Doctornauts Barry Marshall and Lukas Wartman: Living Proof of the Urgent Need for the Doctornaut Act
In my first book, Drug Discovery, The Pending Crisis, published way back in 1972, I wrote, “Our present system of drug discovery is almost designed not to cure the great diseases that confront us. There is no doubt that many will be cured in the distant future, but it is unfortunate that many of us must wait.” In the book I pointed out that one critical reason that drug discovery was and would be in the future dramatically inhibited was due to governmental barriers to conduct clinical studies. Penicillin and insulin were not medically discovered in scientific laboratory studies but in patient studies. Block that step and you block medical discovery. Unfortunately, history has proved me correct. Ask yourself, “With all our modern marvelous technology, when was the last cure?”
Later on, in order to reduce the barrier to clinical research, I proposed the Doctornaut Act which would permit doctors or doctornauts to volunteer to participate in clinical studies much more easily than non-doctors (www.fimdefelice.org). This would immediately open the doors for more potential medical breakthroughs to be tested resulting into the discovery of superior therapies and cures. For a number of reasons we have a cultural blind spot regarding the importance of clinical studies, and, in fact, view them with suspicion, and I have run into stone walls trying to educate health policy makers and the media how doctornauts can delay death and reduce patient suffering on a wide scale. There was one exception. Physician and former Senate Majority Leader Bill Frist agreed with the doctornaut concept and had a preliminary draft of the Doctornaut Act circulated. He also ran into a stone wall, and it was cast into the dust pin of current history- very bad news for patients, indeed.
Not too long ago, Australian physician, Barry Marshall along with his pathologist colleague, Dr. J. Robin Warren, who discovered in gastric ulcer patients an unusual bacterium later named, H. pylori, worked together to follow the unheard of possibility that a microbe may be the cause of gastric ulcers. When Dr. Marshall tried to gather support both for his idea and to conduct a clinical study, he, like Senator Frist, also ran into a stone wall. So the good doctor decided to experiment on himself. He performed a biopsy on one of his patients, isolated the H. pylori bacterium, placed into a broth to ferment and swallowed it himself. He then experienced serious symptoms and proved that his bacterium is a cause for gastritis, gastric ulcer and offering the promise to prevent gastric cancer. He won a Nobel Prize for this historic clinical study.
Recently, one of our great medical writers, Gina Kolata, wrote a fascinating and beautiful article featured on the front page of the New York Times regarding how physician Lukas Wartman was in the final stages of acute lymphoblastic leukemia and how his colleagues came to the rescue. His colleague, Dr. Timothy Ley, gathered his team of gene experts and told them that they had come up with a creative treatment for their friend soon. He told them, “It’s now or never. We will only get one shot.” They explored the cancer cell and healthy genomes using a technique known as genome sequencing and discovered there was a normal gene in the leukemic cells responsible for the production of a protein which was fueling the tumor’s growth. And, both luckily and happily, there was an approved drug already available for the treatment of late-stage kidney cancer that could be effective in controlling the gene’s activity. Dr. Wartman volunteered for the study. He received the drug and responded dramatically. In addition to his response, it added a boost to gene sequencing research where the genes and not the tumor cells themselves are the targets for therapy.
It took only two doctornauts, one healthy and the other at death’s door, to make possible two major discoveries. There are approximately 800,000 physician doctors in the United States. Let’s assume the Doctornaut Act was passed by Congress and only 10 percent of volunteered to be doctornauts which would make 80,000 available for clinical trials! Now use your imagination of the potential discovery of new medical breakthroughs that could follow- very soon. It’s breathtaking!