It’s always nice to be told you are doing a good job. It’s even nicer when it’s unexpected. That’s certainly the case when we, the Communications Team at the California Institute for Regenerative Medicine, found out we’d been named as a finalist for the Patient Advocacy Award (non-profit category) as part of the Phacilitate Advanced Therapies Awards.
To be honest, we didn’t even know we’d been nominated. But who cares. We are now in the final. And we are in good company. Our friends at Americans for Cures, were also nominated. They are advocates for stem cell research in California and were hugely instrumental in getting Proposition 14 passed in 2020, that’s the voter initiative that refunded CIRM with $5.5 billion.
While we may focus on different areas we all share a common goal, a desire to ensure that the voice of the patient is front and center in all that we do. At CIRM we have patient advocates on our Board and on the panel of experts who review applications for our funding. We have patient advocates helping guide the clinical trials we fund. And now, as we expand our efforts to reach out in every community in California, we have patients and patient advocates guiding that work as well.
We do this work because it’s important and because, without the support of the patient advocacy community, we wouldn’t be here.
It’s an old cliché that when you are in this position you say, “it’s an honor just to be nominated.” But in this case, it’s true.
At the California Institute for Regenerative Medicine (CIRM) we are fortunate in having enough money to fund the most promising research to be tested in a clinical trial. Those are expensive projects, often costing tens of millions of dollars. But sometimes the projects that come to our Board start out years before in much more humble circumstances, raising money through patient advocates, tapping into the commitment and ingenuity of those affected by a disease, to help advance the search for a treatment.
That was definitely the case with a program the CIRM Board voted to approve yesterday, investing more than $11 million dollars to fund a Phase 2 clinical trial testing a cell therapy for dysphagia. That’s a debilitating condition that affects many people treated for head and neck cancer.
Patients with head and neck cancer often undergo surgery and/or radiation to remove the tumors. As a result, they may develop problems swallowing and this can lead to serious complications such as malnutrition, dehydration, social isolation, or a dependence on using a feeding tube. Patients may also inhale food or liquids into their lungs causing infections, pneumonia and death. The only effective therapy is a total laryngectomy where the larynx or voice box is removed, leaving the person unable to speak.
Dr. Peter Belafsky and his team at the University of California at Davis are developing a therapeutic approach using Autologous Muscle Derived Progenitor Cells (AMDC), cells derived from a biopsy of the patient’s own muscle, elsewhere in the body. Those AMDCs are injected into the tongue of the patient, where they fuse with existing muscle fibers to increase tongue strength and ability to swallow.
The $11,015,936 that Dr. Belafsky is getting from CIRM will enable them to test this approach in patients. But without grass roots support the program might never have made it this far.
Ed Steger is a long-term survivor of head and neck cancer, he’s also the President of the National Foundation of Swallowing Disorders (NFOSD). In 2007, after being treated for his cancer, Ed developed a severe swallowing disorder. It helped motivate him to push for better treatment options.
In 2013, a dozen swallowing disorder patients visited UC Davis to learn how stem cells might help people with dysphagia. (You can read about that visit here). Ed says: “We were beyond thrilled with the possibilities and drawing on patients and other UCD contacts our foundation raised enough funds to support a small UCD clinical trial under the guidance of Dr. Belafsky in mouse models that demonstrated these possibilities.”
A few years later that small funding by patients and their family members grew into a well-funded Phase I/II human clinical trial. Ed says the data that trial produced is helping advance the search for treatments.
“Skipping forward to the present, this has now blossomed into an additional $11 million grant, from CIRM, to continue the work that could be a game changer for millions of Americans who suffer annually from oral phase dysphagia. My hat is off to all those that have made this possible… the donors, patient advocates, and the dedicated committed researchers and physicians who are performing this promising and innovative research.”
Our hats are off to them too. Their efforts are making what once might have seemed impossible, a real possibility.
It’s traditional this time of year to send messages of gratitude to friends and family and colleagues. And we certainly have much to be thankful for.
