All of the work of SFLF is ultimately for the patients—past, present and future— who have been and will be treated successfully with adult stem cells. We recognize and appreciate the contributions made to the field by these patients. These unlikely heroes didn’t set out to change the world, but are changing the world of science and medicine everyday with their remarkable stories of courage and triumph in the face of adversity.
We have had the opportunity to recognize some of these heroes at our Vatican Conferences and to feature some of their stories in our Life Lines newsletter. We have shared their stories below, and urge you to read more about their inspiring journeys.
Heather Abrams was diagnosed with Hodgkin’s lymphoma in 1999 when she was 15. She was treated with standard COPP/ABVD protocol and radiation and attained remission quickly. However, in July 2000, she relapsed. She was referred to Dr. Mitchell S. Cairo at Columbia University Medical Center and was treated at the Morgan Stanley Children’s Hospital of New York.Dr. Cairo treated Heather with three rounds of ICE chemotherapy protocol, radiation, and an autologous stem cell transplant that was the same protocol offered at other institutions. However, the reason Dr. Cairo was chosen to treat Heather was that he also proposed the use of Rituximab infusions and most importantly that Heather receive an allogenic umbilical cord blood transplant. Because Heather was a full-size adult at age 17, securing an appropriate sample was difficult. After much searching of the existing cord blood bank databases, a sample was found in Milan, Italy, and the transplant occurred in June 2001.Heather is a graduate of the University of Vermont with dual degrees in Animal Science and Geography, and in 2011, worked as a nature educator at the South Fork Natural History Museum in Bridgehampton, New York, led spinning classes at the local gym (despite having been treated with bleomycin), and had recently applied for admission to veterinary school.
Roxane spoke at the 2013 Vatican Conference and currently serves as a Student Ambassador for the Cellular Age
Roxane Beygi was 14 years old in 2007 when she was diagnosed with multiple sclerosis (MS). Prior to being afflicted and diagnosed with MS, Roxane was a straight ‘A’ student, an avid swimmer and tennis player. During her battle with MS, she was put on conventional treatments for MS designed to slow the progression of the ailment. Despite the best attempt and care of the treating physicians, her fanatical adherence to medical orders, and maintaining injection and medication schedules, Roxane’s condition deteriorated.
In 2009 Roxane was evaluated for autologous stem cell transplant and her treating physician referred her for the procedure. In September 2010, she underwent a stem cell transplant at the Northwestern Medical Center in Chicago. Three months after the procedure, she started college, where she was also able to restart regular exercise. Roxane is a Student Ambassador and we thank her for her service dedicated to spreading the word about the benefits of adult stem cell therapies.
In 2004, Michael Carlat suffered a major heart attack. With a young family, Michael did not want to die. His doctors had tried multiple stents and an angioplasty without much success. Michael was placed on a heart donor list which had 5,000 people ahead of him. Being a diabetic further decreased his chances of finding a match. Instead of waiting for a heart donor that might never come, Michael underwent a transplant utilizing his own adult stem cells. Today, the part of Michael’s heart that was dead is now alive and healthy. Recently, when asked what he thought about adult stem cells, he stated, “I am living proof. Without my own stem cells, I wouldn’t be here right now. They saved my life.”
Ciaran Finn Lynch
Ciaran Finn-Lynch made medical history in March 2010 as the first child in the world to undergo a pioneering stem cell-supported tracheal transplant. Ciaran was born under dire circumstances. He was diagnosed with long segment tracheal stenosis, a condition which leaves sufferers with a very narrow windpipe and difficulties breathing. The transplant, performed at Great Ormond Street Hospital in the United Kingdom, used adult stem cells to rebuild the airway in his body. Once the trachea was transplanted, researchers continued to infuse growth proteins into the organ to continue stem cell generation. Because this was an emergency procedure, this technique allowed for researchers to transplant the organ faster instead of having to wait for the organ to grow outside of the body.When we saw Ciaran in Rome, he was doing well post-transplant. Doctors said he has grown 11 centimetres in height and has returned to school. The doctors on his transplant team believe that the success of Ciaran’s procedure points to a future in which these cells can be used to grow more complex organs which are made up of different types of tissues.Read more about Ciaran’s treatment »
Watch a video about Ciaran’s treatment »
Jim spoke at the 2013 Vatican Conference, and contributed an article to the October 2013 Life Lines newsletter
James (Jim) Danhakl had no way of knowing what his future would hold when he stepped out of bed one morning in the summer of 2009 and noticed an odd tingling sensation in his toes. Jim was eventually diagnosed with a rare and malignant form of chronic inflammatory demyelinating polyneuropathy (CIDP), an autoimmune disease that attacks the peripheral nervous system. There is no cure and only limited treatment for CIDP. Over the next five months, Jim’s condition dramatically worsened.
