Durham, N.C.-based Aldagen and UC Davis Health System will collaborate to explore the activity of Aldagen’s unique adult stem cell population in a preclinical model of ischemic stroke.
Adult stem cells hold promise in the treatment of this condition and other neurological disorders, including Parkinson’s disease, amyotrophic lateral sclerosis, traumatic brain injury and cerebral palsy.
According to the American Stroke Association, nearly 800,000 people a year suffer a new or recurrent stroke.
It is the third leading cause of death and the leading cause of disability in adults.
This year, Americans will pay about $65.5 billion for stroke-related medical costs and disability.
Current treatment for an ischemic stroke calls for the immediate use of a clot-busting drug, which must be administered within three hours of the event.
Stem cells as therapeutics may represent a novel approach to the treatment of ischemic stroke.
Aldagen’s technology isolates specific populations of adult stem cells that express high levels of aldehyde dehydrogenase (ALDH-bright cells).
Preclinical studies conducted by leading research institutions and academic centers have shown that ALDH-bright cells from bone marrow and cord blood may be able to repair neural tissue, which hold promise for the treatment of damaged tissue in the brain from ischemic stroke.
In ischemic stroke, for example, both blood vessels and neural tissue in the brain need to be restored.
ALDH-bright cells can induce formation of new vessels in animal models, and can restore functional nerves and ameliorate symptoms in mouse models for inherited lethal human nervous system diseases.
Aldagen has two clinical trials currently under way in patients with chronic vascular disease to test the effectiveness of revascularization of blood vessels in the leg and the heart using ALDH-bright cells from a patient’s bone marrow.
Aldagen will be collaborating with Martha O’Donnell, a professor of physiology and membrane biology, and with Jan Nolta, director of the new UC Davis Institute for Regenerative Cures, a facility supported by the California Institute for Regenerative Medicine.
“Aldagen’s technologies for stem cell isolation offer an approach for developing therapies that may be successful in clinical practice,” said Nolta, who recently participated in groundbreaking ceremonies for UC Davis’ $100 million regenerative cures facility in Sacramento. “The promise and potential of our stem cell work is greatly enhanced by this type of collaborative partnership with private industry.”
UC Davis could begin participating in clinical trials using adult stem cells by the end of 2009.
Nolta said she looks forward to testing Aldagen’s stem cell populations and hopes the research will lead to a novel cell therapy for clinical testing.
“We’re planning to have some of our key laboratories in the stem cell institute’s new building up and running by late next year,” Nolta said. “Those facilities will play a key role in enabling us to advance cellular therapies for treatments of numerous life-threatening diseases.”
Orphan Drug Designation For ALD-101
In related news, the company announced that it had received orphan drug designation from the FDA for the use of ALD-101 to improve patient outcomes by decreasing time to platelet and neutrophil engraftment in patients with inherited metabolic disorders undergoing umbilical cord blood transplantation.
Aldagen is conducting a Phase 3 trial infusing ALD-101 in patients with inherited metabolic disorders undergoing umbilical cord blood transplantation.
Cord blood transplantation is commonly used to treat children with inherited metabolic diseases, including Krabbe syndrome, metachromatic leukodystrophy, Hurler syndrome and adrenoleukodystrophy.
These diseases are progressive, degenerative and often fatal.
In many cases, the only treatment available to these patients is a transplant of blood-forming stem cells found in cord blood.
Umbilical cord stem cells are located in the umbilical cord of a newborn at birth.
The FDA orphan drug designation, administered by the Office of Orphan Products Development, provides potential incentives such as funding for clinical studies, study design assistance, waiver of FDA user fees, tax credits and, importantly, up to seven years of market exclusivity upon marketing approval.
“FDA orphan drug designation for ALD-101 is an important asset in Aldagen’s development of ALD-101,” said CEO Tom Amick. “We are encouraged by the reductions in engraftment times seen in the Phase 1 ALD-101 trial and look forward to continuing our on-going Phase 3 trial of ALD-101.”
ALD-101 is the population of stem cells that Aldagen produces from a portion of an umbilical cord blood unit using our proprietary stem cell isolation technology.
The product is infused into the patient shortly after the transplant of the remaining portion of the cord blood unit.
In a 24 patient Phase 1 clinical trial, Aldagen observed a reduction in the time to platelet and neutrophil engraftment in patients receiving ALD-101 following their cord blood transplant, as compared to similar patients who had received a cord blood transplant without ALD-101 in an earlier independent clinical trial.
In each case, these reductions were statistically significant, with p-values of 0.05 or less.
The pivotal Phase 3 clinical trial of ALD-101 is designed to further evaluate its ability to accelerate engraftment following cord blood transplants in pediatric patients with inherited metabolic diseases.
Aldagen is a biopharmaceutical company developing proprietary regenerative cell therapies that target significant unmet medical needs.
The company has four product candidates in clinical trials. Aldagen’s most advanced product candidate, ALD-101, is currently in a pivotal Phase 3 clinical trial to evaluate its efficacy in improving umbilical cord blood transplants used to treat inherited metabolic diseases in pediatric patients.
The company also is conducting or supporting Phase 1 or Phase 1/2 clinical trials of three other product candidates: ALD 151 to improve umbilical cord blood transplants used in the treatment of leukemia, ALD-301 to treat critical limb ischemia, and ALD-201 to treat ischemic heart failure.
Aldagen’s product candidates consist of specific populations of adult stem cells that the company isolates using its proprietary technology.