This is going to be my last blog for a while. I do not know how long. Certain personal matters have come up and I simply cannot focus on writing. I thank you all for your readership and support. I wish you the best of health.
An incredible update appeared on LinkedIn (thanks to my dear friend Philip Libman for linking to it). The post in LinkedIn is here, and the original article is linked to by Ruti Alon, via here. The title of the post is “Israeli Drug Makes Alzheimer’s Symptoms Disappear in Mice”.
Such a dramatic statement requires a qualifier which appears in the article itself.
Medical science has been this optimistic before about finding a cure for Alzheimer’s, but in most cases the scientists, patients and their families have suffered serious disappointment. The bitter truth is that over 99 percent of all clinical trials for Alzheimer’s treatments end in failure.
I must say that as I read the beginning of this article, despite knowing that this was animal-based research and thus was conducted under very specific conditions that do not perfectly mimic humans, I was nevertheless greatly excited. I don’t think that there is anyone I know who has not felt some degree of pain related to watching a loved one or the loved one of a loved one, going through the stages of Alzheimer’s.
It sounds strange to anthropomorphize diseases by giving them adjectives such as “cruel”, “insidious” and “nefarious”. And although Alzheimer’s is a disease that in many ways causes greater suffering to the people around the patient than the patient him or herself, it is still seen as a vicious malady that urgently needs a cure.
The study of Alzheimer’s is actually an opening into other diseases. If the cure for Alzheimer’s ends up being some enzyme or hormone that re-animates brain neurons/nerve cells, then the same cure may work for Parkinson’s disease and ALS [Lou Gehrig’s disease]. It would be the cruelest of jokes if the myriad of diseases that rob people of all of their quality of life, end up all being a variation on a theme that is treatable using a single method. Whatever the cure is, it is needed as soon as possible, because no one is laughing at this “joke”.
As I read this article, despite the warning about false starts in the past, I could not help but think to myself that there are probably a tremendous number of people who would volunteer without reservation for trials based on such new findings. Ethics committees exist almost exclusively to avoid taking advantage of false hope that is triggered by such articles. Imagine if every breakthrough in a lab working only on an animal, or even just a cell culture model, was immediately made available to the general public. In many cases, it would be equivalent to injecting poison into people whose only crime is wishing to have their normal life restored.
I have mentioned before that I am relearning my basic math skills in order to eventually understand the field of machine learning. I am presently studying probability and I am reminded of the famous quote “there are lies, there are damned lies, and there are statistics”. I have read too many papers in my life that speak of the total of complete and partial responders as being a significant number. In practice, these papers may very well be speaking about a treatment that adds a few months of life to a person, but at the cost of constant hospital residence as well as persistent pain and suffering. Although statistics speak in absolute terms [such as, a 22% greater likelihood of cure], the real-life experience of using statistically significant treatments may be horribly disappointing.
This study on Alzheimer’s speaks of a different level of results. In this particular mouse study, the author states that the “new drug … led to the complete disappearance of symptoms in mice used as a model for the sickness”. This statement is far beyond one such as “a 22% greater likelihood”. Unfortunately, none of this means that this particular drug will have any practical value in humans. Or, it might be that this drug has value, but also causes serious side effects that are intolerable to the vast majority of patients.
It is almost impossible for a patient with any serious disease to make a fully informed decision about how to proceed. Firstly, most patients do not have any degree of medical education and do not even understand the most basic terms such as “cell”, “neuron” or “plaque”. When the majority of patients are recruited for a study, the only thing or perhaps the only word that they truly hear and internalize is “cure”. When my own brother joined a study being run at the Sloan-Kettering hospital, a world center in cancer research, we didn’t think about the money involved in being part of this trial or the likelihood of success. All we understood in our hearts was that there was a chance of cure.
The question we have to ask is whether a person should be denied the right to try something even if the risks are enormous and the potential for success is small. Almost every month, you will hear about some failed attempt at climbing Mount Everest. The number of lives that have been lost over this quest is astounding. And to be blunt, the ultimate outcome of succeeding is to be able to tell a cool story around the campfire. This hardly equates with curing oneself of an incurable disease. This also hardly equates with being a person who by virtue of being in a clinical study, proves the value of a medication that can then go on to help countless people.
