Multiple sclerosis (MS) is a chronic inflammatory and demyelinating disease that affects the central nervous system. It is the most common disabling neurological disease in young adults worldwide. Though the underlying cause of MS is largely unknown, it is considered an autoimmune disease where the body’s own immune system attacks the protective myelin sheath surrounding the nerve fibers and causes demyelination. This damage to the myelin sheaths disrupts the transmission of signals between the brain, spinal cord and the rest of the body. There are different subtypes of MS including relapsing-remitting MS, primary progressive MS, and secondary progressive MS which are classified mainly based on their clinical courses. The exact symptoms vary greatly among patients depending on the location of damage but common symptoms include fatigue, numbness, impaired coordination and balance, vision problems, bladder issues, pain and cognitive impairment.
Current Treatments for Multiple Sclerosis Therapeutics
Currently there is no Multiple Sclerosis Therapeutics cure for MS and treatment focuses on managing its symptoms, preventing relapses, and delaying disease progression. The FDA has approved over a dozen disease-modifying therapies (DMTs) that act as immunomodulators or immunosuppressants to reduce immune system attacks on myelin. The first-line treatments for relapsing forms of MS include interferon beta preparations such as Rebif, Avonex and Betaseron or glatiramer acetate like Copaxone. These injectable medications work to modify the disease course but do not stop disease activity completely. For patients with highly active relapsing MS or worsening progressive forms, stronger medications like Tecfidera (dimethyl fumarate), Aubagio (teriflunomide), Gilenya (fingolimod), Lemtrada (alemtuzumab), Ocrevus (ocrelizumab) or MAVENCLAD (cladribine) may be prescribed. Steroids are commonly used for MS relapse management. In addition, many safe and effective symptomatic therapies are available to specifically target symptoms like pain, mobility issues, spasticity, fatigue and cognitive dysfunction.
Limitations of Current Treatments
While current FDA-approved disease-modifying therapies have significantly improved the treatment landscape for MS over the past few decades, they still have some important limitations. Foremost, none of them are able to completely stop MS progression or cure the disease. Their mechanisms of action involve modifying the immune system in broad ways which can lead to side effects. The injectable therapies require lifelong adherence to daily, weekly or monthly dosing schedules which impacts quality of life. Their treatment effects also decrease over time in many patients. The oral and more potent therapies have more stringent eligibility criteria and safety monitoring requirements due to risks of adverse effects like infections. Lastly, the high treatment costs pose a significant financial burden for MS patients and healthcare systems. Therefore, there remains a major unmet need to develop newer MS therapies with more targeted mechanisms that provide cure or at least have a more permanent treatment impact with fewer side effects and more convenient dosing regimens.
Promising Future Treatment Directions
Scientists around the world are actively pursuing multiple novel therapeutic approaches for MS that aim to overcome the limitations of existing treatments. Some of the most promising future treatment directions currently in clinical trials include:
Stem cell therapies – Autologous hematopoietic stem cell transplantation (AHSCT) is being studied which involves reinfusion of a patient’s own blood-forming stem cells after high-dose chemotherapy. This helps reboot the immune system in a way that can stop MS activity. There have been very promising results so far.
Neuroprotective agents – Drugs that can directly protect neurons or promote repair of damaged myelin are being investigated. Some examples are anti-Nogo antibodies, remyelinating agents like opicinumab, and mitochondria-targeting agents.
Gene therapies – Using gene therapy techniques to deliver protective genes directly toareas of active MS. For example, delivering neurotrophic factors or immunomodulatory molecules. Some therapies involve genetically modifying a patient’s own stem cells before transplantation.
T-cell therapies – Harnessing the power of chimeric antigen receptor T-cell (CAR-T) therapies developed for cancer but modifying them to selectively deplete pathogenic T-cells linked to MS.
Oral monotherapies – Novel oral drugs that can achieve similar efficacy to injectables but with better safety, tolerability and adherence profiles. Drugs targeting sphingosine-1-phosphate, B-lymphocytes and other pathways are being evaluated.
Combination therapies – Strategically combining 2 or more existing or new mechanisms of action to maximize treatment impact in a synergistic manner. For example, combining immunomodulation with neuroprotection.
If ongoing and planned clinical studies continue yielding promising safety and efficacy data for some of these novel approaches, they have the potential to revolutionize MS treatment in the coming years by moving the field closer to cure or disease modification goals. Scientists remain optimistic that advanced mechanisms will ultimately overcome the immune challenges in MS.
The expanding armamentarium of FDA-approved disease-modifying therapies over recent decades has greatly improved life quality for millions suffering from MS worldwide. However, more work still needs to be done to address the key limitations of current treatments. Exciting new therapeutics harnessing stem cells, gene therapies, T-cell depletion and other pathways in development hold immense promise to completely transform MS management. With continued research momentum and collaborative efforts, the future indeed looks brighter for people impacted by this difficult disease.
*Note:
1.Source: Coherent Market Insights, Public sources, Desk research
2.We have leveraged AI tools to mine information and compile it
About Author - Priya Pandey
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