Tasigna (Nilotinib) as a treatment/cure for SCA?

[Goodlifee]

Hi,

I am wondering if anyone has tried Tasigna (Nilotinib - FDA-approved treatment for cancer of the white blood cells) as a treatment/cure for cerebellar ataxia.

There has been a recent study by renowned researchers in South Korea: ncbi.nlm.nih.gov/pubmed/286...

Some people in Korea have been taking the drug off-label, and some patients who have hereditary cerebellar ataxia have seen some fast improvements.

There was also a study by Georgetown University that Tasigna helped Parkinson's patients. There's a second phase of clinical trial going on right now: michaeljfox.org/foundation/...

If anyone has tried Tasigna as a treatment for your cerebellar ataxia, could you share it here please?

[sunvox]

Unfortunately you are asking for a miracle. Nilotinib is a LONG way off from being prescribed for someone with Ataxia. The science is good and encouraging and if I had worse symptoms I would be asking my doctor for an "off label" prescription, but I suspect you will not find any ataxic patients that have tried it yet.

-

What form of ataxia do you have"

[Nilotinib_SouthKorea]

Hi

Yes, here in Korea, after the small clinical trial with nilotinib, a few SCAs are taking it off lable. and almost every one feels positive.

Good luck.

[Goodlifee]

Thank you for your replies. Tkdrl, could you give me contact info for phD Joo? Also, are you still taking tasigna? Are you doing ok? Have you had any side effects? Thank you.

[Nilotinib_SouthKorea]

One of us let me forward following message to you.

We are Korean patient who are only diagnosed with SCA not MSA. The drug ONLY makes effect who are dignosed with SCA which is inherited desease and can be dignosed with blood CAG repeatiton.

As far as MSA , acquired desease is concerned, I'm afraid that the drug is not effective. I have been taking this drug for 18months. And And for me, the drug is perfecrt!!!before taking this medication, I wish you consult your doctor. Good luck.

[earthangel81 in reply to Nilotinib_SouthKorea]

Hello, thank you for this information. Have there been any studies showing that Nilotinib is ineffective for MSA? Thank you

[Nilotinib_SouthKorea in reply to earthangel81]

As I know, There was the small clinical trial in Seoul National University hospital two years ago. there was a dramatic result for SCAs but a subtle result for others.

sciencedirect.com/science/a...

[Farooqji]

all I know that price of Tasigna is exuberant

[Nilotinib_SouthKorea in reply to Farooqji]

Yes, it's the BIG problem.

[Farooqji in reply to Nilotinib_SouthKorea]

there is a forum member who is getting it manufactured with Chinese company at a much cheaper cost

[Nilotinib_SouthKorea in reply to Farooqji]

Isn't there a patent issue or something? I hope not.

[Goodlifee]

Thank you for your replies again. For those of you who have tried tasigna, have there been any side effects? Especially related to heart (I heard heart issues could be side effects)?

[Nilotinib_SouthKorea]

He said No side effect yet. The dosage we've got is much smaller than CML's.

[Goodlifee]

As I do more research, it does seem that some people with hereditary SCA are actually seeing fast improvements with this medicine. With so many SCA patients anxiously waiting for a cure, how come there's no research going on about this in the UK or in the US? Even if Tasigna is expensive, if it can actually improve SCA patients' conditions with minimal side effects, I think it is groundbreaking.

[tkdrl]

저는 19개월째 TASIGNA를 복용하고 있는 한국인 SC. 보조자입니다.

저는 증상의 70%를 회복했습니다. 회복률은 환자마다 다르지만, 거의 모든 SCA환자들이 회복하고 있다.

이는 SCA(SpinoCerebellaratocia)에만 유효합니다.

영국도 좋은 결과가 있길 바래.

가능한 한 빨리 약을 드세요.

효과를 느끼는 데는 일주일에서 한달이 걸린다.

우리 의사는 서울 대학교 병원에서 일한다.

그리고 그의 이름은 ' 준 '이고 그는 이 약에 대한 논문을 썼습니다.

그는 이 약에 대한 많은 자료를 가지고 있다.

질문이 있으면 그에게 물어 보세요.

I am a Korean SCA7 sufferer who has been taking TASIGNA for 19 months.

I recovered 70% of symtoms. Although the rate of recovery is various upon patients, almost every SCA sufferers are recovering.

It is ONLY effective for SCA (spino cerebellar ataxia).

I wish you British also has good result.

Take the medication asap.

It takes one week~a month to feel effect.

Our doctor works for Seoul National University hospital.

And His name is ‘주 건’ and he has wrote papers about the medication.

He has lots of data about this medicine,

If you have any question, consult him.

[HarryB Administrator in reply to tkdrl]

Hi tkdrl

Ataxia UK believes that any treatment for any of the ataxias should be offered on the basis of the results of rigorous trials in patients that assess both the risks and benefits of the intervention, both short-term and long-term. Nilotinib does not meet these criteria and therefore we cannot recommend it.

I am pleased for you that you think Nilotinib is helping you but until there larger trials into this drug for the treatment of ataxia, Ataxia UK cannot recommend taking it. I do hope you don't think this reply is overly negative. Hope is obviously very important but it is equally important that we ensure as far as possible that hope is within realistic, achievable and safe parameters.

Best wishes

Harriet

[tkdrl in reply to HarryB]

i talk you my experience. choice is yours. good luck

[Eunseo]

Im south korean with sca2. I have taken the medcine for 2years and 6 months. But i feel some degeneration. But I am sure that Tasigna slowed my desease and I could work yet. I dont regret taking it.

[Eunseo]

And first tme when I took this medicine, I felt really positive symptom for 1.5years. No fatigue and clear head and enjoying movement