Stem Cell Therapy for the Lysosomal Storage Diseases, the Major Cause of Neurodegeneration in Children

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

Children with lethal inherited degenerative diseases of the brain, the lysosomal storage diseases or LSDs, should be among the first to benefit from novel approaches based on stem cell therapy (SCT). This belief is based on a number of medical and experimental observations including:

1) These diseases cause profound mental retardation or lead to death in most affected children; 2) SCT has already been shown to work in the milder forms of LSD that do not affect the brain; 3) The safety of neurotherapy for stem cell transplantation is already being established in the LSDs; 4) Many of the pertinent regulatory hurdles have already been overcome; and 5) Experimental work has clearly shown that SCT can be used to treat the brain in the severe forms of LSD.

This combination of medical and experimental data strongly suggest that the LSDs are uniquely poised to benefit from novel SCT and that this therapy is likely to be successful, a prediction that can less reliably be made for SCT applied to other brain and spinal cord diseases and injuries. Additionally, results of clinical trials of novel stem cell neurotherapies for the LSDs will be directly pertinent to proposed stem cell neurotherapies for some other neurological diseases, including Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, and stroke, as specific targets for these and other brain and spinal cord diseases and injuries are better identified.

Efforts aimed at moving this new SCT into clinical practice, however, will be complex and involve basic, translational, and clinical research as well as the collaboration of multiple clinical centers. As a result, we have already recruited 20 superbly qualified clinicians and translational research scientists to work as a team in moving stem cells into clinical practice to better treat children with LSDs.

Statement of Benefit to California: 

Most patients with severe diseases or injuries that might be treatable with stem cells have multiple medical problems and must be seen by multiple specialty physicians. In addition, the translation of experimental stem cell therapies to clinical practice requires the participation of basic and clinical research scientists from a variety of academic backgrounds. Thus, the Disease Team approach to these problems, supported by the California Institute for Regenerative Medicine (CIRM), is an entirely appropriate way to move stem cells from the laboratory to the patient.

Our team is focusing on a class of childhood brain diseases that causes the child’s brain to degenerate and results in severe mental retardation or death. These diseases also affect other organs of the body. Many of these organs can already be successfully treated with stem cell therapy. Our team proposes to take these lessons of success and apply them to treating the brain as well. Because of this established stem cell success, this new stem cell therapy, we propose, has a high probability of success. This will not only provide a potential cure for the children that are treated with this new stem cell therapy, but will also benefit California by 1) reducing the State’s burden for caring for these children and 2) providing a successful model of stem cell therapy of the brain that will both bolster public confidence in CIRM’s mission to move stem cell therapies into the clinic, and lay the groundwork for using this type of therapy with other brain diseases such as Alzheimer’s disease, Parkinson’s disease, stroke, and multiple sclerosis.