Meanwhile in Imukin-land

Just me again… Ranting about Imukin as usual 😛

So a pandemic just happened and making research on FA move even slower than before. Our patience have been tested in many ways this year, but if you have FA you are in hurry for real. No lockdown, one meter rule, face mask or antibac can keep us safe from the progressive force of FA.

I’m lucky to have Imukin while all this is going on,

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My 6 years Imukin anniversary speech

The 27th of August 2013 is a Wednesday.

I have have gotten my very first package of Imukin from the pharmacy. I’ve hung up the phone call with doctor Harald for the last instructions. We are ready for the first shot.

I lie down on the sofa with pillows under my knee. I don’t know how this injection will feel like so I make sure I’m comfortable. My father, Morten and my mum is in the room. My mum is nervous. She will do the injection. As an experienced nurse she is never nervous before an injection, but it’s different with us kids. She always gets nervous treating us even now that we are not kids anymore.. She never shows it. She is the calmest person on the ouside as she fixes the syringe and desinfect my thigh.

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Status – Imukin May 2019

My name is Gunnhild Lystad, I’m now 40 years old. In 2009 I was diagnosed with friedreich’s ataxia (FA). In 2011 I started my blog because I wanted to share my “insider” tips to others being a physiotherapist myself and living with this condition. In august 2013 I stared taking Imukin injections. In October I was certain about the positive effects I had and decided to share it on my blog. The years to follow more people started taking Imukin if they could find a doctor willing to prescribe it and if they could afford it. Two big “ifs”. Here I will sum up some of the feedback I’ve got from others taking Imukin. In the end of the document you’ll find more recent updates and finally my own update.

 

 

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Do you want to read the Imukin stories?

Thank you for all the replies you have sent me so far. It is quite interesting to read, right? I hope we will keep getting many more replies. Comment below or send me an email: fafysio@extremeliving.no

Thank you so much dear translators for helping me spreading the word; Ahmet Sami Konca, Marisa Condurso de Nohara, Marilou Mertens, Dorival Ruiz, Amalia Maranhao and Michela Mogavero.

Also a big thank you to the amazing Gian Piero Sommaruga for coordinating translations and always for your great support and patience.

If you are not following the Babel Family group on Facebook than you really must go there and like the page https://www.facebook.com/groups/52801039038/

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New SARA score after 5 months on Thiamine

Today I took another SARA score at my doctors and it is very exciting. In this score the best result is 0 – zero.

31.08 – scored 9,75 (right before my first injection)

26.10 – scored 7,2

25.01 – scored 5,1

Today I was able to take some steps  in tandem walk and stand for longer in tandem stance. It didn’t look pretty, that’s for sure, but I did it. Imukin and Thiamine seems to be a good match ❤

Many people are very curious about Thiamine. If you have tried it, do you mind leaving me a comment below? Your experience is important because it can help others. Thank you 🙂

Here is more about my own experience, dosages, link to reasearch and so on.

It’s been to long since I posted a picture of my cat, I know. Her she is 🙂

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When significant isn’t significant

I know this weekend has been filled with disappointment and sadness in many homes all around the world. The news that Horizon ended their study on interferon gamma struck the FA-family hard last Thursday. The research on using interferon gamma as a treatment for FA “did not meet its primary endpoint of a statistically significant change from baseline in the modified Friedreich’s Ataxia Rating Scale (FARS‐mNeuro) at 26 weeks versus treatment with placebo.”

I cried. Not because it changes anything for me. I have the medication I need to keep going. My heart broke thinking about all the children and people with FA that never will be able to test if IFG will improve their life or not.

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Interferon gamma may improve cardiac function in Friedreich’s ataxia cardiomyopathy

Do you like what you’re reading? I love it!

Thanks to the research team at Akershus University Hospital in Oslo with clever doctor Wyller in charge and not least, thanks to amazing Emilie, the scientific article about Imukin and cardiomyopathy is now published. This is a case study so the research is done one person, but pretty promising right?