Thanks to the voters of California, who passed Proposition 14, we have a bright, and busy, future. We have $5.5 billion to continue our mission of accelerating stem cell treatments to patients with unmet medical needs.
That means the pipeline of promising projects that we have supported from an early stage can now apply to us to help take that work out of the lab and into people.
It means research areas, particularly early-stage work, where we had to reduce our funding as we ran out of money can now look forward to increased support.
It means we can do more to bring this research, and it’s potential benefits, to communities that in the past were overlooked.
We have so many people to thank for all this. The scientists who do the work and championed our cause at the ballot box. The voters of California who once again showed their support for and faith in science. And the patients and patient advocates, the reason we were created and the reason we come to work every day.
As Dr. Maria Millan, our President & CEO, said in a letter to our team; “We are continually faced by great opportunities brilliantly disguised as insoluble problems.” Here’s to the opportunities made possible by CIRM and for its continuation made possible by Prop 14!”
And none of this would be possible without the support of all of you. And for that we are truly Thankful.
From everyone at CIRM, we wish you a happy, peaceful and safe Thanksgiving.
We often talk about the important role that patient advocates play in helping advance research. That was demonstrated in a powerful way last week when the CIRM Board approved almost $12 million to fund a clinical trial targeting a rare childhood disorder called cystinosis.
The award, to Stephanie Cherqui and her team at UC San Diego (in collaboration with UCLA) was based on the scientific merits of the program. But without the help of the cystinosis patient advocate community that would never have happened. Years ago the community held a series of fundraisers, bake sales etc., and used the money to help Dr. Cherqui get her research started.
That money enabled Dr. Cherqui to get the data she needed to apply to CIRM for funding to do more detailed research, which led to her award last week. There to celebrate the moment was Nancy Stack. Her testimony to the Board was a moving celebration of how long they have worked to get to this moment, and how much hope this research is giving them.
Nancy Stack is pictured in spring 2018 with her daughter Natalie Stack and husband Geoffrey Stack. (Lar Wanberg/Cystinosis Research Foundation)
Hello my name is Nancy Stack and I am the founder and president of the Cystinosis Research Foundation. Our daughter Natalie was diagnosed with cystinosis when she was an infant.
Cystinosis is
a rare disease that is characterized by the abnormal accumulation of cystine in
every cell in the body. The build-up of
cystine eventually destroys every organ in the body including the kidneys,
eyes, liver, muscles, thyroid and brain.
The average age of death from cystinosis and its complications is 28
years of age.
For our
children and adults with cystinosis, there are no healthy days. They take
between 8-12 medications around the clock every day just to stay alive –
Natalie takes 45 pills a day. It is a
relentless and devastating disease.
Medical
complications abound and our children’s lives are filled with a myriad of
symptoms and treatments – there are g-tube feedings, kidney transplants, bone
pain, daily vomiting, swallowing
difficulties, muscle wasting, severe gastrointestinal side effects and for some
blindness.
We started
the Foundation in 2003. We have worked
with and funded Dr. Stephanie Cherqui since 2006. As a foundation, our resources are limited
but we were able to fund the initial grants for Stephanie’s Stem Cell studies. When CIRM awarded a grant
to Stephanie in 2016, it allowed her to complete the studies, file the IND and
as a result, we now have FDA approval
for the clinical trial. Your support has changed the course of this
disease.
When the FDA
approved the clinical trial for cystinosis last year, our community was filled
with a renewed sense of hope and optimism.
I heard from 32 adults with cystinosis – all of them interested in the
clinical trial. Our adults know that
this is their only chance to live a full life. Without this treatment, they
will die from cystinosis. In every
email I received, there was a message of hope and gratitude.
I received an
email from a young woman who said this, “It’s a new awakening to learn this
morning that human clinical trials have been approved by the FDA. I reiterate
my immense interest to participate in this trial as soon as possible because my
quality of life is at a low ebb and the trial is really my only hope. Time is
running out”.
