Exciting News

Members of the NPC team....THANK YOU!

Dear families and friends of the NPC community,

There has been a large, collaborative effort to initiate a cyclodextrin clinical trial at the National Institutes of Health (NIH) to systematically evaluate the safety and efficacy of cyclodextrin therapy for the treatment of Niemann-Pick type C (NPC) disease. As many of you are aware, we met with the Food and Drug Administration (FDA) this past Tuesday, November 1, 2011, to discuss the development program for cyclodextrin

The exceptional work that has been done in NPC animal models has guided the design of a human clinical trial. Together with the Therapeutics for Rare and Neglected Diseases (TRND) group at the NIH, as well as several NPC researchers, Johnson & Johnson, and consultants from RRD International, LLC, we are working to submit an Investigational New Drug (IND) application to FDA.

The first step in submitting the IND application to FDA (the perquisite to an initial clinical trial in patients) was to request a pre-IND meeting with FDA to receive the Agency’s feedback on our development program before the IND application is officially submitted. On November 1 we met with the FDA review division staff to discuss the proposed development plan for cyclodextrin and needs for the IND application package. The meeting was positive and the Agency provided helpful feedback focusing on the drug safety and toxicology data. We will have an additional meeting with FDA to focus on the clinical trial design, and FDA is working with us to get that meeting scheduled before the end of the year.

We view this as a very positive step toward pursuing cyclodextrin as a potential treatment for NPC disease. We are planning a scientifically rigorous trial that will allow us to test cyclodextrin in our patients safely and in a way that will provide as much information as possible. While specific details of the trial will not be available until we have agreement from FDA and approval from the NIH ethics review board, we will share information with the NPC community as it is available.

We continue to work toward our goal of starting the trial next year and feel that with the recent FDA feedback, we are on track to do so.

Thank you for your continued support and encouragement as we work together to find a treatment for NPC disease. This fight would not be possible without all of you.

Sincerely,

The TRND Team

New Year New Hope

Now with the Holidays behind us and a new year ahead I look for new hope. Hope that the doctors and researches with come up with a promising treatment for Niemann Pick Type C (NPC) and that it can be made for all children affected by this horrible disease. Over the past couple of years there has been a lot of talk about Cyclodextrin treating NPC. Chris and Hugh Hempel and Bryan and Laura Hadley have both their children on it. I have talked to Dr. Patterson about it many times and he feels there is not enough data to support the effects of Cyclodextrin on NPC to put Tylor through the procedures. But on the other hand how can we just sit here and do nothing? I am in the process of doing more research and will be contacting the Hempels and the Hadleys about the results they are seeing with their children. We are going back to Mayo in February and I plan on talking to Dr. Patterson about this again. The biggest hurdle will be going to the FDA for approval but if the Hempels and Hadleys got it done I am sure they will have some advise for us.

Some of you may be wondering what Cyclodextrin is. Cyclodextrin is a sugar molecule used in common food and household products like Febreze® Fabric Refresher called Hydroxypropyl Beta Cyclodextrin(HPßCD). To find out more please visit the CTD Holdings, Inc website.

God Bless

Could Cyclodextrin be one step closer?

By Amy Dockser Marcus


We’ve been following the efforts of one family, the Hempels, to draw attention to the potential of cyclodextrin, an experimental drug that may have potential against a fatal neurodegenerative disease called Niemann-Pick Type C.

Addison and Cassidy Hempel, twin six-year-old girls, have the disease, and their parents were part of a larger group that earlier this year successfully got orphan drug designation for the compound from the FDA, a key first step for drug development in rare diseases.

The FDA this week gave permission for cyclodextrin to be injected into the spines of the girls. They’ve been receiving the drug intravenously for over a year but new research indicated that it does not cross the blood-brain barrier.

Chris Hempel, the girls’ mother, tells the Health Blog that the family pursued the new approach for delivering the drug in the hopes it will get into the brain and stave off some of the neurological decline associated with NPC. She also hopes that the information they report to the FDA may help bring cyclodextrin one step closer to a trial.

A spokeswoman for the FDA says the agency can’t comment on drugs under agency review or on any individual’s private medical information.

It isn’t easy to get clinical trials going in children with brain disorders. The Children’s Neurobiological Solutions Foundation held a meeting last week with clinicians, researchers, advocates and others to discuss some of the barriers, including the potential risks to participants. (Those can leave drug companies and hospitals skittish.) The foundation is trying to create a national network of sites in the U.S. that will collaborate on setting up trials for pediatric brain disease treatments.

The NIH held a workshop on developing therapies for NPC disease earlier this year. Cyclodextrin played a prominent role there too. A working group of researchers and clinicians has been discussing what scientific questions need to get answered before a trial could be launched.

Hempel tells the HealthBlog that she hopes this latest FDA decision will help propel larger efforts to start a trial. “The biggest hurdle for a trial is showing something is safe,” she says. “If we show this works in the girls, we hope a broader trial can start for kids with NPC.”

Thank you to the Hempels for all their hard work and PERSEVERANCE!! We love you Addi and Cassi.