Dear Member,

I trust all is well with you and you can find some time to read this bumper edition of our charity newsletter. This is the last newsletter before Christmas and may I on behalf of the committee take this opportunity of wishing you a happy and peaceful time.

I mentioned in Newsletter 52 the Myositis Association of America conference in September which Irene, Paula and myself flew to Atlanta to attend. We booked in for the meeting in an orderly and friendly manner at 5pm and by 6pm we were standing outside the hotel in the pouring rain - yes, we left all the good weather behind and got caught up in the final stages of hurricane Isidora. There was a fire alert with an urgency to vacate the hotel. I would mention that we did not need any prompting! Atlanta fire service and police turned out in full force. An air conditioning extractor was faulty and pumping smoke into the fourth, fifth and sixth floors. Fortunately, the situation was not serious and by 6.30pm we were back in the hotel taking refuge in the bar. I trust you appreciate that we needed some sustenance to settle the nerves! Dr Fred Miller was already standing there in bare feet. He had left his hotel room to have a brief reconnoitre when the door shut behind him locking him out! Well I suppose it could have been worse and he was decently attired. Mind you, Dr Miller is the sort of character who would be completely unfazed by the sequence of events. Also evacuated was a meeting of Masonic people who were dressed in their grand costumes which added to a colourful if somewhat bazaar spectacle.

[pictures]

Many of the delegates attending the MAA meeting were in wheel chairs or walking with sticks. You can appreciate the relief on the hotel manager’s face when all was back to normal. One elderly gentleman I spoke to said he sat in his room and had a beer for he had come to the conclusion he would have more chance of being rescued by the fire brigade then if he tried to make it down the stairs.

In the evening we went to dinner and held a friendly and informal discussion with the MAA executive director, Robert Goldberg, chairman, Earl Klein and his wife, Joyce along with Dr Fred Miller. We discussed how the two charities could collaborate along with others in Europe. Medical professionals were already actively involved in these arrangements and this would be a natural development for relevant organisations.

The MAA were quick to respond to one suggestion, which was to have access to our web-site from theirs. This was implemented the following day. Bob Goldberg had just been appointed director to the MAA to continue the MAA aims already established and ensure initiation of its new research programmes and promote international collaboration.

The main topic on the ‘menu’ was to discover more about each other’s organisations and to discuss how to drive forward the possibility of establishing a core myositis association on an international level. This organisation would pool in all the organisations already established in various countries and would centralise sufferer information about the diseases and research. One of the most important considerations to make when establishing an international association of relevant bodies would be to ensure that there is no interruption to the current running of an individual country’s organisation. This is a very important issue, as each country has a different infrastructure of medical health care and support organisations.

Preliminary suggestions on how such an infrastructure could work to ensure continuity yet sustain individuality were discussed. The most favourable at present would be to nominate a representative from each country’s organisation to sit on a central association. The use of the Internet would make this possible for many of the matters involving a committee could be handled in this way. The factors of financial running costs and governance were not discussed. We also discussed the importance of research on an international level for we all recognised because of the rarity of the diseases this was the only way forward.

On Friday we all attended the session on ‘New Direction in Research’ given by Dr Fred Miller. This included talks on genetics, environmental triggers, immunology, molecular pathogenesis, clinical studies, new tools to access myositis disease activity, new combination of older drugs and biologic therapies.

There were many questions asked from the floor and a lively and interesting debate on these issues.

"Progress in myositis research has been disappointingly slow for a decade or so, but there’s reason to look forward to much faster progress in the near future. One very important development of the last few years is the dynamic collaboration of the scientists who are active in myositis research. A good many of the researchers and clinicians who treat and study myositis – rheumatologists, neurologists and dermatologists – are meeting to agree on ways to measure how well patients respond to treatment. Collaboration is an important step (whether defining terms or sharing clinical observations) and there’s another benefit as well: New, more precise definitions of improvement reached by consensus may mean that fewer patients will be needed to establish if a drug is effective in a clinical trial."

"New technology also makes clinical and basic research easier." Dr. Miller noted that doctors now have better ways to distinguish between damage caused by active disease and that caused by scarring. "It’s an important distinction in predicting whether a treatment will be effective. We can expect active disease to improve with treatment," Dr. Miller said. "Scar tissue, of course, will not."

Recently, Dr. Miller asked scientists all over the world to report on their investigations into dermatomyositis (DM), polymyositis (PM), and inclusion-body myositis (IBM). His summary, which will be continuously updated, appears on the MAA web site at www.myositis.org. Dr. Miller reviewed the most promising of these research developments.

"Researchers believe that myositis is caused by a number of genetic factors (those inherited from your parents) and environmental factors (those caused after birth, by stress, viruses, drugs, pollution and the like). Progress has been made in basic science that examines both of these areas."

"New genetic risk factors have been defined in particular ethnic groups, and some of these vary according to geography, confirming the presence of an environmental trigger. The genetic risk factors can’t be used as yet to diagnose myositis," Dr Miller said, "but they are helpful in understanding how the disease develops. Meanwhile, tests for genes that point to other muscle diseases help doctors avoid wrongly diagnosing myositis," he said. "On the environmental front, we still don’t know a great deal about what triggers the disease in those genetically at risk. Some important clues are found in ethnic groups who live in different geographical areas. For instance," Dr. Miller said, "exposure to ultraviolet light from the sun correlates with the proportion of DM in some parts of the world. Certain drugs, like statins and D-pencillamine, probably cause some cases of myositis. The more we look into these causes, the more we’ll understand how the disease develops and how we may actually prevent some cases."

