2. MEDICAL UTILITY
The creation of drugs with high efficacy
and specificity for diseases as complex as
cystic fibrosis, or a way to improve the
health of patients through education
towards good nutrition, represents a
critical point Towards the approach That
must be taken at the time of choosing a
treatment that is really effective and
durable.
For this
reason,
below
are
exposed
Two news that
Put to test the
best treatments
for cystic
fibrosis
and its
Complications
BIBLIOGRAPHY MONOGENIC
DISORDERS
By: Alejandra Jaramillo Montoya
Molecular Biology
Teacher: Lina Martinez
-Matinez Sanchez, Lina Maria .
Biologia Molecular.7
ed.Medellin:
UPB. Facultad de medicina.
-Cysticfibrosis news today
(2017,Sep 20). Antibiotic
Iclaprim Granted FDA Orphan
Drug Status for CF Lung
Infections.
https://cysticfibrosisnewstoday.c
om/2017/09/20/antibiotic-
iclaprim-granted-fda-orphan-
drug-status-for-cystic-fibrosis-
lung-infections/
-Cysticfibrosis news today
(2017, Sep 14). Gaining Weight
May Help CF patients Delay the
Worsening of Symptoms, Study
Finds.
https://cysticfibrosisnewstoday.c
om/2017/09/14/cf-symptom-
exacerbation-delayed-gaining-
weight-uk-study-suggests/
3. INTRODUCTION
Based on Mendel's laws and various
studies on genetics that have been done
over time, phenomena occurring in
meiosis such as linkage and
recombination allow the exchange
of genetic information between
homologous chromosomes and the risk
of new forms of genetic combination, this
can causes alteration of a single gene
and give rise to the so-called: single-
gene disorders
then two articles
will be presented
on
one of the most
famous monogenic
disorders:
cystic fibrosis
ANTIBIOTIC ICLAPRIM AND THE
LUNG INFECTIONS
The U.S food and Drug Administration (FDA)
has classified iclaprim as an orphan antibiotic
for the treatment of one of the pulmonary
infections of cystic fibrosis: Staphylococcus
aureus In vitro laboratory
studies have proven
the efficacy Of this
drug as a powerful
Killer of various bacteria.
Even Iclaprim rapidly
killed practically all
gram-positive methicillin
Staphylococcus
aureus (MRSA),
overcoming
Vancomycin
Student’s opinión
In my opinion is very
interesting the designation made to this drug
by the FDA, due to this advance means for
patients with cystic fibrosis a way to get better
their quality of life in some way, and reduce
their costs inineffective drugs
GAINING WEIGHT MAY HELP
CF PATIENTS DELAY THE
WORSENING OF SYMPTOMS
A new study has shown that those
patients who receive a treatment during
complications of cystic fibrosis, called:
pulmonary exacerbations, show longer
periods to suffer again complications of
CF. the results of this study were
published in the European Respiratory
Journal Open Research, which confirms
that CF patients suffer from malnutrition,
therefore studies have concluded that
nutrition and lung function are related
Student’s opinión
in my
is reflected
as a patient
with good
nutrition
Responds
better
to a treatment for
pulmonary crises
that occur in the
fibrous cystic, unlike
that patient who has a poor diet
4. INTRODUCTION
Cystic fibrosis is a deadly autosomal
recessive disease, in which the epithelial
glands produce a defective chloride
secretion, this causes an imbalance in the
transport of water and ions, and helps the
formation and accumulation of a viscous
mucus that obstructs the intestine and
biliary and pancreatic tracts "in utero", and
that after birth it will accumulate in the
airways.
5. Staphylococcus aureus, including MRSA, is one of
the most common causes of lung infections in cystic
fibrosis, so for Graham Lumsden, CEO of Motif bio,
who are responsible for the development of this
drug, is a very large advance the fact that a drug has
been found that specifically serves this type of
complications.
Motif searches the iclaprim can be applied
intravenously or inhaled by patients with cystic
fibrosis
6. In the year 2015 the FDA granted
Iclaprim as Qualified infectious Disease
Product (QIDP) designation and fast
track status for bacterial skin and her
structure
7. According to clinical studies, Iclaprim
was administered intravenously with a
fixed dose, it was possible to identify
that its dose does not need
modification in patients who have renal
failure or diabetes; on the other hand,
the studies did not observe some
degree of toxicity in the kidneys
In addition, a study was
performed using mice as models
with MRSA infections. These
mice mimicked the lung
conditions of patients with cystic
fibrosis to see the effect of the
drug.
