Sunday, November 19, 2017

A Law Against Lawyers!

December 30, 2007 by  
Filed under Legal

I am posting this message to the entire world to let everyone know that Hugh and I will not sue any doctor, scientist, researcher, hospital or non-profit if anything happens to Addi and Cassi as we embark on trying experimental treatments to save them from Niemann Pick Type C disease.  No matter what happens during this journey, we assure you we will not take legal action against you and we are putting this is writing for the world to see.  We are also willing to sign any release form necessary.  Of course, this release form would need to be drawn up by lawyers!

Oh, the lawyers!  There really needs to be a law against lawyers. The entire medical system is paralyzed because everyone in the ecosystem is living in fear of litigation.  I can tell you that the medical system as it is working today is keeping children like ours from getting the types of treatments they desperately need.  It has come to the point where doctors can’t even dispense an aspirin or an antibiotic out of fear of malpractice.  There are too many bureaucrats who in turn are afraid of the lawyers and who literally want to stand in our way of getting productive treatments accomplished.  I have come to understand that there are people in the medical and research system who do not want to help us, they simply want to keep their jobs and not get sued or are interested in publishing their next scientific paper.

Then there is someone like Mike Alonso at Jones Vargas, a lawyer who has been particularly helpful in making connections for us during this most difficult time.  Talk about world-class.

And of course there is Dr. Marc Patterson, Addi and Cassi’s pediatric neurologist from the Mayo Clinic.  Every single doctor in America should take a lesson from Marc Patterson on how to care for patients and what it means to be a doctor.  Someone who you would expect would have a huge ego since he is one of the brightest people on the planet does not have one.   I have not met one person who has not said, “Marc Patterson and wonderful” in the same sentence.

Dr. Steve Walkley at Einstein University and Dr. Dan Ory at Washington University who are true collaborative research scientists, who believe in openness, data accessibility and science for the people.  These are a few of the people who keep me hopeful that even despite the lawyers and the bureaucrats and the sickness that exists throughout our medical system, there can be a new paradigm for how rare diseases like Niemann Pick Type C are treated and cured in the future.

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Santa Really Is Coming To Town!

December 23, 2007 by  
Filed under Addi and Cassi News

Childhood Alzheimer's Addi and Cassi Christmas
A telephone call from an unknown number came to our house on Friday night.  It was Mrs. Claus on the line calling to inform us that Santa would be arriving at our house from the North Pole between 5:30pm and 6:00pm on Christmas Eve to bring Addi and Cassi their Christmas presents.  This is the time Santa arrives in Reno and apparently he’s very busy so we better be prepared to take video.  Mrs. Claus also reminded us that when Santa leaves the house the girls need to go to the backyard to look for the Rudolph and the rest of the reindeer so they don’t see Kris Kringle getting into his pickup truck!

The kindness people have show us over the past few months is extraordinary to say the least. Yesterday, a friend of mine stopped by with a set of darling CureCaps made by a friend of hers who lives here in Reno.  Carol, our friend in Boston, spent two solid weeks knitting CureCaps day and night and is helping us get the word out about Addi and Cassi on the East Coast.

Our friends from Australia offered to donate their beach house and a round of golf at the Royal Melbourne Golf Club, one of the top golf courses in the world, for our fundraiser and silent auction in May 2008.   We have received pedicure donations, housecleaning service, the condo on Lake Tahoe…the donations go on and on.  Many people are offering their personal time to help us as well.  The outpouring of support on so many different levels is incredibly moving and keeps us focused on our goal to conquer cholesterol and beat NPC.

A number of checks have also come to the house to the Addi and Cassi Fund.  The donations come in large and small amounts — $10,000, $1,000, $500 and even $10.  Many of the checks are coming from people we have never met and they arrive with touching letters of support and encouragement. To everyone this Christmas, we wish you much love, peace and joy.

The Hempel Family

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“I” for Insane, “R” for Ridiculous, “B” for Broken – America’s Institutional Review Boards

December 19, 2007 by  
Filed under HealthCare

Stack-of-Paperwork

Regulations, Regulations, and more Regulations!

Some people have asked me why I am not blogging about different topics every day. I really wish I could be blogging more but besides having 1000 things on my to do list, I am actually scared to death to actually talk about some things that are happening to us right now out of fear we might not get the help and cooperation we need to try and save Addi and Cassi’s lives. There are many doctors, researchers and scientists who are being highly cooperative and it has been a truly remarkable experience. Thank you!