Jim stumbled upon an online support group discussing a stem cell transplant clinical trial being conducted at Northwestern University and because of the urgency of his condition, was fast tracked into the clinical trial. The stem cell transplant was designed to ‘reboot’ his immune system. First, his adult stem cells were harvested, and then his defective immune system was destroyed with chemotherapy. His adult stem cells were then reintroduced to regrow a new immune system, genetically identical to the original, but without the bad habits it learned in its ‘previous’ life. Within weeks, Jim was taking his first steps again. A few years later, Jim has regained nearly all of his normal activities and is once again leading a full and complete life.
Read about Jim’s story in his own words.
I had always had robust health. I spent much of the past 20 years as a Navy fighter pilot and had always taken care of myself. That summer I had commenced an exercise program that involved weight training, aerobics and power walking 50-60 miles per week. I had not taken a day off in several weeks. I decided I would do one last hard day before I granted myself a day of rest. I lifted weights for 2 hours, rode the bike then power walked a 26 mile marathon. When I hit the bed that night, I felt a fatigue deeper than anything I had ever experienced. I had just “overdone it”. I figured it would all be better with a couple of days rest. I was wrong.
Over the course of the next two weeks the tingling feeling changed to numbness and began to creep up my feet. I felt a loss of coordination in my knees. I was losing physical strength. It wasn’t until the 3rd week, when I began to feel the tingling in my fingertips that I became really concerned. I realized that whatever was happening was systemic and not just a training injury. I went to my family doctor who referred me to the local neurologist. It took two weeks before I could get an appointment. The neurologist diagnosed me with Guillane-Barre Syndrome, (GBS) an acute autoimmune disease that demyelinates the peripheral nervous system. At least they knew what was wrong with me. It had a name. We could treat it.
I was admitted to the hospital and spent the next five days receiving daily infusions of Intravenous Immunoglobin (IV-IG). While in the hospital I researched GBS. I was relieved to learn that most people who get hit with this awful disease recover from it. Although, I also realized that my disease profile was different. Where GBS strikes within hours and days, hitting its maximum within a week or two – my symptoms had been going on for over a month and were only increasing. I read that if GBS symptoms persist, the disease can sometimes take on its chronic form – CIDP. CIDP is more rare, and while usually not fatal, is also a permanent disease, much like multiple sclerosis (MS) where patients go up and down, but over time mostly down.
After I left the hospital, I continued to decline. By the two-month point I was having difficulty just walking around the house. I could no longer drive. I was losing sensation in my arms, hands and torso. My sense of taste was diminishing; everyday items began to feel heavy. My world was becoming small.
I cannot begin to describe what it is like to lose so much so fast. So much of our daily world is experienced by movement and by our senses. It felt like I was being robbed of my very being. I would go to bed every night, telling myself that I would be better in the morning, but instead it was worse. I could not sleep this away, pray it away, eat it away or “positive attitude” it away. I was being steamrolled, crushed. I had to find help.
I began to seek out “big league” doctors at major teaching hospitals. But they had no answers either. I looked for answers myself. I found a support blog online for GBS and CIDP patients. Reading these personal accounts was devastating. Most people over a period of years decline to a point of severe disability. Some die from complications. All have their lives dramatically altered. There are no cures or even treatments that change the long-term prognosis.
I was beginning to despair when I found a thread titled “Stem Cell Transplant for CIDP”. The thread chronicled the journey of “Alice” a CIDP suffer and her steadfast determination to undergo a new and possibly game changing experimental treatment for CIDP. We spoke, and compared notes. Our disease profiles seemed remarkably similar. We also shared similar frustrations with the medical world and how little conventional medicine had to offer. Finally someone who truly understood. She was undergoing a procedure called autologous stem cell transplantation, (SCT) a process developed at Chicago’s Northwestern University by Dr. Richard Burt. In simple terms this process “reboots” the immune system much like rebooting a computer after it becomes too slow. The process takes approximately 10 weeks from start to finish, 4 of those weeks in the hospital at Northwestern.
From that moment on, I had a singular focus to get to Northwestern and have this procedure. By this point I was four months into the disease, and had tried steroids, IV-IG and chemotherapy, none of which had any impact or slowed the disease at all. In fact, my progression was unusually fast. I was hitting disability wickets in weeks that most people take years to develop. This decline looked like the rarest and most malignant form of the disease. My loss of physical control was becoming pronounced. I spent most of my days in bed. Getting to the kitchen or bathroom was difficult. I had a walker, but I would often pass out from just standing.
