Walgreen’s Support Allows Our Twins with Niemann Pick Type C To Receive Cyclodextrin Treatments At Home

Addi (l) and Cassi (r) will start their IV Cyclodextrin treatments at home next week after three years in a hospital settting

We are sorry for the lack of posts in recent months! Hugh and I have been extremely busy with rare disease initiatives for Solution Therapeutics, our virtual biotech, as well as helping the Global Genes Project with a number of community based rare disease projects.

We would like to report that after three years of receiving intravenous cyclodextrin infusions (IV Cyclo) in a hospital setting, we have received both hospital Institutional Review Board (IRB) approval and what is considered the “green light” from the FDA to begin our IV Cyclo at home.

We can’t begin to express what this means to our entire family to begin IV Cyclo treatments at home.  Since April 2009, we have been making bi-weekly, and then weekly, visits to our local hospital in order for the twins to receive IV Cyclo.  Their weekly infusion lasts eight hours. By the time we leave our house, initiate the infusion, disconnect the tubing and get home, we’re dealing with a 10+ hour day.

We estimate we have done about 225 IV Cyclo infusions (x2) since we started on this journey to treat Addi and Cassi with Hydroxypropyl Beta Cyclodextrin (HPβCD)– that works out to 2250 hours in the hospital and this does not include our bi-monthly intrathecal cyclodextrin (IT Cyclo) treatments so that HPβCD can reach the twins’ brains.

Why has it taken over three years to get cyclodextrin started in a home setting?  Very good question!

First, we had to contend with the FDA. Our original treatment protocol called for many procedures such as blood pressure checks and having special equipment in place in the event the twins experienced anaphylactic shock from the compound.  Since the twins were the first in the United States to receive this type of treatment, the safety protocol that needed to be in place with the FDA was incredibly strict.   We had to prove the compound was safe and with limited side effects.  (Note: we have not had one reported side effect to FDA in over three years since starting treatment.)

Next, we had issues with how the cyclodextrin was produced from a powder into a sterile liquid form that could be put into Addi and Cassi’s bodies. Our hospital pharmacy was creating our liquid cyclodextrin compound from scratch each week using extensive filters.  This is not a drug you can purchase from a pharmaceutical company, although we are trying to work on an agreement with Johnson & Johnson to provide us with a cyclodextrin solution since they already use it in another drug. Until that agreement in place, we have had to spent tens of thousands of dollars to have it made from scratch and in sterile form.

In order to get an IV Cyclo formula created outside of the hospital, we searched to find a pharmacist and organization willing to help us move to our home. This is where Walgreen’s stepped up to the plate. Ron Vaught, our local Walgreen’s Infusion Services pharmacist, is amazing.  After reviewing our case and meeting with our hospital pharmacist to understand the methods to make the liquid cyclodextrin, Ron contacted Walgreen’s corporate offices to see if the company would allow him to make this formula for our twins.  It was a “long shot” due to corporate liability issue (this seems to be the first thing that comes up before someone can decide to help us) but Walgreen’s was willing to work with us and we are so grateful!

After receiving Walgreen’s commitment to make the IV cyclodextrin formula, we had to alter our treatment protocol for the FDA to allow for home based infusions using Walgreen’s Infusions Services and nurses.  We then submitted the new protocol to the FDA and Children’s Hospital Oakland IRB.  Our protocol called for a “transition period” where the twins would receive their treatments in a clinic setting at the Children’s Specialty Center of Nevada with the formula mixed by Ron at Walgreen’s Infusion Services.  We have been doing that for the past 3-4 months and all has been well.

Critical Care Systems of Reno, NV, worked with Walgreen’s Infusion Services and Dr. Caroline Hastings to get our insurance approval to do these treatments in our home.

This Tuesday, July 17, at 9:30am, two Critical Care Systems nurses will arrive at our home to connect Addi and Cassi’s IV Mediports (which are installed in their chest). With them will be the cyclodextrin solution made by Ron at Walgreen’s and a new portable pump.  The nurses are not allowed to push the “start” button on the home infusion system — that will be left to Hugh and I due to “liability” issues.  We will also be trained on how to “de-access” the ports, which means we will flush them and take the needles out.

After watching the procedure 225 times (x2), I am pretty sure I can de-access the port in my sleep!  And after three years in the hospital, I seriously can’t wait to push that pump button!