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News and EventsPlease refresh or reload this page to ensure you are seeing the most up to date information
g For Your Diary - Forthcoming Events and Notable Dates
Please email the Webmaster with any additions to this list. Please email the Secretary with any changes to addresses or contact details. g 2008 MPS Conference in Christchurch NZ12th National Australian MPS and Related diseases conference - Celebrating their silver Jubilee Incorporating 1st Australasian Lysosomal Diseases Conference Please click on this link to access a Conference Registration Packet Lysosomal Diseases New Zealand is very proud to be hosting the 12th National Australian MPS and Related Diseases conference and the 1st Australasian Lysosomal conference in Christchurch New Zealand, November 20th -23rd 2008. Click on this link to view LDNZ Conference information. This is an especially significant year for the Australian MPS Society as they will be celebrating their 25th Anniversary of supporting families in Australia and New Zealand. LDNZ is accepting expressions of interest now, please e-mail Jenny Noble jenny.noble@xtra.co.nz with your contact details so that information can be posted to you as it comes to hand. Please click on this link to access a Reservation Request Form for Accommodation
The Kiwis will host an ANZAC MPS Conference in the true spirit of the ANZAC tradition. New Zealand and Australia are working together to improve scientific knowledge and clinical care for Lysosomal diseases. LDNZ will host a joint New Zealand and Australian conference in Christchurch in 2008. The first joint conference to be held in New Zealand will take some of the load off the Australian MPS Society which has run these conferences for scientists, clinicians and families every two years for the past few decades, with New Zealand families often attending the Australian events. In a statement released by LDNZ Chairman John Forman it was confirmed that LDNZ would organise the event and host their Australian cousins in Christchurch for the next biennial conference. “What makes it extra special is that it will be the 25th anniversary of the formation of the Australian MPS Society. LDNZ will be honoured to recognise their important anniversary at this prestigious conference in New Zealand” says John. He went on to say “It is also a testament to the fine work done within the LDNZ back office lead by Jenny Noble. Jenny did the hard yards getting the proposal for this conference prepared and making the important connections that will turn this idea into a reality”. The conference is due to run over three days from 20 to 23 November 2008 and will include 25th birthday celebrations for the Australian Society, and guest speakers from New Zealand, Australia and the UK. “This is really important for LDNZ families as it will make access to such respected speakers as Professor Sillence, Dr Wraith and Professor Hopwood who are some of the worlds leading experts in Lysosomal Diseases, as well as focus on the significant contribution New Zealand scientists have made to knowledge of Lysosomal diseases over many decades.” says Jenny. Conference Registration Fees
Conference Program at a Glance
Conference Speakers
Read the latest LDNZ Newsletter g BioMarin Announces Program for ERT for Treatment of MPS IVA - Morquio A SyndromePlease click on this link to read BioMarin's Announcement. g MPS I Market Survey ResultsPlease keep posted, we will provide these results as soon as authorised to do so. g Morquio Conference (UK)Dear International MPS Societies, I have pleasure in attaching a PDF file of the programme for the first Expert Meeting on Morquio Disease (MPSIVA), to be held at the Northampton Hilton Hotel (UK), 29th – 30th August 2008, and the International MPS Network Meeting that follows it, on 30th – 31st August 2008. Please feel free to download and distribute as you wish. With kind regards, Christine Christine Lavery Chief Executive Society for Mucopolysaccharide Diseases Tel: 0845 389 9901, Fax: 0845 389 9902 MPS Society, MPS House, Repton Place, White Lion Road, Amersham, Buckinghamshire, HP7 9LP, UK., UK, www.mpssociety.co.uk, Registered Charity No. 287034 g Inspiring International Story of an MPS II Affected Adulthttp://www.speakwell.com/well/2004winter/simon.php Please click on this link to read Simon Ibell's story g Annual General Meeting 2008The AGM for 2008 was held on 13 April 2008 in Sydney. The Board and Committee are delighted that David Oliver has agreed to take on the role of President for the next year. The Committee offers its very grateful thanks to Vanessa Ede-Scott, Lillian Lodewyk and Louise Jessop who have stepped down from the Committee. The Committee welcomes Nicole Millis to the Committee. g International MPS Awareness Day - 15 May 2008To increase awareness of Mucopolysaccharide and Related Diseases, the international MPS Community has decided that we should annually hold an Awareness Day on 15th May. The first ever Awareness Day was celebrated on 15 May 2007. Those on our mailing list were sent bumper stickers and postcards to provide to family, friends and medical and other carers to help raise awareness of MPS. The Society would be interested in your ideas for how to promote MPS Awareness at our next MPS Awareness Day on 15 May 2008. g Strategic Planning for the Society for the next 5 yearsThe Committee of Management and Board of the Australian MPS Society are current working on a strategic plan for the Society for the next 5 years. A planning day was held on 21 April 2007 and a further get together was held on 16 June. Details of our strategic plan and reviewed Mission and Aims will be posted shortly. g Annual General Meeting 2007 The AGM for 2007 was held on 22 April 2007 in Melbourne. g Brisbane Conference 2006
