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 MW.

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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MS Support in the headlines

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It was a busy week for announcements in the media for new programs that will feature “big” names  for some big name players in MS.

The first is the news that Brigham and Women’s Hospital in Boston will team with others to open the Ann Romney Center for

Neurologic Diseases in 2016.  Romney and her husband Mitt – yes, those Romneys –  have talked openly about her life with MS and they will lead a $50 million fund raising campaign this next year.  I have not seen how much they personally donated, but the sum must have been significant to have the center  named after her.  The center will do research work on primarily five neurologic disorders: MS, ALS, Alzheimers, Parkinson’s and brain tumors.  We all know there are over 2.2 million people worldwide who live with MS, but we are just a small part of the 50 million who live with all of these diseases combined; research for these is seriously underfunded and this commitment of resources is significant.

 

Other news from the Boston area came from the launch of new patient outreach initiatives by MS pharmaceutical companies Biogen Idec and Genzyme.

 

Biogen’s MS Blueprint™ features Gretchen Rubin as the celebrity spokesperson.  Rubin authored The Happiness Project, a New York Times Bestseller, about living in the moment and allowing how we face each day shapes our future.  She will be sharing tips and

From the Biogen Idec press release, “Much like my happiness project has allowed me to focus on the positive and bring more joy to my life, the MS Blueprint program is designed to do the same for the MS community,” said Gretchen Rubin. “I believe every small step—including the activities achieved through MS Blueprint—is a victory toward living a more enjoyable life. When you feel every day reflects how you want your life to be, that’s when you’re truly happiest.”

The release goes on to explain how the program will work.  “By regularly visiting the MS Blueprint website, those impacted by MS can continue to set new goals, create new MS Blueprints, and contribute their suggestions for themes and activities they would like to see added to the site. In addition to the personalized activity plans, the website features frequently asked questions about living with MS, and a blog from Gretchen on how to incorporate happiness into your daily routine.”

 

I wonder if she developed the title of happiness expert during her years as a student at Yale Law School. Her bio from Wikipedia (yes, I hate to quote them but it was easily available ) reads: ‘Rubin received her undergraduate and law degrees from Yale University, was editor-in-chief of the Yale Law Journal and won the Edgar M. Cullen Prize. She clerked on the U.S. Supreme Court for Justice Sandra Day O’Connor and served as a chief adviser to Federal Communications Commission Chairman Reed Hundt. She has also been a lecturer at the Yale Law School and the Yale School of Management. She lives in New York City. She is the daughter-in-law of former US Treasury Secretary Robert Rubin. It sounds like she has a lot to be happy about every day and could probably give some tips about finances.

 

I took a few minutes to explore MSBlueprint and found an easy to use site, surprisingly free of any advertisements for their MS drugs, and constructed a sample blueprint list for myself to see what the site it about.  I’m not familiar with Rubin’s writings, but will soon be if I use this for a while, since it appears the helpful prompts and tips will be given in her blog.

 

The other program unveiled was Lights, Camera, Take Action on MS™, announced by Genzyme.  Madeleine Stowe, the television and film actor, is at the center of this new campaign.   I am not much into following the careers of people on the screen, so I didn’t know much about her. She grew up with her father who had MS, and I understand he died at a fairly young age over 20 years ago, in an era before the disease modifying therapies were available.  She can talk from her own personal experience as a caregiver for a loved one who had this disease from that time.

Stowe,  again quoting from Wikipedia – “appeared on stage and television in early career, before her breakthrough role in the 1987 crime-comedy film Stakeout. She later starred in films Revenge (1990) Unlawful Entry(1992), The Last of the Mohicans (1992), Blink (1993), Bad Girls (1994), 12 Monkeys (1995), The General’s Daughter (1999), and We Were Soldiers (2002).

Stowe had left the movie screen in 2003, and in later several years starred only in two made for television films. In 2011, she began starring as Victoria Grayson, the main antagonist of the ABC drama series Revenge. For performance in show she was nominated for a Golden Globe Award for Best Actress – Television Series Drama in 2012.”

 

 

Thanks to a promotional outreach  from a marketing firm to bloggers to talk about this program, I was able to speak by phone with Dr. Ann Bass, the director of The Neurology Center of San Antonio and the lead neurologist for this program. Lights, Camera, Take Action on MS will be comprised of  three live events held on different dates in Chicago, Orlando and Houston.  The Chicago and Orlando events will also be available to view on the web and taped for on demand viewing at their website.  Each of these events will last about two hours, and have a panel comprised of Stowe, Bass, other as yet unspecified medical lifestyle support people, and their patient ambassadors.   I won’t be anywhere near any of these locations to attend the live program and will have to settle for the rerun.

