A new treatment option may finally be on the way for patients diagnosed with a rare cancer called adrenocortical carcinoma (ACC). 

ACC affects the adrenal glands, which produce hormones that control key body functions like metabolism, blood pressure, immune response and the body’s response to stress.   

Around 350 people each year are diagnosed with ACC in the UK. Treatment options are limited, but because the disease is so rare, recruiting enough people for clinical trials is a challenge.

A new phase 2 clinical trial called CLARITY (ISRCTN15479264) hopes to change that. It will test a new drug, developed by the biotech company Cytovation, in people with advanced ACC.

The trial will run as an international partnership between Cancer Research UK, the Norwegian Cancer Society and Cytovation, which could help researchers recruit more people than if it were run in a single nation.

The team have already opened the trial in hospitals in the UK and plan to expand into Europe, and hope it will soon lead to a new treatment option for people living with ACC.

The urgent need for a new treatment 

Treatment options for people with ACC haven’t really changed for more than 50 years.  

Currently, the only drug approved to treat ACC is called mitotane. Though the chemotherapy drug does help some patients, it can cause side effects and doesn’t work for everyone.  

For Jennie, a retiree from West Berkshire, the side effects were so severe, she had to stop taking the drug altogether. 

For Jennie, and others with ACC, a new treatment option is urgently needed. And now, with the announcement of the new clinical trial, there’s hope that one may be on the way soon.

Hope on the horizon 

The CLARITY trial will test a new drug called CY-101 (getacatetide).  

It’s made up of tiny dagger-like molecules (peptides) that poke holes in the cancer cells. As the damaged cells spill their contents, they signal to the body’s defence force (immune system) to investigate the source of the spill and clear the cancer cells.  

At the same time, the peptides also help the immune system stay focused on the tumour. Some cancer cells can send signals that distract or supress immune cells, but CY-101 is designed to block these signals so the immune system can recognise and destroy cancer cells.   

The researchers hope that this two-pronged approach could make CY-101 an effective treatment against ACC.  

And they have good reason to be optimistic. According to Professor Debashis Sarker, lead investigator of the study, CY-101 “demonstrated encouraging outcomes in people with solid tumours, including ACC” in a phase 1 clinical trial.  

Now, this new clinical trial will look specifically at how effective CY-101 is in treating ACC. 

For Ingrid Stenstadvold Ross, Chief Executive of the Norwegian Cancer Society, the trial’s announcement is exciting news.  

“With the opening of this trial, the treatment is finally reaching patients in the UK and soon also in Norway and other European countries.”

Moving research for rare cancers forward

“When you have a rare cancer, it can feel like you’re an orphan,” says Jennie. “You don’t have enough numbers for it to matter, so you don’t get as much research as other diseases might.” 

Cancers like ACC may be rare, but behind every diagnosis is a person in need of better treatment options. That’s why Cancer Research UK funds research for over 200 different types of cancers, including rare ones, to help get those treatments to people like Jennie faster.  

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

As the trial gets underway, Jennie will be watching its progress closely. 

“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.”