ReportMucopolysaccharide & Related Diseases Society Aust. Ltd held their 11th National MPS Conference in Brisbane from 29 September to 1 October 2006. The conference brought together over 85 delegates from across Australia and New Zealand and two special guest speakers from the United Kingdom. Also in attendance at the conference were 46 children either affected with Mucopolysaccharidoses (MPS) or siblings of those affected. These children were looked after on their own specially designed program by 35 volunteer carers. The 11th National MPS conference theme was dedicated to forming and strengthening partnerships. The conference was about individuals who have MPS, families with children with MPS, friendships, happiness, sadness, much laughter and the sharing of advancement in research, treatments and information. Most importantly the conference was about the empowerment for all individuals and families in attendance whose everyday role is as a mother, a father, or a carer for these special people affected by MPS. MPS National conferences are very unique as they give those dealing with research and treatment a first hand chance to see the human side of the work they are doing. MPS conferences also provide a precious vehicle for individuals or parents to empower themselves by getting first hand knowledge from those with the most to give and to be able to take this knowledge back to their own local medical professionals. Families and individuals with MPS in attendance at this year’s conference benefited by having the opportunity to meet with other families or individuals with MPS, to share and discuss concerns and health issues and discuss ways to improve their life styles. The possibility of the inclusion of a chat room on the MPS Society’s web site was proposed, the committee of management will now look into this possibility so that families and individuals will be able to communicate frequently. Families and individuals with MPS were given ample opportunities to meet with professionals working in the field of MPS to discuss the latest advancement in research and treatment options that may be available to them to manage their child’s or own disorder. The children in attendance at the conference enjoyed the activities provided in the children’s program. They visited Movie Word and The Lone Pine Koala Sanctuary where they were all able to cuddle and be photographed with the koalas. Sunday morning was spent relaxing doing craft and painting followed by lunch at the local McDonalds Restaurant. Just a few photos of the conference have been posted below - See our Conference 2006 Photo Gallery for more photos.
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“Crossing Oceans for a Cure” benefit weekend saw families from Italy, America Venezuela, Canada and New Zealand present. Our speakers came from Australia, Belgium, Germany and America.
Photos: 1. Ashton Keddy I-Cell 2. David Sillence, Autumn Tobey and Kelly Crompton 3. Sergio and Maria Elena Cardenas 4. Zack Hagget, Andre Daniels, John Haggett, Marjory and Luis Catozzi (Click on any photo to see a larger copy)
On behalf of ISMRD, I want to thank the Australian MPS Society for their very generous support of Prof David Sillence.
Our conference was a huge success with 31 families attending, but for the first time ever in the History of Lysosomal diseases I am very proud to announce that we had 28 ML2 and ML3 families joining together on mass, with 3 families with Mannosidosis also present. We also had several families with MPS joining us and giving us their support.
For many of the ML families this was their first time in attending a conference of this nature and the first time they had ever met another ML family, and some of our families were newly diagnosed.. This meeting has seen the beginning of many friendships being made and much sharing of information. There were a lot of tears and much laughter. The isolation for ML families has finally been broken.
Our Drs did an incredible job. Prof Sillence arrived on Wednesday evening and spent all day Thursday seeing families and their children before making two presentations on Friday. Once again I saw him going far beyond the call of duty. I must also acknowledge the work of Drs LeRoy and Patterson. Many families commented on how accessible they were. These 3 men did an incredible job in seeing that each and every family went home with more information than they came with, and of course I cannot forget Stephan Tiede from Germany, and his work on Gene Mutations in ML, and the information and hope he has given all families with ML.
The feed back from the families has been absolutely incredible some of the comments are as follows.
Saturday 24th April saw the families heading off to ISMRD’s first ever major fundraiser. The day was cold in fact it started to snow, so there were very few walkers but people turned up just to register, pick up their tee shirts and leave their donations. How incredibly poignant that Penguins is our logo. The penguins had gathered in Michigan to raise funds for research only to have it snow.
If I was to pick one special moment in the whole weekend it would have to be all about the children. Seeing all our young ladies together just talking like normal girls was very special. Many of them are on Pamidronate and hearing them share how this drug has help them bought tears to my eyes. They were also very informative to other young ladies who have not yet started treatment. Some very special connections have been made with this group.
Language was no barrier for the children. Seeing Zack Haggett from America and Sergio Cardenas from Venezuela playing together are memories we will never forget.
The whole weekend has created many special moments for all the ML families, but I think for our Drs seeing so many ML children and young adults all together for the first time was an incredibly defining moment.
Stephan Tiede from Germany stated that we would continue in his work on Gene Mutations. Seeing all the children together made it all seem so real. He said he would no longer see the samples he works with as just samples.
ISMRD are unreadily grateful for the support given to this benefit weekend by the Australian MPS Society. It has given us the opportunity to raise awareness for these super orphan diseases and to raise much needed funds for research.
