A pre-clinical study published in Nature this week indicates that a geranylgeranyl transferase inhibitor GGTI-2418, known as PTX-100, plays a key role in mitigating a new cancer pathway discovered by Professor Michele Pagano at New York University’s Langone Medical Center, in New York. 

In the study, Professor Pagano’s group demonstrated new details about the tumour suppressor gene, PTEN, which is defective in 30-60% of certain breast, brain and uterine cancers.

“When defective, PTEN cannot control a protein known as FBXL2, which is thought to be responsible for cancer growth in many patients,” said Professor Pagano.

Professor Pagano’s study also showed in mouse models that when administered with Prescient’s drug candidate PTX-100, plus photodynamic therapy, FBXL2 is “switched-off” allowing abnormal cells to self-destruct.

Therefore, patients whose tumours harbour defective PTEN may also be more likely to respond to a combination of PTEN and photodynamic therapy.

PTX-100 was developed by Professor Sebti, Chair of the Department of Drug Discovery at H. Lee Moffitt Cancer Center, and Chief Scientific Officer at Prescient Therapeutics, and NYU President Andrew Hamilton while he was at Yale University.

Professor Said Sebti said, “These findings have important translational implications, as patients whose tumours harbour defective PTEN may be more likely to respond to a combination of PTX-100 and photodynamic therapy. Furthermore, given that PTEN is known to also suppress the Akt tumour survival pathway, patients with PTEN defective tumours could respond to a combination of PTX-100 and an Akt inhibitor like PTX-200.”

PTX-100 has already been tested as a single agent in patients with advanced solid tumours in a Phase 1 trial and will be the focus of studies in rare haematological malignancies.

Source: BusinessWire