TMA’s Past and Future

TMA is fortunate to have an outstanding Medical Advisory Board consisting of 22 myositis experts who are well known and well-respected. We have members from the most prominent research institutions – Harvard, Mayo Clinic, Johns Hopkins, NIH, etc. We have now had over 60 researchers serve on our MAB. They review all publications TMA produces such as Myositis 101 which is now being updated and will be distributed later this year, Physicians Guide to Myositis which was updated last year, etc. All medical information that TMA distributes is reviewed by our Medical Advisory Board. As I mentioned previously, one of the MAB’s most important functions is to review the research applications we receive and make funding recommendations to our Board of Directors. It is important to know that our MAB members do not receive any compensation from TMA. They are volunteers wanting to help find treatments and a cure for the myositis diseases.

TMA funds the best research regardless of where it is taking place. It used to be that America was where the great majority of myositis medical research took place and the U.S. was clearly the leader. However, there is now very good research also being conducted in Europe, Asia and South America. TMA’s Medical Advisory Board reflects this in that nearly a third of the TMA’s Medical Advisors are from outside the U.S. And, the percentage of research funded by TMA being conducted outside the U.S. is growing each year as the quality of the research around the world improves.

Moderate exercise is beneficial for myositis contrary to what was believed in the past. Certainly if you have challenges with activities of daily living and work related issues, an occupational therapist can be very helpful.

TMA began funding research in 2002. It was only because of a large bequest of $1.6 million that TMA was able to begin funding research. Over the next 15 years other bequests and fundraising conducted by TMA allowed us to fund over 50 research projects so far. As for the type of research we are looking to fund, we do not have a strategy. What I mean by that is that there is still so little known about these diseases that we want to be open to any type of research that seems promising. Many drug discoveries have been by chance and often surprise even the researchers most familiar with a particular disease. We are often asked if stem cell treatment is effective for myositis. To date, that is not the case but perhaps at some point in the future it may be.

Medical research is VERY expensive. Yes, TMA has thus far been able to fund grants and fellowships totaling nearly $7 million. Most of these funds have come through planned giving where an individual leaves money to TMA in their will or designates TMA as a beneficiary of a life insurance policy. Of course, all donations help in providing TMA with the funds needed to operate and bring together the best minds to focus on research that can lead to better treatments and ultimately will lead to a cure. I believe there are individuals among the myositis community with the means to really help accelerate the research that is needed. We need those persons who can make a big difference to decide it is time that they step up and make a large gift to TMA now or through their estate when they pass. It is important and I often say to those few who know about myositis and have the means, “if not you, who??” Few people know about this disease, and TMA is relying on those who do to make a difference.

 Big metropolitan areas like Chicago, New York, Los Angeles, Boston, etc. are too expensive for TMA’s Annual Conference. We need to hold the Conference in hotels that are more affordable for our members and their care partners. We move the Annual Conference around the country to hopefully hold it in an area accessible to many of those who have the disease. There are a lot of considerations that go into which cities and hotels we use such as handicap accessibility, location, affordability, etc. It is not easy to find hotels that have reasonable room rates, enough meeting space for our Conference, and accommodations suitable for those with physical limitations.

Sorry for the difficulties you are having and we appreciate your willingness to lead the group in the Carolinas. The new TMA website, which will be going live in a few weeks, will fix some of the problems with the current site and should be an exciting addition to our services for TMA members and the public. An announcement will go out when the new site is up. Probably early June.

Board members are elected to a one year term and can be reelected to two 2-year terms after that. They can only serve a maximum of 5 consecutive years. We welcome anyone interested in serving on the Board to send an email to TMA@myositis.org. We look to have patients, care partners, professionals and others with an interest in myositis to serve on the Board. There are currently 10 members on our Board of Directors.

What we are learning more and more is that myositis is not one disease. It is many different diseases, and each person’s experience of the disease is unique. Most researchers believe that the autoimmune response (that is, the targeting of the body’s own tissues as a foreign substance that needs to be destroyed) is responsible for the muscle fiber damage and muscle weakness in myositis diseases. There is probably some event that triggers the development of myositis in people with a genetic predisposition. However, it is not possible to know what the one trigger might be. It could be an infection, environmental exposure, virus or a number of any other things.

Studies have found associations between various forms of myositis and specific autoantibodies. Anti-Jo-1 antibody, for example, is associated with myositis with antisynthetase syndrome. This is important because these autoantibodies are created as part of the body’s autoimmune mechanism. They do not cause the disease, but they do give doctors some idea about how the disease might progress and what treatments might work better than others.

We have presentation of all the diseases– IBM, DM and PM — and anyone interested can apply. We look to maintain a balance of the diseases and skills — currently we need a CPA to add to our financial expertise on the Board

Yes, TMA has funded research into the benefits of exercise for myositis. Much of the best research has taken place in Sweden and TMA has those involved in that research presenting at our Annual Patient Conference in Louisville this September.

TMA funds grants and fellowships. Grants are designed to promote innovative ideas and promising avenues for research that might lead to a clinical trial. Fellowships are to interest young researchers in myositis and encourage them to make myositis the focus of their research as they develop research interests. TMA is very careful to only fund projects that we believe are promising and worth the investment of our limited funds. Our Medical Advisory Board and other reviewers objectively and impartially consider each research application based on the science and its relevance to myositis. Finally, TMA’s Board of Directors will decide which projects to fund based on the recommendations from our Medical Advisory Board.

The Arimoclomol trial is now getting underway. There are 10 centers in the U.S. that will be participating in the trial. Right now the University of Kansas is the only one in the U.S. recruiting patients. You can contact LHERBELIN@kumc.edu to find out more about the trial and where other centers that will be participating are located. Criteria regarding eligibility to be in the trial can be found at clinicaltrials.gov and searching for arimoclomol.

