Father created a medicine to save his son from a fatal disease (3 photos)

Terry Pirovolakis was told by doctors that his son Michael would never be able to walk or talk and would need support for the rest of his life. However, the father did not give up.





Terry Pirovolakis, an IT director from Toronto, Canada, decided to create his own medicine for his child, who was diagnosed with a rare, fatal disease.

The man's third son was born in December 2017. It was a healthy birth, but six months later he and his wife Georgia noticed that their baby, Michael, was not lifting his head.

After months of doctor visits, physical therapy, and genetic testing, which Pirovolakis calls "an 18-month diagnostic odyssey," a neurologist diagnosed baby Michael with spastic paraplegia 50 (SPG50), a neurological disorder that affects fewer than 100 people worldwide.

"They told us to just go home and love him, and they said he would be paralyzed from the waist down by the time he was 10. They said he would never walk or talk and would need support for the rest of his life," Pirovolakis said.



The publication notes that spastic paraplegia 50 (SPG50) is a neurological disorder that affects a child's development, gradually leading to cognitive impairment, muscle weakness, speech impairment, and paralysis, according to the National Organization for Rare Disorders.

Most people with the disorder die by age 20.

A Father's Mission

Currently, the U.S. Food and Drug Administration has not approved any treatments for SPG50.

After the shock of the diagnosis, Pirovolakis immediately began research, focusing on finding a gene therapy that could help his son. He visited Sheffield and the National Institutes of Health at the University of Cambridge in England, where scientists were studying the disease.

"We then pulled out our savings, refinanced our house, and paid the team at the University of Texas Southwestern Medical Center to create a proof of concept to begin gene therapy for Michael," says Pirovolakis.



After successful trials showing that the gene therapy was effective in stopping the disease from progressing in mice and human cells, Pirovolakis partnered with a small pharmaceutical company in Spain to manufacture the drug.

On December 30, 2021, Health Canada gave approval for gene therapy for Michael.

"On March 24, 2022, my son became the first person to receive gene therapy at SickKids in Toronto. The procedure, which involves injecting spinal fluid through a lumbar puncture, comes with risks, but the potential benefits are lifesaving. I couldn't let him die," the child's father said. .

Pirovolakis said it costs about $1 million to make the drug for each child, and about $300,000 to treat a patient in a U.S. hospital. He approached pharmaceutical companies, but they all refused to produce the drug.

"We want to make sure that the trials continue and that these children get the treatment. No investor is going to give you money to treat a disease that is not profitable," he said.