Join our Podcast on NMO Studies and Clinical Trials: What Should I Know?

Teaser_May_2015What are the various studies and clinical trials in NMO? How do these different drugs work? What is their mechanism of action? How do I know which one is right for me? How does a clinical trial benefit me? What is a placebo-controlled trial? What if I have an exacerbation during the study? What happens when the study is over?

These and more questions will be addressed during our Podcast tomorrow With Dr. Benjamin Greenberg from The University of Texas Southwestern Medical Center and Dr. Michael Levy from The Johns Hopkins University School of Medicine

In addition to our experts, four special guests from the pharmaceutical industry will be joining us to talk about novel therapies for individuals diagnosed with NMO and NMO Spectrum Disorder that are currently open for enrollment.

Please share your questions and concerns with the experts here.

Make sure to register for the podcast to learn more!

ABOUT OUR SPEAKERS

Benjamin M. Greenberg, MD, MHS

Associate Professor at UT Southwestern Medical Center, Director of the Transverse Myelitis and Neuromyelitis Optica Program, Dallas, TX

Dr. Greenberg is recognized internationally as an expert in rare autoimmune disorders of the central nervous system. He splits his clinical time between seeing both adult and pediatric patients. He routinely consults on the inpatient units of Zale-Lipshy University Hospital, Parkland Memorial Hospital, and Children’s Health. His research interests are in both the diagnosis and treatment of transverse myelitis, neuromyelitis optica, encephalitis, multiples sclerosis, and infections of the nervous system. He is actively involved in developing better ways to diagnose and prognosticate for patients with these disorders. He has led an effort to improve biorepository development and has created uniform protocols for sample handling and analysis. As part of this initiative his research has identified novel biomarkers that may be able to distinguish between patients with various neurologic disorders. He also coordinates trials that study new treatments to prevent neurologic damage and restore function to those who have already been affected. He currently serves as the Director of the Neurosciences Clinical Research Center at UT Southwestern and is a Cain-Denius Foundation Scholar. Dr. Greenberg is a member of The Board of Directors of The Transverse Myelitis Association and also Chairs The TMA Medical and Scientific Council.

Michael Levy, MD, PhD

Assistant Professor of Neurology at the Johns Hopkins Hospital and Medical Director of General Neurology at the Johns Hopkins Hospital in Baltimore, MD

Dr. Levy is Assistant Professor of Neurology at The Johns Hopkins University and Medical Director of General Neurology at The Johns Hopkins Hospital in Baltimore, MD. Dr. Levy also directs the Neuromyelitis Optica Clinic and follows over 200 patients with NMO and related disorders. In addition to clinical work at The Johns Hopkins Hospital, Dr. Levy runs the Neuromyelitis Optica and stem cell research labs. The focus of his research is animal modeling of neuro-inflammatory disease and development of strategies to regenerate the nervous system with stem cell technology. Dr. Levy completed the MD/PhD program at Baylor College of Medicine in 2004, Neurology residency training at Johns Hopkins Hospital in 2008 followed by neuroimmunology fellowship training at Johns Hopkins focusing on NMO research. In Oct 2009, he was appointed to the faculty. Dr. Levy is a member of The TMA Medical and Scientific Council.

ABOUT OUR SPECIAL GUESTS

Dr. Athos Gianella-Borradori 

Chief Medical Officer | Chugai Pharmaceutical Co., LTD.

Dr. Gianella-Borradori is the Chief Medical Officer and head of Translational Research at Chugai Pharma USA, LLC and Chugai Pharmaceuticals, LLC in Tokyo, Japan.  He has over 20 years of domestic and global clinical research and development experience in the pharmaceutical industry.  Dr. Gianella-Borradori earned his doctorate in medicine from the University of Bern (Switzerland) and is a board certified hematologist and oncologist. During his career, Dr. Gianella-Borradori worked in teams developing medicines in several therapeutic areas including neurology, he pioneered the use of biomarkers in early clinical development, and kept a strong focus on the needs of patients.

Ms. Angela Melia

Senior Clinical Operations Leader, Project Manager | Chugai Pharmaceutical Co., LTD.

