Clinical Development of Gene Modified Mesenchymal Stromal Cells for Stroke Disability

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

Stroke leaves hundreds of thousands of Americans disabled every year. Beyond drugs that dissolve the blood clots causing the stroke, there is no effective therapy. These "clot-busting" drugs can only be used within the first few hours of the onset of symptoms, leaving many patients without treatment options.

New advances in regenerative medicine offer hope to those with otherwise untreatable stroke disabilities. Several cell therapy products have advanced to human clinical trials. Effective therapies may be only a few years away.

One of these new therapies uses a cell type obtained from adult bone marrow, called mesenchymal stromal cells. These cells are obtained from healthy donors, grown in the laboratory and modified with a gene that improves their ability to promote regeneration of the patient's own cells.

These cells have advanced to the initial stage of human testing. A small number of stroke patients are being implanted with cells to determine if the therapy is safe. They will also be monitored for improvement in their disabilities. If this trial is successful the product will move to the next phase of human testing, determining the appropriate dose and timing needed for the product to be effective and determining which patients are most likely to benefit. This next phase will require the assembly of a multidiciplinary team of scientists and physicians to move forward. The formation of the team and the planning required to carry out this next human clinical trial is the goal of this proposal.

This 6-month project will involve the planning of the details of the clinical trial, the recruitment of physicians and scientists to the team and the identification of university hospitals where the trial will take place. Also, cells must be prepared under strict guidelines to be used in the trial. Finally, plans for additional research on how exactly these cells promote the brain's regeneration after stroke injury are required in order to better select which patients will benefit from therapy and to more effectively test and predict the cellular product's effects.

After the successful conclusion of this planning stage, the next phase of human testing can begin. Armed with the information obtained from this clinical trial, it is hoped that a conclusive clinical trial showing safety and efficacy will move forward, ultimately resulting in FDA approval and the availability of this regenerative medicine product to the many patients who suffer a stroke.

Statement of Benefit to California: 

Like the rest of the developed world, stroke is the number one cause of permanent disability in the state of California. Regenerative therapies that promote recovery from stroke disability would reduced the costs of long-term medical and supportive care, increase independence and improve productivity for thousands of Californians. Stroke costs the state over $6 billion per year.

This project continues the development of a regenerative cell therapy product currently in the earliest stages of human clinical testing. These cells are derived from the bone marrow of healthy donors and have proven very effective at treating stroke disability in animal models.

The clinical trial described in this proposal will take place at a number of university hospitals. Many of these will be in the State of California, providing direct health benefits to patients living nearby and resources to the pioneering scientists and clinicians devoting time to this project.

This work will continue the trend establishing California as the wolrd's leading center for regenerative medicine. Importantly, this project has already advanced to human clinical testing, overcoming challenges in manufacturing and regulatory hurdles not yet addressed by earlier stage products.