A possible VWD treatment could offer long-sought options

Last week, I had two dental procedures that required pretreatment because of my hemophilia B and von Willebrand disease (VWD).

After prepping one of my teeth for a crown, my dentist took an X-ray that revealed an abscess, which meant I needed a root canal, too. Both procedures required pretreatment because needles would be used to numb the area. I’ve had situations where those injections triggered a bleed and caused my jaw to lock mid-procedure, an experience I’m in no rush to repeat.

It’s also notoriously difficult to get me numb, which often means multiple injections are needed.

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A shift in treatment and perspective

My von Willebrand factor levels have improved recently, so with my hematologist’s support, I opted to treat only with my hemophilia B medication (BeneFIX, or nonacog alfa), skipping my usual VWD treatment (Humate-P). Treating requires finding a vein, starting an IV, mixing the medication, and infusing it. It may sound simple, but when you’re doing it one-handed, it’s anything but. And to complicate matters further, I’m not an easy stick; my tiny veins are notorious for rolling and blowing.

That said, I was struck by how much simpler it was to treat with just the factor IX replacement. The volume of medicine was noticeably less — just two vials of BeneFIX compared with the multiple vials of Humate-P. For the first time ever, I found myself thinking: This isn’t so bad. If I only had to treat with BeneFIX, I could handle more frequent infusions.

Living in both the hemophilia and VWD communities, I often find myself pointing out the stark contrast in treatment development. While hemophilia treatments continue to advance at lightning speed, progress for VWD has felt more like a slow crawl. In fact, my VWD medication hasn’t changed in over 20 years.

That’s why I was especially excited to meet with two representatives from STAR Therapeutics this week. They’d reached out to me through my website, Girls Bleed Too, to ask if I’d be open to sharing my lived experience as a woman with VWD. We met via Zoom, where I shared the challenges and frustrations I’ve faced over the years with both treatment and medical providers. I felt like they got it and that they’re genuinely working to meet an unmet need in the VWD community.

They’re developing a new treatment, VGA039, designed to work across all types of VWD. Even more exciting? It’s subcutaneous, which means no more IVs; just a quick injection, similar to how insulin is given for diabetes. VGA039 received fast track designation from the U.S. Food and Drug Administration in January, a promising sign for the VWD community.

For those of us living with VWD, this kind of innovation is both encouraging and deeply personal. It represents not just progress in treatment, but validation that our needs are finally being recognized. I’ll be watching closely as VGA039 moves through clinical trials, holding on to the hope that one day, treating VWD might be as manageable and advanced as treating hemophilia has become.

Note: Hemophilia News Today 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 another 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 Hemophilia News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hemophilia.