Gene Therapy.

In 2019, Terry Pirovlakis’s son was diagnosed with SPG50. Terry was determined to find a cure for his son, Michael, and he was able to fundraise MILLIONS of dollars toward this mission. His incredible efforts have led to the development of a gene therapy treatment, called Melpida.

A copy of the working AP4M1 gene is injected into the spinal column. This may slow down or stop the progression of the disease. Terry’s son was the first child to receive a dose in 2022 in Canada. In 2023, four additional children received a dose in the US. So far, the results are promising, and the FDA has approved Melpida to move into a phase 3 trial. The tentative start date is August 2024 with the intent to deliver 8-12 more doses to affected children! This is a herculean feat, but the battle is not over yet. Large grants that the program was reliant on to see it through success has been denied.

We want to keep this trajectory moving forward. We don't want Lincoln, or any other child with SPG50, to get left behind. Treatment is within our grasps.

Research and drug development is an expensive, lengthy process. It is difficult to obtain funding and support from large pharmaceutical companies as ultra-rare diseases are seen as a market that is not financially beneficial. This is considered an orphan disease.

For perspective, one dose of Melipida costs $250,000 to $300,000 to manufacture. 8-12 doses will cost upwards of $3 million.

Research on rare disease is reliant on fundraising and generous donations. Please consider supporting this effort to not only see this trial through to success, but to make this treatment accessible to all 100 children in the world diagnosed with SPG50.

For more information on Terry’s story (it’s truly remarkable) click on the button below.

For more information on the clinical trial, click the button(s) below.

“Being proactive isn’t about working harder. It means doing what you can to change a circumstance, rather than grinding against it.”

-Adam Grant