Regardless, my spirits were up. My family members were immensely supportive and encouraging. Despite my worsening condition, I now had hope. I cannot tell you how important that was to me. I felt a sense of total optimism about the prospect of a stem cell transplant. The science behind the transplant was solid and it made logical sense to me. In simple terms, the procedure requires first harvesting a patient’s adult stem cells from his bone marrow and then killing the existing immune system using chemotherapy. The stem cells are then reintroduced into the body which grows an immune system genetically identical to the one that you were born with, but with none of the memory or bad habits it picked up along the way. Dr. Burt developed the protocol initially for MS patients, but found the procedure effective against many other autoimmune diseases such as lupus, Crohn’s, systemic sclerosis and thankfully, CIDP.
I was admitted into the clinical trial a month later. I was in a wheelchair at this point, but in high spirits. I was receiving IV-IG on a weekly basis to retain enough strength to make it through the procedure. From the moment I arrived at Northwestern I knew I had made the right choice.
I won’t go through all the details of the stem cell transplant. But I went through the procedure and was taking my first steps within a couple of weeks. Within six months I had 90% of my function back. Within a year I had all my function back and today, three and half years out from the transplant I am as strong as ever. There have been challenges along the way. I did have significant pain during the first year following the procedure but most of that has thankfully resolved.
Although I have 100 percent of my strength back, I cannot run and cannot walk more than a few miles without taking a break. But I live a full and complete life. And from where I came from this is a miracle. There were some psychological challenges. When you get hit with a serious illness, it’s as though your body betrays you. We are all imbued with a confidence in our physicality. It enables us to function and take risks in life. That confidence took a major hit, but over time has largely returned. While I will never forget what happened, I consider myself cured, and that feeling is like being innocent all over again. The best part about not forgetting is the enormous gratitude you get as a survivor. Every morning I put my feet on the ground and stand, I am filled with gratitude, for just being able to do that. I owe my life to Dr. Burt, my wonderful friend Alice who guided me through it, my family and close friends whose love support literally saved my life, and of course God for gracing me with a second chance. What a lucky man I am.
Francesco spoke at the 2013 Vatican Conference
In 2001, Francesco Ferrari, a 47-year-old Italian native was diagnosed with primary progressive multiple sclerosis (PPMS) and was told that there was no treatment for him. From 2001 to 2008, his condition progressively worsened. He was first seen in May 2008 at the International Multiple Sclerosis Management Practice in New York City. He complained of left lower limb weakness and dragging, an inability to walk lengthy distances, and bladder dysfunction. He was started on adult stem cell therapy in 2010, and by August 2011 he had normal bladder control. He also noted improvement in his left leg weakness and he could walk without a discernible limp. On examination, he had improved strength in his left leg and could jump easily as well as hop on either leg. However, he stopped therapy at the end 2011. He was re-examined in November 2012 and felt he had regressed and wanted to re-start therapy. When Francesco joined us in Rome, he had hopes that with the stem cell therapy, he would no longer face a future in a wheelchair and that he would regain almost normal function.
Elizabeth was honored at the 2013 Vatican Conference and featured in our March 2013 Life Lines newsletter
Born with osteogenesis imperfecta (OI), Elizabeth Lobato was given a dire prognosis. Children with OI, also known as “Brittle Bones” disease, usually grow no taller than three and a half feet and have severe bone deformities. Most spend their lives in wheelchairs and even the slightest jolt or bump can cause their bones to fracture, causing incredible pain.
Elizabeth was born with many broken bones and her family fought for her survival. They learned about an innovative stem cell transplant procedure that would use her father’s adult stem cells to encourage her growth. While Elizabeth was undergoing this stem cell treatment, she grew six inches and was able to leave her wheelchair to walk, and even dance. When Elizabeth joined us in Rome, she was continuing her stem cell treatments with the hope to be able to walk on a regular basis. Her doctors were hopeful that with more stem cell treatments that Elizabeth could continue to lead a normal life.
Read My Daughter’s Journey
by Mary Lobato, Elizabeth’s mother.
We felt that Elizabeth was a gift and we weren’t going to give up that gift so easily. My husband said, “As long as she fights we’re fighting with her.” Doctors consistently communicated that there were no treatments, no cure and Elizabeth would live the rest of her life in pain.
When Elizabeth came home, Terry reached out to a doctor that he researched who was featured in an article about a series of bone marrow transplants. That doctor put us in touch with Dr. Edwin Horwitz who instantly felt that he could help Elizabeth at only three weeks old.
When Elizabeth was 10-years-old, Dr. Horwitz had Elizabeth approved to enter a stem cell study. Terry was the one fortunate enough to donate his bone marrow. The doctors took his cells, infused them into Elizabeth and they repeated that over 2.5 years. It was working. Instead of her body deteriorating, her bones were growing. Elizabeth grew 6 inches, if not more, since the treatment began. She’s gotten taller which is the opposite of what happens to a lot of kids with osteogenesis imperfecta. It was unbelievable that her bones grew, which is something he wasn’t even anticipating, so we were very grateful for this.
