StemCells, Inc.’s Phase I BATTEN Trial Data Featured at AANS Annual Meeting
News May 05, 2010
StemCells, Inc. has announced that Nathan Selden, M.D., Ph.D., F.A.C.S., F.A.A.P., will give a feature presentation today at the American Association of Neurological Surgeons (AANS) 2010 Annual Meeting.
Dr. Selden was co-principal investigator of the Company’s Phase I clinical trial in neuronal ceroid lipofuscinosis (NCL, also often referred to as Batten disease), a fatal neurodegenerative disorder in children. In his presentation, Dr. Selden will summarize the positive safety data from the trial and will note that additional data from the ongoing long-term follow-up study continue to affirm the safety profile of the Company’s HuCNS-SC® cells.
“This Phase I trial was a very important first step toward finding a viable treatment and extending the life of children with this devastating disease,” remarked Dr. Selden. “Initial results regarding safety associated with the transplantation of a significant cell dose are certainly promising. Further investigation of HuCNS-SC cells is warranted for infantile and late-infantile NCL, as well as for exploring this cell therapy approach for other conditions of the central nervous system.”
Dr. Selden is Campagna Professor of Pediatric Neurological Surgery and head of the Division of Pediatric Neurological Surgery at Oregon Health & Science University (OHSU) Doernbecher Children’s Hospital and OHSU School of Medicine.
The Phase I NCL trial was the first ever FDA-authorized clinical trial to use purified human neural stem cells as a potential therapeutic agent, and was designed primarily to evaluate the safety of the Company’s HuCNS-SC product candidate. The trial was completed in January 2009, and the results were submitted to the FDA in September 2009.
The trial data demonstrated that the HuCNS-SC cells, the transplantation procedure, and the immunosuppression regimen were all well tolerated, and that the patients’ medical, neurological and neuropsychological conditions, following transplantation, appeared consistent with the normal course of the disease. A total of six patients in advanced stages of either infantile or late infantile NCL were enrolled and treated in the trial. One patient succumbed to the disease approximately 11 months post-transplant.
The five patients who completed the 12-month evaluation period prescribed in the trial protocol were subsequently enrolled in a separate four-year observational study. Thus far, data from this ongoing follow-up study show no evidence of serious adverse reactions directly associated with the HuCNS-SC cells or other safety concerns associated with the stem cell treatment. All five patients survived at least two years after being transplanted with HuCNS-SC cells, and magnetic resonance imaging (MRI) at the two-year post-transplant point reveal no signs of cyst or tumor formation. Two patients passed away at approximately 2.5 years and 3.4 years post-transplant, respectively, and these deaths are also believed to be due to progression of the underlying disease. The three surviving patients are now approximately 2.3 years, 2.7 years, and 3.0 years post-transplant, respectively.
“All of the families and patients involved with this clinical research should be recognized as true medical pioneers,” commented Stephen Huhn, MD, FACS, FAAP, vice president and head of the CNS program at StemCells, Inc. “Their courageous decision to participate in this groundbreaking trial has enabled us to advance the search for an effective treatment for NCL and other neurological disorders, and we owe them all an enormous debt of gratitude.”
Dr. Huhn added, “Given the very sick and fragile nature of the patients involved in this trial, we believe these safety observations are especially meaningful. The data we have gathered to date encourage us to continue our clinical development, and we are currently preparing for a second NCL trial.”
StemCells recently submitted a protocol to the FDA for initiation of a second clinical trial of its HuCNS-SC cells in NCL. The proposed new trial is designed to further assess the safety of HuCNS-SC cells in NCL, while also examining the ability of the cells to affect the progression of the disease. Because intervention prior to the final stages of the disease will likely be key to demonstrating a therapeutic benefit, the Company plans to enroll patients in its second trial who have less neuronal degeneration and brain atrophy.