Tale of Two Illnesses

The disease we have been calling as leukemia (blood cancer) for ages now was once known as the suppuration of blood. In case you were wondering “suppuration” meant “spoiling” (in this case, spoilt to become a pus). So what, you must be thinking, it’s just a name change, how does it matter?

Except that the choice of name had unintended consequences, writes Siddhartha Mukherjee in The Emperor of All Maladies:

“An illness, at the moment of its discovery, is a fragile idea, a hothouse flower – deeply, disproportionately influenced by names and classifications.”

The term “suppuration of blood” suggested the root cause was that blood was getting spoilt. Whereas it was just a description of what was being observed. A key difference. That “humility of the (new) name” was transformative. Once we start from “We don’t know”, we are open to all options.

Mukherjee points out that our understanding of another disease, a century later, got transformed similarly when we renamed it. From gay related immune disease (GRID) to acquired immuno deficiency syndrome (AIDS). This time, the name change was from one with moral connotations to a descriptive one!

The importance of names aside, cancer and AIDS would end up with similar stances in another area: clinical trials. Clinical trials are conducted to check if a “cure” works in a statistically significant number of cases. Additionally, such trials are double blind studies. Here’s what a double blind study means:

1)      Patients don’t know whether they’ve been assigned to the medicine group or to the placebo group;

2)     Nor do doctors! This part was added to eliminate the bias that doctors were unconsciously introducing if they knew which patients were getting the placebo.

With cancer and AIDS, patients “fled from clinical trials”, writes Mukherjee. Why? Because they were afraid that “they might be assigned to the non-treatment arm by what amounted to a coin flip”. In life-and-death diseases like cancer and AIDS, nobody wanted to be on the non-treatment group:

“Scientific uncertainty is no excuse for inaction… We cannot wait for ‘proof’”.

But without proper clinical trials, how could one know whether a cure really worked? As one researcher argued:

“If you start making exceptions and deviating from your protocol, then you get a lot of patients whose results are not going to help you understand whether a drug works or not.”

It has been an argument that has gone back and forth… with no good answer.