STORY HIGHLIGHTS
- Approved stem cell
therapies for cardiac patients
could be available in five
years
- Researchers are finding
that methods that worked in
animal studies are effective in
humans
- Mesenchymal stem cells have
a natural homing ability toward
heart injury site
- Further research is needed
to determine how many cells are
optimal in each infusion
(CNN) -- If you've just had
your first heart attack, doctors may one
day be able to reverse the damage done with
stem cell therapy.
An intravenous method of injecting stem
cells into patients who had experienced
heart attacks within the
previous 10 days suggested that this method
works to repair -- not just
manage -- heart damage, a
recent study found.
The study is a step forward in a field
in which a lot of approaches have been
tried in animals and preliminary human
trials, but none has been approved for
widespread clinical use for heart
patients.
The new results are a milestone in
stem cell research, and for
patients, said Jeffrey Karp, a researcher
at Brigham and Women's Hospital in Boston,
Massachusetts, who runs a stem cell biology
lab at Harvard University. He was not
involved in the study.
Most current clinical approaches are
focused on managing problems, not
addressing the root of the damage, he
said.
"Many patients who have a heart attack
will go on to suffer heart failure," he
said. "It's imperative to try and fix the
root of the problem as quickly as
possible."
We're looking on the time
frame here of five years,
in the best-case scenario,
to have approved cardiac
stem cell therapies.
--Dr. Joshua
Hare
The research, published in the
Journal of the American College
of Cardiology, were part of a
phase I study that set out to
show safety. The trial has
moved on to phase II, which is
taking place in 50 hospitals in
the United States, said Dr.
Joshua Hare, director of the
Interdisciplinary Stem Cell
Institute at the University of
Miami's Miller School of
Medicine and lead author of the
study.
"We're looking on the time frame here of
five years, in the best-case scenario, to
have approved cardiac stem cell therapies,"
Hare said.
Coronary heart disease, which causes
heart attacks and angina -- chest pain
resulting from the heart not getting enough
blood -- is the leading cause of death in
the United States, with nearly 450,000 in
2005, according to the American Heart
Association. About 1.1 million people have
attacks occur in the United States each
year, according to the National Heart Lung
and Blood Institute.
The particular kind of cells used in
this research are called mesenchymal stem
cells, and come from adults, not
embryos.
The researchers are using a mesenchymal
stem cell therapy that is marketed by
Osiris Therapeutics
Inc. under the name Prochymal.
The drug, which consists of stem cells from
donor bone marrow, gets injected into the
vein. The cells then travel through the
bloodstream and take up residence in the
heart.
Mesenchymal stem cells have some natural
homing capability, and injury serves as a
homing beacon for them, Karp said.
The stem cells reduce the amount of scar
tissue and increase the pumping strength of
the heart in heart attack patients, Hare
said. To a limited extent, they also grow
new heart muscle.
The phase I results from 53 patients are
not definitive proof that the treatment is
effective, but do suggest so, Hare said. In
accordance with clinical trial regulations
set by the Food and Drug Administration,
phase I is meant to show safety, while
phase II and phase III trials are done on a
larger group of subjects and evaluate how
well the drug works.
In this trial, patients' doses ranged
from 35 million cells to 350 million cells.
There was no change or increase in side
effects in treatments getting higher doses
of cells, but it seemed that the treatment
was more effective -- at least in terms of
reducing electrical problems -- in the high
dose group, he said.
In separate trials, researchers are
looking at how the technique works in
patients who have had heart attacks many
month or years in the past, Hare said.
The study shows the results of a
six-month follow-up with patients in 2007,
and researchers are presently working with
data from the two-year follow-up.
More broadly, besides bone marrow
transplantations, there have not been any
major successes in the stem cell field in
terms of helping large numbers of patients,
Karp said.
"Mesenchymal stem cells are poised to
really be the next major success in cell
therapy that could be used to treat tens of
thousands of patients," he
said.
There are several advantages to using
mesenchymal stem cells for heart therapy.
First, they are adult stem cells, so there
are no ethical issues that surround
research on embryonic stem cells. Secondly,
at least from a safety perspective,
mesenchymal stem cells do not require
matching -- any donor can give cells to any
other donor, and no immunosuppresant drugs
are necessary, he said.
Still, millions of cells are required in
order for enough stem cells to reach the
heart and have a therapeutic benefit, and
it's costly to get the required numbers of
cells for each patient, Karp said. An area
of improvement would be making the stem
cell treatment more efficient, he said.
"If we could increase the number of
cells that could reach the heart, that
would have significant advantages to this
approach," Karp said.
This method of intravenous injection
means that the procedure theoretically
could be performed in a doctor's office or
clinic, increasing the accesibility to
patients, Karp said.
A more invasive stem cell delivery
technique has been showing positive results
in other clinical trials, involving
injecting patients' own stem cells directly
into heart muscle.
The largest national stem cell study for
heart disease, sponsored by Baxter Inc.,
uses this surgical method, and recently
announced success in a 12-month phase II
trial for patients with severe angina.
Researchers found that participants had
less pain and an improved ability to
walk.
Because stem cells are delivered through
a catheter in this method, there is a risk
of perforation of about 1 percent, Dr.
Douglas Losordo, cardiologist at
Northwestern Memorial Hospital in Chicago,
Illinois, told CNN earlier this year. The
drug, GCSF, which mobilizes stem cells,
also carries a small risk of blood
clotting.
For the Osiris trials, further research
is ongoing determine whether patients would
benefit more from more infusions of the
stem cells, or if it's better for the stem
cells to come from their own bodies instead
of from a donor, Hare said.
While there's less chance of a person's
body rejecting his or her own stem cells
than from a donor, there is a time delay,
he said. Taking bone marrow and amplifying
the stem cells in it could take up to five
weeks, whereas donor cells could be made
readily available, he said. On the other
hand, it is unclear whether a donor could
potentially transmit a disease to the
recipient in the process.
If you are interested
in enrolling in a clinical trial, you can
find one at clinicaltrials.gov. The
Orisis
trial is still recruiting
participants.