To our European Friends

Pitolisant (Wakix) was approved by the European Medicines Agency in fall 2015 (http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002616/human_med_001955.jsp&mid=WC0b01ac058001d124) for the treatment of narcolepsy. The drug is expected to be available in Europe in fall of 2016. As far as we know, the makers of the drug currently have no intention of testing the drug in the PWS population. They also have not begun the process of bringing pitolisant to the United States.

Pitolisant represents the first of a new class of drugs. This means that there is very little clinical experience with the drug. Early evidence suggests, however, that it has an excellent safety profile.

Pitolisant targets the histamine 3 receptor (H3R) and is an inverse agonist. The H3R is found primarily in the nervous system and gastrointestinal tract. It has many functions throughout the body and its role in health and disease is under active investigation. Thus far, scientists know that the H3R regulates many neurotransmitters (brain chemicals) and plays a role in the regulation of sleep and hunger. Scientists learn and publish more about the H3R every month.

Recently Holger Stark, PhD of the Heinrich Heine University of Dusseldorf in Germany presented his research on pitolisant and the H3R at the International Prader-Willi Syndrome Organization meeting in Toronto. Dr. Stark is an international leader in research on the H3R and his presentation raised the possibility that pitolisant may benefit individuals with PWS.

We at the Chion Foundation hypothesize that pitolisant may be uniquely positioned to meet the needs of patients with PWS. We propose that it will normalize sleep, behavior, and cognition. We are also eager to explore whether or not it will relieve other burdens of the complex syndrome.

You will have access to this drug before anyone else. We suggest that, as soon as possible, you initiate a conversation with your child’s doctors to see if pitolisant is a reasonable option for your child. While pitolisant is approved for adults, a pediatric trial is currently underway (https://clinicaltrials.gov/ct2/show/NCT02611687).

If your doctor is open to trying pitolisant, please consider sharing your clinical experience (positive and/or negative) with us. You can do this via email to info@chionfoundation.org.

Even better, we ask that you enroll in TREND Community (email maria@trend.community to request an invitation) so that you capture as much of your experience in as systematic manner as possible. We are asking for 15 minutes of reporting each weekend. We will also ask that you upload sleep study results and report body temperature, body mass, and percent body fat.

While we will welcome any document measurements, we are encouraging people to purchase:

1) A Withings scale (https://www.amazon.com/Withings-Composition-Wi-Fi-Scale-Black/dp/B01F3LJ2RW/ref=sr_1_2_s_it?s=hpc&ie=UTF8&qid=1469715771&sr=1-2&keywords=withings+scale). Have your child weigh him/herself nude first thing in the morning after going to the bathroom. (You will have to set up the scale on the cloud so that it stores your data.)

2) A thermometer (https://www.amazon.com/gp/product/B00Y2CJWKQ/ref=oh_aui_detailpage_o00_s00?ie=UTF8&psc=1). Take your child’s temperature in the morning before s/he drinks anything. Write down the temperature and the date and if you think there is a problem (sickness?) make a notation of it.

Start now

Please bring up the topic of pitolisant to your physicians. If they have any questions, they can feel free to contact our Chief Medical Officer, Charles Johnston, MD at cjohnston@chionfoundation.org.

If your physician thinks that it is reasonable to provide pitolisant to your child, then what would be the most beneficial for our community is if you can start capturing baseline data in advance of initiating pitolisant. This would mean enrolling in TREND (link here), buying the scale and thermometer listed above, and reporting on your child’s sleepiness, cognitive function, and mood.

TREND will also ask you to:

• Upload the results of a recent sleep study and, if your child has not had a sleep study in the past year, to schedule one before starting pitolisant and upload the results.

• Obtain a speech assessment if your child sees a speech therapist.

• Obtain fasting triglyceride levels (blood test).

Finally, we have reason to believe that pitolisant may improve processing speed, and possibly attention and memory. One way to capture this is via a brain training program called Lumosity. Please consider purchasing a subscription to Lumosity (www.lumosity.com) and have your child start the program. Lumosity suggests three 15 minute sessions per week. It would be ideal if your child could do this or even more. Remember, you cannot play with your child. The program will be capturing data and you want to make sure that the data reflect your child’s ability and not yours.

Please email us at info@chionfoundation.org if you have any questions. We have approached the manufacturer of pitolisant and he has declined to study the drug in our rare disease population. Absent a license to perform a clinical trial, we have decided to gather clinical experience with pitolisant. Please help us if you are able.

The Pitolisant Program has two main goals:

1. Determine if pitolisant can relieve the burden of PWS

2. If pitolisant is beneficial for patients with PWS, we will work to provide access in the United States

This fall we will provide funding for five families to travel to Europe to receive pitolisant. These patients are currently documenting baseline information and will record the effects of pitolisant on this baseline data. If pitolisant is able to effectively normalize many of the symptoms of PWS, then the Chion Foundation will work to provide access to pitolisant in the United States.

We will work towards a compassionate use indication from the FDA. We will also attempt to persuade the manufacturer to bring pitolisant to the US.

Subscribe below if you would like to be updated on this process.