Ms. Melia has over 20 years of experience in domestic and global clinical research and operations with Hoffmann-La Roche in Nutley, NJ and most recently, with Chugai Pharma USA, LLC in Berkeley Heights, NJ.  Ms. Melia’s clinical programs were Phases I-IV from initiation through close-out in various therapeutic areas in virology, infectious disease, metabolism, oncology, neurology, and inflammation. She led and managed project teams, regional monitors, and in house CRAs. Ms. Melia completed her Bachelor of Science in Biology at Seton Hall University, South Orange, NJ that earned her Magna cum Laude, and completed a mini-MBA Certificate Program in BioPharm Innovation at Rutgers University in New Brunswick, NJ.  Ms. Melia is a PMP Certified by Project Management Institute, Inc. (PMI).

Eliezer Katz, MD FACS

Senior Director | MedImmune LLC

Dr. Katz is currently senior director at RIA (respiratory, inflammation, and autoimmunity) clinical development, MedImmune LLC. At RIA Dr. Katz oversees clinical development in neuro-inflammation, inflammatory bowel disease, and myositis. Dr. Katz earned his MD degree from the Hebrew University-Hadassah Medical School in Jerusalem Israel. Dr. Katz joined the pharmaceutical industry in 2004 following a 20 years of academic career as a liver transplant surgeon. His last position in academia was associate professor of surgery and director of liver transplant at University of Massachusetts Medical Center. In Industry, Dr. Katz was vice president at CTI clinical trial and consulting services, and senior director at Medicine Development Group, Pfizer Inc. Dr. Katz is an author in more than 50 peer review publications.

Robert Glanzman, MD

Executive Director, Neuroscience Lead, Global Medical Science | Alexion Pharmaceuticals, Inc.

Dr. Robert Glanzman hails from the Midwest of the USA. After graduating medical school at Wake Forrest University, he completed a residency in neurology at the University of Michigan and fellowship in diagnostic nuclear medicine at Duke University. He spent 7 years as Adjunct Clinical Professor at Michigan State University, practicing general neurology and nuclear cardiology/neurology in the Upper Peninsula of Michigan, an area of highly endemic multiple sclerosis. During this time he taught residents and was Primary Investigator for numerous clinical trials in neurology. He came to Pharma in 1999, holding titles of increasing responsibility at both large Pharma companies (Pfizer, Novartis,Hoffmann La Roche) and small biotech companies (Nektar Therapeutics). From 2009 to 2012 he was the Global Development Team Leader for Ocrelizumab in MS at Roche.

You Can and Should Dream Big!

My favorite question to ask our teenagers in the TM and NMO clinic is “What do you want to be when you grow up?” This provokes a wide array of answers, from teacher to CSI agent to rocket scientist. Helping our teenagers identify how to reach their goals despite their disability is one of my passions.

Occasionally a young patient does not have an answer, and after digging deeper I learn that their hope of being independent from their caregivers has been lost. There is a feeling of anxiety that they will not be able to move away from their parents to go to college or start working. Sometimes the perspective of the teenager affected by TM, AFM, NMO, ADEM, or ON, or even the perspective of the family, is the greatest limiting factor preventing them from reaching their full potential. Their expectations of what life would be have not been met, and they are disillusioned about their future. This mental hurdle is by far the most challenging. When young people buy into the lie that they cannot go to school or work because they have issues with their sight or ambulation, then they cheat themselves and those around them out of reaching their full potential.

After diagnosis, the trajectory of the teenager’s life is altered. How much it is altered depends on factors like the severity of the illness, its impact on daily activities, the degree of recovery, and psychosocial support. The child’s dependence on their caregivers and others increases and their independence decreases. Hopefully this is for a short period of time, and as treatment interventions and rehabilitation are put into place the child resumes their previous trajectory. When that doesn’t happen we assess for various barriers affecting independence and look for solutions. It is possible to overcome the mental barriers, but it requires getting help from others and a willingness to take a chance. There will be many trials, but it is important to remember that many times success is defined by our ability to overcome challenges and push through; it is the journey, not the destination that makes us.

