When people ask me what I do at CIRM I sometimes half-jokingly tell them that I’m the official translator: I take complex science and turn it into everyday English. That’s important. The taxpayers of California have a right to know how their money is being spent and how it might benefit them. But that message can be even more effective when it comes from the scientists themselves.
Recently we asked some of the scientists we are funding to do research into COVID-19 to record what’s called an “elevator pitch”. This is where they prepare an explanation of their work that is in ordinary English and is quite short, short enough to say it to someone as you ride in an elevator. Hence the name.
It sounds easy enough. But it’s not. When you are used to talking in the language of science day in and day out, suddenly switching codes to talk about your work in plain English can take some practice. Also, you have spent years, often decades, on this work and to have to explain it in around one minute is no easy thing.
But our researchers rose to the challenge. Here’s some examples of just how well they did.
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!
This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two additional discovery research projects as part of the $5 million in emergency funding for COVID-19 related projects. This brings the number of COVID-19 projects CIRM is supporting to 15, including three clinical trials.
The Board awarded $249,999 to Dr. Evan Snyder at the Sanford Burnham Prebys Medical Discovery Institute. The study will use induced pluripotent stem cells (iPSCs), a type of stem cell that can be created by reprogramming skin or blood cells, to create lung organoids. These lung organoids will then be infected with the novel coronavirus in order to test two drug candidates for treatment of the virus. The iPSCs and the subsequent lung organoids created will reflect diversity by including male and female patients from the Caucasian, African-American, and Latinx population.
This award is part of CIRM’s Quest Awards Program (DISC2), which promotes promising new technologies that could be translated to enable broad use and improve patient care.
The Board also awarded $150,000 to Dr. Steven Dowdy at UC San Diego for development of another potential treatment for COVID-19.
Dr. Dowdy and his team are working on developing a new, and hopefully more effective, way of delivering a genetic medicine, called siRNA, into the lungs of infected patients. In the past trying to do this proved problematic as the siRNA did not reach the appropriate compartment in the cell to become effective. However, the team will use an iPSC lung model to help them identify ways past this barrier so the siRNA can attack the virus and stop it replicating and spreading throughout the lungs.
This award is part of CIRM’s Inception Awards Program (DISC1), which supports transformational ideas that require the generation of additional data.
A supplemental award of $250,000 was approved for Dr. John Zaia at City of Hope to continue support of a CIRM funded clinical study that is using convalescent plasma to treat COVID-19 patients. The team recently launched a website to enroll patients, recruit plasma donors, and help physicians enroll their patients.
“The use of induced pluripotent stem cells has expanded the potential for personalized medicine,” says Dr. Maria T. Millan, the President & CEO of CIRM. “Using patient derived cells has enabled researchers to develop lung organoids and lung specific cells to test numerous COVID-19 therapies.”
Sometimes it’s the smallest things that make the biggest difference. In the case of a clinical trial that CIRM is funding, all it takes to be part of it is four teaspoons of blood.
The clinical trial is being run by Dr. John Zaia and his team at the City of Hope in Duarte, near Los Angeles, in partnership with tgen and the CIRM Alpha Stem Cell Clinic Network. They are going to use blood plasma from people who have recovered from COVID-19 to treat people newly infected with the virus. The hope is that antibodies in the plasma, which can help fight infections, will reduce the severity or length of infection in others.
People who have had the virus and are interested in taking part are asked to give four teaspoons of blood, to see if they have enough antibodies. If they do they can then either donate plasma – to help newly infected people – or blood to help with research into COVID-19.
As a sign of how quickly Dr. Zaia and his team are working, while we only approved the award in late April, they already have their website up and running, promoting the trial and trying to recruit both recovered COVID-19 survivors and current patients.
The site does a great job of explaining what they are trying to do and why people should take part. Here’s one section from the site.
Why should I participate in your study?
By participating in our study, you will learn whether you have developed antibodies against SARS-CoV-2, the virus responsible for COVID-19. To do so, you just need to donate a small sample of blood (approximately 4 teaspoons).
If testing show you have enough antibodies, you will have the option of donating plasma that will be used to treat severely ill COVID-19 patients and may help save lives.
If you don’t want to donate plasma, you can still donate blood (approximately 3.5 tablespoons), which will be studied and help researchers learn more about COVID-19.
By donating blood or plasma, you will help us gain information that may be of significant value for patient management in future epidemic seasons.
You don’t even have to live close to one of the clinical trial sites because the team can send you a blood collection kit and information about a blood lab near you so you can donate there. They may even send a nurse to collect your blood.
The team is also trying to ensure they reach communities that are often overlooked in clinical trials. That’s why the website is also in Spanish and Vietnamese.
Finally, the site is also being used to help recruit treating physicians who can collect the blood samples and help infuse newly infected patients.
We often read about clinical trials in newspapers and online. Now you get a chance to not only see one working in real time, you can get to be part of it.
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) continued its commitment to help with the coronavirus pandemic by awarding $749,999 to Dr. John Zaia at City of Hope. He will be conducting a clinical study to administer blood plasma from recovered COVID-19 patients to treat those with the virus. This marks CIRM’s first clinical study for COVID-19 after approving emergency funding a month earlier.
Plasma is a component of blood that carries proteins called antibodies that are usually involved in defending our bodies against viral infections. Blood plasma from patients that have recovered from COVID-19, referred to as convalescent plasma, contain antibodies against the virus that can be used as a potential treatment for COVID-19. Currently, there are challenges with this approach that include: properly identifying convalescent plasma donors i.e. recovered patients, determining eligibility of those with convalescent plasma that want to donate, collection of the plasma, treating patients, and determining if the plasma was effective.
Dr. Zaia and his team at City of Hope will create the COVID-19 Coordination Program, which addresses solutions for all of the challenges listed above. The program will partner with the medical teams at CIRM’s Alpha Stem Cell Clinic Network, as well as infectious disease, pulmonary and critical care teams from medical centers and community hospitals across the state. Potential donors will be identified and thoroughly screened for eligibility per the established National and State blood banking safety requirements. Finally, the convalescent plasma will be collected from eligible donors and administered by licensed physicians to COVID-19 patients, who will be evaluated for response to the treatment and potential recovery.
“We are in the midst of very challenging times where there is not yet an approved treatment for COVID-19. In response to this, CIRM launched and executed an emergency COVID-19 funding program, which was made possible by our Board, patient advocates, California scientists, external scientific expert reviewers, and our dedicated team,” said Maria T. Millan, MD, President and CEO of CIRM. “With CIRM funding, the City of Hope COVID-19 Coordination program will tap into CIRM’s network of researchers, physicians, and our Alpha Clinics to deliver this treatment to patients in need. It will also serve the critical role of gathering important scientific data about the plasma, safety, and clinical data from treated patients.”
The Board also approved a discovery stage research project that utilizes stem cell models for a novel approach to vaccine development against the virus causing COVID-19 and another project that uses a unique lung stem cell organoid to identify an effective drug against the virus.