Personalized drugs

Presented by
Ankit Bikram Poudel
Masters in Pharmacy
(Industrial group)

Kathmandu university
 How different are we?
Youre one of a kind

Its not just your eyes, smile, and personality.

Your health, risk for disease, and the ways you respond to medicines
are also unique.
Medicines that work well for some people may not help you at all.
They might even cause problems.
Wouldnt it be nice if treatments and preventive care could be
designed just for you?
 Our current apporach lack of ability to predict an individual
patients treatment success for most diseases and conditions
means that clinicians have no choice but to follow a less than
optimal approach to prescribing drugs and other treatment
options.
A patient being treated for high blood pressure, for example,
might be placed on one of a number of blood pressure
medications. The patients doctor makes a decision about what
medication to prescribe based on only general information
about what might actually work for that particular patient. If the
medication does not work after a few weeks, the patient might
be switched to another medication. This somewhat trial-and-
error approach can lead to patient dissatisfaction, adverse drug
responses and drug interactions and poor adherence to
treatment regimens.
 The careful matching of your biology to your medical care is known
as personalized medicine. Its already being used by health care
providers nationwide.
The story of personalized medicine begins with the unique set of
genes you inherited from your parents. Genes are stretches of DNA
that serve as a sort of instruction manual telling your body how to
make the proteins and perform the other tasks that your body needs.
These genetic instructions are written in varying patterns of only 4
different chemical letters, or bases.

The same genes often differ slightly between people. Bases may be
switched, missing, or added here and there. Most of these variations
have no effect on your health. But some can create unusual proteins
that might boost your risk for certain diseases. Some variants can
affect how well a medicine works in your body. Or they might cause a
medicine to have different side effects in you than in someone else.
 Introduction
Personalized medicine is a young but rapidly
advancing field of healthcare that is informed by each
person's unique clinical, genetic, genomic, and
environmental information. Because these factors are
different for every person, the nature of diseases
including their onset, their course, and how they might
respond to drugs or other interventionsis as
individual as the people who have them.
Personalized medicine is about making the treatment
as individualized as the disease. It involves identifying
genetic, genomic, and clinical information that allows
accurate predictions to be made about a person's
susceptibility of developing disease, the course of
disease, and its response to treatment.
 A familiar, small-scale example of this manifests in blood typing.
Every time a blood transfusion is given, a doctor checks the
patients personal blood type and selects the blood for his
transfusion accordingly. Its understood that if the blood type isnt
compatible with the patient, his body will reject the transfusion in
a potentially fatal response of the immune system.

