Adult Stem Cells Treat Scleroderma

MyCentralJersey.com, July 19, 2009

Mike George, a retired junior high school principal, had been suffering from an advanced case of systemic scleroderma. Translated literally as "hard skin", scleroderma is a chronic autoimmune disease characterized by fibrosis (tightening and hardening of tissue) and for which conventional medicine has no known cure. Of the two main versions of the disease, the systemic version (also known as diffuse cutaneous scleroderma) involves internal organs as well as the skin. In the version of the disease known as limited cutaneous scleroderma, symptoms are limited primarily to the skin, although secondary complications may manifest in the pulmonary system. Vascular complications are not uncommon in the systemic version, and when one or more internal organs are affected, the disease can be fatal.

Fourteen months ago, however, Mr. George underwent autologous adult stem cell therapy as part of a clinical trial at Northwestern Memorial Hospital in Chicago. Now, according to Mr. George, "I feel really good. I feel I was reborn. It's great to be alive."

Prior to the therapy last year, Mr. George's skin was stiff, his face was tight, he could only swallow with difficulty, any type of physical movement was an effort, and his doctors were concerned that the disease had begun spreading to his heart and lungs. Upon his return back home after the therapy, however, Mr. George was able to lift his luggage out of the trunk of his taxi - an accomplishment which had not been possible prior to the stem cell therapy. The next month, as Mr. George describes, his physician didn't recognize him. "In April, the doctor said, are you sure I didn't give you a lung transplant, instead of a stem cell transplant?" Although the stem cell therapy has not totally cured Mr. George, it has stopped the progression of the disease and to some extent reversed it, with noticeable improvement not only in his skin but also in his heart and lungs as well.

On May 17, 2005, it was announced that Northwestern Memorial Hospital and the Northwestern University Feinberg School of Medicine in Chicago together launched the Northwestern Scleroderma Program, which offers patients with scleroderma a unique program of comprehensive care. According to Dr. John Varga, a rheumatologist and director of the Northwestern Scleroderma Program, "Patients who are diagnosed with scleroderma are often told that there is little that can be done for them. At Northwestern, our integrated team of experts specializes in the treatment of scleroderma and all of its related conditions. We can offer patients treatment options they can't find elsewhere, like bone marrow transplants, while also giving them access to other important disease management services such as physical and rehabilitative therapy and nutritional counseling and support." As described on their website, "The Northwestern Scleroderma Program offers the latest advances in diagnostics and treatment for scleroderma, including bronchoscopy and lavage, high-resolution CT scanning, right heart hemodynamics (blood circulation), advanced esophageal studies, innovative treatments for pulmonary hypertension and scleroderma lung disease, as well as autologous stem cell therapies."

In fact, on this website we have previously reported a number of times in the past on various clinical studies conducted at Northwestern Memorial Hospital in which patients have shown dramatic success after having received autologous adult stem cell therapy, usually for the treatment of other types of autoimmune diseases such as multiple sclerosis. In particular, Richard Burt, M.D., Chief of the Division of Immunotherapy for Autoimmune Diseases at Northwestern Memorial Hospital, is gaining increasing attention for his pioneering use of hematopoietic stem cells in the treatment of various autoimmune diseases which include not only multiple sclerosis but also rhematoid arthritis, lupus, and Chron's disease, among others. In 2006 Dr. Burt was named within The Scientific American 50, which is the magazine's annual list of outstanding leaders in science and technology. According to John Rennie, editor-in-chief of the magazine, "The Scientific American 50 pays tribute to individuals and organizations who, through their efforts in research, business and policy-making, are driving advances in science and technology that lay the groundwork for a better future. Not only does our list honor these prime movers, it shines a spotlight on the critical fields that are benefiting from their achievements." Continuing his distinguished and pioneering use of autologous adult stem cell therapy, Dr. Burt is currently involved in ongoing randomized clinical trials for a number of autoimmune diseases which include systemic scleroderma.

Usually, in clinical trials, patients are neither charged nor remunerated for their participation in the trial. However, even though the scleroderma clinical trials at Northwestern are FDA-approved and tightly controlled, nevertheless Mr. George had to pay for the medical services that he received as a participant in the study, which came to more than $200,000. Family members, friends, and the communities at his church and school district helped contribute to the cost of his medical expenses.

