On Being Normal

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There’s a lot to be said about the value of people with multiple sclerosis being in the company of others who also have MS; there is a special comradery when we get together.  It used to surprise me when I met up with others with MS that we talk very little about our personal health situations.  Beyond the perfunctory chat of how are you doing or answering brief questions about therapy choices, we talk very little about our own relationship with this MonSter; perhaps it’s because we already know all too well the ups and downs of living with a chronic disease.

 

Discussions among people with MS are usually unlike how we interact with non-MSers – who want details about our life but mainly about our health and our MS. Those conversations which focus on our MS, while often necessary, are tiring.

 

When we are engaged with other people who have MS, we are among our peers – in other words, we are normal. Gathering with other people who have MS may be one of the healthiest mental activities for me to do.  We compare notes about life in general, and hold the same conversations others might have with their friends.  We share family news, we talk about future plans, we relax and just enjoy the company without having to explain our MS.

 

From left: Nicole Lemelle, Laura Kolaczkowski, Lisa Dasis, Dave Bexfield, Yvonne DeSousa, Matt Cavallo, Jodi Dwyer, Lisa Emrich, Jon Chandonnet, Joan Wheeler

From left: Nicole Lemelle, Laura Kolaczkowski, Lisa Dasis, Dave Bexfield, Yvonne DeSousa, Matt Cavallo, Jodi, Lisa Emrich, Jon Chandonnet, Joan Wheeler

I recently had the opportunity to rendezvous in Chicago with a number of other people who blog about multiple sclerosis – these are the trusted voices of our MS community.  We were at a Blogger Summit hosted by Novartis, and they provided transportation, lodging and food for the weekend.

 

The company refrained from discussing their particular MS drugs and instead wanted to know more about the needs of the greater community.  We were given unrestricted access to three experts in the MS field – a neurologist, urologist and MS nurse- for a panel discussion and Q&A on recent changes in the treatment of MS.  The majority of our meeting was focused on the Quality of Life (QOL) items that can make a huge difference.

 

My own QOL numbers spike each time I get to meet up with these bloggers and others who understand the 24/7 life of someone with MS. That evening after the meetings, a number of us were still in Chicago until our morning flights home. We ate deep dish pizza we had delivered to a sitting area in the hotel lobby, spread out and as comfortable as if we were in someone’s living room.  We strategized a bit about next steps in advocacy for the MS community but that’s a discussion removed from our personal daily lives. We talked about travel and our families and life in general and much more.  But we mainly laughed and relaxed, and in the moment we were normal.

be well,

Laura

 

 

 

 

Shhhhh! Don’t Tell the Neighbors

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We have wonderful neighbors – they’re not so much like the old-fashioned ‘can I borrow a cup of sugar’ type that were around for decades.  Now that the women have gone to work, including me, there isn’t time for much homemade baking or knocking on each other’s door just for a visit.

 

Instead, the new ideal neighbor we have are the men.  They are the ones who  don’t bring baked goods, but instead show up with their power tools.  They’ve fixed our deck.  They’ve repaired leaking plumbing.  We even had new electrical upgrades done by a neighbor (he’s a licensed electrician, fortunately).   They do these gratis – I wouldn’t think of offering them money at this point and instead find things to give in gratitude for their help.  It might be an ice cream cake from a local creamery or some strip steaks from the butcher shop. If I’ve had the luxury of time and able to crank up the BBQ smoker, I might gift them with racks of ribs or dishes of pulled pork.  You get the idea – I try to show our appreciation for their neighborliness.

 

So what is it I don’t want the neighbors to know?  I cut the lawn today.  Seriously, I cut the grass and it felt good.  You see, these helpful guys also own the ultimate power tool –  zero turn riding mowers, and it is fairly common for us to come home from work and find our lawn cut if it has grown a bit long and shaggy..  I hear you thinking now – what’s wrong with that?  Honestly, nothing EXCEPT I need the exercise.  It’s a great, and easy workout for me to mow our lawn with the self-propelled mower we bought two years ago specifically because it is easy to use.   Our daughter also helps when she is available..     I just start it, and hang on to the handle and it makes me walk.  I can adjust the speed from crawling to sprinting, and tend to do it on the low and slow side.

 

We have a half-acre lot with significant inclines and it is also a good upper body work out to keep the mower moving in a straight path, up and down those hills. It takes one us with our mower about 90 minutes to complete the job.  The guys can get this done in about 15 minutes.  But not only when they cut our lawn, am I cheated out of the opportunity to exercise they aren’t getting any workout either.

 

Two weeks ago I attempted to cut the lawn and  I stopped after about 20 minutes to take a break.  That’s a reasonable length of time in the heat and I know not to push and try to do it all at once.  I came inside and was sitting, chugging ice water when I hear a familiar sound ….  my husband went out to check and sure enough, one of the helpful neighbors was zooming around our yard on his rider.  Hubby signaled him to stop and we would do it, but this nice neighbor would have nothing to do with that, he wouldn’t even slow down, and in 10 minutes he finished the task I had begun.

 

Later, talking with his wife, I told her they are cheating me out of my exercise and she said he was  watching me  mowing, and was worried about me doing it myself. Never mind that he stopped a task he was doing for her to hop on the mower and help me.

 

At this point, I must tell you I have my own man and he’s great for minor car work and gas fill-ups, grocery shopping, and taking me out to eat,  but he has his own  physical and skill limitations. We have it worked out pretty well between his problems and my multiple sclerosis, we get along by doing tasks our way, in our own time.

