What makes an expert an expert?

When we launched our Facebook Live “Ask the Expert” series earlier this year we wanted to create an opportunity for people to hear from and question experts about specific diseases or disorders. The experts we turned to were medical ones, neurologists and neuroscientists in the case of the first two Facebook Live events, stroke and ALS.

Then we learned about a blog post on the ALS Advocacy website questioning our use of the word “expert”. The author, Cathy Collet, points out that doctors or scientists are far from the only experts about these conditions, that there are many people who, by necessity, have become experts on a lot of issues relating to ALS and any other disease.

Cathy Collet ALS

 

Here’s Cathy’s blog. After you read it please let us know what you think: should we come up with a different title for the series, if so what would you suggest?

 

 

 

“Over the years I’ve experienced many “Ask the Experts” sessions related to ALS.  It’s always a panel of neuroscientists who talk a lot about ALS research and then take a few questions.

The “Expert” crown defaults to them.  They speak from the dais.  We get to listen a lot and ask.  They are by default “The Experts” in the fight against ALS.

But wait, there are all kinds of people with superb and valuable knowledge related to ALS –

  • There are people who know a lot about insurance.
  • There are people who know a lot about communication technology.
  • There are people who know a lot about low-tech hacks.
  • There are people who know a lot about suction machines.
  • There are people who know a lot about breathing.
  • There are people who know a lot about the FDA.
  • There are people who know a lot about moving a person on and off a commode.
  • There are people who know a lot about taxes.
  • There are people who know a lot about drugs.
  • There are people who know a lot about data.
  • There are people who know a lot about choking.
  • There are people who know a lot about financing research.
  • There are people who know a lot about stem cells.
  • There are people who know a lot about feeding tubes and nutrition.
  • There are people who know a lot about what’s important in living with the beast ALS.
  • There are people who know a lot about primary care in ALS.
  • There are people who know a lot about constipation.

Our default implication for the word experts being neuroscientists is revealing. There are many people in the fight against ALS, including those living with it, who know a lot.  We still live in a hierarchy where people with ALS and caregivers are at the bottom.

Words matter.  “Expert” is not a royal title to be owned by anyone by default.

It’s time for simple changes to some traditions.  “Ask the Neuroscientists,” anyone?

 

By the way, our next Facebook Live “Ask the ?” feature is targeting Sickle Cell Disease. It will be from noon till 1pm on Tuesday August 28th. More details, and maybe even a new name, to follow.

 

ALS is in the spotlight in CIRM’s “Ask the Expert About ALS & Stem Cells” Facebook Live event

The Catch

San Francisco 49ers Dwight Clark makes his iconic “Catch” against the Dallas Cowboys

American Football great Dwight Clark was renowned for having the safest hands in the game when he played for the San Francisco 49ers. But in September 2015 he was diagnosed with ALS (also known as Lou Gehrig’s disease) after not being able to use those hands to open a package of sugar. Less than three years later he was dead.

Amyotrophic lateral sclerosis – ALS’ formal title – is a nasty disease that relentlessly destroys the nerve cells in the brain and spinal cord that control movement and breathing. It is always fatal. There are only two drugs approved for ALS and they don’t work for most people. There is no cure.

AskExpertsALSJUL2018

That’s why CIRM chose ALS to be the subject of its latest Facebook Live Ask the Expert event (click here for the event’s FaceBook Live page). There’s a real need for new approaches to help people battling this deadly condition. And CIRM is funding two clinical trials that hope to do just that.

This Ask the Expert event will feature Clive Svendsen, PhD, Director of Cedars-Sinai’s Board of Governors Regenerative Medicine Institute, and Robert Baloh, MD, PhD, Director of Neuromuscular Medicine at Cedars-Sinai. They’ll be joined by Ralph Kern, MD, Chief Operating Officer and Chief Medical Officer at  BrainStorm Cell Therapeutics. The panel will be completed by CIRM Senior Science Officer Lila Collins.

The four will discuss the clinical trials that CIRM is funding with Cedars-Sinai and BrainStorm, and look at other promising research taking place.

Ask the Experts About ALS and Stem Cells is an opportunity for everyone in the ALS community to hear about the very latest in stem cell research targeting this devastating disease,” Svendsen said. “There has recently been some progress in the search for new treatments, which has energized all of us looking for effective therapies—and one day, a cure.”

Because Facebook Live is an interactive event people will be able to post comments and ask questions of the experts.

Dr. Baloh says we are now at a crucial time in the search for new approaches to help people with ALS.

“Many researchers believe that stem cells and gene therapies hold great promise for finding effective treatments, and more trials are needed to explore that potential.”