And a mom of a 19 year old young man who wants to be the first
patient in the trial wrote and said this, “On the day the trial was announced I started to cry tears of pure
happiness and I thought, a mother somewhere gets to wake up and have a child who
will no longer have cystinosis. I felt so happy for whom ever that mom would
be….I never imagined that the mom I was thinking about could be me. I am so
humbled to have this opportunity for my son to try to live disease free.
My own daughter ran into my arms that day and we cried tears of
joy – finally, the hope we had clung to was now a reality. We had come full
circle. I asked Natalie how it felt to
know that she could be cured and she said, “I have spent my entire life thinking
that I would die from cystinosis in my 30s but now, I might live a full life
and I am thinking about how much that changes how I think about my future. I never
planned too far ahead but now I can”.
As a mother, words can’t possible convey what it feels like to know that my child has a chance to live a long, healthy life free of cystinosis – I can breathe again. On behalf of all the children and adults with cystinosis, thank you for funding Dr. Cherqui, for caring about our community, for valuing our children and for making this treatment a reality. Our community is ready to start this trial – thank you for making this happen.
*************
CIRM will be celebrating the role of patient advocates at a free event in Los Angeles tomorrow. It’s at the LA Convention Center and here are the details. And did I mention it’s FREE!
Tue, June 25, 2019 – 6:00 PM – 7:00 PM PDT
Petree Hall C., Los Angeles Convention Center, 1201 South Figueroa Street Los Angeles, CA 90015
And on Wednesday, USC is holding an event highlighting the progress being made in fighting diseases that destroy vision. Here’s a link to information about the event.
Yesterday the CIRM Board approved funding for our 50th clinical trial (you can read about that here) It was an historic moment for us and to celebrate we decided to go back in history and hear from the very first person to be treated in a CIRM-funded clinical trial. Rich Lajara was treated in the Geron clinical trial after experiencing a spinal cord injury, thus he became CIRM’s patient #1. It’s a badge he says he is honored to wear. This is the speech Rich made to our Board.
Hello and good afternoon everyone. It’s an honor to be here today as the 50th clinical trial has been officially funded by CIRM. It was feels like it was just yesterday that I was enrolled into the first funded clinical trial by CIRM and in turn became California’s’ 1st embryonic stem cell patient.
I became paralyzed from the waist down in September 2011. It was Labor Day and I was at a river with some close friends. There was this natural granite rock slide feature next to a waterfall, it was about 60 feet long; all you had to do was get a bucket of water to get the rocks wet and slide down into a natural pool. I ended up slipping and went down head first backwards but was too far over and I slid off a 15’ ledge where the waterfall was. I was pulled from the water and banged up pretty bad. Someone said “look at that deformity on his back” and tapped my leg and asked if I could feel that. I knew immediately I was paralyzed. I thought this was the end, little did I know this was just the beginning. I call it being in the wrong place at the right time.
So, after a few days in the hospital of course everyone, as well as myself, wanted a cure. We quickly learned one didn’t exist. A close family friend had been making phone calls and was able to connect with the Christopher & Dana Reeve Foundation and learned about a clinical trial with “stem cells”. One of my biggest question was how any people have done this? “Close to none”, I was told.
I was also told I most likely would have no direct benefit as this was a safety trial? So why do it at all? Obviously at that time I was willing to overlook the “most likely” part because I was willing to do anything to try and get my normal life back.
Looking back the big picture was laying the ground work for others like Kris or Jake (two people enrolled in a later version of this trial). At the time I had no clue that what I was doing would be such a big deal. The data collected from me would end up being priceless. It’s stories like Jake’s and Kris’ that make me proud and reinforce my decision to have participated in California’s first stem cell clinical trial funded by prop 71.
Like I said earlier it was just the beginning for me. A couple of years later I became a patient advocate working with Americans for Cures. I have been able to meet many people in the stem cell industry and love to see the glow in their face when they learn I was California’s first embryonic stem cell patient.
