Children's Hospital of Philadelphia doctor making strides in finding cure for "holy grail of childhood cancers"
PHILADELPHIA (CBS) -- A doctor at Children's Hospital of Philadelphia (CHOP) is making huge strides in finding a cure for some cancers. One of her patients was indeed cured, and she's hoping that there are more success stories in the near future.
"The science is taking us now to a place where we can start to think about what's called drugging the undruggable," Dr. Yael Mossé said.
Mossé is working on groundbreaking science at the Children's Hospital of Philadelphia. The once undruggable neuroblastoma -- a rare cancer of nerve cells -- is now responding to treatments developed by the team at CHOP.
"This is a game changer," Mossé said. "This is going after the holy grail of childhood cancers."
The experimental drug Lorlatinib has worked to stop cancer in Philip Steigerwald.
He had the same genetic mutation as Alex Scott, the little girl who started the Alex Lemonade Stand Foundation, which is funding the research.
"The impact that her foundation that she started has had on children like Philip, the impact is immeasurable," Mossé said.
Alex's Lemonade Foundation is now funding the Crazy 8 Initiative.
CHOP and research centers around the world are collaborating on targeting a second neuroblastoma gene called MYCN -- the protein that fuels the cancer.
"Now we have incredible technologies to be able to take a disordered protein, dock it to something else that allows the protein to now have a structure," Mossé said, "and then to make what we call a degrader that actually just takes this gene and melts it."
It's still being researched, but the hope is it will eventually be able to treat more patients with deadly cancers.
"Our goal is to cure patients," Mossé said, "and to minimize side effects."
Mossé has dedicated her life to taking care of sick children and researching new ways to treat once-incurable cancers.
"It's humbling," Mossé said "It's exciting. It's a bit onerous. I feel in many ways a heavy weight on my shoulders to bring the science to life."
A mission started by Alex, who lost the battle herself but it lives on through her foundation with science and research, changing the world and saving lives.
