CIRM funded researchers discover link between Alzheimer’s gene and COVID-19

Dr. Yanhong Shi (left) and Dr. Vaithilingaraja Arumugaswami (right)

All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. Today we focus on groundbreaking CIRM funded research related to COVID-19 that was recently published.

It’s been almost a year since the world started hearing about SARS-CoV-2, the virus that causes COVID-19.  In our minds, the pandemic has felt like an eternity, but scientists are still discovering new things about how the virus works and if genetics might play a role in the severity of the virus.  One population study found that people who have ApoE4, a gene type that has been found to increase the risk of developing Alzheimer’s, had higher rates of severe COVID-19 and hospitalizations.

It is this interesting observation that led to important findings of a study funded by two CIRM awards ($7.4M grant and $250K grant) and conducted by Dr. Yanhong Shi at City of Hope and co-led by Dr. Vaithilingaraja Arumugaswami, a member of the UCLA Broad Stem Cell Research Center.  The team found that the same gene that increases the risk for Alzheimer’s disease can increase the susceptibility and severity of COVID-19.

At the beginning of the study, the team was interested in the connection between SARS-CoV-2 and its effect on the brain.  Due to the fact that patients typically lose their sense of taste and smell, the team theorized that there was an underlying neurological effect of the virus.  

The team first created neurons and astrocytes.  Neurons are cells that function as the basic working unit of the brain and astrocytes provide support to them.  The neurons and astrocytes were generated from induced pluripotent stem cells (iPSCs), which are a kind of stem cell that can become virtually any type of cell and can be created by “reprogramming” the skin cells of patients.  The newly created neurons and astrocytes were then infected with SARS-CoV-2 and it was found that they were susceptible to infection.

Next, the team used iPSCs to create brain organoids, which are 3D models that mimic certain features of the human brain.  They were able to create two different organoid models: one that contained astrocytes and one without them.  They infected both brain organoid types with the virus and discovered that those with astrocytes boosted SARS-CoV-2 infection in the brain model. 

The team then decided to further study the effects of ApoE4 on susceptibility to SARS-CoV-2.  They did this by generating neurons from iPSCs “reprogrammed” from the cells of an Alzheimer’s patient.  Because the iPSCs were derived from an Alzheimer’s patient, they contained ApoE4.  Using gene editing, the team modified some of the ApoE4 iPSCs created so that they contained ApoE3, which is a gene type considered neutral.  The ApoE3 and ApoE4 iPSCs were then used to generate neurons and astrocytes.

The results were astounding.  The ApoE4 neurons and astrocytes both showed a higher susceptibility to SARS-CoV-2 infection in comparison to the ApoE3 neurons and astrocytes.  Moreover, while the virus caused damage to both ApoE3 and ApoE4 neurons, it appeared to have a slightly more severe effect on ApoE4 neurons and a much more severe effect on ApoE4 astrocytes compared to ApoE3 neurons and astrocytes. 

“Our study provides a causal link between the Alzheimer’s disease risk factor ApoE4 and COVID-19 and explains why some (e.g. ApoE4 carriers) but not all COVID-19 patients exhibit neurological manifestations” says Dr. Shi. “Understanding how risk factors for neurodegenerative diseases impact COVID-19 susceptibility and severity will help us to better cope with COVID-19 and its potential long-term effects in different patient populations.”

In the last part of the study, the researchers tested to see if the antiviral drug remdesivir inhibits virus infection in neurons and astrocytes.  They discovered that the drug was able to successfully reduce the viral level in astrocytes and prevent cell death.  For neurons, it was able to rescue them from steadily losing their function and even dying. 

The team says that the next steps to build on their findings is to continue studying the effects of the virus and better understand the role of ApoE4 in the brains of people who have COVID-19.  Many people that developed COVID-19 have recovered, but long-term neurological effects such as severe headaches are still being seen months after. 

“COVID-19 is a complex disease, and we are beginning to understand the risk factors involved in the manifestation of the severe form of the disease” says Dr. Arumugaswami.  “Our cell-based study provides possible explanation to why individuals with Alzheimer’s’ disease are at increased risk of developing COVID-19.”

