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Personalized Medicine: transitioning from target-based to individual-based treatment

Joshua Hartman
University of Maryland Global Campus
USCP 615 Orientation to Graduate Studies
Caren Doyle
September 7, 2020
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Personalized Medicine: transitioning from target-based to individual-based treatment

Personalized medicine is defined by treatments individualized through the

patient’s own genetics, which can better predict drug effectivity, response and dosage. Patients

with the same disease respond differently to every treatment. It is time to advance from the “one

size fits all” and trial-and-error method of target-based treatments that respond differently to

each person, and support individualized treatments that can be much more effective using many

sources of data, including the patient’s data (Ho et al, 2020). Data coming from genetic

databases, medical records, tissues banks and others will be analyzed to find groups of

individuals who will respond to certain treatments and help tailor specific treatments for these

groups (Paneth & Cooper, 2020). This data has the benefit of being available anywhere at any

time, as an alternative to tests at a doctor’s office. There are many advantages to personalized

medicine, including less sides effects, better response and more accurate dosage. These benefits

make personalized medicine as a worthwhile and logical progression from target-based medicine

of today.

More accurate diagnoses, treatments and doses

Personalized medicine can be used to identify, treat and dose specifically to the patient’s

needs. Genetic markers can be used to see the risk or the presence of disease before the signs of

symptoms. This could help focus on prevention or early intervention. An example of this is the

BRCA1 and BRCA2 gene, which when in a mutated form can be a sign of susceptibility to

breast cancer (up to 85%) compared to normal BRCA1 and BRCA2 genes (13%) (E. Ellsworth

et al., 2010). These women with the mutated genes can take preventative steps to increase
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chances of survival (E. Ellsworth et al., 2010). There are many biomarkers that when found can

help diagnose diseases early and increase outcomes. Prognostic markers can be used to see how

the patient’s disease will progress, predicting the best treatment (Paneth & Cooper, 2020).

Diagnostic tests can test for genetic characteristics that could improve outcomes instead of trying

different treatments for effectiveness. Pharmacogenomic tests can predict what treatment would

be best for the patient as genetics plays a role in the adverse effects a drug may have (THE

CASE FOR PERSONALIZED MEDICINE 2014 4TH EDITION, 2014). Doctors can use the

patient’s particular variability of these genes to find an ideal dose that lessens or prevents these

adverse effects.

Aiding in drug development

Today, drugs are developed with a target, not the patient, in mind. The safety and

effectivity are identified in clinical trials, and the drug is then tried by trial and error by the

patients to determine which is most effective. With personalized medicine, the patient’s genomic

data can help decide whether a drug or treatment is compatible and effective. Clinical trials can

include or exclude ideal candidates for developing drugs by screening the genomes of potential

patients (Paneth & Cooper, 2020). This will lead to smaller and faster clinical trials, making to

the drug arrive on market more quickly. The new way drugs will be developed with personalized

medicine is one of a heuristic method rather than a linear one (Ho et al., 2020). Scientific data

will be integrated into a system that connects all the steps in the development process, from gene

discovery to Phase IV (THE CASE FOR PERSONALIZED MEDICINE 2014 4TH EDITION,

2014).
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Personalized gene editing through CRISPR

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) has the potential

of editing the genes that cause disease. CRISPR uses a guide RNA to target a DNA sequence,

then the Cas9 protein slices the DNA. Base editing is a new development that can change the

individual bases (A, C, T, G,) permanently correcting the deficient gene (Hong, 2018). Work is

also underway to make these changes reversible through a mutated version of Cas9 (Hong,

2018). Experiments can be run on the patient’s cells in a lab (ex vivo) instead of in the patient

itself to reduce risk. Animal subjects may also be used in the discovery process to apply to

humans. As with personalized medicine in general, clinical trials will use patients with the

genetic defect, saving the burden of testing a wide range of healthy volunteers (Hong, 2018).

One downside to using CRISPR is that cutting the genome activates p53, a DNA repair protein.

