SpinCura contains
Nusinersen that belongs to a group of medicines known as antisense oligonucleotides
(ASO). Treating 5q Spinal Muscular Atrophy (5q SMA) is the only application for
Nusinersen.
The "survival motor neuron" (SMN) protein is deficient in people
with SMA. The nerve cells that assist movement of muscles are called motor neurons,
and they depend on this protein to function. The brain and spinal cord include motor
neurons, which are responsible for contacting muscles.
Human cells need the SMN1 and SMN2 genes to make the SMN protein. The SMN2 gene,
which typically generates just 10% of the SMN protein and is not as functional as a
full-length protein. The SMN1 gene is mutated or deleted in the SMA patients.
SpinCura (Nusinersen) assists these patients making the SMN2 gene to produce full
length (100%) SMN protein essential for normal function of motor neurons.
SpinCura is a synthetic ASO (manufactured genetic material) that
targets the SMN2 gene and enables it to produce more functional, full-length SMN
protein.
Jodas Expoim Pvt.Ltd., manufactures Nusinersen under the brand SpinCura.
SpinCura (Nusinersen)
cannot cross the blood brain barrier so it has to be delivered directly into the
cerebrospinal fluid (CSF) through intrathecal injection into the spinal canal. Upon
injection, Nusinersen reaches the motor neurons and other cell types in the spinal
cord. It may have limited ability to reach other parts of the body. It is possible
that the quantity of SMN protein required in other bodily areas is still unknown.
Cerebrospinal fluid (CSF)
is the direct site of delivery for SpinCura. Doctor delivers SpinCura through lumbar
puncture, which involves inserting a needle through the skin into the area between
the vertebrae (back bones) of the spine. To determine the ideal location for the
insertion, doctors might need to employ imaging techniques like CT or X-rays.
Typically, they use a topical anaesthetic, such "numbing cream." Older children and
adults may be given inhalation anaesthesiaduring the treatment, although some may
require a sedative, and in rare cases, a general anaesthetic may be deemed required.
You and the treatment facility will speak individually to determine the best course
of action for you/ your child.
The procedure involves collecting a small amount of CSF and
injecting Nusinersen over one to three minutes, with healthcare professionals
receiving specific training for this procedure.
As the treatment can only be delivered by lumbar puncture, this
procedure needs to take place within one of the neuromuscular treatment centres.
Any drug, whether
prescribed or sold over the counter, can cause potential side effects. These effects
are documented during clinical trials and monitored through ongoing systems. The
Patient Information Leaflet, provided with any medication, lists all possible side
effects. They are grouped as:
very common
common
uncommon
rare
For nusinersen, side effects related to the lumbar puncture may
occur while nusinersen is being given or afterwards. The majority of these side
effects are reported within 72 hours of the procedure.
Very common: may affect more than 1 in 10 people
Back pain
Headache
Vomiting
These and any rare or very rare side effects are listed in
SpinCura Patient Information Leaflet which covers other questions as well
Multiple factors will
determine how long the therapy is given to the patient. The process may proceed
quickly or slowly. The best person to tell you how long therapy for you or your
child could take is the treatment facility.
Injections are administered as follows:
SpinCura is injected into the CSF in a series of initial doses known as "loading doses" in order to raise its level to an efficient concentration.
Loading doses are administered as follows:
On day 0, the first day of therapy;
Following doses on day 14, day 28, and day 63.
The minimum interval between dosages should always be 14 days.
Once the loading doses have all been delivered, there will be maintenance doses every 4 months to maintain the drug concentration level in the CSF.
The duration of recovery depends on each individual. The treatment will be for a short span if the patient received nusinersen multiple times previously. The treatment may be prolonged accordingly, if the visit of patient is for the first time, or if the delivery method has complications, or if sedation is employed.
Treatment needs to continue unless the treating clinician and you / you and your child agree that it should stop.
The amount of time it takes for therapy to have an impact varies from person to person and is dependent on multiple factors. It may take few months for some people to experience results with loading doses, while it might take up to a year for others. On occasion, families with young children have reported answers prior to their appearance on motor assessment scores. Generally speaking, the longer a person has had SMA or the more severe their muscular weakness, the longer it may take for their therapy to start showing results.
The following dosage should be given as soon as possible if one dose is delayed. The timing for this will depend whether you / your child are within the treatment schedule. Consult with your child's or your own treating clinician if you have any questions or concerns about giving a dosage later than scheduled or about making sure there is a safe interval between doses.
You will find the answer to many of your questions in SpinCura’s Patient Information Leaflet and will have an opportunity to discuss any further questions you may have with your clinician. The leaflet covers:
What SpinCura is and what it is used for
What you need to know before you or your child take SpinCura
How to take SpinCura
What are the possible side effects?
How to store SpinCura
Contents of the pack and other information
