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#apaperaday: N-of-1 Healthcare: Challenges and Prospects for the Future of Personalized Medicine

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: N-of-1 Healthcare: Challenges and Prospects for the Future of Personalized Medicine

Today’s pick is an editorial from Frontiers in Digital Health on challenges for N-of-1 healthcare by You et al to commemorate that Tim Yu is visiting LUMC Leiden tomorrow. DOI: 10.3389/fdgth.2022.830656.

Traditionally medicines are developed in a population based manner using a one size fits all approach. Personalized medicine aims to tailor treatments for each individual – the term is misused frequently. Authors points out they really mean n-of-1 (truly personalized).

Authors point out that n-of-1 treatment poses several challenges. First you only have the patient’s own data to select and modify the treatment. This is a combination of genetic and phenotypic data. Genetic: for tumors whether the cancer will respond to a certain treatment.

For genetic treatments whether a mutation is eligible for a genetic treatment (ASOs & gene addition (both approved), gene editing (though this is not yet clinically applied). The phenotypic data allows measuring response to treatment (positive and negative).

Note that side effects can sometimes also be predicted by genetic data, thus allowing selection of treatment and dose for cancer and immunosuppressive treatments. Phenotypic data also involves biomarkers for the disease subtype or the treatment response.

Finally authors outline that cell lines derived from the individual patient can also help to show a treatment response, especially with differentiated IPSCs. e.g. for cystic fibrosis patients with rare mutations, the response to drugs approved for other mutations can be tested.

Data collection is important the authors outline, as is sharing the data. E.g. Curate AI is a platform for combination therapies used in oncology and immune suppression that can be helpful to tailor treatments for individual cases.

Authors stress with new opportunities come new challenges. It is not possible to develop n=1 treatments in the traditional way: it is too costly and more importantly takes too long: by the time the treatment is ready, the patient may have passed or lost all/important functions.

In the IRDIRC N=1 taskforce we try to design a path to develop individualized treatments so safety and efficacy is ensured as much as possible, while balancing the risk of taking too much time to develop the treatments for patients where not treating is a worse risk.