We want to inspire our young patients to DREAM BIG! I tell them there are politicians who are in wheel chairs running entire states and there are chefs that are blind. I explain that their medical condition is not the driving force in their life, just a challenge. We hope to help them chase after their dreams and break down the obstacles into manageable steps to ultimately be the best they can be.

This is the first blog in the series on Transition of Care. Audrey Ayres, RN, BSN, MSCN is a clinical nurse at University of Texas Southwestern Department of Neuro-immunology. She was recently award the 2014 Excellence in Nursing Award by the Dallas magazine. Audrey provides care for adult and pediatric patients with Multiple Sclerosis, NMO, TM, AFM, ADEM and Limbic Encephalitis.  She is also the primary nurse for the Pediatric Demyelinating Disease Clinic for Children’s Medical Center in Dallas, TX.

Data are Shadows of Stories

Today, I (Roni Zeiger) had the privilege of speaking at RWJF’s #Data4Health conference in San Francisco. Here I’ll share a written version of my brief talk and my two slides.

I love data. In some contexts, that might sound like a confession. Obviously not here. I grew up loving math, then science. I decided the most interesting secrets were probably hidden in biology, in particular molecular biology.

I worked in labs in college, learned how to splice DNA, to make antibodies that were part mouse and part human. It was awesome. It was also a little lonely. The lab was a place where I was looking down more than looking up.

I then discovered medicine, and found a place where I could do science and stories at the same time. Data and humanity… but oh man, the data!

My Palm Pilot was always with me, and I learned how to write computer programs. You see, the medical center was producing so much data, we just needed better tools to analyze it. I also ended up getting a degree in medical informatics and cemented my identity.

I still saw patients, something about that kept me grounded, especially when I went to work at Google.

brid-connect-a-dot

 

Google… holy cow, best data ever. I analyzed all the health questions people across the globe were putting into the Google search box. Symptoms, diagnoses. Drugs, supplements. Results of a scan. Good news. Bad news.

And I started seeing that those searches represent people. Data are tiny shadows, projections of rich complex stories.

Sometimes you can connect the dots, see how a series of searches reveals a new life, or tragedy, hope or fear. And there is SO much more in between those data points.

Even a child, perhaps especially a child, can show us that. (In this case, one of my children.)

bird-colored

To be clear: we need more data, and we need better access to data. And we need the stories that bring them to life, that give them color and context.

The data may hold the answers, but stories tell us which questions to ask and stories tell us why the answers matter.

~  Roni Zeiger

This blog was originally published in the Smart Patient Blog. Click here to view the original article.

 

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What Drug Companies and the FDA Need from Us

If you are interested in learning more about clinical trials, how new drugs come to market and what this means for NMO, TM, ADEM, Dr. Benjamin Greenberg’s presentation at the 2013 Rare Neuro-immunologic Disorders Symposium shares what we need to know and what the future looks like!

[youtube]http://youtu.be/lemEpjSGcj0[/youtube]

To view other videos from the 2013 Rare Neuro-immunologic Disorders Symposium hosted by the TMA, please click here.

AXS Map

For those who use three or four wheels for personal mobility, you might be interested.  I have recently stumbled upon a website that lists places of business utilizing the Google GPS Format for mapping cities and towns in the US and other countries, and showing places of interest such as restaurants, hotels, parks, etc. as a general locater. The website allows its members to locate different areas of interest, click on a specific place shown and see if it has been rated according to accessibility or not.  Currently, since the site is so new, not many have been rated.

How it works; you can join for free and give your own personal rating of places you have visited according to certain accessible categories; such as entrance, bathrooms, crowded or spacious, noisy, accessible parking, etc.  The website is “Source Dependent”, meaning places are not automatically rated, but if someone has visited and rated a place, it will show that person’s ratings.  That someone is a member who took the time to put their evaluations into the program and rated places they have visited.  If it is already rated and you visit the place, you can also give it your rating.  Things may have changed, or even improved, since the previous rating had been posted.