"The fascinating way that the immune system is involved in myositis is being studied, and new autoantibodies and immune markers on lymphocytes and muscle cells are being described in children and adults with myositis. Scientists are researching ways to block these specific immune reactions as we move towards a more targeted treatment that doesn’t interfere with the entire immune system. Some particular immunology research shows promise."

"New studies have found new genes that are turned on or off in the muscle of myositis patients. Some of these genes may be related to the overproduction of interferons which themselves activate other genes."

"Like many of the males in this room, I have some of my mother’s cells circulating in my body," Dr. Miller told his session. "This phenomenon, called ’microchimerism’ occurs in myositis, and may be the rule rather than the exception in certain autoimmune diseases. This transfer of cells from mother to baby and from baby to mother during pregnancy and birth and from mother to baby during nursing – is known to happen in a higher rate in women with scleroderma and in children with myositis. In the case of children with juvenile myositis, it’s likely that stem cells from mothers are transferred and eventually appear in affected muscle and skin. Maternal cells are different genetically from the child’s cells and may cause an immunological attack on them. The finding of microchimerism has opened up an entirely new way of looking at autoimmunity, infections and cancer, since the chimeric cells may be either helpful or harmful depending on their surroundings and the nature of the disease."

"As with other autoimmune diseases, doctors are turning towards new combinations of older drugs, like prednisone with either cyclosporin or methotrexate or both, in a British study to determine which combination is best for PM and DM. Biologic therapies show promise, as they do in other autoimmune diseases. These are antibodies that look like human antibodies to our immune system. Doctors hope that these biologic approaches may more selectively block the immune system and result in less toxic treatments. Many of these are now FDA-licensed as new drugs to treat rheumatoid arthritis or cancer. Treatment of childhood-onset and adult-onset PM/DM with anti-TNF agents (Infliximab/Remicade or Etanercept/Enbrel), anti-complement antibodies (anti–C5 antibody h5G1.1) and anti-B lymphocyte agents (Rituximab/Rituxan) are being studied now in clinical trials to determine their risks and benefits."

"Because a few rheumatoid arthritis (RA) patients with cancer were cured of both their cancer and RA after destroying their immune systems and replacing them with new stem cells from their prior bone marrow, the concept of using stem cells to reset the ‘immunostat’ of patients with immune diseases has become more popular. Adults and children with very severe life-threatening myositis are undergoing irradiation, chemotherapy and autologous stem cell infusions to see if these new cells from their own bone marrow will differentiate into new lymphocytes throughout their body that will not attack muscles and other tissues."

"Oxandrin (Oxandrolone) – an anabolic steroid - has been recently shown to be relatively safe, but unfortunately to have a very minor effect on increasing upper body strength in IBM patients in the safety phase of study. More studies are needed to understand the long-term risks and benefits associated with this treatment."

"B-interferon 1a (Avonex) has been recently shown to be relatively safe but to have little effect on muscle strength in IBM patients. A Phase 2 (efficacy) trial, doubling the dose by intramuscular injection each week, is just finishing up in IBM patients. Results will be available early next year." Besides the new drugs being studied, Dr. Miller talked about the value of patient registries that record the natural history of disease. Large patient registries of adult-onset and juvenile-onset myositis volunteers help scientists better understand these diseases and to predict which cases are likely and unlikely to respond well to treatment. Spurred by greater collaboration and funding; supported by breakthroughs in basic science and technology, "We’re entering a new era of myositis research," Miller said. "The recent and future funding by MAA will have an enormous impact on high-quality myositis research. These factors combine to give hope to physicians and their patients for safe, effective treatment."

In the afternoon Paula attended a session on Managing Side Effects and Irene and myself went to a session on exercise.

The exercise session was given by Nancy Harden and took us through the Feldenkrais method. This is a method through awareness through movement and functional integration lessons. Nancy who had been unwell became frustrated by the experience of being on the different side of the fence. She decided to look at ways to help herself. Her philosophy is to not to be afraid to try something. She said, "Don’t give up after you first attempt, commit yourself to five or six classes to give your body time to make adjustments. Classes to try include Yoga, Ti-Chi Massage, Dance etc. Before you start you should know what you want from it. You should go slow go gentle and be easy with yourself and listen to your body. You should be able to feel the movement but not pain stress or discomfort and take care of yourself, your comfort and well-being." Nancy then took the whole class through some exercises using the Feldenkrais method. Even those on scooters and wheelchairs were able to take part. In brief you focussed on an area on the ceiling that you could comfortably look up to without causing pain. You then performed a few gentler exercises and at the end you looked up again and found that you were focussing on a different part of the ceiling further back then your first spot without feeling any discomfort. To Irene’s surprise she found it did work for her! And further development of the regime over time would have proved helpful. For me, I found it all too confusing and confess to not taking part but was more interested in the actions of others! If anyone is interested in the Feldenkrais method please contact, Feldenkrais Guild of North America, 3611 SW Hood Avenue, Suite 100, Portland OR97201 or www.feldenkrsia.com

The managing side effects session was presented by Dr Steven Miller and the following is Paula’s report.