Jenny Noble Board Member ISMRD, The International Advocate for Glycoprotein Storage Disease |
Shire presents positive results of Hunter syndrome pivotal clinical trial at the American Society of Human Genetics Annual Meeting
Shire Pharmaceuticals Group announced 28 October at the Annual Meeting of The American Society of Human Genetics, further results of its pivotal clinical trial evaluating its investigational human enzyme replacement therapy, idursulfase, for the treatment of Hunter Syndrome.
The primary efficacy endpoint of the trial was a composite of two clinical measures – forced vital capacity (FVC) and 6-minute walk test (6MWT). As previously reported, patients receiving the weekly dosing regimen of 0.5 mg/kg of idursulfase showed a statistically significant difference (p=0.0049) compared to placebo. Results presented by Joseph Muenzer, M.D., Ph.D., of the University of North Carolina at Chapel Hill, indicated that patients receiving the every other week dosing regimen of idursulfase also showed a statistically significant difference (p=0.0416) compared to placebo when measuring the composite. Treatment with idursulfase was generally well-tolerated by patients in the trial.
Other results presented included urine GAG levels, liver and spleen volume, cardiac left ventricular volume, and joint range of motion.
Commenting on the trial results, Dr. Muenzer said “Data from this study are very
encouraging for patients and families living with Hunter syndrome. I am excited about the potential of idursulfase as the first treatment option for patients, once approved.”
As previously announced, Shire expects to file for regulatory approval of idursulfase in both the United States and Europe in the fourth quarter of 2005.
Trial Design
The AIM study (“Assessment of I2S in MPS II”) was a Phase II/III double-blind, placebo- controlled clinical trial conducted at nine sites around the world, including the United States, the United Kingdom, Germany and Brazil. The primary goal of the study was to evaluate the safety and efficacy of 0.5 mg/kg of idursulfase administered weekly compared to placebo. Additionally, the trial evaluated 0.5 mg/kg of idursulfase every other week compared to placebo. Ninety-six patients with Hunter syndrome were randomized to one of three groups with each patient receiving a total of 52 infusions of either idursulfase, idursulfase alternating weekly with placebo, or placebo. Of the 96 who enrolled, 94 completed the 52 week study and they all elected to participate in the open-label extension study of idursulfase at a dose of 0.5 mg/kg weekly.
Trial Results
Six minute walk test (6MWT)
In the weekly dosing regimen, the difference in meters walked was 35 meters compared to placebo (p=0.0131) and in the every other week idursulfase group, the difference in meters walked was 24 meters, compared to placebo (p=0.0732).
Forced Vital Capacity (FVC)
Model adjusted percent predicted FVC in the weekly group was 4.3% greater compared to placebo (p=0.0650); the every other week group showed no treatment difference in percent predicted FVC over placebo (p=0.9531).
Urinary GAG Levels
Urinary GAG levels were significantly lower in patients being treated with idursulfase in both the weekly or every other week dosing regimen compared to patients receiving placebo (p<0.0001). Among all 64 idursulfase treated patients, 26 patients had normalized urine GAG levels at week 53. None of the placebo patients had normalized urine GAG levels by week 53.
Liver and Spleen Size
Combined liver and spleen volume, as determined by MRI, decreased significantly from baseline to week 53 for patients receiving idursulfase weekly (-25.8%) or every other week (-23.7%), (p<0.0001 for both). Patients receiving placebo showed a volume increase of 0.3% in these organs over the same time period.
Left Ventricular Mass (LVM)
Of the 96 enrolled patients, 33 had an enlarged left ventricle at the initiation of the clinical trial (15 in the weekly group, and 9 each in the every other week and placebo groups). Patients in the idursulfase weekly group who had an enlarged left ventricle at the initiation of the clinical trial showed a mean reduction in LVM index of 14.1% following 52 weeks of treatment, compared with patients receiving placebo who exhibited a mean increase of 4.3% (p=0.1524). For patients treated every other week, there was a mean decrease in LVM index of 9.6% as compared to the 4.3% increase in the placebo group (p=0.2893).
Joint Range of Motion
There were no statistically significant differences between treatment groups for Global Joint Range of Motion Score, however there were improvements in elbow (p=0.0476) and shoulder joint mobility (p=0.0797) with weekly idursulfase compared to placebo.
Safety
Treatment with idursulfase was generally well-tolerated by patients in the trial. The most common adverse events observed were associated with the clinical manifestations of Hunter syndrome. Of the adverse events considered possibly related to idursulfase, infusion related reactions were the most common and were generally mild. There were two patient deaths during the study, both of which were considered unrelated to treatment with idursulfase. Of the 64 idursulfase-treated patients, 6 patients tested positive for IgG antibodies, and 2 patients tested positive for IgM antibodies at some time during the course of the study. No IgE antibodies were observed. No patient withdrew from the trial due to an adverse event considered related to idursulfase.
Proposed acquisition of Transkaryotic Therapies Inc.
Shire Pharmaceuticals Group plc announced July 27,2005, that the proposed acquisition of Transkaryotic Therapies, Inc. (“TKT”) was approved by Shire’s shareholders on July 27, 2005. The acquisition is now expected to complete on July 28, 2005.
Shire expects to pay approximately $1.6bn or $37 per common share in cash as consi
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