I invite you to first just visit the website http://www.axsmap.com and look around, watch the videos to learn how it works before joining.  If you are interested, join and help populate the data for others to see.  It will be a great help for out-of-town visitors or even “locals” who would like to go a place but would like to know if it is wheelchair accessible or not.  This is a non-profit organization and the site was designed by a fellow wheel user.

I personally think it is a great idea and have already started putting in my ratings for the area in which I live, and other places outside my area that I have visited.  You can also do it from your Android smart phone or tablet while you are there.  There is a comment section where you could rate an entrance a four star out of five but it has a three, four or two inch step entrance, or several steps, but the facility has a ramp they will put down for a wheelchair to gain access.  A short tutorial video is also on the website showing how actual people rate places.

Remember, the website will only work if people like us populated it with data.  It works anywhere there is a built in Google mapping capability, I think.  I’m still trying it out.  Those in the UK might try it also.  If you like it, pass it onto your contacts and friends.  (p.s. Able Bodied people can also join in on it).  If you are a member of other organizations besides the TMA, pass this information on to those who are members there with you.

I’ve been in a wheelchair as a paraplegic with one arm using a power chair for mobility since 1994 and could really have made use of this information when traveling outside my area.  I’m looking forward to lots of data being put in so others can use it in the future.

Happy wheeling; I’m off to see what I can rate with my I-Phone.

~ Bob Cook from Spring, Texas

Videos of the 2013 Rare Neuro-Immunologic Disorders Symposium Available!

KEYNOTEVIDEOSSYMPOSIUM

We’ve got good news for all those who weren’t able to attend the 2013 Rare Neuroimmunologic Disorders Symposium! You can now view the first day of Presentations on our website! Please go to https://myelitis.org/portfolio-view/2013-rare-neuroimmunologic-disorders-symposium and browse through them!

My Story Prompted Me to Start a Support Group

I volunteered to be a support group leader for The Transverse Myelitis Association because of the need for awareness and support for our members and caregivers living with this condition.  I agreed to help as a support group leader because of my experiences and my story, which I am sharing with you.

On March 14, 2012, I visited my primary care physician with numbness in my feet and legs.  My primary care physician ordered an X-Ray and did not think it was anything serious to worry about.  However, on March 15,, I was completely numb from my waist down.  I could not feel anything below my waist.  I was scheduled for surgery on March 19 for other reasons.  I had been having many medical problems including internal bleeding, blood in my urine, hair loss, weight loss, and GYN problems.  All of the doctors treated my problems separately.

On March 15, my OB-GYN physician instructed me to go directly to the Emergency Room (ER) as he felt that it was something very serious and needed immediate attention.  Upon my arrival at the ER, the on-call neurosurgeon was called in to review my case and they thought that I might need to have emergency surgery.

I was very blessed that the neurosurgeon assigned to my case was very experienced with Transverse Myelitis.  He diagnosed me with Transverse Myelitis (TM) that night and admitted me to the hospital for further testing.  I did not have to have any type of surgery.

The doctors could not determine what else was causing my problems.  Therefore, one doctor said Lupus and one doctor said Sjogren’s syndrome.  Finally, it was determined that I had TM and Lupus.  I was treated with intravenous steroids, cellcept and hydroxychloroquine and a long list of other drugs.

I was also informed that my other previous medical problems were due to the TM and Lupus.  Therefore, my surgery that was scheduled for March 19 was cancelled.  On May 22, my neurologist informed me that my final diagnosis would be TM, NMO, Lupus and Sjogren’s.

I was so confused and did not understand all of this information.  My doctor tried to explain the conditions and took time to speak with me during my appointments, but it was not enough time to comprehend this new medical condition.  I tried to find local support groups, a medical center that provided support as well as other individuals who understood and had experienced such a rare disease and who could answer my questions.  There was no one locally.  I was able to connect with TMA and find many of the answers through the website and other electronic information.  But there was not a physical local support group.  I had to answer my own questions through my research.  I spent many hours each day researching my medical condition and trying to find more answers.

