Stemcells Discontinues Batten Disease Program

StemCells, Inc. has announced that it has discontinued its Phase Ib clinical trial in neuronal ceroid lipofuscinosis (NCL, also referred to as Batten disease), a rare and fatal neurodegenerative disorder in children, due to lack of patient accrual.

In 2009, the Company completed a Phase I safety trial of its HuCNS-SC® human neural stem cells in six patients with advanced stages of NCL at Oregon Health & Science University (OHSU) Doernbecher Children’s Hospital.

In October 2010, the Company initiated a Phase Ib trial at OHSU to evaluate the cells in an additional six patients in earlier stages of the disease, and therefore most likely to benefit from a timely neural stem cell transplant. However, no eligible patients have been identified or enrolled despite diligent efforts by the clinical investigators over the past six months.

Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc., stated, “In clinical trials for rare diseases, timely patient enrollment is always of concern to study sponsors. Unfortunately, this concern has materialized in our Phase Ib NCL study. Out of 22 initial prospects, not one patient has met the entry criteria. This experience has also highlighted the significant challenges the Company would face in completing, within a reasonable period of time, the much larger studies in the target patient population that would ultimately be required for marketing approval. These factors, combined with our limited corporate resources, have prompted the decision to shelve our clinical development program in NCL at this time.”

Martin McGlynn, President and CEO of StemCells, Inc., commented, “We wish to thank the clinicians and staff at OHSU for their dedication and contributions to this pioneering stem cell research. We are also deeply grateful to the families of the six patients involved in our Phase I trial. No doubt, this decision will come as a great disappointment to the NCL community, but I would like to point out that their support has greatly contributed to the prospect that this groundbreaking technology will one day result in a treatment for a broad range of debilitating neurodegenerative disorders.

As a result of this clinical research, we have established that we can safety transplant meaningful doses of HuCNS-SC cells directly into the brain, and that patients can tolerate both the surgical intervention and the immunosuppression regimen. We also now have evidence that, once transplanted, the cells are capable of engrafting and surviving long-term, suggesting the possibility of durable clinical benefit. And, importantly, we have not observed any evidence of aberrant cell behavior or tumor formation in patients who are now more than three years post-transplant, and whom we continue to follow as part of a long-term observational study.”