New drug for rare cancer could offer for hope for hundreds with limited options

Patients with a rare and aggressive cancer with “limited” treatment options could soon benefit from a new drug after a study was launched to test the effectiveness of the treatment.

Scientists have said that they are “excited” about the potential of a new treatment for advanced adrenocortical carcinoma (ACC), which affects the adrenal glands, called CY-101 (getacatetide) – an immunotherapy made by biotech company Cytovation.

The treatment has a “dual function” and is designed to harness the body’s immune system to selectively target and destroy cancer cells, while inhibiting a key driver of immunotherapy resistance in cancers such as ACC, Cancer Research UK (CRUK) said.

It works by converting immune-resistant “cold” tumours into immune-active “hot” tumours by stimulating local immune activation directly within the tumour micro-environment, it added.

CRUK, which announced the new global trial alongside Norwegian Cancer Society and Cytovation, said the Phase 2 study, known as Clarity, will assess the safety and efficacy of the treatment.

The trial operates in seven sites across the UK, with further sites planned across the UK and Europe.

The trial aims to recruit 41 people aged 16 years and over with ACC who have received at least one but no more than two previous lines of treatment for their cancer.

It is expected to run to July 2032.

One ACC patient said that “any new drug is extremely welcome”.

Retiree Jennifer Rayner was diagnosed with ACC in 2018 with scans revealing a 19cm tumour wrapped around her spleen and left kidney.

After surgery, the 67-year-old from Newbury, West Berkshire, was put on mitotane but suffered side effects including vomiting and migraines.

Ms Rayner, who is involved with the charity ACC Support and helping others with the same diagnosis, said: “When you have a rare cancer, it can feel like you’re an orphan. You don’t have enough numbers for it to matter, so you don’t get as much research as other diseases might.

“The only drug that has been approved is terribly toxic, and it doesn’t always work.

“Some people tolerate it beautifully, but others get all kinds of side effects.

“Everybody’s body is different, so any new drug is going to be extremely welcome.

“It’s great to see this new trial has started, and I look forward to seeing the results and what they might mean for people like me.”

Around 350 people in the UK are diagnosed with ACC every year, according to CRUK.

And around 130 patients die from the disease each year.

CRUK said the new study follows a phase 1 trial which had “encouraging” outcomes in people with solid tumours, including ACC.

Dr Lars Erwig, director of the Centre for Drug Development at Cancer Research UK, said: “People living with ACC deserve better treatment options and we are pleased the Clarity study has reached this exciting stage.

“We thank our partners, Cytovation and the Norwegian Cancer Society for their ongoing collaboration to help bring us closer to a world where people with rare cancers live longer, better lives, free from the fear of cancer.”

Chief investigator of the study, Debashis Sarker, professor of experimental oncology at King’s College London and consultant medical oncologist at Guy’s, St Thomas’ and King’s College Hospitals, said: “Advanced ACC is a rare and highly challenging cancer with very limited treatment options and clinical trials available for patients, leading to poor outcomes.

“This makes the dosing of the first patient in a new clinical trial for advanced ACC a very important milestone.

“We are excited by the scientific rationale and potential of CY-101 and deeply grateful to the patients and clinical research teams participating in this trial.”

Dr Lars Prestegarden, chief executive of Cytovation, added: “Reaching this milestone is an important step for patients with ACC, where treatment options remain very limited.

“The activity we observed in the patients with ACC enrolled in our Phase 1 Cicilia trial gave us strong reason to advance into a dedicated Phase 2 study in this disease.”

Jo Grey, cheif executive of the Association for Multiple Endocrine Neoplasia Disorders (AMEND), said: “ACC is a very rare form of cancer with extremely limited treatment options.

“AMEND therefore welcomes the Clarity study and will follow its progress with great interest and hope whilst providing information and support to our ACC community.”