 

So this morning, while it was still cool(er), and my neighbors were gone, I put my cell phone in my pocket in case I ran into problems and needed to call 911 for help, slipped on my nasty mowing shoes, and started the task.  It felt good to get out there but after 20 minutes or so, my body temperature rose, I was no longing pushing the mower but being dragged, even though I had it at the slowest speed,  and I knew I was at my limit.  I  put the mower aside and came into the house to cool off.  As I sit here with an ice pack on my neck, stripped half-naked,  and trying to cool down, I can’t help but think for at least a fleeting moment …..wouldn’t it  be nice if someone else finishes the job? Shhhhh!!  Don’t tell the neighbors.

You May Want to Read these posts on MS.net

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May was a busy month – between wrapping up the MS news from AAN in April to covering the annual Consortium for MS Centers meeting in the end of May, my typing fingers have worked overtime to keep up.  I’ve been busy writing for multipleslcerosis.net  and here are the links and a bit of a tease for each of my  articles published on their site in May.  I hope you find something worth your time to read ….


How to Find a Good Neurologist

By Laura Kolaczkowski—May 31, 2015

 

Some of us have been fortunate to connect with doctors and other medical care providers who we are pleased with, and we often feel like  winners in some big lottery; but then there are those people who are still seeking the right team members. People will often ask how to find a good doctor and I’ve accumulated some tips that might be useful if you are in the still searching group.

http://multiplesclerosis.net/living-with-ms/how-to-find-neurologist/

 

When Can We Stop Taking Our MS Drugs?

By Laura Kolaczkowski—May 27, 2015

It’s easy to be lulled into thinking we are better in our health and stop taking our medications. That is why there is that warning notice on the bottles of antibiotics you might be prescribed for infections – the one that says ‘important! Take this medicine until gone.’ The medical experts know we need to take all of these drugs to really eradicate the cooties they are prescribed to cure. If we stop them too soon, it is always possible the infection is still there and will come back and create more problems.

http://multiplesclerosis.net/living-with-ms/when-can-we-stop-taking-our-drugs/

 

For Frequent & Not-so-Frequent Flyers

By Laura Kolaczkowski—May 19, 2015

 

Through the advocacy work I am doing with the Accelerated Cure Project and the iConquerMS™ patient registry (have you joined yet?) for PCORnet, I have been making frequent trips by commercial airline to attend meetings.  Until this began I would make maybe one flight per year for a leisure trip.  Anyone who has flown in recent years knows the additional precautions that are taken when going through airport security.  If you travel by air, whether often or infrequently, I thought it might be useful to share this with you because you also know about these challenges to get to the other side of the checkpoints.

http://multiplesclerosis.net/living-with-ms/for-frequent-not-so-frequent-flyers/

 

Your MS Isn’t My MS

By Laura Kolaczkowski—May 13, 2015

 

It sounds like a variation of a school yard taunt or bragging – but my MS isn’t your MS.  So often when we are in conversation on line with others and talking about our particular course of multiple sclerosis, it is only natural to compare where we are with this disease to how others are doing with their own MS.

And that is where we have to put the brakes on and bring  the conversation train to a screeching halt. Your MS is never going to be like my MS, and that is part of what is so confounding and confusing about this disease for us, our family and friends, and even the doctors and researchers.

http://multiplesclerosis.net/living-with-ms/your-ms-isnt-my-ms/

 

Sticking with Tysabri, but Less Often May be Possible

By Laura Kolaczkowski—May 7, 2015

 

There has been quite a bit of exciting news from the American Academy of Neurology (AAN) conference this year; a lot of it has been about new ways of looking at existing drugs. A study that has my particular interest is a look at natalizumab dosing and if it can be given less frequently and still be an effective multiple sclerosis disease modifying therapy treatment.

http://multiplesclerosis.net/living-with-ms/sticking-with-tysabri-but-less-often-may-be-possible/

 

The Name Game

By Laura Kolaczkowski—May 4, 2015

 

Are you old enough to remember the song The Name Game, where letters in names were replaced with other letters and you came up with a nonsensical line such as this one for the name Shirley –

Shirley, Shirley bo Birley Bonana fanna fo Firley
Fee fy mo Mirley, Shirley!

Those names made out of Shirley are much like what I’ve been experiencing this week.  For some reason I lost the proper names of many long time friends and even family members, and have been creating nonsense names in their place to fill in the gaps.

http://multiplesclerosis.net/living-with-ms/the-name-game/

 

For a further look into the archives and my writing published through MultipleSclerosis.net, you can find my index of over 160 articles,  at http://multiplesclerosis.net/author/laura/

 

 

Considering A Break

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I’m contemplating taking a break from my DMT.  I’ve been on natalizumab (Tysabri) for over three years and this winter I have been plagued with recurring infections.  In particular I’ve had conjunctivitis on and off since the end of January.  It goes away with treatment and then comes back as soon as the drugs stop.  It’s been medically treated in a variety of ways and with assorted  meds and nothing seems to stick.  I’ve felt worn down and dragging , as this wicked little infection continues to have its way.  The only thing that makes sense to me is my immune system is tired, too.  Perhaps the Tysabri has my system so suppressed that it doesn’t have anything remaining to fight this one.

