Crissy Field was filled with vibrant colors and massive energy for the 10th annual Moving Day San Francisco event on Saturday, May 4th. Even with the rain and wind, community members showed up to demonstrate their commitment to “moving to end Parkinson’s.”
Moving Day is an annual fundraising walk that unites people living with Parkinson’s Disease, their caregivers, and loved ones to raise awareness about Parkinson’s disease and embrace the power of exercise. This celebration encourages the Parkinson’s Disease community to move, which has been proven to help manage symptoms related to the disease.
The California Institute for Regenerative Medicine (CIRM) was present at the event, engaging with attendees to share information about its commitment to continuing to fund cell and gene therapy research to find treatments for Parkinson’s Disease.
Community members of all ages stopped by the CIRM booth to learn more about us and our impact on Parkinson’s Disease. Information shared included highlights about Aspen Neuroscience and Kenai Therapeutics‘ clinical trials and work being done by BrainXell Therapeutics. We also shared information with younger attendees through coloring pages and word searches.
The event was an amazing demonstration of a community’s dedication to coming together, rain or shine, to show their support for a common cause, in this case, Parkinson’s Disease. The rain did not dampen the energy, giving us a positive boost to continue spreading the message about CIRM and what we are doing to support the Parkinson’s community.
See more photos from the Parkinson’s Moving Day event in the gallery below. To learn more about CIRM’s investments in Parkinson’s Disease research, explore this page on our website. To learn more about the Parkinson’s Moving Day event, visit their official website.
The California Institute for Regenerative Medicine (CIRM) recently awarded $1.5 million to Denise Al Alam, PhD, of the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center to support research that aims to understand lung disease in individuals with Trisomy 21, also known as Down Syndrome.
Although Trisomy 21 affects multiple organ systems, respiratory complications are a major cause of death in both children and adults with this genetic condition. Dr. Al Alam’s project will use patient-derived pluripotent stem cells from ethnically diverse backgrounds to model alveolar defects inherent to Trisomy 21.
The study is poised to generate new Trisomy 21 cell lines to study defects specific to this condition. Researchers hope to uncover the genes and pathways associated with these defects, paving the way for targeted therapeutic approaches.
In California, about 667 babies are born with Down Syndrome every year, with the highest rate among Latinx infants.
“Respiratory complications are a significant cause of mortality in both children and adults with Down Syndrome. We are thrilled to support this research that aims to deepen our understanding of lung disease in individuals with Down Syndrome. This knowledge holds immense potential to intervene early and improve outcomes for those with this condition,” added Dr. Canet-Avilés.
Understanding lung disease in individuals with Trisomy 21 is crucial to improving outcomes for those living with this genetic condition. This innovative research project has the potential to lead to targeted therapeutic approaches and improve the quality of life for those affected by Trisomy 21.
The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, is proud to announce the launch of “Championing Change for Sickle Cell in California,” a pioneering campaign aimed at increasing awareness of Sickle Cell Disease (SCD) and research aimed at improving the treatment landscape for the condition.
With more than 100,000 Americans and 20 million people worldwide affected by this debilitating condition, CIRM is committed to funding cutting-edge cell and gene therapy programs that have the potential to transform the lives of SCD patients.
Sickle cell disease is a genetic disorder that causes red blood cells to assume a sickle shape under stress, leading to clogged blood vessels, excruciating pain crises, and organ damage.
This disease disproportionately affects people of African American and Latinx heritage. It is estimated that SCD occurs among about 1 out of every 365 Black or African-American births. Latinx Californians make up 7.3 percent of sickle cell disease cases—and Latinx newborns make up 11 percent of new cases.
In collaboration with key organizations such as sickle cell patient advocacy group Axis Advocacy, the National Heart, Lung, and Blood Institute’s (NHLBI) Cure Sickle Cell Initiative, and the UCSF Sickle Cell Center of Excellence, CIRM is working to champion change and drive progress for sickle cell treatments.
Flyers and social media graphics are available for download at CIRM’s website.
Community partners and patient advocates play a crucial role in the development and success of these treatments, and CIRM recognizes their importance. Among the key calls to action of the campaign include becoming a sickle cell disease advocate, spreading awareness of clinical trial options for sickle cell, downloading a fact sheet and learning more about the NHLBI Cure Sickle Cell Initiative.
