August 31, 2011

October Niemann Pick Awarness Challenge

Walk for a Cure in Memory of Riley Corbitt 2010
This year for Niemann Pick Awareness Month we are challenging people to find 11 people to donate $11 each. This is such a simple way to raise a lot of money. I know we are a month away but I wanted to put a bug in every ones ear so you could start making your list of the 11 people you are going to ask. Also remember to challenge others to find 11 people to donate $11 each!!

Stay tuned for more details as they come along from the National Niemann Pick Disease Foundation.

2nd Annual Walk for Wishes

Come join the Walkin' 4 Tylor team at Murphy Park on Sunday, October 2nd from 8am to 12pm. Make A Wish of Iowa granted over 176 wishes in 2010 and unfortunately the list of children in need of a wish is growing.

Last year the Walkin' 4 Tylor team raised over $1100 and had over 40 people walk on our team! WOW...we even won an award for the largest walk team. Well we are set out to surpass lasts years numbers so we need your help. Please join our team at Walking for Wishes. There are instructions on how to join our team on the website.

We will be looking for sponsors for our team again this year but instead of T-shirts we are going to be selling spots on Tylor's wheelchair. So if you have any businesses or families that would like to buy a spot please let me know.

Hope to see you there!!!

Rachel Taylor "Here for You Always"

A video/slideshow montage of NNPDF families created in honor of those who love and support us. Many thanks to singer/songwriter Rachel Taylor for allowing us to use her song, and thanks to the families who responded to our request for photos! Special thanks to the NNPDF volunteers who so lovingly created this video. For more of Rachel's music, please visit For more information about Niemann-Pick Disease visit

August 18, 2011

Friends from Camp

Wednesday night we had some of the crew from Camp Albrecht Acres come over for dinner. Rich, Nicola, Katie, and Emily wanted to visit with Tylor a little more before heading to L.A. on Friday. They entertained Tylor while I was cooking the burgers....or maybe it was the other way around! Tylor was really into a movie that was making him it was kinda entertainment for us as well!

Emily, Rich, Nicola, Katie and Tylor

After eating dinner we sat out side so the gang could meet Ty's new dog Josie, yes I said new dog (puppy I mean). I know most of you don't know about her so here she is....

Josie and Bono

While visiting Rich mentioned that the Sherill Fire Department would be at camp on Thursday spraying the kids with the hose and that I should bring Tylor out. So this morning we heading out there. Tylor fell asleep on the way out and didn't really wake up the whole time we were there until....they got him with the hose!!!

Tylor woke up fast

Rich and his camping buddy with Ty

Ty got to hold the hose after the water was gone

Thanks for the fun today! We love all of you and will miss you dearly!

NIH Cyclodextrin Trial

National Institues of Health Announces Cyclodextrin Clincial Trial For Fatal Cholesterol Disease Called Niemann Pick Type C

August 16, 2011 by Chris Hempel
Over the past three years, many Niemann Pick Type C families as well as physicians treating NPC patients worldwide have contacted Dr. Caroline Hastings and I regarding Addi and Cassi’s treatments with hydroxypropyl-beta-cyclodextrin.

Since sharing our intravenous (IV) and intrathecal (IT) cyclodextrin treatment protocols with NPC families and doctors in the U.S. and other countries, many NPC patients have started cyclodextrin therapy.

Currently, I know of 11 NPC patients in various countries receiving cyclodextrin treatments – six are receiving intrathecal (IT) treatments to allow cyclodextrin to reach the brain.

With many Niemann Pick Type C families scattered around the world, it’s difficult for people to obtain information on cyclodextrin and determine how they might be able to get started with treatments. In the past month alone, I have been contacted by families located in Italy, Spain, Germany, Dubai and Japan and all are interested in cyclodextrin treatments.

I am writing this blog in the hopes it can help NPC families in other countries who are seeking information on cyclodextrin and help them understand how they may be able to treat their loved ones with this potentially life saving sugar compound.

NIH Announces Intracerebroventricular Clinical Trial with Cyclodextrin
In the United States, the National Institutes of Health (NIH) has announced an intracerebroventricular (ICV) clinical trial with cyclodextrin. Dr. Forbes “Denny” Porter is running the clinical trial with an NIH Bench-to-Bedside Grant and with the help of the NIHs Therapeutics for Rare and Neglected Diseases (TRND) program and the National Center for Advancing Translational Sciences (NCATS).

Details are limited on what the inclusion/exclusion criteria will be for the NIH cyclodextrin clinical trial and they are still preparing their IND for the FDA and conducting pre-clinical studies.

The exciting news for NPC families is that the NIH has announced that they will pursue delivering cyclodextrin directly into the brains of patients using an Ommaya reservoir. An Ommaya reservoir is an intraventricular catheter system – a catheter is implanted into the brain and it is attached to a reservoir implanted under the scalp.

