Stem Cell Therapy for Scleroderma


Scleroderma is a chronic systemic autoimmune disease characterized by fibrosis (or hardening), vascular alterations, and autoantibodies. There are two major forms:

Limited systemic sclerosis/scleroderma cutaneous manifestations mainly affect the hands, arms and face. Previously called CREST syndrome in reference to the following complications: Calcinosis, Raynaud’s phenomenon, Esophageal dysfunction, Sclerodactyly, and Telangiectasias. Additionally, pulmonary arterial hypertension may occur in up to one third of patients and is the most serious complication for this form of scleroderma.

Diffuse systemic sclerosis/scleroderma is rapidly progressing and affects a large area of the skin and one or more internal organs, frequently the kidneys, esophagus, heart and lungs. This form of scleroderma can be quite disabling. There are no treatments for scleroderma itself, but individual organ system complications are treated.[1] [2] Other forms of scleroderma include Systemic sine scleroderma, which lacks skin changes, but has systemic manifestations, and two localized forms which affect the skin, but not the internal organs: morphea, and linear scleroderma.

Prognosis is generally good for limited cutaneous scleroderma patients who escape pulmonary complications. Prognosis is worse for diffuse cutaneous disease, particularly in older age, and for males. Death occurs most often from pulmonary, heart and kidney complications. In diffuse cutaneous disease, 5-year survival is 70%, 10-year survival 55%.[3]

The cause is unknown. Scleroderma runs in families, but the genes have not been identified. It affects the small blood vessels known as arterioles, in all organs. First, the endothelial cells of the arteriole die off, along with smooth muscle cells, by a process of apoptosis. They are replaced by collagen and other fibrous material. Inflammatory cells, particularly CD4+ helper T cells, infiltrate the arteriole, and cause further damage. Many of the inflammatory and destructive protein signals have been identified, and they are potential targets for drugs that could interrupt the process.[1]

Stem Cell Therapy:  There are many theories for the use of stem cell therapies many of which are centered on the concept of modulating the immune system and decreasing inflammation as their primary actions.

The literal explosion of clinical trials and reported results, including substantial remissions for expended periods including those in the severe advanced stages of the disease is very exciting. Treatment with stem cells coupled with limited chemotherapy has been in the forefront of many of the trials. The availability of this treatment and the startling reversals seen in our patients is significant. We will be posting an interview with a patient, treated 6 months ago in her final stage of the disease, now in full remission with no residual symptoms.

Autologous Stem-Cell Transplant: Phases of the Procedure:
After a review of your medical records and discussions with medical staff, a protocol is designed especially for you. Specifics of your condition are addressed along with any special needs.  It may be similar to the one illustrated below:

Day 1:

At the clinic you will be examined by our physicians. Information including any risks and expectations concerning your treatment, plus answers to any questions you may have will be addressed.  A blood draw, to determine cell counts and other chemistries will be collected and cell expansion medication may be administered. Then you will return to your hotel for a restful day or a good nights sleep.

Day 2:

At the clinic our physician/s will review the laboratory results, determine if the cell count is within range, and discuss the response to the stimulation. They may or may not provide additional cell expansion medications and may add adjunctive treatments. The levels of your response will determine if you would return to the hotel, with little restriction on your activities, or possibly go forward with harvesting and processing your cells.

Day 3:

If the cell count and viability is appropriate for harvest either a bone marrow or adipose collection will be utilized.  We typically use local anesthetics for adults and general anesthesia for youngsters. The entire procedure normally takes less than 30 minutes. Although some pain is felt when the needle is inserted, most patients do not find the bone marrow or adipose collection procedure particularly painful.

We recently placed a number of videos on our website interviewing our patient’s who discuss the procedure and their lack of discomfort.

After the collection you may return to the hotel, with some restrictions. The bone marrow or adipose collected is processed in our contract State-Of-Art laboratory by trained staff, under the supervision of the lab physician.

As an alternative to the above, cord blood may be used based on the patient’s individual medical condition and options.

Day 4:

At the clinic or at the hospital you will be treated by IV infusion and/or a lumbar puncture, which injects the stem cells into the cerebrospinal fluid. This route transports the cells into the spinal canal and the brain directly influencing the nervous systems, thereby eliminating the brain/blood barrier. If a lumbar puncture is performed, the patient will be required to restrict their activities and potentially spend the day in the hospital or at their hotel.

Day 5:

At the clinic or hospital the patient will receive a post-treatment examination and evaluation prior to their release. Additional therapy and treatments may also be utilized to maximize the placement and activities of the procedure.

Day 6: Optionally there may be the use of additional ancillary therapies to enhance the procedure.

What makes our treatment different ?

Our approach includes stimulation, prior to collection, processing and expansion of the cell along with the use of growth factors, together with an integrated medical approach. This maximizes the growth and implantation potentials yielding optimized potentials of making changes in your disease.

Our staff physicians are all board certified, in their field with years of experience. Your team includes both primary and ancillary care professionals devoted to maximizing your benefits from the procedures. We enroll you in an open registry to track your changes independently, for up to 20 years.

As our patient we also keep you abreast of the newest developments in stem cell research. This is an ongoing relationship to maintain and enhance your health.

Our promise is to provide you with travel and lodging support, access to bilingual staff members throughout the entire process and most importantly the best medical care possible.

References and Articles:

Nat Clin Pract Rheumatol.2008 Apr;4(4):184-91. Epub 2008 Feb 19. Technology Insight: hematopoietic stem cell transplantation for systemic rheumatic disease.“Transplantation of HSCs for the treatment of autoimmune diseases aims to fundamentally correct the dysregulated immune system, which could result in sustained clinical remission or potential cure.”Curr Opin Hematol. 2008 Nov;15(6):594-600. Haematopoietic stem cell transplantation for autoimmune disorders. Saccardi R, Di Gioia M, Bosi A.Updates of published trials and data from the registries indicate a long-lasting, immunosuppression-free condition in about 50% of the patients who underwent an HSCT for a severe, progressive autoimmune disease. For all diseases, autologous HSCT is largely preferred for safety reasons, whereas allogeneic HSCT is to be considered only for carefully selected cases.J Rheumatol. 2009 Jul;36(7):1460-3. Epub 2009 Jun 16.            Autologous Hematopoietic Stem Cell Transplant in Systemic Sclerosis: Quantitative High Resolution Computed Tomography of the Chest Scoring  David Launay et alAfter a median 60 months’ followup, the overall disease extent score from HCRT scans decreased from 10 (0–45) to 4 (0–36) (p = 0.04) 6 months after AHSCT, and thereafter increased up to 36 months and stabilized; the modified Rodnan skin score fell (p < 0.05).Conclusion. The extent of SSc lung involvement on HRCT rapidly but transiently regressed after AHSCTPhase I/II trial of autologous stem cell transplantation in systemic sclerosis: procedure related mortality and impact on skin diseaseAn improvement in skin score of >25% after transplantation occurred in 20/29 (69%) evaluable patients, and deterioration in 2/29 (7%).1. Gabrielli A, Avvedimento EV, Krieg T (May 2009). “Scleroderma”. N. Engl. J. Med. 360 (19): 1989–2003. 2. Klippel, John H.. Primer On the Rheumatic Diseases 11ED. Atlanta, GA: Arthritis Foundation.3. Harrison’s Principles of Internal Medicine, 16th ed., Ch. 303, Systemic sclerosis (scleroderma) and related disorders, Bruce C. Gilliand

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