News, Agenda, Information of recent congresses
Gene Mutation Responsible for WM identidied by Dana-Farber Researchers
Researchers at Dana-Farber Cancer Institute have identified the first gene mutation that may characterize the vast majority of Waldenstrom's macroglobulinemia (WM) cases.
More specifically, research led by Dr. Steven Treon and Zachary Hunter, and funded in part by the IWMF, discovered:
A single gene mutation in 90% of Waldenstrom patients whose DNA was sequenced.
This mutation will help to distinguish WM from other types of lymphoma, multiple myeloma, and MGUS.
This mutation produces an abnormal protein which activates the NF-kB pathway, essential for the growth and survival of WM cells.
When researchers shut down this pathway with drugs that blocked the abnormal protein, the tumor cells died. Healthy cells did not.
This suggests that new, effective treatments that target WM tumor cells directly are on the horizon.
This doesn't yet mean a cure, but we are one step closer than ever.
Dr. Treon announced the new findings on December 12th at the American Society of Hematology's (ASH) annual meeting in San Diego. A copy of the Dana-Farber press release of the ASH presentation is on the IWMF website.
The importance of this discovery is enormous. Quoting from the Dana-Farber press release, "Drugs that block the abnormal protein or other proteins in the NF-kB pathway could, theoretically, short-circuit the disease process in many patients. Some of these drugs already exist, having been developed for other conditions. Treon and his colleagues are currently working to develop others and are testing them in experimental models."
Update on WM 2012 by Dr. Shirley D’Sa can be found at http://bit.ly/P2dXu6
Important issues on WM have been the focus of attention at a series of International Workshops that have been held since 2000, the most recent being in Newport, Rhode Island, USA on 23-26 August 2012. This article describes some of the recent advances that were presented at Newport 2012.
Award for Lia van Ginneken
The Dutch Foundation "Stichting Eerlijke Geneesmiddelen Voorziening"(Fair Supply of Medicines) has awarded the chair of EWMnetwork Lia van Ginneken with the "Cees van Bezooijen Award". She was awarded for her great efforts in promoting patient rights and access to treatment in Europe and the Netherlands during the last eight years.
Awards for Dr. Veronique Leblond and Dr. Pierre Morel
At the 7th International Workshop on Waldenström's Macroglobulinemia two members of the EWMnetwork Medical Advisery Board were awarded with prizes: Dr. Veronique Leblond was the recipient of the 2012 Waldenstrom Award, Dr. Pierre Morel was the recipient of the 2012 Robert Kyle Award.
Gene Mutation Responsible for WM identidied by Dana-Farber Researchers
Researchers at Dana-Farber Cancer Institute have identified the first gene mutation that may characterize the vast majority of Waldenstrom's macroglobulinemia (WM) cases. See Dr. Steve Treon's Powerpoint Presentation below.
For more information click here for the page "News".
WM Community is on line !!
This is the link to find the site http://www.rareconnect.org/en/community/waldenstrom-macroglobulinemia. Here you can learn more about WM, discuss with other patients and find information and resources.
New European Commission proposal would give patients faster access to medicines. The European Commission has issued a proposal that would cap the amount of time European Union Member States have to take a decision on the pricing and reimbursement of newly authorised medicinal products. The proposal would allow 120 days for decisions on innovative medicinal products as a rule, and for generic medicinal products only 30 days, instead of 180 today. The Commission also proposes strong measures designed to enforce the time limits. The Directive proposal would repeal and replace Directive 89/105/EEC, relating to the transparency of measures regulating the prices of medicinal products for human use and their inclusion in the scope of public health insurance systems. Orphan drugs, frequently expensive, are often slow to be brought to market, as their high prices can engender lengthy negotiation and health technology assessment procedures. The Directive proposal is sure to be subject to careful scrutiny by the rare disease and orphan drug community and could be very good news for rare disease patients across Europe as well as the professionals treating them.
More information on: http://ec.europa.eu/enterprise/sectors/healthcare/competitiveness/pricing-reimbursement/transparency/index_en.htm
Information of recent congresses, meetings etc.:
EWMnetwork meeting, London, 16-03-2013, impression and presentations:-
- Phil Manning, An Impression, click here
- Lia van Ginneken, PP: some EU institutions relevant for WM, click here (PDF 211 kB)
- Marta Campabadal, PP: RareConnect, and the WM on line community in 5 languages, click here (PDF 1.43 MB)
International Patient Forum on WM, London, 17-03-2013, impression, summary and presentations:
- Phil Manning, Impression of the International Patient Forum click here
- Phil Manning, Summery of the International Patient Forum click here
- Dr. Monique Minnema, PP: Diagnoses and Treatment of WM in The Netherlands. (PDF 600 kB) click here
- Dutch Guideline for Diagnosis and Treatment of WM. (PDF 167 kB) click here
- Survey on diagnostic methods and treatment strategies used in patients with WM in The Netherlands (2013). (PDF 167 kB) click here
- Questionnaire on Peripheral Neuropathy (Dr. Minnema e.a.) click here
“Questionnaire that you can fill in if you notice any unusual tingling, weakness or pain in your hands or feet.