Dosing is still being determined by work done on NPC animal models, including the naturally occurring NPC cat model. I am sure dosing will be quite similar to what we are already giving to Addi and Cassi (possibly every two week cycle) as the FDA likes to base clinical trials from animal data. The NPC cats receive bi-monthly doses of cyclodextrin and Dr. Hastings based our initial bi-monthly IT treatment plan from the same cat studies.

Getting Cyclodextrin Into The Brain and Past Blood Brain Barrier
In my previous posts, I have detailed our plans with trying to get cyclodextrin into the twins’ brains. In October 2010, Dr. Hastings received FDA approval to start the twins on intrathecal injections of cyclodextrin into their spines. Since Addi and Cassi were the first in the world to ever receive this treatment, safety was a primary concern.

We initially started with intrathecal injections into the spine because we had to prove that the twins would not have an adverse reaction or die from an injection of cyclodextrin into their central nervous systems. Also, intrathecal treatment was less invasive. Per our FDA approved protocol, the twins must be hospitalized and monitored after intrathecal treatments. At first, they were monitored for 24 hours. Currently, we’re down to three hour observations following IT treatments.

Since October 2010, Addi and Cassi have received over 20 intrathecal injections of cyclodextrin into their spines as we work towards a permanent solution. The twins were going deaf before we initiated intrathecal cyclodextrin therapy. We have now restored their hearing to normal levels which is quite encouraging.

We also continue with our weekly IV infusions of cyclodextrin into their bloodstreams and are also looking for ways to deliver cyclodextrin into the lung as IV and IT treatments do not appear to reach the lung.

Medtronic SynchroMed Pump To Deliver Cyclodextrin
Over the past year, we have been creating an intrathecal protocol with the help of Medtronic and Johnson & Johnson and plan to implement a Medtronic SynchroMed pump system to deliver cyclodextrin into the brains of the twins. Medtronic is currently conducting final pump studies with cyclodextrin and we hope to re- submit data to the FDA and our hospital Institutional Review Board (IRB) in September.

We are working towards a SynchroMed pump solution because the NPC mice data shows that low continuous dosing is effective in completely stopping NPC. NPC mice are now being treated like diabetics. Cyclodextrin may need to be given on a frequent basis (perhaps daily or even weekly). We simply don’t know. We will start with a weekly bolus of cyclodextrin through the pump.

The Ommaya port does have many advantages, however, it does not offer frequency of dosing — it’s an access port versus an automatic system. In addition, it is my understanding that the Ommaya requires a patient to be in a hospital setting to receive a treatment (in the United States). SynchroMed pump refills can be made in an outpatient setting once the pump is installed and the pump lasts about seven years.

Choosing Between Ommaya and SynchroMed
The beauty of the Ommaya system is that you know for certain that cyclodextrin is reaching the brain. I believe the NIHs plan to pursue the Ommaya option is the right decision for the clinical trial and to determine efficacy. I hope many NPC patients will be eligible to participate in the clinical because I believe cyclodextrin could be life saving.

Since Addi and Cassi have been receiving cyclodextrin treatments for almost three years, they will not be eligible for the NIH trial. Many other NPC patients, especially those in foreign countries, will also not be able to participate in a U.S. clinical trial and therefore may want to pursue treatments in their individual countries.

Hugh and I continue to look at a longer term and permanent solution for cyclodextrin treatments as it appears cyclodextrin will be a life-long treatment. We are looking for a solution that provides flexibility on dosing and also keeps our kids out of a hospital setting as much as possible.

Medtronic’s data shows high catheter placement into the spine will allow cyclodextrin to reach brain and we will be taking this chance. We know that our intrathecal treatments in the lower spinal area are reaching the twins’ brains as we have restored the twins’ hearing to normal levels. But there are still lots of unknowns and many risks too. There is no easy decision and no right answer.

Also, I am not sure if an infant can get a SynchroMed pump placed so the only option may be an Ommaya. The NPC animal data shows that the sooner the animals start cyclodextrin treatment (BEFORE symptoms are evident) the healthier the animals stay.

Exciting Treatment Options
The good news for NPC patients and families worldwide is there are now promising treatment options to pursue which were not available before. Doctors can look at both Ommaya or SynchroMed solutions to get cyclodextrin into the brain and consider these options today for their patients.

I believe we’re ultimately going to need combination therapy to treat NPC as the disease impacts every cell in the body. We’ll need ICV or IT to reach the brain, IV to reach the organs and tissues and probably some type of inhalable HPBCD to reach the lung.

These are the options we are pursuing to try and save Addi and Cassi’s lives. We hope this information will help other families as they look for any possible way to save their loved ones from this wretched disease.

For more on Addi and Cassi go to their website.

No fair weather riders here!