Almost every day, you hear about the aging baby boomer population. Amongst the concerns over money and living situation, there is a true terror over the possibility of acquiring a disease like Alzheimer’s. We could soon find ourselves in the midst of an epidemic, where floor after floor in hospitals is filled with patients with varying degrees of Alzheimer’s. There have been articles about the crushing effect that this could have on insurance companies. As it stands, the United States pays out trillions of dollars every year for healthcare. And it is often stated that 50% of these costs are related to 5% of the population. These statistics will worsen as the number of baby boomers, over the age of 65, increases. Finding a cure for Alzheimer’s could be just as important to the worldwide economy as it is to the individuals suffering from the disease.
If all of these facts are explained to a patient who has sufficient residual cognition to understand them, what ethical right do we have to deny him or her even a fraction of a percent success rate? If a loved one of mine had Alzheimer’s, I would try to find out what this drug is, and then to find out if there is any legal [and even illegal] way to obtain the drug. It would ultimately be the decision of my loved one with the disease. But if he or she wanted to try, I would fight with every fiber in my body to get access to this medication.
Ethics are a strange thing. Morality, a Jewish invention, is far from absolute. Once upon a time not that long ago, homosexuality was thought of as one of the worst possible “diseases” that a person could have. This went hand-in-hand with societies, including all Western societies, that saw it as totally legitimate that 12-year-old girls get married to much older males. My wife’s grandmother was 14 years old when she got married to her husband who was 11 years her senior. Speaking to my wife’s grandmother, all she had were memories of adoration and love for her husband. This was not an abusive situation. It was, for all intents and purposes, a totally ethical coupling, that has resulted in generations of offspring. It is a simple truth that ethics/morality are actually very flexible. It is often more so by votes rather than by intellect that the cut off for ethical behavior is set.
One could easily argue that publishing any medically related research until it clearly identifies a positive outcome that can already be used as a medication, is unethical. To give people false hope, or to tell them that we have found something amazing that may become a life-saving drug in 15 years from now, is beyond cruel. If people knew that they could already sign up for human trials, even at great personal risk, then no one could argue that people are being denied their only possible chance. Imagine holding a cup of water in your hand in front of a man dying of thirst, but being unwilling to give the man the water due to a very real fear that it contains a pathogen that itself could kill the man. Does anyone really think that it is ethical to deny this dying man the cup of water? I for one unquestionably believe that the right thing to do is to give the man the water. And in the same way, when lab results are so dramatically positive, I think it is cruel and inhumane to deny people the choice to become part of an early clinical study.
Sometimes, what happens in these scenarios is that the drug being tested ends up being legally shared with other laboratories around the world. And there are places in the world where money and influence are far more powerful than any ethics committee. The ultimate irony is that the results from using a medication in such an “un-ethical” manner, may be the impetus for advancing its use in countries with stricter ethical standards.
I have no answers at the moment as to what should be the cut off for success in the lab before even considering early human trials. I have no mechanism for properly measuring success in such individuals, given that they have not been put through the rigorous selection process that goes along with formal ethical studies. It is definitely possible that perceived success from studies, not done according to formal protocol, could lead to fraud and abuse. It is very easy to imagine a clinic somewhere in the world announcing tremendous success with a given treatment, which ends up swaying the opinion of an American ethics committee, in favor of early human trials. And the result of all of this could be needless deaths, millions of dollars wasted, and wasted resources that could have been put into researching other potential treatments.
I have never understood the attraction of gambling. Taking good money and risking it on a selection of cards or the results of two dice seems utterly foolish to me. On the other hand, if one wants to truly experience the thrill and risk of gambling, invest the same money in research. Perhaps, the same gambler should sign him or herself up for clinical research, i.e. to be a test subject. If one really enjoys the unknown, and furthermore is willing to risk personal well-being, then I can see no greater “game” than becoming involved in clinical research.
So perhaps the next time you are in Vegas, you can put 100 on Alzheimer’s research rather than “lucky seven.”
Thanks for listening.