Hi
Does anybody know if RILUTEK is effective medication for SCA3 please thank you
Quick Answer: No-one really knows yet.
See next post for detals of known studies.
Basically, though Rilutek shows some promise as a potential treatment for Ataxia generally, in theory and a small study, no large scale studies have yet been done. No studies just concentrating on a subtype such as SCA3 have been done either. There is no information on long-term effects where people take it for years and are followed up. So, most of the studies about Rilutek still comes from it's use in ALS (Motor Neurone Disease).
Why do you ask? Have you been offered it? Or to take part in a trial? Or simply found out about it?
Abigail
For information anyone interested, here are the few studies done or going on now for Rilutek in Ataxia.
(1) The first small scale "safety and efficacy" study (40 people, mixed causes and types of ataxia). There were only 40 people, so the trial was a small one, and only lasted two months. But they claim some people showed up to a 5-point drop in their overall Ataxia symptom severity in the ICARS Ataxia Rating Scale, being some improvement in speech and movement:
http://www.ncbi.nlm.nih.gov/pubmed/20211908
Did any have SCA3? I'm unclear. A copy of the full paper can be seen here:
http://xa.yimg.com/kq/groups/21616406/92363872/name/Riluzole%20in%2...
But that version of the paper presumably has some typo's:
(a) "6 had SCA3" according to "Results, Patient Disposition and Characteristics" on page 3
(b) Yet the table on page 4 shows cases of SCA2 and SCA1, but no SCA3.
Obviously, they can't both be right. According to the WeMove.org website, "none had SCA3" in that trial. See under "Treatment":
http://www.wemove.org/ataxia/ata_sca3.html
(Fuller details at http://www.mdvu.org/emove/article.asp?ID=1248 )
So, I'm still unclear if any had SCA3 or not - possibly not!
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(2) Here is the same team's newer "safety and efficacy" trial, which began in 2010. They hoped to get 60 people in it this time, and it lasts for 12-months, but mixed Ataxia types again, and no specific mention of SCA3. No results yet, as it finishes in January 2013, results expected to be published in May 2013.
http://clinicaltrials.gov/ct2/show/record/NCT01104649
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(3) Here is a grant which was awarded for research on Rilutek in the Mouse version of SCA3:
http://www.ataxia.org/research/studies/2011/naf-research-jana-boy.aspx
Now you know about as much as anyone..... Rilutek sounds potentially good, but they're only just starting trials, but there's other avenues of research going on too. So, it's all still "hope for the future" more than it is available as definite "help right now".
Abigail
Here is a short video (lasts 3:34) about SCA3 in the EUROSCA research programme, which has found certain substances in Celery and Parsley which have stopped degeneration in cerebellar cells in the Lab. They hope to turn this into a treatment.
http://www.youtube.com/watch?v=YUH5pUROCBs
Dr Klockgether, who is mentioned in this video, also did a good study on "Sporadic Ataxia" and MSA-C.
Yours,
Abigail
I feel for you, having read your profile info. Like other similar conditions, these Ataxias are not a nice situation to be in, whether one is the patient, partner, family, friends or carers. Emotional support from such groups as this and Ataxia UK is important for patients and carers. But it sounds like you're "in the loop", so if a trial or study does happen more locally though, try and get in it!
Personally, right now, I'm waiting for tests and results of MRI etc., to know what further tests to do - so I haven't actually got any proper diagnosis yet. As my main symptoms are Ataxia, Dystonia, and Peripheral Neuropathy, SCA3 is on the list of possibles for me too. But there's many others (SCA2, SCA17, AOA2, other degenerative or metabolic conditions). I can only wait and see.
Yours,
Abigail
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Hi Abby,
for me it `s clear. There was no SCA3 patient in this study. 6 SCAs from which where four SCA2 and two SCA28. Nine Patients from twenty were responding.. We don`t know if anyone of the SCA´s responded, - it`s too much anonymized.
(a) "6 had SCA3" according to "Results, Patient Disposition and Characteristics" on page 3
There is a comma between "SCA" and "3", which changes the understanding. Please look again on the paper to see if i am right or not.
(b) Yet the table on page 4 shows cases of SCA2 and SCA1, but no SCA3.
