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 But two small companies, Athersys Inc and Mesoblast Ltd, 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.
"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.
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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.
"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)
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