Stem
cell
transplants
that
are
a
standard
treatment
for
certain
blood
cancers
are
out
of
reach
for
many
patients.
The
procedure
requires
cells
from
a
matched
donor,
but
a
match
isn’t
always
available.
A
new
cell
therapy
derived
from
umbilical
cord
blood
now
has
European
Commission
approval,
a
regulatory
decision
that
provides
eligible
patients
with
another
option
and
gives
the
therapy’s
developer,
startup
ExCellThera,
its
first
commercial
product.
The
Wednesday
regulatory
decision
covers
the
treatment
of
adults
with
hematological
malignancies,
including
leukemias
and
myelodysplastic
syndromes,
that
require
an
allogeneic
hematopoietic
stem
cell
transplant.
To
be
eligible
for
this
therapy,
suitable
donor
cells
must
not
be
available
to
patients.
The
new
product
of
Montreal-based
ExCellThera,
known
in
development
as
UM171
cell
therapy,
will
be
commercialized
under
the
brand
name
Zemcelpro.
Suitable
donors
for
stem
cell
transplants
can
be
unavailable
for
various
reasons.
Besides
the
inability
to
find
a
suitable
match,
the
age
and
health
of
the
donor
are
also
considerations.
In
other
cases,
a
patient
may
already
have
antibodies
that
would
reject
donor
cells.
Cord
blood
has
cells
that
are
less
likely
to
be
rejected,
but
this
source
also
has
few
cells.
ExCellThera’s
technology
works
with
a
small
volume
of
stem
cells,
multiplying
them
in
cell
culture
to
provide
the
allogeneic
stem
cells
required
for
blood
cancer
patients
who
lack
a
suitable
donor.
The
technology
is
based
on
research
from
the
University
of
Montreal;
ExCellThera
spun
out
of
the
university
in
2015.
ExCellThera
advanced
Zemcelpro
to
a
Phase
2
clinical
trial
conducted
in
the
U.S.,
Canada,
and
Europe.
The
study
enrolled
patients
with
high
or
very
high-risk
acute
leukemias
and
myelodysplasias,
patients
who
have
had
a
transplant
procedure
and
require
another
one,
and
those
who
have
refractory
or
active
disease.
Results
showed
an
overall
survival
rate
of
67%
and
a
progression-free
survival
rate
of
63%.
The
relapse
rate
was
low
at
19%;
7%
of
participants
developed
moderate-to-severe
chronic
graft-versus-host
disease.
These
data
were
presented
during
the
2023
annual
meeting
of
the
American
Society
of
Hematology.
The
European
Commission
decision
for
Zemcelpro
is
a
conditional
marketing
authorization
based
on
the
Phase
2
data.
This
pathway
reserved
for
therapies
addressing
unmet
medical
needs
and
serious
diseases
with
no
available
treatments.
Conditional
authorizations
can
be
renewed
annually,
but
a
company
must
continue
to
provide
data
supporting
therapeutic
benefit.
ExCellThera
said
a
Phase
3
clinical
trial
is
planned.
In
ExCellThera’s
approval
announcement,
Dr.
Jurjen
Versluis,
internist-hematologist
at
Erasmus
MC
in
Rotterdam,
The
Netherlands,
and
principal
investigator
in
the
Zemcelpro’s
clinical
trials,
said
the
lack
of
suitable
donor-derived
blood
stem
cells
leaves
many
patients
without
access
to
a
potentially
life-saving
treatment.
“Zemcelpro
is
an
innovative,
one-time
cell
therapy
with
curative
intent,
developed
to
give
blood
cancer
patients
without
access
to
suitable
donor
cells
the
transplant
they
urgently
need,”
Versluis
said.
“By
enabling
more
patients
to
receive
a
life-saving
transplant,
Zemcelpro
has
the
potential
not
only
to
save
lives
but
also
to
reduce
the
healthcare
and
societal
burden
associated
with
these
devastating
conditions.”
The
regulatory
decision
permits
marketing
of
Zemcelpro
in
all
European
Union
member
states
as
well
as
Iceland,
Norway,
and
Liechtenstein.
ExCellThera
said
availability
of
Zemcelpro
will
depend
on
several
factors,
including
completing
reimbursement
procedures
in
each
country
covered
by
the
authorization.
Cordex
Biologics,
an
ExCellThera
subsidiary,
is
seeking
strategic
partnerships
to
accelerate
commercialization
of
Zemcelpro
in
Europe
and
other
markets.
ExCellThera
said
it
has
been
speaking
with
the
FDA
about
a
potential
regulatory
filing
for
Zemcelpro.
The
company
has
also
filed
an
amendment
for
an
investigational
new
drug
application
to
proceed
to
a
Phase
3
test
of
the
cell
therapy
in
patients
with
high-
and
very
high-risk
leukemias
and
myelodysplasias.
U.S.
blood
cancer
patients
already
have
a
cell
therapy
option.
In
2023,
the
FDA
approved
Omisirge,
a
cord
blood-derived
cell
therapy
developed
by
Gamida
Cell.
The
company,
which
had
shaky
finances
prior
to
Omisirge’s
approval,
sought
strategic
partnerships
or
other
transactions
to
support
commercialization
of
its
new
product
but
failed
to
land
any
deals.
Last
year,
Gamida
Cell’s
largest
lender
acquired
the
biotech,
taking
it
private.
Omisirge,
Gamida
Cell’s
only
commercialized
product,
is
not
approved
in
Europe.
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