Dr Steven Miller’s talk centred on managing the side effects of corticosteroids. Corticosteroids have a therapeutic benefit in reducing inflammation and thus are prescribed for dermatomyositis and polymyositis patients. Unfortunately, there is a long list of unwanted side effects that prednisolone can induce. Dr Miller emphasised the importance of taking preventative measures to prevent osteoporosis, a common side effect associated with long-term use of steroids. Calcium and vitamin D supplements should be taken in addition to those obtained from a balanced diet to ensure sufficient intake. Exercises to maintain strength and balance will also minimise the risk of osteoporosis and associated weakness which may potentially lead to a fall and a broken bone. Dexter scans measure bone density and are useful in patients on long term steroid treatment as these scans can monitor the effect steroids are having on bones. The frequency which these scans should be performed depends on an array of factors, including, size of person, family history and age of person. Ideally these scans should be performed prior to treatment as well as during treatment and after treatment (especially if you have been on steroids for many years).

We all attended this session which gave information of board members, how to become a board member and an explanation of the role of board members. It was a general information session on how to lobby senators, co-ordinate an awareness programme and ways of promoting the organisation. Bob Goldberg talked of the main focus for the next year, research, international collaboration with doctors and organisations.

[picture] Earl Klein, Les, Irene & Paula Oakley, Joyce Klein and Bob Goldberg

In the morning the medical panel addressed the first session.

Dr Oddis - Clinical and Treatment Update on the Inflammatory Myopathies.

Dr Rider - Similarities and differences between Adult and Juvenile Myositis.

Dr Sontheimer - Skin problems experienced by Dermatomyositis patients.

Dr Miller - Research.

The following are preliminary reports from this session of Dr Oddis, Dr Rider and Dr Sontheimer talks. (Dr Fred Miller’s report has already been printed earlier in this newsletter).

"Adults and children can both be affected by dermatomyositis (DM) but with several differences," says Dr. Lisa Rider. Dr. Rider addressed the crowd after breakfast on Saturday morning at the Annual Conference in Atlanta. She joined Drs. Oddis, Sontheimer, Miller, and Bradley as a part of the medical panel there to present and answer questions on specific topics. Dr. Rider is a well-known and well-respected paediatric rheumatologist, working with children affected by myositis.

"DM affects adults and children at different rates, hitting five to ten adults per million but only one to five kids per million. In adults, the average age of onset is 30 to 50 years; 7.5 to 9 years in children."

"Autoantibodies found in children and adults are the same but are found in different numbers. Children have less anti-synthetase and myositis-associated autoantibodies than adults. Children tend to experience calcinosis more often than adults, with almost one in three JDMS patients affected. With aggressive treatment, calcinosis is becoming less of a factor. Ulcers, diarrhoea, constipation, and other stomach-related problems affect children, but medicines are available to treat these symptoms."

"Adults with DM may have lung problems, but children do not often have any lung problems at all. Fortunately, heart problems are not common in adults or in children."

"Children can also have polymyositis (PM), but this is far less common. DM causes muscle weakness because of swelling in the muscle’s blood vessels. The muscle loses some blood supply, so the muscle wastes away or fails to grow. In PM, the swelling attacks the muscle fibres directly."

"In one study of 375 children, researchers found that 80 percent (or 4 of 5) of the children with myositis have DM, while 10 percent (or 1 of 10) have polymyositis. The remaining 10 percent have some other form. In adults, DM and PM occur at about the same rate."

"When your child becomes ill, you want to know what caused it and how to make them better. There are several theories about what triggers myositis but no known cause. There are factors in the environment that are similar for both adults and children: viruses, bacteria, medicines, foods, and others. Some do differ though, including different medicines taken by adults and children and job-related stress and risks that affect adults. Hormones and genes may be common in all DM patients, so these are being studied further."

"For treatment, corticosteroid medicines like prednisone are often tried first. Children begin with a higher dose than adults usually do. Children tend to accept methotrexate better than adults. With all medicines, children and adults take different doses for different periods of time. Some medicines may affect children’s growth, so doctors do not like to keep children on these medicines for a long period of time."

"Physical therapy exercises include range-of-motion, strength, and endurance training for adults and children with DM. The doctors will decide which exercises each DM patient is allowed to do at a certain time in their disease."

"Most importantly for adults and children, the survival rate is high – somewhat higher in children. Children will more likely have some type of remission or period of one year with no symptoms of the DM. About one in three kids will have a full remission; between one in three and one in ten will have partial remissions. Children tend to have less disability, possibly because they begin with better strength and fewer other complications."

"Overall, adult DM and childhood DM are very similar. More studies looking at adults and children together will help doctors better understand both conditions."

"It’s a complex and widely varying collection of symptoms that we recognise as the inflammatory myopathies," said Dr. Chester Oddis, "but there are some common symptoms: muscle weakness with inflammation, involvement of the whole body and – as yet – an unknown cause or causes. We group the diseases together in several categories: adult PM, adult DM, JDM, malignancy-associated myositis, myositis overlapping with other autoimmune diseases, and IBM."

