A drug for the treatment of metastatic kidney cancer, still incurable? This idea originated in 2008 “on the edge of the lab bench” by Dr. Gilles Pagès, now director of research at the National Institute for Health and Medical Research (Inserm) first class and team leader at the Cancer and Aging Institute (Ircan ) in Nice. And to complete this project, this Friday he will receive the prize of the Amgen France Fund for Science and Man, selected from 90 entries. Dr Pages returns with 20 minutes about the significant progress he has made with his team, doctors and chemists, in optimizing kidney cancer treatment, and even others.
What treatments exist today for metastatic kidney cancer?
Before 2008, a patient’s life expectancy was three months. Little by little we have reached an average of three years. We gained years and quality of life. First, by a drug that targets the compound VEGF, which makes blood vessels. It was first conceived for colon cancer, then lung cancer, breast cancer, and finally kidney cancer. It was a revolutionary treatment because it could extend life expectancy. But doctors realized that it was not effective for all patients, sometimes it even caused the opposite effect. Then there was immunotherapy, which also delayed death, but only in 20% of patients. In reality, there are as many tumors as there are patients.
Why is kidney cancer so difficult to cure?
You should know that kidney cancer is the cancer where most drugs have been developed in recent years. In fifteen years, there has been almost one approved treatment per year for this disease. It is a very active field because the disease is associated with certain mutations. For example, the VEGF protein uses hypoxia to grow. It then makes blood vessels to supply the cancerous tumor with nutrients and oxygen.
Where are you after fifteen studies on the subject?
After all these years of research, patent applications, strong collaborations, especially with the Nice Institute of Chemistry, we have developed a molecule to create a new drug. This molecule is redundant with the molecule that targets the VEGF protein and inhibits tumor growth. It is also effective for tumor models that do not respond to immunotherapy [qui vise à induire ou amplifier la réponse immunitaire anticancéreuse]† Our treatment, combined with the treatments that already exist, will hopefully cure this disease. But the mere possibility of turning it into a chronic disease and allowing patients to live is a source of immense pride.
Could this molecule be used for other cancers?
By being part of the Lacassagne center, which is a reference in Europe for the treatment of certain cancers, we discovered that our molecule could be effective for other diseases. Especially in eye melanoma, which develops in the eye and is very aggressive. The 30% of patients who escape radiation treatment can live off the drug we’ve made. It is also possible for cancers of the ENT sphere.
When can this medicine be used?
To speed up the process, we decided to launch our own start-up, Roca Therapeutics, in April 2021, which has collected all possible awards and funding from the academy and is even an i-Lab winner. † We are currently in the regulatory toxicology phases, which will be completed by the end of 2022 or even early 2023. Then we have to submit the treatment to the health authorities of the European Medicines Agency, with a first clinical trial in 2023. Depending on the validation, with no toxicity in a patient, we move to phase 2 with more patients than phase 3 and the blind study. In short, it’s tomorrow! (laughs)
What will the Amgen France fund bring you?
It is already the recognition of our colleagues because there were doctors and researchers on the jury. It is very important to see that our work is recognized by these professionals. But it is also essential to be recognized by the pharmaceutical industry, without which it is impossible to conduct clinical trials. Financially, it was crucial because it is difficult to get research funding these days. We must knock on all doors to prevent all those years of work for this program from ending now.
How did your program stand out?
The fact that we won this prize, among more than 90 registrations, is also due to our originality in offering personalized treatment according to the patient. We are entering a new era of molecular diagnostics. And then we tested and compared our molecules to developed compounds that had already been released, particularly at GSK. We were able to apply for a patent because ours were better. It is the result of an adventure sometimes interrupted by failures, but by collaborations between researchers, doctors and chemists. And this vicious circle may allow us to bring in our building block for the treatment of kidney cancer. Again, it’s a very big pride.