Thoughts On Stem Cell Research & Treatments

Many will have read about the story earlier this month of a young woman with cystic fibrosis, Roisin Kelleher, who travelled to the Dominican Republic in order to undergo a controversial stem cell procedure. Here, our Chief Executive, Ed Owen, reflects on the difficult issues the case raises and why the Trust cannot recommend others pursue the same course:

There’s been much interest and debate on the use of stem cells as a potential transformational therapy for cystic fibrosis in the wake of publicity around Roisin Kelleher’s story.

Roisin is 20 years old and lives in Kent. She is dependent on oxygen, has been recently diagnosed with pulmonary hypertension and has been told by her clinical team that a lung transplant is probably the only way to prolong her life. Her mum Anntoinette told me when I spoke to her this week, “our lives caved in” at the news and they were desperate to find alternative routes to bring hope for her daughter.

After undertaking a great deal of research on the internet and through other networks, Anntoinette came across the work of a Florida-based physician, Dr Zannos Grekos, who has been promoting a particular stem cell therapy for a range of debilitating conditions, including heart and lung failure. Under his procedure, patients travel to the Dominican Republic where stem cells are introduced into the body intravenously.

Roisin and Anntoinette decided to give it a chance, and raised the funds they needed to travel to the Dominican Republic and undergo the procedure. Roisin is now back home and you can read her personal story on her Facebook page, ‘Breathe Hope for Roisin’.

So what is our view of this particular therapy? It is the question we have been asked by many, including some in the same situation as Roisin who, inevitably, are asking themselves whether they too should seek to undergo this procedure as a way to bring hope of future life.

In answering this question it is important to start by saying that all of us understand the desperation felt by the Kellehers. Theirs is a heartfelt story experienced by many other families touched by cystic fibrosis – and all of us wish Roisin well over the next few weeks and months. Like every other member of the wider cystic fibrosis community, they deserve and should receive full support and love from us all.

But the Cystic Fibrosis Trust does not recommend that others follow their example because it is a procedure that has not undergone the minimum essential procedures to guarantee patient safety. Indeed, the very reason it is carried out in the Dominican Republic is because it is not authorized by the US regulators, the FDA.

I have been in contact with a number of cystic fibrosis experts in the UK and in the US over the last few days, and none believes that this procedure has any scientific or clinical basis. It should also be pointed out that, in 2013, the Florida Board of Medicine removed the medical license of Dr Grekos for committing medical malpractice in performing an “unproven” stem cell therapy on a patient who later died. The board's vote was unanimous and without discussion, although I understand he may be appealing this decision.

I admire Anntoinette’s spirit and deep love for her daughter.  She accepts their decision was a “leap of faith”, and is also keen to point out that Roisin continues to take her existing treatments and is still going through a transplant assessment here in the UK.  She also understands the position the Trust takes on this issue.

And what we all very much agree on is the need for more research into the use of stem cells in cystic fibrosis.

For all the professional scepticism about Dr Grekos’ procedure, there is a great deal of enthusiasm within scientific and clinical circles for the longer-term potential offered by stem cell research. The Cystic Fibrosis Foundation in the US is looking to invest heavily in this field  and we, too, are exploring some innovative areas of work in this area which we hope to announce soon.

This is not work that is going to bring potential clinical therapies soon, and we are some way from even early stage clinical trials. But it is a genuinely exciting area as part of our mission to beat cystic fibrosis for good, and we will not stop until we get there.

In the meantime, our hearts go out to Roisin at this time, and to all those families facing the daily battle to ensure their loved ones remain with them. You are, quite simply, our inspiration in the fight against this cruel condition.

We are continuing to look into this issue, as we do with all developments in cystic fibrosis care and research. Please do keep an eye out on the Trust’s Facebook, Twitter and website, as well as this blog for updates.