Patient specific vs. medication specific, or, the old world vs. the new one

24 May 2020

It is clear that the medical world is going through a major change. Increasing amounts of data suggest that medicine should take the next step, to be even more precise and accurate. It is very reasonable to assume that in the future, probably in our children’s lifetime, medicine and medication will be patient-specific, “tailor made”, rather than the “one size fits all” we are accustomed to.

Why is it so important? Every treatment incorporates potential risk. Patient-specificity will allow an optimal answer of the most important question in any medical treatment: “will the benefit outweigh the potential risk?”

Being patient-specific will allow clinicians to asses which patients are prone to adverse effects or treatment failure, and on the other hand, to recognize those who will benefit most from a specific treatment. For example, the story of thalidomide.

One of the most infamous (‘There is no such thing as bad publicity’…) drugs in the pharmaceutical world, the one drug every pharmacy student is familiar with, is thalidomide.

When the drug was presented in 1957, it was found to act as a tranquilizer, sedative, and a painkiller. Thalidomide was originally marketed as treatment of insomnia, cough, and headaches. Then, physicians found that thalidomide was also an effective antiemetic which had an inhibitory effect on morning sickness. As a result, thousands of pregnant women were prescribed this drug to relieve such symptoms.

In the early 1960’s,anAustralian obstetrician namedWilliam McBride and aGerman paediatrician,Widukind Lenz,identifiedapossiblelink between the increase in somedevastating birth defects andthe use ofthalidomideamong pregnant women.Within a year, Grünenthal, the German pharmaceutical company who marketed the drug and their representatives worldwide, took the drug completely off the market.

Following the thalidomide tragedy,in 1961, after the suspected relation was finally confirmed, the US congress enacted strict rules, requiring pregnancy testing for drugs. Similar laws were applied in other countries as well. Thalidomide usage stopped, and the drug was completely banned. But that was not the end of thalidomide.

Thalidomide’s somewhat ‘illegal’ rebirthbeganwithProf.Jacob Sheskin, a dermatologist working with Leprosy patients in Hansen Leper Hospitalin Jerusalem. Hefirst prescribed thalidomide in 1964 as a sedative toa critically ill patient with ErythemaNodosum Leprosum (ENL), a painful complication of Leprosy,which causes diffuse red nodular lesions along with fever, weight loss, arthritis, and general malaise.In order to relieve the symptoms in one such patient, Prof. Sheskin sedated him with some thalidomide, after other tranquilisers and painkillers failed.Although the drug was banned, it was still available in Hansen hospital, as it was widely used in Israel before its ban. This wasa“hail Merry” attempt to relieve pain, possibly becoming one of the most patient-specific treatments ever, since at the time, if considerations were based solely on “medication specific” point of view, it would have been deemed too dangerous to even mention thalidomide to the FDA or other health regulators worldwide, let alone administrating  it to patients.

A dramatic improvement in patient’s condition was noted after consuming only four tablets. Although there was no relief in pain, the patient woke up after a good sleep, and got out of bed without any assistance, a fete that was not possible for him before the thalidomide. Prof. Sheskin then treated another six patients with ENL and noted the same dramatic results. Further studies involving much larger numbers of patients again showed significant responses.

This research waslater reproduced and reported by Sheskin in 1965 [Sheskin, 1965a, 1965b]. In 1971 the World Health Organization (WHO)published the results of a randomized trial that confirmed the efficacy of thalidomide in ENL [Silverman, 2002; Iyer et al. 1971].Only in 1998 did the FDA approve thalidomide for treating Leprosy.

From the 1970s Grünenthal has been supplyingthalidomide tablets to Leprosy hospitals around the world, to help manage ENL. The drug isdistributed under anagreement with the WHO, while national health authorities strictly monitor it’s distribution.

The renewed use of thalidomide remains controversialalthough the positive effects are undeniable. There is a risk of new thalidomide-effectedbirths, particularly in countries where birth controls may not be widely spread. But there is no argument that what Prof. Sheskin had in mind was only his patients’ well-being, putting them at the center of his attention.

As per our times, this is by no means a call for healthcare providers and medical staff to act against guidelines or regulatory instructions, rather a demonstration of how being innovative and thinking outside of the box could benefit many patients and bring the “new world” closer to them.

Nowadays, legacy clinical decisions support systems, originally designed to address and help manage drug related problems, will cause ‘Alert-Fatigue’ as they alert on many drug-drug interactions, based on “old world” scheme, being drug-specific instead of patient-specific, as they do not take patient parameters into account.

But it is crucial to be able to present or avoid alerts while taking into consideration a slew of various data points, including patients’ genetic profile, concomitant medication which could mitigate unwanted effects, patients real time lab results, and many more.

Seegnal offers a “new world”, smart, patient specific platform to prevent Drug-Related Problems, putting the patient and their unique properties in the center, just like Prof. Sheskin did.

Patient specific Vs. medication specific, or, the old world vs. the new one