Dr. Harbo:

• MMN is treatable, unlike ALS
• MMN is “an ultra rare disorder” affecting 1 out of 100,000 people.
  • More common for men
• The onset of symptoms begin between the ages of 35 to 45, but can start at 20 years old.
• Purely motor with some sensory and cognitive impairments
• MMN is often Misdiagnosed with common neurological disorders like ALS

Pam

• The average time to diagnosis is 6 years and it took Pam 7 years to receive a diagnosis.
• Started with a twitchy finger and eventually she could not curl that finger. She also noticed her typing was impacted.
• Started limping while she was pregnant at 40
• Tried osteoplasty and acupuncture, but it did not help
• A podiatrist eventually noticed that one of her legs was very thin compared to the other leg. This made the podiatrist recommend a neurological consult.
• Saw 3 neurologists before receiving a proper diagnosis
  • The third neurologist dismissed her self-diagnosis of MMN, because she did not have the anti-GM1 antibody.
  • So, she kept pushing her neurologist to receive IVIg. When she tried IVIg, she first got really sick, and then started to see results.

Chrissie

• A major challenge can be “coming to terms” or the initial and ongoing adjustment to accepting one’s diagnosis and prognosis
• This happens differently for every patient at their new normal
• Anxiety and Depression are very common
• Patients say: “it feels like I’ve been hijacked and being held hostage”

What advice would you give a patient, who is trying to balance treatment, a career, familial responsibilities, etc?

• Be gentle with yourself.
• Everyday will look different with your capacity to do things.
• Be mindful when you feel like you are struggling.
• Get counseling support.
• Talk to patient volunteers at the Foundation

It is ok to be stoic and still go through the 5 stages of grief (denial, anger, bargaining, depression, and acceptance).

Therapy can make a lasting difference!

Dr. Harbo

• Typical treatment for MMN is immunoglobulin (Ig), given intravenously or subcutaneously.
• Plasma Exchange and steroids are not beneficial for MMN.
• Some patients feel a decline in their baseline, even when they use IVIg.
• There are ongoing trials for MMN and clinical trials in the US, Europe, Denmark, and most of the world.
• Treatment options for MMN are still currently limited.

Pam

• Started with IVIg, and then tried SCIg, but did not see the same results, so she went back to IVIg.
• Hydration, and specifically Hydrolyte helped reduce side effects of Ig
• Her first few infusions made her nauseous and gave her headaches, so her neurologist reduce the rate of infusion.
• In Ontario, the standard rate is 200ml per hour, but her rate was reduced to 150ml per hour and now she is at 180 ml per hour
  • The different between 200ml to 180ml makes a big difference for Pam and does not experience headaches at 180ml.
• As a personal preference for her schedule and lifestyle, Pam enjoys her two-day infusion treatment every 3 weeks. She explains that, for her, it is very calming and similar to a spa treatment.

Companies are targeting Anti-complement treatments.

• Complements are proteins in the blood, part of the immune system, that clean bacteria.
  • In MMN, they damage the body’s nerves, so blocking them will theoretically reduce damage to nerve cells.
• Researchers are looking into less invasive administrations of IVIg.

What are the side effects of complement Inhibitors?

• There is a concern that patients could get a bacterial infection.
• Trials typically require vaccination against bacteria that could put patients at risk.

• Some compounds only block antibodies that hurt nerves, so bad complements may still be active in the body.

Dr. Harbo

• Currently, we have not seen any studies testing Rituximab’s effectiveness for MMN patients
• There is a percentage of MMN patients who do not respond well to IVIg.
  • In this case, some neurologists may consider Rituximab.
  • Note: Rituximab is not a magical treatment of CIDP either.
  • Rituximab is not toxic as other immunosuppressant drugs.
• Cyclophosphamide has also been tried, but is very toxic because it is an old fashioned chemotherapy.

Chrissie

• Trust and good support is important.
• If you don’t feel comfortable, seen, or heard, you need to change the people in your team.
• Advocate for yourself.
• Care givers are unsung heroes, and need to make sure that they are also taking care of themselves as well.
• Open, honest, and vulnerable communication is needed for caregivers to succeed.
• Do not give up on trying to find the right place for care.
• Finding the right members of your care team is important.
• Nurses are the core of the team; they often know patients more than their own family members do.
• Caregiving is hard because the responsibilities of daily life do not go away.

Dr. Harbo

• Mentioned he is not optimistic for a simple biomarker for MMN.
• We have some helpful tests for diagnosis, including ganglioside antibody testing and electro-physiological exams.
• We are dependent on patient reports, clinical examination, objective measures of grip strength and muscle strength, but these measures are not always effective.
  • Neurofilament light chain as a biomarker is only effective when the disease is very active.
• There is work on testing through peripheral nerves, but it is not an effective biomarker.

Dr. Harbo

• Every patient is different because every patient has different nerves.
• Manual Muscle test are good indicators.
  • Handwriting, cramping when walking, or drop foot after walking 10 meters versus 50 meters (30 feet versus 150 feet)

Pam

• The reality is that no one knows what is going to happen. This can be a dark cloud, making days different and sometimes really hard.
• Diagnosis was a relief for Pam and she kept repeating: “I am lucky, I should do what I can”.
• Found satisfaction in volunteering and participating in support groups.
• Some days, the mental state will impact the physical state.
• If you notice a drop off after infusion, record it and tell your physician.

Chrissie

• The only guarantee in life is that everything changes.
• You will become stronger with a new sense of meaning and purpose.
• Endurance and perseverance will be a gift you can give to other people.
• Be comfortable with the unknown.
  • Our human nature is to know and understand everything.
• This disease is unpredictable, so every morning is an opportunity to try again.

Dr. Harbo

• Accept that this is a chronic disorder.
• Look at the possibilities and not its limitations.
• Find a team in the hospital that you trust.
  • They are important when receiving treatment, correct information, and understanding your options.
• This period is about what feels best for you.
• If you go through a period where you are getting weaker, the medical team is extremely important to adjusting your treatment.
• Exercise is important.

Dr. Harbo

• In Denmark, both are seen as equal.
  • Hizentra is only immunoglobulin.
  • Hyqvia is a mix of hyaluronidase and immunoglobulin.
    • You can receive higher doses at faster rates.
• Every patient is different so if one does not work, try the other.