 

The panel members will talk about their drug, of course, because that is required by the FDA laws governing contact with potential users of their drug.  But Bass says the program will focus on more than just the Genzyme products and include mini-seminars that will offer information on ways to gain better health and be more active as people with MS.  She is particularly excited to share the perspective Stowe will add to the caregivers’ side of this disease; this is a special group of people that does so much but continues to be in the background with little support or resources

 

I applaud these efforts to reach more people with MS through research and patient support programs.  The patient programs come under criticism from many because ultimately the companies want to expose us to their particular product in the hopes we will use their therapy, but I find they can also provide valuable support, especially for the newly diagnosed.  No one should criticize the efforts of the Romneys to give their valuable backing with their high profile support to an important project.

 

Like I said, Big Names are being used for these new programs and I hope that helps to generate Money, Motivation and Knowledge, all of which we could use more of.

 

Be well,

Laura

Post Script – An addendum to this post on Friday afternoon 10/17:  You can add Novartis to the mix of promotions using star power for MS campaigns.  Novartis will unveil their program with David Osmond, of the Osmond family, next week.  According to the Wall Street journal – ““I Can Do This,” the new music video from David Osmond of the musical Osmond family, has the clan’s trademark upbeat positive style, with images of men and women overcoming barriers and obstacles to reach a goal.”   Clink on the link to view the video.

I guess we should stay tuned for who is next to bring on a ‘star’ to help us with our attitude toward life and activity.    Four major programs unveiled in one week might be a record for the MS world.

 

 

 

 

 

If you only do one thing

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Do one thing today to make this a better place – 

I wrote this entry for World MS Day in May, but the message is still needed – the United Nations Convention on the Rights of Persons with Disabilities (CRPD) has still not been passed by our Senate.

http://multiplesclerosis.net/news/armchair-activism-world-ms-day/

 It is scheduled to be before the Committee this Tuesday, July 22.

You can read the details in in my blog, but in particular I urge you to notice that Ohio’s Rob Portman is one of the few who are holding up this important ratification. You can reach Senator Portman on twitter @PortmanPress,
FB Senator Rob Portman

Email him using his webform –https://www.portman.senate.gov/public/index.cfm/contact-form
or the old fashion way of sending a letter or making a phone call
The Honorable Rob Portman
United States Senate
448 Russell Senate Office Building
Washington, D.C. 20510-3506
DC Phone: 202-224-3353

There are others on this list who also could use hearing the voice of reason supporting this resolution.  if you need help tracking down their contact information, let me know.  I am happy to help.


I thank you and the world community thanks you.

Laura

 

Experience Shapes What we See

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We have an assortment of wildlife living in our suburban neighborhood, much beyond the normal squirrels, chipmunks and song birds.  We have birds of prey – the red shoulder hawk in particular – that keep watch from above.  Another fox family has been born in the thick trees of our neighbor’s backyard and they wander around day and night.  In case you still wonder what does the fox say, it is an other worldly squawking and calling sound, especially in the middle of the night when we are trying to sleep.

 

Most recently we have an adult doe who has been making the rounds of our back yard- she wanders through, nibbling on the weeds of which there are plenty, and she doesn’t seem particularly bothered by my stepping out on the deck to watch her and snap a photo or two.   I posted this picture on my Facebook page and immediately was given a lesson in how we apply our own experiences and perceptions to a particular situation.

 

What do you think of when you see my pictures?

deer

I am always in awe of these creatures even though I know they are not necessarily benign.   Many people responded to the beauty of this deer peacefully lounging in my backyard.  Who could argue with that view?

 

 

 

 

A friend immediately chimed in that she wished she could see the beauty but all she could picture was ticks.  You see, she had been ill for several years from Lyme Disease, a serious infection caused by the bite of the tick carried by deer and other wildlife.  From her experience, there was nothing beautiful in my photo.

 

My sister had the  flashback of having extensive damage done to her car when one of these beautiful creatures bounded onto the highway in front of her one autumn evening.  Our daughter had that same misfortune and totaled her car with a deer strike, but she saw this backyard deer and was still mesmerized by its presence.

 

Then there are my hunter friends  – they spend hours and more hours in fields and woods waiting to cross paths with a deer this size.  They are also the same people who process the meat and eat off their kill for many months.  Their pulse quickened a bit when they saw the picture of this deer just hanging around in my yard.

 

 

deer 2 What does this have to do with anything other than I have some interesting  photos of wildlife in my otherwise quiet neighborhood?

