The trial, the final one before possible approval for widespread use of the therapy, is expected to include about 200 patients. The California component is being conducted at UC Irvine for the firm, BrainStorm Cell Therapeutics.
The $3 billion California stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), awarded the cash to BrainStorm last July. The agency's reviewers said of the application:
"Overall, the proposed phase 3 study is well-designed. If executed successfully, it will provide a very meaningful outcome, generating data to define the overall value of the program and the path to regulatory approval and marketing.
"The investigators have now included slow vital capacity as a clinical measure in the revised clinical trial per (earlier reviewer) recommendations .
"The current protocol will still not answer the question of whether the product is better than untreated autologous bone marrow-derived MSCs. However, reviewers felt this is not critical for the current study as a positive outcome in this trial may allow a demonstration of superiority over MSC alone in a future study."The firm has matched the California award with another $16 million. Completion date of the trial is 2019. The trial is one of two phase three trials backed by CIRM that are currently recruiting. The principal investigator at UC Irvine is Namita A. Goyal. More information on the trial can be found here.
About 5,600 people in the United States are diagnosed annual with ALS, which has severely disabled British physicist Stephen Hawking.