Right to Try

My view on Right to Try:  PRO Right to Try.

This post was not made because of any political view at all. I hope it doesn’t get caught up in the political climate that is out there.

I never want anyone to have to go through what I have been through! I have been on different sides of this debate but when you don’t get approved for 5 clinical trials ad Compassionate Use wasn’t so Compassionate to me. I also hope that nobody has to sit in the doctor’s office and hear “I am so sorry but we don’t have any other medicines for you, like I have done. The words you are terminal is something I never wish on anybody!

I am writing this so hopefully nobody else has to hear I am so sorry but because you have 3 very active diseases I doubt any experimental medicines will work on you. So as you can see I am not for Right to Try for me. I am fighting so there wont be too many more like me! I am also not writing  this for pity, NOT AT ALL!!! I want people to hear and ACTUALLY LISTEN to what a patient has to say!!! Stop thinking you all know better for me then I do!!!

I am Pro for many reasons.
My first reason is that I don’t believe that someone who doesn’t know me should decide whether or not I should be able to try a clinical trial or drug that may have a chance to save a life of a patient especially those that are terminally ill. When there is a chance to help someone who is terminally ill they should be able to. The way that clinical trials go now they can take up to 7+ years just to start a clinical trial that patients are involved. Most terminally ill and chronic patients don’t have that much time to wait.
Another reason I am for it is due to my history with the system . When doing a clinical trial right now the pharmaceutical companies are only going to take the perfect person who fits the trial. So many people are denied because they don’t fit the criteria because the pharmaceutical companies need the best results so the FDA will approve the drug. With the Right to Try bill terminally ill patients have the chance to try the same medicines that are in the trials, first of all earlier, they can get into the trial after the FDA approves the first level instead of waiting for the third FDA level of approval.
Another great part of this bill is that if a terminally ill patient goes into a trial it doesn’t affect the trial results.
Why I feel that this will help the Sarcoidosis community is because there are only two approved drugs for Sarcoidosis, prednisone and acthar gel. Neither is a cure and at best only suppresses the inflammation plus the side effects of those medicines are almost as bad as the medicine itself. Also because Sarcoidosis attacks any part of the body the two approved medicines don’t help all those parts. In the past 18 months over 50 Sarcoidosis patients have died. If they were allowed to decide if they wanted to try a clinical trial drug for Sarcoidosis, maybe some of those patients might not have died. Even if it saved one person then I believe that it is worth it.
I know that other Rare Disease Organizations were against this bill because they believed that it would give people false hope. I understand that but I feel they are underestimating the patients, doctors and the families. I believe that each patient should know the risks of each trial and either the patient or the family or caregiver should have the chance to say yes or no. Even if they make a rush decision that is up to the patient who is terminally ill. Also when a patient is terminally ill they don’t have many or any other choices. Are they suppose to just give up? I am a terminally ill patient and I refuse to give up.
The medicines that are being offered after the first level clinical trials still have to be approved by the FDA to get to the next level. So these drugs or treatments are at least initially approved by the FDA.
To wrap up I know as a terminally ill patient that I would investigate the treatment first to see if the benefits will outweigh the side effects or negatives. If I feel that it wouldn’t help me I wouldn’t use it and I truly hope that the patients, caregivers and most importantly the doctors research any trial medication before giving it to a patient.

I have always said it is never too late to have HOPE!
Personally I have a wife and daughter to live for. My daughter is turning 16 in December and I want to see her sweet sixteen, her graduation, go to college and see her get married. I will not just roll up into a ball and give up. I have always been a fighter and always will be.

Here is an article that I am in on my stance on Right To Try:  https://alsnewstoday.com/2018/06/04/rare-disease-advocates-line-up-to-both-praise-and-criticize-us-right-to-try-law/?amp

Here is my NBC NIGHTLY NEWS INTERVIEW on Right to Try:

https://www.nbcnews.com/nightly-news/video/-right-to-try-bill-advances-would-allow-some-patients-access-to-experimental-treatments-1242943555808

 

Advertisements

My Story Got Published!!

