Evolution of multiple sclerosis treatments: an interview with Mike Panzara, Head of MS & Neurology Clinical Development, Genzyme

insights from industryDr. Michael PANZARAGroup VP and Therapeutic Area Head, Multiple Sclerosis
​and Neurology, Genzyme

Please can you give a brief history of multiple sclerosis (MS) treatments? 

The last 20 years have been defined by progress and innovation in the multiple sclerosis (MS) field, with now 10 approved treatment options available for people living with relapsing MS, the most common form of the disease.

Though none of these treatments are a cure, they do offer people living with MS and the physicians who treat them a wide range of different therapy options, each that carries its own unique benefits and risks in terms of efficacy, safety and convenience. 

Finally having a variety of treatment options to choose from has changed the lives of many of those with this disease but has added complexity, making it that much more important for people living with MS and their physicians to take the time to discuss the benefits and risks associated with each treatment. 

The selection of treatment is highly individualized and is ultimately based upon the combination of expected benefits, risks, tolerability, and convenience that offer the best chance for a favorable outcome and an improved quality of life.

Despite periods in which people afflicted by MS may appear free of physical signs of the disease, it is progressive, and research tells us that the disease is silently worsening, even from its earliest stages, in most cases leading to a more overtly progressive disease course resistant to treatment.  Therefore, it has become increasingly common to start treatment as early as possible following diagnosis in the hopes of slowing the accumulation of permanent disability. 

When I first began working in the MS area nearly 20 years ago, these kind of treatment options just did not exist, so it’s exciting to see how this landscape has evolved with continued innovation. 

Genzyme has one treatment on the market in the U.S. (AUBAGIO), with another under FDA review (LEMTRADA), and is building a pipeline not just focused on treating relapsing MS, but also progressive forms of the disease where the unmet need for new treatments is greatest.   

How has our understanding of the causes of MS changed over this time and what impact has this had on MS management?

Despite significant progress in our understanding of the causes of MS, there is still much that is unknown.  MS is an autoimmune disease where the body’s immune system attacks cells in the brain and spinal cord.  These cells are unable to function properly and eventually die, leading to the clinical signs and symptoms people with MS experience.

It is clear that both genetic and environmental factors play important roles in the onset and progression of the disease.  An understanding of the details of this process has grown over the past many years, and these insights have led to many of the treatments for relapsing MS that we have today.

However, while we have many options available to treat relapsing MS, lack of understanding of the mechanisms of disease progression has been a barrier to the development of treatments for progressive forms of the disease. 

This year at AAN, Genzyme announced the formation of a research collaboration with the Cleveland Clinic specifically focused on deepening understanding of the pathogenesis and progression of MS, and to address needs for additional research and the development of treatments in this area.

As clinicians and researchers, we know we have a lot of work yet to be done to ensure that all people living with this devastating disease receive treatments that make their lives better.  We are committed to doing what we can to make this possible.

How successful are current treatments for MS?

Current treatments for MS offer patients many different options for how they want to treat their disease.  Each of these treatments has its own unique set of benefits and risks, and physicians play a critical role in educating patients about these considerations and discussing a personalized treatment approach that will help them determine which therapy option is right for them. Connecting the right patients with the right therapy is essential to successful treatment. 

For a long time, MS therapies were all self-injectables, which some patients find frightening and difficult, especially when you consider that MS is a chronic disease that often requires a lifetime of treatment.

Some of the more recently approved treatments are taken orally, which many patients consider to be an important benefit that better fits their lifestyle.  For example, Genzyme’s once-daily oral therapy AUBAGIO is one of these therapies and offers efficacy and manageable risks with the convenience of once-daily dosing without the hassle of self-injections.

For most people, MS continues to worsen over the course of their lifetime.  MS may also become particularly active at times, requiring intervention with treatments that may have higher risks, but offer strong efficacy that can delay, and even prevent, disease progression. 

This is why there is a lot of excitement around LEMTRADA, which is currently approved in over 30 countries worldwide and under review by the FDA in the U.S.

In clinical trials, LEMTRADA has demonstrated efficacy greater than one of the injectable therapies, reducing the risk of disease progression, with many also experiencing an improvement in disability scores.

While LEMTRADA comes with some potentially serious risks, these have been successfully managed in the clinical trials with a robust risk management program similar to that in place in the countries where it is currently approved.

What are the main drawbacks of current treatments?

None of our current treatments offer a cure for MS and many have significant side effects or are poorly tolerated. 

Further, they are only effective for treating relapsing forms of the disease, so there is a high unmet need for therapies that effectively fight progressive forms of MS as well.

