First-ever Therapy to Demonstrate Survival Benefit in Progeria

April 24, 2018: A new study published in The Journal of the American Medical Association (JAMA) reports that lonafarnib, a farnesyltransferase inhibitor (FTI), helped extend survival in children with Progeria. Authors from Boston Children’s Hospital and Brown University tracked more than 250 children from six continents to demonstrate a link between lonafarnib treatment and extended survival.

Click here to read the abstract of the study

मीडियासाठी: 

• Press release
• इन्फोग्राफिक 
• B-roll video

चे परिणाम the clinical drug trial for children प्रोजेरिया मध्ये आहेत आणि ते अधिकृत आहे!   The study using lonafarnib, a type of farnesyltransferase inhibitor (FTI) originally developed to treat cancer, supports extension of lifespan in children with Progeria. In the clinical trial, 27 children with Progeria received oral lonafarnib twice daily as a monotherapy. The control arm of this study consisted of children with Progeria with similar age, sex and continent of residency as the treated patients, who were not part of the clinical trial and therefore did not receive lonafarnib. The results demonstrated that treatment with lonafarnib alone compared with no treatment was associated with a significantly lower mortality rate (3.7% vs. 33.3%) after a median of 2.2 years of follow up. Results of the study, which was प्रोजेरिया रिसर्च फाउंडेशनद्वारे निधी आणि समन्वयित, were published April 24, 2018 in The Journal of the American Medical Association.

Gordon et. al.,Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome, JAMA, April 24, 2018 Volume 319, Number 16Results Bring New Hope and Optimism
The children traveled to Boston Children’s Hospital to receive comprehensive medical testing and study medications. All received oral lonafarnib, an FTI supplied by Merck & Co. “This study published in JAMA shows evidence that we can begin to put the brakes on the rapid aging process for children with Progeria. These results provide new promise and optimism to the Progeria community,” said Leslie Gordon, MD, PhD, co-founder and Medical Director for PRF, and lead study author.

“At PRF, we are working tirelessly to fund new scientific breakthroughs for children living with Progeria.  This study shows us that the clinical trials conducted today are our best hope for saving for these children in the future. Based on the promising results from this study with lonafarnib, we feel a stronger sense of urgency than ever. Today’s milestone marks a new beginning for these children and their families. Every day and every moment count. PRF’s goal is to find the cure for Progeria and this study brings us one more step towards that goal,” said Meryl Fink, President and Executive Director, PRF

Media Coverage

Here is a sampling of the press coverage received:

• MedPage Today
  Drug Could Make Progeria Manageable
• बोस्टन ग्लोब
  A pediatric researcher whose son died of progeria sees promise in new treatment April 24, 2018
• Brown News
  Research suggests promising drug therapy for premature aging disease April 24, 2018

“This is a story that is inspiring on many levels. There is the potential to transform the lives of a group of very special children. But it is also exemplary of the scientific method gone right, where rigorous basic science and a smart application of an existing drug combined to produce outcomes that are genuine breakthroughs.”

– F. Perry Wilson MD, MSCE, Yale School of Medicine, MedPage Today

आम्ही या अद्भुत दिवसापर्यंत कसे पोहोचलो?
Following the 2003 discovery of the gene mutation that causes Progeria, PRF-funded researchers identified FTIs संभाव्य औषध उपचार म्हणून. प्रोजेरिया-उद्भवणाऱ्या उत्परिवर्तनामुळे प्रथिनांचे उत्पादन होते प्रोजेरिन, जे सेल फंक्शन खराब करते. शरीरावर प्रोजेरिनच्या विषारी प्रभावाचा एक भाग "फार्नेसिल ग्रुप" नावाच्या रेणूमुळे होतो, जो प्रोजेरिन प्रोटीनला जोडतो आणि शरीराच्या पेशींना नुकसान होण्यास मदत करतो. FTIs प्रोजेरिनवर फर्नेसिल ग्रुपचे संलग्नक अवरोधित करून, प्रोजेरिनची हानी कमी करून कार्य करतात.

अधिक अभ्यास तपशीलांसाठी, Click here for press release

“Demonstrating effectiveness of treatments in this small population of children with this rare fatal disease is a major challenge. Thus, I’m particularly encouraged by these latest finding.”

– Dr. Francis Collins, Director of the National Institutes of Health

प्रोजेरिया सामान्य वृद्धत्व प्रक्रियेशी जोडलेले आहे
Research shows that the Progeria-causing protein प्रोजेरिन सामान्य लोकांमध्ये देखील तयार होते आणि वयानुसार वाढते. संशोधकांनी FTIs च्या प्रभावाचा शोध सुरू ठेवण्याची योजना आखली आहे, ज्यामुळे शास्त्रज्ञांना लाखो लोकांना प्रभावित करणाऱ्या हृदय व रक्तवाहिन्यासंबंधी रोग, तसेच आपल्या सर्वांना प्रभावित करणाऱ्या सामान्य वृद्धत्वाच्या प्रक्रियेबद्दल अधिक जाणून घेण्यास मदत होऊ शकते.

Thank you ALL – आम्ही तुमच्याशिवाय हे करू शकलो नाही!
One of the main reasons we achieved these breakthrough results is because of the tremendous supporters who provided funding and other support, helping to get us one step closer to achieving our ultimate goal – a cure for Progeria.