Superdoctors – Miracle cures

Robert Winston introducing the programme

Commenting in the second episode of the three part Superdoctors series that “one of the most exciting frontiers of our age is stem cells“, Robert Winston goes on to ask “how will these cutting edge technologies change the way that you, and I, and are children are treated?” (Start – 00:04:02). Stem cell therapy is at the beginning of its expected transition from the laboratory to the clinical application. The programme seeks to distinguish the hype from the genuine developments and to examine some of the hard decisions that need to be taken. Several of the key ethical issues associated with stem cell research have been considered in posts about other programmes (see for example Are hybrid embryos an ethical step too far? – The Big Questions and Bioethics Briefing – Stem cells). This episode, however, is particularly useful for consideration of two issues:

  • Public understanding of science and the management of expectations
  • Clinical trials and “therapeutic misconception”

Public understanding of science and the management of expectations

Robert Winston searching the internet for available stem cell treatments

Inevitably, there is a gap between the knowledge of scientist directly involved in a new biomedical breakthrough and the general public’s understanding of the issues. Unfortunately this difference allows for irresponsibly excessive and unrealistic claims from some researchers and clinicians regarding the current availability and effectiveness of treatments. As Winston puts it, this leads to a situations where “blind faith in unproven treatments often ends disastrously” (00:03:45 – 00:05:52).

By way of illustration, Winston meets with the parents whose son unfortunately contracted meningitis and fell into a ‘permanently awake’ coma (00:05:38 – 00:13:06). His mother could not accept the verdict of the doctors that his situation was incurable; “I wanted my son back”, she says. Searching the internet for treatments or cure, they discovered an American psychiatrist practicing in the Dominican Republic and an Argentinean clinic, claiming to have treated many diseases such as Parkinson’s disease, diabetes, multiple sclerosis and brain damage with stem cell therapy. The family travelled to both of the clinics so that their son could receive treatment with stem cell. The mother was convinced that there was some progress in his health, but having any possible spent over £180,000, their money ran out and their son eventually died. 

Robert Winston and the consultant brain surgeon examine the MRI scans

Robert Winston and the consultant brain surgeon examine the MRI scans

 
The Argentinean clinic showed the family MRI scans taken before and after treatment as evidence that there was real improvement in the health of their son. Unconvinced, Winston took these scans to a consultant brain surgeon (00:13:06 – 00:15:39). The consultant’s conclusion was that the treatment “appears to have done harm rather  than good, there is no new improvement at all after the treatments he’d had”.

Robert Winston (00:15:39 - 00:16:56) "What is shocking, is that of course the hype, the exaggeration, the certainty that we professionals who are involved in the stem cell field, people like me. The claims that have tended to allow to be fostered have contributed to this couples huge distress; and therefore to put these patients through this kind of trauma at huge expense is immoral. All the overblown claims about stem cells hide the genuine promise that stem cells hold, and the responsible work done ethically by doctors that might revolutionise the way medicine can treat people."

 
The following questions could be considered:
  • Do scientists have a moral obligation to ensure that the public has a realistic understanding of the current parameters of science and medicine?
  • Should scientists and doctors do more to manage patient expectations of how beneficial a treatment might be?
  • How do the media influence public understanding of science and medicine?
  • Should the regulation of health services be the responsibility of national or international authorities?

Clinical trials and therapeutic misconception

The remainder of the programme focuses on the potential of stem cells to treat severe heart disease (00:16:56 – End). Every year in Britain over a quarter of a million people suffer from a heart attack, caused when one of the branches of the major arteries that supply the heart with oxygen becomes blocked. Through a procedure called angioplasty doctors can re-open the blocked artery. This procedure, however, does not repair the sections of dead heart tissue that result from a lack of oxygen (00:16:56 – 00:19:40). It has long been an ambition of doctors to find a way of regenerating the damaged tissue of the heart and the hope is that stem cells will provide the answer.

Robert Winston speaking with Alec

Alec, now 77, had his first heart attack when he was 30 years old and has since had five heart operations, leaving him unable to walk more than 20 yards (00:19:40 – 00:21:15). Doctors here now say there is nothing more that they can do for him, and so he travels to Germany for stem cell-based treatment. When asked why he had made this decision he replies “I don’t know of anything else that would give me what I need, which is virtually a new heart. Unless you’ve got some hope what is the point? Even if it doesn’t work, I’d have tried.”

Winston visits Alec’s doctor to discuss his condition (00:21:15 – 00:24:35). It is clear that Alec has had every available treatment; his doctor believes that despite the unknown potential of this treatment, as long as it is done in an ethical sound manner, he approves of the trip to Germany. Winston, however, has some concerns, believing the trip might be too risky for Alec. Nevertheless, he admits that he would find it very difficult to tell Alec otherwise.

