Controversial: "Milasen", the first drug created for a single patient

"Milasen" is the name of a very special new medicine: it can only be used by one person, Mila Makovec, who suffers from a unique genetic disease.

It's ultrapersonalized medicine. Its emergence opens a regulatory and ethical debate.

The new drug has recently been described in an article in the New England Journal of Medicine.

Mila is eight years old and lives with her mother in Longmont, Colorado (United States).

He suffers from a rapidly progressing neurological disorder that is fatal. His symptoms started at 3 years old.

According to a newspaper article The New York TimesIn a few years, Mila has lost her sight and can not stand up or lift her head. I needed a food probe and I was doing up to 30 seizures a day.

Rare rare disease

The diagnosis was Batten's disease, but a special case.

It is a recessive disorder: the patient must inherit the mutated MFSD8 gene from her mother and father.

Mila had only one mutated gene, but still had the disease.

Researchers at the Boston Children's Hospital discovered that the normal gene contained a strange DNA fragment, which resulted in failure.

As you go

Scientists have created a drug, which is actually an RNA fragment that blocks the effects of foreign DNA.

It's an expensive drug, but Mila's mother has created a foundation and raised three million dollars.

The drug has been tested on rodents and finally, the US Food and Drug Administration. authorized its use in humans: only at Mila.

Mila has stopped having seizures and no longer needs to be powered by a cannula. Despite this, the girl is still unable to speak and has little mobility.

Open debate

This is the first medication for a single patient and opened the debate. There are more than seven thousand rare diseases and only 10% have an authorized treatment.

With which there would be many patients in the same situation as Mila. The case leaves questions:

• Can science give answers to everyone individually?
• What type of evidence is needed before exposing a human to a new drug?
• To what extent is it justified to develop ultra-expensive drugs that do little to improve the patient's health? How to evaluate the effectiveness of a personalized medicine?
• And more: who will pay for this ultra-customized medicine?

The Cordoba case

A case known in Cordoba a few months ago opened a similar debate.

The parents of a patient with spinal muscular atrophy (AME) have managed to get Justice to order them to work in the social service of the University of Daspu and that the state pays expensive treatment.

SMA is a rare disease that progressively destroys lower motor neurons.

The drug Nursinersen (Spinaza NR) is not registered in the country. Nor is it approved by the National Administration of Medicines, Food and Medical Technologies (Anmat).

The cameramen understood that, the life of a child being in danger, the cover should be imminent. And then they asked Social Work and the Nation to import the medicine.