I sent a message to my neuro about taking a break – perhaps  just a short one of a few months to see if I might feel better or at least this pink eye will stop and I’ll at least look healthier.   My doctor’s  reply – ‘let’s talk. We need to have a plan.’ So we will talk – which means I will talk and he will listen.  And then he will talk and I will listen.  And then he will ask me what I want to do, because that is how he treats his patients .  And most probably I will doubt whatever choice I make because I also know about the rebound effect coming off Tysabri can be nasty, but somewhat controlled with steroids.

I’m not wanting to stop for forever because I’m not willing to bet against the house in this poker game and I believe the science that DMTs delay progression.  But if I had to decide on my own at this very moment I would be packing my bags for my first DMT  break since I was diagnosed in 2008. Fortumately I have an expert to confer with before making a final decision, but I can’t help but think everyone loves a holiday, right?

Patient Powered Research Networks (PPRN) & You

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Do you wonder how you might get involved with research in a simple way? You can always connect with one of the Patient Powered Research Networks (PPRN’s) that are actively looking for people to share their health data and research questions. PPRN’s are an offspring of the Patient Centered Outcome Research Institute (PCORI) and cover a wide range of chronic diseases and conditions.

 

I write often about the Multiple Sclerosis PPRN, iConquerMS™, and my role as the Lead Patient Representative and now also the Chair of the Governing Board.  I would love to have all of you join our PPRN and join the Big Data movement to find the answers about multiple slcerosis.

You can learn about PPRN’s, and PCORI through our portal at www.iconquerms.org.  But perhaps you have other conditions you live with and want to do more.

 

Following is the complete list of PPRNs and some of their identifying information.  I encourage you to read through the list, explore the associated links and see where you might help advance research.  At the very least, we all qualify for Health eHeart, an ambitious heart history registry that only takes a few minutes to complete and is for everyone, whether we  have heart disease or not.

 

We can only build this new way of doing research by getting the  help of many people,  and that includes you.

Be well,

Laura

 

PPRN’s of as May 1, 2015 and their information, from data I have compiled. Let me know if you spot omissions or errors so I can make the corrections.

 

ALD Connect

Network name:  ALD Connect

Website: http://www.aldconnect.org/

Project summary available at:  http://www.pcori.org/research-results/2013/ald-connect

Organization: ALD Connect, Inc.

Condition: Adrenoleukodystrophy

Population Characteristics: Pediatric and Middle Aged

 

ABOUT – American BRCA Outcomes and Utilization of Testing Patient-Powered Research Network (ABOUT Network)

Network Name:   ABOUT  Network

Website: https:aboutnetwork.org

 

Project summary available at http://www.pcori.org/node/4408

Organization: University of South Florida

 

Condition: Hereditary Breast and Ovarian Cancer

Population Characteristics: Female

 

AR-PoWER

Arthritis Patient Partnership with Comparative Effectiveness Researchers (AR-PoWER)

Network name: AR-PoWER

 

Website:  in progress

Project summary at http://www.pcori.org/node/4401

 

Organization: Global Healthy Living Foundation

Project summary available at http://www.pcori.org/node/4401

 

Condition: Arthritis (rheumatoid arthritis, spondyloarthritis), musculoskeletal disorders (osteoporosis), and inflammatory conditions (psoriasis)

Population Characteristics: All

 

The CCFA Partners Patient-Powered Research Network

Network name:  CCFA PPRN

 

Project summary available at http://www.pcori.org/node/4397

Website:  https://ccfa.med.unc.edu/user/sign_up

Organization: Crohn’s and Colitis Foundation of America, Inc.

Condition: Inflammatory bowel disease (Crohn’s disease and ulcerative colitis)

Population Characteristics: All

 

CENA – Community Engaged Network for All

Network name: CENA

Website: http://www.jsrdf.org/JSLIFE

 

Project summary available at http://www.pcori.org/node/4400

Organization: Genetic Alliance, Inc.

Condition: Alström syndrome, Dyskeratosis congenital, Gaucher disease, Hepatitis, Inflammatory breast cancer, Joubert syndrome, Klinefelter syndrome and associated conditions, Metachromatic leukodystrophy, Pseudoxanthoma elasticum (PXE), Psoriasis

 

Population Characteristics: All

 

The COPD Patient-Powered Research Network

Network name: COPD PPRN

 

Project summary available at http://www.pcori.org/node/4396

Website: www.COPDPPRN.org

 

Organization: COPD Foundation, Inc.

Condition: Chronic Obstructive Pulmonary Disorder

Population Characteristics: Multiple co-morbidities

 

 

Duchenne Connect Patient-Report Registry Infrastructure Project

Network name: DuchenneConnect Patient Registry

Project summary available at http://www.pcori.org/node/4404

 

Organization: Parent Project Muscular Dystrophy

Condition: Duchenne and Becker muscular dystrophy

Population Characteristics: Pediatric

 

Health eHeart Alliance

Network name:  Health eHeart Registry

Organization: University of California San Francisco

Website: https://www.health-eheartstudy.org/

Project summary available at http://www.pcori.org/node/4407

Condition: Cardiovascular health

Population Characteristics: Multiple co-morbidities

 

ImproveCareNow: A Learning Health System for Children with Crohn’s Disease and Ulcerative Colitis

Network name: ImproveCareNow Registry

Website: https://improvecarenow.org/

Project summary available at

Organization: Cincinnati Children’s Hospital Medical Center

 

Mood Patient-Powered Research Network

Network name: MoodNetwork

Website: www.moodnetwork.org

 

Project summary available at http://www.pcori.org/node/4403

Organization: Massachusetts General Hospital (The General Hospital Corp.)