Just wanted to let you all know I actually got a story that was amazingly written by Nate Davis and was published by a website called Thrive Global, who is none other run by Arianna Huffington!! If you don’t know who she is check out The Huffington Post!!

You don’t know what this means to get my story out there for all of the rare disease community! I am beyond happy!

I have been through so many rough times in the past 6-8 months that I really want to make sure we get our story told and raise awareness for the whole rare disease community and especially for the Adult Rare Disease Community! We need our stories to be heard too!!

Here is the link to the story:

https://www.thriveglobal.com/stories/25259-my-life-as-a-rare-disease-patient

My life in a nutshell.

Today I woke up early. I wake up early these days not because I have to but because my body says so. Today is because of the pain.

But you know what? I am very thankful that I get to wake up! You see, somebody with a rare disease like myself can’t and don’t take waking up for granted.

I am so blessed to be here! I realize I wake up in pain everyday and can’t do so many things that others do take for granted, but I am here and I am still a force to be reckoned with! I have a voice! I will make sure people will hear me. Some may not like what I have to say but I speak so others can understand what it is like for a rare disease patient like me to live each day. I also speak for many others who can’t speak up for themselves.

I took some time to do a little recouping and soul searching recently. I realized a some things lately.

One thing I realized is that no matter what I am destined to be me. You may say well of course you are. Well sometimes we forget what it is to be ourselves. I know who I am. I am someone who cares, sometimes too much, about others and myself. I need to be me. I need to do what I do the way I do it. I can’t be someone else who people want me to be.

Just know that the person I am may not be to everyone’s taste, but I am really here for the greater good for the Sarcoidosis and Rare Disease Community. I am in it for the whole not for me.

I have said in past blog posts that I most likely won’t be here for a cure for me, but I am here for a cure and to make a difference for so many others that have a rare disease.

I really am not a person who thinks I am better than anyone else, as a matter of fact I truly believe the opposite. Ask many that know me, I really don’t know why people even know who I am.

All I am here to do is to make sure WE as a Rare Disease Community have a voice. I am one of so many people who try to make sure we aren’t forgotten.

I want one day when the over 7000 rare diseases have a cure not only 5%.

I hope to see it and get to say I had a say in that! I want to be able to say I made a difference.

So these are my thoughts when I wake up at 3am in the morning.

I hope I make a difference in your life! Many of you have made a HUGE difference in mine!

My thoughts as a follow up to What about Us?

Hey everyone I know I wrote a blog yesterday that may have caused waves among the Rare Disease Community…

Do I regret it?? ABSOLUTELY NOT!!

Will I stop talking about it?? If you think YES, you don’t know me by now!

I have been thinking more about this today. I am not against anyone at all! I am not against that organizations have said that 50% of Rare Disease Patients are children.

I just want to not be forgotten. I also want to make sure that in our community there isn’t a feeling of separation.

I know we don’t discuss this often. I FEEL WE NEED TO AS A COMMUNITY!

Why shouldn’t we discuss this as a Rare Disease Community?

Why should I or anyone else be afraid to discuss this or feel sorry if we bring up an adult that has a rare disease?

I really have been thinking about this so much lately. I guess partly because as an adult with Sarcoidosis, Sjogren’s, and Parkinson’s I shouldn’t be ashamed. I am not saying anyone has made me feel this way personally, but I always have felt that this has been the elephant in the room.

I am not here to pull apart the Rare Disease community. I am here to bring the community together. If 50% of the rare disease community are children the other 50% are adults. We are all part of the community! I never had a thought on my mind that I was advocating for a child or an adult. I was advocating for the 30 million rare disease patients.

What actually had me write the blog yesterday was not just seeing the Today Show spot yesterday morning but also just laying here as usual in so much pain and thinking how much as an adult patient people don’t realize what we go through also. Also being told by some TV and newspapers and I quote “That there isn’t an interest in knowing about an adult patient.” Are you kidding me??? Also when interviewed I have been asked on multiple occasions “How many Children have your disease?”

So then I think.. What are we doing as a Rare Disease Community to help raise Awareness for adults with Rare Diseases? I realized NOT ENOUGH!

