Four families are fighting to finance the treatment of GENIQUE therapy SPG50 for their children

Four American families are desperate to save their children from a devastating genetic disease. Healing exists – but it’s up to them to raise $ 1.15 million to finance a clinical trial.

Each of these families has a child with spastic paraplegia 50 (SPG50), a rare neurological disorder that affects less than 100 people in the world.

In an interview with the camera with Fox News Digital, families talked about their race against time to get treatment. (See the video at the top of the article.)

Mother FRANTIC to save a clinical trial that could heal her daughter: “Treatment is seated in a refrigerator”

The objective of families is to raise $ 1.15 million by the end of October to start the trial in the United States, they created a non-profit organization called Jack’s corner To help in fundraising efforts.

What is SPG50?

SPG50 slows down a child developmentGradually leading to a cognitive impairment, muscle weakness, speech problems and ultimately paralysis, according to the national organization of rare disorders.

(From left to right) Naomi Lockard, Mila Wood, Cade Jobsis and Lincoln Medeiros were all diagnosed with the SPG50 genetic disease. (Lindsey Jane Photography; Erika Mills; Leah Serena photography; Candice Jean Photography)

“The prognosis varies from one person to another, but it is generally a progressive condition,” said Dr. Eve Elizabeth Penney, epidemiologist in Texas Department of State Health Services and medical contributor for Drugwatch, previously told Fox News Digital.

“Children with SPG50 can undergo early development delays, muscle weakness and spasticity,” she said. “Over time, the symptoms can worsen, which makes people difficult for people affected to walk and perform daily activities.”

There is no treatment currently approved by the American Food and Drug Administration (FDA) for SPG50. However, there is, thanks to Terry Pirovolakis, a father in Canada whose son was diagnosed in 2017.

The father created a medication to save his son from a rare disease, now other families are desperate to obtain it

“They told us to go home and love it – and said it would be paralyzed in size at the age of 10 and quadriplegic at the age of 20,” Pirovolakis told Fox News Digital of his son’s diagnosis. “They said he would not work or speak, and need support for the rest of his life.”

By refusing to accept this result, Pirovolakis embarked on a mission to save his son – he liquidated his savings, met many experts and scientists paid to create gene therapy.

Emma and Dylan Jobsis, from Juneau, Arkansas, have a 4 -year -old son, Cade (photo), who was also diagnosed with SPG50 in 2023. (Leah Serena photography)

In March 2022, his son received gene therapy and was now prosperous. Pirovolakis then opened a phase 2 study in the United States, which treated three other children. All reported that the disease has stopped progressing and that their cognition has improved.

Now, the objective is to go to a phase 3 test, which would allow more children to be treated – but because the drug is not yet approved by the FDA, families will have to collect funds for the trial themselves.

Four families, a rare disease

Rebekah Lockard, from Littleton, Colorado, is desperate to treat her 4 -year -old child, Naomi, who received a diagnosis of SPG50 in 2023.

Her young child, Jack, also received a diagnosis of SPG50 and received therapy in a previous test because of an early age.

The Lockard family, from Littleton, Colorado, is fighting to treat their 4 -year -old child, Naomi, who received a diagnosis of SPG50 in 2023. (Lindsey Jane Photography)

“Naomi remains unable to walk and has a wheelchair for mobility, while Jack moves quickly with the help of a walker,” Lockard told Fox News Digital. “Naomi has a few sounds and gestures she uses to communicate, while Jack sails with her gestures and his understanding of what we say.”

“Every day, we are so grateful that Jack has received a significant treatment at an early age. And every day, we feel desperate that we could not give Naomi the same quality of life.”

Mother with a rare ALS boils the “miracle drug” which has stopped her illness

Emma and Dylan Jobsis, from Juneau, Arkansas, have a 4 -year -old son, Cade, who was also diagnosed in 2023.

“Right now, Cade is still able to travel short distances with his walker, but without treatment, we know that he will lose this capacity,” Emma Jobsis told Fox News Digital.

“There are eight doses, sitting and waiting to be given to children who need it desperately.”

“Right now, we are living with the daily fear of looking at our son to lose skills and independence that he worked so hard to win. Without treatment, the future of Cade is that of decline – finally losing the ability to walk, eat and do things that make him who he is.”

Jordan and Cody Medeiros, from Scio, Oregon, were devastated to learn that their 4 -year -old son, Lincoln, A SPG50.

Emma and Dylan Jobsis, from Juneau, Arkansas, have a 4 -year -old son, Cade, who received a diagnosis of SPG50 in 2023. (Candice Jean Photography)

“Since the diagnosis of Lincoln two years ago, SPG50 has slowly removed its ability to walk safely,” Jordan Medeiros told Fox News Digital. “He often falls – several times a day. And he cannot verbalize his pain or his frustrations.”

“The worst part is that there are eight doses, seated and waiting to be given to children who need it desperately.”

Click here to obtain the Fox News app

In Susquehanna, Pennsylvania, Jami and Cody Wood welcomed twins in 2022. In August 2025, they were devastated to learn that one of the twins, Mila, had SPG50.

Three weeks later, Mila took her first steps, an important step that her parents described as “bittersweet”.

“Without the medication, we will be condemned to look at it slowly escape.”

“We know that there is only one treatment at his disposal, and without him, she will lose this stage probably in as long as she took him to win it,” Jami Wood told Fox News Digital.

“Genetic therapy is currently the other treatment option for Mila, and this test is our daughter’s only chance to stop the progress of this terrible disease. Without the medication, we will be condemned to look at her slowly escape.”

Ahead

The hope is that after a successful phase 3 test, the drug will obtain the approval of the FDA, which would mean that the insurance companies and the government would cover this “huge cost burden”, told Pirovolakis to Fox News Digital.

Click here to register for our Health Newsletter

“More importantly, it will be on the newborn screening panel, so we will have a chance to catch and eradicate this disease.”

Pirovolakis and his team actively work with the FDA to obtain the approval of this therapy, but “the process is anything but simple,” he said.

Jami and Cody Wood welcomed twins in 2022. In August 2025, they were devastated to learn that one of the twins, Mila, A SPG50. (Erika Mills)

The challenge is that the FDA is currently applying the same manufacturing standards with small programs like Pirolakis – which can only serve a few children in the United States – as for diseases affecting hundreds of thousands of children.

“For large -scale conditions, economies of scale help reduce costs – but in our case, these requirements become almost impossible to respond,” said Pirolakis. “We need flexibility of the FDA for these ultra-rare conditions.”

For more health items, visit www.foxnews.com/health

Anyone interested in learning more about SPG50 and families’ efforts can visit the Jack’s corner website.

Fox News Digital contacted the FDA to comment.