Condition: Major Depressive Disorder (MDD) and Bipolar Disorder (BP)

Population Characteristics: Multiple co-morbidities.

Seeking to enroll : individuals with mood disorders (unipolar depression and bipolar disorder)

 

Why should someone join this PPRN: To help prioritize the research questions for mood disorders, to share data to better understand mood disorders and create better interventions, and to join a community of people actively seeking answers to the diagnosis and treatment of mood disorders.

(thanks to Dan Goodman, Mood PPRN Patient Rep, for the additional information)

A Multiple Sclerosis Patient-Powered Research Network

Network name: iConquerMS™

Organization: Accelerated Cure Project for Multiple Sclerosis

Website: www.iConquerMS.org

Project summary available at  http://www.pcori.org/node/4391

 

Condition: Multiple Sclerosis, including RRMS, SPMS and  PPMS,  as well as CIS and RIS

Population Characteristics: 70% Female

Enrolling controls: Yes

Why should someone join this PPRN?  iConquerMS is a unique opportunity to  share data and research questions from  a patient centered approach to find the cause of multiple sclerosis, and identify ways to improve the quality of life of people living with MS.

 

NephCure Kidney Network for Patients with Nephrotic Syndrome

Network name: NephCure Patient Registry

Organization: Arbor Research Collaborative for Health

Website:  https://www.nephcurekidneynetwork.org/

 

Project summary available at http://www.pcori.org/node/4394

Condition: Primary Nephrotic Syndrome (Focal Segmental Glomerulosclerosis [FSGS], Minimal Change Disease [MCD], and Membranous Nephropathy [MN])

Population Characteristics: All

 

The Patients, Advocates and Rheumatology Teams Network for Research and Service (PARTNERS) Consortium

Network name: PARTNERS

 

Project summary available at http://www.pcori.org/node/4398

Organization: Duke University

Condition: Juvenile Rheumatic Disease

Population Characteristics: Pediatric

Enrolling controls:

 

 

Phelan-McDermid Syndrome Data Network

 

Organization: Phelan-McDermid Syndrome Foundation       Phelan McDermid Syndrome Foundation

Website:  https://pmsiregistry.patientcrossroads.org/

 

Project summary available at http://www.pcori.org/node/4405

Condition: Phelan-McDermid Syndrome

Population Characteristics: Pediatric

 

PI Patient Research Connection: PI-CONNECT

Network name: Pi-Connect

Organization: Immune Deficiency Foundation

http://primaryimmune.org/about-primary-immunodeficiencies/

 

Project summary available at http://www.pcori.org/node/4402

Condition: Primary Immunodeficiency Diseases

Population Characteristics: All

 

Rare Epilepsy Network (REN)

Network name: Rare Epilepsy Network Registry

Organization: Epilepsy Foundation

Website: https://ren.rti.org/

 

Project summary available at http://www.pcori.org/node/4399

Condition: Aicardi Syndrome, Lennox-Gastaut Syndrome, Phelan-McDermid Syndrome, Hypothalamic Hamartoma, Dravet Syndrome, and Tuberous Sclerosis

Population Characteristics: Pediatric

 

Sleep Apnea Patient Centered Outcomes Network (SAPCON)

Network name: MyApnea

Organization:American Sleep Apnea Association

Website: https://myapnea.org/

Project summary available at http://www.pcori.org/node/4393

Condition: Sleep Apnea

Population Characteristics: Multiple co-morbidities

 

 

The Vasculitis Patient-Powered Research Network

Network name: Vasculitis Patient-Powered Research Network

The Trustees of the University of Pennsylvania

Website: http://www.vpprn.org/

 

Project summary available at http://www.pcori.org/node/4406

Condition: Vasculitis

Population Characteristics: Adults

 

 

 

 

 

 

 

PCORI CER Results

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Last month the PCORI group charged with looking into Multiple Sclerosis clinical effectiveness research questions  convened a meeting of  people who had various interests in the task.  I was asked to participate  because I am the lead patient representative for iConquerMS™  with the Accelerated Cure Project for the MS PCORnet.

I wrote about this meeting and asked for input from anyone who cared to share their preferences of choices  for possible research questions.  Thank you if you took the time – I did include your preferences in noting the selections i entered.

If you did not see it, you might want to back up and read my original post before you go any further with reading  this one so you understand the context of the rest of what I am going to write.

PCORI and Me and You!! – Help Prioritize Research Topics

Now the prioritized questions have been returned to us and I have those in two different forms; the first is the overall ranking of the questions and the second group is a look at how each interest group cast their preferences.  Keep in mind  there were four people with MS out of 40+ people who sat in on this discussion, and our voice did not carry the weight to match that of the other parties. The number of participants in each group were:

Clinician/Medical Professional (7)

Industry (12)

Other (5)

Patient Advocacy Organization (5)

Patient (4)

Payer (5)

Researcher (3)

PCORI will further refine these questions and then possible issue a call for proposals from researchers to do one or more of these.  The earliest that might happen would probably be August or September.

