Annual Report

How the Tooth Fairy is helping unlock the secrets of autism

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Our 2021-22 Annual Report is now online. It’s filled with information about the work we have done over the last year (we are on a fiscal calendar year from July 1 – June 30), the people who have helped us do that work, and some of the people who have benefited from that work. One of those is Dr. Alysson Muotri, a professor in the Departments of Pediatrics and Cellular & Molecular Medicine at the University of California, San Diego.

Dr. Alysson Muotri, in his lab at UCSD

For Dr. Alysson Muotri, trying to unlock the secrets of the brain isn’t just a matter of scientific curiosity, it’s personal. He has a son with autism and Dr. Muotri is looking for ways to help him, and millions of others like him around the world.

He created the Tooth Fairy project where parents donated more than 3,000 baby teeth from  children with autism and children who are developing normally. Dr. Muotri then turned cells from those teeth into neurons, the kind of brain cell affected by autism. He is using those cells to try and identify how the brain of a child with autism differs from a child who is developing normally.

“We’ve been using cells from this population to see what are the alterations (in the gene) and if we can revert them back to a normal state. If you know the gene that is affected, and autism has a strong genetic component, by genome sequencing you can actually find what are the genes that are affected and in some cases there are good candidates for gene therapy. So, you just put the gene back. And we can see that in the lab where we are correcting the gene that is mutated, the networks start to function in a way that is more neurotypical or normal. We see that as highly promising, there’s a huge potential here to help those individuals.”

He is also creating brain organoids, three-dimensional structures created from stem cells that mimic some of the actions and activities of the brain. Because these are made from human cells, not mice or other animals, they may be better at indicating if new therapies have any potential risks for people.

“We can test drugs in the brain organoids of the person and see if it works, see if there’s any toxicity before you actually give the drug to a person, and it will save us time and money and will increase our knowledge about the human brain.”

He says he still gets excited seeing how these cells work. “It’s amazing, it’s a miracle. Every time I see it, it’s like seeing dolphins in the sea because it’s so beautiful.”

Dr. Muotri is also a big proponent of diversity, equity and inclusion in scientific research. He says in the past it was very much a top-down model with scientists deciding what was important. He says we need to change that and give patients and communities a bigger role in shaping the direction of research.

“I think this is something we scientists have to learn, how to incorporate patients in our research. These communities are the ones we are studying, and we need to know what they want and not assume that what we want is what they want. They should be consulted on our grants, and they should participate in the design of our experiments. That is the future.”

Celebrating a life that almost didn’t happen

/ Kevin McCormack / 1 Comment
Evie Vaccaro

You can’t look at this photo and not smile. This is Evie Vaccaro, and it’s clear she is just bursting with energy and vitality. Sometimes it feels like I have known Evie all her life. In a way I have. And I feel so fortunate to have done so, and that’s why this photo is so powerful, because it’s a life that almost ended before it had a chance to start.

Evie was born with a rare condition called Severe Combined Immunodeficiency (SCID). Children with this condition lack a functioning immune system so even a simple cold or diaper rash can prove fatal. Imagine how perilous their lives are in a time of COVID-19. These children used to be called “bubble babies” because they were often kept inside sterile plastic bubbles to keep them alive. Many died before their second birthday.

Today there is no need for plastic bubbles. Today, we have a cure. That’s a word we use very cautiously, but in Evie’s case, and the case of more than 40 other children, we use it with pride.

Dr. Don Kohn and a child born with SCID

Dr. Don Kohn at UCLA has developed a method of taking the child’s own blood stem cells and, in the lab, inserting a corrected copy of the gene that caused SCID, and then returning those cells to the child. Because they are stem cells they multiply and renew and replicate themselves, creating a new blood supply, one free of the SCID mutation. The immune system is restored. The children are cured.

This is a story we have told several times before, but we mention it again because, well, it never gets old, and because Evie is on the front and back cover of our upcoming Annual Report. The report is actually a look back on the last 18 months in CIRM’s life, reporting on the progress we have made in advancing stem cell research, in saving and changing lives, and in producing economic benefits for California (billions of dollars in sales revenue and taxes and thousands of jobs).  

