Eight-month-old boy suffers from spinal muscular atrophy
By
Yalilé LoaizaFrom Quito
Maikel and his parents. (Photo: The EC Observer)
An eight-month-old Ecuadorian baby needs the most expensive medicine in the world to treat his spinal muscular atrophy (SMA), it is Maikel Pisco who is now in intensive care at the Baca Ortiz pediatric hospital in Quito . The medicine to treat her ailment is called Zolegsma and has a value of USD 2.1 million .
Four months ago, Maikel was diagnosed after, on a family trip, he began to drown and had to be transferred to Quito to receive medical attention. The baby’s parents received tests carried out in the United States where the condition was confirmed.
Spinal muscular atrophy is due to deficiency in the translation of survival motor neuron (SMN) protein, which is needed by the brain to send commands to the muscles via nerves that travel down the spinal cord. This disease has varying degrees hence some types have been classified. Type 1, for example, is for patients whose disease begins between 0 and 6 months of age, according to a scientific study.
Spinal muscular atrophy has a worldwide incidence of approximately 1/10,000 births, that is to say that 1 in 40 people in the world is a carrier. In Ecuador, according to information from the Ministry of Health, 18 more children have spinal muscular atrophy, according to data up to October 2021.
In 2021 the Ministry of Health reported that three children with a complex type of SMA would receive the medicine. However, now Maikel’s parents ask that their son’s case be also considered in order to obtain the medication.
With a broken voice, Fernando Pisco, tells the local press that her son “was independent, he moved his little arms, he laughed, he really wanted to live, he fed himself”, but now the degenerative disease affects his mobility.
Desperate for help, Maikel’s parents sent a letter to the Minister of Health, Ximena Garzón, and President Guillermo Lasso to ask them to attend to their little boy’s case, but they have not received a response: “I haven’t heard anything, they haven’t done anything (…). Madam Minister, put your hand on your heart…”, said the father.
Zolgensma, which has been sold since 2019 and whose price amounts to USD 2.1 million, is a drug produced by the Swiss pharmaceutical company Novartis, and consists of a genetic treatment that seeks to repair the genes of infants so they can produce these proteins in normal amounts.
Novartis has insisted that it “works with governments and health insurance companies to get them to cover the cost of the medicine and to pay for it in installments”.
The pharmaceutical company Novartis has stressed that the application of Zolgensma, which is performed only once and costs USD 2.1 million, avoids more medical expenses for families. Treatment costs are often in the six figures in dollars.
There is currently an alternative to Zolgensma, it is Spinraza, a treatment for life, which is taken four times each year. The price of the medicine is USD 750,000 the first year. The following years the treatment costs USD 350,000. This implies that a patient must invest at least USD 4 million every decade.
The Food and Drug Administration of the United States (FDA, for its acronym in English ) plans to approve until 2025 more cell and gene therapies. As of 2019, there were at least 2,600 gene therapy clinical trials underway or completed in 38 countries around the world.
In July 2021, one of the Ecuadorian babies who suffered from this disease died without being able to receive treatment.