Successful Phase 1 Trial for Potential New Cervical Dystonia Treatment

Bontulinum Neurotoxins (BoNTs) are a protein that are used to treat a wide array of conditions including cervical dystonia, blepharospasm, neurogenic detrusor overactivity, and sialorrhea to name a few. These proteins are classified into seven different sterotypes, however the vast majority of BoNTs used commercially are BoNT-A products. This treatment helps to prevent muscles from contracting, reducing their excess activity in diseases like cervical dystonia. It can be administered by injection directly to the affected muscle.

However, just because BoNTs have been effective for some patients, doesn’t mean researchers have slowed down in their search for ways to improve treatments for these conditions.

Ipsen has just released data from their first human trial examining a recombinant neurotoxin called rBoNT-E.

The Study

This was a Phase 1 study that utilized healthy volunteers. Data from the trial will be presented in Copenhagen at the TOXINS International Conference.

The trial included 28 healthy males who were between the ages of 18 and 49. None of the participants had received a BoNT treatment of any kind, at least in the 6 months leading up to the trial. For the study, 21 of the individuals received the rBoNT-E treatment and 7 received a placebo.

The trial indicated the drug was well tolerated. While there were some TEAEs, they were not considered to be related to treatment. Among all of the participants, no one seroconverted and no one experienced local diffusion to adjacent muscles.

The study was significant because it showed that rBoNT-E was not only safe and tolerable for patients, it also had a quicker time to onset of action than BoNT-A. Additionally, it had a shorter duration of effect and the treatment had a faster time to peak activity when compared to BoNT-A.

Of course further studies are needed to confirm this treatments potential. However this initial study was an exciting step in the development of what could be a new therapeutic option for patients dealing a condition like cervical dystonia.

Looking forward

Researchers are particularly excited about rBoNT-E following this study because providing more treatment options which have different onsets and durations of action, allows clinicians the opportunity to pick what is the best option for the individual patient. Too often there are no options. There is one treatment that is typically effective and that is what the patient receives. Studies like this show promise for the development of more individualized treatment plans for patients. After all, we are all different, it only makes sense that to be most effective, our medicine should be too.

You can read more about this trial and the impact this research could have for cervical dystonia patients here.

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