In addition to spreading awareness of this condition, the campaign highlights how CIRM is funding research into stem cell, gene therapy, and other regenerative medicine procedures for sickle cell disease. To date, CIRM has invested over $65 million towards research for sickle cell, ranging from basic discovery to clinical trials.
Evie Junior participated in a CIRM-funded clinical trial for sickle cell disease at UCLA. Photo: Jaquell Chandler
CIRM’s new campaign comes at the heels of two recently FDA-approved gene therapy treatments for sickle cell disease. Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have brought forth a new era of treatment options for people living with the condition.
CIRM introduced the “Championing Change for Sickle Cell in California” campaign at Oakland’s Black Joy Parade in February 2023. Pictured is CIRM’s Community Outreach Manager Aditi Desai.
“As the first regulatory authorization for a CRISPR-based gene-editing therapy in the world, this approval not only marks a milestone in the advancement of cell and gene therapies, but it also marks a transformative step in the treatment landscape for the millions of people impacted by sickle cell disease,” said CIRM Vice President of Therapeutics Development, Abla Creasey, PhD.
For more information on CIRM’s Sickle Cell Disease campaign, ongoing research projects, and ways to champion change, please visit cirm.ca.gov/sickle-cell-disease. To get involved in the campaign, please contact CIRM’s Community Outreach Manager Aditi Desai at adesai@cirm.ca.gov.
Approximately 22,000 women are diagnosed with ovarian cancer—the most lethal gynecologic malignancy—each year in the US. At diagnosis, more than 70 percent of patients are already late stage with abdominal metastases, leading to a dismal 34 percent 5-year survival rate.
The standard of care includes aggressive chemotherapy, which often results in toxic side effects and the development of chemoresistance, underlining the need for safer, more effective treatment options to improve clinical outcomes for these patients.
This proposed treatment uses neural stem cells to target an oncolytic virus directly to abdominal ovarian tumor sites. The virus infects and kills the tumor cells, even if they are chemo resistant, which then stimulates the patient’s immune system to recognize, and fight the cancer.
If successful, this stem cell-delivered therapy can potentially lead to a more effective, less toxic treatment for Stage III ovarian cancer patients, improving survival and quality of life.
“The funding from CIRM enables us to complete the preclinical studies, product manufacturing, and clinical trial design needed to receive FDA approval to move this novel treatment to patients within 2-3 years,” said Dr. Aboody.
CIRM has previously supported Aboody and the City of Hope research team with an award for earlier-stage translational research.
The California Institute for Regenerative Medicine (CIRM) has approved an $8 million award to Aspen Neuroscience to advance an investigational stem cell-derived dopaminergic neuron replacement therapy for Parkinson’s disease (PD).
This individualized potential therapy is being explored in a First in Human Phase 1/2a clinical trial for patients with moderate to advanced PD.
Affecting more than one million Americans, PD is a devastating neurodegenerative disorder that causes walking and motor problems, as well as impaired balance and coordination. Existing therapies alleviate symptoms but do not treat the disease, leading to a significant unmet medical need for those suffering from this chronic condition.
“We would like to thank CIRM for their support of this program to investigate an autologous cell therapy for Parkinson’s disease, which is a very personalized condition,” said Damien McDevitt, PhD, President and CEO of Aspen Neuroscience, Inc.
“This first-in-human trial is the culmination of many years of work by a remarkable team of researchers and clinicians. Our approach to provide patients with their own dopamine neuron cells has the potential to impact the entire field of neurodegenerative disorders.”
This clinical trial aims to evaluate the safety, tolerability, and preliminary efficacy of this one-time therapy that, if successful, would eliminate the need for daily medications.
“This clinical award represents a significant step forward in the treatment landscape of Parkinson’s disease by advancing individualized therapy that has the potential to restore motor function in those impacted by this devastating condition,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.
The streets of Oakland were alive with vibrant colors, infectious energy, and a powerful sense of community as the 2024 Black Joy Parade took center stage on Sunday, February 25.
Black Joy Parade-goers with colorful noisemakers walking down Franklin and 19th St.
The Black Joy Parade is a yearly celebration of the Black community and its contributions to history and culture. The event started with a parade and culminated in a celebration featuring local businesses, community-based organizations, and musical artists.
