the both the CIRM and ISSCR websites, including a handbook for patients that includes questions to ask anyone offering you a stem cell treatment, and also some fundamental facts that everyone should know about stem cells. https://www.closerlookatstemcells.org/patient-resources/

What kinds of techniques do we have now that we didn’t have in the past that can help us better understand what is happening in the brain of a child with autism.

We covered this in the online presentation. Some of the technologies discussed include:

– “disease in a dish” models from patient derived stem cells for studying causes of autism

–  new ways to make human neurons and other cell types for study

– organoid technology, to create more realistic brain tissues for studying autism

– advances in genomics and sequencing technologies to identify “signatures” of autism to help identify the underlying differences that could lead to a diagnosis

Alysson, you work with things called “brain organoids” explain what those are and could they help us in uncovering clues to the cause of autism and even possible therapies?

We blogged about this work when it was first published and you can read about it on our blog here.

Can stem cells help people who have had a stroke? Ask the experts.

Stroke is the third leading cause of death and disability in the US. Every 45 seconds someone in the US has a stroke. Every year around 275,000 people die from a stroke many more survive but are often impaired by the brain attack. The impact is not just physical, but psychological and emotional. It takes an enormous toll on individuals and their families. So, it’s not surprising that there is a lot of research underway to try and find treatments to help people, including using stem cells.

That’s why CIRM is hosting a special Facebook Live ‘Ask the Stem Cell Team About Stroke event on Wednesday, March 25th at noon PDT. Just head over to our Facebook Page on the 25th at noon to hear from two great guests.

We will be joined by Dr. Tom Carmichael, a Professor of Neurology and the Co-Director of the UCLA Broad Stem Cell Center. He has a number of CIRM grants focused on helping repair the damage caused by strokes.

CIRM Senior Science Officer, Dr. Lila Collins, will also join us to talk about other stem cell research targeting stroke, its promise and some of the problems that still need to be overcome.

You will have a chance to ask questions of both our experts, either live on the day or by sending us questions in advance at info@cirm.ca.gov.

Why “Ask the Stem Cell Team” Remains Important

These are definitely strange, unusual and challenging times. Every day seems to bring new restrictions on what we can and should do. All, of course, in the name of protecting us and helping us avoid a potentially deadly virus. We all hope this will soon pass but we also know the bigger impact of the coronavirus is likely to linger for many months, perhaps even years.

With that in mind a few people have asked us why we are still going ahead with our Facebook Live ‘Ask the Stem Cell Team About Autism’ event this Thursday, March 19th at 12pm PDT. It’s a good question. And the answer is simple. Because there is still a need for good, thoughtful information about the potential for stem cells to help families who have a loved one with autism. And because we still need to do all we can to dispel the bad information out there and warn people about the bogus clinics offering unproven therapies.

In many ways Facebook Live is the perfect way to deliver this information. It allows us to reach out to large numbers of people without having them in the same room. We can educate not contaminate.

And we have some great experts to discuss the use of stem cells in helping people with autism.

The event features Dr. Alysson Muotri from UC San Diego. We have written about his work with stem cells for autism in the past. And CIRM’s own Associate Director for Discovery and Translation, Dr. Kelly Shepard.

But we also want you to be a part of this as well. So, join us online for the event. You can post comments and questions during the event, and we’ll do our best to answer them. Or you can send us in questions ahead of time to info@cirm.ca.gov.

If you were unable to tune in while we were live, not to worry, you you can watch it here on our Facebook page

Enabling the Best Choice for Patients: The Need for Effective Patient Navigation

Making sure patients get the treatment they need and not a “snake oil” substitute

We are at a turning point in regenerative medicine as the first wave of treatments have obtained FDA approval. But at the same time as we see the advance of scientifically rigorous research and regulated products we are also witnessing the continued proliferation of “unproven treatments.” This dueling environment can be overwhelming and distracting to individuals and families trying to manage life-threatening diseases.

How does a patient navigate this environment and get trusted and reliable information to help sort through their options?