I can’t even fathom all the year’s of hard work and countless hours of research that had lead up to my long anticipated surgery, but when I see their glowing smile I know they knew what it took.
I also enjoy sharing my story and bridging the gap between myths and facts about stem cells, or talking to students and helping inspire the next generation that will be in the stem cell industry. As a matter of fact, I have 13 year old sister, Maddie, dead set on being a neurosurgeon.
Fast forward to today. Life in a wheelchair is not exactly a roll in the park (no pun intended) but I have grown accustomed to the new normal. Aside from some neuropathic pain, life is back on track.
Not once did I feel sorry for myself, I was excited to be alive. Sure I have bad days but don’t we all.
In the last 14 years CIRM has funded 50 human clinical trials, published around 2750 new peer-reviewed scientific discoveries, and they’ve transformed California into the world leader in stem cell research. As I look around the posters on the wall, of the people whose lives have been transformed by the agency, I can’t help but be struck by just how much has been achieved in such a short period of time.
While my journey might not yet be over, Evie and 40 other children like her, (children born with SCID) will never remember what it was like to live with the horrible condition they were born with because they have been cured thanks to CIRM. There are hundreds of others whose lives have been transformed because of work the agency has funded.
CIRM has proven how much can be achieved if we invest in cutting-edge medical research.
As most of you here probably know CIRM’s funding from Proposition 71 is about to run out. If I had just one message I wanted people to leave with today it would be this. Everyone in this room knows how much CIRM has done in a little over a decade and how many lives have been changed because of its existence. We have the responsibility to make sure this work continues. We have a responsibility to make sure that the stories we’ve heard today are just the beginning.
I will do everything I can to make sure the agency gets refunded and I hope that all of you will join me in that fight. I’m excited for the world of stem cells, particularly in California, and can’t wait to see what’s on the horizon.
Let’s face it, when you are feeling crummy all you want to do is be quiet, rest and not have to deal with anyone else. So, it’s not surprising that a new survey of people with primary mitochondrial disease (PMD) found that many were often less than enthusiastic about taking part in a clinical trial.
It’s not surprising because PMD, caused by problems with the mitochondria which provide energy within our cells, can lead to a wide variety of debilitating conditions including muscle weakness, visual problems, hearing problems, heart disease, liver disease, kidney disease, gastrointestinal disorders, breathing problems, neurological problems and dementia. Any one of those is bad enough, but if you combine several you can see why it would be hard for a person with PMD to get to a clinical trial site for an experimental therapy.
That’s unfortunate because right now there are no effective treatments for PMD so it’s vitally important that people take part in clinical trials that might lead to new therapies.
Obstacles and opportunities
Fortunately, this study, published in the journal PLOS One, did more than just identify the barriers to taking part in a clinical trial, it also identified some strategies to overcome those barriers.
The barriers included not just the individual’s state of health but also:
Requiring patients to discontinue current medications
Daily blood tests
Requiring patients to pay for the cost of the clinical trial
Ways to encourage increased participation include:
Direct communication with a physician involved in the trial
Better education and outreach to people with PMD
Working with patient advocacy groups
The study says this last point in particular is extremely important.
“We propose widespread, coordinated efforts that involve PMD patient advocacy groups to organize community education sessions that clarify the components and need for efficacious clinical trial design.”
CIRM CAP meeting
This is something that CIRM knows a lot about. Whenever we fund a clinical trial – or, in some cases a late stage pre-clinical program – we create a Clinical Advisory Panel (CAP) to support it. Each CAP consists of an independent, outside expert in whatever disease the trial is targeting, a CIRM Science Officer, and a Patient Advocate. The Patient Advocate plays a vital role in making sure this project works.
Researchers know the science, but the Patient Advocate knows what it is like to live with the disease and the limitations it may impose. They can help guide and advise the researchers on how to design a clinical trial that works for the patients and makes it as easy as possible for them to be part of the trial.