Evie’s story, Evie’s photo, is a reminder of what is possible thanks to the voters of California who created CIRM back in 2004. Hers is just one of the stories in the report. I think,  you’ll enjoy reading all of them.

Of course, I might be just a little bit biased.

Performance, Passion and Progress: and that’s just page one of our 2018 Annual Report

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It’s hard to sum up the activities and achievements of a year in a single document, let alone one that’s just 24 pages. But that’s what we have done in putting together our 2018 Annual Report.

It’s a look back at the year just gone, the highlights, the low lights (spoiler alert – there weren’t any) and the impact we had on the field of stem cell research. But it’s far more than that. It’s also a look ahead. A look at the challenges we face, and profiles of the people who are going to help us overcome those challenges and maintain our progress.

And people are truly at the heart of this report, from UC San Francisco’s Dr. Tippi MacKenzie who is on the front cover for her work in developing an in-utero treatment for the almost always fatal disorder alpha thalassemia major (and the photo of the baby and mom whose lives were changed by that therapy) to Rich Lajara on the back cover, the first person ever treated in a CIRM-funded clinical trial.

Inside are an array of simple images designed to reflect how we as a state agency have performed this year. The numbers themselves tell a powerful story:

  • 50 clinical trials funded to date, 7 this year alone
  • $2.6 billion in CIRM grants has been leveraged to bring in an additional $3.2 billion in matching funds and investments from other sources.
  • 1,180 patients have been involved in CIRM clinical trials

We know people don’t have a lot of time to read Annual Reports so we have made this as visually engaging and informative as possible. We want you to get a real sense of who we are, what we have done and who has helped us do that without you having to wade through a document the size of War and Peace (great book by the way – the Russians beat Napoleon).

We think we have a great story to tell. This Annual Report is one chapter in that story. We hope you like it.

 

The power of one voice: David Higgins’ role in advancing stem cell research

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David Higgins: Photo courtesy Nancy Ramos @ Silver Eye Photography

As we start a new year, we are fine tuning our soon-to-be-published 2018 Annual Report, summarizing our work over the past 12 months. The report is far more than just a collection of statistics about how many clinical trials we are funding (50 – not too shabby eh!) or that our support has generated an additional $3.2 billion in leveraged funding. It’s also a look at the people who have made this year so memorable – from patients and researchers to patient advocates. We start with our Board member David Higgins, Ph.D.  David is the patient advocate on our Board for Parkinson’s disease. He has a family history of Parkinson’s and has also been diagnosed with the disease himself.

How he sees his role

As a patient advocate my role is not to support any Parkinson’s program that comes in the door and get it funded. We have to judge the science at the same level for every disease and if you bring me a good Parkinson’s project, I will fight tooth and nail to support it. But if you bring me a bad one, I will not support it. I see my role as more of a consultant for the staff and Board, to help advise but not to impose my views on them.

I think what CIRM has done is to create a new way of funding the best science in the world. The involvement of the community in making these decisions is critical in making sure there is an abundance of oversight, that there is not a political decision made about funding. It’s all about the science. This is the most science-based organization that you could imagine.

The Board plays a big role in all this. We don’t do research or come up with the ideas, but we nurture the research and support the scientists, giving them the elements they need to succeed.

And, of course the taxpayers play a huge role in this, creating us in the first place and approving all the money to help support and even drive this research. Because of that we should be as conservative as possible in using this money. Being trustees of this funding is a privilege and we have to be mindful of how to best use it.

On the science

I love, love, love having access to the latest, most interesting, cutting edge research in the world, talking to scientists about what they are doing, how we can support them and help them to do it better, how it will change the world. You don’t have access to anything else like this anywhere else.

It’s like ice cream, you just enjoy every morsel of it and there’s no way you can find that level of satisfaction anywhere else. I really feel, as do other Board members, that we are helping people, that we are changing people’s lives.

