Potential First-in-Class Therapy for Spinocerebellar Ataxia

I am SCA2. Tuesday 8/8 will finish up phase 3 and will let everyone know how it goes.

Hello everybody, Well, I just came back from my Extension Phase Visit 2 (Week 8) from Columbia University and there is nothing new to report. I decided to continue with trial only by eliminating ALL my supplements from diet and will follow up then.

will post a new thread = Placebo

As I understand it, Trigriluzole is a ‘finely tuned’ form of Riluzole and is less harmful to the liver when absorbed.

The drug has a few known uses. In the case of Cerebellar Ataxia it’s thought to prevent further damage to certain brain cells (motor neurons) responsible for controlling muscle function.

Final results of the trial are said to be available early 2018.
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Hi everyone. The second part of this trial for bhv-4157 is over and the FDA did not see enough evidence to approve it. However the 3rd part continues until the end of 2018. My daughter has had significant improvements. They did a test on her called a SARA test. Her score was at an 11. Her score went down to a 2. She still continues to tic when she is exhausted. She says learning seems to be easier as well. Her speech is much clearer. The doctors all agreed that the 8 week blinded placebo test was not long enough. My daughter continues with this medicine and will through 2018. I will post new findings.

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A post was merged into an existing topic: Cocktails anyone?

Trigriluzole (BHV-4157) is a prodrug of Riluzole. I know that this form isn’t utilized by the liver as much and more drug is introduced to the brain. I know Riluzole has been around for a while and hopefully Trugriluzole will help many Ataxic patients. I know it is helping my daughter and I hope it will get approved in 2018. :pray:t3:

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Hi Cinthia if the trial failed then why is your daughter still on the drug?

Is there still hope for this and ataxia?

I do hope so

Hi. The trial is in the 3rd phase. They will have us on trial until late 2018. I was hoping the 2nd part of the trial would of given us the go ahead but all of the physicians agreed that 8 weeks is not long enough. So there is still hope. My daughter is improving and her name came up during a talk between all of the physicians on this trial. I don’t know why some people show improvement and why some people may not. She has sca8, young, and female. Maybe these factors have to be taken into consideration. So much to study but at least there is a study.

I want nothing more than for every ataxia patient to find a cure, but the fact is this drug is like levedopa in PD. It is a treatment for symptoms. Please, please, please talk to your doctor about some of the other molecules I have linked to in my posts.

Hi Andy. We are seeing one of the top doctors in the nation studying Ataxia. My daughter is very fortunate. There are only a few Ataxia clinics in the United States The top doctors around the nation are excited about this study. For now there is not a cure but this medicine may be blocking the progression. This is not covering the symptoms but stopping the progression and hopefully letting my daughters brain heal. In the future they may find a way to stop the replication process with DNA manipulation but for now my daughter is improving and I thank the Lord for this miracle and I hope it will help others. :pray:t3::two_hearts:

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I’ve been on “real” drug for 12 weeks through the clinical trial at UCLA. I have noticed significant improvement in standing and walking. My diagnosis is SCA1. The drug works by stopping the death of Perkingy cells in the cerebellum. The brain is capable of making new brain cells, but only if the rest of the body is in very good health. Even doctors were not aware of the brains ability to regenerate until the 1990”s. Read Dave Asprey’s book, HEAD STRONG. I’ve changed my way of eating, exercise, meditation and several other factors. Biohavens drug is not a magic pill, but if you’re willing to make lifestyle changes along with taking the drug; it does feel like magic. In May I was shopping for canes and walkers. Now I walk normally and started playing golf again. Keep strong and keep trying.

Hi. I am so happy to hear about your success. We are also at UCLA. Love the Doctor. My daughter thank goodness is taking nutrition in College. She had to drop out last year because of her Ataxia now is able to concentrate again. She is taking good care of her body with food and exercise. She still has the depression but has doctors helping her with this. She is 21 and has started showing symptoms at 16. This was hard for her to cope at such a young age. I am so excited about her future with this new treatment. How was your SARA score and how much did it change if I may ask. Thank you so much for your voice_. I’m am so happy for you!! They better approve this in 2018. Lol.

I saw Dr. Perlman on Thursday and my score improved another 2 points. When I walked down the hall for her, she said if she didn’t know, she wouldn’t notice anything unusual with the way I walk. People don’t whisper to my wife, “What’s wrong with him?” anymore. I didn’t get diagnosed until age 55, I’m 59 now. I know it progresses faster when patients show symptoms at a younger age. My nephew is 41 and has worse problems walking than I do. I wish your daughter the best; good luck.

Yes it seems people are showing symptoms younger and younger. Thank you for sharing. I may be the carrier of the disease but have not shown any significant symptoms. I am 55 years old. I wish your nephew the best in the future. Maybe he can start the meds next year. I am sure you are giving him hope with your participation in the study.

My daughter has sca2 and to be honest a cure would be great but right now some form of treatment to halt progression would be all we could wish for

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AMEN!!! I wish it got approved already. Now there is hope.

Hey Punk, I feel like you do. I have SCA 2 and tomorrow marks being on this clinical trial for 22 weeks!!! I wish this would halt my progression. It has been such a waste of time.

Hi Henry I’m glad you are still in the study. I thought you gave them back the meds. I hope you feel some results soon. Don’t give up!! Thinking about you. :pray:t3:

Hi Henry have you any feeling that progression may be halting in any way? Have the doctors said anything? Hope you keep on fighting mate.

The thing I don’t understand about this trial is that I thought it had failed so why are you both still taking the meds?