In the last few years we have created 68 CAPs, ensuring the voice of the patient, and the needs of the patient, are front and center in everything we do.
The easier it is for the patient, the easier it will be to recruit people for the trial and the more likely it is they will stay with the trial to the end. It won’t guarantee the therapy will succeed, but it gives it the best possible chance.
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.
Speeding up the way you do things isn’t always a good idea. Just ask someone who got a ticket for going 65mph in a 30mph zone. But at CIRM we have found that doing things at an accelerated pace is paying off in a big way.
When CIRM started back in 2004 we were, in many ways, a unique organization. That meant we pretty much had to build everything from scratch, creating our own ways of asking for applications, reviewing those applications, funding them etc. Fast forward ten years and it was clear that, as good a job as we did in those early days, there was room for improvement in the way we operated.
So we made some changes. Big changes.
We adopted as our mantra the phrase “operational excellence.” It doesn’t exactly trip off the tongue but it does reflect what we were aiming for. The Business Dictionary defines operational excellence as:
“A philosophy of the workplace where problem-solving, teamwork, and leadership results in the ongoing improvement in an organization.”
We didn’t want to just tinker with the way we worked, we wanted to reinvent every aspect of our operation. To do that we involved everyone in the operation. We held a series of meetings where everyone at CIRM, and I do mean everyone, was invited to join in and offer their ideas on how to improve our operation.
The end result was CIRM 2.0. At the time we described it as “a radical overhaul” of the way we worked. That might have been an understatement. We increased the speed, frequency and volume of the programs we offered, making it easier and more predictable for researchers to apply to us for funding, and faster for them to get that funding if they were approved.
For example, before 2.0 it took almost two years to go from applying for funding for a clinical trial to actually getting that funding. Today it takes around 120 days.
But it’s not just about speed. It’s also about working smarter. In the past if a researcher’s application for funding for a clinical trial failed it could be another 12 months before they got a chance to apply again. With many diseases 12 months could be a death sentence. So we changed the rules. Now if you have a project ready for a clinical trial you can apply any time. And instead of recommending or not recommending a project, basically voting it up or down, our independent panel of expert reviewers now give researchers with good but not great applications constructive feedback, enabling the researchers to make the changes needed to improve their project, and reapply for funding within 30 days.
This has not only increased the number of applications for clinical trials, it has also increased the quality of those applications.
We made similar changes in our Discovery and Translation programs. Increasing the frequency of each award, making it easier for researchers to know when the next round of funding was coming up. And we added incentives to encourage researchers to move successful projects on to the next level. We wanted to create a pipeline of the most promising projects steadily moving towards the clinic.
The motivation to do this comes from our patients. At CIRM we are in the time business. Many of the patients who are looking to stem cells to help them don’t have the luxury of time; they are rapidly running out of it. So we have a responsibility to do all we can to reduce the amount of time it takes to get the most promising therapies to them, without in any way compromising safety and jeopardizing their health.
By the end of 2016 those changes were very clearly paying dividends as we increased the frequency of reviews and the number of projects we reviewed but at the same time decreased the amount of time it took us to do all that.
But we are not done yet. We have done a good job of improving the way we work. But there is always room to be even better, to go even faster and be more efficient.
Patients and Patient Advocates are at the heart of everything we do at CIRM. That’s why we are holding three free public events in the next few months focused on updating you on the stem cell research we are funding, and our plans for the future.
Right now we have 33 projects that we have funded in clinical trials. Those range from heart disease and stroke, to cancer, diabetes, ALS (Lou Gehrig’s disease), two different forms of vision loss, spinal cord injury and HIV/AIDS. We have also helped cure dozens of children battling deadly immune disorders. But as far as we are concerned we are only just getting started.
Over the course of the next few years, we have a goal of adding dozens more clinical trials to that list, and creating a pipeline of promising therapies for a wide range of diseases and disorders.
