A new drug, discovered and developed in the Triangle, shows promise in treating cancer.
It's a story CBS North Carolina first reported on last year. At the time, the drug had only been tried in petri dishes and mice, but now a little girl in Philadelphia has become the first person with cancer to use the experimental drug.
Philomena Stendardo, 8, was diagnosed with diffuse intrinsic pontine glioma last September. DIPG is a rare, aggressive brain tumor. The tumor wrapped around her brain stem is inoperable.
Her mother, Mina Carroll, said her daughter received radiation that initially helped, but Philomena's health began declining in March to the point that her right side was paralyzed.
"We were pretty much told that unless a clinical trial comes up, there's nothing else they could do for us," Carroll said.
Miami father Raymond Rodriguez-Torres knew how the Stendardos felt.
He lost his 10-year-old daughter, Bella, to cancer four years ago.
After her death, he created the "Live Like Bella Foundation" to support childhood cancer research.
When Rodriguez-Torres learned North Carolina State University scientist Dr. Kenneth Adler discovered a drug that stopped cancer cells from spreading in the laboratory, he sent samples of Bella's tumor to Adler's lab to see if the drug worked on her cancer cells.
"It was bittersweet in the sense it was joy it was, 'oh my God. It worked,'" recounted Rodriguez-Torres. "At the same time, for my wife and my other daughter and I, it was 'how did we not find this? How did we not know about this?'"
The discovery came too late for Bella Rodriguez-Torres, but maybe not for other children with cancer.
So, Rodriguez-Torres urged Adler and Biomarck, the Durham company developing the drug, to make it available for compassionate use in children.
That means once the FDA approved the drug for clinical trials in adults, some would also be available, as a last resort, for kids with certain cancers.
The Stendardos didn't know anything about the research or the experimental drug when they contacted the "Live Like Bella Foundation" for help.
Rodriguez-Torres told them about the drug, now known as Bio-11006, just after it had been approved by the FDA for clinical trials in adult lung cancer patients.
Those trials had not yet begun, but the drug had been tested in the lab on the same kind of cancer Philomena is fighting, and the "Live Like Bella Foundation" helped her get it under the terms of compassionate use.
Philomena's family said Biomarck and the FDA approved the request within a matter of days and the Rodriguez-Torres family traveled to Philadelphia to see Philomena her receive the first dose.
"Philomena is patient 001. The first patient in the entire world to receive this medication for cancer," noted Philomena's mother.
Within a couple of weeks, her parents said they saw signs of improvement in Philomena.
"She gets a little bit stronger each day," Carroll said. "She did just finish her second round of small, like palliative radiation, but I believe it's 006 that's making her better."
Philomena can now sit up on her own, walk with assistance and move her right side.
Her family said she is also able to speak and think much more clearly than she was before she received the drug.
Adler said it's too soon to say whether the drug he discovered is responsible for Philomena's progress, but he wanted to meet the little girl for himself.
He traveled to her home in Philadelphia and administered the medication, which she inhales twice a day. As Adler held the medication to her lips, her family prayed.
Her parents said they rely on their faith to get them through Philomena's illness.
Back at his N.C. State lab where he first began testing this drug on cancer cells in petri dishes five years ago, the scientist described the experience as "humbling."
"This is what we all do research for. We want to end up helping people who are sick, and here is a child who is sick and not only am I helping, I'm actually physically helping her get the medication," Adler said.
But Adler warns it's still not clear whether this drug is actually helping Philomena.
"This is just a single patient. Perhaps it was the other therapy she was getting coming to fruition I don't know," Adler said. "It works great in mice. Humans are not mice."
No one knows what the future holds for Philomena, but for now, her family is grateful.
"Thank you to 'Live Like Bella' and Dr. Adler. To all the people who have invested in this project," Carroll said. "If they didn't have the support and the people who believed in them then we wouldn't be here."
Her father, Mark Stendardo added, "We're hopeful. We believe."
While the drug 11-006 is being used under "compassionate use" in Philomena's case, it has received FDA approval for clinical trials in adults with lung cancer.