She received Dr. Horwitz’s treatment in the summer of 2010. It’s just been great, but our new challenge is funding her treatment. We have applied for a compassionate use exception to continue Elizabeth in the study. Just for a piece of the treatment, we have raised about $25,000 on our own and through the support of our community.
The hardest part as a parent is to know that treatment exists and that only funding is lacking. Dr. Horwitz just has to verify what kind of protein that the stem cells secrete, concentrate them and infuse them into the patients. We think the global impact that this could have on any kind of patient with skeletal or bone disorder is huge. We’ve come so far and she’s 14-years-old. To live that long and fall short is going to feel like a failure if there is no way for her to continue treatment.
Dr. Horwitz’s current clinical trial is the most advanced in his field, and the only cell therapy treatment for OI currently being tested in humans. Every child being treated under this experimental protocol has shown new bone growth—a stunning finding. Dr. Horwitz is now focused on sustaining that bone growth over time and isolating the proteins that stimulate this growth.
In 2000, Charles Melber experienced a heart attack that resulted in repeated hospital stays. By 2004, he had more than 30 stents placed in his heart. When he was accepted into an adult stem cell clinical trial in 2006, run by Dr. Richard Schatz, his health had deteriorated further. He was using nitroglycerin in excessive doses, and his physical ability to perform daily tasks was severely compromised.In September 2006, he had adult stem cells injected into his heart muscle at the Scripps Green Hospital in San Diego, California. He underwent stem cell mobilization, a process that includes injections of a stem cell mobilizing agent over the course of several days, followed by apheresis, where the mobilized stem cells are collected by filtering them from his whole blood into a centrifuge.After the collection process, he spent the night in the hospital and, early the next day, Dr. Schatz injected the collected cells into his heart muscle through a catheter in a process very similar to an angiogram. He spent the balance of that day and one night in the hospital before being released. He started noticing improvement within three weeks.Dr. Schatz continued to monitor Charles’ progress, running tests at various intervals. Charles continued to improve with longer treadmill times and positive responses to other tests. Within a few months, he showed remarkable improvement.
In 2007, Don Robinson of Georgia, then 58 years old, suffered a severe heart attack. He felt stabbing pains between his shoulder blades and a scan verified substantial heart muscle damage. His doctors explained to him that such weakness of the heart can lead to congestive heart failure and possibly death. With that grim knowledge, Don enlisted in a Phase I clinical trial of a product candidate called AMR-001. The trial was sponsored by NeoStem and uses a patient’s own adult stem cells to try to prevent further heart damage.Clinicians extracted bone marrow from Don’s hip, isolated the cells intended to enhance blood vessel formation, and administered the cells back into his damaged artery via a catheter, thereby preserving at-risk muscle tissue and protecting it from further damage. The overall outcome was that there appeared to be improved blood supply to the area affected by the heart attack. Six months later, a scan showed additional active muscle. Six years after the treatment, Don described that he has more energy and that feels back to normal.Watch a video about Don’s story
Read Healing a Heart: Don Robinson’s Story
In the fall of 1995, Stephen Sprague was diagnosed with chronic myelogenous leukemia. Unfortunatley, after 18 months of chemotherapy and in desperate need of a bone marrow transplant, he found himself in “blast crisis”—the end stage of his disease. An only child with no siblings as possible donors and unable to find an unrelated marrow match in any of the donor registries, he was realistically told to “get his affairs in order.”In October 1997, Stephen was presented with an unexpected, but high-risk, opportunity to become one of the first adult patients to participate in a pioneering clinical trial being conducted at Hackensack University Medical Center, New Jersey, using umbilical cord blood as an alternative to bone marrow as a stem cell source.As of 2011, following his successful transplant more than 13 years before, Stephen as completely cured of his leukemia and in relatively good health. He was proud to have lived to see both of his children marry and prosper and was a most-proud grandfather to five grandchildren.Stephen demonstrates the importance of the clinical trial process and that cord blood stem cells can be used effectively on full-sized adult patients if the human leukocyte antigen match is optimal. His case has been published by Dr. Andrew L. Pecora and his colleagues in the April 2000 issue of Bone Marrow Transplantation. Stephen is often requested to appear and testify before the FDA, various U.S. House and Senate Committees and the White House, each investigating promising, non-embryonic adult stem cell applications. In October 2007, he was appointed by the U.S. Secretary of Health and Human Services to serve on the Health Resources and Services Administration’s Advisory Council on Blood Stem Cell Transplantation.In 2011, Stephen served as director of patient advocacy for CORD:– USE, a family cord blood bank in Orlando, Florida.