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.

The Top CIRM Blogs of 2019

This year the most widely read blog was actually one we wrote back in 2018. It’s the transcript of a Facebook Live: “Ask the Stem Cell Team” event about strokes and stroke recovery. Because stroke is the third leading cause of death and disability in the US it’s probably no surprise this blog has lasting power. So many people are hoping that stem cells will help them recover from a stroke.

But of the blogs that we wrote and posted this year there’s a really interesting mix of topics.

The most read 2019 blog was about a potential breakthrough in the search for a treatment for type 1 diabetes (T1D).  Two researchers at UC San Francisco, Dr. Matthias Hebrok and Dr. Gopika Nair developed a new method of replacing the insulin-producing cells in the pancreas that are destroyed by type 1 diabetes. 

Dr. Matthias Hebrok
Dr. Gopika Nair

Dr. Hebrok described it as a big advance saying: “We can now generate insulin-producing cells that look and act a lot like the pancreatic beta cells you and I have in our bodies. This is a critical step towards our goal of creating cells that could be transplanted into patients with diabetes.”

It’s not too surprising a blog about type 1 diabetes was at the top. This condition affects around 1.25 million Americans, a huge audience for any potential breakthrough. However, the blog that was the second most read is the exact opposite. It is about a rare disease called cystinosis. How rare? Well, there are only around 500 children and young adults in the US, and just 2,000 worldwide diagnosed with this condition.  

It might be rare but its impact is devastating. A genetic mutation means children with this condition lack the ability to clear an amino acid – cysteine – from their body. The buildup of cysteine leads to damage to the kidneys, eyes, liver, muscles, pancreas and brain.

Dr. Stephanie Cherqui

UC San Diego researcher Dr. Stephanie Cherqui and her team are taking the patient’s own blood stem cells and, in the lab, genetically re-engineering them to correct the mutation, then returning the cells to the patient. It’s hoped this will create a new, healthy blood system free of the disease.

Dr. Cherqui says if it works, this could help not just people with cystinosis but a wide array of other disorders: “We were thrilled that the stem cells and gene therapy worked so well to prevent tissue degeneration in the mouse model of cystinosis. This discovery opened new perspectives in regenerative medicine and in the application to other genetic disorders. Our findings may deliver a completely new paradigm for the treatment of a wide assortment of diseases including kidney and other genetic disorders.”

Sickled cells

The third most read blog was about another rare disease, but one that has been getting a lot of media attention this past year. Sickle cell disease affects around 100,000 Americans, mostly African Americans. In November the Food and Drug Administration (FDA) approved Oxbryta, a new therapy that reduces the likelihood of blood cells becoming sickle shaped and clumping together – causing blockages in blood vessels.

But our blog focused on a stem cell approach that aims to cure the disease altogether. In many ways the researchers in this story are using a very similar approach to the one Dr. Cherqui is using for cystinosis. Genetically correcting the mutation that causes the problem, creating a new, healthy blood system free of the sickle shaped blood cells.

Two other blogs deserve honorable mentions here as well. The first is the story of James O’Brien who lost the sight in his right eye when he was 18 years old and now, 25 years later, has had it restored thanks to stem cells.

The fifth most popular blog of the year was another one about type 1 diabetes. This piece focused on the news that the CIRM Board had awarded more than $11 million to Dr. Peter Stock at UC San Francisco for a clinical trial for T1D. His approach is transplanting donor pancreatic islets and parathyroid glands into patients, hoping this will restore the person’s ability to create their own insulin and control the disease.

2019 was certainly a busy year for CIRM. We are hoping that 2020 will prove equally busy and give us many new advances to write about. You will find them all here, on The Stem Cellar.

What would you like to know about stem cell research? This is your chance to ask the experts.

There’s a lot of fiction, a lot of misinformation surrounding stem cells and stem cell research. There are claims that are not based on solid science and clinics that are offering so-called “treatments” that are unproven, even dangerous for patients. Now you have a chance to talk to the experts in the field and get solid answers from them about what’s working, what’s not, and how you can find a therapy that might be appropriate for you.

Do you have questions about the latest in research using stem cells to help people recovering from a stroke? We’ll have someone who can answer them.

Want to know if stem cells can help people battling cancer? Or what’s happening in finding a stem cell treatment for diabetes or sickle cell disease, even autism, Alzheimer’s or Parkinson’s disease? We’ll have experts to answers those.

This is all happening in a special Facebook Live “Ask the Stem Cell Team” event on Thursday, December 12th from 10.30am to 11.30am PDT. To take part all you have to do is tune in on the day and post a question or you can send us one ahead of time at info@cirm.ca.gov

We will do our best to answer as many of them as we can during the Facebook Live event, and those we don’t have time to get to we’ll answer in a blog at a later date.

So join us.