Patient testimonial – Marie G – March 23, 2024
My name is Marie, I am 36 years-old, and I leave in a small French village in the Vosges region. I have had lupus disease since I was 15 years-old. At that time, my lupus was quite mild. I only presented one blood symptom (a very low level of blood platelets count) and after a year under steroid therapy, everything was back to normal. Then, at the age of 25 years, I started to increasingly suffer from skin manifestations, arthralgia and muscular pain that progressively limited my daily activities. I had to stop working due to repeated disease flares, awaiting for the effects of corticosteroids to control the disease symptoms. At that time, I was still able to hide my lupus disease hallmarks: a touch of make-up was enough and perfect ! ( my skin lesions were effectively hidden under the face powder). At the same age, I became pregnant. I was so pleased ! I could not imagine this would happen to me, but a few months later I had a miscarriage; afterwards, the lupus signs worsened. For ten years thereafter, the number, the frequence and the severity of all lupus symptoms increased tremendously, as well as the intensity of my pains ( with extended deep red skin lesions on my face, chest, arms and consequent scar injuries on my fingers and arms, toes and feet, face; with articular and muscular pain ; with oedema when awaking). I remember that one morning, I suffered so much from diffuse articular pain, that I could not even walk to the bathroom. My skin was opening on its own, it oozed. Sometimes, I had the feeling that a welding torch was hurting and my eyes were burning so much that I could not even sleep.
Throughout the years, I did try a multitude of successive or combined therapies, namely hydroxychloroquine, azathioprine, disulone, photopheresis, lenanidomide, Belilumab, Thalidomide, Methotrexate, Isotretinoïn, colchicine, ustekinumab, alitretinoïn, mycophenolate mofetil, IL-2, mesenchymal stromal cells, cyclophosphamide. I thought that they could ease the symptoms. I was hoping to find the treatment which would control the disease and give me some relief. However, despite all the attempts, my kidneys, my lungs, my skin and my eyes conditions and functions continued to worsen dramatically. In addition, these treatments also accounted for a number of adverse events: allergic reactions, herpes zoster and other infections, including two episodes of pneumonia which led me to the intensive care unit and once to be under artificial sedation. Fortunately, my husband, my family, my friends and my employer I always supported we. Since December 2021, my health condition had even become worse. I suffered from several episodes of acute respiratory distress syndrome (ARDS) and, over the months, my respiratory capacity deteriorated, with need for continuous oxygen support at night, and sometimes for 24/24h.
I had to realize that, despite all treatment attempts, this damned lupus was taking over and was effectively killing me. I was scared of dying due to respiratory distress; my only wish, when I realized there was no more solutions, was to simply die without any pain; it was my only prayer: to avoid suffering.
In autumn 2022, by chance, I read an article in the press (in the Santé Magazine journal) and discovered that in Germany, , near Nuremberg, a few professors had treated five lupus patients and all of them had become symptom-free. I shared this paper with the medical doctors who were and who are still in charge of me. I wish to underline that this discovery was a tremendous hope for me.
After many discussions and a lot of perseverance and tenacity from my side towards the medical teams, and thanks to both the French and the German teams, I received this therapy in February-March 2023.
Today, it is one year since I have received the CAR-T cells therapy and my clinical evolution has been unbelievable. I feel much better than before. I have no more pain from my fingers or feet and I no need for oxygen support (neither in day time, nor at night). I can do exercice, which were before impossible for me: I can use my hands normally, I can take a shower and be stand-up, I can ride an electric bike, I can walk, I play music, I can cook being pain-free, and I can now wear shoes ! I can go to the toilet from my kitchen without being exhausted, I can walk several kilometers, I am not anymore afraid about my respiratory function, and I do not have to think before breathing. I am again discovering what is life without pain: it is fantastic!
For more than two years, I had completely stopped working and I have now returned to my professional life part time: being back into the world of work was a dream for me that has become came true.. A few weeks ago, I went as a tourist in Prague and could walk over 9 kilometres in one day : this was an effort that I couldn’t even have imagined nor hoped anymore to achieve before.
I remember that when my respiration started to improve and I could start breathing without the need to focus, something came up, which made me laughing and I laughed, I laughed, I laughed again until I started to crie without even thinking about my breath. That day, I realized that I did not remember when was the last time, I could have laughed with all my body muscles …that was several years ago, no doubt.
Today, I also have less fatigue; when I wake up in the morning, I feel that my body has rested. Plus, I have no more proteinuria (this is a good sign for my kidneys). Now, I can have some projects for my life.
I do not take any more specific treatment for lupus, as hydroxychloroquine, corticosteroids, or any immunosuppressives drugs. I continue to receive intravenous immunoglobulins when necessary and to perform my physical readaptation, twice a week. In order to exercise my lung capacity at best and to train my body to effort, I practice regular physical activities, namely walking, rowing and bodybuilding machine exercise and/or electric bike.