What were the main takeaways from the recent American Academy of Neurology (AAN) Meeting with regards to MS research?

The AAN has increasingly become a hub for some of the most innovative, exciting research being done in MS and across the entire neurology landscape.  From a Genzyme perspective, this meeting was especially exciting because of the breadth of data presented from our ongoing work with both AUBAGIO and LEMTRADA.

For AUBAGIO, we presented data from the Phase III TOPIC trial, which was designed to assess whether early initiation of AUBAGIO in patients who experienced their first neurological symptoms consistent with Clinically Isolated Syndrome (CIS) can prevent or delay a second clinical attack (i.e., conversion to clinically definite multiple sclerosis (CDMS)). 

In this trial, patients receiving AUBAGIO 14 mg and 7 mg were significantly less likely to develop a second clinical attack compared to patients on placebo with a similar safety profile to that seen in other stages of MS. The results reinforce AUBAGIO’s consistent efficacy and safety across a spectrum of MS patients.

For LEMTRADA, new magnetic resonance imaging (MRI) data from the two Phase III CARE-MS studies (both treatment-naïve patients and patients who had active disease on another therapy) demonstrated that slowing of brain atrophy and reductions in new MS lesions were sustained at three years in patients treated with LEMTRADA, suggesting durable effects beyond the two year treatment period in clinical trials.

This was supported by the observation that approximately 80 percent of patients treated with LEMTRADA did not receive a third course of treatment in the first year of the extension trial.

In our Phase III studies, patients on LEMTRADA received two courses of treatment, the first administered via intravenous infusion on five consecutive days, and the second administered on three consecutive days, 12 months later – in other words, the last course of treatment with Lemtrada for 80% of patients in the first year of the extension study was two years prior, yet efficacy was maintained.

What do you think the future holds for MS treatments?

Obviously, no one can predict the future, but given the rapid expansion of efforts to understand and fight MS, I am optimistic that we will get to a cure for this disease. 

As we work to get there, it is important that we continue to optimize treatment with the therapies we do have to maximize outcomes, while continuing to research and develop new therapies with improved efficacy, tolerability and safety profiles for relapsing MS, and therapies that target disease progression, even leading to repair of the brain and spinal cord. 

What are Genzyme’s plans for the future?

At Genzyme, our priority continues to be becoming leaders in MS and building a long-term partnership with the MS Community.  We are a company with a strong legacy of focusing on the patients we serve, and we are passionate about addressing unmet needs for people living with MS.

Looking ahead to the future, Genzyme plans to continue to innovate in MS treatment beyond AUBAGIO and LEMTRADA. The company is committed to developing the next generation of therapeutics for relapsing and progressive MS.  

If all people with MS are to benefit from the ongoing progress in this field, development of therapies that protect the brain and spinal cord from damage and repair the damage that has already been done is critical.  This will be the next great innovation in this area.

Where can readers find more information?

Genzyme offers a great educational resource called MS Atrium (MSAtrium.com), which was developed with a leading panel of independent MS experts, and offers disease information, as well as perspectives on emerging treatment trends and other MS topics of interest.

People can also visit Genzyme.com and MSOnetoOne.com to learn more about our MS Franchise and long-term commitment to innovation in MS.

Several advocacy groups, such as the National Multiple Sclerosis Society (NMSS) and the Multiple Sclerosis Association of America (MSAA), also offer great information on their websites for people living with MS, as well as ways for people to find support and connect with others in the MS Community.

About Michael Panzara

Michael Panzara, M.D., M.P.H., joined Genzyme in 2009 and is the Group VP and Therapeutic Area Head, MS and Neurology, responsible for the clinical development strategy within the therapeutic area, including for LEMTRADA. 

Dr. Panzara was previously Chief Medical Officer for Neurology at Biogen Idec, Inc., where he led the development of late-stage neurology products, including PLEGRIDY ™, Tysabri®, and Tecfidera®.

Dr. Panzara is a board certified in neurology who received his M.D. from the Stanford University School of Medicine and trained in neurology at Massachusetts General Hospital.  He completed his master’s degree in public health at the Harvard University School of Public Health.

April Cashin-Garbutt

Written by

April Cashin-Garbutt

April graduated with a first-class honours degree in Natural Sciences from Pembroke College, University of Cambridge. During her time as Editor-in-Chief, News-Medical (2012-2017), she kickstarted the content production process and helped to grow the website readership to over 60 million visitors per year. Through interviewing global thought leaders in medicine and life sciences, including Nobel laureates, April developed a passion for neuroscience and now works at the Sainsbury Wellcome Centre for Neural Circuits and Behaviour, located within UCL.


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