Catherine Jayaysuriya
Dusty’s Mom
Coalition Duchenne
Founder and Executive Director
Monday, April 25th's Advisory Committee meeting felt more like a cross examination in a criminal case than a
fair discussion about the efficacy of a promising treatment for Duchenne. As the clinical data on eteplirsen was
scrutinized, minor details became exaggerated, such as doubts on validity of the 6 MWT, and ignorant
opinions such that boys with Duchenne can walk if they put their minds to it, to
be the same level as key facts. I question their competence to even be on the
panel.
The Advisory panel stood
in judgment of something they don’t fully understand, which is the complexity
of the Duchenne situation. They characterized the parents as
‘emotional’ which diminishes our input as we actually have the best ‘fact
based’ knowledge and first hand experience of Duchenne. And they didn’t seem convinced by the
testimony of parents, and most importantly, the boys who are on the eteplirsen
trial themselves. There is a strong need
to break away from herd mentality of bureaucracy and business as usual.
I advocate for the generation of boys
who have lived the natural progression of the disease. I feel certain that there were many on the
panel who do not understand that when Duchenne is left untreated, the
progression is cruel and relentlessly progressive.
 |
| Dusty Brandom, age 23. |
My 23-year-old son Dusty Brandom will
not benefit from eteplirsen but would benefit from the follow on drugs
targeting other exons once approval is made. Dusty has been
waiting for this treatment ever since we funded Steve Wilton's oligos work through
the Dusty Brandom Fellowship in 2004. It feels like Dusty has been hanging on
to a cliff, but now by his fingertips. By the FDA not approving, we will lose a
generation of boys including my precious son. We really are running out of
time.
 |
| Steve Wilton and me. |
The judgment made at the 11th
hour literally, felt like a series of bullets that deeply wounded our Duchenne community.
But we don’t fall easily. In spite of my sadness and disappointment, I do have
a glimmer of hope, because FDA Director Janet Woodcock, in her opening remarks,
reminded the panel of the harm that could be done by failing to approve a drug
that does work. The final judgment comes May 26th.
Thank you Senator Marco Rubio, for championing our
Duchenne cause in your speech to Congress today.