Early diagnosis and treatment offers the best chance for controlling your symptoms and preventing further damage to your peripheral nerves. It is advisable to fill in the questionnaire at the start of each treatment cyclus and discuss it with your doctor”.
Annual Symposium of CMWP (MM&WM Patient Association) the Netherlands, April 13 2013.
The WM part of the symposium was attended by around 100 patients and their partners. The Dutch WM specialist Dr. Monique Minnema from University Medical Center, Utrecht, gave an interesting Power Point presentation of the present state of diagnosis and treatment of WM. There was time for questions and of course people wanted to learn more about Ibrutinib. Dr. Minnema explained that this medicine is very promising. However, even if all goes well, it will take time before this medication will be available in Europe, since the necessary procedures are very lengthy.
Preceding the Symposium CMWP’s Annual General Meeting took place.
A historic moment at this annual meeting was the decision to dissolve the 30 year old CMWP and to form a new hematological patient organisation: Hematon. This new organisation is a merge of all four existing Dutch hematological patient organisations: CMWP (MM&WM), SCL (Leukemia), LVN (Lymphoma) and CST (Stem cell transplants )
The reason for merging is the fact that all concerned are convinced that joining forces offers huge benefits to the patients and their families, particular in the field of advocacy, innovation, and administration. However, all parties pledged that they will keep their own identity with respect to disease specific topics like support groups, newsletters, web-sites etc.
More information (in Dutch language): www.waldenstrom.nl
7th International Workshop on Waldenström's Macroglobulinemia, Newport, Rhode Island, August 23-26, 2012.
- Update on WM/Newport by Dr. Shirley D'Sa Click here.
- Click for an overview of this workshop at www.wmworkshop.org and you will find information about Faculty, Program, Abstracts, Sponsors and Media Gallery. A synopsis of this workshop is expected soon, link will follow.
17th IWMF Annual Ed Forum, Philadelphia, PA, June 2, 2012.
On www.iwmf.com/services/ed-forum.aspx you can find the Special Bulletin Ed Forum 2012 and order the 2012 Ed Forum DVD.
Dr. Steve Treon's Powerpoint Presentation 3rd International Patient Forum on WM, London, Sunday 11th March2012.
You can download Dr. Steve Treon's Powerpoint Presentation on the Genetic Basis and Therapy of Waldenstrom Macroglobulinemia and also other information and pictures of the Forum on www.iwmf.com/services/ed-forum.aspx
The 6th symposium on WM in Belgium (Dutch speaking) took place on 19 November 2011 at the University Hospital in Brussels.In the capital of Europe nearly 40 interested people come together to attend the sixth symposium on WM: patients, partners, caregivers. Dr. Fabienne Trullemans, connected to the above mentioned hospital as a hematologist, starts with a very clear exposition of WM, the signs and symptoms, diagnosis and treatment. Newly diagnosed are reassured. All patients receive information about the current therapeutic arsenal available to treat the disease.
After lunch the audience can hear a very interesting presentation from a lawyer working at the research department of the Flemish League against Cancer: ‘The cost of care for hematologic disease for the patient’.
Satisfied and with positive feelings everyone goes home, ready to be back next year for the seventh symposium. More information: www.cmp-vlaanderen.be
The 2nd symposium on WM in France (French speaking) took place on September 24 2011in the "Plateforme Maladies Rares" at the Hôpital Broussais in Paris. NB. Waldenström France has 80 members ; 150 persons are connected to the blog on the website of the Medicalistes.
In Paris 43 persons (including 25 patients) coming from all over France attended the meeting which took place between 11.30-18.00 hours. Each participant got a packet with information on WM and on membership of Waldenström France (costs € 20 a year, deductable from tax payment) and was asked to distribute these in their home town/department.
Professor Dr. Véronique Leblond, hematologist at “Groupe Hospitalier Pitié Salpêtrie” in Paris gave a very informative lecture about stemcell transplantation, (new) medicines, clinical trials in Europe, Guy Sherwood (IWMF) presented a €1000 cheque to WM specialist Dr. Xavier Leleu (Hôpital Claude Huriez , Lille).
More information: www.medicalistes.org/waldenstrom