The California Institute for Regenerative Medicine (CIRM) was present at the event, engaging with attendees and spreading awareness about Sickle Cell Disease (SCD). With approximately 100,000 Americans impacted by this disease, primarily from black and brown communities, raising awareness and funding research is crucial.
At the event, CIRM’s booth drew crowds of all ages eager to learn more. With individuals stopping by to chat and collect materials and countless others exposed to CIRM’s mission through strategic placement, the impact was felt throughout the parade.
CIRM marketing and communications director, Esteban Cortez, in a discussion with a parader-goer.
But it wasn’t just about spreading awareness; it was about fostering connection and joy. Attendees embraced CIRM’s presence, participating in activities like the Blood Cell Plinko board, which became an instant hit!
CIRM medical affairs & policy project manager Emily Crotti, MPH, explains the game Blood Cell Plinko!
Photo Courtesy of Neurona Therapeutics/Curtis Myers
Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, recently announced the successful completion of a $120 million financing.
Neurona’s financial success is an encouraging sign for the stem cell and gene therapy sector, highlighting partnerships with investors and public organizations like the California Institute for Regenerative Medicine (CIRM).
CIRM recently awarded Neurona an $8 million clinical project grant to test NRTX-1001—a neural cell therapy derived from human stem cells—for drug-resistant epilepsy. CIRM has supported this research from the initial discovery research stage to the ongoing first-in-human clinical trial.
NRTX-1001 is being evaluated in an ongoing clinical trial and has potential application in Alzheimer’s disease and other disorders of the nervous system.
“This financing is a testament to the hard work and dedication of the Neurona team, commitment of our collaborators, and encouraging preliminary data from the first patients in the ongoing clinical trial of NRTX-1001 cell therapy,” said Cory R. Nicholas, PhD, Neurona’s chief executive officer and co-founder.
The Potential for Suppressing Seizures in a Single Dose
One patient in the CIRM-funded clinical trial is Annette Adkins, who has experienced a remarkable transformation in her life since receiving the single dose of the neuronal cell therapy NRTX-1001.
In the past, Annette endured frequent seizures, but thanks to this groundbreaking single-dose therapy, she is now hiking again and doing other activities she loves.
Neurona clinical trial patient Annette Adkins, after receiving the single dose of NRTX-1001. Photo Courtesy of OHSU/Christine Torres Hicks
In a recent announcement, the Neurona research team reported that Annette had undergone the procedure safely and had a greater than 90% reduction in seizure frequency.
Annette’s story underscores CIRM’s dedication to investing in meaningful research that provides real-world solutions for unmet medical needs.
Seizing Life episode, featuring Annette Adkins, who discusses the impacts of epilepsy on her professional and personal life, and how it led her to participate in the CIRM-funded Neurona clinical trial.
Updates from the Trial
Data from the first cohort of five participants in Neurona’s ongoing clinical trial were shared in December 2023.
The five participants entered the study with a history of seizure activity that was not controlled by anti-seizure medications. These subjects received a one-time administration of NRTX-1001 as well as temporary immunosuppression to promote the long-term persistence of cell therapy.
The first two subjects, who experienced 32 and 14 seizures per month, respectively, during the six-month baseline, have continued to report a reduction of more than 95% in overall seizure counts more than one year after taking the dose of NRTX-1001.
The remaining three subjects were treated with NRTX-1001 more recently and have been followed for three months after the dose. Two of the three subjects demonstrated reduced monthly seizure frequencies of 76% and 87% from baseline levels (26 and 30 seizures per month, respectively) since the first month, with elimination of their more severe focal impaired-awareness seizures.
South San Francisco, CA, Jan. 29, 2024 – The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded almost $26 million to fund various clinical research — from treating bipolar depression to advancing a therapy for spinal cord injury.
The awards will support four projects in the Agency’s clinical program which provides funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development.
In addition, CIRM approved a concept proposal for the Community Care Centers of Excellence (CCCE), a new model for increasing clinical trial access to diverse communities throughout California.
