Stem Cell

Tiny stem cell companies close in on goals

The early hope that stem cell therapy would make the paralysed walk, the blind see and cure diabetes have given way to a long list of failures, highlighted by early stem cell champion Geron Corp abandoning the field in 2011.

But two small companies, Mesoblast Ltd in Australia and Athersys Inc in the US, are beginning final stage trials in hundreds of patients that they – along with loyal investors – say could change the course of devastating heart failure and stroke.

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.

Australia’s Mesoblast, with a market value of about $US500 million ($A652.86 million), is attempting to alter advanced heart failure, a leading cause of hospitalisations and deaths and an enormous cost burden.

Cleveland-based Athersys, with a market value of about $US200 million, 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.

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.

Perception of the field has also been hurt by unscrupulous actors selling unapproved, possibly unsterile cell therapies for everything from cancer to lung disease and ALS, prompting a US. crackdown.

“The market tends to test these companies sometimes to the brink,” said Tom Dobell, who manages a fund for UK-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 four per cent.

Athersys’s experimental Multistem treatment was one of the first companies to be designated by the US Food and Drug Administration as a promising cell-based therapy with potential to address unmet needs for serious or life-threatening…