Researchers at UCLA receive $21.8 million in funding from CIRM for the advancement of innovative stem cell treatments
California Institute for Regenerative Medicine Advances Stem Cell Therapies for Neuropsychiatric Disorders, Alpha Thalassemia, and Angelman Syndrome
The California Institute for Regenerative Medicine (CIRM) is making significant strides in the development of stem cell-based treatments for various medical conditions, including neuropsychiatric disorders, alpha thalassemia, and Angelman syndrome. While specific projects funded by CIRM on these conditions may not be detailed in the search results, the broader context of stem cell and gene therapy advancements supported or associated with California-based research institutions provides valuable insights.
In the field of neuropsychiatric disorders, stem cell and gene therapies are being applied towards cell replacement and gene editing strategies to address neurological and psychiatric conditions. Notable work involves the use of induced pluripotent stem cells (iPSCs) derived from patients to model diseases at the cellular level and develop targeted interventions. This research is gaining attention at major regenerative medicine conferences in California, where progress in using stem cells for therapeutic screening, cell replacement, and emerging immune approaches in neurodegenerative and psychiatric diseases is highlighted [3].
Alpha thalassemia, a genetic blood disorder, can potentially be treated through hematopoietic stem cell gene therapies that correct the underlying mutations. Recent clinical advances at UCLA include gene-engineered blood-forming stem cells that continuously generate therapeutic blood cells after transplantation. These strategies employ gene-editing and gene transfer to hematopoietic stem cells, aiming to provide durable cures, as reflected by ongoing clinical trials in cancer and other blood disorders funded at California centers [1][3].
Though no direct mention of Angelman syndrome was found in the search results, efforts to develop gene therapies and stem cell-based approaches for rare genetic neurological disorders like Angelman syndrome are active in California’s regenerative medicine community. These often focus on restoring expression of impaired genes or replacing defective neural cells, aligning with the themes of cell replacement therapy and gene therapy highlighted in recent CIRM-supported events [3].
Notable among these advancements is the work of Dr. Aparna Bhaduri, assistant professor of biological chemistry at UCLA, who has received a $10.3 million foundational research award from the California Institute for Regenerative Medicine. The award is for the development of a gene therapy to treat Angelman syndrome, a rare genetic disorder affecting the nervous system [4]. Dr. Bhaduri and collaborators will use stem cell-derived 3D brain organoid models grown from samples from patients with schizophrenia and autism spectrum disorder to compare metabolism in a healthy versus disrupted environment [5].
Another significant development comes from Dr. Roger Hollis, a project scientist in Dr. Donald Kohn's laboratory, who has received a $5.8 million translational research award. Hollis's gene therapy approach involves modifying a patient's own blood stem cells using a viral vector to add a healthy copy of the mutated gene that causes Angelman syndrome [6]. The data to date, funded by TransformaTx Biotherapeutics, has demonstrated complete symptomatic correction in the commonly used adult mouse model of the disease [6]. The team will use the new CIRM grant to prepare a pre-IND package submission to the U.S. Food and Drug Administration, which is the first step toward launching a phase 1 clinical trial to evaluate the therapy in humans [7].
The need for more effective therapies that address the root biological causes of neuropsychiatric disorders is evident, as current treatments mainly manage symptoms. The team comprising UCLA, UCSF, and UCSC experts in metabolism, computational biology, and neurodevelopmental disorders aims to fill this gap [5]. This work immediately paves the way for innovative, targeted therapies that include dietary interventions and new drugs to transform care [8].
It's important to note that the treatment for Angelman syndrome, if successful, is intended to be curative or to greatly diminish the debilitating symptoms of the disorder. Angelman syndrome causes a universal lack of speech, impaired motor function and balance, and profound difficulties with sleep, requiring lifelong care [9]. With one in five people in the U.S. experiencing a neuropsychiatric disorder, many cases starting in adolescence, the potential impact of these advancements cannot be overstated [10].
In addition to the work on Angelman syndrome, Dr. Donald Kohn, distinguished professor of microbiology, immunology, and molecular genetics at UCLA, is working on gene therapy for alpha thalassemia major, a blood disorder [11]. This fact, while not directly related to the main topic of the article, is another grant-funded research project at UCLA's Broad Center of Regenerative Medicine and Stem Cell Research.
References:
- California Stem Cell Report
- California Stem Cell Report
- Regenerative Medicine Research in California
- UCLA Newsroom
- UCLA Newsroom
- TransformaTx Biotherapeutics
- UCLA Newsroom
- UCLA Newsroom
- Angelman Syndrome Foundation
- National Institute of Mental Health
- UCLA Newsroom
- In response to the advancements in the development of stem cell-based treatments by the California Institute for Regenerative Medicine (CIRM), researchers at UCLA are exploring the utilization of stem cell therapies to address medical conditions like Angelman syndrome, a neuropsychiatric disorder.
- Beyond neuropsychiatric disorders, the use of stem cell and gene therapies is being investigated for the treatment of various health-and-wellness issues, such as alpha thalassemia, a genetic blood disorder, where hematopoietic stem cell gene therapies are being employed to correct underlying mutations.