On the other hand, there are others who are not as cooperative. I have found that there are a lot of major egos in the medical and science community. A lot of science is about publishing papers about experiments in mice and petri dishes, not about finding near term treatments and therapies that can save the lives is kids like Addi and Cassi. The system is certainly not designed around sharing and collaboration – it’s really a collection of silos all competing against each other for funding or who will get the next Nobel Prize. Someday I will have the opportunity to explain more.

What I can and will talk about is IRBs – in my mind the term stands for "Insane, Ridiculous and Broken" ethics oversight committees. I had never even heard of the term “IRB” until a few months ago when a doctor said we have to, “get this idea through the IRB and this is going to take lots of time.”  An institutional review board (IRB), also known as an independent ethics committee (IEC) or ethical review board (ERB) is a committee that has been formally designated to approve, monitor, and review biomedical and behavioral research involving humans with the aim to protect the rights and welfare of the research subjects if they are involved in research studies.

One of the biggest problems we are facing at the moment with Addi and Cassi centers around the IRBs. These IRBs now standing in the way of Addi and Cassi getting critical tests done which will ultimately lead us to therapies more quickly. The bottom line is that IRBs are severely impeding progress of medical research in the United States today and something needs to be done about it at a national level.  I know my rights and I want testing done.

Let me give you an example of just how ineffective an IRB can be when you are facing a rare and fatal disease like NPC and you’re in a sprint, not a marathon, to save your kids lives. The National Institutes of Health (NIH) needs spinal, blood and urine samples from healthy children to compare against NPC children who they are currently studying. Both Addi and Cassi are in the study. If the NIH can compare normal kids samples directly against NPC kids samples, they might actually find something called a biomarker (a sign of illness in the bodies of NPC kids). Piece of cake to get samples, right? Wrong. The problem is it can take MONTHS to put together a “protocol” and get it approved through an IRB. Once you get a “protocol” designed, you need to get group of committed physicians (most likely ER doctors) who will be at the front lines to get consent from families to obtain these specimens.

I have been working with the NIH and my local hospital, Renown Regional Medical Center, to try and make this happen.They both have been very cooperative and are trying to move as quickly as possible under the rules but I’m now seven weeks into this process between getting the protocol, the consent form, and begging our local doctors to participate. I still have to get approval from the local IRB so cross your fingers. Why am I even having to do this? If I don’t do this, it will take months, if not years, to get these samples because there are not enough resources in the NPC community to accelerate the process more quickly.

I told this story to a friend who offered to send her child down to get the blood and urine sample done so we could send them to the NIH immediately. Problem is, you can’t do this, it’s against the "rules."  So while millions of samples are being flushed down the toilet or disposed of on a daily basis at hospitals around the country, kids are dying. These samples literally could provide clues into Niemann Pick Type C that could lead to treatments more quickly. 

This is an absolute nightmare of all nightmares. No parent should be faced with getting a diagonosis of Niemann Pick Type C and then enter into a totally dysfunctional medical and drug development system on top of it. While I realize that IRBs do serve a purpose, the regulations have simply gone too far and therefore are becoming ineffective. It seems like IRBs are far more worried about litigation when they should be focused on saving lives.

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Hope, Faith and Love – Our Nations Robotic Heros!

December 7, 2007 by  
Filed under Genetic Testing

National-Chemical-Genomics- We have returned from the National Chemical Genomics Center (NCGC) and are busy working on pushing forward new therapy ideas for Addi and Cassi and Niemann Pick Type C disease using state-of-the-art cheminformatics methods.

The robotic equipment the NCGC has established for testing cells and profiling compounds for large collections of chemicals is incredible.  The whole facility is like something out of a Sci-Fi movie.  It was fascinating to see these three robots in action and we decided to name them, “Hope, Faith and Love.”

It has been an absolute insane six weeks for us since receiving the Niemann Pick Type C diagnosis.  Besides the NCGC trip, we have made trips with the girls to the Mayo Clinic and the National Institutes of Health for a week long study (thankfully with our new portable DVD player and CandyLand CD). I have literally been immersed in trying to get a handle on Niemann Pick Type C, what this means for Addi and Cassi and our family and what we can do to accelerate research into the disease and find therapies.

