The first patient

I was hesistant to cover the impressive study of GDC-0449 because it's an early phase trial. But after hearing the work that these cancer researchers have done, I was incredibly moved by a number of things, including the success of the investigational drug and the courage of the first human patient to take it.The study looked at a rare type of inherited basal cell carcinoma called Gorlin's syndrome that affects maybe a couple thousand people in the United States. GDC-0449 is a drug that inhibits the hedgehog pathway, the first and only hedgehog-targeted drug that is currently in human clinical trials. (You can learn more about the hedgehog pathway here.)The clinical response of the drug appears to be remarkable. The session presented at the Plenary yesterday detailed the success of the drug in patients with basal cell nevus syndrome, a condition that presents as hundreds of lesions all over the body, including the face. Dr. Ervin Epstein, at the Children's Hospital of Oakland Research Institute and who presented the phase 2 trial results, remarked that one of the patients in the trial no longer went out in public because people would stare at him. The before and after photos Epstein presented showed visible evidence of the disease disappearing with the drug. The bad news is the condition returned once treatment was stopped, and the side effects could be uncomfortable to say the least. A number of patients dropped out of the trial due to those adverse events, which could include muscle cramps, hair loss and taste and weight loss. Those side effects also make it illogical to use the drug with common basal cell carcinoma, which presents as one or two lesions and can be treated with simple surgery alone. After the presentation of the phase 2 results by Epstein, Dr. Dan Von Hoff, who worked on the original phase I study, went into the background that led to Dr. Epstein's study. He showed imaging scans of a man who had metastatic basal cell carcinoma--his name is Jerry. Jerry was no unidentified patient number. Dr. Von Hoff recalled meeting Jerry for the first time, shuffling in with his oxygen tank because the metastases in his lungs made it difficult to breathe.Jerry did not have Gorlin's syndrome, though. While BCC is a very common type of skin cancer, it very rarely metastasizes. However, Jerry had developed BCC metastases in his lungs, spine, liver and hips. He entered Dr. Von Hoff's phase 1 trial three years ago and became the first human to take GDC-0449. We've covered the ethics of clinical trials before, and they are quite different than the phase 3 trials CURE typically reports on. Phase 1 trials usually only test the safety and dosage of a drug, and patients who enroll are often those who have few or no treatment options. To say that Jerry's experience is not common would be an understatement, but it truly speaks to the power of what cancer research can accomplish. Jerry has been on the drug for three years and has improved incredibly--no more oxygen tank and he's even going to the gym now. At the end of his presentation, Dr. Von Hoff included a photo slideshow of Jerry, complete with recent photos of Jerry and his wife, Jerry and the clinical trial team with the cake he brought in as a thank you, and his personalized license plate thanking the team for essentially saving his life. Dr. Von Hoff showed these slides not only to show the audience of cancer researchers that what they do matters to people like Jerry, but to also thank Jerry for having the courage to be the first patient.