A short while ago, I was seeing my doctor to talk about some side effects I experienced from a routine medication I have been taking for years. After my preferred formulation recently went off market, I have been rotating between different formulations of the same drug to find one to suit me. The doctor asked me again what the active compound was in the drug I was taking before. I confirmed that all the preparations I tried had the same active compound in the same dose, yet I experienced varying side effects with all of them. With a puzzled look, she said that she had never heard about that before and didn’t know what to advise me. So, based on my own research, I convinced her to write me a prescription for yet another brand of the same drug. This experience was not even new to me. Years before, I had faced some severe side effects that made me stop another medication and I had similar difficulties convincing my doctor of an experience that felt very real to me.
Clinical trials aim to improve treatment for all
The fact is that actions and side effects of drugs are based on the results of large-scale studies and clinical trials, where the experiences of individual patients can get lost. A drug gets approved because of its improved effect compared to placebo, or to an existing drug regimen, but it is based on an average response observed in hundreds or even thousands of people. During the same trials, side effects are monitored and recorded so that they can be added to the drug prescription (“bipacksedel” in Swedish) based on how many people reported them, with a minimum of 1 to 10 in 10 000 people.
A rather innocent und unavoidable consequence of this setup is that individual people have a certain risk of reacting unexpectedly to new medication. That’s why it is always advised to read the reported side effects and watch out for any changes after you start taking a new medication. However, the risks involved get more serious with the severity of the condition that is being treated. Take multiple myeloma for example, a malignant bone marrow condition, for which since the turn of the century several new agents were approved after clinical trials showed clear benefits compared to previous treatments. Consequently, the standard of care was updated based on these groundbreaking results and since then, most newly diagnosed myeloma patients have been treated with one of these novel agents. This has provided statisticians with a wealth of new response data and recent retrospective analysis has shown that, although the average prognosis has improved significantly since the introduction of the new regimens, certain groups of patients have hardly benefited at all – while still suffering through the side effects of the new drugs. New myeloma trials thus often focus on such specific subtypes of patients for whom a lot of progress is still to be made.
So, after the drug gets approved, there is still no guarantee that every patient will benefit even though it showed improvements in large cohorts of people. On the other hand, we have the extraordinary phenomenon of exceptional responders. These patients are defined as responding to treatments in an unexpectedly positive way, to drugs other patients with the same condition normally don’t, or with a depth of response that is unusual. It is of course important that these people do have access to these drugs, although they might not be recommended on a large scale for other patients. Dedicated studies of these cases aim to unravel the cause of their response so that these treatments can be targeted to the right people.
Personalized medicine just right for you
These efforts are part of a larger movement called personalized medicine. This term captures the idea that every treatment should be tailored to each patient individually. A major part of this field is taken up by immunotherapy studies. Activation of the patient’s own immune system through vaccination or transfusion with donor immune cells can be used to target the specific disease phenotype of individual tumors. Some of these trials have shown exceptionally promising results and optimistic reports estimate that such vaccines could be available in the next decade.
Personalized medicine is also in view for neurological disorders, with wearable diagnostic tools to follow up patients outside the hospital or by further subdividing large disorders. A Belgian start-up has developed a patch that measures a patient’s clinical values during epilepsy attacks, paving the way to a faster tailoring of their treatments, while other projects aim to define subtypes of Alzheimer’s diseases so these patients can receive specialized care.
A personalized future
When I picked up my new medication at the pharmacy and explained the issue I was having, the pharmacist did admit that different formulations of the same drug could affect the uptake of the active compound and wished me good luck. Now I can report that I found my personal match and I’ll make sure to stock up on this formulation. The future, it seems, will be personal. And I mean that beyond the personalized ads on your social media feeds. Your treatments will also evolve from a one size fit all to a personal fit adapted just for you.
A myeloma trial for a subtype of patients harboring TP53 defects:
Pages about exceptional responders to cancer treatments:
A review article on personalized immunotherapy:
The site of Byteflies that is developing an epilepsy patch:
A recent study identifying subtypes of Alzheimer’s and the clinical implications:
Pictures from PublicDomainPictures and Arek Socha via Pixabay