My medical situation changed my life style completely and my family provided all of the rehabilitation for me.  I had to learn how to walk again as the doctors told me that I may never walk again or may only gain some mobility with restrictions.  I can now walk again, but I have my limitations.

The medication is hard on my system and I had to adjust to the severe pain and also had to limit my activities because of the rest I needed to get through the day.  Many people look at me as if nothing is wrong with me because I look so healthy; but what people do not understand is that I fight every morning to get started and fight every night to get to sleep because of the burning, banding and pain.

Therefore, I want to be a support for others so that they will have a local resource to call, meet and ask questions and also get help with understanding it all.  This is a very complex medical condition and it is very important that we provide the support to help others get the support and awareness that they need.

It is also important to me that people do not have to experience the long time pain and medical problems before they are diagnosed.  It is important to know that we can fight this medical condition together; but alone it can be hard and bring on other life-changing issues.

If you are in the Richmond, VA area, please contact me and I look forward to us getting together and forming a support group for these rare neuro-immune diseases.

Together we all can form the vision and provide long-term support and awareness.

~ MaLinda Washington, Richmond, VA

 

Life on hold: the experience of living with Neuromyelitis Optica

A research paper was recently published from the Northern UK NMO Service by Drs. Kerry Mutch, Abigail Methley, Phil Moore and Anu Jacob to develop an improved understanding of the experiences of people living with NMO.  15 patients from the Northern UK NMO Service were interviewed and five major themes were identified – diagnosis and treatment, symptoms, adjustment, identity and support. The authors reported that NMO is a difficult condition to live with due to the unpredictability of relapses and accrued disability of visual or spinal symptoms occurring with each relapse. Poor vision, reduced mobility, bladder dysfunction and pain affected participants’ independence and experience of living with NMO. Participants reported that during relapse and recovery they would ‘‘put their life on hold’’. They identified the importance of periods of stability to enable them to adjust to their condition and therefore aim for ‘‘normality’’ of life that they believed was comparable to their peers.

The study also outlined several implications for rehabilitation.

To read and learn more, please click here

Notes from the Southern California (SoCal) TM Support Group Meeting

The SoCal TM support group got together on August 24, 2013 at the Dayle McIntosh Center in Garden Grove, CA.  The Dayle McIntosh Center was gracious to let us use a large conference room for our meetings.

Fifteen people attended the meeting, 13 of whom were diagnosed with TM. One person drove from Bakersfield, at least a 150-mile drive, to join us.  Another drove 70 miles from Encinitas. It was a great meeting and we discussed some topics that were really interesting and opened at least one person’s mind to the benefits of a support group. The mix of people that attended ranged from a former Olympic bobsledder, a librarian, a college professor, a nurse and of course the rest of us that no longer work due to TM.  TM hasn’t stopped us from making the most out of everything in our lives.

Our meeting might seem long to those who don’t attend but at the end of four hours (yes, four!) people are still willing to stay.  We started the meeting with a potluck of great food, conversation and catching up.  Then we went around the group and shared our individual stories.  Some were new people sharing their stories and others provided an update.  Each person has a different story and each was diagnosed differently.  Each person is affected differently, some are in wheelchairs (me for one), some use walkers or canes and others are able to walk unaided.  Below are some of the issues we discussed at the support group meeting.

Pain We were all different in our pain and pain levels, but the discussion was lively.  Almost every one of us has some sort of neuropathic pain.  We talked about how this is such a hard concept for other people to understand.  How can we look normal and have this type of pain? How can we have no sensation and have numbness, but still have pain?  Many of us have experienced the attitude from others that “it must be in your head” so get over it. We discussed the use of medical marijuana for pain control.  Several people have tried it, some have issues with the fact it is illegal (federal government), and some that tried it found it to be a great help.

Fatigue Everyone in the group shared the experience of fatigue.  Not the kind of being tired, but the kind that will just suddenly come over you and you seriously need to lie down.  Many in the group try to push through it because of the attitude family members and friend have towards us.  We talked about how we hear things like “the job is exhausting for all of us, not just you…welcome to the club”.  When we can’t attend a family gathering, a get together with friends or just about any event because of fatigue, we are accused of just using it as an excuse.  Fatigue is something we understand as a group but have great difficulty getting others to understand.

