There is much excitement and discussion in the PWS community about the possibility of a Phase 2 clinical trial for oxytocin currently in development of for PWS. Below is a list of questions that we have been asked, along with answers. 

What is oxytocin?

Oxytocin is a hormone that is normally produced by the pituitary, a small organ at the base of the brain. It is thought to play a role in social behavior and bonding, and is known as the “hugging hormone” or “love hormone”, but its biology is actually much more complex (The Hard Science of Oxytocin)

Why is oxytocin potentially important in PWS?

People with PWS have been shown to have a decreased amount of oxytocin-producing neurons. Low levels of oxytocin have also been implicated in autism-related behaviors. This has led to interest in administering oxytocin via nasal spray to people with PWS to see if it has a positive effect on PWS-related behaviors such as temper outbursts, OCD-type behaviors, or food seeking. 

Has oxytocin been studied before in PWS?

Yes. Results of previous studies have been mixed. A 2014 study by Einfeld et al showed little positive impact and an increase in temper outbursts. However, a 2011 study by Tauber et al showed improvement in several behaviors. A Phase 2 study of an oxytocin analog (Ferring Pharmaceuticals) is completed but not yet published. Additional ongoing / planned studies include administration to infants, children and adults with PWS (see this recent blog for an overview of oxytocin studies in PWS‎).

Why has there been so much discussion about oxytocin lately?

There is currently a Phase I oxytocin trial underway at the University of Florida, University of California Irvine, and Kansas University Medical Center. Although the trial is not complete and the data has not been analyzed, preliminary anecdotes are encouraging.

Is FPWR planning to provide funds for the upcoming Phase 2 trial?

Yes. FPWR has a strong, long -term commitment to supporting the full evaluation of oxytocin as a therapeutic for PWS. There is great community interest in moving forward with this oxytocin study, and as a parent-driven organization, FPWR has set aside a significant amount of existing funds to ensure that this study is fully funded.  In addition, we will continue to raise money for this trial as it develops, and for other important studies of new therapeutic agents for PWS.

How much will this Phase 2 study cost?

The ultimate cost of the study is currently unknown as it is still in the planning stages.  However, it will likely be an expensive study, which that will require a community-wide effort to ensure success.

When will the funds be released to the investigators?

FPWR’s policy is that funds are released to studies that have passed a formal peer review process established by our Scientific Advisory Board. This policy ensures that all studies funded by the FPWR are of the highest scientific quality, and are thus most likely to produce scientifically valid results that will lead to advances in the field of PWS research. This procedure is not unique to the FPWR or to the oxytocin study, and is standard for most funding organizations. The policy also ensures that as donors, your money is well invested. Once the protocol is written and reviewed, with satisfactory resolution of any concerns brought out upon review, the funds can be released.

Aside from this clinical trial, is the FPWR supporting research into the effects of oxytocin in any other way?

Yes. FPWR has funded and continues to fund both basic science studies to understand how oxytocin works, and human clinical trials to evaluate its potential as a therapy. To date, this has included funding of studies in PWS animal models to learn how oxytocin might best be used in humans, funding of Phase 1 and Phase 2 clinical trials of oxytocin in children and adults with PWS, and funding basic biology of oxytocin in individuals with PWS to assist in interpreting clinical trials. (see blog for details) ‎

Why doesn’t the FPWR put all of its resources into the Phase II trial only?

If oxytocin has a proven positive effect in PWS, it is vital that multiple studies confirm it. This will ultimately help our community gain access to oxytocin for the treatment of PWS. It is also critically important to continue to support other areas of research, including upcoming clinical trials for other promising interventions, as we cannot predict the results of any one study.

Can all of the money that I raise for FPWR be restricted to the oxytocin trial?

At this point, the Phase 2 trial is at a preliminary phase; the Phase I study is not complete, and the protocols for the Phase 2 study are in early development. FPWR’s policy is to restrict funds only to studies that have been reviewed and approved by our Scientific Board for the reasons outlined above, so the Phase 2 trial does not qualify for restricted funding at this time. The FPWR Board has set aside funds to ensure that they are available for the upcoming study. FPWR is committed to funding sound scientific research into oxytocin as a potential treatment for PWS, and thus any funds raised are supporting these efforts, as well as the many other important areas of PWS research that are ongoing. 

I have more questions about oxytocin!

Please contact Susan Hedstrom, Executive Director of the FPWR, with any additional questions.