173 world-renowned researchers and clinicians
in the field of Progeria converged from 14 countries to meet in Cambridge, MA in May for PRF’s 8th
International Scientific Workshop entitled, Across the Table, Around the Globe
. Twenty-five speakers and 46 poster presentations showcased important scientific findings, presented the progress in translating bench research to potential therapeutic treatments, and inspired future collaborations between the research and medical communities. The breadth and scope of work is expanding every year, as experts work towards tirelessly to find a cure for progeria and unlock the mysteries of heart disease and aging.
The Meeting Agenda at a Glance
Children & Parents Living with Progeria
: Toddlers & Teens
on landmark pre-clinical research findings: Vicente Andrés García, PhD,
Centro Nacional de Investigiones Cardiovasculares, Spain
Clinical Outcomes and Biomarker Discovery in HGPS
Moderator: Monica Kleinman, MD;
Boston Children’s Hospital, MA
Pharmacological Intervention in HGPS and Aging Models
Moderator: Brian Kennedy, PhD
; Buck Institute, CA
Evenings 1 and 2 were capped off with Poster Presentations
of topics ranging from new potential therapeutic targets for treatment, to potential biomarkers and technology for non-invasive measures used to determine efficacy of treatment, to new discoveries in atypical progeria patients, to recent findings from clinical trials.
Molecular and Cellular Mechanisms in HGPS and Aging
Moderator: Maria Eriksson, PhD
; Karolinska Institute, Sweden
Moderator: Tom Misteli, PhD,
National Cancer Institute, MD
: The Next Phase - Strategies for the Future; Science and Medicine Coming Together.
Scientists presented new data in support of over 20 possible therapeutic avenues for HGPS
, a significant expansion over the prior meeting in 2013. There were wide-ranging discovery strategies that included RNA therapeutics, lamin A post-translational processing pathways, small molecules discovered as a result of high throughput screening, agents adapted from the general aging field such as a senescence regulator, telomere-based therapies; regulators of autophagy and antioxidants; treatments rooted in LMNA structure and function, and a strategy for improving genomic stability. If all of these research efforts yield just one effective treatment for children with Progeria, then these global research efforts will be a tremendous success.
The Workshop kicked off with a Family Panel
, moderated by Leslie Gordon, MD, PhD
(PRF). Researchers had the unique opportunity to meet some of the people their work could help: Meghan Waldron,
her brother Ian
and her parents Tina
; Carly Kudzia
, along with her parents Heather
; and Zoey Penny
with her mother, Laura
. Meghan spoke about her experience living with Progeria and read a poem recently published in the magazine Stone Soup. Parents answered questions from the audience and thanked the research and medical communities for their work towards finding a cure. To top it off, Carly and Zoey led the entire group in a lively game of Simon Says (yes, the scientists jumped up and down while rubbing their heads!)
In a riveting Summation Session, Judy Campisi, PhD
(Buck Institute), Mark Kieran, MD., PhD
(BCH) and NIH Director Francis Collins, MD, PhD
spearheaded a lively discussion about how to work as a community to guide the future for progeria research. As a fitting closure to the conference, Dr. Collins
performed the song Dare to Dream,
which he wrote after speaking with Sam Berns at TEDMED 2012.
Overwhelmingly Positive Feedback!
With a 98% approval rating overall, the most common remarks from attendees reflected excitement about data sharing and collaboration leading to new ideas for future work. Here are a few attendees’ comments:
“The meeting was so much better than I had hoped for -- I learned a tremendous amount, and felt very welcomed into your research community.”
“What a spectacular meeting!”
“I wanted to thank you for giving us the opportunity of being part of such a wonderful experience. Everybody was very committed to their work and to the families of these children, and that is admirable.”
Many thanks to our Workshop Organizers and our Workshop Supporters
Images of Francis Collins and Meghan Waldron courtesy of the Progeria Research Foundation