I do not know how to thank my nearest and dearest family relatives and my friends, nor how to express my gratitude for them: they have been with me unfailingly and relentlessly, when I suffered such hard times and they still support me. It is sometimes difficult to talk about your own feelings, but I would just simply tell them: thank you, thank you, thank you, thank you, I love you!
I also wish to thank, the CRMR MATHEC team (Centre de Référence des Maladies auto-immunes systémiques Rares – Maladies Auto-immunes et Thérapies Cellulaire) at Saint-Louis hospital, AP-HP, France, so sincerely as they worked throughout the all medical and administrative procedures so that I could effectively receive the CAR-T Cells therapy in Germany. Without them, I know that it would have been impossible to receive this treatment.
I also sincerely and deeply thank Professor Schett, Professor Mackensen and Doctor Müller from Erlangen who agreed to treat me, as well as every member of their medical teams who were so kind with me despite the language barrier.
I am certain: this CAR-Tcell therapy has saved my life!
Patient testimonial – Bruno G – June 14, 2021
The results of my mesenchymal stem cell (MSC) allograft are completely positive, even better than I had hoped. I think it is time for me to share the story of the allograft I was lucky enough to receive and how I am feeling today. The protocol that started in June 2019 will be finished in 2 months.
I am a retired, 64-year-old married man with three children. My passion was competitive sports (cycling in regional competitions). I had a healthy lifestyle, with no alcohol or tobacco, and was in perfect health. My scleroderma was diagnosed in 1989, after a power outage, when lightning struck a power station at the Electric Company.
This disease called Systemic Sclerosis has been my permanent companion for 32 long years, with many ups and downs. My psychological battle with the disease was replaced by acceptance in 2013. I decided to dare to refuse its fatality, that was my new state of mind. I have Raynaud’s syndrome, atrophy of the hands as well as esophageal, pulmonary and cardiac involvement.
To be able to participate in the MSC protocol, I stopped immunosuppressive therapy in June 2019 and have not taken it since.
The benefits of the allograft: My skin elasticity has clearly improved, so that I can now swim a little breast stroke in the pool (which was impossible before), can open my mouth more easily, am less tired (1 hour of exercise bike per day (fall, winter, and spring)), can garden and walk 3, 6 or even 7 kms. My general condition has also improved, although I must take a nap now and then. At the same time, there are still many things I am unable to do, like putting on my compression socks, for example. I can still feel the immediate improvements that I had from the stem cell injections, today, but then, my wife’s harvested cells were numerous and of good quality.
The protocol was performed by the team at the APHP St Louis Hospital, headed by Professor FARGE. There was also a local support team (because of Covid 19) at Montpelier St Eloi University Hospital headed by Dr. Alexandre MARIA in liaison with Paris.
I would like to take this occasion to sincerely thank them on behalf of my wife (my bone marrow donor), my daughters who have been at my side and shared my improvement, and myself, the lucky beneficiary of this treatment. I am also deeply grateful to Dr Sophie RIVIÈRE who made all of this possible, and who has always been extremely attentive, ever since 2009 when I began being followed in the Medical Unit in Montpellier. Especially since I am not any easy patient, and all of my hospital stays are extremely difficult for me to deal with. And finally, I would like to thank my wonderful wife who has managed some difficult situations, and has always been at my side. I would also like to extend my thanks to ASF (French Scleroderma Association) for its financial investment in medical research, in particular in the field of rare diseases, and for all of the work and efforts of the local and national members of the association. I have been a proud member of the ASF for the past few years.
Patient testimonial – Myriam F – May 31, 2019
Hello. Three months after my mesenchymal stem cell allograft, I would like to share my experience. I am a woman with a positive attitude, who has kept looking forward, and has always tried to do what needs to be done to limit the consequences of my disease.
I have had systemic Sclerosis since 2000. The skin is affected over my entire body, as well as all of my joints, with more or less severe limitations. My joints are painful, with calcific tendinitis (including the elbow which is blocked at 10% of mobility), my fingers are significantly retracted, and there is moderate oesophageal and pulmonary involvement. I require help to do most activities (bathing, dressing, cooking, cleaning etc). Because the different medications I was prescribed did not help, I only take paracetamol and an anti-acid when needed. Needless to say I was tempted by the proposition of this allograft despite many hesitations… So, at the beginning of February, with no particular problems or side effects, I received an intravenous infusion of cultured stem cells from my 26-year-old daughter for 1 hour. The next day, my hands were warm all day long, which was something unusual enough for me to notice, and the next day, the doctors confirmed that my skin elasticity had already improved.
