Alexander Owens: Decades of Research Brings a Breakthrough Therapy and a Bright Future

In 2012 when Diane Owens took her 2-month-old son Alex for a routine checkup, she never imagined that visit would change their lives forever. She had noticed what looked like birthmarks near his groin and pointed them out to the pediatrician. Those marks turned out to be café-au-lait spots, a common sign of neurofibromatosis type 1 (NF1), a rare genetic disorder that can cause tumors to grow along nerves in the body.

At 4 months old, Alex was officially diagnosed. “It felt like we got hit by a freight train,” Diane recalled. “We were told there was no cure, no treatment, and no way to predict what would happen. All we could do was watch and wait.”

For years, that’s exactly what they did. Regular MRIs tracked the growth of tumors, including a plexiform neurofibroma on Alex’s back and several others near his brain stem. While not cancerous, plexiform neurofibromas can cause many clinical problems including pain and functional deficits, and they can transform to aggressive cancers. Surgery was an option to remove some of the tumors, but others were too large. “We tried everything we could think of, diet changes, eliminating sugar and dairy, but nothing stopped the tumors from growing,” Diane said.

The emotional toll on the family was heavy. At 5 years old, Alex’s older brother, Justinian, struggled to understand the disease. When doctors found a tumor on Alex’s brain, Justy asked if his little brother was going to die. “That was crushing,” Diane said. “We reassured him, but the fear was real.”

For years, Diane’s hope was channeled into advocacy and working with the Children’s Tumor Foundation. She threw herself into fundraising for research, running races, and rallying family and friends. “When you’re told there’s nothing you can do, you hit your ‘no way’ button,” she said. “There’s always something you can do. If there’s no treatment, then research has to happen, and research needs funding.”

When the FDA approved the MEK inhibitor selumetinib (Koselugo) as the first drug for NF1 tumors in 2020, the family decided the time wasn’t right to start Alex on treatment. Alex’s tumors were mostly cosmetic—easily covered with a shirt—and they didn’t want to risk anything that could potentially compromise his immune system during the pandemic.

But the tumors continued to grow and started to limit what Alex could do. “The one on his back became so large he couldn’t lie flat or do simple things like sit-ups in gym class,” Diane said. By then, the FDA had approved another MEK inhibitor, mirdametinib (Gomekli), in February 2025 that showed promise in shrinking NF1 tumors and improving quality of life.

Alex began treatment in July 2025. The regimen required 3 pills twice a day for 3 weeks, followed by a week off, which he tolerated remarkably well. “We were warned about side effects like skin infections, nausea, and hair color changes,” Diane said. “But Alex has had only mild stomach upset. Nothing that makes us think twice.”

The results were stunning. “We were told not to expect measurable changes for 6 to 12 months,” Diane said. “After one cycle, his tumor had already shrunk by a centimeter. That’s huge.” Today, Alex’s tumors continue to shrink, and the pain that once plagued him daily has all but disappeared. He still travels to New York every 3 months for scans, but life feels different now—lighter, more hopeful.

The biggest change is in Alex himself. “He’s more confident,” Diane said. “He’s running cross-country, something he couldn’t do before. He signed up for basketball for the first time in years. He doesn’t feel like the kid with tumors anymore.”

Alex is thriving. He loves music, teaching himself guitar and trombone, and playing in the school jazz band. He’s passionate about cooking, whipping up fried chicken, kale slaw, and Harry Potter-inspired butterbeer from his growing collection of cookbooks. “He’s fearless in the kitchen,” Diane laughed. “He’ll try anything.”

For Diane, advocacy remains central. Her family has raised close to a million dollars for research, and she urges policymakers to keep funding science. “When you fund cancer research, you’re giving life,” she said. “You’re not just helping kids survive, you’re giving them back their childhood. You’re giving families hope.”

Alex’s journey is far from over, but thanks to decades of research and a breakthrough therapy, his future looks brighter than ever. “We may not be able to cure him,” Diane said. “But we can make sure the life he has is full and joyful. That’s what research makes possible.”