Our Facebook Live event, “Ask the Experts About ALS and Stem Cells” is tomorrow – Tuesday, July 31st – from noon till 1pm PST. You can join us by logging on to Facebook and going to the FaceBook Live broadcast link at: https://bit.ly/2uYQ8wM

Also, make sure to “like” our FaceBook page before the event to receive a notification when we’ve gone live for this and future events.

We want to hear from you, so you will be able to post questions in real-time for the experts to answer or, you can email them directly to us beforehand at info@cirm.ca.gov

If you miss the event, not to worry. A recording of the session will be available in our FaceBook videos page shortly after the broadcast ends.

We look forward to seeing you there.

 

Can stem cells help people recovering from a stroke? You asked, and the experts answered

FacebookLive_AskExperts_Stroke_IMG_1656

We recently held our first ever Facebook Live event. It was focused on the use of stem cells and recovery from a stroke and featured three great guests: Dr. Gary Steinberg, chief of Neurosurgery at Stanford, Sonia Coontz, a patient of Dr. Steinberg’s, and CIRM’s own Science Officer Dr. Lila Collins.

We had an amazing response from people during the event and in the days since then with some 6,750 people watching the video and almost 1,000 people reacting by posting a comment or sharing it with friends. It was one of the most successful things we have ever done on Facebook so it’s not surprising that we plan on doing many more Facebook Live ‘Ask the Expert’ events in the future. We will post more details of that as we finalize them.

We tried to cover as many topics as possible during the hour but there were simply too many questions for us to get to all of them. So here is a recap of the key issues we covered, and a few we didn’t have a chance to answer.

Let’s start with Dr. Steinberg’s explanation of the research that led to his current clinical trial:

Dr. Steinberg: “I got interested in this about 18 years ago when I took human cells and transplanted them into rodent models of stroke. What we found was that when we transplanted those cells into the stroke region, the core of the stroke, they didn’t survive very well but when we moved them a few millimeters away from the stroke they not only survived but they migrated to the stroke.

The reason they migrate is that the stem cells have receptors on them that interact with chemicals given off by the stroke environment and that’s why they migrate to the stroke site. And when they get to the site they can turn into different kinds of cells. Very importantly we found these mice and rats that had behavioral problems – walking, moving – as a result of the stroke, we found we could improve their neurological outcomes with the stem cells.

With the help of CIRM, which has been very generous, we were fortunate enough to receive about $24 million in funding over the last 8 years, from 2010, to move this therapy into the clinic to understand the basic mechanisms of the recovery and to start clinical trials

One of the surprising things was that our initial notion was that the cells we transplanted into the brains would initially turn into the cells in the brain affected by the stroke and reconstitute those circuits. We were shocked to find that that was not what was happening, that only a few of the transplanted cells turned into neurons. The way they were recovering function was by secreting very powerful growth factors and molecules and proteins that enhanced native recovery or the ability of the normal brain to recover itself. Some of these processes included outgrowth of neurons, new connections, new synapses, not from the stem cells but from the native cells already in the brain.

This is not cell replacement but enhancing native recovery and, in a simple sense, what the cells are doing, we believe, is to change the adult brain, which has a hard time recovering from a stroke, into an infant brain and infants recover very well after a stroke.”

All this work was focused on ischemic strokes, where a blockage cuts off blood flow to the brain. But people like Cheryl Ward wanted to know: “Will this work for hemorrhagic stroke?” That’s where a blood vessel in the brain leaks or ruptures.

Dr. Steinberg: “I suspect we will be generalizing this therapy into hemorrhagic patients very, very soon and there’s no reason why it shouldn’t work there. The reason we didn’t start there is that 85% of strokes are ischemic and only 15% are hemorrhagic so it’s a smaller population but a very, very important population because when patients have a hemorrhage from a stroke they are often more seriously disabled than from ischemic.”

Dr. Lila Collins: “I would like to highlight one trial for hemorrhagic stroke with the Mayo Clinic and that’s using mesenchymal stem cells (normally found in bone marrow or blood). It’s an early stage, Phase 1 safety study in patients with recent cerebral hemorrhage.  They are looking at improvements in neurological function and patients have to be treated within 72 hours after the stroke.”

Dr. Steinberg explained that because it’s more difficult to enroll patients within 72 hours of a stroke that we may end up offering a combination of therapies spread out over months or even years.

Dr. Steinberg: “It may be that and we may figure this out in the next 5 to 10 years, that you might want to treat patients acutely (right away) with an intravenous therapy in the first 72 hours and then you might want to come in again sub-acutely within a few months, injecting the cells into the brain near the stroke, and then maybe come in chronically a few years later if there are still problems and place the cells directly in the brain. So, lots of ways to think about how to use this in the future.”

James Russell suffered a stroke in 2014 and wrote:

“My left side was affected. My vision was also impacted. Are any stroke patients being given stem cells seeing possible improvement in visual neglect?”

