Zolgensma - The Most Expensive Life Saving Drug
Zolgensma is the most expensive medicine given for the patients of spinal muscular atrophy. It is the best medicine known for gene therapy. It comes under the brand name
- Nov 02 2021
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What is
Zolgensma and Why Zolgensma Drug is So Expensive?
As since humans
are born there is always a development in their genes . As humans are believed
to evolve from monkey , there is a continuous genetic change kept on going.
Also, many diseases with genetic change of human being have also evolved. From
which spinal muscular injury is a rare condition. It is a life threatening
disease that can cause death of a person . It can happen both in children and
adults.
Spinal
muscular atrophy
It is the
condition where there is a loss in motor nuerons and progressive muscle
wasting. There is an uncontrollable muscle weakness which is marked by
disability of the muscles. It is diagnosed in early childhood as the patient is
not able to walk or speak. It may also appear in later stages of life. The
other problems may include :-
-weakness of
voluntary muscles
-weakness of
arms, legs
-weakness of
respiratory muscles
-poor head
control
-difficulty in
swallowing
-scoliosis
-joint
contracture
This is a life
threatening disease if not taken care. So , there is a medicine names Zolgensma
given for patients of spinal muscular atrophy.
What is
Zolgensma?
Zolgensma is the
most expensive medicine given for the patients of spinal muscular atrophy. It
is the best medicine known for gene therapy. It comes under the brand name
Onasemnogeneabeparvovec. It is directly infused to the vein. The
company provides a copy of SMN protein in form of Zolgensma medicine. One
injection of Zolgensma is of 16 crore rupees
Why is
Zolgensma drug is so expensive?
It is so
expensive because of the rarity of the disease as among 10,000 children only
1-2 get these disease. Government has put up heavy taxes on the import of the
medicine. The high cost of this drug is being put up by the company itself as
it dramatically transforms the lives of families who are being affected.
novel gene
therapy zolgensma drug
The spinal
muscular atrophy happens due to the SMN1 gene that codes for survival motor
nueron protien which eventually leads to motor nueron cell survival. Motor
nueron cells are responsible to communicate with our muscles and tell them
about the muscle action that has to be done. But if the SMN 1 gene is not present
then the motor nueron will not work and eventually life the no muscle action
would be there. This disease happens in children with deficiency of SMA.
Hence, Zolgensma
is a drug that is made up of working copy of human gene to essentially replace
the one that is missing or not working properly. It is placed in Adeno
associated virus 9 (AAV 9) vector which simply acts to take the gene where it
is needed. Therefore, it delivers the gene directly to motor nuerons of the
body.
The protien that
accumulate in the nuerons starts producing the SMN functional protien. These protiens bring back
the lost functions of the child and hence, expanding the life span of children.
Clinical
research Zolgensma drug and Clinical Research Associates
The pre- clinical
research includes In Vivo use of animal models and in vitro use no use of
animal models. There are variety in AAV9 vectors that provides the drug to be
safer in all aspects. The clinical trial associates carry out these trials to
see if the drug is safe for the people or not.
AAV has been used
to treat various other diseases such as:-
- cystic fibrosis
- parkinsonism
disease
- caravan disease
- limb girdle
muscular dystrophy
these are list of
diseases that have been significantly cured by AAV.
Clinical
Research and Pharmacovigilance:
So many
Clinical Research has been Going on for the Novel Gene Protein Therapy.
What is the
Novel Gene Therapy and the Clinical Trials for Novel Gene Therapy?
Novel Gene
Therapy is for the treatment of SMA in childrens. The Therapy by inserting a
healthy gene into the genome into the precise spot in the form of Zolgensma
Injection.
The Clinical
Trials has been occurring from so many years. So many Clinical Research
Associates have been involving in the process of the Clinical Research. The
Well Trained Clinical Research Associates with Clinical Research Certification
has been involved.
Clinical Trials for Zolgensma Injection:
Novartis is playing a crucial role in the
development of Zolgensma Injection.
1 st
clinical trial - An in vivo
model has been successfully delivered in to airway of rabbits which had
continued to be naturally expressed indicating that it was a successful
treatment for adult cystic fibrosis patient.
2nd clinical
trial- an in vitro model
where AAV vector was able to bring a vital gene to skeletal muscle and central
nervous system which allows the body to produce various types of protiens
3rd clinical
trial- the AAV vector
was able to produce dopamine to a patient of parkinsonism disease.
then clinical
trials for Zolgensma were done where SMN gene was put into the AAV vector and
three clinical trials were made:-
START
clinical trial-
where 15 patients
were given Zolgensma injection
-12 with high
dose
results showed
that all 12 people were able to breath properly without any breathing support.
75% could sit
without help
17% could stand
and walk without help
- 3 with low
dose
3 patients
required permanent breathing support.
hence, the Zolgensma
is the medicine which is given in the earlier stages of the disease that is
till the age of 2 years can cure the disease effectively.
side effects
the patients
treated with Zolgensma had - elevated liver enzymes
- also they had
vomiting
- low platelets
- elevated levels
of cardiac troponin-I
- Thrombotic
microangiopathy
Important
Safety Information
- patients
receive corticosteroid after the infusion of Zolgensma so proper blood tests
should be taken.
- if the skin
becomes yellow or eyes becomes yellow contact the doctor as it is s sign of
liver toxicity
- viral
infections can become a major respiratory problem after the drug infusion.
- seek immediate
help if there is decreased platelet count.
- make the
patient to wear diapers as some amount of Zolgensma can appear in the patient's
stool.
So, if your child
is suffering from the condition of spinal muscular atrophy and the age is below
2 years you need to contact the doctor immediately for help.
Clin
Infotech who is a well known Clincal Reserach Organization hiring for theClinical Research Associates with Clinical Research Training and Certification
for the various process of Clinical Trails