Research in new vasculitis treatments gives me hope for the future
Advocacy influences whether the next breakthrough drug will get a chance
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If you’ve been living with ANCA-associated vasculitis (AAV), irrespective of the type, you’ve probably felt both sides of hope: the side that feels good, and the frustrating side when treatments don’t quite get you where you want to be.
Today, I want to focus on the hope of new and investigational treatments. This type of hope allows me to imagine an improved quality of life.
One example is the oral therapy Tavneos (avacopan), which targets C5a receptors, a part of the inflammatory cascade driving vasculitis. Tavneos helps to reduce a patient’s reliance on steroids.
We’ve also seen it with Fasenra (benralizumab) in eosinophilic granulomatosis with polyangiitis (EGPA). Fasenra activates natural killer cells to clear eosinophils more effectively. I must say I love the image of natural killer cells attacking the things that cause me damage.
But these aren’t just new drugs. They represent a shift in thinking away from broad suppression and toward targeted treatment. And what gives me even more hope is what’s coming next.
What’s in the pipeline (and why it matters)
Active clinical trials are happening right now across granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and EGPA. These aren’t hypotheticals; they’re actual studies with real patients.
After a brief search, a few stood out to me:
- Depemokimab (GSK3511294) is being studied in a Phase 3 trial called the OCEAN study for EGPA, comparing it to existing IL-5 therapies (therapies designed to block interleukin-5) like Nucala (mepolizumab).
- Telitacicept is a B-cell–targeting therapy being studied in GPA and MPA, aiming to better control immune activity.
- Obinutuzumab is a next-generation, anti-CD20 antibody-based therapy (similar to rituximab, but potentially more potent) currently being studied for AAV.
- Complement pathway therapies, like next-generation C5 inhibitors, could refine how we block inflammation at its source.
- Low-dose naltrexone is being studied for quality-of-life improvement (including fatigue and pain) in EGPA, GPA, and MPA, among other conditions.
Other trials are exploring things like microbiome changes, relapse prevention strategies, and even entirely new biologics for kidney involvement in AAV.
The importance of all of this, to me, is what those potential treatments mean in real life. It’s the difference between having a quality of life that feels like a 3 on a scale of 1 to 10, and a 6 or a 7. It’s the difference between simply existing and actually participating in life.
Every time a new mechanism is discovered, a trial moves forward, or something gets approved, it creates for us patients another possibility, another option, and another shot at something better. If you’ve ever had a treatment change your life, even by a little, you know how big that is.
The part we don’t talk about enough
Here’s the part that’s easy to overlook: None of this happens without good public policy.
It takes over $1 billion to develop a single medication, and 90% of clinical drug development fails. That’s a harsh reality. Therefore, every success exists because of massive investment, risk, and support structures that allow research to happen in the first place. Someone chose to invest in our potential treatments.
Public policy directly impacts research funding, incentives for rare disease drug development, access to clinical trials, and drug approval pathways. If those systems weaken, the pipeline slows. And when the pipeline slows, fewer options will inevitably reach us. This is why advocacy matters — even if it feels far removed from our day-to-day lives.
Advocacy helps determine whether the next Tavneos will come along, or whether the next Fasenra will be approved. It influences whether the next breakthrough drug will even get a chance.
Personally, I don’t think hope means believing there’s a cure around the corner. But I do believe that we’re ahead of where we used to be.
Treatments are becoming more targeted. Options are expanding. Clinical trials are active across GPA, MPA, and EGPA. For those of us living with vasculitis, that matters — because we’re not just waiting for the future; we’re living in the middle of it.
Note: ANCA Vasculitis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of ANCA Vasculitis News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to ANCA vasculitis.
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