All you need to know before your scleroderma stem cell transplant
Prior to undertaking a scleroderma stem cell transplant it is important to get up-to-date with what is involved in the procedure and why recent research has shown the tremendous benefits that this form of treatment has to offer.
Scleroderma comes from the Greek words skleros (hard) and derma (skin) and means a disease that results in progressive skin hardening and damage to other structures. Scleroderma is one component of systemic sclerosis, a body-wide connective tissue disease that also involves skin, subcutaneous tissue, muscles, and organs.
It is characterized by the excessive deposit of collagen and other large molecules into the skin and multiple internal organs, with scarring and damage to small blood vessels. The immune system is damaged, and scleroderma is considered an autoimmune disease whereby the body attacks itself.
Systemic sclerosis is a complex and multifaceted disease with clinical forms ranging from skin involvement only, usually areas like the forearm, hands, and fingers, to forms with diffuse skin sclerosis and severe, progressive internal organ involvement. Some skin areas affected might be the face and neck.
One form of skin involvement is called the CREST syndrome. This relates to:
although not all of those signs are needed for the disorder to be called CREST.
Diffuse cutaneous systemic sclerosis refers to skin thickening of the trunk, extremities, and involvement of the face. Other organs that can be affected are the stomach, kidneys, heart, muscles, hormone-producing organs, and bladder.
The exact cause of systemic sclerosis is not known but it does not appear to be inherited, although a genetic predisposition probably plays a role in its development. Environmental factors may trigger or accelerate the development of systemic sclerosis if the person has the genetic propensity [1,2,3,4,5]. These include the following:
Skin signs and symptoms of scleroderma are as follows:
Progressive skin tightness and hardness usually preceded by swelling and puffiness that do not respond to drugs to induce water excretion from the tissues—diuretics.
Raynaud phenomenon occurs initially in 70% of patients with systemic sclerosis; 95% eventually develop it as the disease progresses but may precede the systemic disease by many years. The female-to-male ratio is 4:1, and it starts in adolescence.
Other vascular manifestations of systemic sclerosis include:
There is a lot of information relating to GI manifestations as they relate to scleroderma. GI findings are:
Musculoskeletal complaints may include the following:
Cardiac signs and symptoms in severe systemic sclerosis include the following:
Heart failure due to scarred heart muscle
Renal signs and symptoms include the following:
Patients may have the following:
Patients may also present with the following signs and symptoms:
There are a number of ways that scleroderma may be diagnosed. It is most often diagnosed following laboratory testing. Laboratory testing may include the following:
X-rays (abdomen, chest, hands, jaw)
Endoscopy of the esophagus and GI tract
Lung function studies
Holter monitoring for 24-hours to detect arrhythmias
To date, treatment of SSc patients is for symptom relief since there is no cure. Drugs aimed at alleviating Raynaud’s phenomenon, gastro-oesophageal reflux, pain, and immunosuppressants (methotrexate, mycophenolate mofetil, and cyclophosphamide), or organ transplantation in case of severe heart-lung involvement . There have been new drugs for the treatment of PAH , but the scleroderma person’s prognosis and mortality have not changed in the last 40 years .
Mesenchymal stem cells (MSCs) are a promising therapeutic approach primarily due to their anti-fibrotic (anti-scarring), angiogenic (vessel growth), and immune-stimulating or modulating capacities. They act on the different processes that are dysregulated in the disease. Recently, the therapeutic effectiveness of MSCs has been demonstrated in different preclinical animal trials and currently in phase I human trials. Both foreign (allogenic) and autologous (self) derived stem cells from bone marrow, or fat tissue are being evaluated. The reason for using allogenic MSCs in Scleroderma is because it is an autoimmune disease, and’ self’ MSCs may be already damaged and not work as well.
MSCs are ‘generic’ adult cells that have the capacity to differentiate into many different but specific cell types and have been identified in the bone marrow (BM), adipose, umbilical cord blood, placenta, and dental pulp [9,10].
How does a stem cell transplant work in regards to scleroderma?
In Scleroderma stem-cell transplantation, the main functions of MSCs are thought to be anti-inflammatory to counteract the messed up immune system, anti-fibrotic by reducing the excessive collagen deposition into the skin and internal organs that cause hardening, and pro-vessel forming to fight the vessel- damaging effects of scleroderma [11,12,13]
One study comparing adipose-derived stem cells (ADSC) from a donor to those collected from the person with scleroderma showed that the function and results from both types are identical. The only difference was the reduced proliferation and migration capacity of ADSC from scleroderma patients .
In a study conducted by Scuderi et al., six scleroderma patients were treated with ASC in affected skin areas (face or limbs) combined with the injection of acid hyaluronic, with a good reduction of skin thickness and no local complications .
In 2011, a German team published four more cases of refractory Systemic Scleroderma (SSc) treated with allogeneic bone marrow (BM)-MSC . At 18-month follow-up, four of the five patients had an improvement in overall skin score for disease, digital ulcers or distal limb necrosis with no major complications.
Can Stem Cells Cure Scleroderma?
As the recently conducted trials clearly show, stem cell transplant treatment for scleroderma continues to be a great reason for optimism. As the effectiveness of this treatment method continues to gain traction from various clinical studies, finally scleroderma sufferers can look forward to a brighter future.
How much does a stem cell transplant cost?
Prices for treatment with stem cells varies significantly based on the individual patient’s needs, the location of the treatment center, and other factors. The treatment center that you intend to visit should be contacted for all matters regarding pricing.
How long does a stem cell transplant take?
The length of treatment will vary considerable for a stem cell transplant and this will be based on a number of individual factors relating to the severity of the scleroderma experienced by the patient attending a clinic.