"The unknown cause is part of the formal name for myositis: in ‘idiopathic inflammatory myopathies,’ ‘idiopathic’ means ‘unknown’. Actually," said Dr. Oddis, "we know some of the causes: genetic predisposition and environmental agents such as infection, toxins (harmful chemicals) and exposures through food and drugs that aren’t ordinarily toxic to everyone."

"Although the weakness is in the muscle tissues, there are other systems myositis attacks," Dr. Oddis said. "It can target the skin, as in dermatomyositis, the joints, especially where the skin is stretched, and the lungs, causing shortness of breath and scar tissue. Other targets are the gastrointestinal tract, where it affects the swallowing mechanisms, and can cause ulceration’s."

"We do know that it’s an auto-immune response that causes the symptoms," Dr. Oddis said, "meaning that the body’s immune system attacks its own tissues."

Dr. Oddis reviewed the clinical features of IBM: "Onset usually after age 50, more common in males, muscle weakness in hands and feet as well as arms and legs, quadriceps prominently involved, slow progression and difficulty in swallowing. The latest reports indicate that there are probably a number of causes, with ageing as a prominent feature. Other features: accumulated protein in muscle fibers, cholesterol within the muscle fibres and a role – though less clear than in PM – of autoimmunity. Some of the difficulties in treating IBM are the confusion of IBM and PM, a number of incorrect diagnoses, and inadequate design of treatment trials, making it difficult to tell if a drug is really effective. Although IBM sometimes responds to various drugs – in particular IVIG (intravenous immunoglobulin), which needs more study – for most purposes it isn’t a treatable disease except by intervention with physical therapy, swallowing therapy, adaptive devices and the like."

"There are a number of issues in treating myositis", Dr. Oddis said, "and there have been improvements in accurate diagnosis. To treat myositis properly, researchers must measure muscle strength accurately, correctly identify the type of myositis and observe the impact of the disease on the patient’s daily life. In some cases, doctors make use of MRIs to differentiate between PM and IBM." Dr. Oddis said, "Always the risks of treatment should be discussed and weighed against the potential benefits." He listed the treatment options and spent some time discussing the side effects of prednisone. Like Dr. Fred Miller, Dr. Oddis noted that ‘combination therapy’ is much more common in treatment of all rheumatic diseases, allows greater flexibility, and may dramatically affect the speed of patient response."

Dr. Oddis sees a better future for treatment of myositis patients, as clinicians and researchers meet to set trial measurement standards and more well designed multi-center trials are begun. Also improving the picture is the identification of more reliable markers of disease activity and damage and the increasing availability of new and better treatments.

Dr. Richard Sontheimer is Professor and Head of Dermatology at the University of Iowa College of Medicine, where he works in the Rheumatic Skin Diseases Center of the hospital. He sees many patients with lupus and dermatomyositis (DM) and is especially interested in the skin changes common to DM patients.

"Common skin changes that are needed for a diagnosis of DM include swelling and reddish-purple colouring, both easy to see for the experienced doctor. Less common changes include swelling with no redness, zebra-like stripes, and ‘mechanic’s hands’, or dry, scaly hands – all changes that are very important when they are there. Changes in the skin of lupus patients are like the skin changes in DM patients in the early stages of the diseases. When the diseases continue, however, the skin begins to look different." From head to toe, the following is a list of skin changes important to note in DM patients:

• Scalp itching, burning, and redness with some hair loss possible in small areas – the hair most often grows back when the rash is treated;
• Heliotrope rash, or reddish-purple rash around the eyes that looks like a flower; Shawl sign, or rash at the back, base of neck, and shoulders, covering the same area a shawl would cover;
• Gottron’s sign, or rash at the elbows and knees;
• Gottron’s papules, or bumps with craters in the centre that form over the knuckles;
• Visible blood vessels at the base of the nail below the cuticle (nail-fold);
• Patches of redness around the trunk;
• Holster sign, or redness around the hip area. A combination of these changes makes it easier and possibly quicker to reach a correct diagnosis.

"Those patients who don’t have muscle weakness with the skin changes have clinically-amyopathic (a mee’ o path’ ik) DM, or DM sine (sie’ nay) myositis. Muscle weakness may develop over time. In classical DM, skin and muscle changes both usually occur within six months. No studies have been published on how to determine the risk for patients with only skin symptoms to later develop muscle weakness. It is not uncommon for the muscle weakness to go away and leave the skin symptoms."

"To treat DM, there are three main types of therapies. Local medicines fight the DM by using creams and lotions spread over the skin. These include sunscreen, moisturisers, anti-itch creams, and topical forms of some myositis medicines often taken in pill form, like prednisone and tacrolimus.

Systemic medicines are taken by mouth to attack DM throughout the body. These include antihistamines, anti-malarials (like plaquenil and dapsone), and oral forms of prednisone, methotrexate, azathioprine, and others.

Parenteral therapy is given by shots in the muscles or through intravenous (IV) medicines. These include IV immunoglobulin (IVIG) and TNF-a inhibitors. Doctors usually choose these therapies only after trying local and systemic therapies."

