Last Wednesday, the Scleroderma Foundation announced a research breakthrough in the search for a scleroderma cure. A team of University of Pittsburgh Medical School researchers, including the Foundation’s board vice chair Dr. Carol Feghali-Bostwick, has discovered a peptide that blocks skin and lung fibrosis.
The so-called E4 peptide was discovered serendipitously when the team was investigating agents that promote fibrosis—thickening of connective tissue. This particular peptide (a chain of amino acids that make up part of a protein) blocks fibrosis in mice. Testing the discovery further, the investigators found that E4 also reduces fibrosis in human skin samples maintained in the lab.
Exciting news, since lung fibrosis is the leading cause of death in patients with scleroderma, and excessive skin thickening is at the core of the disease process. The discovery of E4 is not a cure, but an important step forward in that quest.
I’ve been thinking about this announcement all week and wondering: Is it really possible that a cure for this disease could be found in my lifetime?
It’s an idea that I have not let myself entertain, for decades. Scleroderma is disease that affects about 300,000 people in the U.S., just under one percent of our nation’s population. It seems like a lot of people to me, but not enough to attract significant research dollars, compared to a killer like cancer.
According to language in the Scleroderma Research and Awareness Bill (HR2408 and S1545), which has been pending in Congress since 2009, NIH funding of scleroderma research was then about $20 million annually. The Scleroderma Foundation and Scleroderma Research Foundation each raise and distribute a little more than $1 million for research each year. So, that’s around $22 million, give or take. Just about the same as Red Sox first baseman Adrian Gonzalez will earn this year.
Would that our nation made health as high a priority as sports franchises. But I digress.
For my own part, I’ve tried to help the search for a scleroderma cure by participating in studies ever since I was diagnosed 25 years ago by Dr. Virginia Steen, a leading researcher in the field, then at the University of Pittsburgh, now at Georgetown University.
Data about my blood samples are in research databases around the country. I contributed a tissue sample from my placenta when I gave birth to Emily. I’ve helped to test new drugs for Raynaud’s (I think I had the placebo) and have allowed images of my hands, pre- and post-surgery, to be published in medical journals.
I’ve also explained my case history to numerous rheumatology fellows at Boston Medical Center over the years, and I’ve talked to classes of second-year BMC med students, helping them to learn how to diagnose scleroderma.
I don’t often fit the criteria for research trials at this point, because I’ve had the disease for so long. And I don’t participate in any research that involves procedures that sound painful or too risky. I’ll leave those to someone braver. But I’m glad to help when I can. It’s the least I can do to try to find a cure and improve the chances that some new patient suffering from the disease will be diagnosed sooner, with better odds of survival.
As for the E4 peptide discovery, all I can do is hope that the progress of my disease continues slowly enough, and that the research proceeds apace, so that maybe, just maybe, as my health gets worse, there will be better treatment options available when I need them.
And when I finish this post, I’m going to make a donation to both the Scleroderma Foundation and the Scleroderma Research Foundation. I don’t really care who claims credit for the next breakthrough. I just want us to get there, already.
Evelyn Herwitz blogs weekly about living fully with chronic disease, the inside of baseballs, turtles and frogs, J.S. Bach, the meaning of life and whatever else she happens to be thinking about at livingwithscleroderma.com.
Hi Evelyn,
I just found your website and have read all of your postings. Your story is also my story and you write about it so well. So good to know someone else gets it. I even have a hula finger (actually several) and a golden retriever named Lily.
Cathy
Thanks, Cathy! Nice to meet another kindred spirit. Ginger says hello to Lily!
Hi Evelyn: My daughter age 34, was diagnosed about 5 years ago with Raynaud’s Disease first, Then with tests and coming home from Kitchener/Waterloo (where there was no one there that could help her), she was diagnosed with Scleroderma. Hers has attacked her lungs, skin hard and very white, (we are white anyways) but it looks like its been bleached, pains, digestive problems, fingers curling, pain in her back and body, ulcers on her knuckles of her hands. and etc etc. I pray for a cure for her disease. My husband had a auto-amunion disease called Crohnes and it and hospital pneumonia killed him in 2007. I pray there is a cure for her. She has no boyfriend, no relationship with a guy, and probably will never had children. I bless all of you with Scleroderma. LETS HAVE A MIRACLE AND A CURE FOR SCHOLERODERMA.
Lil, I hope your daughter is able to get the care she needs. We all hope and pray for a cure. Blessings to you and your family.
