PWS Grants & Discoveries, Message to FDA on Drug Development [Webinar]

On December 14, FPWR gave its quarterly webinar reporting on our PWS grants, and on recent research discoveries. In the webinar, which you can watch below, I recapped a recent meeting at the FDA, shared information on the new grants we've funded, and presented two recent advances in PWS research that are of interest to our community. You can download slides from the December 2016 FPWR Research Update webinar here

 

 

Talking to the FDA on PWS Drug Development

On December 13, 2016, I represented FPWR at the FDA Commissioner's Listening Session with the purpose of sharing with the FDA the priorities and concerns of the PWS community with respect to drug development. With thousands of rare disease groups to chose from, it was quite notable to have FPWR represent PWS at this session. Each organization invited to the session was asked to bring their priorities and concerns to the conversation. The concerns and priorities presented by FPWR on behalf of the PWS community included:

  • Priority: Improve instuitional knowledge of PWS at the FDA, and maintain an ongoing dialog as we accumulate new data on the patient perspective in PWS. (This is one initiative of the Prader-Willi Clinical Trials Consortium, PWS-CTC)
  • Priority: Improve interaction and meaningful engagement with FDA, throughout the drug development process, with particular emphasis on time-sensitive issues.
  • Priority: Work with the FDA to provide additional clarity on the FDA’s thinking regarding PWS drug development.
  • Concern: Perception of conflict of interest as patient organizations work with pharmaceutical companies to improve efficiency of clinical trials with respect to the needs and priorities of the patient community.

New PWS Studies

This webinar also discussed new studies funded by FPWR. Since its inception, the FPWR grant program has developed a broad portfolio of projects, investing over $8M into more than 125 PWS related research projects. FPWR has supported more than 100 new advances that have been published in the medical literature and has funded the development of key resources for use across the scientific community (new animal models, cellular models, bioinformatics). During this year's second round of grant applications, 8 grants were selected to receive funding, and in the webinar I reviewed each of these projects and their potential impacts.

Advances in PWS Research

Also in the webinar, I reviewed two advances recently published in the medical literature that are changing the PWS research landscape. The first publication, "Deficiency in the convertase PC1 impairs prohormone processing in Prader-Willi syndrome," suggests PC1 deficiency may explain many of the symptoms of PWS and provides a potential target for therapeutic development. The second publication, "Targeting the endocannabinoid/CB1 receptor system for treating obesity in Prader-Willi syndrome," identifies the endocannabinoid/CB1 receptor as target for treating obesity in PWS and suggests a new compound for treating PWS that has effectively reduced weight (fat mass), decreased hyperphagia, and improved metabolic parameters and energy profile in PWS mice.

For more details, please watch the full webinar above, or download the slides here

If you have questions on the content of the webinar, please email me.

Understanding PWS - Slide Deck 

Topics: Research

Theresa Strong

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Theresa V. Strong, Ph.D., received a B.S. from Rutgers University and a Ph.D. in Medical Genetics from the University of Alabama at Birmingham (UAB). After postdoctoral studies with Dr. Francis Collins at the University of Michigan, she joined the UAB faculty, leading a research lab focused on gene therapy for cancer and directing UAB’s Vector Production Facility. Theresa is one of the founding members of FPWR and has directed FPWR’s grant program since its inception. In 2016, she transitioned to a full-time position as Director of Research Programs at FPWR. She remains an Adjunct Professor in the Department of Genetics at UAB. She and her husband Jim have four children, including a son with PWS.

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