Here are the results in both forms –

OVERALL RANKINGS

Question
Score
# who ranked it at all
# who ranked it #1 or #2
J.  What are the comparative benefits and harms of non-pharmacological and pharmacological approaches in relation to key symptoms (e.g., emotional health, fatigue, cognition, pain) in people with MS?
101
23
14
F.  In people with progressive MS, what is the comparative effectiveness of different care delivery approaches (i.e., MS specialty center vs. community neurology; direct care vs. telemedicine; “specialized medical home” vs. community neurology delivery of care) in improving outcomes such as functional status, quality of life, symptoms, ER use, and hospitalization?
93
29
7
I.  Does an integrative model of care along with DMT in a newly diagnosed individuals affect disability progression and symptoms (physical, emotional and cognitive) compared to treatment with DMT alone?
89
24
9
A:  What are the comparative benefits and harms of different disease-modifying therapies in newly diagnosed relapsing, remitting multiple sclerosis on disease activity, disease progression, symptoms, and quality of life?
87
20
11
B.  Among MS patients receiving a DMT who experience disease activity, what are the benefits and harms of continuing the same therapy versus changing to a new medication?
85
22
9
C.  Is treatment escalation using DMTs as effective as starting treatment with higher efficacy treatments in early active, previously untreated patients?
65
19
5
E.  What is the comparative effectiveness of stopping versus continuing therapy after a period of prolonged disease stability in patient with MS?
60
16
6
L.  What are the benefits and harms of early vs. delayed treatment with DMTs, in terms of symptoms, function, QOL, and disease activity in treatment-naive patients recently-diagnosed patients (meeting McDonald criteria within 12 months)?
60
21
5
M.  In patients who recently transitioned from relapsing to progressive MS or were recently diagnosed with SPMS, what are the benefits and harms of continuing compared to discontinuing DMTs on outcomes including but not limited to symptoms, QOL, function, disease activity, disability, and/or mortality?
59
17
7
H.  In people with relapsing MS, what is the comparative effectiveness of physician-directed vs. allied health-directed vs. navigator-directed, vs. technological-enabled self-management tools for improving initial decision making, patient care experiences, decision regret, quality of life and adherence to therapy?
54
18
2
G.  In people with relapsing MS within 2 years of diagnosis, what is the comparative effectiveness of changing DMT using a NEDA strategy (no relapse, no new MRI or enhancing lesion, no change in disability) vs. not changing DMT in terms of functional status, quality of life, symptoms, ER use, and hospitalization?
45
16
3
K.  What are the comparative benefits and harms of specific dietary regimens in people with MS?
37
14
3
D.  What is the comparative effectiveness of smoking cessation efforts upon disease activity, progression, symptoms, and quality of life in MS?
26
7
1
Total
 
246
82

 

 

This attachment is an Excel spreadsheet with all of the various stakeholder groups itemized.  I have to share this as an attachment because the file is so wide and spreads across a large number of columns and rows.

MS CER Ideas per interest group

Please be sure and tell me what you think after you have the chance to review these results. And a special thanks to everyone who  helped me in the first round to rate them on our behalf.

be well,

Laura

PCORI and Me and You!! – Help Prioritize Research Topics

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Forty-one stakeholders with a keen interest in Multiple Sclerosis, gathered in Washington DC on April 2, to develop, discuss and submit topics to be considered by PCORI for Comparative Effectiveness Research (CER) in MS.  In preparation for the meeting, the stakeholders were invited to submit topics for consideration and the lead science writers and researchers for PCORI consolidated the ideas into central themes; you can read those ideas in the summaries available at http://www.pcori.org/events/2015/prioritizing-comparative-effectiveness-research-questions-treatment-multiple-sclerosis. Also available at this link are the other meeting materials, a list of attendees and their affiliations, and recordings of each session.

I was invited to participate because of my connection with the PCORI funded PCORnet MS-PPRN, and my role as the lead patient representative for Accelerated Cure Project’s  iConquerMS™  (please enroll today, if you haven’t already done so!) patient powered research network.

 

There were four central themes identified for the day’s discussions, and we worked within an assigned group to come to a consensus of how a CER question might reflect our individual submissions.

The four groups were:

Group 1 – Comparison across DMTs, including differential effects in subgroup

Group 2 – Care Strategies

Group 3 – Non-pharmacologic and non-DMT therapy for specific symptoms and overall health

And

Group 4 – Timing of therapy and study design

 

.

As you might already know,  PCORI begins with the words Patient Centered, and I raised concerns that of the 41 participants, only four of us identified ourselves as people living with multiple sclerosis.  There were a few more representatives from the Accelerated Cure Project, National MS Society and the Multiple Sclerosis Foundation.  The remaining participants were connected with MS pharmaceutical companies, government agencies, managed care (both medical and pharmacy benefits) organizations and physician groups.  The patient voice was definitely in the minority in these sessions.  That said, I was a participant in group #2 and felt my ideas were well received and the chair of the discussion, Dr. Alex Rae Grant, actively sought my input at several points.

 

Toward the end of the day, we reconvened and each group presented their revised questions, and it was then opened for discussion. There was little discussion or debate about the proposed questions and the chair of the talks turned the questions toward the people with MS who were in the room, asking for our thoughts about the day, the process and the proposed topics.  Although it felt as if there was empty time on the schedule that needed to be filled with the impromptu shout-out for our reactions, I believe we all welcomed this additional opportunity to speak to the larger issues.

 

The proposed CER topics for consideration for funding are now being prioritized by the 41 participants, myself included.  We have been polled electronically and asked to rank the importance of these topics, and from those rankings the PCORI decision makers will choose what might be put forth in a request for proposal (RFP) to fund Multiple Sclerosis research.  If you are familiar with PCORI, there are some restrictions on what they will fund – they must always look at CER, and the cost of treatment can never be considered.

