CIRM approves $8 million award for plasma cell disorder therapy, $27 million to boost collaboration among California researchers
FOR IMMEDIATE RELEASE
Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, July 25, 2024 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, awarded nearly $35 million to fund clinical research to address a rare plasma cell disorder and six new Shared Resources Laboratories, an important component of CIRM’s infrastructure program.
The $8 million clinical award given to Ilya Rachman, MD, PhD, of Nexcella, Inc. will advance a therapy aimed at treating Amyloid light-chain amyloidosis (AL amyloidosis), a rare plasma cell disorder in which the body’s immune system produces abnormal forms of antibodies called “light chains.”
The award is made through the Agency’s clinical program, which provides funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development. This award brings the number of CIRM-funded clinical trials to 109.
In the United States, approximately 4,000 new cases of AL amyloidosis are diagnosed annually. Patients with this condition face a significant reduction in their quality of life due to the debilitating symptoms and the need for frequent medical interventions. The burden of living with a rare disease also places emotional and financial strains on patients and their families.
There is no cure for amyloidosis and the current standard of care is ineffective in preventing the disease progression. The proposed therapy aims to eliminate the faulty plasma cells with a single treatment. It uses the patient’s own immune cells, which are genetically altered, to precisely target and destroy the problematic cells.
“Nexcella is thrilled to be selected as a recipient of this prestigious award, and to join California’s thriving biotechnology innovation ecosystem,” said Dr. Rachman, the principal investigator of the project. “We believe this award highlights the importance of NXC-201 as a potential treatment for relapsed/refractory AL Amyloidosis patients, who have no FDA-approved therapies today.”
“AL amyloidosis has an unmet medical need. Currently approved therapies for this rare disease are mostly not well tolerated and have not led to sustained remissions,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. “This one-time therapy would be an innovative treatment for patients with AL Amyloidosis and the preliminary data are encouraging.”
Fostering Collaboration Through Shared Labs
The CIRM Board awarded nearly $27 million to additional facilities as part of the network of Shared Resources Laboratories (SRLs) for Stem Cell-Based Modeling, bringing the total of funded SRLs to 11.
The CIRM-funded SRLs are designed to address the challenges that hinder progress in the regenerative medicine field.
The approved SRL awards include:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| INFR6.1-15363 | Stem Cell-Based Partnership Resource for Investigating Human Diseases and Training (SPRINT) | Al Alam, Denise — Lundquist Institute | $5,400,000 |
| INFR6.1-15413 | Resources for Expanding Stem cell-derived Tissues and Organs for Regenerative Engineering (RESTORE) | McCloskey, Kara E — UC Merced | $5,396,133 |
| INFR6.1-15517 | Shared Resource Laboratory for Human Stem Cell-Based Modeling (SRL-hSC) | Sprowles, Amy — Humboldt State Foundation | $4,399,888 |
| INFR6.2-15440 | Shared Resource Laboratory for Stem Cell-Based Modeling: Resources for Exploring the Biological Underpinnings of Aging and Age Associated Pathologies | Gage, Fred H — Salk Institute | $3,641,064 |
| INFR6.2-15475 | Shared Resource Laboratory for Advanced Stem Cell-Based Modeling | Sharma, Arun — Cedars-Sinai | $3,991,879 |
| INFR6.2-15457 | Shared Resources Laboratory for Stem Cell-Based Modeling in Stem Cell Biology and Engineering | Clegg, Dennis O — UCSB | $3,999,995 |
The goal of the CIRM-funded Shared Resources Laboratories (SRLs) is to foster collaboration among California researchers, break down research silos, and provide students and researchers access to top-notch resources and training using stem cell-based models, which include various cell types of the brain, cardiovascular system, and other organ systems. These stem cell-based models will help advance knowledge of human diseases to identify potential therapies, biomarkers, and drug candidates.
There are two SRLs categories: SRL6.1 which will serve California researchers, educators, and students in geographic areas where access to stem cell models is limited; and SRL6.2 which will enable leading experts in stem cell-based modeling to share their models and expertise locally and broadly across California.
A $4.4 million SRL award will support Amy Sprowles, PhD, of the Humboldt State Foundation to establish a SRL at California Polytechnic University Humboldt, the northernmost California State University located in one of California’s most rural, remote, and medically underserved geographic areas.
The proposed SRL in Humboldt will provide the facilities and equipment for regional researchers to conduct human stem cell-based modeling and create well-characterized, diverse hiPSC lines to serve different medical needs.
The Cal Poly Humboldt SRL will also provide educational workshops and research experiences for college, high school, and middle-school students; professional development opportunities for local clinical professionals in Humboldt’s medically underserved rural region; and facilitate educational outreach to communities in the region.
“We are honored by the opportunity to extend CIRM’s ability to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to our remote yet vibrant area of California through research, education, and outreach,” said Dr. Sprowles.
Another approved SRL award for $3.6 million will support Fred H. Gage, PhD of the Salk Institute.
The award will enable the Salk Institute to distribute quality-controlled stem cell resources to create numerous avenues for California researchers to make important strides toward understanding aging and neurodegeneration, potentially leading to breakthroughs in diagnostics and therapeutics.
“Over the past five years we have banked cells from a unique aging cohort and with CIRM’s generous support we will be able to distribute these quality-controlled resources to California’s stem cell and aging research communities,” said Dr. Gage. “Our goal is to enable research into a range of age-associated pathologies, including neurodegenerative diseases.”
With CIRM support, the Salk Institute will emphasize training and the standardization of cell resources and protocols to foster reproducible research, bring new Californians into the stem cell field, and ensure that the state remains at the forefront of stem cell biology.
“By investing in shared resources laboratories, we are not only providing essential infrastructure for stem cell research but also positioning California at the forefront of this transformative research. These new SRL awards showcase the remarkable progress in stem cell research and highlight CIRM’s pivotal role in propelling stem cell research forward,” said Dr. Rosa Canet-Aviles, VP of Scientific Programs and Education at CIRM. “Through these awards, CIRM will continue to drive progress, now focusing on cutting-edge disease modeling using human stem cells.”
About the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.
With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.