CIRM grant will advance work on multiple sclerosis treatment
A UC Irvine immunologist will receive $4.8 million to create a new line of neural stem cells that can be used to treat multiple sclerosis.
The California Institute for Regenerative Medicine awarded the grant Thursday, May 24, to Thomas Lane of the Sue & Bill Gross Stem Cell Research Center at UCI to support early-stage translational research.
CIRM's governing board gave 21 such grants worth $69 million to 11 institutions statewide. The funded projects are considered critical to the institute's mission of translating basic stem cell discoveries into clinical cures. They are expected to either result in candidate drugs or cell therapies or make significant strides toward such treatments, which can then be developed for submission to the Food & Drug Administration for clinical trial.
Lane's grant brings total CIRM funding for UCI to $76.65 million.
"I am delighted that CIRM has chosen to support our efforts to advance a novel stem cell-based therapy for multiple sclerosis," said Peter Donovan, director of the Sue & Bill Gross Stem Cell Research Center.
MS is a disease of the central nervous system caused by inflammation and loss of myelin, a fatty tissue that insulates and protects nerve cells. Current treatments are often unable to stop the progression of neurologic disability - most likely due to irreversible nerve destruction resulting from myelin deficiencies. The limited ability of the body to repair damaged nerve tissue highlights a critically important and unmet need for MS patients.
In addressing this issue, Lane - who also directs UCI's Multiple Sclerosis Research Center - will target a stem cell treatment that will not only halt ongoing myelin loss but also encourage the growth of new myelin that can mend damaged nerves.
"Our preliminary data are very promising and suggest that this goal is possible," said Lane, a Chancellor's Fellow and professor of molecular biology & biochemistry. "Research efforts will concentrate on refining techniques for production and rigorous quality control of transplantable cells generated from high-quality human pluripotent stem cell lines, leading to the development of the most therapeutically beneficial cell type for eventual use in patients with MS."
The best estimates indicate that there are 400,000 people diagnosed with MS in the U.S., with nearly half - about 160,000 - living in California. The economic, social and medical costs associated with the disease are in the billions of dollars, placing a significant burden on the state's healthcare system.
As an MS patient and research advocate, Nan Luke sees support for Lane's work as a positive step toward a regenerative therapy. The Irvine attorney was diagnosed with MS more than 20 years ago, and current treatments have slowed its progress but cannot undo damage to critical areas of her brain.
"This new research gives me and others like me real hope that our nerve damage may be repaired and that we may regain lost function," said Luke, who serves on the Sue & Bill Gross Stem Cell Research Center's patient advocacy committee.
Lane will collaborate with Australian MS researcher Claude Bernard at Monash University in Melbourne, who will help validate the cell line's effectiveness. Australia's National Health & Medical Research Council will provide a supplemental $1.8 million as part of CIRM's new collaborative funding program.
Additionally, Jeanne Loring, director of the Center for Regenerative Medicine at The Scripps Research Institute in La Jolla, will work with Lane to develop the neural stem cells to be used in the study.