From My Friend Nabiha In Pakistan
 

Dear Chris,

I am not a very interesting person…and as of now only a broken hearted one.  I had a very nice childhood, a loving family and fun loving friends, got what I wanted, studied what I wanted or where I wanted.  I was taught never to do anything bad with others, only to help the less privileged ones.  I was a top student, had many distinctions during my academic career and somehow or the other became the first on camera female reporter of the state run news television in Pakistan.

I was the youngest one in the media circles to have worked with the decision makers at the highest corridors of power and travelled with my country’s President and the head of the Government, the PM to their visits abroad.  During my international travelling, I met with the world leaders, was praised for my work for being the one who would not compromise on any news story for political expediency and yet was surviving well.  Whenever I used to go out with my family - people would recognize me and ask for my autograph.

But that was not what was the best thing that happened to me…it was Hisan….. who although lost his father very early in his life was a wonderful and delightful child.  Being a single mother, I thought I would give Hisan the best of everything so that no one would think that he did not get what he desired since he had no father.  I wanted so much to give Hisan and education abroad as I had to miss my scholarship when I got married.  I guess it’s the same unfinished agenda on my life that I wanted so much for have Hisan have.  I believe every parent looks towards his or her child in terms of their unfulfilled aspirations.  I had many offers in my hand but I regretted to all as I wanted Hisan to be near to me.  I left the most coveted beat and resigned from my media position when I thought I was missing Hisan on the way.  People thought that I was crazy for doing that…..a professional suicide.  As for me, I never once regretted my decision.

You see it is not me who is important in this picture..it is Hisan…the ‘miracle boy’ who had left the most experienced and seasoned doctors in awe owing to his resilience and bravery to fight back this pathetic Niemann Pick Type C disease.  Once in a code blue situation, Hisan came back after 45 minutes of CPR, all soaked in a pool of his own blood…and went home to spend some very memorable time with his family. 

Hisan was the favorite of all…..be it doctors, nurses, therapists…he would have the pricks done on him without crying…would keep quiet even in extreme pain and his eyes would always exude compassion…tolerance and a certain peacefulness that would put me to shame.  When everyone had lost hope Hisan would fight back and lead the doctors to think otherwise.  His fighting spirit would prove everyone wrong.

I would become the proud Mom of a very special child.  He would never let me down..never..and it became an altogether different story that my heart would cut in shreds upon every needle that was poked in him.  Never mind missing Stanford.  One after another he lost everything except his beautiful eyes that would tell everything to his Mom and his beautiful spirit.  He died so peacefully.  So becoming of him.  I cry for him each time and I mourn him.  My world is not only blank, it is meaningless as I have lost the will to live anymore.  The only thing matters to me is the long drawn wait that I have to go through to meet him some time when he is happy and without the monster.   You ask for my religion?   For me it is probably not a bad bargain after all to give him back soon and then to have him forever afterwards…that’s my belief as a human being and as a Muslim too.

Love to your Princesses!
Nabiha

There are new reasons for hope not only for families of NPC kids but for families with children suffering from Duchenne muscular dystrophy, a fatal recessive X-linked form of muscular dystrophy that strikes 1 in 3500 boys and leads to paralysis and ultimately death.

According to a research paper released from the University of Washington, understanding the mechanism of Niemann Pick Type C (NPC), a fatal progressive neurodegenerative disease that is trying to take the lives of Addi & Cassi could help researchers find new treatments for Duchenne Muscular Dystrophy (DMD) and possibly muscular dystrophy in general.

In the studies conducted by the University of Washington, “transgenic expression of Neimann Pick Type C 1 (NPC1) in skeletal muscle ameliorates muscular dystrophy in the Dtna-/- mouse (which has a relatively mild dystrophic phenotype) and in the mdx mouse, a model for DMD. These results identify a new compensatory gene for muscular dystrophy and reveal a potential new therapeutic target for DMD.”

In addition, the researchers report that “molecular abnormalities in NPC1-null cells suggest possible links to known causes of muscle degeneration. One particularly intriguing connection involves the caveolins. Caveolin-3, the muscle-specific form, binds directly to the dystrophin complex members, β-dystroglycan at the sarcolemma and is required for the correct targeting of the dystrophin complex to cholesterol-sphingolipid rafts/caveolae. A link between caveolin-3 regulation and the muscular dystrophies is well established. Muscles from Duchenne muscular dystrophy patients and mdx mouse muscles have elevated caveolin-3 levels.”

The Muscular Dystrophy Association (MDA) through the help of Jerry Lewis has raise approximately $2 billion for neuromuscular patient care and research since its inception yet a cure has not been found to date.  This paper suggests that new therapies could be found for muscular dystrophy by looking at the NPC1 gene, a gene that causes a rare and fatal childhood disease that strikes only 500 children worldwide.   Does anyone have Jerry Lewis’ number?

