Foundation for Prader-Willi Blog | Research

GDF15: A New Appetite-Regulating Pathway With PWS Therapy Potential

A series of recent discoveries have defined a new pathway for regulating appetite and satiety suggesting that a naturally occurring protein in our bodies could have the potential to be a safe and effective therapy for obesity. This protein, known as growth differentiation factor 15 or GDF15 (or MIC1), is mostly made in the liver but also in other tissues. It normally circulates in the blood and functions in many cellular processes such as inflammation, response to stress, energy expenditure etc. However, its levels can increase in several disease states including cardiovascular disease, liver disease, diabetes, dementia and especially advanced cancer.

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PWS Clinical Trial: Effects Of GLWL-01 On Hyperphagia, Phase 2

GLWL Research is now recruiting patients for its Phase 2 study to evaluate the efficacy, safety and pharmacokinetics of GLWL-01 in the treatment of patients with Prader-Willi syndrome. The study will compare the effects of GLWL-01 on hyperphagia as compared to a placebo. You can learn more about GLWL-01 in the video below, from the clinical trials session at the 2017 FPWR conferece:

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Dr. Nectow Researches Role of DRN Brain Region and Hyperphagia in PWS

Researchers throughout the world are hard at work trying to identify the biological mechanism(s) that drive hyperphagia (excessive eating) in PWS. One of these scientists is Dr. Alexander Nectow at Princeton Univeristy.

Dr. Nectow's group studies a region of the brain called the dorsal raphe nucleus (DRN). Two populations of neurons in the DRN help control food intake and can cause animals to either binge eat or to stop eating.

 

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Intellectual Disability and PWS Researcher Godler Awarded $500,000

FPWR–funded researcher Dr. David Godler, of Murdoch Children’s Research Institute and University of Melbourne in Australia, was recently awarded nearly $500,000 AUD from the Turnbull Government’s Medical Research Future Fund (MRFF). The award funds a four-year project to further his work investigating the significance of mosaicism to intellectual disability in pediatric disorders. Dr. Godler's work and that of other grantees is described in this news release.

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PWS Registry Data: Developmental Milestones in PWS [INFOGRAPHIC]

Since the launch of the Global PWS Registry in 2015, nearly 1,400 participants have enrolled. This month, the registry is highlighting results from the developmental milestones survey. This survey is aimed at understanding the age individuals with PWS reach various milestones. The survey includes physical milestones such as sitting, crawling, and walking, as well as social and cognitive milestones such as first words and reading.

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Dr. Dimitropoulos Uses Telehealth To Bring PWS Behavioral Therapies Home

There are numerous efforts in the healthcare community to increase health care accessibility, education, and support. One of these approaches is “telehealth”, the use of telecommunications methods such as computers and phones to deliver health related services and information. Dr. Anastasia Dimitropoulos at Case Western University is working on a project to determine the feasibility and effectiveness of using telehealth to bring behavioral therapies and interventions to families of those with PWS. They are working on two interventions.

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Developing a Clinical Trial Network for Prader-Willi Syndrome

The number of clinical trials for PWS continues to increase at a rapid pace. In 2017, clinicaltrials.gov listed 13 PWS clinical trials, compared with only two trials 10 years ago. With the increase in PWS clinical trials, our community is presented with a challenge we have never faced before: potential delays in trial completion due to patient enrollment. A small patient population coupled with a limited number of PWS clinical trial sites (see map below) increases the risk of inadequate recruitment and delay in the conduct of these trials. In response to these issues, FPWR is assessing the feasibility of building a database of PWS clinical care centers and providers in the U.S and Canada.

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PWS Registry Data: 61% Report Sleep Issues In PWS [INFOGRAPHIC]

Sleep is a common challenge among people with PWS. Whether it is daytime sleepiness, getting up at all hours of the night, early waking, or another sleep related issue, sleep challenges not only affect the individual with PWS but the entire family. The PWS Global Registry has gathered useful survey data on sleep issues in PWS, which is shared in the infographic below.

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PWS Clinical Trials Consortium: 2017 Update

The PWS-Clinical Trial Consortium (PWS-CTC) combines the expertise and perspective from multiple disciplines and stakeholders, bringing together members of academia, industry, and patient organizations from around the world in order to address the unmet needs of clinical trials. PWS-CTC was launched in 2015. This blog describes highlights of the consortium's work to date.

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Good's Research Explores Downstream Targets of SNORD116

Of the genes on chromosome 15 that are missing or inactivated in PWS, the SNORD116 gene cluster is known to be critical. But it's still unclear how disruption of SNORD116 contributes to the symptoms of PWS. In a new study funded by FPWR, Dr. Deborah Good is researching how SNORD116 regulates the genes NHLH2 and PCSK1, which will contribute to a better understanding of potential targets for new PWS therapies.

Topics: Research

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