CIRM teamed up with the CURA Foundation to organize a roundtable discussion intended to answer this question. The conversation included thought leaders involved in patient advocacy, therapy research and development, public policy and research funding. The roundtable was divided into three segments designed to discuss:

  1. Examples of state-of-the-art patient navigation systems,
  2. Policy, research and infrastructure needs required to expand navigation systems, and
  3. Communication needs for engaging patients and the broader community.

Examples of Navigation Systems:

This session was framed around the observation that patients often do not get the best medicines or treatments available for their condition. For example, in the area of cancer care there is evidence that the top 25% of cancers are not being treated optimally. Historic barriers to optimal treatment include cost pressures that may block access to treatments, lack of knowledge about the available treatments or the absence of experts in the location where the patient is being treated.  Much of the session focused on how these barriers are being overcome by partnerships between health care provides, employers and patients.

For example, new technologies such as DNA sequencing and other cell-based markers enable better diagnosis of a patient’s underlying disease. This information can be collected by a community hospital and shared with experts who work with the treating doctor to consider the best options for the patient. If patients need to access a specialty center for treatment, there are new models for the delivery of such care. Emphasis is placed on building a relationship with the patient and their family by surrounding them with a team that can address any questions that arise. The model of patient-centered care is being embraced by employers who are purchasing suites of services for their employees.

Patient advocacy groups have also supported efforts to get the best information about the patients’ underlying disease. Advocacy organizations have been building tools to connect patients with researchers with the aim of allowing secure and responsible sharing of medical information to drive the patient-centered development of new treatments. In a related initiative, the American Society of Hematology is creating a data hub for clinical trials for sickle cell disease. Collectively, these efforts are designed to accelerate new treatments by allowing critical data to be shared among researchers.

Essential Policy Infrastructure for Regenerative Medicine:

Session two dovetailed nicely with first discussion. There was continued emphasis on the need for additional evidence (data) to demonstrate that regenerative medicine treatments are having a significant effect on the patient’s disease. Various speakers echoed the need for patients in clinical trials to work with researchers to determine the benefits of treatments. Success stories with gene therapies in blood diseases were cited as proof of concept where treatments being evaluated in clinical trials are demonstrating a significant and sustained impact on diseases. Evidence of benefit is needed by both regulatory bodies that approve the treatments, such as the FDA, and by public and private payers / insurers that pay for treatments and patients that need to know the best option for their particular disease.

In addition, various speakers cited the continued proliferation of “unproven treatments” being marketed by for-profit centers. There was broad concern that the promotion of treatment where there is no evidence of effectiveness will mislead some patients and potentially harm the scientifically rigorous development of new treatments. Particularly for “stem cell” treatments, there was a desire to develop evaluation criteria that are clear and transparent to allow legitimate treatments to be distinguished from those with no evidence of effectiveness. One participant suggested there be a scorecard approach where specific treatments could be rated against specific indicators of safety, medical benefit and value in relation to alternative treatments. The idea would be to make this information widely available to patients, medical providers and the public to inform everything from medical decision making to advertising.

Communicating the Vision

The final session considered communication needs for the field of regenerative medicine. Patients and patient advocacy organizations described how they are using social media and other networking tools to share information and experiences in navigating their treatment options. Patient advocacy groups also described the challenges from providers of unproven treatments. In one case, a for profit “pop up” clinic had used the group’s videos in an attempt to legitimize their unproven treatment.

There was general consensus among the panelists that the field of regenerative medicine needs “trusted intermediaries” who can evaluate claims and help patients distinguish between high quality research and “snake oil”. These intermediaries should have the capacity to compile the most reliable evidence and utilize it to determine what options are available to patients. In addition, there needs to be shared decision making model where patients have the opportunity to explore options in an unbiased environment so they may make the best decision based on their specific needs and values.

Creating this kind of Navigation System will not be easy but the alternative is unacceptable. Too many vulnerable patients are being taken advantage of by the growing number of “predatory clinics” hawking expensive therapies that are both unproven and unapproved. We owe it to these patients to create a simple way for them to identify what are the most promising therapies, ones that have the highest chance of being both safe and effective. The roundtable discussion marked a starting point, bringing together many of the key players in the field, highlighting the key issues and beginning to identify possible solutions.