8. According to Motif
Bio, EL Iclaprim has
a low tendency to
develop bacterial
resistance, it has a
targeted Gram-
positive spectrum of
activity, its dose in
fixed in all the
patients and it has a
profile favorable to
the tolerability
9. the company concluded
that the use of this drug
represents great
benefits for health
centers, as doctors will
not have to make
constant dose
adjustments and
treatment costs may
decrease
10. STUDENT’S OPINION
In my opinion, scientific advances like these
represents a benefit for all areas related to this
disease. Because patients can access a more
effective treatment that requires less toxicity to their
body, and improve their complex economic situations
by trying to pay for all the treatments that involve the
complications of CF.
On the other hand, health centers can provide
more resources to patients, and better implement
their income in other weak points of the treatment
of cystic fibrosis and its complications.
11. To check for improved treatment in patients with good nutrition, the study
used data from a group of patients treated at two CF centers in the UK.
GAINING WEIGHT MAY HELP CF PATIENTS DELAY THE WORSENING OF
SYMPTOMS,STUDY FINDS
this group of patients was treated for 14 days with antibiotics, most of them
only gained 1 kg. When reviewing the clinical histories, it was observed that
98% of the patients had pulmonary exacerbations again in a very short time
After a constant follow-up from July 2009 to March 2012, 59 patients were
diagnosed with a chronic lung infection called Pseudomonas aeruginosa.
12. the researchers concluded
that weight gain during
treatment was directly
proportional to a decrease in
the occurrence of pulmonary
exacerbations again
Patients who gained 1 kg
during the course of their
treatment increased the
time of onset of another
lung crisis due to
pulmonary exacerbations
by 0.79% compared to
those patients who did not
gain weight.
13. Besides,other experts things that althoung
these investigations permited to conclude the relation
between good nutrition and good pulmonary functioning,
there are still many
unresolved doubts, for
example: if it is possible that
weight gain is the total
solution to a decrease in
pulmonary exacerbations or
if these two events occurred
at the same time and are
erroneously associated,
and, on the other hand, if
weight gain may help other
complications of cystic
fibrosis
14. However, the study concluded that weight gain
modifies the risk factor for complications from
pulmonary exacerbations
15. Student’s opinión
I think this study significantly helps to
promote a much healthier way of developing
treatments for pulmonary exacerbations and
its application to other complications of
Cystic Fibrosis, since this study shows that
many times the patient can take many
medications to treat their illnesses, but if the
patient does not start with the minimum that
is a good diet and good mental health, so he
uses the most effective drugs, the treatment
will not be effective
16. M E D I C A L U T I L I T Y
the specific development of a drug for
a complication in this case of CF
contributes so much to medicine,
since it favors a way to reduce the
risk of allergies to drugs, the constant
change of dose or intake that helps
that the patient may develop poor
treatment, the discovery of
components of this medication can
help to create other drugs that
decrease other complications of the
same disease etc
17. M E D I C A L U T I L I T Y
On the other hand, iclaprim kills one
of the most multiresistant antibiotic
bacteria, since antibiotics like
vancomycin could not kill it, and one
of the biggest problems that medicine
has is how to fight microorganisms
that cause so much damage to
humans, which also does not cause
adverse reactions in patients and
does not aggravate basic diseases
18. M E D I C A L U T I L I T
Y
Additionally, concluding the relationship
between the state of nutrition and the
effectiveness of a treatment, helps the
medicine to find that perhaps not all
treatment should be guided in a schematic
way, investigating frequently have focused
on the minimum and the metabolic
component and nutritional maybe they are
leaving aside, perhaps the solution to many
diseases should start to be guided because
it is what you need to cell
19. M E D I C A L U T I L I T
Y
Maybe the treatments alone are based on the treatment to cure an
organ, and the experts are not taking into account that all the organs that
make up the body are related to each other, and the deficiency of a
component in an organ can worsen the another organ already affected,
and there may be the solution to many diseases
20. BIBLIOGRAPHY
-Matinez Sanchez, Lina Maria . Biologia Molecular.7 ed.Medellin:
UPB. Facultad de medicina
-Cysticfibrosis news today (2017,Sep 20). Antibiotic Iclaprim Granted FDA
Orphan Drug Status for CF Lung Infections.
https://cysticfibrosisnewstoday.com/2017/09/20/antibiotic-iclaprim-granted-
fda-orphan-drug-status-for-cystic-fibrosis-lung-infections/
-Cysticfibrosis news today (2017, Sep 14). Gaining Weight May Help CF
patients Delay the Worsening of Symptoms, Study Finds.
https://cysticfibrosisnewstoday.com/2017/09/14/cf-symptom-exacerbation-
delayed-gaining-weight-uk-study-suggests/
