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Sofia is the name of the girl under six months old who suffers from SMA treated at the Santobono pediatric hospital in Naples thanks to a new gene therapy developed by Novartis. The Swiss multinational operating in the pharmaceutical sector has commercialized one of the most expensive drugs in the world for the treatment of spinal muscular atrophy: it is called Zolgensma and was authorized in Europe in May 2020 and in Italy on November 17 of the same year.
Zolgensma, what do we know about the spinal muscular atrophy drug SMA?
As reported by the Santobono hospital, it is the first time that treatment with Zolgensma has been administered in our country. Mom How does the therapy work?
Zolgensma uses harmless, genetically modified viruses to increase levels of SMN proteins (or survival motor neurons), which are necessary for the survival of motor neurons. Giving treatment (by ‘intravenous injection’) allows these viruses to travel through the body and reach a variety of different cells, helping to restore some of the missing or non-working SMN protein in SMA patients.
The virus used in Zolgensma gene therapy begins to produce antibodies as soon as it is administered to a patient, for this reason only a single injection of the treatment can be used effectively, a second, on the other hand, would not work, since the effects would be neutralized by antibodies developed by the ‘body.
In addition, the virus produced in the laboratory To create the drug, Zolgensma can be found in the environment, therefore some people, including people with SMA, may have developed a natural immunity to it, which means they would not get any improvement from treatment. Consequently, any child, youth or adult who is considered for a trial with Zolgensma should always be screened to see if they are immune and therefore unsuitable for treatment.
Zolgensma, how much does it cost and how does the new gene therapy work?
The drug administered to Sofia is currently the most expensive in the world: a single treatment with Zolgensma costs 1.9 million euros.
The administration of the drug Zolgensma is drip and lasts approximately 60 minutes. As explained above, in addition, only one dose is needed to verify its effects, the same dose that once injected transmits a healthy copy of the SMN1 gene to the cells of the patient’s body and reaches those of the spinal cord. At this point, the healthy SMN1 gene begins to produce the SMN protein which, for the first time in the patient, begins to function normally, helping to improve motor neuron function and increasing the chances of survival.
Zolgensma has been approved by the Food and Drug Administration (FDA) for patients with all forms and types of SMA who have less than 2 years at the time of administration. As with all treatments, time is key. Babies treated with Zolgensma after diagnosis fared better than those who waited for treatment.
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