The full results to this study were published in Cell Stem Cell.

Anticipating the Future of Regenerative Medicine: CIRM’s Alpha Stem Cell Clinics Network

All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. Today we take a deeper dive into CIRM’s Alpha Stem Cell Clinics Network.  The following is written by Dr. Geoff Lomax, Senior Officer of CIRM Therapeutics and Strategic Infrastructure.

The year 2014 has been described as the regenerative medicine renaissance: the European Union approved its first stem cell-based therapy and the FDA authorized ViaCyte’s CIRM funded clinical trial for diabetes. A path forward for stem cell treatments had emerged and there was a growing pipeline of products moving towards the clinic. At the time, many in the field came to recognize the need for clinical trial sites with the expertise to manage this growing pipeline. Anticipating this demand, CIRM’s provided funding for a network of medical centers capable of supporting all aspect of regenerative medicine clinical trials. In 2015, the Alpha Stem Cell Clinics Network was launched to for this purpose.

The Alpha Clinics Network is comprised of leading California medical centers with specific expertise in delivering patient-centered stem cell and gene therapy treatments. UC San Diego, City of Hope, UC Irvine and UC Los Angeles were included in the initial launch, and UC San Francisco and UC Davis entered the network in 2017. Between 2015 and 2020 these sites supported 105 regenerative medicine clinical trials. Twenty-three were CIRM-funded clinical trials and the remaining 82 were sponsored by commercial companies or the Alpha Clinic site. These trials are addressing unmet medical needs for almost every disease where regenerative medicine is showing promise including blindness, blood disorders (e.g. sickle cell disease) cancer, diabetes, HIV/AIDS, neurological diseases among others.

As of spring of 2020 the network had inked over $57 million in contracts with commercial sponsors. High demand for Alpha Clinics reflects the valuable human and technical resources they provide clinical trial sponsors. These resources include:

  • Skilled patient navigators to educate patients and their families about stem cell and gene therapy treatments and assist them through the clinical trial process.
  • Teams and facilities specialized in the manufacturing and/or processing of patients’ treatments. In some instances, multiple Alpha Clinic sites collaborate in manufacturing and delivery of a personalized treatment to the patient.
  • Nurses and clinicians with experience with regenerative medicine and research protocols to effectively deliver treatments and subsequently monitor the patients.

The multi- site collaborations are an example of how the network operates synergistically to accelerate the development of new treatments and clinical trials. For example, the UC San Francisco Alpha Clinic is collaborating with UC Berkeley and the UC Los Angeles Alpha Clinic to develop a CIRM-funded gene therapy for sickle cell disease. Each partner brings a unique expertise to the program that aims to correct a genetic mutilation in the patients’ blood stem cells to effectively cure the disease. Most recently, City of Hope has partnered with UC Irvine and UC San Diego as part of CIRM’s COVID-19 research program to study how certain immune system antibodies might be used as a treatment for respiratory disease in infected patients. In another COVID-19 study, UC Irvine and UC Davis are working with a commercial sponsor to evaluate a treatment for infected adults.

The examples above are a small sample of the variety of collaborations CIRM funding has enabled. As the Alpha Clinics track record grown, sponsors are increasingly coming to California to enable the success of their research programs. Sponsors with trials running across the country have noted a desire to expand their number of Alpha Clinic sties because they consistently perform at the highest level.

Back in 2014, it was hard to imagine over one hundred clinical trials would be served by the CIRM network in just five years. Fortunately, CIRM was able to draw on the knowledge of its internal team, external advisors and the ICOC to anticipate this need and provide California infrastructure to rise to the occasion.

Month of CIRM: Battling COVID-19

All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the people of California approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future.

Dr. John Zaia, City of Hope stem cell researcher

The news that effective vaccines have been developed to help fight COVID-19 was a truly bright spot at the end of a very dark year. But it will be months, in some countries years, before we have enough vaccines to protect everyone. That’s why it’s so important to keep pushing for more effective ways to help people who get infected with the virus.

