Email Response #2

All-

I started this morning at 4:45 a.m., first answering emails to patients and their families.  I noticed a remarkable number of emails coming from Seattle, Washington; hence, I thought I would send a more lengthy reply to the Seattle Mito Family as a group.

First- thank you. Your vocal support and advocacy is great and well-needed, exactly what I would do. It is the squeaky wheel that gets the oil, and it was exactly that type of passion that resulted in Edison Pharmaceuticals being started as a patient- and physician-founded company. "We cannot and will not wait in line for a treatment– period!" I hear and applaud your message.

Second, on to your question as to the delay at Seattle. Simple truth is that we never expected EPI-743 to exhibit the promising results, albeit preliminary ones, that have been generated to date. We initiated a very closely watched and controlled treatment in one little girl ~ 2 years ago, and from that we are now at the request of FDA treating close to 85 children worldwide with a variety of inherited respiratory chain diseases. Dr. Enns - who is the principal investigator of this study - has presented some of the results at the UMDF meeting with our extended EPI-743 team. This data has just been tallied and submitted for publication.

Edison has been working very closely with the FDA, physicians, and hospital administrators and ethics review boards to expand access to EPI-743. There are three aspects that must be addressed to make this a success. We must have in place the appropriate safety guidelines set forth by the FDA and oversight at each institution; we must manufacture drug according to very strict specifications; and we must agree on budgets at each institution and contracts that are lengthy and unfortunately expensive.

As many of you know Edison is not a typical pharmaceutical company. Our mission is defined by our tag line--- "more tomorrows." We were founded by patient families who were/are blessed to have the financial resources to fund such an endeavor, and today we continue to only "invite" investors who share our singular focus… treatment for "our kids." In the last weeks, we have raised a considerable amount of money to fund studies in Seattle and worldwide, and we are working with FDA as recently as today is gaining their go-ahead to expedite treatment and pivotal clinical trials hopefully paving the way for a 1st approved drug for mito disease. We will not stop our mission there- we will work until we exhaust all of our talents and resources, and until all mito kids and adults have the potential for clinically meaningful treatment. 

We will not hide behind any excuses at Edison- too much is at stake. We know we can never move as fast as not only we want, but as you and your doctors and families want. The Seattle Children's group has been tremendous, but the fact is each institution has their polices and procedures, as so does Edison, and we both must respect them, and navigate them, and come to an agreement on all aspects of oversight, review and budget before we can begin treating patients.

As of today's discussion with administrators at Seattle Children's we have an agreed upon budget, and ALL roadblocks from Edison's side have been resolved. Next steps are for Seattle Children's hospital and Dr. Saneto's team to mobilize - then  a first subject can start. We are awaiting a response from Seattle Children's on their time estimates for this process. I have copied Lorraine Gilmore and will ask her to schedule a group conference call next Monday so I can field any open questions folks may have.

Thank you again for your advocacy and vocal championing of your children. We will need this and very much more to be successful. 

Best/g

Guy Miller, MD, PhD
Chairman, CEO
Edison Pharmaceuticals, Inc







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A great big THANKS to all who joined me in writing a letter.
I even got emails from people that don't have mito in their families, but felt compelled to help anyways. I appreciate each and every one of you who took the time to help us out in this matter. I guess we will see what happens here in Seattle....



********

Just got this from Childrens's after I forwarded Dr. Millers response....



Ohmygosh, Girl, I think you did it!!!!!  I mean, it’s not just “coincidence” that they decide this today after all the letters from here!
 
Russ is going to “mobilize his team”!!  Stay tuned!!
 




I did get a follow up email that Dr. S mobilizing his team will take a little time, but sounds like we definitely got things pushed ahead! Wahhhoooo!!!! This is such great news!

Email Response #1

I have to say...
I am impressed with the quick response, although it should be quickly addressed, so many times you sit and wait for a response.

Here we go...



Debbie

Dr. Miller here- I am the CEO of Edison. Thank you for your email below.
We have a call with the Seattle Children's hospital today to hopefully
finalize getting started.