"Dermatologists often work with other doctors to treat DM, especially when the skin and muscles are both involved. The changes to the skin are very important, but they don’t tell what is happening to the muscles. When doctors work as a team, they treat the disease more completely and efficiently."

In the afternoon there were question and answers session with members of the board. Irene attended the PM session with Dr Oddis and I attended the JDM session with Dr Rider. These were informal sessions of questions and answers where individuals could talk directly to the doctors. Paula attended a session by Dr Bradley on IBM and as we have not yet received a preliminary report from him Paula has put together the following report on his session.

Dr Bradley held the IBM question and answer session. He described how in his opinion IBM is a relatively new disease. First described in 1969 and a subsequent full description in 1972, IBM is an inflammatory myositis characterised by muscle inflammation and the presence of ‘inclusion bodies’. IBM is often under diagnosed and misdiagnosed on initial examination, however it can be conclusively diagnosed and likewise ruled out. IBM like PM affects the proximal limbs, shoulders and hip girdle but also more significantly affects the hands to an effect where the sufferer can no longer grip. IBM is not hereditary and is often confused with hereditary diseases like Inclusion Myopathy (inclusion bodies are present but there is no inflammation of the muscle), the gene for this disease is known. Hereditary Inclusion Myopathy has similar features to IBM but because IBM has an element of inflammation Dr Bradley believes that initially IBM should be treated. The observation that IBM does not respond to treatment may be false. As IBM is very slowly progressive the sufferer rarely notices any problems, as a result the muscles become more and more damaged. When an IBM sufferer eventually realises they have a problem it is too late for treatments which dampen inflammation to have an effect and the muscle damage is so great that regeneration of the muscle is not possible. In the USA today there is a drive to promote initial treatment of IBM in case the disease is diagnosed at a stage where treatment may have a beneficial effect and encourage exercise but not to a stage of exertion or muscle pain.

Bob Goldberg announced that The Myositis Association of America was awarding $901,000 in grants for research into the rare autoimmune diseases of polymyositis, dermatomyositis, inclusion body myositis and juvenile dermatomyositis.

Nine projects were selected for funding from 20 applications submitted in response to the association’s first-ever call for research proposals.

These significant research awards were made possible in part by a $1.6 million bequest from Dr. Martha Bishop, a Canadian inclusion-body myositis patient, who died last year. One of the grants, designated as the Dr. Martha Bishop Challenge Grant, totals $200,000 over the course of two years and has been awarded to Valerie Askanas, MD, PhD, who will examine the role of excess cholesterol in the disease process of inclusion-body myositis. Dr. Askanas is a professor of Neurology and Pathology at the University of Southern California Keck School of Medicine.

Steven A. Greenberg, MD, of Brigham and Women’s Hospital, Harvard Medical School. Dr. Greenberg, who is joined in this multi-centre study by Drs. Anthony Amato, Richard Barohn, Alan Beggs, Robert Griggs, Carlayne Jackson, John Kissel, Jerry Mendell and Rabi Tawil, will receive $100,000 over two years for a study of gene expression in myositis.

Carol M. Artlett, BSc, PhD, of Thomas Jefferson University, who was awarded $100,000 over two years for her study of the transfer of maternal cells to males with juvenile dermatomyositis.

Xiao-Feng Yang, MD, PhD, of Baylor University College of Medicine for a study of RNA exosome-specific immune responses and autoimmune myositis. Yang will receive $100,000 over two years for this study which focuses on polymyositis.

Lauren Pachman, MD, of Children’s Memorial Hospital at Northwestern University, who will receive $91,332 over two years for her work studying the activation of lymphocytes in juvenile dermatomyositis patients after ten years.

Sabine Krause, MD, PhD, of Friedrich Bauer Institute at the University of Munich, Germany, for a fellowship to study the MHC processing and presentation machinery for clues for the immunopathogenesis of idiopathic inflammatory myopathies. This award is for $90,000 over the course of two years.

Goran Rakocevic MD, who received $90,000 for a two-year fellowship at the National Institutes of Health to study the molecular immunology of inclusion-body myositis.

Gulnara O. Mamyrova, MD, PhD, for a two-year $90,000 fellowship to complete the database for the Childhood Myositis Heterogenity Study sponsored by the Environmental Autoimmunity Group at NIEHS, NIH.

In addition, the Association will help support the work of Dr. Ann Reed of the Mayo Clinic in Rochester on the influence of genomics on chimerism in juvenile dermatomyositis.

Bob Goldberg announced that from January 2003 the ’America’ from their name will be dropped and they will be know as the ’Myositis Association’. With the Internet giving access to easier communications it was agreed that by dropping the ‘America’ made them more welcoming to international members and organisations. This is a good starting point for ourselves and other myositis organisations in other countries to work and collaborate together.

Bob continued to outline different initiatives for the group’s future.

[picture]

MAA members enjoying a working breakfast on the last day of the conference

Communications director, Theresa Curry writes, "It was wonderful to meet you at the conference. All of us are discussing ways to provide more co-operation and support for you as we move towards being an international association. We’re excited about working with you and taking the first steps in what we hope will be a long and very productive collaboration."