When CRISPR cuts and fixes the DNA, p53 tries to “fix” the DNA, making future gene editing

difficult (Hong, 2018). This can, however be avoided by editing cells that lack this protein,

which is about half of tumor cells (Hong, 2018).

Cancer treatments

Cancer is widely understood to be a genetic-based disease, so it may be possible to

discover more effective treatments or potentially a cure from personalized medicine. Harmful

DNA mutations cause abnormal control of replication, the growth of these cancer cells and the

growth eventually proves to be fatal. Finding the driver mutations and blocking their effects

could be a way to find a cure (Paneth & Cooper, 2020). Looking at the present however, genetic

tests have been used to match the patient to the best treatment. Drugs that might not have had
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positive results in clinical trials or with the global population can be used on small subgroups

with particular genetics that favor the drug (THE CASE FOR PERSONALIZED MEDICINE

2014 4 TH EDITION, 2014). Though it may not find the cure, personalized medicine can treat

patients individually to meet the patient’s needs.

Conclusion

Personalized medicine can change the way we approach treating disease. Every person

has different genetics and other variables that make the response to a drug or treatment

unpredictable. Using the patient’s genetic data can pair them with the ideal treatment, reaping all

the benefits while avoiding adverse effects. The entire process from drug development to clinical

trials can be streamlined to group patients by their genetics and provide the best drugs, testing

and response to every patient. The traditional trial-by-error method of medicine must be

examined and compared to the benefits of personalized medicine. Individualizing treatment with

personalized medicine can and most likely will promote a healthier word of individuals.
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References

E. Ellsworth, R., J. Decewicz, D., D. Shriver, C., & L. Ellsworth, D. (2010). Breast Cancer in the

Personal Genomics Era. Current Genomics, 11(3), 146–161.

https://doi.org/10.2174/138920210791110951

Ho, D., Quake, S. R., McCabe, E. R. B., Chng, W. J., Chow, E. K., Ding, X., Gelb, B. D., Ginsburg, G.

S., Hassenstab, J., Ho, C.-M., Mobley, W. C., Nolan, G. P., Rosen, S. T., Tan, P., Yen, Y., &

Zarrinpar, A. (2020). Enabling Technologies for Personalized and Precision Medicine. Trends in

Biotechnology, 38(5), 497–518. https://doi.org/10.1016/j.tibtech.2019.12.021

Hong, A. (2018). CRISPR in personalized medicine: Industry perspectives in gene editing. Seminars in

Perinatology, 42(8), 501–507. https://doi.org/10.1053/j.semperi.2018.09.008

Paneth, N., & Cooper, R. (2020, February 10). Will Precision Medicine Lead to a Healthier Population?

Issues In Science and Technology. https://issues.org/precision-medicine/

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THE CASE FOR PERSONALIZED MEDICINE 2014 4 TH EDITION. (2014).

http://www.personalizedmedicinecoalition.org/Userfiles/PMC-

Corporate/file/pmc_case_for_personalized_medicine.pdf

‌
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THE CASE FOR PERSONALIZED MEDICINE 2014 4 TH EDITION. (2014).

http://www.personalizedmedicinecoalition.org/Userfiles/PMC-

Corporate/file/pmc_case_for_personalized_medicine.pdf

‌Hong, A. (2018). CRISPR in personalized medicine: Industry perspectives in gene editing. Seminars in
Perinatology, 42(8), 501–507. https://doi.org/10.1053/j.semperi.2018.09.008

E. Ellsworth, R., J. Decewicz, D., D. Shriver, C., & L. Ellsworth, D. (2010). Breast Cancer in the

Personal Genomics Era. Current Genomics, 11(3), 146–161.

https://doi.org/10.2174/138920210791110951

‌
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COOPER, R., & PANETH, N. (2020). WILL Precision Medicine LEAD TO A HEALTHIER

POPULATION? Issues in Science & Technology, 36(2), 64.

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