lecture 19: community concerns and expectations

Question 1

What captures the imagination of members of the community re: stem cells?

Answer 1

• media headlines
• as scientists we have a responsibility to try and influence how our work is reported

Question 2

Are there many proven stem cell treatments available?

Answer 2

• no
• haemopoietic stem cell transplantation
  
  • leukaemia, thalassaemia
  • bone marrow transplant
• can give patient a drug to release the HSCs into the circulating blood and then collect blood
• cell separating technique used to isolate and purify HSCs
• reinfused into a patient
• have been used for 40 or 50 years
• treatments are very restricted in clinical use
  
  • AML, ALL, MDS, NHL, other leukaemia, CML, HL
  • i.e. really only treatments of blood or immune system
  • replacement strategy where new HSCs are added into their bone marrow
  • cells given back intravenously and they hone into the bone marrow to restore its regenerative capacity
  • numbers of these transplants are quite high

Question 3

What is the difference between allogeneic and autologous?

Answer 3

• allogeneic = from a donor
• autologous = from the patient

Question 4

What is the growth in ‘novel’ applications of stem cells?

Answer 4

• novel clinical trials (involving MSCs)
  
  • being investigated the most in clinical trials
• novel clinical trials (not involving MSCs)
  
  • foetal tissue
  • hESc
  • iPSc
  • umbilical cord
• non-novel clinical trials
• a long way from delivering on hype
• overall increase in clinical trials involving stem cells
• large proportion is refinements of treatments using HSCt

Question 5

What are some examples of companies involved in clinical trials to evaluate possible treatments with stem cells?

Answer 5

• mesoblast (ASX) bone marrow → cartilage, bone, heart
• Advanced cell technology
  
  • precursors from embryonic stem cell → rare type of blindness
  • stargardt’s macular degeneration and macular degeneration

Question 6

How can stem cell science be translated?

Answer 6

• demonstrated efficacy and risk assessment in preclinical models
  
  • peer review
  • clinical trials
  • proven therapy
  • this is a long and expensive process - can be very frustrating
• rationale and preclinical evidence of efficacy and safety
  
  • peer review
  • medical innovation, involving few seriously ill patients → passionate use
  • clinical trials when possible
  • proven therapy
• no rationale or preclinical evidence of efficacy
  
  • no peer review
  • stop

Question 7

What is the problem with Dr Google?

Answer 7

• many conditions will have impressive looking websites offering ‘miracle’ stem cell treatments
• very hard to work out what is legit and what is bogus
• more marketing than science
• implication of the magic of stem cells
• apparently they will know exactly where to go and how to work

Question 8

What are hazards of unproven stem cell treatments?

Answer 8

• outside clinical trials by non-experts
• lack of proven benefits
• endorsed by patient testimony
• risk of physical harm
• financial exploitation
• **diversion from existing proven therapies **
• disqualification from future clinical trials

Question 9

What are the findings of ‘Capturing experiences of Australians’?

Answer 9

• considered pro-active and well informed
  
  • did not seek advice from local doctor
• no choice but to travel abroad
  
  • australia ‘fallen behind’
  • condition deteriorating quickly
• aware possible risks and benefits
  
  • risks defined as financial
  • were happy to have minor improvements - did not expect to be cured
• high costs
  
  • financial and emotional
• all reported benefits - ‘it works’
• Lisa: “It came down to the worst that could happen was nothing really… we could spend our money and have gotten no result…”
• Natalie: “I have not regretted it for a day. It certainly made a difference and as I said, it’s given me hope…”

Question 10

What is the cost of ‘medical tourism’?

Answer 10

• more at risk than “doing your money”
• unexpected growths
  
  • bone fragments growing around her eye after stem cell treatment in the face
  • stem cell ‘cure’ boy gets tumour
  • stem cell treatment causes nasal growth in woman’s back: 8 years after treatment with olfactory stem cells
• death
  
  • forestdale man died after operation to help him walk again
  • europe’s largest stem cell clinic shut down after death of baby
• a few cases but not as many as we might expect
• but N=1 - not a proper evaluation

Question 11

Are unproven SCTs being offered in Australia?

Answer 11

• increasing number of doctors offering autologous “stem cell” or SVF treatment
  
  • usually liposuction derived but also peripheral blood
  • $9k+ per treatment (multiple encouraged)
  • offered for many conditions including osteoarthritis, autism, retinal neuropathy, MS, MND, and SCI
  • intra-articular and IV delivery
• marketed as ‘medical practice’

Question 12

How is this a regulatory challenge?

Answer 12

• it was the intention of the australian government through the therapeutic goods administration to regulate stem cells
• introduced new biological framework where stem cells were strongly included
• wanted to include a caveat where standard medical practice could continue on without having to go through this rigorous process
• however the way that it is written when it comes to cells and tissues is so broad that it has opened up this opportunity for these entrepeneurial doctors to start treating and selling
• at what cost?
• at what cost to the patient?
• what cost to the field in general?
• often extracted by not even quality scientists
• pulmonary ebolism?
• will there be a backlash?

Question 13

What is the support for stem cells vs perceived risk?

Answer 13

• most support
• lowest perceived risk
• expectations gap
• Stamina
• need to manage community expecations