Practice English Speaking&Listening with: BabelFAmily's interview with Ron Bartek (FARA) - part 3 of 7
Transcription by Marisa Condurso de Nohara Subtitles by Krešimir Babic and Hasan Çiçek
This is a wonderful illustration of how collaborative we've all been.
A funding for the HDAC inhibitors program has come from
FARA, from GoFAR here in Italy,
from the Muscular Dystrophy Association,
from the National Institutes of Health in the US.
And if you look at the work in the mice
of the HDAC inhibitor that has been supported by Ataxia UK and other associations.
It's been a widely collaborative effort around the world.
There is a new drug that we haven't said much about yet,
because it's so new!
It's from the same company - Edison Pharmaceuticals -
they gave us A0001 recently.
This is in the same class of molecules as other mitochondrial agents,
like Idebenone, CoQ10, A0001.
It has already been approved for emergency treatments of very ill patients.
For the most part, once they have been released to hospice care,
and given 30 days to live, for example, in other indications... not FA.
So, the second drug I'd like to tell you about that is coming into our pipeline,
is called EPI-743 - Edison Pharmaceutical Inc. 743.
It comes to us from the same pharmaceutical company that gave us A0001.
It's a very promising new drug and is in the same category, the same class of molecules,
as the other mitochondrial agents like CoQ10, Idebenone, A0001.
It's already been approved for emergency treatments of very sick patients,
in other indications that are related to FA,
like Leber's hereditary optic neuropathy, Leigh syndrome, or SURF-1 mutation.
GP: ...it's already tested on humans.
RB: It's not being tested; it's being administered.
It's being used as a treatment on an emergency basis.
Let's say for example, there are 2 children with Leigh syndrome,
and the FDA said they were being released to their hospice care, giving 30 days to live.
The first little girl that was given 743 was in that category.
Instead of 30 days she lived three quarters of a year.
And not only live longer, but with a considerably higher quality of life.
She was initially a rag doll and her mother had to hold her,
so that she would sit up for the camera,
and just a few months later, with 743 being given to her,
she was holding onto her mother's fingers as she was trying to walk down the hall.
So, this is a phenomenal and remarkable improvement this little girl,
whose smile would win your heart.
She looked up into the camera trying to walk.
She was just beaming.
Unfortunately, she did not survive the year,
but her family was very grateful for that year of her life
at such an improved quality of life.
And the presumption is that, had she been given that drug earlier
- in her experience - before all that damage was done,
that it may have been even more profoundly therapeutic.
So, that drug has been in several patients with that disease, Leigh syndrome.
It's also been in a handful of patients with Leber's hereditary optic neuropathy,
in which vision goes very quickly...
they go from full vision to blindness in a very short order,
and in cases where 743 was administered, there are a couple of cases
in which vision has returned sufficiently for the patient to return to work.
So it's now being in one very ill FA patient
and the preliminary results are encouraging.
There's some return after just weeks on the drug, sort of colour capability in vision
much more energy, sleeping through the night without difficulties,
and speech improvement, that's remarkable!
So we are very encouraged by this.
MariLuz (ML): And you said she was blind before taking this drug.
RB: Before taking this drug, she was legally blind.
She could not see anything. Now, she can differentiate colours.
This is not a scientific analysis.
The data is not in yet, but from the parents and friends and the neurologists
they are all reporting that there's some recognition of colour now,
but her speech is recognizable by the speech therapist who didn't even know she was on a drug, asked :
"What's going on with this little girl, that her speech is vastly improved, and I can have a conversation?"
ML: Is this drug going to be tested in more patients?
RB: The FDA has approved it for trial in Friedreich's ataxia, Leber's hereditary optic neuropathy, and dominant optic atrophy.
We hope together to full up Phase II clinical trial going later this year.
ML: Yes. And this clinical trial, is it going to be only in the USA or also in Europe?
RB: Initially, it will probably be done just in the US, just the Phase II,
it will be fairly short, almost a proof-of-principle, a proof-of-concept trial.
If it shows promise in the Phase II in the US, I'm sure it would be spread remarkably.
ML: Because this drug is already in the market. I mean, if it is or not.
RB: No, no. This is not available.
ML: Is it not on the counter?
RB: No. It is not available anywhere. This is an experimental drug
ML: It is not available even for other diseases?
RB: Absolutely. This is experimental in all diseases. ML: In all diseases!
RB: This is a discovery of Edison Pharmaceuticals.
RB: Right now, it's available only from Edison pharmaceutical, and it's totally, completely an experimental drug.
And it is approved only by the FDA for emergency treatment in those three diseases.
GP: As a last option.
ML: And only with a limited number of patients.
RB: Right. The ones that are specifically approved. It's been tested in only about five or six people.
GP: If it shows to be promising, it would be interesting to incorporate this candidate drug in the EFACTS project, I think.
RB: Absolutely. And then the clinical network will be involved, I am sure.
ML: Is it an antioxidant?
RB: It's a mitochondrial agent.
We've always referred to antioxidants but it appears that these drugs are able to do more than anti-oxidation.
And that's great! Maybe, they are able to help transfer electrons to the mitochondrial membrane;
maybe they increase our energy levels and decrease oxidative stress.
So, I prefer to call them mitochondrial agents.
ML: And the mechanism is not clear yet?
RB: It is not entirely clear yet.
It seems to go beyond. We initially thought Idebenone, for example, was a pure antioxidant that would just sop up;
it would bind oxidative radicals - was just not true! It proved to be a mitochondrial agent that goes beyond that.
The fact is that we're trying to use these agents to strike upstream of the exit
prevent the damage by increasing mitochondrial energy production by helping transport electrons
so we get more energy and don't get as many radicals.
So, instead of sopping them up, addressing them later,
we're hoping these mitochondrial agents prevent the formation of so many radicals.