It is not fair for anyone to have a rare disease! I know life isn’t “FAIR.” But to be pushed away from everyone and then to feel like this is a taboo subject is ridiculous.

We as a community shouldn’t have any taboo subjects!

I really started thinking of all of the conferences, all of the government officials I have talked to, all of the rare disease community events, and also all of the television spots about rare diseases we mainly hear the numbers of children. Not that it is wrong, but what about us as an adult community.

I have been thinking about this for months. I decided to speak up now because with my health getting tougher to deal with and with so much to look forward to like moving forward as an organization President and also on a personal level. Knowing that I most likely won’t see a cure for any of my diseases is really so tough to get through my mind.

I am not here to make you feel sorry about me at all.

I am just talking about how I feel as an adult rare disease patient.

I don’t know how it is to be a rare disease parent and hopefully never will.

I do know how hard it is as a patient and do know hard it is on my family and worrying if I may pass it down to my daughter since we aren’t sure if it is genetic or not yet! I also understand on how hard it is to raise a family on a very limited budget due to my disease. I also know what it is like to see the ones you love worry everyday about me!

I also know what it is like to be looked at like there is nothing wrong with you! To be told you look great! To be drug tested when I go to the hospital! To be told there is nothing wrong from emergency room doctors!

The worse parts of these diseases are:

To be looked at from people you know and think that I am faking it!

To be worried what people think of you!

To be told by specialists that there is nothing else we can do for you!

To be in pain EVERY SINGLE DAY!!

I am an adult patient advocate!

I am an adult rare disease patient first and foremost!

Rare Diseases… What about Us?

So here I am laying down thinking to myself why? So many whys to ask about…

Why do I have a rare disease that nobody can treat?

Why am I in so much pain everyday?

Why is the “Rare” disease community still not being respected?

Why don’t 30 million Americans count??

And today I am going to talk about a very tough topic…

WHY ARE THE 50% OF RARE DISEASE PATIENTS THAT ARE ADULTS ARE  HARDLY EVER TALKED ABOUT? 

Let me set this disclaimer out there first.. This is only my opinion! Nobody else or any organization has anything to do about this post. Also I am not diminishing the 50% of the children that have rare diseases. I also understand it hits the heartstrings of people when you talk about children.

But let me tell you from an adult perspective of having a rare disease called Sarcoidosis.

I was laying down today watching the Today Show and saw a great clip by Richard Engel about how the Tax Reform Cuts will affect the Rare Disease Community, especially the children. I wanted to thank Peter Saltonstall, President/CEO from NORD and Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical Inc. for expressing their opinions and supporting the rare disease community.

Okay so now I am going to talk to you from my adult patient perspective…

I cried watching that clip on the Today Show! There were many thoughts going through my mind! First I watched and felt the pain of those children and also the parents.

Then came the other reason I was crying… I looked at that piece and so many others like that and they all have one thing in common… NO ADULTS DEALING WITH RARE DISEASES ARE BEING SHOWN. WHY???

I was crying because I lay there in pain that I wouldn’t wish on my worse enemy! I lay there because if I move too much I don’t know if I am going to pass out. I lay there because my pain level is at an 8-9 level from 1-10. I also lay there knowing that being a realist that I will never see a cure in my lifetime, but yet I fight for the rare disease community to possibly make it better for someone else.

Adults with a rare disease are a different breed. Either we were having and living a normal life then all of a sudden our lives changed in an instant… We didn’t ask for this.. We get looked at like we are lazy, looking for attention or just making things up. We deal with trying to either work if we can or have to because we can’t get disability or even if we do we can’t live on it.

We also have the pressures of trying to have a support system, finding someone who will support us mentally and sometimes even physically and financially.

Someone like me that has been the hardest thing for me! I want to work! I can’t work! I went from a runner to someone who has had 9 surgeries in 7 years. I want to be able to support my family but I can’t. I have an amazing wife who I see has to deal with the everyday pressures of being the bread winner and then also being a Mom and worse yet  wondering everyday what is going to happen to me! People don’t realize being an adult with a rare disease is so much pressure on your significant other as well as your child. I can’t do things a normal Dad and husband can do. I try my best, but the reality is that I wouldn’t be here if it wasn’t for my wife and my daughter and I will never be able to do what other Fathers do!

