Clinical studies

The future of research

Regenerative medicine
Venous cord blood is populated by six cell lines with equal levels of stem cellularity (see “Stem cells: what they are”) .  In addition to haematopoietic  cells, there are another five populations of stem cells which, if differentiated in vitro, create a series of cell types that can be used to treat the following diseases:

This sphere of application, called “regenerative medicine”. is still being tested.  It is the key for other possible therapeutic applications of stem cells (especially of  the  “mesenchymal” and “endothelial” type) as shown in orthopaedic studies, as well as acute myocardial infarction, Type I insulin dependent diabetes, cornea transplant and infantile cerebral paralysis.

(Hollands, 2009 – Haller. Exp Hematol, 2008 – Zhao and Mazzone, Autoimmun Rev. 2010 – Harris DT: Br J Haematol. 2009 – Arien-Zakay H, Best Prct Re Clin Haematol. 2010)

Clinical trials using autologous mesenchymal stem cells:

• Crohn’s disease (Northwestern University 2006)
• Type I diabetes(American Diabetes Association, 2007)
• Infantile cerebral paralysis, hypoxic encephalopathy (Duke University 2009)
• Multiple sclerosis (Saccardi et al 2010)
• Traumatic brain lesions
• Gene therapy for Ewing’s sarcoma
• Brain tumours – sarcomas

Clinical trials using allogeneic mesenchymal stem cells:

• Crohn’s disease – Type I diabetes
• Spinal cord injuries
• Breast cancer
• Ewing’s sarcoma
• Renal cell carcinoma

(www.clinicaltrial.gov; Parent’s guide to cord blood)

Expansion in vitro (or ex-vivo)

The current expansion protocols, in the process of being defined and validated – albeit limiting because potentially the cause of greater contamination, of selection of expanded cells, of limited post-transplant vitality and of longer times prior to the transplant (2-3 weeks) – permit a greater stem cell progenitor development and shorter transplant attachment times. This protocol is operative in the appropriate facilities for minor or major manipulation (according to AIFA and EMEA rules) and involve an additional charge.

Cellular therapy

Cellular therapy uses stem cells as pharmacological vectors and as vectors for developing gene therapy (replacement of a mutated gene with a healthy gene) often with the aim of an autologous transplant.

Tissue engineering

Involves the regeneration of human tissue by seeding stem cells on scaffolds of suitable materials and characteristics, their cultivation in special reactors (bioreactors) until the scaffold is colonized and a new tissue produced (extracellular matrix). Tissue engineering is still not able to substitute prostheses for repairing damaged tissue or for restoring function to compromised organs, but some applications are starting to be found, for example in manufacturing skin or corneas, and in bone, cartilage and ligament regeneration^[1].

Pharmacological trials

An important and innovative objective is the use of stem cells for testing drugs in vitro and not directly on the patient. This is of fundamental importance in choosing personalized chemotherapy for tumours.

Diagnosis

The diagnosis of a pathology requires a family medical history. Neonatal stem cells represent the “biological witness” of the newborn child and are an important diagnostic source for the retroactive assessment of an infection or tumour occurring during the individual’s lifespan.

Induced Pluripotent Stem Cells (iPS)

Since 2007, following a revolutionary publication by Yamanaka in Science, some 200 laboratories worldwide have focused their research on pluripotent stem cells obtained from differentiated cells, inside which a viral vector was introduced containing four genes (OCT4, SOX2, KLF4 and c-Myc) able to create pluripotent cells with indefinite replication capacity, the iPS.  These experiments have reached such a sophisticated level that it is no longer necessary to introduce a viral vector but instead “small molecules” capable of transforming a cell and bringing it back to a condition of primordial potential without any ethical issues in using embryos. Recent experiments have demonstrated that cells derived from the cord blood are excellent candidates for producing iPS thanks to their immaturity, their clonal identity and the total absence of mutations (damages) accumulated by other types of cells after birth^[10,13,18].

Clinical trials on the autologous use of umbilical cord blood: