April 19, 2012
In the April 16, 2012 Wall Street Journal, one article dealt with an ALS (Lou Gehrig’s disease) patient having problems having access to a new potential therapy which is being clinically studied in a specific ALS population therapy. The patient is too far advanced to qualify as a volunteer for the study. Even if the results of the ongoing study are positive, he will probably not be alive by the time of the FDA approval. So what did he do? He somehow found out what he believes to be the new therapy, and it is now taking it through his feeding tube.
Awhile back I designed and managed a multi-clinic study in patients with ALS who were treated with brain extract nerve growth factors called gangliosides. I included a broad spectrum of patients, including late stage, because there is not much of a placebo response and positive effects could be easily detected in a small number of patients. Regarding late stage patients, the rationale is based on the assumption that there still are viable neurons in the brain but they are not functioning because they are in the late stages. If we could make them function again then we would hopefully see a dramatic, reversal effect which would then speed-up the process of FDA approval and availability to patients. If doctornauts or physician volunteers for clinical research with late stages of disease were not permitted in the current clinical study, then the drug could be studied in them increasing the probability of success within a shorter period of time.
An interesting historical note: In my studies, one of the ALS patients was a famous physician who had lost power in his hands along with other neurologic manifestations. He told me, and I cannot confirm this fact, that he was the doctor who treated Lou Gehrig. It defies probability.
In the op-ed section of the WSJ there’s a piece by Andrew von Eschenbach, former Commissioner of the FDA, making the argument that FDA needs more resources to speed up medical discovery. He wrote, “Breakthroughs were and still are a long way off.”
In my first book published way back in 1972, I wrote, “Our present system of drug discovery is almost designed not to cure the great disease that confronts us. There is no doubt that many will be cured in the distant future, but it is unfortunate that many of us must wait.”
So Dr. von Eschenbach and I agree. After 40 years little has changed. I’ll discuss whether increasing FDA resources will speed up medical breakthroughs discoveries in a future posts. But, though this was not his intent, he makes a powerfully strong argument that the discovery process is stalled due to FDA regulations. A simple, rapid and effective way to remove these regulations is to pass the Doctornaut Act where doctor patients can directly volunteer for clinical studies conducted by doctor clinical researchers.