Rees, Tylor, and Rylee (good buds of Ty's)
Can't forget about Fat Head
The 2nd Annual Ryde 4 Tylor went on even threw the rain! We had about 30 bikes came and weather the storm with us.  Shortly after Noon we saddled up and headed for Buenie, we went through some rain but we missed the down pour by 45 minutes. But look at the view....

At Hank's Ty got a tshirt and a koozie....what a stud (he loves being the center of attention).

Hang Ten!
Sy and Kirsten
Ty and his Dad
After the first stop we lost some bikes because the weather station was calling for rain all day but most continued on. And let me tell you God was watching over us....the rain came when we were at our stop and then let up for us to make it to the next. I let my friend, Kirsten, ride with Sy so I jumped on the bus. The bus got lost, even though I did the practice ride I wasn't much help! I never pay attention when I am not driving, duh! So the bus group missed the third stop and didn't know if we had enough time to make the forth stop but we went anyway. I am glad we did because this is what we pulled up to....

Our dear friends from Farley wanted to surprise me, to say the least they did! It brought tears to my eyes!! Thanks again to the gang in Farley, you guys are the best!

Ty's Crew
Even through the rain we were able to spread the word about NPC and raise some money! Thank you to all you helped make the day a success, to all the riders, bar owners, and people we meet along the way! It is becasue of you our researchers can keep fighting for a CURE!!

Love You Buddy!

August 12, 2011



Ride will be leaving Knicker's at Noon sharp. Sign up starts at 10:30am. $10 donation per rider.

We did the practice run last week and the ride is awesome!! Here are the stops....

Hank's in Buenie (they will have Brats)

The Damn Bar in Guttenberg

The Detour in Petersberg

Dirty Ernie's in Farley

Back to Knickers for food and music!!

Last years Ryde 4 Tylor was awesome so if you are free this Saturday come out and ride for a great cause. All donations with go towards finding a cure for Niemann Pick Type C. Even if you don't have a bike bring your car!!

July 14th-Mayo check up

Tylor's last check up was very uneventful, but we like them that way!

Lisa said his height and weight are good. He weighs 123lbs and is a whopping 5'-11"! His tube feedings are going well and he continues to eat orally. After a little more discussion about his eating habits she suggested we keep everything the same unless something were to change.

The G-tube specialist looked at the site before changing the tube, she commented on how good the site looks. There is no skin break down, no scar tissue, and no leakage. The tract has moved due to Tylor's growth but she didn't have any concerns to warrant a GI investigation. So she changed the tube and sent us on our way.

Dr. Patterson continues to be impressed that Ty is remaining stable. His seizures are under control, he is down to about 15 per month were before he would have anywhere from 35 to 45 per month. He was very alert during the exam....smiling and laughing when appropriate. The doctor contributed Ty's stability to the care we provide and the reduced seizure activity. So unless something changes we will see them again in six months.

August 3, 2011


I had to share these pictures with everyone. I think they are so cute and yes Tylor actually fell asleep in the funny is that!!

Mr. Hollywood himself

Ty's summer in a nutshell

Crackle pop, crackle pop pop, buzz, buzz, splash, cluck, cluck....uh the sounds of CAMP!  Tylor went to Camp Albrecht Acres again this summer...his favorite part of summer break.

His time at camp was full of swimming, fishing, arts & crafts, movies, campfires, smores, dancing, an awards ceremony (Ty won #1 camper and the biggest cheese head award), oh and did I mention smores....

Rich was his counselor for the week he was there (Rich was there last year too). Tylor and Rich really hit if off. Rich is so good to Tylor and when you see them together Ty always has a HUGE smile on his face. He also got to spend a lot of time with Jason, his friend from last year. Jason is also a die hard Cubs fan.

Tylor and Rich
Tylor and Jason

It's hard to believe that camp is over. It makes me sad to have to say goodbye to Rich. He is a special person and I am glad he is a part of Tylor's life.

Thank you to the staff and counselors for making the 2011 camp season a wonderful experience not only for Tylor but for all the campers.

Tylor is ready for next year and believe or not there are only 331 more day until the 2012 camp season!! See you next year!

Walk 4 Wishes

Grab your tennis shoes, comfy pants, the stroller for the kids, the dog and join us on October 2nd at Murphy Park for the 2nd Annual WALK 4 WISHES from 8:30am to 10:30am.

All donations will help grant wishes to kids with life threatening illnesses in Iowa. Make a Wish of Iowa grants an average of 200 wishes a year. So we need your help to keep granting more wishes for children like Tylor.

Tylor was granted his wish in 2008! Make A Wish flew him to New York to meet Brett Favre. This is a memory no one can ever take from our family. So we as a family are challenging you to come walk with us and if you can't walk with us please donate. Every little bit can either contact Make A Wish or myself to make a donation.

Thank you for your support!!