Month: February 2024

CIRM awards more than $56 million for clinical research aimed at treating Parkinson’s disease, autoimmune conditions and a variety of cancers

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded $56 million to fund various clinical research aimed at treating Parkinson’s disease, autoimmune conditions and a variety of cancers.

The awards will support seven projects—five clinical trials and two preclinical 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.

The approved clinical awards include:

Application # Program Title Principal Investigator/InstitutionAmount 
CLIN1-14845Neural stem cell delivered oncolytic viro-immunotherapy for ovarian cancerAboody, Karen – City of Hope$5,314,547
CLIN1-15337Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and Adolescents and Young Adults (AYA) with Recurrent Solid TumorsAsgharzadeh, Shahab – Children’s Hospital Los Angeles$6,000,000
CLIN2-14801Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid TumorsKalbasi, Anusha – Stanford$10,211,085
CLIN2-15547Phase 1/2a Dose Escalation Study of Autologous Neuron Replacement in Sporadic Parkinson’s Disease (PD)Wirth, Edward – Aspen Neuroscience$8,000,000
CLIN2-15562Phase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ GlioblastomaOkada, Hideho – UCSF$10,927,618
CLIN2-16063A phase 1/2 study to evaluate a bi-specific CD19/CD20-directed CAR T-cell, in refractory lupus nephritis (LN) and systemic lupus erythematosus (SLE)Benjamin, Jonathan – ImmPACT-Bio, Inc.$8,000,000
CLIN2-16303A Phase 1 Study in Participants with Moderate to Severe Active Systemic Lupus ErythematosusHickingbottom, Barbara – Fate Therapeutics$7,934,448

Included in the awards is 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.

Another award is a $7.9 million award to Barbara Hickingbottom, MD, of Fate Therapeutics to advance clinical research for FT819, an induced pluripotent stem cell (iPSC)-derived CD19 CAR T-cell therapy for Systemic Lupus Erythematosus (SLE). SLE is a debilitating autoimmune disease and affects more than 200,000 Americans, particularly women of color.

Another project added to CIRM’s clinical program includes a $5.3 million award to Karen Aboody, MD of City of Hope for late-stage preclinical research to develop a neural stem cell mediated treatment for a chemo-resistant, metastatic ovarian cancer. CIRM has previously supported Aboody and the City of Hope research team with an award for earlier-stage translational research.

To learn more about CIRM-funded clinical trials, click here. To explore more CIRM awards, visit our grants database.

Neurona Therapeutics Raises $120M Financing, Cell Therapy Shows Positive Outcomes in CIRM-Funded Trial

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
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.

To read the full release and more updates from the trials, visit the Neurona website.