With an experimental treatment for a young child with Duchenne muscular dystrophy last week, UC Davis Medical Center inaugurated its new Alpha Stem Cell Clinic, which will serve as a launch pad for testing novel cellular therapies in a wide spectrum of medical conditions.
The HOPE-2 clinical trial for Duchenne – a devastating and fatal genetic disorder mainly affecting boys and young men -- is the first of what will be a range of leading-edge studies at UC Davis’ Sacramento campus, where the Alpha clinic will be part of a consortium of top-tier institutions in California offering FDA-approved clinical trials.
The network, funded by the state’s stem cell agency, the California Institute for Regenerative Medicine (CIRM), is focused on developing new resources and enhancing existing research expertise – such as UC Davis’ work in regenerative medicine – to address the unique challenges of taking investigational products from the laboratory bench to a patient’s bedside.
The key to the Alpha clinic success will be its ability to accelerate the delivery of therapies and treatments through partnerships with patients, medical providers and clinical trial sponsors. With the HOPE-2 trial, the Alpha team is working closely with Craig McDonald, UC Davis’ internationally recognized clinical and research expert in Duchenne, and the trial’s sponsor, Capricor, Inc., which received a grant from CIRM to fund, in part, its earlier Hope-Duchenne clinical trial.
"We have the full range of resource experts in regenerative medicine, from the cellular to the clinical trials level," said Jan Nolta, director of the university’s stem cell program and its Institute for Regenerative Cures. "We’re also excited about the prospect of being able to link with other Alpha Stem Cell Clinics around the state to help speed the process of testing and refining treatments so we can get therapies to patients in need.”
The new clinic is staffed by personnel from the UC Davis Institute for Regenerative Cures and is designed to provide a centralized space at the medical center to safely administer stem cell therapies, gene therapies and immunotherapies, as well as manage biospecimens and conduct the follow-up visits that are essential components of a successful clinical trial.
The Duchenne muscular dystrophy trial is the first of what will be a number of leading-edge clinical opportunities for patients in Northern and Central California, studies that will also include the testing and evaluation of unique and promising stem cell therapies.
McDonald, who is a professor and chair of physical medicine and rehabilitation, is the national principal investigator on the multicenter HOPE-2 trial for Duchenne. The disorder only has limited treatment options, and no cure.
“The HOPE-2 trial is testing the safety and efficacy of a cellular therapy known as CAP-1002,” said McDonald. “We’re infusing patients with allogeneic cardiosphere-derived cells, which represent a unique population of cells containing cardiac progenitor cells. Collaborating with the Alpha clinic team enables us to capitalize on their research infrastructure and expertise for clinical trials focused on cell-based therapies. It complements the skills of UC Davis’ Neuromuscular Research Unit, which is a national leader in conducting Duchenne trials. We believe this unique partnership could be a model for translating stem cell discoveries into meaningful treatments for patients with muscular dystrophy and other serious progressive neurologic diseases.”
McDonald said the CAP-1002 contains cardiac progenitor cells which are a type of adult stem cells derived from adult human cardiac muscle tissue that can only undergo differentiation along a cardiac cell lineage; meaning that unlike human embryonic, pluripotent stem cells, they cannot become any cell type. Rather than working through engraftment and growth, the primary mechanism of the CAP-1002 therapy is for the cardiosphere-derived cells to diffusely secrete extracellular vesicles, including exosomes and microvesicles, which are membrane-covered vesicles, or sacs, containing a variety of potent growth and secretory factors known to promote muscle regeneration, decrease inflammation, improve mitochondrial health, and help maintain or perhaps improve critical cardiac and skeletal muscle function.
In the fall of 2017, the California Institute for Regenerative Medicine (CIRM) awarded a nearly $8 million grant to Nolta and colleague Mehrdad Abedi, a professor of internal medicine and a specialist in bone marrow transplantation, to launch the Alpha Stem Cell Clinic program in Sacramento. The clinic has partnered with the UC Davis Clinical and Translational Science Center on studies and has its infusion site directly adjacent to the hospital. Upcoming studies currently in the planning stages include several other stem cell studies, as well as immunotherapies for patients with relapsed cancer.
Through the HOPE-2 clinical trial for the potential treatment of Duchenne muscular dystrophy, UC Davis is playing a leading role in advancing the overall science of regenerative medicine. All of the programs and facilities within UC Davis complement a focus on turning stem cells into cures. For more information, visit www.ucdmc.ucdavis.edu/stemcellresearch.