TMA hosts a meeting of our Medical Advisory Board members annually for them to discuss myositis research as a group. There are several other meetings each year where the various specialties meet separately. TMA’s meeting is the only annual meeting where all the specialties that treat myositis patients are together to talk about myositis research.

To help TMA, you can educate your physician(s) about the disease since in Idaho it is likely you are one of their first myositis patients and they will be better prepared if they see other myositis patients in the future. TMA offers its Physicians Guide to Myositis to educate physicians. To request copies (they are free), email TMA@myositis.org or call us at 1-800-821-7356.

Donors can restrict their funds to research and to research into specific types of myositis such as IBM or dermatomyositis, etc. They also can indicate that they want their funds to be used only for Annual Conference scholarships to enable people to attend TMA’s Annual Patient Conference who would not have been able to attend otherwise. It is, however, important to remember that unrestricted donations can be used to support all of the services of TMA including research, but if you restrict your donation to research only then those funds cannot be used to support the Annual Conference, physician education efforts, myositis awareness activities, etc.

While TMA funds more myositis research than any other patient-based nonprofit organization, there is much more expensive research funded by the pharmaceutical and biotech industries. One of the most exciting developments in the past few years is the growing interest in myositis of big drug companies like Pfizer, Bristol-Myers Squibb, Novartis among others. TMA assists these companies in having patient input in the design of their clinical trials and recruiting patients for their trials. This is an activity that TMA has become very involved in the past few years and which we feel is important. Patient input will result in more efficient trials and measures of improvement that are most meaningful to the patients who have the disease and know best the issues they have to deal with due to their disease.

I know it is very frustrating for those who have IBM to not have any medications that are effective. However, you should be encouraged that there are 4 trials that have been or are soon to be underway looking at potential treatments for IBM. In the meantime, those with IBM can help themselves best by taking precautions to avoid falls and be careful when swallowing. Doing any exercise that stretches your muscles can help and eating well to maintain your nutrition and health also can help to slow down the progression of the disease. Remain positive and do not give up hope.

IVIG and Rituxan are treatments that are not approved for myositis meaning that the FDA has not seen research that convinces them o the effectiveness of these drugs for myositis. TMA is working to change that by our support and assistance of studies now underway to collect data on the effectiveness and safety of these treatments for myositis. If we can prove their effectiveness for treating myositis, then Medicare and other insurers will include these treatments on their formularies, meaning they will be considered an approved treatment and no longer an experimental myositis treatment. This will lead to improved reimbursement and coverage of these drugs for the treatment of myositis.

Same thing I said about drugs for myositis applies to physical therapy for myositis. If we can demonstrate the effectiveness of physical therapy in maintaining the fitness and strength of myositis patients then Medicare and other insurers are likely to cover these treatments. Physical therapy for myositis is an area that TMA is beginning to focus on more.

There are many things I am proud of looking back over my 15 years here, but the one that stands out the most is the incredible group of people I have had the pleasure working with. Not only are the myositis patients a remarkably courageous and resilient group trying to cope with a terrible disease, but the physicians and researchers focused on myositis are as committed and determined as you are likely to find anywhere. Of course, the staff and Board of Directors have also been of great assistance in helping me to make TMA what it is today.

Yes, having the time and strength to lead a support group is not easy for those who work and especially for those physically depleted by the disease. While we have some great support group leaders, we need more to help as co-leaders. And, for patients who find it too difficult to travel to a support group meeting or do not have any nearby, TMA has a program we call MyoConnect which means we will find someone with your form of myositis willing to talk with you by phone or through email to share information and help each other cope with this disease. For those interested, in this program email TMA@myositis.org  or call us at 800-821-7356.

Concern about privacy and protection of your personal health information is a major issue and will likely be an even bigger issue going forward as technology allows more capturing of data. Greater protections need to be in place. I can tell you that TMA has never shared any personal health information with any outside parties (commercial enterprises, drug companies, insurers, or other organizations). We only share contact information between TMA members who are in a TMA support group and want to be able to contact others. Any surveys conducted by TMA of our members has the data de-identified so that researchers using the data cannot in any way associate responses with individuals who provided data. The only studies and surveys that TMA conducts which collect personal data are overseen by an IRB (Institutional Review Board) that sets very strict rules for how data can be collected and used.

 Stems cells to treat myositis has been studied and the evidence so far is not convincing that there is any significant benefit.

I am proud of a lot of things but most proud of promoting compassion and professionalism among the TMA staff and leading by example. Those who suffer with this disease need someone willing to listen and willing to do the heavy lifting that is needed to allow the organization to best serve its members. There has never been any favoritism shown toward, staff, physicians, or patients during my tenure. All were treated with equal respect and received what they deserved. I have no regrets.

I cannot tell whoever is to replace me what their top priorities ought to be. Hopefully, they will bring to the organization a fresh set of eyes and a perspective that might identify opportunities for growth going forward. I will say that maintaining the integrity of the organization and remaining in touch with the patients and care partners is essential to having the long-term support needed to maintain the organization’s strength and loyalty among the patient and physician community. Do not cut corners, be true to your beliefs, respect the opinion of others, and remain focused on the big picture are some qualities that the next leader should have.

The future is bright for TMA. There is more myositis research underway now than ever before. There is more interest in myositis research among scientists, researchers and the pharmaceutical industry than ever. TMA is recognized as the leader of the myositis community and has a stellar reputation. However, one cannot be complacent. The organization needs to continue to push for more financial support to provide more services and to fund more research. While there are other organizations helping to serve the myositis community and each in its own way contributes, TMA remains the leading organization because it serves the entire myositis patient and research community. It needs to remain so.