New cancer hope as pill kills tumors – thanks to little girl

Medicine

By Marc Lallanilla

August 2, 2023 | 17:45

Cancer has a powerful new enemy – and it is driven by a 9-year-old girl with an unforgettable smile.

Researchers have developed a drug containing a molecule called AOH1996 that “appears to wipe out all solid tumors” in preclinical research – while leaving healthy cells unharmed.

The drug AOH1996 is named after Anna Olivia Healey, a cancer patient from Indiana who was born in 1996.

“I knew I wanted to do something special for that little girl,” said Linda Malkas of City of Hope in Duarte, Calif., a leading cancer research center, in a statement obtained by The Post.

“She died when she was only 9 years old from neuroblastoma, a childhood cancer that affects only 600 children in America each year.”

Malkas met Anna’s family just before their little girl died in 2005 after a devastating battle with cancer. Neuroblastoma starts in very early forms of nerve cells, which are most often found in an embryo or fetus, according to American Cancer Society.

“I met Anna’s father when she was terminal … he asked if I could do something about neuroblastoma and he wrote a check to my lab for $25,000,” she said. “That was the moment that changed my life – my fork in the road.”

AOH1996 represents the culmination of two decades of research for Malkas and City of Hope.

The new drug works by targeting a protein called PCNA, or proliferating cell nuclear antigen. In its mutated form, PCNA helps cancer tumors thrive and grow.

“PCNA is uniquely altered in cancer cells, and this fact allowed us to design a drug that targeted only the form of PCNA in cancer cells” while leaving healthy, normal cells untouched, said Malkas, 68.

“Our cancer-killing pill is like a blizzard that shuts down a central airline hub and shuts down all flights” — but “only in planes carrying cancer cells.”

Their AOH1996 drug is currently in a phase 1 clinical trial at City of Hope. In previous tests, AOH1996 selectively killed cancer cells by successfully interrupting the cell’s reproductive cycle.

Young Anna’s fatal battle with neuroblastoma was also that inspiration for the ANNA Foundation (Anna Needs Neuroblastoma Answers), a charity that has raised more than $400,000 for neuroblastoma research since 2002.

“My wife and I thought there was no way to thank friends, family and strangers for their outpouring of love and support for our daughter. No way before the establishment of ANNA,” wrote Anna’s father, Steve, 57 the family website, which he runs with her mother, Barb Healey, 56.

“Through awareness and financial support from people like you, we will be able to pass on the same love and concern to other families in our situation,” he added.

Because AOH1996 killed cancer cells in several cancer cell lines, it gives hope that the drug may one day be used to treat breast, prostate, brain, ovarian, cervical, skin and lung cancers.

“No one has ever targeted PCNA as a therapeutic agent because it was thought to be ‘inducible,'” said Long Gu, associate professor at City of Hope and lead author of the study. published in Cell Chemical Biology.

“Now that we know the problem area and can inhibit it, we want to dig deeper to understand the process of developing more personalized, targeted cancer medicine.”

An additional benefit of AOH1996 is its ability to make cancer cells more vulnerable to agents that cause cellular DNA or chromosomal damage, such as the chemotherapy drug cisplatin.

This means that the new drug may one day be a useful tool in combination therapies and in the development of new chemotherapeutics.

“We were too late to help Anna, but we could help others like her,” Malkas said. “I always say when you see me that there is a little 9-year-old girl sitting on my right shoulder. She is my touchstone.”