A determined father in Toronto has taken extraordinary steps to save his young son from a rare and devastating neurological disorder.

Terry Pirovolakis, who received regulatory approval for a drug he created, said, "I couldn't let him die," as he aimed to save his six-year-old son and others affected by a rare neurological disorder.

According to Daily Mail Online, Pirovolakis embarked on this medical odyssey to help his son and potentially benefit others with the rare disease.

Father’s Breakthrough Treatment for Son’s Rare Disorder

Pirovolakis, 44, received regulatory approval in December for a clinical treatment he developed to combat spastic paraplegia 50 (SPG50), the condition affecting his 6-year-old son Michael.

SPG50 is an extremely rare neurodegenerative disorder that impacts fewer than 100 people worldwide. The condition leads to cognitive impairment, muscle weakness, and eventual paralysis, often resulting in death before patients reach 30 years of age.

When Michael was diagnosed in 2018, doctors painted a grim picture, telling the family to "go home and love him" as he would likely be paralyzed by age 10 and quadriplegic by 20.

From IT Director To Medical Researcher

Unwilling to accept this fate for his child, Pirovolakis, an IT director by profession, took drastic measures. He liquidated his life savings, refinanced his home, and dedicated himself to researching potential cures rooted in gene therapy. His efforts involved reading countless medical journals and meeting with experts in the field.

Within a month of Michael's diagnosis, Pirovolakis had signed a contract to initiate a gene therapy program. This innovative treatment involves injecting cerebral spinal fluid into the patient's back. After years of intensive laboratory work, the treatment showed promise, leading to government approval on December 30, 2021.

On March 24, 2022, Michael became the first person to receive this groundbreaking gene therapy treatment at SickKids hospital in Toronto. Pirovolakis shared with Fox News:

They said he'd never walk or talk, and would need support for the rest of his life. They told us to just go home and love him - and said he would be paralyzed from the waist down by age 10, and quadriplegic by age 20.

Expanding Treatment To Help Other Children

Pirovolakis's dedication to finding a cure for his son has expanded into a broader mission to help other children affected by SPG50. He quit his job and established a nonprofit organization in California called Elpida Therapeutics, named after the Greek word for "hope."

The organization now employs five people and works with 20 consultants. Their efforts have led to treatment for three additional children in the United States as part of a Phase 2 study in 2022. Among these children was 6-month-old Jack Lockard, whose mother, Rebekah Lockard, reported significant improvements following the treatment.

Lockard told Fox News:

Jack has thrived since then. He is sitting independently, banging toys together, drinking from a straw cup and working really hard on crawling. Doctors and therapists share the same sentiment. The treatment works!

Challenges In Scaling Up Treatment Production

Despite the promising results, Pirovolakis faces significant hurdles in making the treatment widely available. The drug currently costs about $1 million to produce for each child, and pharmaceutical companies have been hesitant to manufacture it on a larger scale.

Pirovolakis explained the dilemma: "No investor is going to give you money to treat a disease that is not going to make money. That's the dilemma we're in." As a result, parents are currently forced to pay out of pocket for the treatment.

The next step for this potentially life-saving drug is a clinical trial at the National Institutes of Health, scheduled for November. Pirovolakis stressed the urgency of the situation, noting that eight doses have already been flown to the US and are ready for use.

Why This Story Matters

This narrative isn't just about disease; it's about how individual resilience, when harmonized with scientific exploration and communal effort, can challenge the status quo of medical possibilities. It sheds light on the broader issues faced by those dealing with rare diseases — a topic needing more visibility, understanding, and action.

Conclusion

Terry Pirovolakis's journey from an IT professional to a medical innovator showcases the power of parental love and determination. His efforts have provided hope for his son, Michael, and other families affected by SPG50.

The upcoming clinical trial represents a crucial step in making this treatment more widely available, potentially changing the lives of children diagnosed with this rare and devastating disorder. As the medical community watches closely, the Pirovolakis family's story serves as an inspiring example of how far a parent will go to save their child.