We are 100% supportive of having EPI-743 available at Seattle Children's.
You can be certain of this. You need not in any way convince Edison or
myself of urgency or petition on behalf of your son or others. We were
founded by physicians and parents of children with mito disease with one
goal clinically meaningful treatment. While I will never second guess
what it is like to have a child with mito disease, I can tell you we are
heart felt in our mission.

Emails like this and others focus our attention even more and that is a
good thing. I will email you back after our call today and set
expectations on both the going forward process and time lines.

Thank you so much for taking the time to get in touch with us.

Best regards/g

Guy Miller, MD, PhD
Chairman, CEO
Edison Pharmaceuticals, Inc

My Washington and surrounding mito families ....

You've got homework....

We need to put the "heat" under Edison Pharmacueticala, to push forward EPI-743 so it is available at Seattle Children's Hospital TODAY!!!
Children's has done their part...there is so much frustration because it should be here and it is not.
I am asking you to write a letter to Guy Miller, and his assistant,Lorraine Gilmore.
gmiller@edisonpharma.com, LGilmore@edisonpharma.com

Any of my friends here, that personally know Hudson and our family, feel free to write as well....grandparents, aunts, uncles, any family members or friends, ANYONE who feels compelled to write, go for it!!!
Thanks!

Here. Is a copy of my letter:


Dear Mr. Miller,
Dear Ms. Gilmore,

I am writing in regards to my son Hudson, and the study EPI-743.
Hudson suffers from Mitochondrial disease, and is one of those individuals who it attacks almost every part of his body in catastrophic ways.

We have been waiting patiently, praying this study drug would become available at Seattle Children's Hospital, in hopes that Hudson would be able to participate.
We need hope. At this time, as you know, there is no cure for this catastrophic disease that so many children suffer from, EPI-743 gives hope. Hope for a change for the better in the lives of children who desperately need help with a disease that gives no hope.

I am not sure why it is still not available at Seattle Children's. What I do know, is time is running out for so many children, the clock is ticking and we need this to be an option NOW for our precious babies. I say "our" because I am connected to other Mito families here in Seattle, that are sitting, waiting patiently like myself. We wait, we wonder if this could be the drug that makes a difference in the quality of our child's life, we wonder how we could scrape enough money and find the time to put our lives on hold and fly our kids to another hospital, in another state to give our child this option.
For me it is impossible. I am single parenting three children, I can not scrape the money or put my life on hold....this needs to be available here, in Seattle, where Dr. Saneto knows Hudson personally and all his struggles and history with this disease.

I do not want to be one of those parents left to wonder if this drug would have helped my child.
Whatever the road block, whatever is causing this to be in a holding pattern and making it not available today at Seattle Children's, I am personally asking things to be feverishly pushed forward NOW. There is no time time to waste.
If you were to look in the eyes of my very sick Hudson, you would know he and all of his mito buddies here, are worth putting forth the extra effort to make this available.
I am a mom trying to do the very best for my son, please do what you can to help me do that.

Thanks for your time,

Debbie

Humbled

I am so humbled by so many who have crossed our path on this journey with Hudson.
Not everyone has been on the mito path...some started this journey with us as we unraveled why Hudson was having infantile spasms, without finding a structural, or tangible cause.
There are so many pieces of this journey that you can dissect and then actually relate to people with whom you have never met.

As we close out what has been labeled Mitochondrial Disease Awareness Week, I stumbled across my heart connected friend's blog tonight,with whom I have not physically met, but who gets me on so many levels.
I am teary eyed at her post...
Because, even though her son is not faced with this disease, she has been touched by my sons face, and the mighty weight of this disease and what it means to our family.
She has listened to my daily plea this week, to spread the word, and so she posted on her blog my sons face and the face of mito.

Thank you D....my heart thanks you, my mito baby boy thanks you....I am so humbled to be on this journey with you, even though are paths are so incredibly different....

Blessings to you my friend...thank you for not only hearing my plea but feeling it....




And so here is most of her post that I copy and pasted...to see it in full click on side bar of my blog list...happy being trevy.(because as an iPad post I am pretty limited)







for my Mito mom friend
 

Infantile Spasms is a diagnosis. 

 

 

And a marker.

 

 

Or symptom…if you will.

