How do you know a treatment works

Question 1

Explain the cancer kinetics growth graph

Answer 1

-It takes 10 years before a tumour gets to a certain number of cells
-where it produces symptoms and patient can be diagnosed.
-There is a short window of opportunity between diagnosis and death to treat the patient

Question 2

What current cancer treatments do we have and what are their objectives

Answer 2

treatments
Surgery – physically removing the tumour
Chemotherapy – using anti-cancer drugs to destroy the tumour
Radiation – using radiation to destroy cancer cells
Hormone therapy – turning off the hormones that are driving the cancers.
Immunotherapy – using patient’s immune system to fight the cancer
Biological therapies – interfering with the biological pathways that are driving the cancer

Objectives of treatment
Cure the patient.
kill or remove ALL cancer cells
Prolong patient survival (if they have too advanced disease)
By killing MOST cancer cells
Palliate symptoms when the cancer is very advance. Here you need to:
kill SOME cancer cell

Question 3

What is remisson

Answer 3

• although cancer may not be visible
• there may be some cancer cells left after removal
  **- when cancer is no longer visible it is called being in remission
  **- so tests need to happen to make sure the cancer hasn’t returned
• what you do when you treat is that you shift the curve left so that you have more time to treat the cancer in case of relapse

Question 4

treatment response

complete response

Answer 4

disapperance of all target leisons

Question 5

partial disease

Answer 5

at least 30% decrease in sum of diameters of target lesions

Question 6

stable disease

Answer 6

there hasnt been sufficent shrinkage, to qulaify for partial reponse, or enough to increase in leisons to qualify for proggressive disease

Question 7

how do we assess treatment efficacy for target leison

Answer 7

• so you take a patient and you pick up to 5 lesions
• then you physically measure the length of the longest diameter of the lesions
• then you add those 5 numbers up to give you measures of the burden of disease
• then they go away and have treatment
• rescan them and measure the 5 lesions ago
  
  • a complete response means all lesions are gone
  • 30% decrease partial response
  • anywhere between stable disease
  • 20% increase -> progressive

cannot guarantee a responding tumour= longer survival time

Question 8

survival

Disease free survival time

Answer 8

the time prior to the release post post-radical treatment

Question 9

progression-free survival time

Answer 9

• survival time prior to tumour progression
• can be beneficial to a patient’s symptoms

Question 10

Overall survival time

Answer 10

time from start of treatment to date of death

Question 11

How much evidence do you need to show a treatment works?

Answer 11

• a large group of patients from bare places
  
  • so you would need to do a clinical trial

Question 12

What is a clinical trial

Answer 12

A clinical trial compares the effects of 1 treatment with another

• patients consent to trial
• patients agree to take drugs, to see whether the treatment works and is good
• some patients may not get the drug

Question 13

What happens in a single arm clinical trial

Answer 13

-ppts recieve same experimental treatment no control group recieving placebo
-effectiveness and safety of treatment assesed

Question 14

How would we know if the treatment in a single arm trial is effective or works

why can this be a problem

Answer 14

• from other data
  
  • historical data you can look at the other 5 year survival rate
• however people from historical patients may not have had good supportive care, they may have had smaller tumors in the other groups

Question 15

What is the aim of a RCT

Answer 15

to directly compare new treatment against standard
-randomise eligable patients, allocate them to either arm
-treat them
-then follow them up
-compare what happens

Question 16

phase of trial

Answer 16

• phase 1: trying to fine the right dose, that doesn’t cause extreme side effects (looing at pharmacology)
• phase 2: once you find a safe and hopefully effective dose, you try out treatment for a group of patients to see if there’s a shrinkage in tumors
  
  • quite quick, small amount of patients (atp wanna work out if you should do the trial)
• phase 3: looking at survival times (long term efficacy), aim is to compare standard treatment to new one
  
  • 250-1000 patients

Question 17

Hazard ratios

Answer 17

> Measure of relative difference in survival between the NEW and STANDARD treatment

• so risk of dying on new treatment compared to old

HZ= risk of death on NEW/old

results
1= no difference

less than 1 means new treatment decreases risk of death

more than 1 means new treatment increases risk of death

Question 18

Stratified Medicine Trial Design to Incorporate Potential Predictive Biomarkers

what is it

Answer 18

from what i understand, you think you have a particular biomarker say havin a k-ras mutation, and then your like hmmm do people respond differenlty to treatment

so you would run 2 RCTs in each biomarker group to see if new treatment was beneficial in either biomarker +ve or –ve.