 

An email was sent to all participants, and by Wednesday, April 8, I am asked to prioritize the order of these suggested topics and indicate which SIX (6) I would rank with the highest priority and add any additional comments I might wish to include to support my choices. Following are the proposed CER questions:

 

A: What are the comparative benefits and harms of different disease-modifying therapies in newly diagnosed relapsing, remitting multiple sclerosis on disease activity, disease progression, symptoms, and quality of life?

B: Among MS patients receiving a DMT who experience disease activity, what are the benefits and harms of continuing the same therapy versus changing to a new medication?

C: Is treatment escalation using DMTs as effective as starting treatment with higher efficacy treatments in early active, previously untreated patients?

D: What is the comparative effectiveness of smoking cessation efforts upon disease activity, progression, symptoms, and quality of life in MS?

E: What is the comparative effectiveness of stopping versus continuing therapy after a period of prolonged disease stability in patient with MS?

F: In people with progressive MS, what is the comparative effectiveness of different care delivery approaches (i.e., MS specialty center vs. community neurology; direct care vs. telemedicine; “specialized medical home” vs. community neurology delivery of care) in improving outcomes such as functional status, quality of life, symptoms, ER use, and hospitalization?

G: In people with relapsing MS within 2 years of diagnosis, what is the comparative effectiveness of changing DMT using a NEDA strategy (no relapse, no new MRI or enhancing lesion, no change in disability) vs. not changing DMT in terms of functional status, quality of life, symptoms, ER use, and hospitalization?

H: In people with relapsing MS, what is the comparative effectiveness of physician-directed vs. allied health-directed vs. navigator-directed, vs. technological-enabled self-management tools for improving initial decision making, patient care experiences, decision regret, quality of life and adherence to therapy?

I: Does an integrative model of care along with DMT in a newly diagnosed individuals affect disability progression and symptoms (physical, emotional and cognitive) compared to treatment with DMT alone?

J: What are the comparative benefits and harms of non-pharmacological and pharmacological approaches in relation to key symptoms (e.g., emotional health, fatigue, cognition, pain) in people with MS?

K: What are the comparative benefits and harms of specific dietary regimens in people with MS?

L: What are the benefits and harms of early vs. delayed treatment with DMTs, in terms of symptoms, function, QOL, and disease activity in treatment-naive patients recently-diagnosed patients (meeting McDonald criteria within 12 months)?

M: In patients who recently transitioned from relapsing to progressive MS or were recently diagnosed with SPMS, what are the benefits and harms of continuing compared to discontinuing DMTs on outcomes including but not limited to symptoms, QOL, function, disease activity, disability, and/or mortality?

 

Which brings me to the point I made in the meeting – I am just one person with MS and there are at least 400,000 of us in the United States alone, trying to make sense of living with multiple sclerosis.  Our perspective on what is important to study may be totally different than the perspective of these other stakeholder groups and I would like your input on this list of topics and what you believe should be prioritized.

 

Would you look at these topics, consider their significance to you, and tell me what you would rank toward the top of your list?   If you have reasons why they are important, I would love to hear that too.  I will consider all the comments and put those together with my own and submit ‘our’ rankings to PCORI. You don’t need to give me six but if you would at least give me a top three, that would be helpful to consult as I propose our slate.

 

This is due to PCORI  on Wednesday, so please help me by commenting no later than Tuesday afternoon.  Your help with this important assignment is greatly appreciated and I thank you in advance.

 

Be well,

Laura

 

A Familiar Tale

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It was the daily routine: wake, dress, pop a handful of pills, and then slowly make the way out the door.    Each morning begins with the same question, what is the day going to hold?   Not daring to look back or forward, she focuses to stay in the moment – life at age 55 could be so much better but she realizes it could also be worse.  She wanders home after being out for the day, feeling the stress through her legs, torso and up into the brain.   Life is sure a challenge living with MS.  She eases into the routine of taking more pills, selecting a spot on her weary body for the next injection, and pauses to think how much better life could be if it weren’t for this damn multiple sclerosis.  Physically spent, she drops heavily into her favorite chair and the fatigue washes over her.  Almost instantly, she falls into a heavy sleep.

 

When she awakens, it’s with the usual sluggishness that tells her the body is again under the spell of MS and awaiting the next pharmaceutical jolt to get moving for the day.  She reaches to the table beside her and is immediately gripped by panic  –  Where are the pills?  Her home has a lineup of bottles and elixirs and syringes, all in order and serving a purpose – although not perfect, these medicines slow the progression of her MS and keep her moving. Where are they now??

 

She tries to stand but can’t move – looking down, she is gripped with confusion because she is not in her favorite easy chair but seated in a wheelchair.  How could this have happened ?  She was taking her medicines and although life with MS was a struggle, she was still functioning.  Why was she no longer moving on her own?  She releases the brake and wheels herself across the room and catches sight of a reflection in the mirror – it is a younger vision of herself, possibly no older than 25.  She is confused and spins around to head the other direction and is faced with a calendar hanging on the wall open to 1985. How could that be?  Slowly it occurs to her that she’s no longer in the present, but has somehow slipped into an era where there were no treatments for MS and people were left to languish, destined to finish the final years of their  lives in wheelchairs or worse.