Posted by Chris (MOM - Mom On a Mission)

Important news.  In a recent issue of Nature Medicine, Oxford University reported that Niemann Pick Type C disease is a sphingosine storage disease that causes deregulation of lysosomal calcium.  According to Oxford researchers, there is a block in the late endosome to lysosome transport in NPC disease, resulting in the downstream storage of cholesterol and glycosphingolipids (a lipid derived from a ceramide that contains a carbohydrate such as glucose or galactose).

For the lay person, this means that cholesterol is not the ultimate culprit in Niemann Pick Type C disease — there is a cascade of events happening in the cell with sphingosine using up the calcium in the cell which then triggers a cholesterol traffic jam in the cells of NPC patients.  Oxford conducted extensive studies and published a paper which shows many complex experiments.  One experiment showed that Curcumin is helping to correct the loss of calcium and rejuvenate the calcium in the cell.

Typically, NPC mice die at 13 weeks of age.   Above is a picture of a 9 week old Niemann Pick Type C mouse — the one on the top is not treated with Curcumin, the one on the bottom has been treated with Curcumin.  Curcumin is obviously extending its life.  Curcumin is considerd a "spice or supplement," not classified as a drug.  This is amazing research by Oxford and I am grateful for their research as my 4 year old identical twin girls, Addi and Cassi, are on high doses of bioavailable Curcumin (delivered to them 2x a day in applesauce!) 

The data from Oxford suggests we must pursue a new clinical intervention strategy for treating Niemann Pick Type C, this horrible neurological disorder that is trying to take the lives of my precious twins.  What Oxford has also shown us is that NPC patients potentially have another "treatment option" available to them in addition to Zavesca.  For many NPC patients who have no access to drugs of any kind, Curcumin could help their suffering children.   Non-toxic drugs similar to myriocin that reduce sphingosine levels could be a therapeutic targets for NPC patients or drugs that act to reguvinate the calcium in the cell could help as well.

If you have a brain disease and have been told that Curcumin can help you and are interested in obtaining the bioavailable Curcumin UCLA Alzheimer’s reseachers have developed for humans, contact Verdure Sciences.   As I reported in my last blog, Verdure Sciences has a compound of bioavailable Curcumin that crosses the formidable blood brain barrier in humans.

What next?  A 2008 Nobel Prize winning scientist!   Apparently, a UCSD scientist is one of the leading authorities in the world on cytosolic free calcium.   I am hoping this Nobel winner will provide some insights and guidance on how we can accelerate research into lysosomal calcium to not only help Addi and Cassi but other kids who suffer from this horrible fatal disease.  Wish me luck!

Posted by Chris (MOM - Mom On a Mission)

Verdure Sciences - Never heard of this company?? Neither had I until I started trying to find a natural botanical compound called Curcumin to help save Addi and Cassi, our four year old identical twins who suffer from a fatal disease that mimics Alzheimer’s in young children. It’s called Niemann Pick Type C (NP-C) and I learned last October that it’s trying to destroy my twins’ brains and kill them.

There is only one experimental drug for NP-C that costs a whopping $160,000 per year so I began looking at every available alternative to treat our twins and save them from dementia. After learning through a special MRI brain scan that Addi and Cassi have brain inflammation I focused my efforts on anti-inflammatory products that get into the brain. 

I had heard about a natural “wonder compound” called Curcumin that was working well in NP-C mice studies and also in Alzheimer’s mice studies. Curcumin is not a new idea.  It’s a component of Turmeric, an Indian spice, and people have been trying it as therapy for a variety of diseases and there are thousands of scientific papers on its amazing properties.   But I found out there was a major problem with Curcumin.  The BBB.

Curcumin was a “lab superstar” and working well in test tubes but when tested in humans the absorption rate was extremely low. I was told by our doctors that the Curcumin currently available on the market, even pharmaceutical grade, would not cross the blood brain barrier (BBB) to help Addi and Cassi with brain inflammation. I decided I would try and create my own “bio-available” version of Curcumin and started reading everything I could get my hands on. I even arranged for a formulation pharmacy to help me make a Curcumin suspension. It was then that I read about Dr. Greg Cole.

Dr. Cole is a leading Alzheimer’s researcher at UCLA and during my research I found out that he is the leading authority on Curcumin and had written dozens of papers on the neuro-protective properties of the substance. I decided to email him to see if he could help me understand the bio-available aspects of Curcumin.