The clinical awards approved at the CIRM January Independent Citizens’ Oversight Committee (ICOC) meeting include:
Application #
Program Title
Principal Investigator/Institution
Amount
CLIN1-14840
Prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies
de Vries, David – Tr1X Inc
$4,000,000
CLIN2-15085
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Kim-McManus, Olivia – UCSD
$985,713
CLIN2-15395
A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach
Etkin, Amit – Alto Neuroscience
$15,000,000
CLIN1-15450
Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)
Tuszynski, Mark – UCSD
$6,000,000
Improving the Treatment Landscape for Bipolar Depression
A $15 million award to Alto Neuroscience and Amit Etkin, MD, PhD, President and CEO of Alto Neuroscience will advance a Phase 2b trial to test a novel therapeutic treatment for bipolar depression (I or II), a condition that impacts 4.4% of U.S. adults at some point in their lives.
The drug is designed to act on neural progenitor cells by enhancing the brain’s ability to create new neurons and improve connections in the hippocampus, a specific region of the brain implicated in bipolar depression.
Bipolar depression is a severe, life-long psychiatric condition associated with a significant burden of illness and risk of suicide.
“Given the prevalence of bipolar depression and the high unmet medical needs of those facing this challenging disorder, this potential new therapy could expand treatment options to the hundreds of thousands of Californians impacted by this chronic neuropsychiatric disease,” said Abla Creasey, PhD, Vice President of Therapeutic Development at CIRM.
Unlike current treatment options which consist of a regimen of antipsychotics and mood-stabilizing medications, this small molecule drug has the potential to offer better tolerability, and includes a diagnostic approach designed to identify patients who are most likely to benefit.
Using Stem Cells to Restore Function after Spinal Cord Injury
Another project added to CIRM’s clinical program includes a $6 million award to Mark Tuszynski, MD, PhD, from the University of California San Diego (UCSD) to explore the use of human embryonic stem cells to treat severe spinal cord injury (SCI).
The proposed therapy works by implanting neural stem cells at the injury site to regenerate and repair damaged axons associated with SCI.
SCI impacts more than half a million Americans and is associated with significant morbidity, including long-term and severe disability, chronic neuropathic pain as well as high healthcare costs.
“The potential therapy represents a unique and forward-looking approach in the treatment landscape of severe spinal cord injury and underscores CIRM’s commitment to advancing groundbreaking solutions to transform patients’ lives,” said Dr. Creasey.
Improving Access to Treatments Across California
Additionally, CIRM approved a plan for the Community Care Centers of Excellence (CCCEs). The program is projected to award up to $60 million in CCCE awards.
The California Stem Cell Research, Treatments, and Cures Initiative of 2020 (Proposition 14) mandates the establishment of the CCCE Program to promote access across the state to clinical trials, treatments, and therapies arising from CIRM-funded research.
The CCCEs are designed to serve as a hub for expanding access to clinical trials, regenerative medicine treatments, and CIRM education and training programs. The program will seek to enlist community-based partnerships to engage populations that can benefit from these treatments.
A strategic aim is to provide equitable access to communities and populations that would otherwise have more limited opportunities in the absence of these centers.
Please direct any inquiries to Koren Temple-Perry, Senior Director of Marketing & Communications at press@cirm.ca.gov.
The California Institute for Regenerative Medicine (CIRM) applauds and acknowledges the historic significance of the Food and Drug Administration (FDA) approval of Vertex and CRISPR Therapeutics’ CRISPR-based gene therapy (Casgevy) for sickle cell disease.
As the first regulatory authorization of a CRISPR-based gene editing therapy in the world, this brings forth a new era of treatment options for people living with sickle cell disease, a condition that affects more than 100,000 Americans and 20 million people worldwide.
“As the first regulatory authorization for a CRISPR-based gene-editing therapy in the world, this approval not only marks a milestone in the advancement of cell and gene therapies, but it also marks a transformative step in the treatment landscape for the millions of people impacted by sickle cell disease,” said CIRM Vice President of Therapeutics Development, Abla Creasey, PhD.
By leveraging CRISPR gene editing, this therapy has the potential to provide a more effective and potentially curative solution compared to traditional treatments. The FDA also approved another gene therapy for sickle cell disease (Lyfgenia) from bluebird bio based on long-standing lentivirus vector technology.
Through the Cure Sickle Cell Initiative, CIRM, with funding from the National Heart, Lung, and Blood Institute, remains committed to funding cell and gene therapy programs that will accelerate the development of gene therapies to treat sickle cell disease, as well as create a collaborative, patient-focused research environment.