That day I returned home, feeling a little lost in the face of these incredibly rapid improvements…
In fact, I felt as if I had finally been freed from a straightjacket. Obviously my joint movements were still limited, but I could move much more easily and rapidly, without pain!!!
After that I developed new muscle and tendon pains, because I was moving more and my muscles and tendons, which had been atrophied for 18 years, were reminding me of this… like anyone who starts playing sports again. Today, my body is stronger and the pain is gone so that I no longer need to take pain killers. And the icing on the cake is that the fatigue is gone. Before the transplantation EVERYTHING was an effort…And also I can now recognize myself again in the mirror, which is a great pleasure for a woman.
I would like to mention the following, because this could make certain patients hesitate to try this procedure. Since I live far from Paris, I was asked to agree to travel by plane, with transfer to airports in national health service-approved taxis, thus, I didn’t have to pay any of the travel costs. And since I am not autonomous, I travel on AIR FRANCE and am assisted. They pick me up with a wheelchair and manage my baggage. In this way I was able to travel alone, with no problem.
I would like to conclude by mentioning the kindness and availability of the entire medical team at the Internal Medicine Unit of the Hopital St Louis. They always answered all my questions, and there were many of them !!!!!
For the first time, I have a bit of hope that this treatment will be effective, I am more and more convinced that these stem cells will be the key to combating our defective cells, and I wish the best of luck to Pr FARGE and his team !!!
Patient Testimonial – Eric S – August 27, 2018
Three years after autologous hematopoietic stem cell transplant for systemic sclerosis
I was a person who was passionate about sports and life.
On January 6, 2014 I was admitted to emergency for a pulmonary infection and a bilateral pleurisy. Other attacks soon followed …
In June 2014, I was diagnosis with severe systemic sclerosis.
On October 5, 2014, I met Professor Farge from St Louis Hospital in Paris to sign-up for the autologous stem cell transplant program. I felt and knew this was my one chance. I am here to express my gratitude to the Saint-Louis team.
I could no longer walk, my body knew only suffering, like being entombed in a sarcophagus. Every breath required intense effort. At the height of my illness, I had a Rodnan’s score of 42. My organ involvement was complete (pulmonary, digestive, cardiac, and cutaneous). My hands were like those of a playmobil figurine, with finger ulcerations.
On November 4, 2014, I returned to Pr. Farge’s department at St. Louis Hospital to begin the autologous stem cell transplant treatment. The first step was to harvest my stem cells.
On December 26, 2014, I returned to St Louis Hospital in the hematology transplant department to receive a transplant of my own stem cells.
After the autograft, which was a very difficult experience, I was able to go home and start an intensive rehabilitation. I had to relearn everything, including to walk and grasp objects, but the important thing was that I was alive and with my family. In time, I gradually began to play sports again, always with objective of correcting my handicaps.
Today my skin has returned to normal, and I have a Rodnan score of 0. I was able to put my wedding ring back on without having to change the size. During my first stay in intensive care, my hands were so swollen that my ring had to be removed. A nurse with a lot of patience and experience removed it without having to cut it, but it was impossible for me to put it back on. Today I can put it on and remove it as easily as I did before.
I am here thanks to this precious gift from the entire St-Louis team. Thank you for this second lease on life and what better testimony than a little video showing what I am capable of now.
Eric S
Testimonial from patient – Eric L – January 12, 2017
Hello,
Since 2012, I have had severe systemic sclerosis (affected kidneys, lungs, heart and digestive system). After several months of difficult treatments with Endoxan and Myfortic, I was fortunate to benefit from a donor mesenchymal stem cell transplant treatment in the internal medicine department of Saint Louis Hospital in November 2016.
Very quickly I felt an improvement in skin flexibility, especially on the face and hands. For many months the impact of the disease had been very problematic, particularly the effects on the face, with occasional periods of severe acne breakouts that were very debilitating because of the stares – which were difficult to bear. I take Tétralysal which inhibits those unsightly, and painful, pimples. At the same time I hydrate my skin (face and body) with a dry oil (Monoi Florescence) from Lea nature that I find in supermarkets (8 €) or on the website. This oil is very effective, it makes the skin soft and penetrates immediately. The whole family has begun to use it. Be careful, it does not protect from the sun. I use it morning and evening, and I saw a clear before and after change.
I was fortunate to be surrounded by medical and paramedical teams who were very professional and attentive.
I try to keep up with my daily activities and time for recreation while reserving moments of rest, both in the morning and in the early afternoon.
I try not to think of myself as sick but as a full individual even if sometimes it is difficult. I wish you all a happy 2017 New Year. Let us not forget to plan projects but also that ensure that every day there is a small moment of joy in the present, that before the disease we took for granted and which today represents happiness.
E. L