Investigational Agents for the Treatment of Myasthenia Gravis


A. Gordon Smith, MD, FAAN, describes the landscape of investigational therapies for myasthenia gravis, including plasma cells, CAR-T, antigen-specific tolerance.

A. Gordon Smith, MD, FAAN: These are really glory days in terms of therapeutic development in myasthenia gravis, and we have new therapies coming at us at a very rapid pace, which is very exciting. It is going to prompt a great deal of really interesting discussion about how these treatments are used. Several of the treatments that are in the pipeline are from similar disease classes to those that we've already discussed. So here I would include other FcRn inhibitors, other complement inhibitors, or B-cell depleting, for instance, anti-CD19 targeted therapies. In these, the variation between the drugs would be the extent to which they are successfully engaging in that target, but I think a lot of it's going to be dosing frequency and dosing route. All of our therapies right now are intravenous. We expect to have subcutaneous formulations coming along, and this will be helpful for patients in their small molecule approaches to some of these targets, as well, that are in development.

What I'm really excited about, one I've already mentioned is the idea of developing a scientific base for predictive validity: which agent should we be using? This is going to require us to think a lot about immunophenotyping and biomarker development. The other area, of course, is what targets haven't been explored, and there are some really interesting targets in early therapeutic development. An obvious one would be what about targeting plasma cells as opposed to B cells? Obviously, if we deplete B cells, they are still tissue-resonant plasma cells making antibodies, so it's possible that targeting plasma cells may be helpful. There are even very innovative approaches, for instance, using CAR T therapy. We are all familiar with CAR T cells as a mechanism to treat lymphoma and leukemia, but this technology's being leveraged to treat myasthenia gravis or even ways of engaging in the T-cell aspect of myasthenia gravis to induce antigen-specific tolerance. Fr instance, a nanoparticle-type approach is being explored right now. I think we've got advances in current classes of medications that we have, new formulations, new routes of administration, as well as innovative targets that are being explored.

Transcript edited for clarity

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