The effectiveness of a new anti-cancer compound called AOP208 is being evaluated for the first time in a clinical trial conducted by Spanish researchers. The first patient in the world to receive AOP208 did so within the framework of the phase 1 SERONCO-1 clinical trial, led by Dr. Irene Braña, oncologist at the Vall d’Hebron University Hospital and head of the Head and Neck Tumors Group. of VHIO.
“The treatment of the first patient with AOP208 in the trial represents a significant step towards understanding the safety and tolerability of the drug,” explained Dr. Irene Braña. “All the drugs that we test on patients in a clinical trial have previously demonstrated efficacy in preclinical models, but now we must demonstrate, first, that it is safe in humans and, second, that this antitumor activity also occurs in the patient,” he adds.
The Molecular Cancer Therapy Research Unit (UITM-CaixaResearch) of Vall d’Hebron is the only center in the world that is carrying out the phase 1 clinical trial SERONCO-1, which evaluates the new compound developed for the first time in patients. initially by Leukos Biotech, a spin-off of the Josep Carreras Leukemia Research Institute. This drug acts through a completely new mechanism of action in oncology, being classified as “first-in-class”.
Compound AOP208 is being evaluated in clinical trials in international collaboration with AOP Health. The SERONCO-1 trial, led by Leukos Biotech with support from AOP Health, is the first study within a global program. In 2022, both entities signed a collaboration and licensing agreement to advance the development of the drug, with the aim of benefiting patients with leukemia and other types of cancer worldwide.
A compound with potential in leukemia and solid tumors
AOP208 blocks the serotonin 1B receptor, a protein located on the surface of cancer stem cells, which in the oncological context adopts a particular conformation. This protein regulates metabolic pathways that, when blocked, could be key to treating the root of the disease. The compound is administered orally and was designed by Leukos Biotech, based on research by Dr. Ruth Risueño since 2012 at the Josep Carreras Leukemia Research Institute, with the support of the Josep Carreras Foundation.
“We wanted to find a new way to attack cancer stem cells, since they are responsible for the initiation, maintenance of the tumor and the appearance of recurrences, a serious problem particularly in some leukemias. Our work at the Josep Carreras Leukemia Research Institute demonstrated that the serotonin 1b receptor is key to this process in leukemic stem cells. As there was no suitable molecule that could block the receptor, at Leukos we implemented a chemical development program that allowed us to reach the AOP208 molecule. Now we are delighted to begin a clinical trial and take our compound to the next level for the benefit of leukemia patients and some solid tumors,” stated Dr. Risueño, who is also the scientific director of Leukos Biotech.
AOP208 acts on a cancer stem cell receptor that had not been explored in oncology, a pathway that could be relevant in leukemia and solid tumors such as breast and lung tumors.
Dr. Christoph Klade, Chief Scientific Officer at AOP Health, underlines the need to research new approaches in cancer therapy. “AOP208 targets a receptor on cancer stem cells that until now has not been the focus of cancer therapy, making it a first-in-class investigational drug in oncology. “This pathway may have a role in several types of leukemia and also in several solid tumors, including breast and lung cancer.”
Based on the safety and efficacy data that will be obtained from the SERONCO-1 trial in patients with advanced solid tumors, AOP Health plans to conduct a second clinical trial to evaluate AOP208 in patients with acute myeloid leukemia, a disease with a currently poor prognosis. .