I lost my very best friend October 2012 due to complications as a result of his having scleroderma. He had this chronic illness for more than 25 years. I pray for a cure…..this new break through gives me hope for those struggling with this awful illness. God bless you and all of those who suffer whether the patient, family or caregiver.
I’m sorry to hear of your loss. Thank you for your good wishes.
I don’t have scleroderma, however I walked along with my sister as she fought her battle for 18 years. I read and sometimes comment on a Facebook page, but it really isnt my place. I pray everyday for a cure for this disease.
Thanks, Barbara, for sharing. Maybe in our lifetimes, a cure will be found.
Barbara, of course it is your place. I don’t have it either, my oldest daughter 34 has it, but we all learn from each other and you telling people about staying close to you dear sister for 18 years, holds dear, to our hearts. I hope everyone has some one like you to love and care for them and stay right by her side. Thank you for printing out your story. and God Bless Your Dear Sister, and you Barbara, for all the love you shared together.
Thank you for allowing me to express on here… I dont want to be a downer, but I do think I know things that might help others. I.e. ask for vitamin shots, as Janice’s stomach became scarred and to help with malnutrition. I was with Janice the last days…
Barbara: See you do know things that alot of people don’t know, my daughter doesn’t take vitamins, what kind of vitamin shots are available. And I’m sure, you were there for Janice the last days, You were there for 18years!!! And Janice knows how much you loved her and knew you were with her to comfort and support and love her. Tell us all what you know, what helped her feel abit better, tell us anything you want. You and I and many others who are care/love supporters and those ill need to hear your journey through the painful 18 years.
Very sincere Lil
G’day Evelyn, Your blogs give me hope. I was told I would be lucky to survive a year or two with my condition. I have now been living with Systemic Scleroderma for 5 years.
My left hand a permanent claw now, my right not as bad. My strength or lack there of has been surprising. Lifting even a couple of shopping bags is a trial, lucky I live in a small town as they all know I need assistance even if I do protest. They even don’t get frustrated when I take for ever to get change from my purse. I do need to move to a capital city so I can start to look into treatment, I gave it up after my first specialist nearly gave me kidney failure with incorrect medication. I’m 38 and going to live for at least 20 more years, that would make me happy. But to live without Scleroderma would make me ecstatic.
G’day to you, too, Liza! I’m glad my blog has given you some inspiration. And I do hope you get appropriate treatment. I think there is a great need for physician education about scleroderma, especially for doctors who are not practicing near major teaching hospitals, where much of the research is being done. Good luck with that, and I’m glad for you that you have kind neighbors who help you when your hands are too tired. I agree, we would all be ecstatic to live without this disease! In the meantime, we just have to do our best. Thanks for sharing.
Hi Good luck and am praying for you and all scleroderma sufferers.
I lost my younger brother – he battled for 3 years and lost in 2009 due to complications of the lungs. He was mis-diagnosed for over a year or so and when the doctors finally agreed it was Sceloderma it was too late. as I read the research and university names I realize how help was so close – Walied (that’s my brothers name) did his MBA and previously started on PhD @ Penn State Univ – my other brother lives in Boston – when he got to Boston but when He got there, it was too late for him to take part in any research!
He was full of life and hope – just moved to homeland Sudan to set up a business – as he was suffering from what he thought at that time was diabetes – developed during his MBA years- unfortunately the Sceloderma – was never detected.
All the doctors worldwide Sudan, london Dublin Saudi misdiagnosed him! even when a doctor in Saudi guessed it was Sceloderma a leading UK rheumatologist laughed it off (really) saying there was a zero possibility . It was had we listen to him maybe Walied would have had a chance.
I hope the miracle is found TODAY!
There is too little research on this disease. I am praying for everybody – I hope a cure will be discovered Evelyen and thanks for sharing.
Thanks for sharing your brother’s story, Lubna. In addition to a cure, we need better physician education about scleroderma! So many of you have written about misdiagnoses leading to unnecessary suffering.
Hi,
Could you please tell me where I can find the above article on the breakthrough for skin and lung fibrosis. I like to share the facts with my doctors.
Thanks
Desiree
Hi Desiree,
You can find a short summary of the research here: http://www.niams.nih.gov/News_and_Events/Spotlight_on_Research/2012/endostatin_fibrosis.asp
I wrote this post when the study was first publicized in the summer of 2012. Research continues.