Dr Nicola Pipitone is hoping to conclude this trial shortly. However he still needs a few more participants to ensure the validation of the trial. If you are a sufferer and wish to help in the vital research for the benefit of sufferers please contact Nicolo at, Weston Education Centre, Rm 3.52, King’s College Hospital, Cutcombe Road, London, SE5 9PJ. Telephone 0207 848 5786.

The trial is going well with centres participating in Dudley, Liverpool, Manchester, Birmingham, Newcastle and London. These may well extend to Budapest and Germany participating in the future.

Recruitment to take part in the trial is very important (see our web link). If you are suffering from the adult form of Polymyositis or Dermatomyositis you may meet the criteria to participate. The trial is not using new drugs but may well be the ones you are already taking. These being, Prednisolone plus methotrexate or cyclosporin. If you have just been diagnosed you may well be the ideal person to take part. However, even sufferers of many years can be involved.

The trial will not mean you will have to leave your present consultant. But what it does mean is that you have the added bonus of the best medical teams and support staff treating your illness and monitoring your health. This is well worth keeping in mind.

Barbara Mason is co-ordinating the trial and can be contacted at Academic Department of Rheumatology, Room 3:55 Weston Education Centre, Guy’s, King’s and St Thomas’ School of Medicine, Cutcombe Road, Denmark Hill, London SE5 9PJ or Telephone 0207 848 5791. She is very approachable, very helpful and a good listener! If you wish to have any questions answered, further information or advice about the trial she would love to hear from you.

In the rich tapestry of life there is never a straight line of events. There are always deviating lines, criss-crossing, broadening and tapering. It could be considered very confusing and yet in reality some conclusions can be considered plain and simple - let me explain. Two of the Group’s research grants are named in memory of the late Dr Christine Saunders who tragically died sixteen years ago. As many of you know without Christine the Group would have taken much longer, if at all, to get off the ground. We are fortunate that we have kept in touch with Christine’s parents by means of the newsletters, etc. During this time a close friend of their family, Hilda Gallimore, had also watched Christine’s life and career develop. I presume like us she was devastated when Christine died at a time when her career was about to make ground breaking progress with all the due rewards after many years of hard work and training. Well, the years have moved on and sadly Mrs Hilda Gallimore has died leaving the charity over £29.000 in her will. Our paths have never personally crossed but somehow they have to unite in different ways in the fight to cure these miserable diseases and continue the memory of Christine, for in life I am sure would still be battling our cause.

Member, Carol Wilkins writes, "My daughter Stacy is now in her second year at Plymouth University doing Teacher Training and absolutely loving it! She has met a nice ‘young man’ and is extremely happy. I would never have thought all those years ago she would have got to this stage and been so healthy! There was light at the end of the tunnel."

How about this following news to lift even the most heavy of hearts! Mrs Strother writes, "This letter is to say that fortunately I have no further need of the Myositis Support Group. I first developed Dermatomyositis in 1995 and it turned out to be an indicator of stomach cancer. I had a partial gastrectomy, which has proved extremely successful, and after long treatment with steroids the muscle and skin problems have gone.

I am now 81 and I can still play golf and lead a normal active life. I have nothing but praise for the NHS. I still see a rheumatologist every few months but he seems happy with my condition. I am grateful for the bulletins I had from your group, which have always been interesting and informative, but I think you can now cross me off your mailing list. I also enclose a cheque for £25 as a small contribution to your funds."

Paula, our secretary, is now studying hard for three years to gain a PhD. She is working at the Royal Free hospital in London and is researching liver disease.

Paula will continue to help the Group but her day to day work will be put in the capable hands of Irene and the incapable hands of myself!

On behalf of the Trust, Director, C P Ward writes, "We have pleasure in enclosing a cheque made payable to the Support Group for £1000, representing a charitable donation and we have no doubt you will put these funds to good use."

Member, Pam Edwards writes, "We held a raffle and a coffee evening on the 12th September with all the proceeds going to the Myositis Support Group. I have great pleasure in enclosing a cheque for the amount of £350. Gifts were donated by business people and individuals, then raffled."

"I was helped and supported by the congregation of Eglwys Dei Sant, (Saint David’s Church) Holyhead, who served tea and coffee on the evening. Also a show of old slides (pictures) by the Maritime Museum. I would like to thank my son and grandson for their support and all the people who sold raffle tickets."

Fl. Lt. W Ellis of the squadron rites, "We are delighted to enclose a cheque for £1000 for the Myositis Support Group. The cadets of 863 Thurston Squadron raised this money by partaking in the Thetford Chase Walk back in May of this year. I think you will agree that this was a splendid effort by them. Please accept this with our good wishes for your Support Group."

Myositis Group member, Simon Millyard, made this wonderful effort possible for us by asking the cadets to help us. He writes, "Twenty five cadets took part in the annual Thetford Chase walk and had to walk and navigate fifteen miles round Thetford woods. The Myositis Support Group was selected from other charity nominations as my daughter Emily aged seven who has Dermatomyositis has three brothers in the ATC at Thurston. I consider this to be an excellent effort by the cadets – well done."

