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
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!
From Day One CIRM’s goal has been to advance stem cell research in California. We don’t do that just by funding the most promising research -though the 51 clinical trials we have funded to date clearly shows we do that rather well – but also by trying to bring the best minds in the field together to overcome problems.
Over the years we
have held conferences, workshops and symposiums on everything from Parkinson’s
palsy and tissue
engineering. Each one attracted the key players and stakeholders in the
field, brainstorming ideas to get past obstacles and to explore new ways of
developing therapies. It’s an attempt to get scientists, who would normally be
rivals or competitors, to collaborate and partner together in finding the best
It’s not easy to do,
and the results are not always obvious right away, but it is essential if we
hope to live up to our mission of accelerating stem cell therapies to patients
with unmet medical needs.
For example. This
past week we helped organize two big events and were participants in another.
The first event we
pulled together, in partnership with Cedars-Sinai Medical Center, was a
workshop called “Brainstorm Neurodegeneration”. It brought together leaders in stem
cell research, genomics, big data, patient advocacy and the Food and Drug
Administration (FDA) to tackle some of the issues that have hampered progress
in finding treatments for things like Parkinson’s, Alzheimer’s, ALS and
ambitiously subtitled the workshop “a cutting-edge meeting to disrupt the field”
and while the two days of discussions didn’t resolve all the problems facing us
it did produce some fascinating ideas and some tantalizing glimpses at ways to
advance the field.
Two days later we partnered with UC San Francisco to host the Fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium. This brought together the scientists who develop therapies, the doctors and nurses who deliver them, and the patients who are in need of them. The theme was “The Past, Present & Future of Regenerative Medicine” and included both a look at the initial discoveries in gene therapy that led us to where we are now as well as a look to the future when cellular therapies, we believe, will become a routine option for patients.
different groups together is important for us. We feel each has a key role to
play in moving these projects and out of the lab and into clinical trials and
that it is only by working together that they can succeed in producing the
treatments and cures patients so desperately need.
As always it was the patients who surprised us. One, Cierra Danielle Jackson, talked about what it was like to be cured of her sickle cell disease. I think it’s fair to say that most in the audience expected Cierra to talk about her delight at no longer having the crippling and life-threatening condition. And she did. But she also talked about how hard it was adjusting to this new reality.
Cierra said sickle
cell disease had been a part of her life for all her life, it shaped her daily
life and her relationships with her family and many others. So, to suddenly
have that no longer be a part of her caused a kind of identity crisis. Who was
she now that she was no longer someone with sickle cell disease?
She talked about how
people with most diseases were normal before they got sick, and will be normal
after they are cured. But for people with sickle cell, being sick is all they
have known. That was their normal. And now they have to adjust to a new normal.
It was a powerful
reminder to everyone that in developing new treatments we have to consider the
whole person, their psychological and emotional sides as well as the physical.
And so on to the third event we were part of, the Stanford Drug Discovery Symposium. This was a high level, invitation-only scientific meeting that included some heavy hitters – such as Nobel Prize winners Paul Berg and Randy Schekman, former FDA Commissioner Robert Califf. Over the course of two days they examined the role that philanthropy plays in advancing research, the increasingly important role of immunotherapy in battling diseases like cancer and how tools such as artificial intelligence and big data are shaping the future.
CIRM’s President and CEO, Dr. Maria Millan, was one of those invited to speak and she talked about how California’s investment in stem cell research is delivering Something Better than Hope – which by a happy coincidence is the title of our 2018 Annual Report. She highlighted some of the 51 clinical trials we have funded, and the lives that have been changed and saved by this research.
The presentations at
these conferences and workshops are important, but so too are the conversations
that happen outside the auditorium, over lunch or at coffee. Many great
collaborations have happened when scientists get a chance to share ideas, or
when researchers talk to patients about their ideas for a successful clinical
It’s amazing what happens when you bring people together who might otherwise never have met. The ideas they come up with can change the world.
Blood is the lifeline of the body. The continuous, unimpeded circulation of blood maintains oxygen flow throughout the body and enables us to carry out our everyday activities. Unfortunately, there are individuals whose own bodies are in a constant battle that prevents this from occurring seamlessly. They have something known as sickle cell disease (SCD), an inherited condition caused by a mutation in a single gene. Rather than producing normal, circular red blood cells, their bodies produce sickle shaped cells (hence the name) that can become lodged in blood vessels, preventing blood flow. The lack of blood flow can cause agonizing pain, known as crises, as well as strokes. Chronic crises can cause organ damage, which can eventually lead to organ failure. Additionally, since the misshapen cells don’t survive long in the body, people with SCD have a greater risk of being severely anemic and are more prone to infections. Monthly blood transfusions are often needed to help temporarily alleviate symptoms. Due to the debilitating nature of SCD, important aspects of everyday life such as employment and health insurance can be extremely challenging to find and maintain.
An estimated 100,000 people in the United States are living with SCD. Around the world, about 300,000 infants are born with the condition each year, a statistic that will increase to 400,000 by 2050 according to one study. Many people with SCD do not live past the age of 50. It is most prevalent in individuals with sub-Saharan African descent followed by people of Hispanic descent. Experts have stated that advances in treatment have been limited in part because SCD is concentrated in poorer minority communities.
Despite these grim statistics and prognosis, there is hope.
The New York Times and Boston Herald recently released featured articles that tell the personal stories of patients enrolled in a clinical trial conducted by bluebird bio. The trial uses gene therapy in combination with hematopoietic (blood) stem cells (HSCs) to give rise to normal red blood cells in SCD patients.
Here are the stories of these patients. To read the full New York Times article, click here. For the Boston Herald article, click here.
Emmanuel “Manny” Johnson was the very first patient in the SCD trial. He was motivated to participate in the trial not just for himself but for his younger brother Aiden Johnson, who was also born with SCD. Manny has a tattoo with Aiden’s name written inside a red sickle cell awareness ribbon.
In the article Manny is quoted as saying “It’s not only that we share the same blood disease, it’s like I have to do better for him.”
Since receiving the treatment, Manny’s SCD symptoms have disappeared.
For Brandon Williams of Chicago, the story of SCD is a very personal one. At just 21 years old, Brandon had suffered four strokes by the time he turned 18. His older sister, Britney Williams, died of sickle cell disease at the age of 22. Brandon was devastated and felt that his own life could end at any moment. He was then told about the SCD trial and decided to enroll. Following the treatment, his symptoms have vanished along with the pain and fear inflicted by the disease.
The NY Times piece also profiles Carmen Duncan, a 20 year old from Charleston, South Carolina. She had her spleen removed when she was just two years old as a result of complications form SCD. Duncan spent a large portion of her childhood in hospitals, coping with the pain in her arms and legs from blocked blood vessels. She enrolled in the SCD trial as well and she no longer has any signs of SCD. Duncan had aspirations to join the military but was unable to because of her condition. Now that she is symptom free, she plans to enlist.