It’s a bit strange not to be directly involved in the workings of the AAT as a Trustee after more than forty years, since its foundation back in the Hammersmith days.  Still I very much look forward to supporting the Charity as it moves onwards into what will surely be an effective future. I knew it was high time to step down when I realised it is more than 15 years since I left the research scene and 10 since I left the clinical side. The pace of research and clinical practice is so rapid that one soon gets left behind with what is happening and I am happy to retire, I hope gracefully, and leave the future to more to  up to date and current experts to explain.

So much has been learned about the nature and treatment of aplastic anaemia since 1968 when I first became involved in trying to make sense of the rare and devastating disease, that it is easy to forget that there is so much more to do. On reflection there seem to have been a series of steps, some big some small, which led to the state where a previously usually fatal condition is now often managed successfully.

The success of current treatments

The steps that have led to today's successes:

  • First there was the identification of the condition clearly amongst other similar rare causes of bone marrow failure slowly leading to a more general awareness of the condition amongst clinicians and patients.
  • Then there were the early attempts at treatment using oxymetholone extracted from the Mexican yam and supplied by the pharmaceutical company Syntex.

This was an early example in my career of working in harmony with a pharmaceutical  group, in this case with the enthusiastic support of the medical director, Miriam Moore-Robinson. The collaboration was important  because it provided the impetus for a controlled comparison  with the bone marrow transplant regime developed by Don Thomas, Reg Clift, Rainer Storb and others in Seattle. The study also produced the first classification based on the severity of bone marrow destruction. The discovery of cyclosporin for facilitating engraftment of transplanted marrow came from the work of Jean Borel and colleagues at Sandoz in Basel, another great collaboration with industry.

The suggestion from Georges Mathé in Paris that AA might be an immune disorder was strengthened by the work of Bruno Speck in Basel showing anti lymphocyte globulin (ALG) could be effective and this became another treatment of great benefit to many patients who did not have a suitable donor.

Bone marrow transplantation

The first hesitant use of volunteer donors from the Anthony Nolan Fund is now a standard and international therapy for many. The founding of the European Bone Marrow Transplant Group, which collects and collates data in the registry  on all cases of AA irrespective of treatment methods, was another hugely important international initiative. None of these measures would have been effective without the expansion of support services, new antibiotics, anti viral drugs, transfusion services and so on.

What's been the role of the AAT?

The AAT provided the wherewithal for the UK to play a major part in these developments and will, I am sure, continue to support and encourage the work needed to go on to more understanding and success in treatment.

What does the future hold?

What more is there? Some of the initiatives seek to identify in greater detail of the nature of the autoimmune attack and to see if new understanding and manipulation of the normal immune response can lead to specific treatment  and perhaps helping to cope with some of the major challenges of transplantation.

There is a lot more known about the processes of blood cell production which may yet be of importance for AA. The fascinating discoveries about the genetic anomalies and their consequences in the inherited bone marrow failure syndromes  may shed light on the acquired genetic changes in AA.

I am confident that the AAT will continue to be a prime sponsor for the UK to keep its place in this global community working towards ever better treatment for AA and will bring the benefits of this collaboration in timely fashion for all our patients and their families.  I wish it every success and thank all its supporters, past and future, for their so important interest.

 Blog by Prof Ted Gordon-Smith