Sharon Hesterlee of the Muscular Dystrophy Association Speaks on New Therapy, Vamorolone

Applied Clinical Trials: What sets Vamorolone apart from other therapies in this market?

Sharon Hesterlee: Vamorolone is a little bit different because we've heard a lot about gene therapies and some pretty high tech, very specific, genetically focused medicines in the Duchenne space. Vamorolone is actually a twist on an old medication. Corticosteroids have been the first line of defense in Duchenne for many years. There are strong anti-inflammatory benefits that help the inflammation that you get as you have muscle breakdown. So we know from experience that the corticosteroid use can delay the loss of ambulation by as much as two years. So they're actually quite effective at preserving ambulation and muscle strength. But the side effects obviously are well known for glucocorticoids. So weight gain, cushingoid features, they cause irritability, behavioral changes, loss of bone density, they delay puberty, and they lead to short stature. So there's a whole host of pretty significant side effects. It's been this good-bad situation where corticosteroids are effective, but they're limited by the side effect profile that not everyone can deal with. And so, Vamorolone is different, because it is a twist on this medication, designed to have the benefits without as many side effects.

ACT: We’ve heard, “this is the drug that the community brought to market.” Could you explain that and touch on the community’s role in this?

Hesterlee: Vamorolone was developed by a company called ReveraGen, and it was actually founded by an academic investigator who was one of the people who was involved in the original identification of the dystrophin gene and dystrophin protein, so he has long roots in community and that's Eric Hoffman, PhD, president and CEO of ReveraGen. He was really committed to this idea of: we want to bring this drug to market with as little interference as possible; we don't want to be driven by a fiscal bottom line. And to do that, he worked with the community and with some government funders, so that all the money that he took was non-dilutive. So there wasn't a group of shareholders or investors who were dictating what happened with the drug. And in total, I think there were 17 different foundations involved in providing funding for this in multiple government agencies in both the US and in Europe. And it was a total of about $24 million, that this nonprofit/governmental community put into the development of the drug. But I think that's also something that's pretty unique about it.

ACT: How important is the role of patient advocacy groups in bringing any potential therapy to market?

Hesterlee: There's a huge role for advocacy groups for any therapy. Often your therapy that you end up with is better if you involve advocacy groups from the beginning, because then you're getting better feedback about what patients are looking for in a drug, what they want to see that's effective, and how you design your trials; is what you're planning realistic? So there's all these different ways that patient groups and patient advocacy can weigh in on the drug development process; funding is just one of them. A lot of it is actually how the drug is developed and then when it comes to drug approvals, it also can be testifying at some of these FDA advisory committees about the unmet need and what the risk benefit tolerance is of the community if there were side effects, for example. So I think there's all these different roles at every stage of drug development where advocacy groups can be really helpful.

ACT: Could we see this R&D funding model more frequently in the future? Potentially across other rare disease areas?

Hesterlee: Yeah, I think so. I think it's really a matter of: as a lot of patient organizations get more sophisticated and motivated, they say these are the drugs we want to see and if we can support these ourselves. If they're not being driven by a for-profit company, we can band together and get the drugs on the market that we see a need for and that we want to see come to market. So I think it's going to be a more common strategy and it has been successful in other areas.