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

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

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

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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

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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.

 

Stem cell heroes: patients who had life-saving, life-changing treatments inspire CIRM Board

 

It’s not an easy thing to bring an entire Board of Directors to tears, but four extraordinary people and their families managed to do just that at the last CIRM Board meeting of 2016.

The four are patients who have undergone life-saving or life-changing stem cell therapies that were funded by our agency. The patients and their families shared their stories with the Board as part of CIRM President & CEO Randy Mill’s preview of our Annual Report, a look back at our achievements over the last year.

The four included:

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Jake Javier, whose life changed in a heartbeat the day before he graduated high school, when he dove into a swimming pool and suffered a spinal cord injury that left him paralyzed from the chest down. A stem cell transplant is giving him hope he may regain the use of his arms and hands.

 

 

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Karl Trede who had just recovered from one life-threatening disease when he was diagnosed with lung cancer, and became the first person ever treated with a new anti-tumor therapy that helped hold the disease at bay.

 

brenden_stories_of_hopeBrenden Whittaker, born with a rare immune disorder that left his body unable to fight off bacterial or fungal infections. Repeated infections cost Brenden part of his lung and liver and almost killed him. A stem cell treatment that gave him a healthy immune system cured him.

 

 

evangelinaEvangelina Padilla Vaccaro was born with severe combined immunodeficiency (SCID), also known as “bubbly baby” disease, which left her unable to fight off infections. Her future looked grim until she got a stem cell transplant that gave her a new blood system and a healthy immune system. Today, she is cured.

 

 

Normally CIRM Board meetings are filled with important, albeit often dry, matters such as approving new intellectual property regulations or a new research concept plan. But it’s one thing to vote to approve a clinical trial, and a very different thing to see the people whose lives you have helped change by funding that trial.

You cannot help but be deeply moved when you hear a mother share her biggest fear that her daughter would never live long enough to go to kindergarten and is now delighted to see her lead a normal life; or hear a young man who wondered if he would make it to his 24th birthday now planning to go to college to be a doctor

When you know you played a role in making these dreams happen, it’s impossible not to be inspired, and doubly determined to do everything possible to ensure many others like them have a similar chance at life.

You can read more about these four patients in our new Stories of Hope: The CIRM Stem Cell Four feature on the CIRM website. Additionally, here is a video of those four extraordinary people and their families telling their stories:

We will have more extraordinary stories to share with you when we publish our Annual Report on January 1st. 2016 was a big year for CIRM. We are determined to make 2017 even bigger.