A prayer was answered the day I emailed Dr. Cole.  His team at UCLA has developed a formulation of “Optimized Curcumin” that works more like a drug with “super bio-availability.” See the chart.  UCLA’s Curcumin was not only crossing into the blood stream it was crossing through the blood brain barrier (BBB). The BBB is a membranic structure in the central nervous system (CNS) that restricts the passage of various chemical substances and things like bacteria between the bloodstream and the neural tissue itself, while still allowing the passage of substances like oxygen that is essential to metabolic function.

UCLA put me in touch with Verdure Sciences, an innovative bio-nutritional company, making the super bio-available version of Curcumin.  UCLA and Verdure Sciences have done extensive testing and spent several years creating this special formulation. I immediately put Addi and Cassi on significant doses of Curcumin and noticed improvements in their mood, balance and coordination right away. While this is anecdotal, I cannot attribute these positive effects in my girls to anything other than Verdure Sciences optimized Curcumin formulation. Except for a modest amount of diarrhea when we initially started the girls on it, there are no negative side effects from what I can tell. It’s all natural and not even a drug yet it’s acting like a powerful anti-inflammatory.

Do not be fooled by what other companies claim with their Curcumin products.  If they tell you it’s optimized Curcumin, it’s not!  There are lots of Internet scams and companies that make all kids of false claims.    Verdure Sciences is the only company that has the optimized Curcumin with UCLA mice studies to back it up.   The product is going to be marketed under the name LongVida or “Long Life" but right now it’s only available if you call them directly.

One of the real neuro-protective compounds for the 21st century has finally arrived and it’s not even made by a pharmaceutical company. Thank you UCLA and Verdure Sciences for persevering to bring theraputic Curcumin to people.  While the drug companies are only interested in "drugs" and financial profits, you have shown us a new pathway with Curcumin that is not all about money — it’s truly about health and trying to save peoples lives.  I will be forever grateful and I know others will be too.

Posted by Chris (Mom)

On Monday, September 22nd, 2008, our family will featured on "The Doctor’s," a new Dr. Phil spin-off television show that is in its first season.  Addi and Cassi’s battle against Niemann Pick Type C, also known as the "Childhood Alzheimer’s" disease, will be discussed and we will appear as guests on the show.   Addi and Cassi will also make an appearance towards the end of the segment — you can see the girls getting into all the apples on the set!   Watch us now.
 
Thank you so much to The Doctor’s and to Dr. Phil for helping create this new show to bring reliable medical and health advice to our nation.   We hope this new show will help generate national awareness of our fight to beat this fatal illness that destroys the brain and kills children in childhood.  Please take a moment to read our story and make a wish for us today!

Coriell Institute - Understandings Human Genetics

I had never heard of the term “BioBank” until my 4 year old identical twins, Addi and Cassi, were diagnosed with a fatal disease called Niemann Pick Type C, otherwise known as the “Childhood Alzheimer’s.” 

Since receiving this nightmare diagnosis, I have become familiar with many organizations I had no idea existed, including an nonprofit research organization called the Coriell Institute based in Camden, New Jersey.  Coriell is a leading biobank that is funded by the NIH and they are dedicated to understanding human genetic diseases.

Coriell collects human biospecimens from people with all types of diseases ranging from Epilepsy to Parkinson’s to Rett Syndrome.   As scientists study the connection points between genes and diseases, they are finding many diseases have complex patterns of inheritance.

I have submitted Addi and Cassi’s “biospecimens” — things like tissue and blood samples — to Coriell which are now being analyzed and stored in tanks.   When researchers are interested in studying Niemann Pick Type C diesase, they order biospecimens from Coriell so they can try and understand the molecular characteristics and clinical progression of the disease.    Once Addi and Cassi’s NPC mutation is fully analyzed, it is given an anonymous number and will be represented by a red dot on Chromosome 18 and can be ordered from Coriell. 

Addi and Cassi’s DNA will essentially live on forever in these tanks and my hope is someday their DNA will help solve something researchers have not thought of yet.  Maybe their identical twinship will prove even more important to understanding disease than the genetic disease they were born with?  Who knows, but it’s interesting to think of the possibilities.

Cures for human disease can only happen faster with biobanks providing researchers with critical genetic material.  I am eternally grateful to the NPC families who have already contributed to Coriell’s biobank.   Most of these NPC children have passed away but they live on through current research work and they are now trying to help save Addi and Cassi’s lives.

I hope people who have the opportunity to provide samples to biobanks like Coriell will do so in the future and without fear.  The good news is that the newly enacted genetic discrimination law (GINA) bars health insurers from using genetic information to make coverage decisions.   Many Americans have been reluctant to take advantage of breakthroughs in genetic testing for fear of “genetic discrimination” and that the results could deny them jobs or health insurance. 

