Stem cell-based strategies for treatment of Alzheimer's and other neurodegenerative disease

Funding Type: 
Disease Team Planning
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Alzheimer’s disease (AD) is a progressive brain disorder that gradually destroys a person’s memory and ability to communicate. AD is the most common form of dementia, currently affecting more than 5 million people in the United States. There is no cure for AD, and even the most effective drug treatments only temporarily delay the relentless progress of the disease. Ironically, since AD is a disease of aging, as therapies for other diseases improve and life spans increase, the number of people with AD is increasing. If this trend continues, in the next 50 years 16 million Americans will be afflicted with AD.

Stem cell therapies have been proposed for treatment of AD, but most scientists agree that cell replacement is unlikely to work because the damage is too widespread throughout the brain and involves too many different types of cells. We propose a stem cell therapy approach that doesn’t require replacement of cells, but instead uses stem cells in two novel ways. First, we plan to use stem cell transplantation in a promising new method to deliver substances that protect neurons and help them to regenerate. Second, we will use nerve cells developed from human embryonic stem cells to screen drugs that are currently approved or in development for the treatment of AD.

The unprecedented team approach that we propose starts with a core group of five scientists: a clinician, a pharmaceutical researcher, an expert in creating transgenic mice for testing drugs, a basic researcher studying the molecules involved in AD pathology, and a human embryonic stem cell expert. All of the scientists are leaders in their respective fields and were chosen because they are all excellent collaborators and are excited about the opportunity to learn from the others. After a series of planning meetings among the core team members, a draft plan will be offered for discussion at an off-site meeting for the core team and additional experts, twelve people in all. Finally, the team will devise the best path to successful therapy that they can, using their combined expertise, and will draft a strategic plan to go forward with a formal team effort.

Statement of Benefit to California: 

There are now nearly half a million people in California living with Alzheimer’s disease (AD). AD is the most common form of dementia, currently affecting more than 5 million in the U.S; a new case is diagnosed in the US every 72 seconds (Alzheimer’s Association.) Nearly half of all people over the age of 85 suffer from AD, and it affects one in eight of those over the age of 65. Ironically, deaths from AD have risen more than 30% in the last 10 years, a decade that saw decreases in deaths from heart disease, cancer, and stroke.

As life spans increase, the number of people with AD is increasing; in 2050 years 16 million Americans will be afflicted, The loss of productivity by the family members who care for AD victims costs California businesses $3 billion a year. More than a million people provide unpaid care to AD patients in California each year-as many as in Florida and New York combined. Their unpaid time has been valued at more than $8.5 billion. Nearly half of the nursing home residents in California, more than 100,000 people, have AD, costing the state and federal governments another $10 billion in Medicare and Medicaid each year.

AD is an incurable disease, and there are no therapies that can do more than temporarily reduce the relentless progression of nerve cell death and loss of memory. A therapy that could halt the disease would have immeasurable value; even if it is possible only to delay a person’s decline by a year or two, the cost savings would be billions of dollars, and to a family of an AD victim, every extra year would be priceless.