Personalized medicinetakes this idea and applies it to all medical

treatments. It does not make sense to give a person with type A
blood a type B blood transfusion (even if someone else could
receive that same type B blood successfully). In other words, if a
treatment is incompatible with the genetic makeup of a patient, it
will not work.
The goal of personalized medicine is to streamline clinical
decision making by distinguishing in advance those patients most
likely to benefit from a given treatment from those who will incur
cost and suffer side effects without gaining benefit.
 Thus personalized mnedicine is the
ability to offer
The right drug
To the right patient
For the right disease
At the right time
With the right dosage
 Advantage
Ability to make more informed medical
decisions
Higher probability of desired outcomes
thanks to better-targeted therapies
Reduced probability of negative side effects
Focus on prevention and prediction of
disease rather than reaction to it
Earlier disease intervention than has been
possible in the past
Reduced healthcare costs
 background
Every person has a unique variation of the
humangenome.Although most of the variation
between individuals has no effect on health, an
individual's health stems from genetic variation
with behaviors and influences from the
environment.
One way that biological variation manifests itself is
responsiveness to drugs.
for example, ADHD medicine works only for one of
ten preschoolers, cancer drugs are effective for
25% of patients, and depression drugs work with 6
of 10 patients
 Modern advances in personalized medicine rely on
technology that confirms a patient's fundamental biology,
DNA,RNA, orprotein, which ultimately leads to confirming
disease. For example, personalized techniques such as
genome sequencing can reveal mutations in DNA that
influence diseases ranging from cystic fibrosis to cancer.
Another method, calledRNA-seq, can show which RNA
molecules are involved with specific diseases. Unlike DNA,
levels of RNA can change in response to the environment.
Therefore, sequencing RNA can provide a broader
understanding of a persons state of health. Recent studies
have linked genetic differences between individuals to RNA
expression,[10]translation,[11]and protein levels.[12]
 Application
Diagnosis and intervention:- Having the ability to look at a
patient on an individual basis will allow for a more accurate
diagnosis and specific treatment plan
Genotypingis the process of obtaining an individuals DNA
sequence by usingbiological assays.[17]By having a detailed
account of an individuals DNA sequence, their genome can then be
compared to a reference genome, like that of the
Human Genome Project , to assess the existing genetic variations
that can account for possible diseases. A number of private
companies, such as23andMe,Navigenics, andIllumina, have
created Direct-to-Consumer genome sequencing accessible to the
public.[7]Having this information from individuals can then be
applied to effectively treat them. An individuals genetic make-up
also plays a large role in how well they respond to a certain
treatment, and therefore, knowing their genetic content can change
the type of treatment they receive.
 In addition to specific treatment, personalized medicine can
greatly aid the advancements of preventive care. For instance,
many women are already being genotyped for certain mutations
in the BRCA1 and BRCA2 gene if they are predisposed because of
a family history of breast cancer or ovarian cancer. [20]As more
causes of diseases are mapped out according to mutations that
exist within a genome, the easier they can be identified in an
individual. Measures can then be taken to prevent a disease from
developing. Even if mutations were found within a genome, having
the details of their DNA can reduce the impact or delay the onset
of certain diseases.[16]Having the genetic content of an individual
will allow better guided decisions in determining the source of the
disease and thus treating it or preventing its progression. This will
be extremely useful for diseases likeAlzheimers or cancers that
are thought to be linked to certain mutations in our DNA.
 Drug development and usage
Having an individuals genomic information can be significant in
the process of developing drugs as they await approval from the
FDA for public use. Having a detailed account of an individuals
genetic make-up can be a major asset in deciding if a patient
can be chosen for inclusion or exclusion in the final stages of a
clinical trial.[16]Being able to identify patients who will benefit
most from a clinical trial will increase the safety of patients from
adverse outcomes caused by the product in testing, and will
allow smaller and faster trials that lead to lower overall costs. [22]
In addition, drugs that are deemed ineffective for the larger
population can gain approval by the FDA by using personal
genomes to qualify the effectiveness and need for that specific
drug or therapy even though it may only be needed by a small
percentage of the population.,[
 Cancer genomics:- Cancer Genomics, or
Oncogenomics is the application of
genomics and personalized medicine to
cancer research and treatment.
High-throughput sequencingmethods are
used to characterizegenesassociated with
cancer to better understand
diseasepathologyand improvedrug
development. Oncogenomics is one of the
most promising branches ofgenomics,
particularly because of its implications in
drug therapy.
Example :-
 Personalized medicine for
cancer
Personalized medicine is used to learn about a persons genetic
makeup and how their tumor grows. Using this data, doctors hope to
find prevention, screening, and treatment strategies that may be
more effective. They also want to find treatments that cause fewer
side effects than the standard options. By performing genetic tests on
the cancer cells and on normal cells, doctors may be able to
customize treatment to each patients needs.
Creating a personalized cancer screening and treatment plan includes:
Determining the chances that a personwill develop cancer and
selecting screening strategies to lower the risk
Matching patients with treatments thatare more likely to be more
effective and cause fewer side effects
Predicting the risk of recurrence, whichis the return of cancer
 How is it different ???
Before personalized medicine, most patients with a specific
type and stage of cancer received the same treatment.
However, it became clear that some treatments worked
better for some patients, than for others. The growth in the
field of genetics has led researchers to find genetic
differences in people and their tumors. In turn, this
explained many of the different responses to treatment. A
person with cancer may now still receive a standard
treatment plan, such as surgery to remove a tumor.
However, the doctor may also be able to recommend some
type of personalized cancer treatment. Personalized cancer
treatment is now an active part of the treatment plan or as
part of aclinical trial. A clinical trial is a research study
involving people.
 Examples
Targeted treatments.Atargeted treatmenttargets a cancers specific
genes and proteins that allow the cancer cells to grow and survive.
Researchers are finding new targets each year and creating and testing new
drugs for these targets. This is a few, but not all of the cancers where targeted
treatments are used.
Breast cancer
Colorectal cancer
Gastrointestinal stromal tumor
Kidney cancer
Lung cancer
Melanoma
Multiple myeloma
Some types of leukemia and lymphoma
Some types of childhood cancers
Of course, treatment with a targeted therapy depends on finding out whether
the tumor has the specific target. This is found by testing a sample of the
tumor.
 Pharmacogenomics
Pharmacogenomics.Pharmacogenomicslooks at how a persons
genes affect the way the body processes and responds to drugs.
These changes influence how effective and safe a drug is for a
person. For example, some peoples bodies may process a medicine
more quickly than others. This means that the person would require a
higher dose of that drug for it to be effective. However, someone
elses body may not process a drug as quickly. The drug would then
stay in the bloodstream for a longer time and may cause more
severe side effects.
How can pharmacogenomics be used for cancer treatments? Here is
an example: People with colorectal cancer sometimes have a specific
altered gene. These patients may have serious side effects when
treated with the drug, irinotecan (Camptosar). This gene makes it
harder for the body to break down the drug. In these patients,
doctors prescribe lower amounts of the medicine so patients will
have fewer side effects.
Personalized drug in preventive care
dividual's genome influences his or her likelihood of developing
(or not developing) a broad range of medical conditions,
personalized medicine focuses strongly on wellness and disease
prevention.
For example, if a person's genomic information indicates a higher-
than-average risk of developing diabetes or a particular form of
cancer, that person may choose a lifestyle, or sometimes be
prescribed medications, to better regulate the aspects of health
and wellness over which he or she has control. The person may
benefit in the long run from making preventive lifestyle choices
that will help counteract the biological risk.
Genomic medicine may help determine a person's risk of
developing several specific medical conditions, including:
CancerCardiovascular diseaseNeurodegenerative
diseasesDiabetesObesityNeuropsychiatric disorders
Future of personalized drugs
Despite the promises of personalized cancer treatments, not all
types of cancer have personalized treatment options. Some of
these are only offered through a clinical trial and are not yet
standard treatment options. Genetic testing for patients and
tumor samples may be costly and time-consuming. Also, many
insurance plans may not cover the costs of these tests. In
addition, some personalized treatments, such as targeted
treatments, can also be expensive.
Personalized medicine is an evolving approach to cancer
treatment. Doctors still dont know all about the genetic changes
that occur in a cancer cell. They also dont know how some of
these new cancer treatments work. A targeted therapy may stop
working and a promising treatment is no longer effective. Talk
with your doctor to learn if personalized cancer treatments may
be a part of your treatment plan.