Optimizing Multiple Sclerosis Treatment with Stratified and Precision Medicine

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• Stratified Medicine Explained
• Importance in Multiple Sclerosis
• Clinical Subgroups and Prognosis
• Informing Treatment Decisions
• Personalized Precision Medicine
• Monitoring Disease Activity
• Full Transcript

Stratified Medicine Explained

Dr. Paul Matthews, MD, defines stratified medicine as a clinical approach that recognizes patient heterogeneity. He explains that not all patients with a common diagnosis are the same. Instead, distinct subgroups exist within a larger diagnostic category. These subgroups share similar disease characteristics and clinical behaviors.

This concept is crucial for moving beyond a one-size-fits-all treatment model. It allows clinicians to categorize patients more accurately. This leads to better predictions of disease course and treatment response.

Importance in Multiple Sclerosis

The application of stratified medicine is particularly vital in multiple sclerosis. Dr. Paul Matthews, MD, clarifies that MS is a syndrome, not a single disease with a precise cause. This inherent variability means patients experience vastly different disease trajectories. Their responses to available medications can also differ significantly.

Dr. Anton Titov, MD, discusses this with Dr. Matthews, highlighting its role in improving clinical care. Understanding these differences is the first step toward optimizing therapy. It helps balance the benefits of treatment against its potential risks for each patient subgroup.

Clinical Subgroups and Prognosis

Dr. Paul Matthews, MD, identifies three broad prognostic subgroups in multiple sclerosis. A small group of patients may show very slow disease progression even without treatment. Another small group experiences a highly aggressive, or malignant, disease course.

The largest group of patients falls into an intermediate category. Assigning a new diagnosis to one of these subgroups is a critical early step. This stratification provides invaluable information about a patient's likely future prognosis. It forms the foundation for all subsequent treatment discussions and decisions.

Informing Treatment Decisions

Stratified medicine directly informs initial treatment choices for multiple sclerosis. Dr. Paul Matthews, MD, explains how subgroup assignment guides therapy selection. For patients in a more benign subgroup, the decision might be to monitor without immediate treatment. For those with a higher risk of progression, starting treatment is more urgent.

This approach also influences the choice between first-line medications and higher-efficacy drugs. Medications with greater potency often carry a higher risk of side effects. Stratification helps ensure that the potential benefits of a stronger therapy justify its risks for that specific patient group.

Personalized Precision Medicine

Dr. Paul Matthews, MD, describes personalized medicine as the next evolution after stratification. While stratified medicine defines groups, precision medicine focuses on the individual patient. It aims to understand how one specific person is responding to their current treatment protocol.

The goal is to identify the most appropriate medication for that individual. Dr. Anton Titov, MD, and Dr. Matthews discuss this tailored approach. It represents the highest standard of care, moving from group-based predictions to truly individualized therapy.

Monitoring Disease Activity with Tools

Implementing precision medicine requires careful monitoring of multiple sclerosis disease activity. Dr. Paul Matthews, MD, outlines the key tools used in clinical practice. Physicians track clinical measures like relapse rates and disability progression over time.

Neuroimaging is a cornerstone of monitoring. MRI scans are used to measure disease activity, such as counting new T2 hyperintense lesions. MRI is also critical for assessing brain volume change, a marker of neurodegeneration. This ongoing assessment allows clinicians to adjust treatment based on real-world evidence of its effectiveness for that individual.

Full Transcript

Dr. Anton Titov, MD: Neurological disease treatment is very costly and often is not as efficient as it can be. You have advanced the concept of "Stratified Medicine" to improve quality of clinical care for patients with neurological disease. What is the Stratified Medicine concept? How does it help in clinical treatment of neurological diseases, including multiple sclerosis?

Dr. Paul Matthews, MD: The concept of stratified medicine is that all patients are not similar. Some groups of patients who carry a common diagnosis may behave in a more similar fashion to each other than they do to the group as a whole. There are distinct subgroups of patients with similar disease characteristics within a larger umbrella diagnostic group.

In the context of multiple sclerosis, it is a particularly important idea because multiple sclerosis itself is a syndrome. It is not a specific diagnosis established on the basis of a very precisely recognized common disease-causative factor.

The importance of stratified medicine in disease in general is this: patients with different forms of expression of a syndrome or disease may behave differently with respect to treatments. Patients may also have different prognosis and results of treatment.

Being able to express the difference in results of treatment is important. Patients must balance the benefits and risks of treatment or no treatment. Every patient belongs to such distinct subgroup with similar characteristics of clinical course and prognosis.

Several subgroups form a larger group within a given disease entity. Understanding the subgroup an individual belongs to is important in guiding therapy for other reasons too.

Here is a specific example of Stratified Medicine in the context of multiple sclerosis. We know that there exists a small group of patients with multiple sclerosis who may not show very rapid progression at all, even if we do not treat them.

Then there is another group of patients with multiple sclerosis, also relatively small, who will show very malignant progression. Finally, there are many patients with multiple sclerosis who belong to a middle group.

After initial diagnosis of multiple sclerosis, we can assign patients to appropriate subgroup. This helps to inform clinical decisions regarding initial treatment of multiple sclerosis. This helps patients to make the decision whether to start treatment or not immediately.

Dr. Anton Titov, MD: Assignment to distinct clinical group helps to make the choice of first treatment.

Dr. Paul Matthews, MD: We can use a traditional first-line medication for multiple sclerosis, or we can use medications with a higher efficacy but also with a higher risk profile. All can be informed by that initial decision to assign a patient to a specific clinical group.

This is the Stratified Medicine concept. Personalized Medicine is taking Stratified Medicine one step further. Stratified Medicine tells us about the following classification of patients: “This class of patients with a given diagnosis are more similar. We can have these general expectations for behavior of disease in that group of patients”.

Personalized Medicine is about trying to individualize this understanding of disease course and prognosis more precisely. Precision medicine helps us to understand how a single given patient is responding or not responding to medication or to a treatment protocol.

Dr. Anton Titov, MD: Precision medicine helps us to identify medication that would be most appropriate for that person individually.

Dr. Paul Matthews, MD: Here is the best way we can use today a Stratified Medicine and Precision Medicine for patients with multiple sclerosis. We can carefully observe patients on-treatment or off-treatment.

We can follow patients serially with clinical measures of relapse rate and disability progression of multiple sclerosis. We can use MRI imaging to measure multiple sclerosis activity. We can follow, for example, T2 hyperintense lesion count. We can identify brain volume change on MRI.

We can assess patients who have evidence for no multiple sclerosis disease progression. We can identify patients who have evidence for modest disease activity in multiple sclerosis. We can also find those patients who have evidence for higher multiple sclerosis disease activity.

That is how we can change whatever our initial treatment structure is. We can adjust and tailor treatment protocols to individual patients. Treatment becomes most appropriate for that individual patient with multiple sclerosis.

Dr. Anton Titov, MD: That is stratified medicine. It is identifying groups of patients with similar disease characteristics and prognosis. Personalized medicine is trying to individualize treatment for a single person with multiple sclerosis.