US scientists have reported an ‘experimental drug’ that can be injected directly into tumours made aggressive breast and skin cancers disappear in a small trial.

The treatment, described as ‘an enhanced CD40 agonist’ was successful in its first trial of 12 human participants: six people saw their tumours shrink, while two saw their cancer go completely into remission ‘while not causing serious side effects’ (one had melanoma, the other breast cancer). The researchers noted not only did their tumours shrink, but tumours in the rest of the body disappeared.

In their human phase 1 study, reported in the journal Cancer Cell, researchers from Rockefeller University tested a new CD40 antibody drug, 2141-V11, designed to shrink tumours and reduce side effects.The team focused on immunotherapy (using the body’s immune system to attack cancer) – and, specifically, the CD40 agonist antibody drug class, a type of monoclonal antibody treatment that works by activating the CD40 receptor and causing an immune system response.

But because research on CD40 ‘has not been successful in human trials in the past and has caused serious side effects’, the scientists created an engineered form of CD40 called 2141-V11 ‘designed to improve the issues with CD40 and reduce systemic inflammation and liver toxicity’. And they injected the treatment directly into the tumour instead of administering it intravenously.

For the human study, everyone in the group of 12 participants (aged 42-89) had metastatic cancers including melanoma and breast cancer. They received injections of 2141-V11 into their tumours every 3 weeks, with an increased dosage each time.

Throughout the treatments, researchers monitored the side effects and bloodwork of participants; they were especially concerned with platelet count and liver enzymes since these would indicate a treatment-related adverse event (TRAE).

By the end of treatment, 10 participants experienced adverse events and seven experienced TRAEs. However, none experienced TRAEs higher than grade 3, and the researchers considered the drug ‘well tolerated’.

Study author Dr Juan Osorio told medicalnewstoday.com: ‘We look forward to the results of several ongoing phase 2 studies nationwide, targeting difficult-to-treat cancers, such as malignant gliomas, bladder cancer and prostate cancer.’

SOURCECancer Cell Journal
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