Are We There Yet?
A cure for multiple sclerosis. That is what we all want, correct? While we have not reached the cure yet, we are getting closer. When we think about a cure for multiple sclerosis, that term may mean different things to different people. To those who are newly diagnosed, a cure might mean a completely effective therapy that would completely eliminate any risk of relapses, new lesions on MRI, or progression of disability. For those who have been dealing with multiple sclerosis for a while and may have accumulated some disability, the cure would mean stopping any further progression and erasing any disability. Admittedly, we have come a long way since 1992 when there were no FDA approved treatment options for multiple sclerosis. So where are we right now with current research in multiple sclerosis? Let’s take a look at our paradigms for using existing medications, research on improving current treatments, and finally, research looking at neural repair.
Currently, available disease modifying therapies can all be given a grade or score based upon how convenient they may be, how safe they are and how effective they are. No one medication will be effective for all individuals with multiple sclerosis. The decision as to which treatment is right for a given individual will take into account all of the above factors. When considering which medication to choose, there are two schools of thought as to how we pick a medication. These paradigms might be called escalation therapy versus induction therapy. Escalation therapy means picking a treatment with a great safety profile but perhaps a treatment that might be less effective than those more side effect risks. Many individuals with multiple sclerosis may have good disease control with one of these treatments. If they do not, then we could move on to a more aggressive treatment. The advantages of this treatment approach would be improved safety by hopefully minimizing exposure to higher risk medications. The disadvantage to this treatment approach would be that some patients are going to fail these less effective treatments and may be at risk for relapses, progression of disability and new lesions on MRI. The alternative to escalation therapy is induction therapy. Under this paradigm, we would pick a more effective treatment with perhaps a higher side effect risk as a first-line treatment. The advantage to this treatment approach is the potential for better disease control from day one. The disadvantage to this approach would be a higher side effect risk with the primary side effect being progressive multifocal leukoencephalopathy (PML).
The research horizon also includes the potential for improvements on existing medications. Gilenya (fingolimod) is one of three FDA approved oral medications for multiple sclerosis. Three drugs on the horizon, have similar mechanisms of action but may offer better safety. These are siponimod, ozanimod and ponesimod. Ocrevus (Ocrelizumab) was approved for both relapsing remitting MS and primary progressive MS in 2017. Ofatumumab has a similar mechanism of action, but is administered as a subcutaneous injection. Some might argue that this is more convenient than the slow infusion required with Ocrevus.
The management of multiple sclerosis currently focuses on three areas. These are relapse management, symptom management and modification of the disease course. Hopefully, the future will see a fourth area of treatment, neural repair or the reversal of disability. Areas of ongoing research that may offer the potential for neural repair include anti-lingo (opicinumab), clemastine, biotin and mesenchymal stem cell transplantation. Mesenchymal stem cell transplantation should be distinguished from autologous stem cell transplantation. The latter represents a means of "rebooting" the immune system and halting further progression. Several recent studies have shown that autologous stem cell transplantation may be a viable treatment option for some forms of aggressive relapsing multiple sclerosis.
Hopefully, we are moving closer to the day when we can effectively prevent multiple sclerosis for those who do not have it, stop multiple sclerosis dead in its tracts for those who are diagnosed, and reversed disability for those with multiple sclerosis who have been dealing with the disease for years.