Yes, there was no SCA3 in the study. But scientists don´t worry about this detail. I suppose because it seemed sure for them that the patients with a cerebellar ataxia involved into this study were representative also for the other types in general, "a mixed population of ataxia patients suggested a possible antiatactic action of this compound"
When the researcher on the German Webside of the University of Tübingen underlines the importance of a research project especially for SCA3, he/she wants to bring only an argument to get the funding. Supposedly there exists a reason why SCA3 was chosen for the trial. Often this is done for the patient group with the supposed best chance for success of the trial. So you can be optimistic. They surely have already treated SCA3 patients with Rilutek, and as a consequence, chosen SCA3 as the patient group for this study.
Kind regards,
Margarete
http://www.ncbi.nlm.nih.gov/pubmed/21734495
Curr Opin Neurol. 2011 Aug;24(4):339-45.Update on degenerative ataxias.
Source
Department of Neurology, University Hospital Bonn and German Center for Neurodegenerative Disorders, Bonn, Germany. ■■■■■■■■■■■■■■■■■■■■■■■
Abstract
PURPOSE OF REVIEW:
Degenerative ataxias are a heterogeneous group of disorders that are clinically characterized by progressive ataxia. They can be subdivided into three major groups: the acquired ataxias, which are due to exogenous or endogenous nongenetic causes, the hereditary ataxias, and the nonhereditary degenerative ataxias. On the basis of a review of the literature published in 2009 and 2010, this review gives an update of the most recent developments in the field of ataxia.
RECENT FINDINGS:
Using advanced methods of molecular genetic analysis, novel genes for recessive and dominant ataxias were identified. Recent imaging studies in dominantly inherited spinocerebellar ataxias (SCAs) focussed on the analysis of connectivity in the brain. Novel clinical assessment methods were developed and validated in large patient cohorts. Although a phase 3 trial of idebenone in Friedreich ataxia (FRDA) failed, a smaller phase 2 trial of riluzole in a mixed population of ataxia patients suggested a possible antiataxic action of this compound.
SUMMARY:
Recent molecular advances underline the diversity of degenerative ataxias. With the progress in the development of clinical assessment methods for ataxia, the methodological requirements to run large interventional trials are now met.
- PMID:
- 21734495
- [PubMed - indexed for MEDLINE]
--
i think the subtype makes only a difference when it has special features not existing in the other types.
In general: Abigail: thanks. You are also reading the studies.. Its sometimes fascinating!
"As my main symptoms are Ataxia, Dystonia, and Peripheral Neuropathy, SCA3 is on the list of possibles for me too. But there's many others (SCA2, SCA17, AOA2, other degenerative or metabolic conditions). I can only wait and see." Principally i find diagnoses helpful, but in the watch and wait phase you also can try to act somehow to emeliorate your symptoms. In this regard it would be interesting for me to have a description of what you feel is you Dystonia. Has this already been diagnosed? Are you doing something against it?
There is a successful treatment with "levodopa" described for a SCA3 patients. What is levodopa? Have you heard about it?
Kind regards,
Margarete
Postgrad Med J 2004;80:363-365 doi:10.1136/pgmj.2003.015297
- Case report
Dramatic levodopa responsiveness of dystonia in a sporadic case of spinocerebellar ataxia type 3
Abstract
A genetically confirmed case of spinocerebellar ataxia type 3 (SCA 3), presenting with disabling foot dystonia, peripheral neuropathy, and minimal cerebellar signs is reported. The dystonia improved dramatically with levodopa treatment in the absence of additional parkinsonian feature. A trial of levodopa for dystonia in SCA 3 may be of therapeutic benefit, at least in the initial stage of the disease.
Levodopa
http://www.cancer.gov/drugdictionary?cdrid=42622
Free Fulltext of the mentioned article (not everybody responded to the medication. :
http://pmj.bmj.com/content/80/944/363.full?sid=007eed73-4984-414a-8d0b-f6e3abbf8ad6
i don`t know if its easy to get and/or its side effects, but could perhaps be a chance. Necessary in general to consult a doctor. But what to do, if really nobody wants to treat you till you have the definite diagnosis? i often have got such problems. It`s hard to cope with.
Kind regards, good luck and thinking,
Margarete
In the italian rilutek-study from 19 evaluated patients 13 had improved after 8 weeks by at minimum 5 points, another three with 4 points. The three other were: a man with the worst symptoms of all at the beginning of the study, and two women with relatively few points/fewer symptoms at study onset. All others improved.