Tiny stem cell companies close in on major heart disease goals
18/12/2017 06:00
By Bill Berkrot
NEW YORK, Dec 18 (Reuters) - The early hope that stem cell
therapy would make the paralyzed walk, the blind see and cure
diabetes have given way to a long list of failures, highlighted
by early stem cell champion Geron Corp GERN.O abandoning the
field in 2011.
But two small companies, Athersys Inc ATHX.O and Mesoblast
Ltd MSB.AX , are beginning final stage 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 $200
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.
Australia's Mesoblast, with a market value of about $500
million, is attempting to alter advanced heart failure, a
leading cause of hospitalizations 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.
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 U.S.
crackdown this past summer. urn:newsml:reuters.com:*:nL4N1LE4Q7
"The market tends to test these companies sometimes to the
brink," said Tom Dobell, who manages a fund for U.K.-based M&G
Investment Management that specializes 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
percent stake, and Athersys, with about 4 percent.
Athersys's experimental Multistem treatment was one of the
first companies to be designated by the U.S. Food and Drug
Administration as a promising cell-based therapy with potential
to address unmet needs for serious or life threatening
conditions, potentially easing the approval process.
FDA Commissioner Scott Gottlieb said in a recent interview
the agency is seeking an approval pathway for legitimate stem
cell therapies "that's not overly burdensome."
"The regenerative medicine space has been a tough road
clinically and Athersys is one of the bright, shining stars,"
said Steven Martin, managing member of Chicago-based Aspire
Capital and a long-time holder of Athersys shares.
REDUCING THE RISKS
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.
The body's reaction to stroke may also severely deplete the
immune system, leading to later complications beyond paralysis
or speech impairment. In the Phase II trial, Multistem patients
had significantly fewer incidents of pneumonia, acute pulmonary
distress and other serious complications.
That trial, attempting to extend the treatment window to 48
hours, failed. But researchers were encouraged by patients
treated up to 36 hours post stroke.
"It's reason to be cautiously optimistic," said Dr. Ken
Uchino, a leading Multistem investigator from Cleveland Clinic.
The company plans to begin a 300-patient Phase III study of
Multistem in early 2018, aiming for U.S. and European approval
after regulators agreed to protocols for one pivotal trial.
"This reinforces and cements an efficient path forward for
us," Athersys Chief Executive Gil Van Bokkelen said.
A separate 220-patient Multistem trial in Japan for that
market began enrolling patients through partner Healios KK
4593.T .
"Of the many entities in this field, they have been careful
and deliberate in their movement forward" and do not engage in
"overwrought claims," said Dr. S. Thomas Carmichael, co-director
of UCLA Broad Stem Cell Center, who was not involved in
Multistem trials.
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.
An ongoing Phase III trial of 600 advanced heart failure
patients is currently enrolling. Reducing the severity of a
patient's heart failure "would give them back potentially many
years of good quality life," said Mesoblast CEO Silviu Itescu.
Independent monitors who oversee and can halt blinded
clinical trials for futility gave a green light to continue the
Mesoblast study in April after a review of the first 270 treated
patients, suggesting the therapy might be effective.
A separate U.S. National Institutes of Health-sponsored
Phase II study of 159 end-stage heart failure patients dependent
on an implanted pumping assist device completed enrollment in
September, with results expected in the first quarter of 2018.
Some 250,000 people in the United States alone have been
diagnosed with most serious class IV heart failure, and only
about 9,000 receive a transplant or left ventricular assist
device (LVAD) each year, Itescu said.
If such patients can recover enough to again rely on their
own heart, said Dr. Joseph Woo, a heart surgeon at Stanford
University and an investigator of the NIH trial, "this would be
considered one of the big advances of this century."
(Editing by Michele Gershberg and Edward Tobin)
((bill.berkrot@thomsonreuters.com; 1 646 223 6030; Reuters
Messaging: bill.berkrot.thomsonreuters.com@reuters.net))
Keywords: HEALTH STEMCELLS/