That’s why we are holding these free public events – something we try and do every year. We want to let you know what we are doing, what we are funding, how that research is progressing, and to get your thoughts on how we can improve, what else we can do to help meet the needs of the Patient Advocate community. Your voice is important in helping shape everything we do.
The first event is at the Gladstone Institutes in San Francisco on Wednesday, September 6th from noon till 1pm. The doors open at 11am for registration and a light lunch.
Gladstone Institutes
Here’s a link to an Eventbrite page that has all the information about the event, including how you can RSVP to let us know you are coming.
We are fortunate to be joined by two great scientists, and speakers – as well as being CIRM grantees- from the Gladstone Institutes, Dr. Deepak Srivastava and Dr. Steve Finkbeiner.
Dr. Srivastava is working on regenerating heart muscle after it has been damaged. This research could not only help people recover from a heart attack, but the same principles might also enable us to regenerate other organs damaged by disease. Dr. Finkbeiner is a pioneer in diseases of the brain and has done ground breaking work in both Alzheimer’s and Huntington’s disease.
We have two other free public events coming up in October. The first is at UC Davis in Sacramento on October 10th (noon till 1pm) and the second at Cedars-Sinai in Los Angeles on October 30th (noon till 1pm). We will have more details on these events in the coming weeks.
We look forward to seeing you at one of these events and please feel free to share this information with anyone you think might be interested in attending.
It’s not hard to find people who don’t like the US Food and Drug Administration (FDA), the government agency that, among other things, regulates medical therapies. In fact, if you type “do people like the FDA?” into an internet search engine you’ll quickly find out that for a lot of people the answer is “no”.
But the Agency is trying to change and deserves credit for taking seriously many of the criticisms that have been levelled at it over the years and trying to address them.
The latest example is the news that the FDA has set a date for the first-ever meeting of its first-ever Patient Engagement Advisory Committee (PEAC). On its website, the FDA says the PEAC will be focused on patient-related issues:
“The PEAC is a forum for the voice of patients. It will be asked to advise on complex issues related to medical devices and their impact on patients. The goal of PEAC is to better understand and integrate patient perspectives into our oversight, to improve communications with patients about benefits, risks, and clinical outcomes related to medical devices, and to identify new approaches, unforeseen risks or barriers, and unintended consequences from the use of medical devices.”
In the past, the FDA has created forums to allow patients to talk about the impact of a disease on their daily life and their views on treatment options. But those were considered by many to be little more than window dressing, providing a sounding boards for patients but not actually producing any tangible benefits or changes.
The FDA also has patient representatives who take part in FDA advisory committee meetings, but the PEAC is the first time it has ever had a committee that was solely focused on patients and their needs. The nine core members of the PEAC all have experience either as patients or patient advocates and care-givers for patients. A really encouraging sign.
We tip our CAP to the FDA
At CIRM we support anything that ensures that patients not only have a seat at the table, but also that their voices are heard and taken seriously. That’s why for every clinical trial we fund (and even some pre-clinical projects too) we create what we call a Clinical Advisory Panel or CAP (we do love our acronyms).
Each CAP consists of three to five members, with a minimum of one Patient Representative, one External Advisor and one CIRM Science Officer. The purpose of the CAP is to make recommendations and provide guidance and advice to the Project Team running the trial.
Having a Patient Representative on a CAP ensures the patient’s perspective is included in shaping the design of the clinical trial, making sure that the trial is being carried out in a way that has the patient at the center. Patients can ask questions or raise issues that researchers might not think about, and can help the researchers not only do a better job of recruiting the patients they need for the trial, but also keeping those patients involved. We believe a trial designed around the patient, and with the patient in mind, is much more likely to be successful.
In announcing the formation of the PEAC the FDA said:
“Patients are at the heart of what we do. It makes sense to establish an advisory committee built just for them.”
I completely agree.
My only regret is that they didn’t call it the Patient Engagement Advisory Committee for Health, because then the acronym would have been PEACH. And this is certainly a peach of an idea, one worthy of support.
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The Stem Cellar 