Tag Archives: Clinical trials

What is your number?

Last night I had the honour to speak following the AGM of the Canterbury Medical Research Foundation (CMRF).  The CMRF are a fantastic organisation I’ve talked about before.  They are also one of three sponsors of my current research fellowship.  What I talked about was volunteers and clinical trials.  Two days ago the world celebrated Clinical Trials Day in honour of James Lind who in 1747 took some men aboard a ship and started to feed them citrus fruit to see if the Spanish (who had less scurvy than the British) were on to something.  I don’t know if he used volunteers or not, but I do know that since then millions of people have volunteered to be part of clinical trials.  I salute those volunteers.  I work with people who present to the Emergency Department with chest pain or are seriously ill in the ICU.  These people are vulnerable, often scared, and are asking “Am I having a heart attack?”  Yet, despite this, when approached and asked to participate in a trial they very rarely say no.  This shows to me an incredible generosity of spirit & a heart-warming willingness to do something for someone else, even when that someone else is a mythical patient some time in the future.  I salute those volunteers.  They are my heroes.

I didn’t record the talk last night, but have tried to reproduce it this morning and present it to you now. Click HERE to access from Researchgate. It is not the same as with an audience as some of it was interactive.  However, I hope you enjoy it.  It is about 20 minutes long (100Mb) and deliberately targeted at a lay audience.

logos w uni

The patient volunteer

Me

Curious

Ignorant

Seeker of understanding

Hunter of truths

 

Volunteer

Hurting

Vulnerable

Seeker of healing

Hunter for relief

 

Volunteer

Willing

Generous

Seeker of understanding

Hunter of truths

Hope for Type I diabetics

Living Cells Technology have announced the beginning of phase II trials of their product for helping Type I diabetics (they call it DIABECELL).  The idea behind the technology is to inject cells (called islet cells – normally present in the pancreas) that can sense glucose levels and release insulin.  LCT has managed to isolate these cells from pigs and encapsulate them in a way that the body’s immune system will not react to them – no anti-rejection drugs necessary.  Being xeno-transplant it has gone through quite a process even to get to the stage of Phase I trials.

This is pretty cool and I sure hope it all works out.  A Phase I/IIa (safety trials and first stage efficacy trial aimed to find an optimal dose) has been conducted at Middlemore hospital in New Zealand and another is underway in Argentina.  A press release in September announced the success of the Middlemore trial:

The New Zealand dose-finding trial was led by Dr John Baker at Middlemore Hospital in Auckland and saw 14 patients treated with a single implant of DIABECELL at doses of 5,000, 10,000, 15,000 and 20,000 IEQ/kg (islet equivalents per kilogram of body weight). The trial demonstrated that DIABECELL is a safe and effective treatment which results in: 

  • a statistically significant reduction in unaware hypoglycaemic events at doses of 5,000 and 10,000 IEQ/Kga
  • trend to reduction in HbA1c
  • improvement in patient-reported quality of life 

Note – HbA1c is just a form of haemoglobin (from the blood) proportional to the blood plasma glucose concentration.

Positive preliminary results from the 9 person Argentina based Phase I/IIb trial were announced.  The difference from the NZ trial is that two doses were given 12 weeks apart.  Followup has not been complete.

While this is all good news, I see no indication that the research has been peer reviewed (nothing in the Press release and I looked on PubMed as well).

The announcement this week is of a phase IIb trial of 20 patients with a dose of 10 IEQ/kg.  The announcement mentioned the trial will be in Argentina with 20 patients.  This is a little different from a September press release which mentioned 15 patients. The hope to metamorphosise the Phase IIb trial into a Phase III trial with an additional 10 patients from NZ.  A Phase III trial is to confirm effectiveness and monitor side effects – it is intended as the final step before regulatory approval.

In the press release the CEO announced.

“We remain on track to meet our goal of completing clinical trials of DIABECELL by 2015 and having a product commercially available by 2016.”

When I read this I thought “gulp – that is pretty positive given trials have yet to be completed.”  I gulped twice when I realized the trials to date have yet to be peer reviewed (although some results are public).  My third “gulp” is that these trials are all in small numbers – especially the planned Phase III trial.  That is not to say that I have any suspicions about the results or the technology, merely I worry about the juxtaposition of “completing clinical trials” and “commercially available” without the explicit interim statement of “and if the product is shown to be safe and effective.”

When doc don’t know

I had my 13th general anaesthetic yesterday for the same thing!  It was, yet another, 50/50 call by the surgeon as to whether this new procedure would work.  50/50 calls are not unusual in medicine – to the contrary they are probably the norm.  Most of us have had the experience of having a doctor state they were not sure of the diagnosis (eg. viral or bacterial?) or of the best treatment option.  In the past they may not have told us, just applied their own prejudice and made a decision for us.   Nowadays, we are given the option and expected to make the decision ourselves…daunting!  One wonders why they are paid the big bucks sometimes!  The reality is that very few of us are well prepared to make the decision ourselves.  Few read the research and even fewer have the skills to understand what the numbers mean (a failing of our school system which places more emphasis on algebra than statistics).  So, is there anything we can do?

The number one thing we can do is insist that we become part of a trial.  Two reasons for this, if we are part of a trial then we are more likely to have better health outcomes (there is research to show this, in the meantime, you will just need to believe me).  Second, only by there being trials will answers be found as to which treatment or diagnosis is better/correct. It is a no-brainer really!

Unfortunaetly there are many barriers to trials – labourious ethics proposals (not all bad, just time consuming), lack of willingness in the medical profession (no time, no financial reward), and too few people engaged in analysing the results (my job). This could change if the politicians insisted that all medical professionals be engaged in research because it is a fundamental right of patients to receive the best health care possible!

To summarise:

Being  in a trial is a right.

Being in a trial means better health outcomes

The vision is for all patients, all of the time, to be enrolled in a trial.

As for me,  I read the research (poorly done), I made a decision based on a balance between no op and certain future multiple operations or having the op and if it were not successful more ops with possible additional problems .  I chose hope rather than the status quo.  I suggested a trial but there was no time to get ethics sorted…I may just write a trial protocol for the surgeons and if I can perrsuade them to do better.