Member, Mr Boyce writes, "Please accept the enclosed donation of £10 raised by a, ‘Baked potatoes under the fire’ night at our friendly local, The Red Lion, Outwell. My wife, Michelle, suffers from PM and has gained valuable insight into the disease via your publications and web-site. We hope to make this a regular event over the winter months."

Member, Mary Anne Fishwick writes, "Please find enclosed cheques for £120 as donations to the Group. This money was raised at my 50th birthday party instead of presents for me."

A lady runner unknown to us took part in this race. We received £5 of her £12 entry fee as we were her designated charity. Whoever you are, well done and thank you.

Canadian member, Gordon McGowan, made a beautiful wooden bowl using the stave/segmented method on a lathe and incorporated the woods, maple, mahogany and birch. By donating it to the Group he trusts it will make a good prize for our future fund raising.

Dr Cambridge, our Charity President, is an immunologist working on B cell depletion therapy for the treatment of Rheumatoid Arthritis at University College London.

"Dermatomyositis and polymyositis both result in muscle weakness (there are exceptions). However, the immunological cells which predominate in the muscle and carry out the series of events that leads to muscle damage are different between the two diseases (this can be seen by muscle biopsy). Because of the nature of these immunological events B cell depletion therapy will theoretically be more effective in dermatomyositis than polymyositis. However, B cell depletion therapy is one of many new emerging therapies that could be future treatment for myositis."

B cell depletion therapy has been demonstrated to be effective in Rheumatoid Arthritis patients and Dr Cambridge believes that this therapy has the potential to treat dermatomyositis sufferers. This type of therapy is novel and in its very early stages. No trials have yet been performed in Myositis patients to demonstrate whether it is more effective than conventional therapy. Small scale testing of the therapy is soon to be carried out and Dr Cambridge is optimistic that it may become a therapy for sufferers of dermatomyositis.

"In dermatomyositis, B cells (rather than T cells in polymyositis) are thought to govern the series of inflammatory events. B cells are the white blood cells which when mature develops into ‘plasma cells’ which make antibodies. As well as being a very selective and effective way of defending the body from attack by bacteria and viruses, antibodies can also turn ‘bad’. It is thought that such a ‘bad’ population of antibodies result in the inflammation of joints and other tissues in patients with Rheumatoid Arthritis. Although there is less evidence for such aberrant antibodies underlying the disease process in dermatomyositis, the nature of the disease suggests that they may play a role. B cell depletion therapy removes a large quantity of the B cell population for from 4 to 9 months. In patients with Rheumatoid Arthritis, the patients can remain disease-free for up to 3 years after a single treatment. Thus, this treatment unlike conventional drugs does not have to be given continually."

"One of the drugs in the limelight at the moment is Entercept. Entercept is the name given to one of the anti-TNF drugs. Research in RA and other inflammatory diseases has shown that TNF may be an important mediator in the inflammatory events of the joint. Macrophages are the main source of TNF but activated T cells can also release TNF. TNF is a powerful inflammatory mediator that can perpetuate and amplify the disease process."

"If TNF is released by macrophages, which we know are present in muscle then Entercept and other anti-TNF drugs could benefit dermatomyositis and polymyositis sufferers. However, it is not yet known whether macrophages in the muscle of myositis sufferers release large quantities of TNF, but raised levels of TNF have been found in the blood of patients. The really good news is that a clinical trial of this therapy is underway in the USA."

"Although the anti-TNF drugs are not being widely prescribed in the UK, some patients here have tried the therapy with mixed responses. Anti-TNF drugs are not cures but very effectively block a potentially key player. Until we know what actually underlies the disease process in myositis (both PM and DM), we can only try to target our treatment at the most likely immunological candidates. Future research will need to be adopted to determine whether TNF is important in myositis and to ensure that treatment is given wisely, determined by what type of sufferers are likely to benefit."

"However, until there is a proper controlled trial of B cell depletion or indeed any of the anti-TNF drugs in myositis, we have to be cautious in our optimism. But at least there is some progress and a general feeling that more good options for treatment may be available in the near to very near future."

Professor David Isenberg is the academic director of Rheumatology at University College London. He has had many years of myositis experience and is currently co-ordinating international plans to reassess the ways which myositis is assessed and managed. The group of world-wide specialists is known as the International Myositis Outcome Assessment Collaborative Study Group (IMOACSG).

Professor Isenberg’s presentation was an explanation of the IMOACSG and why it now is essential to get together on an international level to ensure in particular that rheumatologists and neurologists are using the same methods of assessment for their patients so trials of new therapies can be meaningfully compared.

"Dermatomyositis, polymyositis, inclusion body myositis and juvenile dermatomyositis are grouped together as myositis and constitute a group of diseases known as inflammatory myopathies. As more is discovered about these diseases they are more easily distinguished clinically, pathologically and in their response to treatment."

"One question often asked by specialists themselves is, ‘Why does one patient with polymyositis responded very well to treatment and another also suffering from polymyositis not at all?’ Initially, unresponsive polymyositis sufferers found their diagnosis being changed to other conditions such as inclusion body myositis or a muscular dystrophy. Indeed this is often the correct answer in many cases and a re-diagnosis requires reassessment of the muscle biopsy by an expert. However, there is growing evidence to suggest that within a classification there are subtypes."