I also love the learning curve. The amount I have learned about the field that I didn’t know before is amazing. Every meeting is a chance to learn something new and meeting the scientists who have spent years working on a project is so fascinating and rewarding.

 Unexpected pleasure

The other joy, and I hadn’t anticipated this, is the personal interaction I have with other Board members and staff members. They have become friends, people I really like and admire because of what they do and how committed they are.

When I talk about CIRM I tell people if you live in California you should be proud of how your money is being spent and how it’s making a difference in people’s lives. When I give a talk or presentation, I always end with a slide of the California flag and tell people you should be proud to be here.

 

 

A year in review – CIRM’s 2017 Annual Report focuses on a year of accelerating stem cell treatments to patients

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At CIRM we have our focus very clearly on the future, on accelerating stem cell therapies to patients with unmet medical needs. But every once in a while, it’s a good idea to look back at what you have already done. Knowing where you came from can help you get to where you are heading.

So, it’s with a sense of accomplishment that we are unveiling our 2017 Annual Report. It’s a look back at another banner year for the stem cell agency, the research we funded, the partnerships we created and, most importantly, the lives we touched.

It features profiles of several people who received stem cell therapies in CIRM-funded clinical trials and the impact those therapies are having on them. But it also looks at some of the other individuals who are such a vital part of the work we do: patient advocates, researchers and a member of our Grants Working Group which reviews applications for funding. Each one, in their own way, contributes to advancing the field.

The report also highlights some of the less obvious ways that our funding is benefitting California. For example, the additional $1.9 billion dollars our funding has helped generate through co-funding and partnerships, or the number of projects we are funding that have been awarded Regenerative Medicine Advanced Therapy Designation from the Food and Drug Administration (FDA), making them eligible for accelerated review if their results continue to be promising.

It’s a look back at a successful year.

But we are not resting on our laurels. We are already hard at work, determined to make 2018 even better.

 

 

How Tom Howing turned to stem cells to battle back against a deadly cancer

/ Kevin McCormack / 2 Comments

As we enter the new year, CIRM’s 2017 Annual Report will be posted in less than two weeks!  Here’s one of the people we are profiling in the report, a patient who took part in a CIRM-funded clinical trial.

Tom Howing

In March of 2015, Tom Howing was diagnosed with stage 4 cancer. Over the next 18 months, he underwent two rounds of surgery and chemotherapy. Each time the treatments held the cancer at bay for a while. But each time the cancer returned. Tom was running out of options and hope when he heard about a CIRM-funded clinical trial using a new approach.

The clinical trial uses a therapy that blocks a protein called CD47 that is found on the surface of cancer cells, including cancer stem cells which can evade traditional therapies. CD47 acts as a ‘don’t eat me’ signal that tells immune cells not to kill off the cancer cells. When this ‘don’t eat me’ signal is blocked by the antibody, the patient’s immune system is able to identify, target and kill the cancer stem cells.

“When I was diagnosed with cancer I knew I had battle ahead of me. After the cancer came back again they recommended I try this CD47 clinical trial. I said absolutely, let’s give it a spin.

“I guess one is always a bit concerned whenever you put the adjective “experimental” in front of anything. But I’ve always been a very optimistic and positive person and have great trust and faith in my caregivers.

“Whenever you are dealing with a Phase 1 clinical trial (the earliest stage where the goal is first to make sure it is safe), there are lots of unknowns.  Scans and blood tests came back showing that the cancer appears to be held in check. My energy level is fantastic. The treatment that I had is so much less aggressive than chemo, my quality of life is just outstanding.”

Tom says he feels fortunate to be part of the clinical trial because it is helping advance research, and could ultimately help many others like him.

“The most important thing I would say is, I want people to know there is always hope and to stay positive.”

He says he feels grateful to the people of California who created CIRM and the funding behind this project: “I say a very heartfelt thank you, that this was a good investment and a good use of public funds.”

He also wants the researchers, who spent many years developing this approach, to know that they are making a difference.

“To all those people who are putting in all the hours at the bench and microscope, it’s important for them to know that they are making a huge impact on the lives of real people and they should celebrate it and revel in it and take great pride in it.”

