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.
There are a growing number of predatory clinics in California and around the US, offering unproven stem cell therapies. For patients seeking a legitimate therapy it can often be hard finding a reliable clinic, one offering treatments based on the rigorous science required in a clinical trial sanctioned by the US Food and Drug Administration (FDA). That’s one of the reasons why the California Institute for Regenerative Medicine (CIRM) created the CIRM Alpha Stem Cell Clinic Network and we are delighted the clinics have now been chosen as a Core program of the American Society of Hematology (ASH) Sickle Cell Disease (SCD) Collaborative Trials Network.
The Alpha Clinics are a network of top California medical centers that specialize in delivering stem cell clinical trials to patients. It consists of five leading medical centers throughout California: City of Hope, University of California (UC) San Diego, UC Irvine & UC Los Angeles, UC Davis and UC San Francisco.
The mission of the ASH Research Collaborative SCD Clinical Trials Network is to improve outcomes for individuals with Sickle Cell Disease by promoting innovation in therapy development and clinical trial research.
“The key to finding a cure for this crippling disease, and finding it quickly, is to work together”, says Maria T. Millan, MD, President & CEO of CIRM. “That’s why we are delighted to be chosen as a core program for the ASH Sickle Cell Disease Clinical Trials Network. This partnership means we can share data and information about best practices to help us improve the quality of the research being done and the clinical care we can offer patients. We already have 23 clinical stage therapies in cell and gene therapy, including two clinical trials targeting SCD, so we feel we have a lot to bring to the partnership in terms of experience and expertise.”
Sickle Cell disease is a life-threatening blood disorder that affects 100,000 people, mostly African Americans, in the US. It is caused by a single genetic mutation that results in the production of “sickle” shaped red blood cells that can block blood vessels causing intense pain, recurrent hospitalization, multi-organ damage and strokes.
“We hear a lot about the moonshot for curing cancer, but a moonshot for curing sickle cell disease should also be possible. Sickle cell disease was the first genetic disease that was discovered, and wouldn’t it be great if it is also one of the first ones we can cure in everyone?”
It is hoped that creating this network of clinical trial sites across the US will better serve an historically under-served population.
Establishing links and educational materials across these sites can increase patient engagement and recruitment
Standardizing resources across the network can ensure efficiency and coordination
Improving the training of clinical research staff can promote patient safety and trust and increase research quality
The CIRM Alpha Clinics Network has a proven track record of creating a faster, more streamlined approach in running clinical trials. It has developed the tools and systems to simultaneously launch clinical trials at multiple sites; created model non-disclosure agreements to make it easier for clinical trial sponsors to sign up; created a system to enable one Institutional Review Board (IRB) to approve a trial to be carried out at multiple sites rather than requiring each site to have its own IRB approval; developed best practices to quickly share experience and expertise across the network; and set up a database of over 20 million Californians to improve patient recruitment.
An Executive Summary prepared for the Western States Sickle Cell Disease Clinical Trials Network said: “the ASCC provides a formidable clinical trial unit uniquely qualified to deliver the next generation of cell and gene therapy products for SCD.”
CIRM Board approves first program eligible for co-funding under the agreement
disease (SCD) is a painful, life-threatening blood disorder that affects around
100,000 people, mostly African Americans, in the US. Even with optimal medical care, SCD shortens expected
lifespan by decades. It is caused by a
single genetic mutation that results in the production of “sickle” shaped red
blood cells. Under a variety of
environmental conditions, stress or viral illness, these abnormal red
blood cells cause severe anemia and blockage of blood vessels leading to
painful crisis episodes, recurrent hospitalization, multi-organ damage and
On April 29th the governing Board of the
California Institute for Regenerative Medicine (CIRM) approved $4.49 million to
Dr. Mark Walters at UCSF Benioff Children’s Hospital in Oakland to pursue a
gene therapy cure for this
devastating disease. The gene therapy approach uses CRISPR-Cas9
technology to correct the genetic mutation that leads to sickle cell disease. This program will be eligible for
co-funding under the landmark agreement between CIRM and the National Heart,
Lung and Blood Institute (NHLBI) of the NIH.
This CIRM-NHLBI agreement
was finalized this month to co-fund cell and gene therapy programs under the
NIH “Cure Sickle Cell” initiative. The
goal is to markedly accelerate the development of cell and gene therapies for
SCD. It will deploy CIRM’s resources and expertise that has led to a portfolio of over 50 clinical
trials in stem cell and
“CIRM currently has 23 clinical stage programs in cell and
gene therapy. Given the advancements in
these approaches for a variety of unmet medical needs, we are excited about the
prospect of leveraging this to NIH-NHLBI’s Cure Sickle Cell Initiative,” says
Maria T. Millan, M.D., the President and CEO of CIRM. “We are pleased the NHLBI
sees value in CIRM’s acceleration and funding program and look forward to the
partnership to accelerate cures for sickle cell disease.”
“There is a real
need for a new approach to treating SCD and making life easier for people with
SCD and their families,” says Adrienne Shapiro, the mother of a daughter with
SCD and the co-founder of Axis Advocacy, a sickle cell advocacy and
education organization. “Finding a cure for Sickle Cell would mean that people
like my daughter would no longer have to live their life in short spurts,
constantly having their hopes and dreams derailed by ER visits and hospital
stays. It would mean they get a chance
to live a long life, a healthy life, a normal life.”
CIRM is currently funding two other clinical trials for SCD using different approaches. One of these trials is being conducted at City of Hope and the other trial is being conducted at UCLA.
The live broadcast will feature two scientists and a patient advocate who are working hard to bring an end to sickle cell disease, a devastating, inherited blood disorder that largely targets the African-American community and to a lesser degree the Hispanic community.
We want to answer your most pressing questions, so please email them directly to us beforehand at firstname.lastname@example.org.
For a sneak preview here’s a short video featuring our patient advocate speaker, Adrienne Shapiro. And see below for more details about Ms. Shapiro and our two other guests.
Adrienne Shapiro [Video: Todd Dubnicoff/CIRM]
Donald Kohn, MD
Don Kohn, M.D. is a professor in the departments of Pediatrics and Microbiology, Immunology and Molecular Genetics in UCLA’s Broad Stem Cell Research Center. Dr. Kohn has a CIRM Clinical Stage Research grant in support of his team’s Phase 1 clinical trial which is genetically modifying a patient’s own blood stem cells to produce a correct version of hemoglobin, the protein that is mutated in these patients, which causes abnormal sickle-like shaped red blood cells. These misshapen cells lead to dangerous blood clots, debilitating pain and even death. The genetically modified stem cells will be given back to the patient to create a new sickle cell-free blood supply.
Mark Walters, MD
Mark Walters, M.D., is a pediatric hematologist/oncologist and is director of the Blood & Marrow Transplantation Program at UCSF Benioff Children’s Hospital Oakland. Dr. Walters has a CIRM-funded Therapeutic Translation Research grant which aims to improve Sickle Cell Disease (SCD) therapy by preparing for a clinical trial that might cure SCD after giving back sickle gene-corrected blood stem cells – using cutting-edge CRISPR gene editing technology – to a person with SCD. If successful, this would be a universal life-saving and cost-saving therapy.
Adrienne Shapiro is a patient advocate for SCD and the co-founder of the Axis Advocacy SCD patient education and support website. Shapiro is the fourth generation of mothers in her family to have children born with sickle cell disease. She is vocal stem cell activist, speaking to various groups about the importance of CIRM’s investments in both early stage research and clinical trials. In January, she was awarded a Stem Cell and Regenerative Medicine Action Award at the 2018 World Stem Cell Summit.