DRUG DISCOVERY

Question 1

BIOLOGICS ACCOUNT FOR ………… OF NEW MEDICINE APPROVALS

Answer 1

1/3

Question 2

WHAT KINDS OF TREATMENTS ARE CONSIDERED BIOLOGICS

Answer 2

ANTIBODY BASED
VACCINES
RNAI
CELL BASED THERAPY
GENE THERAPY

Question 3

WHAT IS DIFFERENT ABOUT BIOMOLECULES

Answer 3

1. TISSUE BINDING ASSAY
2. X OF TARGET SATURATION
3. T1/2 LONG
4. IMMUNOGENICITY
5. NO PK/PD CORRELATION

Question 4

WHAT IS DIFFERENT ABOUT SMALL MOLECULE DRUGS

Answer 4

1. 14C ADME
2. MAX TOLERATED DOSE
3. T1/2 SHORT
4. TOXIC METABOLITES
5. PK/PD CORRELATION
6. GENETOTOXICITY
7. CARCINOGENICITY

Question 5

WHAT TESTS ARE CARRIED OUT ON BOTH SMALL MOLECULED DRUGS AND BIOLOGICS

Answer 5

1. SAFETY PHARMACOLOGY
2. 1-3-6 MONTH STUDIES
3. DEVELOPMENTAL TOXICOLOGY STUDIES

Question 6

WHAT IS THE PROBLEM WITH BIOPHARMACEUTICALS AND ANIMAL TRIALS

Answer 6

WE MUST USE THE HUMAN MOLECULE AND SO ALL WE CAN DO IS THE ANIMAL CLOSEST IN HOMOLOGY TO HUMANS EVEN IF IT COMPROMISES THE RELIABILITY OF THE DATA

Question 7

WHY CAN YOU NOT FOLLOW SMALL MOLECULE PROTOCOL WITH BIOMOLECULES
GIVE AN EXAMPLE

Answer 7

THEY BEHAVE DIFFERENTLY

EG TGN1412 (2006)
MONOCLONAL ANTIBODY WITH A HIGH AFFINITY TO T CELL CD28
CYTOKINE STORM IN PATIENTS DUE TO MISCALCULATION OF RECEPTOR OCCUPANCY IN PRMATES VS HUMANS

Question 8

HOW WAS PROTOCOL CHANGED AS A RESULTS OF THE TGN1412 DISASTER

Answer 8

REGULATIONS NOW ONLY TEST ON TWO PEOPLE TO BEGIN WITH

Question 9

WHICH PHASE OF CLINICAL TRIALS DO SOME BIODRUGS GO STRAIGHT INTO

Answer 9

PHASE 2

Question 10

WHAT IS IMMUNOTHERAPY

Answer 10

CANCER TREATMENT USING IMMUNE SYSTEM TO ATTACK CANCER CELLS

Question 11

WHAT ARE THE TYPES OF SUCCESSFUL TREATMENTS IN IMMUNOTHERAPY IN RECENT YEARS

Answer 11

1. CHECKPOINT INHIBITORS
2. CHIMERIC ANTIGEN RECEPTOR THERAPY (CART)
3. CANCER VACCINES

Question 12

WHAT ARE CHECKPOINT INHIBITORS

Answer 12

ALLOW T CELLS TO RECOGNISE TUMOURS

Question 13

WHAT IS CAR T THERAPY

Answer 13

ENGINEERING THE IMMUNE SYSTEM TO MAKE PROTEIN THAT FIGHTS CANCER

Question 14

HOW DOES CAR T WORK

Answer 14

1. EXTRACT BLOOD FROM PATIENT
2. TAKE CD3 AND T CELLS AND TRANSFECT WITH POI
3. USE OF CRISPR CAS9 TO PUT BACK INTO THE SUBJECT

Question 15

WHAT ARE THE PROS AND CONS OF CAR T

Answer 15

NEEDS TO BE HIGHLY MONITORED
CURRENTLY TOO EXPENSIVE
CAN CAUSE FATAL IMMUNE REACTIONS

Question 16

CYTOTOXIC T LYMPHOCYTE ASSOCIATED PROTEIN 4 (CTLA-4) ON T CELL SURFACE WHEN BLOCKED CAUSES WHAT

Answer 16

CANCER TO VANISH

Question 17

PROGRAMMED CELL DEATH PROTEIN 1 DOES WHAT

Answer 17

TAMPS DOWN THE IMMUNE SYSTEM

Question 18

PROGRAMMED DEATH LIGAND 1 (PDL1) DOES WHAT

Answer 18

PROTECTS CANCERS FROM T CELLS

Question 19

IF IPLIMUMAB BLOCKS CTLA-4 WHAT IS THE EFFECT

Answer 19

T CELL POTENTIATION

Question 20

IF PEMBROLIZUMAB BLOCKS PDL1 WHAT IS THE EFFECT

Answer 20

T CELL CAN NOW RECOGNISE THE CANCER AS FOREIGN AND BEGIN TO ATTACK IT