This was not a gripe session positive influences come out of this wonderful group of people.  We have made some life long friendships.  We have gained understanding that we are not alone with this monster called TM.  We also share many laughs and tears.

We talked about depression and finding the right doctor.  Many of us have just quit going to a neurologist. We find that our family doc can treat our symptoms just as well.

Among many things we discussed, we talked about was what many of us called “invisible disabilities”.  Some of us are constantly challenged by the fact that we look normal.  What could be wrong with us, why are we using that handicapped parking space?  This is when using a wheelchair or a walker has its benefits.  We don’t have to justify that we are disabled.

At about 3 PM the group was surprised when Debbie Capen, the Secretary of the TMA and a Board Member joined us!  The whole group welcomed her.  Debbie and I started the group many years ago.  We worked hard before the first meeting and became BFF’s.  Debbie spent the night at my house and we got to catch up.

My thanks to Joan, Mona, Jan, Rick, Allen, Bob, Bill, Denise, Midge, Teri, Kathy and Ray, Nichole, and Debbie for making this a terrific meeting.

If you want to read how our meeting had an affect on one of our members, Allen Rucker, read the Support R Us blog published in September 2013!

~ By Cindy McLeroy 

Crowdsourcing Neuro-Immune Disorder Specialists

One of the most frequent requests we receive at the TMA is a referral to a specialist who understands TM, ADEM, NMO or ON. Finding these specialists is critical and also a challenge. It is a challenge because these disorders are rare and there are few centers and physicians who focus their specializations on these disorders. Finding the specialists is critical to people’s care, because there is very little published in the medical literature that offers definitive guidance about clinical care based on scientific studies. Today, almost all clinical care, including acute treatment, is based on expert judgment. A physician becomes an expert by caring for a large number of patients with these disorders and thus developing expertise.

We have just launched a web-based resource, the Physician Network, to address this critical need in our community. The network (www.myelitis.org/resources/physician-network) resource allows anyone to browse through a list of experts in the field of rare neuro-immune disorders. You are able to search based on disease expertise (ADEM, NMO, ON, TM) and by specialization (ranging from pediatric care to psychiatry to urology). The physician network is organized geographically so that you are able to search by location, as well.

The most exciting feature of this new resource is that the members of our community will be able to participate in expanding and growing our network of physicians. The TMA physician network includes the TM and NMO Centers at UT Southwestern and Johns Hopkins University and specialists from Multiple Sclerosis centers at major academic hospitals across the country. We want to hear from you! If you would like to share information about your physician or medical professional in your area who has been able to offer you excellent care through your journey with one of these rare neuro-immune disorders, please add them to the network and help us expand it. The link is www.myelitis.org/resources/physician-network. Using a community collaboration platform to grow our network of physicians and specialists, we can all participate in ensuring that everyone in our community receives care for their disorder. Please don’t forget to ask your doctor for his/her permission to be added to network. The TMA will verify the information that is provided and will confirm that the physicians and specialists added to the network have an open practice and are seeing new patients. Thus, there will be some time between the submissions from our community and when they are posted on the network.

By adding physicians who are offering you great care to the Physician Network, other people in your area will be able to receive excellent care from a specialist. The second very important benefit is that by being identified as a physician or medical specialist in our Network, the medical professional will begin to see and care for more people with these disorders, and thus will develop a more intensive and extensive experience with these disorders. As expert judgment guides acute and long-term care, the development of this experience is of critical benefit to everyone in our community.

It is important to keep in mind that finding a physician in your ‘area’ is going to be a challenge or impossible for some of you. If you live in a rural community or in a state such as Montana or Alaska, that have very few neurologists, scattered across very large distances, you are likely going to have to travel to a different state to find an expert.

Please join us in growing our network. If you have a physician that you think should be on our network, please add him or her to the list by going to www.myelitis.org/resources/physician.network. We look forward to hearing from you and are excited about the potential to improve medical care!