CIRM recognizes that long-term innovation is needed to lower the costs of gene therapies, reduce the burden of the treatment on the patient, and make these therapies more accessible.
CIRM and NHLBI are co-funding two clinical trials testing different types of gene therapy approaches for patients with severe sickle cell disease. Those include a pivotal Phase 2 trial led by David A. Williams, MD of Boston Children’s Hospital and a Phase 1 trial led by Mark Walters, MD of UCSF Benioff Children’s Hospital Oakland.
In the Phase 2 clinical trial led by Williams, investigators will take a patient’s own blood stem cells and insert a novel engineered gene to silence abnormal hemoglobin and induce normal fetal hemoglobin expression. The modified blood stem cells are then reintroduced back into the patient. The goal of this therapy is the production of normal shaped red blood cells that are robust and able to transport normal amounts of oxygen, thereby reducing the severity of the disease.
“As a researcher involved in development of new therapies to treat sickle cell disease and as a clinician caring for children with this terrible disease, I am elated to see FDA approval of two different effective genetic therapies. I anxiously await access of these therapies by patients with severe disease as well as the continued development of additional options for curative therapies for our patients,” said Dr. Williams, who has consulted for Vertex Pharmaceuticals and bluebird bio.
In the Phase 1 trial led by Walters, patients will be transplanted with their own blood stem cells that have been edited with CRISPR-Cas9 to correct the disease-causing mutation in the beta globin gene. This trial will begin enrolling patients in California in 2024.
Please direct any inquiries to Koren Temple-Perry, Senior Director of Marketing & Communications at press@cirm.ca.gov.
John A. Zaia, MD, of Beckman Research Institute of City of Hope
This year marks the 35th commemoration of World AIDS Day, a global event that aims to end human immunodeficiency virus (HIV) related stigma, honor people lost to the disease, and reaffirm the commitment to ensuring HIV is no longer a public health threat.
This year, the theme of the global event is World AIDS Day 35: Remember and Commit.
HIV is a virus that attacks the body’s immune system. If HIV is not treated, it can lead to acquired immunodeficiency syndrome (AIDS). There is currently no effective cure, though much progress has been made to reduce HIV transmissions and make the virus undetectable in people.
Globally, an estimated 38 million people live with HIV. More than 35 million people have died of HIV or AIDS-related illnesses over the past 40 years, making it one of the most devastating pandemics in history.
In California, there are more than 141,000 people living with HIV as of 2021. That same year, there were 4,444 new HIV diagnoses.
While the rate of new diagnoses is on the decline in the state, the number of people living with HIV continues to grow. It is estimated that Californians living with HIV will face an average lifetime cost of $510,000 to treat their infection.
Investing in HIV Research and Therapies at CIRM
CIRM has invested more than $104 million in the search for stem cell and gene therapy treatments for HIV, ranging from discovery research to clinical trials.
Recent CIRM investments includea study at UC Davis health, in which researchers take a patient’s own white blood cells, also called T cells, and modify them so they can identify and target HIV cells to control the virus without medication.
CIRM also funded a clinical trial at UCSF to develop a treatment for HIV. In the study, the UCSF team lead by Steven Deeks, MD will modify a patient’s own immune cells to treat HIV.
The goal of this one-time therapy is to act as a long-term control of HIV so patients no longer need to take antiretroviral therapy (ART), which involves taking a combination of HIV medicines every day.
Recent HIV Research Awards
Other recent CIRM awards include an $11.3 million grant to John A. Zaia, MD, of Beckman Research Institute of City of Hope for a clinical trial that will enroll up to 12 healthy people with HIV to be treated in a first-in-human study.
Zaia’s City of Hope team, in partnership with the University of San Diego, will test a novel approach developed by City of Hope scientists in which a patient’s T cells are engineered to target both the HIV antigen gp120 and a common virus that infects nearly all persons living with HIV called cytomegalovirus.
The goal is for these CAR-T cells to persist and eliminate HIV-infected cells so that antiviral drugs are no longer needed.
World AIDS Day Events
CIRM will be co-sponsoring the upcoming World AIDS Day event in Palm Springs, hosted by HIV+ Aging Research Project and RID HIV. The event will be held at the Camelot Theater on December 1st, with a reception to follow.
To learn more about CIRM-funded research to find treatments for HIV, visit this web page.
The Stem Cellar 