Accelerating stem cell treatments to patients in 2017

/ Kevin McCormack / 1 Comment

As we enter the new year, CIRM’s 2017 Annual Report will be posted in a few short weeks!  Here’s a sneak peek at CIRM’s progress in clinical trials.

2017 CIRM Annual Report

At the start of 2017, we set a goal of finding and funding 12 new, high quality clinical trials. We easily beat that goal, funding 16, in a wide variety of conditions from ALS (also known as Lou Gehrig’s disease) to cancer and diabetes. That means we have now funded a total of 43 different projects in clinical trials and enrolled more than 700 people in those trials.

Here’s a look at the different kinds of stem cells and diseases are involved in those clinical trials:

Funding those 16 new clinical trials means we have now funded 26 new trials in the last two years, putting us ahead of schedule to meeting our goal of 50 new clinical trials by 2020.

When we fund clinical programs, we truly partner with these programs and give them support – financially, operationally and strategically.

CIRM assists investigators in the application process so they can best articulate their research proposal in a way that can be optimally evaluated by our independent peer review group for funding. By putting applications through a rigorous review process, we select programs with the highest probability of success.  You will hear from one of our GWG members, the external panel that reviews our grants for funding, in the Annual Report.

CIRM provides funding at a critical stage when programs are not yet able to get sufficient funding because they are felt to be “too early” or “too risky” for traditional investors. By funding these investigators to conduct important early work, CIRM “de-risks” the projects, and we have already seen how this has allowed “high risk but high reward” programs to attract investors and commercialization partners. We will feature examples of these follow-on investments in the Annual Report.

In addition to funding clinical trials, CIRM brings in critical expertise and resources for these programs. Clinical Advisory Panels (CAPs), composed of CIRM science officers, external experts and patient representatives, meet on a quarterly basis for each program to help them overcome obstacles and meet project milestones. CIRM has created the Stem Cell Center – a stem cell-specific research organization that helps investigators navigate the best regulatory pathways, provides access manufacturing resources, operational clinical trial support and strategic resources for delivering successful products to patients.

In short, we do everything we can to try and ensure those clinical trials have the best possible chance to be successful.

With a growing number of clinical trials to track, and more on the way, we needed a new tool to make it easier to see, at a glance, the trials we are funding, and all the key details of each program.

So, we created the Clinical Trials Dashboard to let you sort each trial by disease type, researcher, company or institution, and phase, as well as how many patients are to be enrolled. It also includes links to the www.clinicaltrials.gov website – a list of clinical trials registered with the National Institutes of Health – with details about patient eligibility and how to apply to be part of the trial.

The Dashboard is our way of making it as easy as possible for you to find the information you need, when you need it.

On Thursday, we’ll introduce you to one of the patients involved in a CIRM-funded clinical trial for cancer.

Stem Cell Profiles in Courage: Brenden Whittaker

/ Kevin McCormack / 3 Comments

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Brenden Whittaker: Photo Colin McGuire

It’s not often you meet someone who says one of their favorite things in the world is mowing the lawn. But then, there aren’t many people in the world like Brenden Whittaker. In fact, as of this writing, he may be unique.

Brenden was born with severe chronic granulomatous disease (x-CGD), a rare genetic disorder that left him with an impaired immune system that was vulnerable to repeated bacterial and fungal infections. Over 22 years Brenden was in and out of the hospital hundreds of times, he almost died a couple of times, and lost parts of his lungs and liver.

Then he became the first person to take part in a clinical trial to treat x-CGD. UCLA researcher Don Kohn had developed a technique that removed Brenden’s blood stem cells, genetically re-engineered them to correct the mutation that caused the disease, and then returned those stem cells to Brenden. Over time they created a new blood system, and restored Brenden’s immune system.

He was cured.

We profiled Brenden for our 2016 Annual Report. Here’s an extended version of the interview we did with him, talking about his life before and after he was cured.

