My daughter was the first pediatric cancer patient in a trial. She died at 16, but the drug has been approved for other children.

Back view of patient woman sitting on bed in hospital ward
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  • Nathan Traller's daughter, Nathalie, was diagnosed with cancer in seventh grade.

  • There were no treatment protocols for her cancer, so she fought to be included in a clinical trial.

  • This is Traller's story, as told to Kelly Burch.

This as-told-to essay is based on a conversation with Nathan Traller. It has been edited for length and clarity.

During spring break when she was in seventh grade, my daughter, Nathalie, complained of a headache. I specifically remember saying, "It's not anything serious." But it was.

Later that spring, Nathalie went to the doctor for something minor, but the doctor was concerned. They ordered a chest X-ray, which showed a mass near her heart. We were immediately admitted to the children's hospital's cancer floor.

There, Nathalie was diagnosed with alveolar soft part sarcoma, a rare form of cancer that occurs mostly in young people. The thing about ASPS is that it can grow and spread without making trouble to alert you to it. By the time Nathalie was diagnosed, she had tumors in her brain, lungs, and chest, near her heart. She had stage 4 cancer.

After a meeting with her oncologist, Nathalie and I went out to lunch. She said, "Daddy, what did the doctor say we could do?" My heart lurched because there was no treatment protocol for ASPS, which doesn't respond to chemo. It was so hard to digest that there was nothing in the books about treating ASPS — you don't expect to hear that in this day in age.

I felt I had to find a cure for Nathalie

Once doctors told us there was no standard of care for treating ASPS, I felt that the burden of treating Nathalie shifted, in some way, onto my shoulders. My wife and I pored over medical journals. We enrolled Nathalie in a clinical trial, but the drug didn't work for her. Meanwhile, Nathalie was having surgeries to remove tumors that had become painful or dangerous, but there were always more popping up.

In my research, I found that some cancers similar to Nathalie's were responding to immunotherapy drugs. There were clinical trials for these treatments, and Nathalie would have been able to enroll in them if only she were 18.

With every roadblock we encountered, I drilled down to the reasoning for it, and it never held up. Doctors had information on safe dosing, and there was no scientific reason to exclude people under 18. In an effort to keep patients safe, the system had inadvertently built barriers for pediatric patients. If you were the youngest, you were the last in line for science's latest innovations.

Our family contacted government officials, drug companies, and the press. All we wanted was access to these trials, which were Nathalie's only hope.

After 8 months, Nathalie was given access

After one surgery to remove a tumor, Nathalie was in the intensive-care unit. But she wanted to walk. The nurses gave their blessing, and our whole family trailed her around the atrium of the hospital. That was always Nathalie's attitude: "What can we do next?" As her parents, we had to keep up.

It paid off. After we advocated for eight months, including many polite but firm conversations with drug companies, a representative from the drug manufacturer Genentech reached out. Through the company, Nathalie was given a compassionate-use allowance that would let her get the experimental treatment.

Nathalie was thrilled to be the first pediatric ASPS patient to take this drug. And it was promising — we could see a tumor on her scalp shrink, and she had minimal side effects. For the first time ever, her cancer was responding to therapy, and we were incredibly grateful to have Nathalie on the drug for about a year.

Unfortunately, the cancer had a head start. Nathalie died in 2015. She was 16.

I continue to fight for better treatments

Even as we endured the absolute horror of losing a child, our family knew our work wasn't done. We continue to advocate for more research on pediatric cancers and access to potentially lifesaving treatments for young patients.

After Nathalie took the drug, Genentech started a pediatric clinical trial. The company told me that wouldn't have happened without Nathalie's work. Her tenacity also changed how clinical trials include people under 18.

In December, Genentech announced Food and Drug Administration approval for the drug that had helped Nathalie. When a representative from the company called me, I immediately had a flashback to that moment when Nathalie asked me what we could do. Now when other families face an ASPS diagnosis, they'll at least have an answer for their children; there's now a treatment protocol.

Nathalie wanted to try everything, right until the end.

This approval doesn't bring Nathalie back, but it speaks to who she was. She wanted access to the best science could offer her. Now she's helped ensure that for other kids.

Read the original article on Insider