Next generation of stem cell scientists leave their mark

One of the favorite events of the year for the team here at CIRM is our annual SPARK (Summer Program to Accelerate Regenerative Medicine Knowledge) conference. This is where high school students, who spent the summer interning at world class stem cell research facilities around California, get to show what they learned. It’s always an engaging, enlightening, and even rather humbling experience.

The students, many of whom are first generation Californians, start out knowing next to nothing about stem cells and end up talking as if they were getting ready for a PhD. Most say they went to their labs nervous about what lay ahead and half expecting to do menial tasks such as rinsing out beakers. Instead they were given a lab coat, safety glasses, stem cells and a specific project to work on. They learned how to handle complicated machinery and do complex scientific experiments.

But most importantly they learned that science is fun, fascinating, frustrating sometimes, but also fulfilling. And they learned that this could be a future career for them.

We asked all the students to blog about their experiences and the results were extraordinary. All talked about their experiences in the lab, but some went beyond and tied their internship to their own lives, their past and their hopes for the future.

Judging the blogs was a tough assignment, deciding who is the best of a great bunch wasn’t easy. But in the end, we picked three students who we thought captured the essence of the SPARK program. This week we’ll run all those blogs.

We begin with our third place blog by Dayita Biswas from UC Davis.

Personal Renaissance: A Journey from Scientific Curiosity to Confirmed Passions

By Dayita Biswas

As I poured over the pages of my battered Campbell textbook, the veritable bible for any biology student, I saw unbelievable numbers like how the human body is comprised of over 30 trillion cells! Or how we have over 220 different types of cells— contrary to my mental picture of a cell as a circle. Science, and biology in particular, has no shortage of these seemingly impossible Fermi-esque statistics that make one do a double-take. 

My experience in science had always been studying from numerous textbooks in preparation for a test or competitions, but textbooks only teach so much. The countless hours I spent reading actually demotivated me and I constantly asked myself what was the point of learning about this cycle or that process — the overwhelming “so what?” question. Those intriguing numbers that piqued my interest were quickly buried under a load of other information that made science a static stream of words across a page. 

That all changed this summer when I had the incredible opportunity to work in the Nolta lab under my mentor, Whitney Cary. This internship made science so much more tangible and fun to be a part of.  It was such an amazing environment, being in the same space with people who all have the same goals and passion for science that many high school students are not able to truly experience. Everyone was so willing to explain what they were doing, and even went out of their way to help if I needed papers or had dumb questions.

This summer, my project was to create embryoid bodies and characterize induced pluripotent stem cells (iPSCs) from children who had Jordan’s Syndrome, an extremely rare neurodevelopmental disease whose research has applications in Alzheimer’s and autism.

 I had many highs and lows during this research experience. My highs were seeing that my iPSCs were happy and healthy. I enjoyed learning lab techniques like micro-pipetting, working in a biological safety hood, feeding, freezing, and passaging cells. My lows were having to bleach my beloved iPSCs days after they failed to survive, and having unsuccessful protocols. However, while my project consistently failed, these failures taught me more than my successes.

I learned that there is a large gap between being able to read about techniques and being “book smart” and actually being able to think critically about science and perform research. Science, true science, is more than words on a page or fun facts to spout at a party. Science is never a straight or easy answer, but the mystery and difficulty is part of the reason it is so interesting. Long story short: research is hard and it takes time and patience, it involves coming in on weekends to feed cells, and staying up late at night reading papers.         

The most lasting impact that this summer research experience had was that everything we learn in school and the lab are all moving us towards the goal of helping real people. This internship renewed my passion for biology and cemented my dream of working in this field. It showed me that I don’t have to wait to be a part of dynamic science and that I can be a small part of something that will change, benefit, and save lives.

This internship meant being a part of something bigger than myself, something meaningful. We must always think critically about what consequences our actions will have because what we do as scientists and researchers— and human beings will affect the lives of real people. And that is the most important lesson anyone can hope to learn.

                                                                                                   

And here’s a bonus, a video put together by the SPARK students at Cedars-Sinai Medical Center.