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Brenden with a CIRM Game Ball – signed by everyone at CIRM

Brenden’s story:

I still think about it, my disease, every few days or so and it’s weird because in the past I was sick so often; before this year, I was sick consistently for about 5 years and going to doctor’s appointments 2 or 3 times a week and being in the hospital. So, it’s weird having a cough and not having to be rushed to the ER, not having to call someone every time the smallest thing pops up, and not having to worry about what it means.

It’s been good but it’s been weird to not have to do that.  It’s a nice problem to have.

What are you doing now that you didn’t do before?

Cutting the grass is something I couldn’t do before, that I’ve taken up now. Most people look at me as if I’m crazy when I say it, but I love cutting grass, and I wasn’t able to do it for 22 years of my life.

People will complain about having to pick up after their dog goes to the bathroom and now I can follow my dog outside and can pick up after her. It really is just the little things that people don’t think of. I find enjoyment in the small things, things I couldn’t do before but now I can and not have to worry about them.

The future

I was in the boy scouts growing up so I love camping, building fires, just being outdoors. I hiked on the Appalachian Trail. Now I’ll be able to do more of that.

I have a part time job at a golf course and I’m actually getting ready to go back to school full time in January. I want to get into pre-med, go to medical school and become a doctor. All the experience I’ve had has just made me more interested in being a doctor, I just want to be in a position where I can help people going through similar things, and going through all this just made me more interested in it.

Before the last few months I couldn’t schedule my work more than a week in advance because I didn’t know if I was going to be in the hospital or what was going on. Now my boss jokes that I’m giving him plans for the next month or two. It’s amazing how far ahead you can plan when you aren’t worried about being sick or having to go to the hospital.

I’d love to do some traveling. Right now most of my traveling consists of going to and from Boston (for medical check-ups), but I would love to go to Europe, go through France and Italy. That would be a real cool trip. I don’t need to see everything in the world but just going to other countries, seeing cities like London, Paris and Rome, seeing how people live in other cultures, that would be great.

Advice for others

I do think about the fact that when I was born one in a million kids were diagnosed with this disease and there weren’t any treatments. Many people only lived a few years. But to be diagnosed now you can have a normal life. That’s something all on its own. It’s almost impossible for me to fathom it’s happening, after all the years and doctor’s appointments and illnesses.

So, for people going through anything like this, I’d say just don’t give up. There are new advances being made every day and you have to keep fighting and keep getting through it, and some day it will all work out.

Related Links:

Stem Cells Profile in Courage: Pat Furlong, Patient Advocate

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Pat Furlong: Photo by Colin McGuire – http://www.colinmcguire.com

One of the true joys for me in helping put together this year’s Annual Report was getting to know the patients and patient advocates that we profiled in the report. These are some extraordinary individuals and the short profiles we posted only touch the surface of just how extraordinary.

So, over the next few weeks we are going to feature four of these people at greater length, allowing them, in their own words, to talk about what makes them tic, and how they keep going in the face of what is often heartbreak and tragedy.

We begin with Pat Furlong, a Patient Advocate and the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (DMD).

DMD is the most common fatal, genetic childhood disorder, which affects approximately 1 out of every 3,500 boys each year worldwide. It’s a progressive muscle disorder that leads to loss of muscle function, meaning you lose your ability to walk, to use your arms, and ultimately to breathe. And because the heart is a muscle, that is often seriously affected. There is no cure, and treatment options are limited. At the time her sons were diagnosed life expectancy was in the teens.

Pat’s story:

“When my sons, Chris and Pat were diagnosed with DMD, at the ages of 4 and 6, there was nothing available for them. Doctors cared about them but they didn’t have the tools they needed, or the National Institutes of Health the money it needed to do research.

Doctors were faced with diagnosing a disease and saying “there’s nothing we can do”. And then parents like me, coming to them hearing there was nothing they could do, no hope, no help. When your son is diagnosed with something like this you are told go home and love them.

When I asked questions, I was often ignored or dismissed by some doctors.

When my sons were diagnosed with DMD I would drop them off at school and go walking and that would help me deal with the anger.