One of those ways is in a clinical study that CIRM is funding with City of Hope’s Dr. John Zaia. Dr. Zaia and his team, in partnership with the Translational Genomics Research Institute (TGen) in Flagstaff, Arizona, are using something called convalescent plasma to try and help people who have contracted the virus. Here’s the website they have created for the study.

Plasma is a part of our blood that carries proteins, called antibodies, that help defend our bodies against viral infections. When a patient recovers from COVID-19, their blood plasma contains antibodies against the virus. The hope is that those antibodies can now be used as a potential treatment for COVID-19 to help people who are newly infected. 

To carry out the study they are using clinical trial sites around California, including some of the CIRM Alpha Stem Cell Network clinics.

For the study to succeed they’ll first need people who have recovered from the virus to donate blood. That’s particularly appropriate in January because this is National Volunteer Blood Donor Month.

The team has three elements to their approach:

  • A rapid-response screening program to screen potential COVID-19 convalescent plasma donors, particularly in underserved communities.
  • A laboratory center that can analyze the anti-SARS-CoV-2 antibodies properties in COVID-19 convalescent plasma.
  • An analysis of the clinical course of the disease in COVID-19 patients to identify whether antibody properties correlate with clinical benefit of COVID-19 convalescent plasma.

There’s reason to believe this approach might work. A study published this week in the New England Journal of Medicine, found that blood plasma from people who have recovered from COVID-19 can help older adults and prevent them from getting seriously ill with the virus if they get the plasma within a few days of becoming infected.

We are used to thinking of blood donations as being used to help people after surgery or who have been in an accident. In this study the donations serve another purpose, but one that is no less important. The World Health Organization describes blood as “the most precious gift that anyone can give to another person — the gift of life. A decision to donate your blood can save a life, or even several if your blood is separated into its components — red cells, platelets and plasma.”

That plasma could help in developing more effective treatments against the virus. Because until we have enough vaccines for everyone, we are still going to need as much help as we can get in fighting COVID-19. The recent surge in cases throughout the US and Europe are a reminder that this virus is far from under control. We have already lost far too many people. So, if you have recently recovered from the virus, or know someone who has, consider donating blood to this study. It could prove to be a lifesaver.

For more information about the study and how you can be part of it, click here.

CIRM-funded development of stem cell therapy for Canavan disease shows promising results

Yanhong Shi, Ph.D., City of Hope

Canavan disease is a fatal neurological disorder, the most prevalent form of which begins in infancy. It is caused by mutation of the ASPA gene, resulting in the deterioration of white matter (myelin) in the brain and preventing the proper transmission of nerve signals.  The mutated ASPA gene causes the buildup of an amino acid called NAA and is typically found in neurons in the brain.  As a result of the NAA buildup, Canavan disease causes symptoms such as impaired motor function, mental retardation, and early death. Currently, there is no cure or standard of treatment for this condition.

Fortunately, CIRM-funded research conducted at City of Hope by Yanhong Shi, Ph.D. is developing a stem cell-based treatment for Canavan disease. The research is part of CIRM’s Translational Stage Research Program, which promotes the activities necessary for advancement to clinical study of a potential therapy.

The results from the study are promising, with the therapy improving motor function, reducing degeneration of various brain regions, and expanding lifespan in a Canavan disease mouse model.

For this study, induced pluripotent stem cells (iPSCs), which can turn into virtually any type of cells, were created from skin cells of Canavan disease patients. The newly created iPSCs were then used to create neural progenitor cells (NPCs), which have the ability to turn into various types of neural cells in the central nervous system. A functional version of the ASPA gene was then introduced into the NPCs. These newly created NPCs were then transplanted inside the brains of Canavan disease mice.

The study also used iPSCs engineered to have a functional version of the ASPA gene. The genetically modified iPSCs were then used to create oligodendrocyte progenitor cells (OPCs), which have the ability to turn into myelin. The OPCs were also transplanted inside the brains of mice.

The rationale for evaluating both NPCs and OPCs was that NPCs typically stayed at the site of injection while OPCs tend to migrate, which might have been important in terms of the effectiveness of the therapy.  However, the results of the study show that both NPCs and OPCs were effective, with both being able to reduce levels of NAA, presumably because NAA can move to where the ASPA enzyme is although NPCs do not migrate.  This resulted in improved motor function, recovery of myelin, and reduction of brain degeneration, in both the NPC and OPC-transplanted Canavan disease mice.