"Another important question a doctor will ask themselves when examining a patient is, ‘What is disease activity and what is disease damage?’ This is a very important question to answer and the most difficult to get right. By ‘activity’ it is implied that there is an active process of inflammation going on whereas ‘damage’ implies permanent change which will not be amenable to immunosuppressive drugs. When a disease is active is must have medical intervention. However, this may be accompanied by side effects of the drugs. It is a balance, enough drug(s) must be given to control activity but not render the patient ill with too many side effects."

"Disease damage can be limited by early effective control of disease activity. One of the areas in development by the IMOACSG are guidelines to clarify ‘what is activity’ and ‘what is damage’. In principle the ideas of disease activity and disease damage are simple however for these guidelines to work they must when applied by the specialist to assess the patient give the same result. There must be a uniform consensus within defined limits if these guidelines are to be of any beneficial use."

"With our knowledge of the diseases expanding, new treatments emerging, and an international drive for reassessment of our current tools, now is the time to take steps to introduce internationally accepted guidelines. This will attempt to insure correct diagnosis, protocols for treatment (depending on the diagnosis and possibly disease subtype) and uniform assessment and management of the disease."

Dr Choy is a Rheumatologist at Kings College Hospital London. He has secured funding of a £360,000 research grant from the ARC to perform a clinical trial to investigate the importance of combination therapy in dermatomyositis and polymyositis.

Dr Choy’s presentation included an explanation of why clinical trials are important and what the SELAM trial aimed to investigate. The acronym of SELAM stands for Second Line Agents in Myositis.

Dr Pipitone is a Rheumatologist at King’s College Hospital London and currently holds the Christine Saunders Memorial Lectureship sponsored by the Myositis Support Group. Dr Pipitone is co-ordinating the final arm of the Creatine Supplementation Trial and hopes to perform some lab based research into myositis.

Dr Pipitone split his presentation into three mini parts. He gave an update on the Creatine Supplementation Trial and explained two areas of research which he plans to pursue. These research areas included further investigation into the HLA expression in myositis and research into microchimerism as a potential trigger of myositis.

The committee has agreed to fund one year’s funding of part time research registrar Dr Shabina Sultan.

Professor David Isenberg writes, "As you know I have been working very hard to establish, with international support, disease activity and damage indices which could be used as part of a drug responder index. This seems to me a major advance in the field and is likely to be very important given that a number of new drugs including the anti-TNF alpha drugs and anti-CD20 are now being contemplated for the treatment of patients with myositis. There is a great need to validate these tools and Dr Sultan who has a background in research in myositis is keen to undertake the research."

The funding is from the 1st October 2003.

This is a reminder to those who may still require Christmas Cards, please can we have your order as soon as possible. We have sold out of Christmas Eve and Snowman at Night. If you have mislaid your order form you can download one from the web-site, www.myositis.org.uk or telephone the office and we will send you one.

The planning is now underway for the conference next year. Having attended the Myositis Association of America’s conference this year it has given us some ideas for ours. If you have any suggestions on what you would like included we would be pleased to hear from you.

I will be visiting the hotel very shortly to finalise the arrangements. I am hopeful of arranging a reduced price for overnight accommodation for those wishing to stay. Full details and booking forms will be sent out with the first newsletter in 2003.

The venue is the Paragon Hotel, Alcester Street, Birmingham and the date is Saturday 12th July 2003.

It gave me great pleasure to present Treasurer Tony with a cheque for £1000. Thanks to all who have donated books.

[pictures]

Piles of paper for some, Piles of hope for others. Paula and Les showing the stack of newsletter 52 before they were stamped and posted.

I am delighted to inform you that my son, David, who, due to injury, had to withdraw from the marathon last year has had confirmation of his place for 2003. He will be running on behalf of the support group.

You may have had an e-mail from or seen on the bulletin board an advert for Japanese Acupuncture. We did not give our permission for this person to solicit our members. We have removed the article from the bulletin board. If you are considering this type of treatment please contact your doctor first.

Rob Fenton our ‘Web Master’ has recently set the ‘Buddies’ list on our web site. This is a map of the UK and Ireland. You can click on your area to see if there is a member who is happy to be contacted by fellow members. If you have an e-mail address and would like it added to the ‘Buddies’ page please contact Rob at.

rob.fenton@ntlworld.co.uk

We are very grateful to Rob for all the time and effort he puts in to running the web-site. I know the Bulletin Board, and now the ‘Buddies’ page is proving to be a great success.

We are aware that not everyone has access to a computer. We are therefore proposing to set up a list of members who are willing to be contacted by other members by post or telephone. If you would like to be included on this list please send a letter giving permission for your name, address and/or telephone number to be included on the list. It would also be helpful if you could include the type of myositis you have, your age and perhaps a brief history. If you already have your e-mail address on the ‘buddies’ page perhaps you would also like to be on this list. We get many enquiries for contacts and it would be of great benefit to be able to put more members in touch with each other.

Please send your letters to Irene at the office.

Thank you for the many kind and helpful letters and individual donations they are much appreciated.

Les Oakley

Chairman