 

She closes her eyes and feels the anguish of helplessness well up inside – at least in her present day there were options with disease modifying therapies.  Here there is nothing but a feeling of hopelessness.  She closes her eyes, and drops her head back, thinking of how someday things  will be different.   Surely a cure can’t be that far away, can it?

 

She allows herself to be lured into that almost unthinkable  thought – a world without Multiple Sclerosis – and startles to find herself whole, as if stepping out of a cloud onto a movie set.  Everything’s perfect, including her body, except for the added years. She may have aged, but she immediately recognizes she hasn’t felt this strong and connected in decades.  How could this be? She grabs a nearby computing device and quickly searches and finds that MS has been cured.  Ways to remyelinate the body were discovered in labs, a vaccination was developed, the cause of MS was found and the risk eradicated, and no one will contract this disease again.   Overjoyed, but also perplexed, she wonders how could this have possibly happened?

 

Reading beyond the headlines, she finds the answer –  Just as brave people with MS had offered themselves for trials to study ways to treat MS, the next generation of forward thinking people gave even more – they gave their history, their samples and their ideas, and together formed the big data that allowed researchers to deconstruct MS once and for all.   She’s left smiling with gratitude to the people who made the evolution of MS care possible.

 

Yes, this is a Dicken’s of a tale, but not so unimaginable.  Multiple Sclerosis research of the present, past and future is shaped by people living with MS. When we commit our time and resources to help, we build on what others have contributed to help move closer to a world free of MS.   Join me in sharing your MS history and ideas at https://iconquerms.org , the gateway to our future and the time when a cure is found.  Don’t wait until tomorrow – do it today!

 

Wishing you well,

Laura

 

 

 

 

 

Learning More about Lemtrada

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The PR firm representing Genzyme Sanofi and the launch of their newest MS drug, contacted bloggers with the offer to speak with one of their neurologists about Lemtrada, the trade name for alemtuzamab.   Never one to turn down the opportunity to speak one on one with these specialists, I was happy to accept their offer and was connected with Dr. Ann Bass, from the Neurology Center of San Antonio.   Fortunately they allotted 30 minutes to talk by phone and we used every bit of that and could have used more.  There is a lot to talk about – this new MS treatment option has the MS community buzzing with excitement about the potential. Following are the questions and answers we exchanged and our discussion:

Q: Lemtrada has been approved for RRMS but will it also be studied for SPMS and PPMS? 

Dr. Bass immediately pointed out something I had not caught –Lemtrada is approved for relapsing forms of MS and that is not limited to just Relapsing Remitting MS, and it could include Secondary progressive MS. She said there are no plans for a study for its use in primary progressive MS (PPMS), and cited an older study that showed the drug, alemtuzumab showed no benefit in PPMS.   I asked if there might be doctors who would try to use it off label for PPMS and she said that would be highly unlikely because of all the serious warnings that accompany this drug, and the extreme exposure to liability a doctor would face using it in an unapproved manner.

 

Q: What criteria might a doctor use to decide if this would be a suitable choice for a person to try?

Dr. Bass  said the most obvious will be if a person fails two other types of drugs and is still relapsing.  This is the use the FDA approved, but she did say if a person had aggressive MS and it was deemed a medical necessity, the neurologist could strongly advocate for a person to be treated with Lemtrada as a first round of MS drugs.   She also said Lemtrada may be a good choice for someone who tests positive for the JC Virus and wants to discontinue Tysabri, because as of now they found no link between PML and Lemtrada.

 

Q: Lemtrada is intended to be given in multiple doses – five doses , five days apart and then nothing for a year.  At the beginning of the second year, the person is given three additional doses.  If necessary, a person might need to take additional rounds of alemtuzumab  for a third or even fourth year.  Can you share those numbers as to how many people needed additional rounds of treatment?

 

Dr. Bass said in the studies, 80% of the people were fine with treatment for just 2 yrs.  20% needed an additional round for the third year and 10 % needed treatment for the fourth year.    The need for additional treatments is based on finding two indications that additional treatment is needed, and could include clinical evidence, EDSS changes, or new lesions and enhancing lesions on MRI exam.

 

Q: Since we all respond to MS treatment in different ways how quickly would a person on Lemtrada see an improvement or would they have to wait the entire year to see cumulative improvement?

Dr.  Bass said the response is noted fairly fast, within the first few months for most people , with the full effect being felt in 6-12 months. It can take some time for the immune system to be rebooted after it is suppressed by the alemtuzumab.

 

Q: Many of the MS  therapies require a washout period (time without a drug so it can leave your system) before trying a different drug.  Is there a washout period if a person stops taking Lemtrada?

It is no surprise that Dr. Bass said there is a neurologist online community that regularly talks and this is a hotly debated question, because no one really knows that answer.  Their big debate is while the  lymphocytes were repopulating would there be a benefit to be on something else? The answer is not known at the present time.

 

Q: We then talked about side effects – there is a 30% rate of thyroid disorders reported among the people in the Lemtrada trial . What percentage of those are thyroid cancers?

She said that hyperthyroidism and hypothyroidism was the majority of that number, but 5% were cases of thyroid cancer.   Of the people with the hyper/hypothyroidism, 80% were reversible with treatment after it was detected.  20% of those cases had surgery to remove the thyroid and will need a lifetime of treatment to compensate for that loss.

 

Q: If a person has a negative reaction to Lemtrada, can the drug be removed from the system like Tysabri?

