Trial participation has huge value

People sometimes think that because they’re invited to participate in a research trial, participation is free. In the sense that free means without cost, it’s not. Trials are enormously complex, time consuming, and expensive. Participants might not be required to pay anything, but someone is paying.

In addition, for participants, there are costs other than money. Participation requires commitment to responsibilities that if they aren’t delivered on, could damage or even invalidate the trial. The most important of these is discretion. It’s rare that participation would need to actively be kept secret but anyone making public that they’re in a trial is simply oversharing.

(Before anyone ever posts anything about their condition or possible treatments in any online forum, they should consider that their post is likely to be read by a newly diagnosed person who will probably misunderstand and/or misinterpret the context.)

The ultimate trial format is double-blind, where neither researcher nor participant knows who’s getting whatever’s being tested and who’s getting a placebo but for several reasons trials can’t always be run that way. In the case of FA there are so few of us that it’s often difficult to get a decent sample size at all. Some trials are for equipment rather than drugs so there is no control sample. As a result, participants often know that they’re getting the treatment being tested and validity of the trial depends on trial protocols being followed exactly. Some people consider this rigidity inconvenient so it could be considered a cost.

The reward stage for a researcher often comes well after the trial itself, when results have been documented, presented and ideally published. That’s what leads to promotion and more grant funding for researchers. But nobody will publish results that are already in public domain. So, another cost might be the requirement for a participant to wait a frustratingly long time before knowing if their own results were representative and meaningful.

The website https://clinicaltrials.gov/, hosted in the US, lists clinical trials throughout the world, planned, recruiting, underway and completed. It’s a searchable database, so this link brings a list of studies related to FA.

When I last checked, the site listed 56 studies. The simple fact that studies are listed is progress already. As learnings are gained from each of those studies, that progress continues towards finding treatments, and even a cure for FA. Any of us who gets to contribute to that progress should seize and appreciate that opportunity, recognise its value and treat it with the immense respect it deserves.

Terry O’Hanlon
fara Ambassador

 

What goes up, must come down

Tan…done!

Hair and makeup…booked!

Dress…hired!

Botox…topped up! (Don’t judge me. If it was in my calves for movement or in my bladder for control, you wouldn’t judge!)

Shoes x4…organised! (I’m not even going to go into that!)

Tomorrow night is the annual fara Gala Ball in Melbourne. This night is my favourite night of the year. It is a night when FA is recognised, celebrated, tears are shed, money is raised and hope is heightened.

Everyone in the room is lovely, understanding, non judgemental, kind, giving…all of those nice words.

It is the one night where I feel a million bucks. No one stares at me because I may look a little different. No one judges me because I talk a little slower (especially after champagne). No one thinks it’s odd that I can’t hear very well in such a noisy environment. Actually let’s rephrase that…I DON’T JUDGE MYSELF!

The line for the disabled toilet is huge and I love it! Gives me a chance to catch up with all of my heroes.

I smile all night. Go to bed smiling. Wake up smiling.

Then I go home.

Home to routine.

My body needs routine to function so I’m not saying it’s a bad thing.

The gala is on the Friday night. On the Monday, I cry and cry and cry some more.

I don’t want to have FA. I don’t want to use a walker. I don’t want to fall over. I don’t want to go to appointments every day. I don’t want to have to calculate and concentrate on every move I make, every word I speak, every single thing I do really. I don’t want my children to have a  mum with FA. I don’t want my husband to have a wife with FA. I don’t want to be scared of the future.

By Thursday, I should be fine again. Ready to take on the world. 

It’s so true though. What goes up, must come down. In my case, crashing down.

Leah Alstin
fara Ambassador

More than lazy

More than lazy

Would you believe you were lazy if you grew up hearing it all the time?

As an eight year old I recall the sunny days with my family walking the dog down to the beach at the end of our street. A casual family outing, normally involving a pit-stop at the local petrol station to buy ice-creams; Gaytime for Dad, Calypso for Mum, two double-choc Cornettos for my little sister and me.

A virtuous cycle

A virtuous cycle

“If you need something done, ask a busy person” they say. And they’re right. You see, what keeps busy people busy is that they’re getting things done. Add the thing you need done and it joins their list. They’ll get it done too.

Or you could set out to start getting things done yourself. Start with small things. Once you set out to accomplish one thing, as soon as it’s accomplished you want to tackle another. It’s contagious. Add the thing you needed done, and you’ll get that done too.

It's only a phone. It lets me be smart!

It's only a phone.  It lets me be smart!

According to Deloitte, smartphone penetration in Australia is at almost the highest level in the world, at 88% in 2017. They're so useful I reckon penetration among FAers should be 100%.

Here are just three apps I have on my phone that are particularly useful considering that I have FA (there are lots of different versions of each of these you should be able to find one that's free):

Do you like big buts? Well this one’s huge!

Do you like big buts? Well this one’s huge!

In the many years since diagnosis, how I describe FA has become pretty standard: with resignation. People would enthuse to me back then about how fast progress was being made in medical research and assure me how convinced they were that there would be a breakthrough in FA that I’d benefit from very soon. As time went on though and my FA progressed, it’s felt appropriate to become stoic. I’m a big supporter of FA research, but I became increasingly convinced that it would benefit FAers much younger than I when a breakthrough was eventually made.

Spiritual Healing

Spiritual Healing

I am at a stage in my acceptance of Friedreich’s Ataxia, where I am not always looking for a cure (although, this would be handy!). Rather, if something pops up in my life, which may better my wellbeing, I jump at it. For example, I have started doing a form of physical therapy called Bobath. It is by no means a cure for FA, but it might just get my body moving more effectively. This may mean a decrease in falls & pain. No, it’s not a cure, but it’s definitely a win.

Finding Hope Amidst a Debilitating Disability

Finding Hope Amidst a Debilitating Disability

The diagnosis

I was a happy, healthy child living on the Gold Coast, Australia when my sister, who is 18 months older than me, started having trouble walking. We would regularly walk the few blocks from our house to the Broadwater and her unsteady gait became more and more obvious. My mum, determined to get to the bottom of it, took her to several doctors. When I was 10 years old the diagnosis was confirmed and my parents were devastated.

A view on meteorology from a physicist

A view on meteorology from a physicist

Neils Bohr, a Danish physicist, is reputed to have said “It’s difficult to make predictions, especially about the future”. He was one of the smartest men to ever have lived. I guess he had an enormous respect for weathermen!

Well for us FAers, predictions are what we seek most in the world. It’s not hard to find out the list of things that’ll go wrong with us. What is hard though is getting an idea of when.