Spanish researchers design the first drug that inhibits a key gene in cancer

by time news

The MYC gene is a key gene in the development of cancer and is involved in multiple tumors, such as breast and prostate. But it seems that he already has someone to stop him.

A group of Spanish researchers has discovered a drug that targets this key gene. This drug has been able for the first time to inhibit the function of this gene in a phase I clinical trial. Until now, no other drug has been able to do this safely and effectively.

Presenting the preliminary results of the trial at the 34th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapy in Barcelona, ​​Elena Garralda, Director of the Unit for Early Drug Development at the Vall d’Hebron Institute of Oncology (VHIO) in Barcelona, ​​«MYC is one of the ‘most sought after’ targets in cancer because it plays a key role in driving and sustaining many common human cancers, such as breast, prostate, lung and ovarian cancer. To date, no drug that inhibits MYC has been approved for clinical use.”

VHIO scientists developed a miniprotein called OMO-103 that can enter cells and reach the nucleus. In experiments in the lab and in mice, they showed that it successfully inhibited MYC’s ability to promote tumor growth by blocking MYC’s role in controlling the flow of information for many common gene mutations found in cancer.

Starting in April 2021, Elena Garralda’s team, in collaboration with researchers from two other Spanish centers, enrolled 22 patients in a phase I clinical trial to assess the safety of OMO-103 and to see if there were any early signs of control. of cancer.

The patients had a variety of solid tumors, including pancreatic, bowel, and non-small cell lung cancer. All had already been heavily pretreated, receiving between three and 13 treatments previously.

The researchers took biopsies of the tumors at the start of the study and after three weeks of treatment to assess levels of MYC gene activity and other biological markers of cancer.

By October 10, 2022, eight of the 12 patients who underwent CT scans after nine weeks had stable disease and showed that treatment had stopped cancer growth.

It is still too early to assess the activity of the drug, but we are seeing the stabilization of the disease in some patients

Of these, two had pancreatic cancer, three had colon cancer, one had non-small cell lung cancer, one had sarcoma, and one had salivary gland cancer.

“It is still too early to evaluate the activity of the drug, but we are seeing the stabilization of the disease in some patients,” says Garralda. He adds that, “Surprisingly, one patient with pancreatic cancer stayed in the study for more than six months, his tumor shrank by eight percent, and there was a reduction in tumor-derived DNA circulating in the bloodstream.” . The patient with a salivary gland tumor has stable disease and is still on study after 15 months.”

Of similar opinion is Lawrence Young, Virologist and Professor of Molecular Oncology at the University of Birmingham (United Kingdom). “These are very exciting results, but it is still early days. Large clinical trials are required to determine whether the observed effects can be replicated in a larger cohort of cancer patients.”

Cancer, Young warns, is a complex disease and the best way to attack tumor cells is to use a “multiple approach, which is why combination therapies are the most effective.”

These are very exciting results, but it’s still early days

Lawrence Young

University of Birmingham

For Garralda, “the most exciting thing is that the biological markers show that we are successfully targeting MYC. Also, adverse side effects are mostly mild, which is important when we start thinking about the next steps and combining OMO-103 with chemotherapy or other therapies”.

The most common treatment-related adverse side effects were mild reactions to the intravenous infusion, including chills, fever, nausea, rash, and low blood pressure. Higher dose levels were associated with more infusion reactions, but were easily treatable. Inflammation of the pancreas was the only dose-limiting reaction, something that occurred in one patient.

In conclusion, Garralda points out that “OMO-103 is the first MYC inhibitor to successfully complete a phase I clinical trial and be ready to move on to a phase II trial.”

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