 

 

A symptom of something else.  Because once you enter Infantile Spasms Ville you quickly learn that seizures in and of themselves are not the final diagnosis…they are a symptom.  Of an underlying cause.  And in the world of Infantile Spasms there are many underlying causes.  Each underlying condition a spectrum.

 

 

Spectrum according to Dictionary.com:

 

  a broad range of varied but related ideas or objects, the individual features of which tend to overlap

 

 

You know.  The same but different. 

 

 

Each IS baby has a unique journey.  And those that share the same Underlying (spectrum) Condition do too.  Each Cortical Dysplasia child.  Or baby who had a neonatal stroke.  Or has Tuberous Sclerosis.  Or Dravets .  Or Mito…

 

 

(to name a few)

 

 

All have unique paths that unfold.

 

 

One thing that rings true for all (that I’m aware of) IS children and their specific underlying conditions…

 

 

There is NO cure

 

 

There are miracles here and there. Some children respond quickly to treatment and move forward without scars until Infantile Spasms is but a distant memory.  That maybe you had a nightmare once-d.  But the majority of families move forward into a journey that…while it brings it’s own sense of joy because our children do bring joy…is much different than the one we ever imagined they would travel.  Much harder.  Much darker.  Much more medically complex.  Drug and therapy saturated.  Tear and prayer drenched.  Than we could have imagined.

 

 

And we live each day trying desperately to love our children to the best holistic health humanly possible. 

 

 

And we spend our free time dreaming about the day there will be a Cure. 

 

 

This week is Mitochondrial Awareness Week. 

 

 

And while Trevy’s underlying condition is not Mito…

 

 

he does have a sweet little (arranged) friend on the other side of the country who does.  And his friend has an extraordinary mommy…to whom my heart has connected. 

 

 

And today…I’m praying and dreaming for Hudson. 

 

 



 

 

And this post is my way of helping create more awareness for all the children and their families courageously living with Mitochondrial Disease.  

 

 

XOXO Hudson & Mommy

 

 

…danielle























for my Mito mom friend
 

Infantile Spasms is a diagnosis. 

 

 

And a marker.

 

 

Or symptom…if you will.

 

 

A symptom of something else.  Because once you enter Infantile Spasms Ville you quickly learn that seizures in and of themselves are not the final diagnosis…they are a symptom.  Of an underlying cause.  And in the world of Infantile Spasms there are many underlying causes.  Each underlying condition a spectrum.

 

 

Spectrum according to Dictionary.com:

 

  a broad range of varied but related ideas or objects, the individual features of which tend to overlap

 

 

You know.  The same but different. 

 

 

Each IS baby has a unique journey.  And those that share the same Underlying (spectrum) Condition do too.  Each Cortical Dysplasia child.  Or baby who had a neonatal stroke.  Or has Tuberous Sclerosis.  Or Dravets .  Or Mito…

 

 

(to name a few)

 

 

All have unique paths that unfold.

 

 

One thing that rings true for all (that I’m aware of) IS children and their specific underlying conditions…

 

 

There is NO cure

 

 

There are miracles here and there. Some children respond quickly to treatment and move forward without scars until Infantile Spasms is but a distant memory.  That maybe you had a nightmare once-d.  But the majority of families move forward into a journey that…while it brings it’s own sense of joy because our children do bring joy…is much different than the one we ever imagined they would travel.  Much harder.  Much darker.  Much more medically complex.  Drug and therapy saturated.  Tear and prayer drenched.  Than we could have imagined.

 

 

And we live each day trying desperately to love our children to the best holistic health humanly possible. 

 

 

And we spend our free time dreaming about the day there will be a Cure. 

 

 

This week is Mitochondrial Awareness Week. 

 

 

And while Trevy’s underlying condition is not Mito…

 

 

he does have a sweet little (arranged) friend on the other side of the country who does.  And his friend has an extraordinary mommy…to whom my heart has connected. 

 

 

And today…I’m praying and dreaming for Hudson. 

 

 



 

 

And this post is my way of helping create more awareness for all the children and their families courageously living with Mitochondrial Disease.  

 

 

XOXO Hudson & Mommy

 

 

…danielle