“Thanks to funding from CIRM and the hard work of my team here at City of Hope and collaborators at Center for Biomedicine and Genetics, Department of Molecular Imaging and Therapy, and Diabetes and Metabolism Institute at City of Hope, as well as collaborators from the University of Texas Medical Branch at Galveston, University of Rochester Medical Center, and Aarhus University, we were able to carry out this study which has demonstrated promising results,” said Dr. Shi.  “I hope that these findings can one day bring about an effective therapy for Canavan disease patients, who currently have no treatment options.”

Dr. Shi and her team will build on this research by starting IND-enabling studies using their NPC therapy soon.  This is the final step in securing approval from the Food and Drug Administration (FDA) in order to test the therapy in patients.  

The full study was published in Advanced Science.

Want to help us solve a mystery?

Patient that has recovered from Covid-19 donating blood plasma. Photo courtesy Science Photo

Convalescent plasma has been in the news a lot lately as a potential treatment for people infected with the coronavirus. In August the US Food and Drug Administration (FDA) granted emergency use authorization (EUA) to use these products based on preliminary data that suggested it might help people battling COVID. But there are still a lot of unanswered questions about this approach.

And that’s where you come in.

Plasma is a component of blood that carries proteins called antibodies that are usually involved in defending our bodies against viral infections.  We also know that blood plasma from patients that have recovered from COVID-19, referred to as convalescent plasma, contain antibodies against the virus that can be used as a potential treatment for COVID-19. 

That’s the theory, but the reality is that there are still a lot we don’t know, basic questions such as does it really work, how does it work, does it work for everyone or just some patients? A clinical  grant includes testing the plasma in COVID-19 Positive patients that CIRM is funding with City of Hope, UC Irvine and Translational Genomics Research Institute (TGen) hopes to answer those questions. 

The first step is getting the plasma from people who have recovered from COVID and then testing it to make sure it’s safe and to identify what blood type it is, so you can match that blood type with the person receiving it.

But plasma doesn’t contain just one kind of antibody, there are many antibodies and each one works in a slightly different way. For example, two antibodies, IGM and IGG, target in on the spike protein on the coronavirus. The goal is to block that spike and prevent the virus from spreading throughout the body. IGM has up to 10 ‘arms’ and so has the potential to bind multiple copies of the spike, whereas IGG has only 2 arms, but lasts longer. Both IGM and IGG also come in many different flavors, allowing them to bind to many different parts of the spike, some being more protective than others.

That’s one of the things that this trial is trying to find out. And you can help them do that. The trial needs volunteers, volunteers to donate the plasma and volunteers to try the therapy.

The team is evaluating changes that occur before and after plasma treatment.  Many recipients have no immediate response, a few get dramatically better, and some continue to have symptoms long after discharge from the hospital.  These so-called “long-haulers” can have debilitating problems, months after becoming infected. The study hopes to evaluate these variable responses to plasma treatment.

But more people are needed if we are to truly understand what works best. We need people who are newly infected, those being treated with plasma, and those that have recovered from the virus.

We are particularly interested in recruiting people from the Black and Latinx communities, groups that are often underserved when it comes to access to medical care.

The team has created a website to make it easy to find out more about the clinical trial, and to see if you are a good candidate to be part of it, either as a donor or recipient.

Lives are at stake and time is short so join us, help us find answers to the most pressing medical issue of our times. It’s a chance to do something that might benefit your family, your friends and your community.

Cures, clinical trials and unmet medical needs

When you have a great story to tell there’s no shame in repeating it as often as you can. After all, not everyone gets to hear first time around. Or second or third time. So that’s why we wanted to give you another opportunity to tune into some of the great presentations and discussions at our recent CIRM Alpha Stem Cell Clinic Network Symposium.

It was a day of fascinating science, heart-warming, and heart-breaking, stories. A day to celebrate the progress being made and to discuss the challenges that still lie ahead.