You can download the article here: Wyller et al_Imukine for cardiomyopathy in FRDA_Int J Cardiol 2016

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Cardiomyopathy and Imukin – promising news!

This morning I woke up to an incredible message of hope! Not a bad way to start a new day?! On my phone there was a message from a happy mother sharing some good news about her daughter Emilie. Emilie is a great inspiration to me in so many ways. She is a musician and is releasing her first single later this summer. We are so excited about this. It is called “Unbreakable” and I’ve been lucky enough to listen to some of it already. It is so good!! 🙂 She is 18 years old and has FA.

Beauty, musician and warrior ❤

Emilie is suffering from severe cardiomyopathy and has been for many years. She has been in and out of hospitals. You can imagine that her doctors had many dilemmas when she last year asked about trying Imukin… No one else with FA and cardiomyopathy is taking Imukin, as far as I know. Her cardiologist made thorough research. After many discussions they decided that Emilie would start injections of Imukin. She started last October and the first dose was 10 mcg and injected at the hospital to be able to monitor her for 24 hours. It all went well. Unfortunately she’s had a bit of a hard time adjusting to the medication and she has been having more side effects than noticable effects for a long time. Side effects has been fatigue, muscle aches, nausea, headaches, depression and sleepiness. Hard to stay motivated feeling like this, but she kept going.

Now look at what her mother wrote me:

Emilie was just on her 6 month follow up with her cardiologist and we have some good feedback.

Emilie has been on the verge of giving up on the Imukin injections altogether, but since about one week ago something changed. The experienced side effects faded and the effects started showing. She has grown a lot stronger (and she was already very strong), she finds it easier to stand up, her movements are more controlled and generally everything seems a lot better.

Her heart markers are a lot better. One of them, pBNP, is now completely normal and Troponin T is almost normal. Both of these markers used to be “sky high” on Emilie. They have analyzed her ultrasound images from before starting Imukin with the images taken after 3 months and now after 6 months. The experts are convinced that her heart function has improved, both the systolic function and the diastolic function. In addition they notice that the walls of her heart have slowly grown thinner.

Emilie is about to increase her dosage to 150 mcg now so it will be very exciting to see if the positive trend continues. We are also very excited to see if her scoliosis has improved. She is going to a back clinic at the beginning of June.

Everything is very promising and more people could possibly also benefit from Imukin, maybe even those with cardiomyopathy.

Great news or what? 🙂

If you have any questions please write them below in the comments field. Emilie will help me with the replies 🙂

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The definition of frustration

frus·tra·tion

frəˈstrāSH(ə)n/

noun

noun: frustration

1. the feeling of being upset or annoyed, especially because of inability to change or achieve something.

When I hear doctors (and parents) refusing Imukin treatment to people with FA who wants to see if it can help it makes me laugh AND cry! It is both hilarious and devastating. Imukin (interferon gamma/ actimmune) might not help everybody, it won’t cure us but many people who have tried it experienced relief of symptoms. There are no words in the world that can justify what kind of relief that is!

How can the risk of flu-like symptoms be a reason not to try Imukin when FA is as terrible as it is? I know many decition makers are scared that there are more serious side effects to Imukin. There might be but it is not common. What kind of medicine does not have serious side effects listed in their description?

The most common side effects to Imukin are flu-like symptoms. What is that:

Flu:

• fever
• headache
• chills
• myalgia

I would take flu-like symptoms every day for the rest of my life as long as my FA symptoms gets better. That said, I hardly feel any flu-like symptoms from taking Imukin anymore. The body adjusts.

This is why I think doctors should let FA patients try Imukin:

Friedreich’s ataxia (FA) is a debilitating, life-shortening, degenerative neuro-muscular disorder.

