New medicine could extend lives of terminally-ill cancer patients

This medicine may prevent cancer patients from losing their hair

ATX-101 is the name of the new cancer treatment. According to the press release, it has now been tested on 20 cancer patients who were terminally ill. All of these patients had tried other treatments before deciding on this one, which was still in the experimental stage. The trials were held in Australia. 

After six weeks, 70 percent of the patients were stable, according to the results of this stage. While 12 of them stayed on the medication and had stable conditions for up to 18 weeks, a woman took the medication for 17 months and remained stable for more than two years.

One of the study's goals was to see if the medication was toxic to cancer patients. According to the researchers, the treatment only affected stressed cancer cells and had no effect on other healthy cells in the body. This breakthrough may prevent cancer patients from losing their hair.

“Cancer cells are more stressed than other cells. However, they don’t die but continue to grow even when they are damaged. Conventional cancer treatment with chemotherapy puts more stress on the cancer cells so that the cells eventually do die. Chemotherapy affects all cells, including the normal ones, such as in the hair follicles, and thus affects the whole body with many side effects like hair loss,” explains Marit Otterlei, a professor of molecular medicine at NTNU.

The Phase 2 trials have started

Previous animal experiments demonstrated that the medicine could "keep cancer at bay and defeat it" in the lab. The testing trials have progressed to Phase 2.

During this stage, the team needs to demonstrate the effectiveness of the medicine. In the United States, patients with sarcoma, a type of connective tissue cancer, are undergoing Phase 2 trials.

“A lot of medicines might work but don’t make it through the development process. The pharmaceutical industry doesn’t take on any projects they can’t profit from. What we develop has to work slightly better than current treatments and preferably be cheaper to produce and have fewer side effects. Only then can an expensive development run pay off. It’s been a long run, and there’s still a long way to go,” said Otterlei.

According to the team, it took 18 years and more than 20 million Euros to develop this medicine.

The study was published in the journal Oncogene.

Study abstract:

Proliferating Cell Nuclear Antigen (PCNA) is a highly conserved protein essential for DNA replication, repair and scaffold functions in the cytosol. Specific inhibition of PCNA in cancer cells is an attractive anti-cancer strategy. ATX-101 is a first-in-class drug targeting PCNA, primarily in cellular stress regulation. Multiple in vivo and in vitro investigations demonstrated anti-cancer activity of ATX-101 in many tumor types and a potentiating effect on the activity of anti-cancer therapies. Healthy cells were less affected. Based on preclinical data, a clinical phase 1 study was initiated. Twenty-five patients with progressive, late-stage solid tumors were treated with weekly ATX-101 infusions at four dose levels (20, 30, 45, 60 mg/m2). ATX-101 showed a favorable safety profile supporting that vital cellular functions are not compromised in healthy cells. Mild and moderate infusion-related reactions were observed in 64% of patients. ATX-101 was quickly cleared from blood with elimination half-lives of less than 30 min at all dose levels, probably due to both, a quick cell penetration and peptide digestion in serum, as demonstrated in vivo. No tumor responses were observed but stable disease was seen in 70% of the efficacy population (n = 20). Further studies have been initiated to provide evidence of efficacy. Trial registration numbers: ANZCTR 375262 and ANZCTR 375319.