Top 10 for 2021: #2 | Novel Drug for Primary Progressive Multiple Sclerosis

Top 10 for 2021: #2 | Novel Drug for Primary Progressive Multiple Sclerosis

It can start with blurry vision, tingling in the arms or legs, or a persistent feeling of tiredness. In individuals with multiple sclerosis, or MS, the immune system attacks the fatty protective myelin sheath that covers their nerve fibers, disrupting communication between the brain and other parts of the body. This leads to a decline in function and increased disability.

Multiple sclerosis is not a single disease. The majority of patients – around 85% – suffer from relapsing-remitting MS. Which means they may experience disappearing and reappearing symptoms. In contrast, patients suffering from a less common primary progressive multiple sclerosis have a continuously worsening disease from the time of diagnosis.

While research and drug development have created new treatments for relapsing-remitting multiple sclerosis, there hasn't been a drug that showed benefit for primary progressive MS – until now. A novel therapeutic monoclonal antibody approved in March 2017 targets a type of immune cell called CD20-positive B cells. It reduces the immune response, stopping the cells from attacking and damaging myelin. Given once every six months by an intravenous or IV infusion, the treatment is the first and only MS treatment to target these specialized immune cells, and treat the primary progressive population.

Positive results from a phase three study of the drug showed significantly slower disability progression over a median treatment duration of three years when compared to placebo. Patients were 24% less likely to have disability progression for three months. This medical innovation brings new hope to patients in an effort to slow the disabling effects of primary progressive multiple sclerosis.

Akhil Saklecha, MD:
Hello, this is Akhil Saklecha from Cleveland Clinic Ventures. We're now at #2 on our Top 10 Medical Innovations list, ‘Novel Drug for Primary Progressive Multiple Sclerosis.’ With me today is Dr. Adrienne Boissy. She's the Chief Experience Officer at Cleveland Clinic and also a Staff Neurologist at Cleveland Clinic’s Mellen Center for Multiple Sclerosis. Dr. Boissy, tell us a little bit more about this innovation.

Adrienne Boissy, MD:
I think I'd start with the patients who have this devastating diagnosis of primary progressive MS. If you think about a lot of them, MS patients look like all of us on this call. Relatively young people, highly motivated, may have a very full career who now are facing a diagnosis that they don't have a lot of control over. Usually starts with frequent falls and difficulty walking, or problems with their gait. To be told that there's nothing we can do is very difficult for these patients and their families. As a clinician, it is one of the toughest conversations we have.
So this is a really pivotal moment in MS history that, for the first time, we can say we have treatments for all forms of MS. This monoclonal antibody offers hope for these patients who have primary progressive MS – the most difficult to find a medication to treat historically. So we're really excited about it.

Akhil Saklecha, MD:
It sounds fantastic. From the video, we learned that these patients with primary progressive MS really didn't have anything prior to this innovation. So how were you treating these patients before this? And how does this fit into the context of your treatment regimen for MS in general?

Adrienne Boissy, MD:
Yeah. Well, if you think about it, most of the time, when we don't have a treatment targeted directly at the disease – instead, we're managing symptoms. We're starting a new medication to help with your fatigue, or we're sending you to physical therapy to get balance training or gait strengthening. So it's symptomatic benefit. It's not targeted therapy.

I think that's really where this has been so exciting for people – it’s a specific treatment meant to impact the disease itself. Both the MRI appearance in this disease, decreasing T2 new lesions, as well as functional measures like walking and being able to maintain the life that they want to live are improved here.

Akhil Saklecha, MD:
You mentioned some of the benefits that you're seeing in this therapy and, to me, I imagine compliance. I mean, just simply giving an IV every six months. It's really a game-changer for both the patient’s upkeep and the physician’s ability to make sure that we're getting better treatment into the patient. Is that the case – is that what you're seeing? Are the benefits worth the risk?

Adrienne Boissy, MD:
I think I would like to remind everybody who doesn't know that much about MS that the vast majority of patients in relapsing-remitting forms wind up giving themselves injections at times every day and other times every week as a first-line therapy. It is incredibly difficult, even for the most highly motivated and engaged patients. Think about how exciting it is to offer a patient injections twice a year as a means of managing their disease.  So as we think about opportunities not just to minimize side effects, but also to enhance the likelihood that people will stick with this treatment, reducing the frequency of it and administrations absolutely a game-changer. A huge win for MS patients and making their lives easier so that they can get the treatment they need.

Akhil Saklecha, MD:
Do side effects play a role here? Are we concerned about that? Or is the game-changing option of this just worth everything to go forward?

Adrienne Boissy, MD:
Yeah. Again, I would normalize this to if you think about relapsing-remitting MS, we're typically giving injections. Although, there are a lot of new infusion and oral therapies in those disease types, it's often a local injection reaction, or swelling, and redness, and pain, and irritation, and itching. To have somebody go and do that same thing in a different part of their body, the next day is very difficult. So the side effects associated with this infusion, though can be serious, are pretty minimal.

Reactions with the infusion itself can be fatigue or headache, itchiness, shortness of breath. At the time you get the infusion as well as infection. It's an immune-suppressing medicine. So activating viruses like hepatitis B or other opportunistic infections are the major side effects that we see in patients. But overall, I would say the general experience of patients getting the drug is pretty positive. The fact that even if they do have side effects, that they only have to experience that hopefully once or twice a year is a pretty big win.

Akhil Saklecha, MD:
Perfect. So finally, how is this impacting patient lives? I mean, I imagine you've had real patients that you've seen this significantly improved their life. Is there something you can share along that line?

Adrienne Boissy, MD:
Yeah. I have a patient that flies from several states away. She's a mom considering having children. She has a full-time career and a husband. I have been having the primary progressive MS conversation with her for a number of years. Given what I just told you about imagining what it feels like to sit in that chair and imagining what it feels like to have that conversation, this offers something pretty magical.

It goes well beyond the physiological benefits – it gives hope. Hope to the patient that they're not going to be on that horrible graph just getting worse and worse and worse. Hope that we could flatten that curve, that she could have a full life, and think about continuing to work and continuing to expand her family. Those are game-changing conversations. And for us as clinicians, being able to offer the hope when we can't cure, is one of the greatest things we do in our entire careers. I can't tell you a single beautiful story. I would just offer you that on the landscape, this has been a pivotal moment in MS history.

Akhil Saklecha, MD:
Thank you, Dr. Boissy, for taking the time today to discuss this ‘Novel Drug for Primary Progressive Multiple Sclerosis,’ which is #2 on our Top 10 Medical Innovations list.

Adrienne Boissy, MD:
Thank you.
 

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