There is a wide selection of topics from “Driving Towards a Cure” – which looks at some pioneering work being done in research targeting type 1 diabetes and HIV/AIDS – to Cancer Clinical Trials, that looks at therapies for multiple myeloma, brain cancer and leukemia.

The COVID-19 pandemic also proved the background for two detailed discussions on our funding for projects targeting the coronavirus, and for how the lessons learned from the pandemic can help us be more responsive to the needs of underserved communities.

Here’s the agenda for the day and with each topic there’s a link to the video of the presentation and conversation.

Thursday October 8, 2020

View Recording: CIRM Fellows Trainees

9:00am Welcome Mehrdad Abedi, MD, UC Davis Health, ASCC Program Director  

Catriona Jamieson, MD,  View Recording: ASCC Network Value Proposition

9:10am Session I:  Cures for Rare Diseases Innovation in Action 

Moderator: Mark Walters, MD, UCSF, ASCC Program Director 

Don Kohn, MD, UCLA – View Recording: Severe combined immunodeficiency (SCID) 

Mark Walters, MD, UCSF, ASCC Program Director – View Recording: Thalassemia 

Pawash Priyank, View Recording: Patient Experience – SCID

Olivia and Stacy Stahl, View Recording: Patient Experience – Thalassemia

10 minute panel discussion/Q&A 

BREAK

9:55am Session II: Addressing Unmet Medical Needs: Driving Towards a Cure 

Moderator: John Zaia, MD, City of Hope, ASCC Program Direction 

Mehrdad Abedi, MD, UC Davis Health, ASCC Program Director – View Recording: HIV

Manasi Jaiman, MD, MPH, ViaCyte, Vice President, Clinical Development – View Recording: Diabetes

Jeff Taylor, Patient Experience – HIV

10 minute panel discussion/Q&A 

BREAK

10:40am Session III: Cancer Clinical Trials: Networking for Impact 

Moderator: Catriona Jamieson, MD, UC San Diego, ASCC Program Director 

Daniela Bota, MD, PhD, UC Irvine, ASCC Program Director – View Recording:  Glioblastoma 

Michael Choi, MD, UC San Diego – View Recording: Cirmtuzimab

Matthew Spear, MD, Poseida Therapeutics, Chief Medical Officer – View Recording: Multiple Myeloma  

John Lapham, Patient Experience –  View Recording: Chronic lymphocytic leukemia (CLL) 

10 minute panel discussion/Q&A 

BREAK

11:30am Session IV: Responding to COVID-19 and Engaging Communities

Two live “roundtable conversation” sessions, 1 hour each.

Roundtable 1: Moderator Maria Millan, MD, CIRM 

CIRM’s / ASCC Network’s response to COVID-19 Convalescent Plasma, Cell Therapy and Novel Vaccine Approaches

Panelists

Michael Matthay, MD, UC San Francisco: ARDS Program

Rachael Callcut, MD, MSPH, FACS, UC Davis: ARDS Program 

John Zaia, MD, City of Hope: Convalescent Plasma Program 

Daniela Bota, MD, PhD, UC Irvine: Natural Killer Cells as a Treatment Strategy 

Key questions for panelists: 

  • Describe your trial or clinical program?
  • What steps did you take to provide access to disproportionately impacted communities?
  • How is it part of the overall scientific response to COVID-19? 
  • How has the ASCC Network infrastructure accelerated this response? 

Brief Break

Roundtable 2: Moderator Ysabel Duron, The Latino Cancer Institute and Latinas Contra Cancer

View Recording: Roundtable 2

Community Engagement and Lessons Learned from the COVID Programs.  

Panelists

Marsha Treadwell, PhD, UC San Francisco: Community Engagement  

Sheila Young, MD, Charles R. Drew University of Medicine and Science: Convalescent Plasma Program in the community

David Lo, MD, PhD,  UC Riverside: Bringing a public health perspective to clinical interventions

Key questions for panelists: 

  • What were important lessons learned from the COVID programs? 
  • How can CIRM and the ASCC Network achieve equipoise among communities and engender trust in clinical research? 
  • How can CIRM and the ASCC Network address structural barriers (e.g. job constrains, geographic access) that limit opportunities to participate in clinical trials?

