NEW YORK • Two small companies are beginning final stage stem cell trials in hundreds of patients that they - along with loyal investors - say could change the course of devastating stroke and heart failure.
Both have overcome major hurdles to manufacturing stem cell treatments on a large scale that are off-the-shelf products derived from healthy donor bone marrow and do not face immune system rejection issues.
Cleveland-based Athersys, with a market value of about US$200 million (S$271 million), has demonstrated evidence in a midstage trial that its therapy may be able to expand the emergency treatment window for major strokes to up to 36 hours, compared with about four hours with current drugs, potentially allowing many more patients to avoid crippling disabilities.
Australia's Mesoblast, with a market value of about US$500 million, is attempting to alter advanced heart failure, a leading cause of hospitalisations and deaths and an enormous cost burden. They are among the farthest along in the stem cell industry at a time when Wall Street investors have focused on potentially big payoffs from immune system-based cancer therapies and rare disease treatments, after the early hope that stem cell therapy would make the paralysed walk, the blind see and cure diabetes gave way to a long list of failures.
"The market tends to test these companies sometimes to the brink," said Mr Tom Dobell, who manages a fund for Britain-based M&G Investment Management that specialises in supporting promising companies during difficult periods. "We're comfortable that the progress that's going on is going to be worth it."
M&G is a long-time holder of Mesoblast, with about a 15 per cent stake, and Athersys, with about 4 per cent.
Athersys' experimental MultiStem treatment was one of the first to be designated by the US Food and Drug Administration (FDA) as a promising cell-based therapy with potential to address unmet needs for serious or life-threatening conditions, potentially easing the approval process.
Athersys believes MultiStem - 1.2 billion cells delivered via simple intravenous drip - dampens the immune system's hyper-inflammatory reaction to signals that the brain is under attack during stroke, and promotes healing.
Mesoblast's MPC-150-IM therapy aims to mitigate advanced heart failure, a progressive condition in which the heart cannot pump enough blood to efficiently serve the body.
The company believes the therapy reduces inflammation, increases blood flow and spurs blood vessel formation that helps repair the heart muscle. A single dose consists of 150 million highly purified stem cells delivered directly to the heart's left ventricle by injection or catheter.
If such patients can recover enough to again rely on their own heart, said Dr Joseph Woo, a heart surgeon at Stanford University, "this would be considered one of the big advances of this century".