Posted by Mom (Chris)

Biggest Little Community In The World Supports Finding Cure

Reno, Nevada, may be known as the “Biggest Little City in the World,” but the City also has the title of the “Biggest Little Community in the World.”  Since our identical twin 4 year old twins, Addi and Cassi, received a devastating medical diagnosis of Niemann Pick Type C — otherwise known as “Childhood Alzheimer’s (featured on ABC’s Good Morning America) – we have received an outpouring of support from the Reno community to help us fight this fatal disease that robs children of their ability to walk, talk and eat.

On May 10th, 2008, the Reno community came together for the “Moonlight Garden Gala - A Night Of Hope and Miracles” at the Downtown Reno Ballroom to help raise money for research into this devastating and mysterious brain disorder.

Millions of American’s are facing Alzheimer’s disease and every 71 seconds someone in America develops the disease.  NPC children like Addi and Cassi may hold important clues to helping researchers understand brain disorders of all types.  No person, whether young or old, should face death of the mind before death of the body and treatments and cures need to be developed.

Here are some of the amazing events that have taken place in recent weeks in Reno to help raise money and awareness of brain disorders and "Childhood Alzheimers."

• Claim Jumper Fundraiser: On April 24th, Claim Jumper hosted an evening to raise money for the Addi and Cassi Fund with 10% of all proceeds earned that evening going to the fund. Thanks to Cara Hamm for organizing the event — we raised $2,358.33! 
• Sierra Strength and Speed: Joe Dibble, one of Nevada’s leading dietitians and nutritionists, donated 100% of the proceeds from “Wednesday’s Workout Evening” at the WB Baseball Softball Training Center.  Everyone worked up a sweat, including Addi and Cassi, and Joe and his volunteers helped raise $1,345.00! 
• On Thursday, May 8, 2008, Bishop Manogue Catholic High School held a "Miracle Minute" to raise funds to aid in the research necessary to find a cure for Niemann Pick Type C. 
• CureCaps party – Jennifer Dolder and her two children organized a CureCaps hat making party at their house – over 50 caps were made to help raise money for research

Thank you Reno for your support!

Posted by Chris (Mom)

Saving Our Brains

We’re 4 year old identical twins, Addi and Cassi Hempel.  We recently went on a trip to Detroit, Michigan, with our Mom, Aunt and Grandma to have PET scans done on our brains.  We are suffering from a rare and progressive neurological disease called Niemann Pick Type C.   If we don’t do something about it, we’re going to lose our minds.

Our PET scans revealed that just like some Alzheimer’s patients, we have a glucose metabolism problem in the frontal lobes of our brains.  This causes dementia.  One of us (Cassi) has an inflammation spot in the middle of our brain, in the parietal lobe and basal ganglia area.  This is an area of the brain where Parkinson’s patients are often impacted. 

We ask that you take a moment to learn more about us and our fight against NPC.  We have posted our brain videos, report and scans for the world to see.   We hope that through a view into our brains, we can bring answers and treatments not only for ourselves but for others who suffer from neurological conditions of all types.

Watch our Brain Video 
Read our Brain Report

Look At Our PET Scans
Glucose FDG Addi
Glucose FDG Cassi
PK Addi
PK Cassi

An Everlasting Promise

Dear Hisan, Ibrahim, & Anthony:

We love you!  We will always fight, our brothers.  Not a war against each other but a war against disease.  Your lives will not be lost in vain.  We promise.  Promises are witnessed by God and forbidden to be broken by Allah.

Your sisters,
Addi & Cassi

I was laying in bed the other afternoon after staying up all night emailing scientists the results of Addi and Cassi’s brain scans when the doorbell rang.  It was Christopher Serio with an envelope in hand.  Chris is 11 years old and is the son of our friend and former neighbor, Bob.   “Here, this is for Addi and Cassi,” he says and proceeds to hand me an envelope decorated with a red heart, a sunshine and pine trees.   “I just counted up all my savings from when I was little.  I have $653 dollars and I want to give Cassi and Addi $150 dollars of my savings for research into Niemann Pick Type C.”  

I open the envelope and it’s full of cash.  I almost burst into tears but I somehow hold it together and give him a great big hug.   I think about how generous this young man is.  It turns out that this is an entire year of his allowance.   Would anyone else we know give us an entire year of their savings to help with research?  I am so inspired by Chris and his generosity.   As I watch him walk down the driveway and get into his Dad’s truck, I hold out hope that Addi and Cassi will someday be competing for his attention.  A lovely young man with a very big heart.  That’s Christopher Serio.

Posted by Mom (Chris)