Remembering a stem cell pioneer in the fight against HIV/AIDS

Timothy Ray Brown. Photo courtesy Seattle Times

Timothy Ray Brown, a man who was the first person to be cured of HIV, giving hope to millions of people around the world, died at his home in Palm Springs this week. He was just 54 years old.

For years Brown was known simply as “the Berlin patient” because that was where he was living when he made medical history. He was diagnosed with HIV in 1995 and began taking medications to keep the virus under control. He was later also diagnosed with leukemia. He underwent several rounds of treatment for the leukemia, but it kept recurring.

By 2007 Brown’s physician decided the best way to treat the leukemia was with a blood stem cell transplant. But the doctor also wanted to see if using the stem cells from a donor who had a natural immunity to the AIDS virus could help treat Brown’s HIV. While such donors are very rare, the doctor succeeded in finding one whose bone marrow carried the CCR5 gene, a mutation that is believed to provide resistance to HIV. The transplant was a success, putting Brown’s leukemia into remission and eliminating detectable traces of HIV. For the first time in years he was able to stop taking the medications that had helped keep the virus under control.

The procedure quickly garnered world-wide attention. But not everyone was convinced it was real. Some questioned if Brown’s HIV had really been eradicated and speculated that the virus was merely suppressed. But with each passing year, and no signs of the virus recurring, more and more people came to believe it was a cure.

Initially Brown remained in the background, preferring not to be identified. But three years after his transplant he decided he had to come forward and put a face on “the Berlin patient”. In an interview with the website ContagionLive he explained why:

“At some point, I decided I didn’t want to be the only person in the world cured of H.I.V.,” I wanted there to be more. And the way to do that was to show the world who I am and be an advocate for H.I.V.”

He proved to be a powerful advocate, talking at international conferences and serving as living-proof that stem cells could help lead to a cure for HIV.

But while he managed to beat HIV, he could not beat leukemia. He suffered relapses that required another transplant and a difficult recovery. When it returned again this time, there was little physicians could do.

But Timothy Ray Brown did get to see his hope of not being the only patient cured seemingly come true. In September of last year researchers announced they had successfully treated a second person, known as “the London patient” using the same technique that cured Brown.

While it wasn’t the role he would have chosen Brown was a pioneer. His experience showed that a deadly virus could be cured. His courage in not just overcoming the virus but in overcoming his own reluctance to take center stage and becoming a symbol of hope for millions remain and will never die.  

Since Brown’s transplant many other scientists have attempted to replicate the procedure that cured Brown, in the hopes of making it available to many more people.

CIRM has funded three clinical trials targeting HIV, two of which are still active. Dr. Mehrdad Abedi at UC Davis and Dr. John Zaia at City of Hope are both using the patient’s own blood forming stem cells to try and defeat the virus.

If they succeed, some of the credit should go to Timothy Ray Brown, the man who led the way.

Explaining COVID can be a pitch

When people ask me what I do at CIRM I sometimes half-jokingly tell them that I’m the official translator: I take complex science and turn it into everyday English. That’s important. The taxpayers of California have a right to know how their money is being spent and how it might benefit them. But that message can be even more effective when it comes from the scientists themselves.

Recently we asked some of the scientists we are funding to do research into COVID-19 to record what’s called an “elevator pitch”. This is where they prepare an explanation of their work that is in ordinary English and is quite short, short enough to say it to someone as you ride in an elevator. Hence the name.

It sounds easy enough. But it’s not. When you are used to talking in the language of science day in and day out, suddenly switching codes to talk about your work in plain English can take some practice. Also, you have spent years, often decades, on this work and to have to explain it in around one minute is no easy thing.

But our researchers rose to the challenge. Here’s some examples of just how well they did.

It’s all about the patients

Ronnie, born with a fatal immune disorder now leading a normal life thanks to a CIRM-funded stem cell/gene therapy: Photo courtesy of his mum Upasana

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 end user, 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 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.

Frances Saldana, a patient advocate for research into Huntington’s disease

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 it 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.

Here’s one of the interviews that was featured at the event. I do apologize in advance for the interviewer, he’s rubbish at his job.

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.