I also live with the fact, being a realist, that I will never see a cure in my lifetime…. let me talk to you all about that!!

Does anyone who is not a patient understand that??? Really, I know my life is not going to get better and most likely will get worse. I know that sounds terrible but it is a FACT! Imagine knowing that the pain I feel today will never go away. Imagine being old enough to realize this but too young to die! Like I said I never know how I am going to be everyday.

Imagine waking up to a phone call from your cardiologist saying “I called an ambulance for you!” “Your heart stopped twice while sleeping!” Also imagine waking up on the floor not knowing how you got there. That is my life, EVERYDAY!

I didn’t write this just to complain about me….

So here I am laying down wondering why don’t people talk about adults with Rare Diseases?? I get upset hearing well you lived your life!! Excuse me??? When is 50 a whole life??? I am grateful that I have made it to 50, but to say that I lived a full life is one of the most insensitive things I have ever heard!!

Like I said before I understand that people will look at children and feel real bad. And as I said before I don’t mean anything bad about the 50% of rare disease patients are children.

Please do remember that the other 50% are young adults and adults. We deserve respect just as much as anyone else! We suffer too! Our Family suffers too!  We have to make dramatic life changes just to stay alive! We live day to day too!!

So when I see something about Rare Disease patients why can’t we get a point of view from both adults and children??? Is that really too much to ask???

As I write this I am crying, yes I am a man who cries and not afraid to say it, because knowing that I might upset those in the Rare Disease community, either parents of children or others who just like to get on me for things I say or do.

I want people to realize that their is another side to this community…. Please don’t forget about us ADULTS!!!

 

 

 

 

Today… What does one of those days mean to you??

I AM TRYING!

Well let me tell you what “One of those days” mean to me….

Today sadness has been my friend. Depressed that my life is changing. I know I shouldn’t be putting this on my blog. But this is part of life… MY LIFE!

Life is always changing… Well change is not always easy too! I am adapting to the NEW ME!

Will I be able to accept this I don’t know. I do know I have said I am adaptable, but I need to really look to my inner self and say “Are you ready to adapt?”

I think I am…. I hope I am!!

I love life even with these diseases I have. I learned to love life!

Just been very hard to know you want to do things but aren’t physically able to do them.

I have taken many steps back, for the better of my health. It has been the hardest decisions I have ever made since finding out I have Sarcoidosis…

I am fighting an inner battle to figure out my strength. I know it is there just have to find it again!

Just know I am always with the Rare Disease Community and will pick my battles. If I do step back from certain events or fights it is only because I need to get better.

I need to have an internal peace… I can honestly say I don’t know what that is! But I am looking for ways to get there.

I will leave it with this:

My new phrase is: ” I AM SCARED, BUT I AM TRYING!” That is all I know how to do!

Just a Day in the life of A Rare Disease patient.

A couple of phrases you never want to hear:

We can’t figure out what is wrong with you.

There is nothing we can do for you.

This is the worse thing to hear:

Just Deal With It!

Well here it is President’s Day. Let us celebrate! Well for me it is another day of extreme pain with no answers in sight.

What did I do to celebrate the holiday? I have been in bed all day barely wanting to eat and drinking Gatorade so I don’t dehydrate anymore than I am.

Everyone says to me go to the hospital… go to the doctor… I have tried that. Nobody has an answer.

I was in the hospital 2 weeks ago for 4 days. What did they say? “We don’t know why you are dizzy, we don’t know why you are having TIAs.

So in other words you just have to deal with it.

What a way to live!

This is something many Rare Disease patients deal with.

I am sick, tired, and wondering what is next for me!

Yes it gets depressing! Everyday it is a new challenge.

But when does your body want to say enough is enough??

I am not sure when that is but I know mentally this is so draining which then affects your body even more.

I am writing this to let you know that I am trying! That is all I can do! I won’t say I am fighting! I am trying!

I don’t ask for pity! Never had never will! I am just writing this to show you that the lows are lows. The highs are highs.

I always put up a brave face, say I am doing fine! But guess what???

I am not fine!