International Pompe Association

The IPA has issued the following press release in regard to the film, Extraordinary Measures. (Download PDF, 393 kB)

 


Extraordinary Measures the Movie

A Discussion by Pompe Disease Support Groups

January 2010
For immediate release

Background

Extraordinary Measures is a new movie scheduled for release around the world starting in the US on January 22, 2010. Starring Harrison Ford, Brendan Fraser and Keri Russell it tells the story of a family struggling with a rare disease-the neuromuscular condition known as Pompe Disease (aka Acid Maltase Deficiency or Glycogen Storage disease Type 2).

The International Pompe Association (IPA) is a federation of nearly 40 national patient groups around the world who collectively support over 1000 patients and their families.

Pompe disease is an extremely rare, progressive neurological condition that affects people of all ages; the classical infantile form is particularly severe as it particularly damages the heart , respiritory muscles and skeletal muscle. Older infants, children and adults are affected to varying degrees and without treatment all may suffer from loss of muscle function and breathing difficulties. Quality of life is reduced and life expectancy is considerably shortened; most untreated infants do not live beyond their first birthday. Every patient has their own unique story, and every family is affected in its own way.

Extraordinary Measures

The film Extraordinary Measures focuses on the actions of one highly-motivated individual and his collaboration with a medical researcher. It shows how Pompe disease, a devastating illness, changes family life forever, something that is true whatever the severity or age of the sufferer.

 

It is a remarkable story about the man and his quest to find a treatment for his children – but it is NOT the story of the development of a treatment for Pompe disease; that is a misconception implied by the film and spread by other commentators.

The real story behind the development of an effective treatment involved close collaboration between families, researchers and industry and was underway long before John Crowley’s involvement. The presence of his company did not lead to the development or acceleration of a therapy for Pompe disease; that process was already making good progress elsewhere.

The missing part of the Hollywood story is that the development of a treatment involved an international Pompe community; this has been airbrushed out of the picture by the movie and the associated book. Since the early 1990’s IPA and its affiliated organizations have together been very closely involved with the research and subsequent development of an Enzyme Replacement Therapy (ERT) for Pompe disease. We have long worked with the Dutch team of researchers at the Erasmus Medical Centre (Rotterdam). These trail-blazing pioneers, proved the principle of ERT for Pompe disease, carried out the first successful clinical trial and led the path towards a commercial therapy. They did so on a shoestring budget, in the face of indifference from the medical establishment. It is therefore particularly disappointing that no mention is made of their contribution.

It is apparent by the end of the movie that the therapy is certainly not a miracle cure and it is for that reason that the Pompe community, through national support groups and the IPA, continues to work with researchers and industry towards future therapies. However, it is important to remember that ERT is helping hundreds of patients around the world today.

Research and patient communities fighting rare diseases such as Pompe yearn for publicity to help them reach out to the public, patients, famililes, medical professionals and politicians, and for that reason we are very grateful to this movie.

As a community we hope that this film will lead to a greater awareness of Pompe disease and thereby improved diagnosis of the condition. We hope that the information about the disease will help families quickly find support from both medical professionals and patient support groups, and that screening programs to accurately identify the disease will be given a high priority.

We also hope that the community at large will realize the critical importance of newborn screening – by far the best way to identify the disease so that timely treatment can limit irrepairable muscle damage, giving those affected the best opportunity to lead a normal life

Recommended Reading

IPA founding member Kevin O’Donnell is currently writing a chronicle of events that led to the development of the commercial Enzyme Replacement Therapy for Pompe disease. “Pompe Disease – The Real Story” can be read online at pompestory.blogspot.com

Whilst we would recommend that you read the whole story, if time is limited there is a search facility to locate subjects of interest.

Contacts

The IPA has connections with a large number of families touched by Pompe disease who are eager to share their stories; if you would like more information about our work or would like to interview these families please use the contact details below.

VSN ( Vereniging Spierziekten Nederland)
Lt. Gen. v. Heutszlaan 6
3743 JN BAARN
The Netherlands
Tel. +31-35-5480480/ 5480475
Fax: 00-31-34-5480499
IPA@vsn.nl
Allan Muir, Chairman
Phone: +44 1730 231554 Email: allan@pompe.org.uk
Website: www.WorldPompe.org

 


 

The release dates for the films are as follows:

  • Canada 22 January 2010
  • USA 22 January 2010
  • UK 26 February 2010
  • Egypt 3 March 2010
  • Spain 5 March 2010
  • Germany 11 March 2010
  • Romania 12 March 2010
  • France 17 March 2010
  • India 19 March 2010
  • Belgium 24 March 2010
  • Argentina 25 March 2010
  • Poland 26 March 2010
  • Sweden 9 April 2010
  • Finland 16 April 2010
  • Norway 16 April 2010
  • Australia 29 April 2010
  • Netherlands 13 May 2010
  • Singapore 20 May 2010