She says the drug itself has a relatively short life in the body and it is gone quickly but it is the effect of alemtuzumab that can’t be reversed  – it is long lasting, and suppresses B cells for 6 months and T cells for 12 months.  Those effects cannot be reversed. Because of the suppressed immune system people can be more at risk for infections, but it is a catch-22 because if you treat the infections, you are counteracting the lemtrada effects  and low counts repopulate sooner and bring back the disease activity.

 

Q: Alemtuzamab was used for many years as Campath, a  chemotherapy drug for leukemia.  Will Lemtrada be administered the same way?

Dr. Bass says Lemtrada will be given by IV, the same as Campath, and the times will be similar.  A treatment of Lemtrada will involve the infusion to take place over 4-6 hours and that an additional 2 hours of observation, making it at least an eight hour day.  Because of the side effects of this drug which is a potent chemotherapy agent,  they recommend pretreating the person with other pharmaceutical to help counteract the side effects.

Q: Can the cost of Lemtrada, versus what it was as Campath be justified?

Dr. Bass said she had not seen the final cost for Lemtrada but feels it needs to be a personal choice and not dictated by price.  She also discussed the long term costs to a person with MS and how those might be decreased if a person with Lemtrada remains healthy and free from MS progression.

 

Since our interview I have thought of a few more questions, but I appreciated the openness Dr. Bass displayed discussing the pros and cons of Lemtrada.  She noted that her clinic was a trial site for Lemtrada and she had several years experience with the drug,  and she is a compensated  spokesperson for Genzyme Sanofi.

 

I hope this gives you some additional insight into Lemtrada and what needs to be considered if you think this might be the next drug choice for you.

Be well,

Laura

 

You & I Conquer MS

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iConquerMS™ sounds like a solitary activity, doesn’t it? But imagine if a bunch of us with Multiple Sclerosis got together and from all of us sharing notes on our individual histories we develop new data and ideas that will give researchers a bigger look at MS from our experiences.

Moving data from being a single contributor to collecting the data from many is often called BIG DATA and iConquerMS™ is looking to create some of the biggest data yet on MS.  We can’t do this alone – we need each and every one of you to join.

iConquerMS™ is an online patient portal led by the Accelerated Cure Project (ACP),  a non-profit organization that  has the ultimate goal – to find a cure for MS.   ACP received the funding from the Patient-Centered Outcomes Research Institute (PCORI) to develop iConquerMS™.  I have been involved with ACP and this project for over a year and can say I have never worked with a more sincere and dedicated group of professionals than their small but mighty staff. They have earned my trust and respect.

Remember the part of PCORI that stands for Patient Centered?  I am proud to say that the majority of people on the Governing Board, and the other committees that are overseeing iConquerMS™ and making the critical decisions and choices are just like you and me – people with multiple sclerosis.  This project could not be more patient centered than it is already.  Patients helped design it, patients run it, and it will be successful because patients will join and contribute their own data.

As people living with Multiple Sclerosis we often complain that we aren’t recognized as experts on the disease.  Who else besides us really knows what it is like to live in a body with MS?  We have knowledge that others don’t … our own experiences.  And now, through iConquerMS™ we can share those experiences and our information and together all of us are poised to make a difference.  If you’ve paid attention to the medical news in recent years, patient centered care is part of our health care reform – isn’t it about time you and I are the important ones in our health care?

The idea behind iConquerMS™ is really quite simple  -there is a web portal at iconquerms.org where people with MS create an account, complete some basic surveys, share their health records, and propose questions that researchers might study.

I am also happy to tell you that every one of the major MS organizations in the United States have endorsed iConquerMS™, including NMSS, MSAA, MSF and CanDoMS.  Not only have they endorsed it, leaders from each of these groups participate on the various committees.  They believe in the work of ACP and what iConquerMS™ can do for the future of MS research. There is a lot more information about this project, who is involved and what you can expect on the website and I hope you will take the time to learn more.

This is my brief outline of what you can expect to do when you join me at iConquerMS™:

  • Register at iconquerms.org. You will find informed consent information that tells you the details on how your data will be used and how it will be de-identified so your individual identity is not attached to the records
  • Enter your demographic data – we really do need to know those basic things about you to help figure out where you fit in the Big Data picture
  • Complete some surveys about life with MS –you have probably done these at other times for your doctor but are very important to add to our Big Data
  • Share your electronic health records – there are great instructions on how you can do this and you can always contact someone at iConquerMS™ for help if you get stuck on this part
  • Submit your questions of what you think researchers should take a closer look at – the research committee, made up of people with MS and key scientists will sort through our questions and make recommendations
  • Then share your experiences with iConquerMS™ with everyone you know – to gather really Big Data, we are aiming for 20,000 participants and we need your help to spread the word and recruit others
  • Expect to receive updates from iConquerMS™ via email (you can always opt out of these) on data gathering and research interests

I won’t lie and tell you all of this is easy – because it isn’t. It’s going to take some time to work through all of these bits of information and iConquerMS™ is designed so you can start and stop your entry of data when you need to take a break.   But it obviously hasn’t been easy to find a cure for MS either since that hasn’t been done yet – and who knows what one key piece of our collective information might unlock the answer.

Isn’t it  worth our time and efforts to try to find the answers? I want to be able to say we conquered MS  through iconquerMS™.  This is only going to happen when we all join together and I hope you will accept this invitation and register today.

Be well,

Laura

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