SCA3/Machedo Joseph Disease has specific symptoms, if she is being seen weekly any change symptoms will likely be monitored. But if she isn’t, it could be worthwhile contacting the hospital for advice.
thank y’all for your comments. But what she is after is anyone experiencing condition getting worse while on this test drug
we do to thank you for your help
I am on BHV-4157 But I have not experienced any significant deterioration. But normal progression of the disease is expected.
My wife would like to know how long you have been taking the test drug BHV-4157.
I took BHV-4157 for 1 year in 2017 -2018 ( doubled blinded study, with probably 3 months of placebo) and started the second study( without any placebo) in the end of January 2019. My neurologist thinks my SARA score hasn’t changed much during the study.
So…just so I understand you correctly Shoaib. In phase 2, you weren’t given the placebo because of improved SARA scores at the time. But overall, scores haven’t improved
There was no improvement or deterioration of my SARA score during the first study in 2017 - 2018. The second study ( which I am enrolled in ) started in January 2019 with the same dose ( 140 mg.) to show if it slows down the progression of the disease. There was an option to increase the dose of BHV-4157 only if the SARA score dropped during the study. Fortunately, my SARA score has not changed since the beginning of the study. The third study began in March 2019 ( which i am not a part of) with increased dosage ( 200 mg.) of BHV-4157 . Some members on this board have reported a dramatic improvement of SARA scores with increased dosage of BHV-4157. But unfortunately, they are false. My neurologist thinks that Biohaven will apply for FDA approval of BHV-4157 for slowing the progression, after all is said and done. Although I am not too excited about BHV-4157 , I have a feeling that the FDA application will light up a fire for other companies to get into the race. We will just have to wait and see.
Thank you Shoaib. From what I’ve read elsewhere, there seems to have been a lot of faith placed on this study. Time will tell…
Shoaib, can you enlarge on ‘false’.
Sure. No data so far has come out on that study. To claim that the SARA score has come down from 12 to 1 is misleading.
I heard their representative explain the findings of clinical trial at the Ataxia conference last year.in Philadelphia. It was all smoking mirrors with their data proving there wasn’t a decrease with progression of the disease using BHV-4157.
but we have no choices,. T_T
I hear you. Biohaven is trying to look at the data and make their investment worthwhile.
My wife has a question for you,
- While you were on this test pill were you in any pain in your body?
she has pain in her stomach area and it is hard to find any medicine that she can take to take care of it. She was wondering if it is cause by this test pill.
I doubt that the BHV-4157 is responsible for any stomach pains. But I would let the investigator team know immediately. There has not been any reports that it causes any stomach pains and this is the second time I had been on it without any adverse effect.
Now do you feel any degeneration?
I don’t understand what you mean by, " if I feel any degeneration". There is natural progression of the disease, but I don’t feel any side effects from BHV-4157. I did not have any follow up MRI to see if there’s any further shrinkage of the cerebellum.
You have progression. I’m sad.
There is nothing that halts the progression. Statistical manipulation by Biohaven is not surprising to show there is slowing only of the progression. But that’s a start. Expanded Analysis from SCA Clinical Trial BHV4157 (2).pdf (147.3 KB)