1) Mission Massimo in Australia
|Leah told me she is working on the sequel to this book: "Fixing the Code". I absolutely loved "Cracking the Code", this is my favorite book for 2015.|
Two major differences here: Massimo's story was written by a Doctor, Leah Kaminsky. As she saw Massimo's disease (her patient, she is a pediatrician) was beyond the realm of her competence, she decided to write about the father's story. Massimo's father is actually at the center of the book: he is the envelope pusher, the pioneer. He got a diagnostic for his son where nobody else could. Leah tells about the diagnostic odyssey and reflects on some of the ways that conventional medicine and genomic medicine could find common ground.#PWSYN France: boy w/ #leukemia found neither @cellectis (T Cells) nor doctors can help -> #crowdfunded Rx in the US https://t.co/sRUsz4oDe7— CATHERINE COSTE (@cathcoste) December 11, 2015
I will meet with Leah, Massimo and his parents in Melbourne in a few days. How can we build synergies to take advantage of what pioneer patients have achieved and democratize genomic medicine? Surgeons and doctors should attend congresses about genomics; not only geneticists. And geneticists should attend medical congresses to show what can be done. Robert Darnell PhD at the NY Genome center showed his work about genomic precision medicine at the Academy of Medicine in NY. See the Genomics and Health Care Conference, two months ago (link to videos). Doctors at the Academy of Medicine had a lot of questions for Darnell, they showed real interest.
The French case is different. Matéo did get a diagnostic, acute leukemia, but there was nothing doctors could do that was helpful. If he does not get a new treatment (CAR-T cells) he will die in a couple of months. French biotech Cellectis was not helpful. It turns out they do have a trial for the new treatment but due to regulation matters, things are being slowed down to such an extent that by the time the new drug trial starts, Matéo will be dead. So the parents looked for solutions, found this innovative treatment in the US and started a crowdfunding action (treatments are 10 times more expensive in the US than in France) to speed things up and save their son's life as no doctors seem to be in charge. The contrast between the media uproar (Matéo is now a star on French TV) and the extreme apathy of French medical establishment is mind boggling.
While doctors are burying their heads in the sand
Matéo is taking the Future of Medicine in his hands.