Dr. Steinberg: “We don’t know the answer to that yet, it’s quite possible. It’s true these vision circuits are not dead and could be resurrected. We have not targeted visual pathways in our work, we have targeted motor functions, but I would also be optimistic that we could target patients who have vision problems from stroke. It’s a very important area.

A number of people wondered if stem cells can help people recovering from a stroke can they also help people with other neurological conditions.

Hanifa Gaphoor asked “What about Parkinson’s disease?” and Ginnievive Patch wondered “Do you feel hopeful for neurological illnesses like Huntington’s disease and ALS? Dr. Steinberg was cautiously optimistic.

Dr. Steinberg: “We’ve extended this kind of treatment not just for ischemic stroke but into traumatic brain injury (TBI) and we just completed a trial for patients with chronic TBI or who have suffered a trauma to the brain. Many other indications may be possible. In fact, now that we know these circuits are not dead or irreversibly injured, we believe we could even extend this to neurodegenerative diseases like ALS, Parkinson’s, maybe even to Alzheimer’s disease in the future. So, lots of hope but we don’t want to oversell this, and we want to make sure this is done in a rigorous fashion.”

Several people had questions about using their own adipose, or fat stem cells, in therapies being offered at clinics around the US and in other countries. Cheri Hicks asked: “I’m curious if adipose stem cell being used at clinics at various places is helpful or beneficial?”

Dr. Steinberg: “I get emails or calls from patients every week saying should I go to Russia, India or Mexico and get stem cell transplants which are done not as part of a rigorous trial and I discourage patients from getting stem cells that are not being given in a controlled fashion. For one thing, patients have been getting hurt by these treatments in these clinics; they have developed tumors and infections and other problems. In many cases we don’t even know what the cells are, there’s not published information and the patients pay cash for this, of course.”

At CIRM we also worry about people going to clinics, in the US and in other countries, where they are getting therapies that have not been approved by the US Food and Drug Administration (FDA) or other appropriate regulatory bodies. That’s why we have created this page on our website to help people who want a stem cell therapy but don’t know what to look for in a clinical trial or what questions to ask to make sure it’s a legitimate trial, one that’s been given the go-ahead by the FDA.

Bret Ryan asked: “What becomes of the implanted cells?”

Dr. Steinberg: We found after transplanting the cells, one week after the transplant, we see a new abnormality in the premotor cortex, the area of the brain that controls motor function. We saw a new abnormality there or a new signal that disappears after a month and never comes back. But the size of that temporary abnormality after one week correlates very closely with the degree of recovery after six months, one year and two years.

One of the interesting things is that it doesn’t seem to be necessary for the cells to survive long term to have beneficial effects. The cells we used in the SanBio trial don’t survive more than a month and yet they seem to aid recovery function in our pilot studies which is sustained for years.”

And of course, many people, such as Karen Smart, wanted to know how they could get the therapy. Right now, the clinical trial is fully enrolled but Stanford is putting together a waiting list for future trials. If you are interested and would like more information, please email: stemcellstudy@stanford.edu.

Sonia Coontz, the patient who was also a key part of the Facebook Live event, has an amazing story to tell. She was left devastated, physically and emotionally, after having a stroke. But then she heard about Dr. Steinberg’s clinical trial and it changed her life. Here’s her story.

We were thrilled to receive all of your comments and questions during our first Facebook Live event. It’s this kind of dialogue between scientists, patients and the public that will be critical for the continued support of our mission to accelerate stem cell treatments to patients with unmet medical needs.

Due to the response, we plan to regularly schedule these “Ask the Expert” events. What disease area would you like us to focus on next time? Leave us a comment or email info@cirm.ca.gov

 

‘Ask The Expert’ on Facebook Live about the power of stem cells to reverse damage caused by a stroke.

facebook-live-brand-awarenessIt’s not often you get a chance to ask a world class stem cell expert a question about their work, and how it might help you or someone you love. But on Thursday, May 31 you can do just that.

CIRM is hosting a special ‘Ask the Expert’ event on Facebook Live. The topic is Strokes and Stem Cells. Just head over to our Facebook Page on May 31st from noon till 1pm PST to experience it live. You can also re-watch the event any time after the broadcast has ended from our Facebook videos page.

Steinberg

We will be joined by Dr. Gary Steinberg, chair of neurosurgery at Stanford University, who will talk to us about his work in helping reverse the damage caused by a stroke, even for people who experienced a brain attack several years ago.

CIRM Senior Science Officer, Dr. Lila Collins, will 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.

We’ll post reminders on Facebook so make sure to follow us. But for now, mark the date and time on your diary and please feel free to share this information with anyone you think might be interested.

It promises to be a fascinating event.