 

 

I offer this as an example of how people can take the same thing – in this case the deer– and come away with very different interpretations of what they see.  I see this happen quite often when people with MS read the same news or research and come away with different ideas.  Take CCSVI or the latest diets or hyperbaric oxygen treatment or any of the other unproven MS treatments and you’ll see this in full action.  There is such a strong desire, no make that a strong need, to find a cure for MS, that it is easy to interpret  the headlines into something other than what it says based on our own biases and preconceived ideas and needs.

 

Each cure we read about means we also must stop and wonder how are we reading the news – is it because it says what we  want  depending on our perspective and experiences, or is it based in fact?  I am a Pollyanna by nature and want to think one of these ‘cures’ might really work,  but the realist in me recognizes the science doesn’t hold up to the proof process.    That doesn’t mean I can’t still stop and wonder if the next ‘cure’ might hold up, and want to learn more about it  – but have to keep in mind my personal perspective helps to complete the story.

 

I will also look at this deer the next time and not only see beauty, but wonder about the ticks and look closer for any bow and arrow yielding hunters closing in.

be well,

Laura

 

 

 

 

 

 

 

The Advertising Pitch

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We had a good laugh as my husband sorted through the coupons that arrived in our mail – it was an envelope full of special offers and near the top of the stack was a coupon for breast augmentation – only $3,850, including anesthesia.  Boy I’m glad to know at least they knock you out while you have this done.  A few more coupons down in the stack, just after the one for the $39 hour massage to celebrate a new studio opening, was an invitation to come to a local funeral home to do our pre-planning.

 

What happened to the simpler times when these coupons involved buy one get one free pizzas and special deals on moonlight bowling? The face of marketing has changed, complex services are being peddled as common merchandise; even the pharmaceutical companies are doing all they can to get their product or service on our radar.

Websites that talk about Multiple Sclerosis are often populated with ads – you know the ones that are promoting the latest treatments or product that might make our lives better.  The cost of maintaining a website, complete with a staff to keep the content fresh, isn’t cheap, and selling ad space to pharmaceutical companies is one way to bring in operating revenue.

 

Virtually all of these ads are specifically targeted to me and what they are guessing might be my interests.  Tracking of what I look at online is captured through cookies and pretty soon,  ads that mimic my interests pop up on my screen.  I was shopping for some special style of orthopedic shoe and for the next month,  my computer screen was visited by shoe ads by a variety of different manufacturers.  Of course I am regularly looking at multiple sclerosis information on line and those visits trigger ads for MS treatments to populate my viewing screen.  Often those are from reputable companies, but I also get ads for special diets and unproven surgical procedures performed in exotic locations like Costa Rica and Bulgaria.

 

When doing research for writing, the  search terms  I might use often lead to an interesting  assortment of pop up ads – try it yourself and see what you get if you google for MS and sexual dysfunction.   Or MS and incontinence.  Or MS and marijuana.   I’m sure you get the gist of my point, when I search the right words,  I get lots of Viagra, adult toys, catheter supplies, and bong  advertisements thrown my way.

 

I’m resigned to those ads and how most websites have to do business with advertisers to cover their expenses and make a profit. Most of them I could simply ignore, but I still often have trouble with some of those ads, mainly because their fancy colors and animation often interfere with my vision, focus and also the speed of my computer.  I could turn off the cookies in my computer, but many sites these days require the cookies to be set before you can even enter their website.

 

I found a great ad blocker software that is free – they ask for donations to support their work and I gladly give them a small amount of money to support their product – and it keeps most of the pop-up ads away from my computer.  Since I installed this software on my new laptop in February, the counter tells me I have been spared seeing   over 100,000 download ads.

 

I say most, because even AdBlockPlus lets some ads through – they call these ‘acceptable ads.’  It is an acknowledgement that advertising revenue is necessary to sustain most of these sites.  There is a long list of criteria that makes an ad acceptable, and this includes no animations, nothing with sound and preferably text only.

 

Social media sites such as Facebook and Twitter also generate income for their sites by selling prime spots on their pages to advertisers. Unfortunately, the ad-block software doesn’t stop those messages and in between messages from friends and sharing jokes and pictures of children and grandchildren, those ads slip into our feeds.

 

Advertisers have their fleeting chance to get us interested in their products and it takes a lot these days to get our attention since we are bombarded at what seems like every opportunity.  Whether it’s a mailing to my home or ads on my computer, most of them end up deleted or in the trash – if I am shopping for MS drugs or breast augmentations, I want to do my own searching.

 

Be well,

Laura