Pennsylvania boy is headed to California for a clinical trial that might help save his life

This isn’t the column I was hoping to write after introducing readers to Jax Ramirez in 2022, a cherubic 9-year-old who lives just outside of Pittsburgh with a rare autoimmune disease.

But it is a column that brings me one step closer to the one I still hope I will one day write. The one where I share that Jax was finally able to receive a lifesaving treatment for the IPEX syndrome that attacks his organs.

For years, that meant a necessary and urgent bone marrow transplant his family — mom, Missy, dad, Peter, and his 6-year-old brother, Lincoln — was singularly focused on.

In less than three years, the family has held more than 200 events to raise awareness and helped register nearly 7,000 new donors in Jax’s honor. Their story and efforts caught the attention of the likes of the second lady of Pennsylvania, Gisele Fetterman, and Philadelphia boxer Jaron “Boots” Ennis, who befriended Jax after learning he was a huge fan.

“He’s a fighter, just like me,” Jax said at the time.

With the kinds of odds Jax faces, he needs to be. Even with an estimated 30 million people on bone marrow donor registries worldwide, about 4,000 patients are searching for a match at any given time, according to NMDP, a nonprofit formerly known as the National Marrow Donor Program and Be The Match. It’s worse for people of color, who make up a small percentage of all donors, making it difficult to find matches, which are based on DNA and heritage. Because Jax’s dad is of Mexican descent, it’s particularly hard for Jax to find a match.

It seemed nothing short of miraculous when three matches were identified in 2022.

“Unfortunately, one they couldn’t find, and two others said no,” Missy Ramirez told me when we recently spoke.

Yet, the family remained surprisingly hopeful.

“It gave me some level of hope because up to that point, I was convinced that he had no matches anywhere,” Ramirez said. “But then I was like, ‘No, they do exist. There are people in this world that match him.’ So I knew I just had to keep pressing on and doing what we’ve always done, which is to try and save his life.”

Last year, the family was approached about a clinical trial of a new gene therapy for IPEX at Stanford Medicine Children’s Health in California, led by a world-renowned expert in IPEX, and where Jax’s local doctor had moved. With no transplant matches and time working against them, the family seriously considered participating. But the risks to Jax’s compromised immune system would only become more acute by flying commercially and staying in communal lodging offered by the hospital.

The decision was gut-wrenching, Ramirez said, but they just couldn’t jeopardize his already fragile health.

“I cried,” recalled Ramirez, a school psychologist for the Pine-Richland School District. “He didn’t even know it was a possibility — we hadn’t told him. But I was just so sad.”

Disappointed, but more determined than ever to find a bone marrow match, the family continued holding events and registering new donors.

Then, a couple of months ago, a second call came from Stanford, again asking Jax to participate in the trial. The family worried the same logistical issues would mean they’d have to decline the offer again. But this time their small borough of Zelienople in western Butler County, with a population of less than 4,000, came to the rescue. A local businessman contacted another local businessman, Aaron Rectenwald, the owner of TRC, a general contracting company based in Zelienople but with projects all over the country. He immediately offered the use of his private plane.

“I have a young family, and I can only imagine what they’ve been going through,” Rectenwald told me.

Loaning his plane, he said, was “the right thing to do.”

The family plans to leave in early June. A friend set up a GoFundMe to help the family stay together in a rental home while Jax receives months of care nearby.

“He talks about it all the time,“ Ramirez said. “He talks about being excited to maybe not have to take as much medication as he takes. He’s very positive about it.”

Still, the trip will be an emotional and financial strain, with no guarantees.

Jax — whose biggest dream is to get on a school bus — still has to pass a battery of tests to make sure he’s healthy enough for the treatment, which includes extracting his T cells, which help protect the body from infection, and eventually transplanting them with his own genetically modified cells.

“The hope is that those genetically modified T cells will replicate, and those replicated T cells are the ones that are going to function normally,” Ramirez said.

It will be a long few months, at the very least, for Jax and his family, one spent inside a hospital while other little boys and girls his age spend their days outside soaking up every moment of summer — playing ball, jumping into pools, and chasing ice-cream trucks down the street.

But they hope that one day, in the not-too-distant future, Jax will be among them, just another healthy and happy little boy.