In the study, the autologous adult stem cells were harvested from Mr. George's own bone marrow and then readministered to him therapeutically after having been isolated, purified and expanded in the laboratory. First, however, he also received a heavy dose of chemotherapy, the purpose of which was to "cleanse" his immune system before he received his own adult stem cells, which not only served as a therapy for his scleroderma but also "rescued" his immune system from deliberate destruction by the chemotherapy. The use of chemotherapy prior to autologous adult stem cell therapy is, unfortunately, not uncommon. Fortunately, however, the scientific logic of such a routine practice is becoming increasingly questioned.

In actuality, other doctors have already demonstrated success in treating various autoimmune diseases with adult stem cell therapy, but without the brutal and deliberate destruction of the immune system with chemotherapy. Known as immunological myeloablation, such a procedure had previously been considered a necessary part of any transplant therapy, even though it exposes the patient to potentially life-threatening risks. Today, however, an increasing number of doctors are questioning the logic and necessity of subjecting their patients to deliberate immune destruction, and with valid scientific reason. In a publication that appeared over two years ago, in the Journal of Translational Medicine in January of 2007, Dr. Neil H. Riordan et al. posed the following question: "...in patients who are not suffering from a disease that is associated with an aberrant bone marrow such as hematological malignancies or immunological dysfunctions, how is it justifiable to subject them to the high levels of morbidity and mortality associated with immune suppression?" Dr. Riordan and his team of scientists then examined compelling evidence which strongly indicates that pre-transplant immune suppression is unnecessary for autologous hematopoietic cell therapies and even for some types of allogeneic therapies, such as those that utilize "universal donor" cells such as mesenchymal stem cells and the CD34+ stem cells that are found in umbilical cord blood, and for which immune rejection is not even a concern. As Dr. Riordan and his colleagues wrote in their 2007 paper in a section that is subtitled, "Mesenchymal stem cells do not need myeloablation for efficacy": "Currently there are several ongoing clinical trials in Phase I-III using 'universal donor' mesenchymal stem cells in non-conditioned recipients of Crohn's disease, GVHD (graft-versus-host disease) and myocardial infarction. Although these cells are bone marrow expanded mesenchymal cells, the superior proliferative potential of cord blood mesenchymal cells may allow them not only to escape immune destruction, but also to expand in vivo and mediate therapeutic effects superior to those derived from bone marrow. The fact that regulatory agencies have allowed advancement of 'off-the-shelf' universal donor mesenchymal stem cells supports the numerous reports of clinical efficacy in an allogeneic setting." Therefore, certainly with autologous (in which the donor and recipient are the same person) adult stem cell therapy, there is no risk of immune rejection so there is no need to destroy the immune system with chemotherapy; but even with many types of allogeneic (in which the donor and recipient are not the same person) adult stem cell therapy, such as with "immune privileged" "universal donor" stem cells, there is also no need to destroy the immune system with chemotherapy.

Nevertheless, for clinical trials such as those conducted at Northwestern University, the autologous adult stem cell therapies offer tangible improvement - at least for those patients who survive the life-threatening destruction of their immune systems from the chemotherapy. One can only conclude, therefore, as has already been demonstrated by other doctors at other clinics, that patients would exhibit even greater and faster improvement if they did not have to recover from the deliberate destruction of their immune systems prior to receiving the stem cell therapy. Additionally, other clinical evidence indicates that even greater patient improvement would be seen if the stem cell therapy would utilize the "superior proliferative potential" of the adult stem cells that are found in umbilical cord blood.

It has been estimated that between 150,000 and 300,000 people in the U.S. alone suffer from scleroderma. Having been one of the fortunate patients who was strong enough to survive the deliberate and unncessary destruction of his immune system prior to receiving his autologous adult stem cell therapy, Mr. George is now a devout believer in autologous adult stem cells. "I'm like an advocate," he says. "All my life, I wanted to help people. Helping kids was my forte. Now to help someone in need who doesn't know what to expect, it raises it to a whole different level."