For me staying in this is to be able to say to Chris and Pat in the universe, when you were here I tried my very best and when you were gone I continued to try my best so that others would have advantages that you didn’t receive.

I haven’t stood back and said I can’t go on.

The family is all scarred, we all suffered this loss. It’s much more apparent when we are together, there are empty chairs, emptiness. If we go to a family gathering we wish Chris and Pat were here, could be married. Now there’s my husband and our two daughters. We have a granddaughter, who is wonderful, but still we are incomplete and we will live with that forever.

I am trained as a nurse and I find DMD equal parts fascinating disease, heartbreaking and painful. I try to emphasize the fascinating so I can keep going. There are frustrations; lack of money, the slow process of regulatory approval, but I have an incredible team of very smart people and we are passionate about change so that helps keep us going.

Your only interest can’t be DMD, it can’t be. For me it’s certainly a priority, but it’s not my only interest. I love to go to an art museum and see how creative people work. I love Cirque du Soleil because they do things with their muscles I can’t imagine. Going outside and seeing these things makes the world better.

I am interested in the expression of art, to see how people dress, to see how people are creative, I love creativity, I think the human spirit is pretty amazing and the creativity around it. I think we are all pretty amazing but sometimes we don’t say it enough.

I recently saw a woman on the subway with a pair of tennis shoes that said “you are beautiful” and people around her were looking at her shoes and smiling, just because of those shoes. We forget to interact, and that was such a simple way of doing that.

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I relax by doing yoga, 90-minute hot yoga, as often as I can. I’ve also done a number of half marathons, but I’m more a walker than a runner. I find getting outside or hot yoga makes me concentrate on what I’m doing so that I can’t think of anything else. I can put it down and think about nothing and whisper prayers to my sons and say am I doing the right thing, is there something I should be doing differently? It’s my time to think about them and meditate about what they think would be important.

You need to give your mind time to cope, so it’s putting your phone down and your computer away. It’s getting rid of those interruptions. To put the phone, the computer down and get in a hot room and do yoga, or run around outside, to look at a tree and think about the changing season, the universe, the sun. It’s an incredible break for the brain to be able to rest.

I think the disease has made us kinder people and more thoughtful. When Chris died, we found a notebook he kept. In it was written “the meaning of life is a life of meaning”. I think that’s where we have all landed, what we all strive for, a life of meaning.

 

 

 

Cured by Stem Cells

/ Kevin McCormack / 1 Comment

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To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual Report for 2016, now online, is our way of checking that we are still on the right path.

I think, without wishing to boast, that it’s safe to say not only are we on target, but we might even be a little bit ahead of schedule.

The Annual Report is chock full of facts and figures but at the heart of it are the stories of the people who are the focus of all that we do, the patients. We profile six patients and one patient advocate, each of whom has an extraordinary story to tell, and each of whom exemplifies the importance of the work we support.

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Brenden Whittaker: Cured

Two stand out for one simple reason, they were both cured of life-threatening conditions. Now, cured is not a word we use lightly. The stem cell field has been rife with hyperbole over the years so we are always very cautious in the way we talk about the impact of treatments. But in these two cases there is no need to hold back: Evangelina Padilla Vaccaro and Brenden Whittaker have been cured.

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Evangelina: Cured

 

In the coming weeks we’ll feature our conversations with all those profiled in the Annual Report, giving you a better idea of the impact the stem cell treatments have had on their lives and the lives of their family. But today we just wanted to give a broad overview of the Annual Report.

The Strategic Plan was very specific in the goals it laid out for us. As an agency we had six big goals, but each Team within the agency, and each individual within those teams had their own goals. They were our own mini-maps if you like, to help us keep track of where we were individually, knowing that every time an individual met a goal they helped the Team get closer to meeting its goals.

As you read through the report you’ll see we did a pretty good job of meeting our targets. In fact, we missed only one and we’re hoping to make up for that early in 2017.

But good as 2016 was, we know that to truly fulfill our mission of accelerating treatments to patients with unmet medical needs we are going to have do equally well, if not even better, in 2017.

That work starts today.