I have very little science background but I think I may be ready for my Ph.D. in biochemistry!   Try taking a shot at reading the Brown and Goldstein papers after reading my posting and let me know what you think (it takes some time to download).  These new papers are major scientific milestones for understanding Niemann Pick Type C and cholesterol binding at the cellular level but it’s not exactly light reading. I believe we have done a fairly good job on our website by simplifying what is happening to Addi and Cassi — cholesterol is building up in their cells and leading to neurodegeneration in their brains and doctors don’t fully understand why.

At this point, we are doing everything we can to stop this horrible disease and are trying to unearth every stone we can to find near term therapies. I have learned overnight that there is a whole different language in science — it’s a combination of Latin and Greek!  There are words like assays and microarrays (translation: sophisticated tests of cells), IRBs ( institutional review boards are really starting to slow us down in trying to find therapies for Addi and Cassi which I will leave for another post).

BC-Theta, a new kind of cholesterol binding probe (translation: you can see cholesterol in cells better and it’s cool new stuff that could be better than filipin staining (I don’t even want to get into what this is!)  There are words I’m learning like sterols, cyclodextrins, sphingomyelin, cytosolic, pathogenic cascades, hepatomegaly and substrate reduction therapy. I don’t really want to learn any of this but I must in order to try and work towards finding therapies to help Addi and Cassi.

I wish I could go back six weeks to when I was making trips to Baskin-Robbins, the library and Walmart.  I have not been able to spend much time with Addi and Cassi since this all happened and my mother has essentially moved in with us.  Our nanny is working overtime to try and keep things somewhat normal in our household while we try and make headway with doctors and researchers to accelerate research.  We really have no choice.   We can’t simply stand on the sidelines and do nothing so we have decided to take action on multiple levels.

For those of you wanting and update on the drug Zavesca, we still don’t have it and I’ve been working to push this along for weeks now.   After hours of back and forth on the phone and email, we should have the drug by Monday.   I really should not complain.  At least we’re going to get the medication.  I had an email exchange with one Mom and their insurance company won’t cover the drug. It costs $80,000 per year to have a child on Zavesca — $160,000 for Addi and Cassi for the year!   To give you an idea of how expensive this is, in 2006, the median annual household income was $48,201.00!   Obvoiusly, these people don’t make enough money to pay for Zavesca so they have to watch their son slowly die without being able to do anything about it.   If you are feeling sorry for us, can you imagine this hopeless feeling?

This makes me so incredibly ANGRY at our system – a drug that could help slow down this disease should not cost this amount of money.   This is also why I am so motivated to do something to bring awareness to this cholesterol disorder and also what is happening in our medical system (more posts to follow on this for sure!) We are literally in a race against time and unfortunately this is not a marathon.  We are in an outright sprint.  We plan to win this race for all kids with Niemann Pick Type C even though we’ve just come out of the starting blocks.  We appreciate everyone cheering us on!

Finally, we need financial donations to help us accelerete research not only for Addi and Cassi but for a novel medical approach we are pursuing that could impact millions of people.  Please donate today to the Addi and Cassi Fund.

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Grandma Cathy’s Zero Oatmeal Raisen Cookies

December 7, 2007 by  
Filed under Recipe Submissions

Ingredients:

2 Sticks of Imperial Margerine
2 egg whites
2 1/2 teaspoons vanilla
4 tablespoons applesauce
1/4 cup oatbran (adds great fiber)
1/4 cut wheatgerm (adds folic acid, vitamin B12, and zinc vitamins)
Grated rind (zest) of 1 orang2
1 3/4 cup flour
3/4 teaspoon baking soda
3/4 teaspoon baking powder
1/2 teaspoon salt
1/2 teaspoon cinnamon
1/4 teaspoon nutmeg

Preparation:

In a mixing bowl, place 2 sticks of Imperial Margerine.  Stir until smooth.  Add the above ingredients in the order given.  Mix well.  Preheat oven to 350 degrees.  Place 1-2 tablespoons of dough on cookie sheet for each cookie.  Bake at 350 degrees for 15 minutes.  Nuts can be added and also dates.  Dough freezes well.  Makes 4 dozen cookies.

 

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