AOH1996 has been developed over two decades to target a protein found in all forms of the disease
A new “cancer-stopping” drug has been found to “annihilate” solid cancerous tumours in early stage studies.
The chemotherapy drug leaves healthy cells unaffected, scientists said.
The AOH1996 drug is named after a child – Anna Olivia Healy, born in 1996, who died when she was only nine after being diagnosed with a rare childhood cancer neuroblastoma.
Prof Linda Malkas and her team spent two decades developing the drug that targets a protein in all cancers, including the cancer that led to Anna’s death.
The protein, proliferating cell nuclear antigen (PCNA), was once thought too challenging to aim targeted therapies at.
PCNA in its mutated form encourages tumours to grow by aiding DNA replication and repair of cancerous cells.
Prof Malkas and her team at the City of Hope in California, one of the United States’ largest cancer research and treatment organisations, said the targeted chemotherapy appears to “annihilate” all solid tumours in preclinical research.
Selectively kills cancer cells
AOH1996 was tested in more than 70 cell lines and was found to selectively kill cancer cells by disrupting the normal cell reproductive cycle, but it did not interrupt the reproductive cycle of healthy stem cells.
Pre-clinical studies suggest the drug has been shown to be effective in treating cells derived from breast, prostate, brain, ovarian, cervical, skin and lung cancers.
The drug still needs to go through rigorous safety and efficacy testing and large-scale clinical trials before it can be used widely.
The first patient received the potentially cancer-stopping pill in October with the phase one clinical trial still ongoing and expected to last for at least two years.
Patients are still being recruited to the trial.
Researchers are also still examining mechanisms that make the drug work in animal studies.
‘Like snowstorm that closes airline hub’
Prof Malkas said: “PCNA is like a major airline terminal hub containing multiple plane gates.
“Data suggests 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.
“Our cancer-killing pill is like a snowstorm that closes a key airline hub, shutting down all flights in and out only in planes carrying cancer cells.”
The professor called the results “promising” but made clear that research has only found AOH1996 can suppress tumour growth in cell and animal models.
Long Gu, the lead author of the study, said: “No one has ever targeted PCNA as a therapeutic because it was viewed as ‘undruggable’, but clearly City of Hope was able to develop an investigational medicine for a challenging protein target.”
The study, titled “Small Molecule Targeting of Transcription-Replication Conflict for Selective Chemotherapy”, was published in the Cell Chemical Biology journal.
TheIdea – The Innovation Agency 