Year 1

Alzheimer’s disease (AD) is an incurable disorder that affects memory, social interaction, and the ability to perform everyday activities. The number of AD patients older than 65 has surpassed 5 million in the US and 600,000 in California, numbers that may triple by 2050. Annual health care costs related to AD have been estimated to exceed $172 billion in the US, even without reflecting either the loss of income or the physical and emotional stress experienced by caregivers. Efforts to discover novel and effective treatments for AD are ongoing, but unfortunately, the number of active clinical studies is low and many traditional approaches have failed in clinical testing. There is a great need for new therapies that will both improve symptoms and alleviate suffering.

AD is characterized by the dysfunction and eventual loss of neurons, the specialized cells that convey information in the brain. Death or dysfunction of neurons results in the characteristic memory loss, confusion, and inability to solve new problems that AD patients experience.

StemCells Inc. is embarking on an initiative to evaluate the use of its proprietary human neural stem cells to treat AD. We believe that neural stem cells transplanted into a patient’s brain may protect neurons and preserve their function. This represents an entirely new approach to standard therapeutic drug development for AD, which has so far resulted in drugs that only temporarily alleviate symptoms in some patients but that do not slow or change the course of the disease. We envision using neural stem cells as a one-time intervention that will improve memory and cognitive function in AD patients. Even a modest improvement in these symptoms could significantly alter the quality of life of a patient with AD.

StemCells Inc. received a Disease Team Planning (DTP) award from CIRM to establish a Disease Team for AD, and to begin organizing the activities required to submit a Disease Team Therapy Development (DTTD) award. We are reporting now on the successful completion of this DTP award. The main deliverables were (i) submission of a DTTD award application and (ii) development of a four year research plan that contemplates an Investigational New Drug (IND) submission to the FDA for the clinical study of neural stem cells in patients with AD, within four years.

To begin evaluating its proprietary human neural stem cells as a potential therapy for AD, StemCells Inc. and its collaborators from UC Irvine needed to first design IND-enabling safety and efficacy studies to test these stem cells in animal models relevant for AD. The DTP funding from CIRM helped support a series of telephone, email and face-to-face meetings over the last 6 months, between investigators at UCI and StemCells Inc., to present and evaluate existing data on neural stem cells and to share information about AD in order to design pilot and definitive efficacy and safety studies. During this time, the team also discussed the logistical details required to conduct these studies.

After a draft research plan had been outlined, StemCells Inc. and its principal collaborator at UCI, Dr. Frank LaFerla, enlisted the help of various experts in the field of AD, including both clinicians and academic scientists, to evaluate this plan. These experts attended a meeting at UCI and provided input into the experimental design of efficacy and safety studies. Many of these experts were also recruited by StemCells Inc. to participate in preclinical and clinical working groups hosted by the Company. These working groups will ultimately evaluate the preclinical experimental results and help design the protocol for the proposed clinical trial.

The DTP award also allowed StemCells Inc. to establish a “Project Team” consisting of highly trained and skilled personnel at UCI, StemCells Inc., and an established Contract Research Organization. This Project Team will be responsible for the production and supply of the human neural stem cells, the execution of all efficacy and safety studies, and the preparation and submission of IND documents to the FDA within the next 4 years.

Finally, the DTP award allowed StemCells Inc. to timely develop and submit its DTTD application to CIRM, in which the Company requested funding in the amount of up to $20 million to facilitate execution of IND-enabling safety and efficacy studies for its proposed breakthrough neural stem cell treatment for AD.