The journey from a potential cancer cure in mice to a viable treatment for humans is a long and arduous one, filled with both promise and pitfalls. This story follows the path of biologist Laura Soucek, who made a groundbreaking discovery three decades ago, and the challenges she faced in translating her research into a safe and effective drug for humans. But here's where it gets controversial...
In 1996, Soucek, then at the University of Rome La Sapienza, designed a mini-protein capable of binding to Myc, a protein that coordinates the proper division of a cell. This mini-protein, named Omomyc, was a potential game-changer in the fight against cancer. However, the road to human trials was fraught with challenges. Soucek's journey illustrates the extremely long, and in most cases insurmountable, gap between a cancer cure in mice and an effective and safe drug for humans.
In 2008, while working at the University of California, San Francisco, Soucek demonstrated that Omomyc was capable of eliminating lung cancer in mice, with only mild adverse effects. This achievement was published in the journal Nature, one of the world's leading scientific publications. However, the same journal rejected Barbacid's study, which was ultimately published in PNAS, thanks to Barbacid's membership in the U.S. National Academy of Sciences.
Barbacid's so-called "triple therapy" consists of three experimental drugs, and his promising results on mice have sparked hope among patients worldwide. However, Soucek and others have warned against creating false expectations. The biologist emphasizes that the cocktail used by Barbacid in mice does not exist for humans, and the journey from mouse to drug is fraught with years of work and no guarantee of success.
The European Society for Medical Oncology has also clarified the numerous obstacles ahead, and the Pancreatic Cancer Association has urged caution following Barbacid's announcement. The development of a safe and effective drug for humans takes a standard timeline of about 15 years, with four or five years to compare thousands of compounds in the laboratory, another year to test hundreds of the most promising molecules in animals, and between five and seven years to study a dozen candidates in humans.
Despite the challenges, Soucek's work with Omomyc has been recognized, and she has received a grant of €2.5 million from the European Research Council to continue the development of the drug. Peptomyc, the company she founded, has secured €31 million in private capital and another €11 million in public funding. However, the company does not yet have any drug candidate, and the future of Omomyc remains uncertain.
The story of Laura Soucek and her journey from a potential cancer cure in mice to a viable treatment for humans is a cautionary tale about the challenges of translating scientific discovery into clinical practice. It highlights the importance of honest information and the need to manage expectations in the face of promising but uncertain results.