Robert Winston speaking with David

Another patient, David, also has severe heart disease (00:24:37 – 00:26:42). After having two angioplasty operations and a pacemaker fitted, only 30% of David’s heart works properly and it is too fragile to undergo a bypass operation. This has also left him unable to walk more than a very short distance. However both he and his wife believe that “you never give up hoping that something will come along and help him.”
Both David and Alec have their stem cells removed by a procedure called bone marrow aspiration. Alec knows he will definitely receive stem cell treatment, whereas David has a 50/50 chance.

Robert Winston (00:26:42 - 00:28:26) "We are now going to follow the stories of Alec and David, both with a very diseased heart.The promise of stem cells is their only remaining hope. They are going to follow two very different paths of treatment; that difference is at the heart of how medicine progresses. David will take part in a double-blind trial in London. Half the patients in the trial will receive stem cell treatment for their heart and half will have an injection of salt water, a placebo (Note: no one, including the doctors will have knowledge of this until the end of the trial). If the treatment works only those who receive the stem cells should show any improvement. That means that half of the very sick patients won't receive any treatment at all. Alec is so ill that he can't take part in a trial. So he's going to travel to Germany. And there he'll receive stem cells from responsible doctors. What we are going to see played out at this frontier of medicine is a serious ethical tension. Doctors sometimes make what seem to be heart-breaking decisions. Can sacrificing one patient be worthwhile to advance medical knowledge?"

The following questions are raised by this section:

  • In a double-blind placebo trial, why do only half the patients get the treatment?
  • Why do scientists keep secret the information of who has received the treatment from both the patients and the doctors?
  • How would a patient feel if they did not receive the treatment in the trial but later found that it did provide a benefit?
  • Do you think double-blind placebo trials are ethical? Suggest two arguments in favour and two against.
  • What alternatives are there to double-blind placebo trials? Why have clinical trials?

Robert Winston (00:41:55 - 00:42:44) "I can't help but feel desperately sorry for Alec; he's travelled hundreds of miles to come here. He is facing very clearly the end of a struggle with his condition. The fact of the matter is that you can see from his attitude that repeatedly today that he thinks of these cells as being ‘magic cells'. He keeps on saying ‘give me double dose'. ‘Give me those wonderful cells'. And so even though I am absolutely sure the doctors are doing their best to manage his expectations. The fact is; he perhaps has to believe that this is something that is going to give him some extraordinary affect. Maybe it will, but maybe it won't"

Additionally, this section (00:40:16 – 00:41:55) again raises the issue of patient understanding and the doctors role in ensuring this. 

  • What factors could have caused Alec to label stem cells as ‘magic cells’?
  • Should doctors make an extra effort to confirm that Alec understands the true benefits of this experimental treatment?

The thoughts above highlight the very important issue of ‘therapeutic misconception’ (discussed in the previous post Wit: a window of tensions in clinical trials). This issue may arise when a patient has no other treatment option and is faced with the choice of no more treatment or trying an experimental treatment. Patients that opt for the latter may believe that these experimental treatments have been specifically designed for them, when in fact it may have little or no benefit. This belief is often encouraged by doctors keen to try out the experimental treatment. Even using the word ‘treatment’ suggests a therapeutic benefit, when in fact this is not yet known.  When Alec talks about those ‘magic cells’, it suggests that he has an overestimation of their benefit, something also known as ‘therapeutic misestimation’.

Six weeks after the stem cell therapy in Germany, Alec believes he feels a little better, as he is not suffering with as much angina. He undergoes an echocardiogram to see if his heart’s ability to pump blood has got any better (00:48:05 – 00:55:35). The results suggest that there has been no improvement. Winston explains that since Alec knowingly received the treatment, rather than as part of a double-blind placebo trial, he may feel better for a number of reasons including the ‘placebo effect’ and not because of the stem cell therapy. In contrast, improvement in David’s condition after participation in the double-blind trial would be a more valid indication if the stem cell therapy outperformed placebo. However, six weeks after the start of the trial, David does not feel any better.

Despite this, David believes “things need to be discovered and invented and if it does me some good; great. But if other people can benefit from what the doctors have done to me, that’s equally as great” (00:55:35 – 00:57:13). This altruistic approach is difficult but at times essential for the progression of medicine, Winston (00:57:17 – end): “Medicine needs willing guinea pigs like David but it’s likely to be others who will benefit most. Like it or not the double blind placebo trial is the only way to answer the critical question about stem cell therapy.”

Superdoctors is an excellent resource for teaching about the intricate nature of patient expectations and the use of double-blind placebo trials. Superdoctors – Miracle Cures (TRILT: 00AC861F) was first shown on Thursday 28th August, at 21:00 on BBC1 and repeated on Saturday 13th September at 01:30.

Other useful sections

(00:28:26 – 00:31:06) When David has his bone marrow aspiration Robert Winston explains what stem cells are and what they do.

(00:31:19 – 00:34:51) This section shows how doctors inject the stem cells into David’s heart tissue

(00:42:44 – 00:48:32) This section shows how doctors use an alternative method to place the stem cells in Alec’s heart

(DJW)

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: