Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
Towards personalised therapy for lymphangioleiomyomatosis: lessons from cancer.
[lymphangioleiomyomatosis]
Lymphangioleiomyomatosis
(
LAM
)
is
a
rare
cystic
,
destructive
lung
disease
occurring
almost
exclusively
in
females
.
Bi-allelic
inactivating
tuberous
sclerosis
complex
(
TSC
)
gene
mutations
occur
in
LAM
cells
,
resulting
in
activation
of
the
mTORC
1
pathway
.
Pivotal
clinical
trials
have
demonstrated
that
inhibition
of
mTORC
1
with
sirolimus
can
induce
a
partial
response
of
TSC-associated
tumours
and
decrease
the
rate
of
lung
function
decline
in
females
with
LAM
.
Many
parallels
have
been
identified
between
LAM
pathogenesis
and
neoplasia
.
Here
,
we
highlight
three
key
nodes
through
which
advances
in
cancer
therapy
can
streamline
future
innovation
in
clinical
LAM
research
with
parallels
to
breast
and
prostate
cancer
.
These
include
:
1
)
hormonally
targeted
therapies
to
achieve
true
disease-free
complete
remissions
;
2
)
the
use
of
vascular
endothelial
growth
factor
-
D
and
other
plasma
biomarkers
to
streamline
early
-phase
clinical
trials
;
and
3
)
the
utilisation
of
histological
and
molecular
features
of
biopsy
material
to
enable
patient
stratification
and
personalised
therapies
.
Diseases
Validation
Diseases presenting
"early-phase clinical trials"
symptom
lymphangioleiomyomatosis
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom