Benjamin Besse, President-Elect of EORTC and Head of Clinical Research at Gustave Roussy, shared a post on LinkedIn:
“Ten years ago, I took care of a 19‑year‑old man with a devastating disease that took his life in less than 3 months: a NUT carcinoma. He was at Gustave Roussy to receive a BET inhibitor – the only therapy targeting the core alteration driving this disease: a NUTM1 gene alteration. Because he practiced sport intensively, his family transformed part of their grief into a charity initiative, organizing a yearly sports event to raise money for NUT carcinoma. Our charity team worked with the family with kindness, attention, and professionalism. Each year, they came to me with a check. Just humans that want to help.
The money accumulated. It wasn’t a lot, but every euro mattered. Every year, I told myself: I should do something. I had ideas, but nothing clearly impactful. fabrice andre always says that “research is mostly humans working together.” And Gustave Roussy has this magic: a small talk in an elevator can spark a project, because everyone works there exactly for that. Just humans that want to help.
The first birabresib trial, a BET inhibitor, was partially conducted at Gustave Roussy and published by my friends Jean-Charles Soria, MD, PhD and christophe massard. Among 11 patients with NUT carcinoma, 3 had strong tumor reduction and 3 stable disease. With Maria Virginia Sanchez Becerra, we started a European based registry to better understand treatment patterns and expectations. Yes, it is a strong efficacy signal: disease control in more than half of the patients, sometimes prolonged – significant for such a fast‑growing cancer. But NUT carcinoma is ultra‑rare (10–20 cases a year in France, a 69‑million‑inhabitant country).
Drug development stopped. Some US trials exist with other BET inhibitors (led by the great Jia Luo), but nothing elsewhere. Maxime Annereau, one of our pharmacists, told me: “With my boss Bernard Do, I can make pills of a BET inhibitor for you … find a drug we can use for free.” So with Virginia, we called the Japanese company holding the birabresib patent, expecting nothing. After 30 minutes, they said: “Use it as you want.” Their focus had moved on. Sometimes money doesn’t rule everything. Just humans that want to help.
Next step was convincing ANSM Agence nationale de sécurité du médicament et des produits de santé to grant early access and reimburse birabresib. With Maxime, Bernard, Virginia, patients and families, we built the dossier, with the unwavering and decisive support of our CEO Fabrice Barlesi. The drug was develpped in six months and all steps to qualify it were completed. At every stage, ANSM staff were incredibly helpful – the opposite of what you expect from a bureaucratic institution. Just humans that want to help.
On Nov 26th, I prescribed my first birabresib pills with Virginia and Maxime at my side. One of the most incredible moments of my life. Just humans that want to help.
Read: Exceptional temporary use protocol for Birabresib for adult and paediatric patients with NUT carcinoma.”
More posts from Benjamin Besse on OncoDaily.