• loss of coordination (ataxia) in the arms and legs
• fatigue – energy deprivation and muscle loss
• vision impairment, hearing loss, and slurred speech
• aggressive scoliosis (curvature of the spine)
• diabetes mellitus (insulin – dependent, in most cases)
• a serious heart condition (enlarged heart – hypertrophic cardiomyopathy)

Again, I am not saying that Imukin is a cure but it might make symptoms improve. Please do something! Something is better than nothing!

Just sayin…

 

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The phase 2 results are out. Exciting!

Last Monday the results from the phase 2 study of Interferon Gamma for the treatment of Friedreich’s Ataxia was presented. There was also a presentation held by the pharmaceutical company Horizon Pharma that owns the right to Actimmune/Imukin.

This event was recorded and is available HERE. You can both listen to the webcast and see the slides they used.

I thought I would try to make a short version of the main points of this presentation. This is a mix of the info that was shared from the different speakers.

Just note that I am not part of the studies in any way. I am not a researcher or a doctor. What I write in my blog is based on my own individual experience and how I understand the facts.

SUMMARY

Actimmune (interferon gamma-1b) has been on the market for many years and is already FDA approved for two other orphan diseases (orphan diseases = rare diseases). The first approval came in 1999 for Chronic Granulomatous Disease (CGD).

There have been about 200 clinical trials done on Actimmune. The research is done on a variety of diseases so the safety and tolerability of the drug is well documented.

The good news for us is that since it already is on the market it is usually easier to get it approved for another indication than if it was a whole new molecule. This can speed things up. Speed is a good thing, right?

Not much of the research on FA going on today is done on children. This is because the majority of substances tested are new molecules. Using children as subjects in research is therefore often restricted, but not on Actimmune. Since this medication already is used on children (with other conditions) they were able to do this phase 2 study on children with FA.

STUDY DESIGN

• 12 people, ages 5- 17
• Doses: from 10 micrograms, gradually increased to the maximum dose at 50 mcg
• 12 weeks duration
• Measurements: Frataxin levels, Neurological tests (including FARS). See the other tests below;

RESULTS

Side-effects:

Almost everyone experienced some kind of reaction, but they were minor and typical for IFG. These are the flu-like symptoms.

Frataxin:

The way that frataxin is measured is through blood cells and buccal cells. Buccal cells are the cells you can sample from a swab of the inside of a person’s cheek. You have probably seen this on CSI 😉

The problem with this measurement is that these cells are not affected in FA. The best thing would be to investigate the neurons affected, but this is not possible at the moment. We cannot go to the lab and deliver samples of our spinal cord every other week, right! Some interesting problems we would get then… 😛

Back to the results;

“While there was statistically significant change in frataxin levels it was not at a level or magnitude that could be clinically meaningful.” You can see every individuals change in frataxin below. Every line shows one person. Injections stopped at v5.

Even though the increase is not high enough to make any conclusions at this point, the researchers find this amount of “noise” on the charts unusual and very interesting for further research.

FARS

FARS can be a bit confusing since 0 is good and high numbers are bad. If you have a lower score it means you have improved. A higher score means that you are getting worse.

After 12 weeks the FARS score decreased by 5 points!

Five points might seem very little, but check this out; They say that 6 point increase is the normal progression rate over two years. The results indicate that these children improved about 18 months in neurological function!

This is a significant neurologic improvement.

The improvements did not last when they stopped the injections as the trial ended.

NEXT STEP

As you can see below the phase 3 study is already in planning. Horizon Pharma and FARA will do what they can to speed up this process. Scheduled start of phase 3 is during the second quarter (April – May) of 2015.

 

Finally a comment about my own FARS score. I started taking Imukin the 27^th of August 2013. My FARS at baseline was 47,5. When I re-tested the 10^th of December it was 44. That means 3,5 points improvement. I was told that this change was very low, but what I learned from hearing JenNifer Farmers speech last week is that 3,5 points improvement equals over 1 year improvement! That is huge! This was after 15 weeks on Imukin.

 

 

 

 

 

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