In statistics, N refers to the number of subjects in the sample, or the sam­ple size. In a scientific study such as a clinical trial, N is the number of patients en­rolled in each arm of the study. Generally speaking, the larger the N, the stronger the validity of the trial.

From a statistical standpoint, an N of 1 would have no real significance. In fact, we were always taught in medical school that an N of 1 is an anec­dote, not nec­es­sar­i­ly worthy of consideration, and often dismissed.

In real life, however, when you are confronting cancer and you are the N, quite the opposite is true. The only thing that matters is the N of 1.

We are all familiar with clinical trial results. They all seem to sound pretty similar and are reported in a some­what standardized fashion.

It goes something like this: 30 percent of patients achieved at least a partial response, average time to pro­gression was x months, and ... I’m zoning out.

I want to get down to the gist of it, the important stuff: What do the results mean for me personally? Am I part of the 30 percent of patients who will respond, or the 70 percent who will not respond? At this point, it’s a crapshoot.

The reason it’s a crapshoot is that there are fundamental biological differences in the multiple myeloma of the pa­tients who respond and those who don’t respond, and we don’t yet understand those differences.

This is the huge disconnect between the clinical trial world and the reality of a single patient.

A number of patients – hopefully enough to be statistically significant – are thrown together in a clinical trial. These patients may all be fundamentally different from each other; some will respond, some won’t. We are just not sophisticated enough yet to understand these differences.

This is the essence of genomics and personalized medicine. Everyone is an N of 1, but we are still using a crude, one-size-fits-all approach in current clinical trials.

At the stage that I am at in my multiple myeloma (having exhausted all standard therapies and being in­el­i­gible for al­most all clinical trials), I have to embark on my own N of 1 experiment.

After months of waiting for approval and then completing seemingly mountains of paperwork, I have finally started a single-patient protocol of elotuzumab ^[1] in combination with Revlimid ^[2] (lenalidomide) and dex­ameth­a­sone ^[3] (Decadron) through a “compassionate use” program.

Since this is a single-patient protocol, we will hopefully have some flexibility – with the approval of the drug company and the U.S. Food and Drug Administration (FDA) – to modify and add other myeloma drugs as we go if the current combination doesn’t work.

Will it work? I have had more than one opinion telling me that it won’t. No one can say for sure. However, in this case, I’m not just a statistic. All that matters is me. I am more than happy to be an anecdote. It all comes down to that N of 1. So, of course, why not try?

However, I’m not putting all of my eggs into one basket. I am actively investigating and pursuing any and all options. Immunotherapy ^[4] and genomics seem to be the hot areas that are currently generating all the buzz. They seem to be the promise of the future.

A great deal of work is being done to try to identify and characterize the genetic mutations that drive multiple myeloma. It is turning out to be a very complex and heterogeneous task. The goal is to identify which specific mutations are driving the disease in an individual and then use specific therapies to target these mutations – truly the N-of-1 approach.

Access to genetic profiling in multiple myeloma is becoming increasingly prevalent and available. I have had the testing done (my insurance even covered it) and obtained some potentially useful information, although it’s way too early to say for sure.

The problem is that what to do with the information is still very much in its infancy.

Work is also being done on “repurposing” existing drugs that have already been approved for other cancers for use in multiple myeloma. The most significant example of this effort is a group of drugs currently ap­proved by the FDA to treat malignant melanoma. These drugs target genetic mutations that also have been found in certain multiple myeloma patients. Some multiple myeloma patients have already started using these drugs with some success.

Clinical trials of new drugs targeted to specific genetic mutations are being introduced or expanded to in­clude multiple myeloma.

All of this is going to be exceedingly complex and will take time. It may even turn out to fall short of ex­pec­ta­tions.

The ultimate goal is for everyone to be an N of 1. After all, that’s the only N that matters.