Title of the medical therapeutic experiment
“The use of mesenchymal cells obtained from Wharton jelly from an incompatible donor unrelated to the treatment of patients with neurological and ophthalmic diseases in which there is no causative treatment and in which the treatment did not bring any improvement in the clinical condition.”
Wharton’s jelly-derived mesenchymal stem cells – WJ-MSC are a population of multipotent stem cells, located in the umbilical cord, classified as gelatinous connective tissue. According to the criteria defined by ISCT these cells have a fibroblast structure, show adherence to a plastic substrate and a specific set of surface antigens (WJ-MSC immunophenotype: CD11b- / CD19- / CD45- / CD34- / HLA-DR- / CD73 + / CD90 + / CD105 +), and also have the potential to differentiate into other cell types (mainly osteoblasts, chondrocytes and adipocytes).
A number of effector functions of these cells have been demonstrated. Immunosuppressive effects due to cytokine stimulation from the proinflammatory environment, such as interferon-γ (IFN-γ), tumor necrosis factor α (TNFα), interleukin 1β (IL-1β) or by interaction with the toll-like receptor (TLR) have been observed. Upon activation, the spectrum of WJ-MSC immunosuppressive activities includes the secretion, inter alia, of human leukocyte antigen (tissue compatibility) of HLA-G, transforming growth factor β (TGFβ), prostaglandin E2 (PGE2), TSG-6 (tumor necrosis factor alpha-inducible) protein 6), haemoxygenase 1 (HO-1), interleukin 10 (IL-10), interleukin 6 (IL-6), IDO1 (Indoleamine 2,3-dioxygenase 1), hepatocyte growth factor (HGF), factor inhibiting leukemia (LIF) and for the Fas (FasL) signal pathway. WJ-MSC therefore have specific properties not only of immunomodulatory and immunosuppressive character, but also anti-apoptotic or anti-inflammatory. The immunosuppressive activity of WJ-MSC requires direct contact with target cells such as B, T cells, dendritic cells and NK cells.
A medical therapeutic experiment is a multi-stage procedure aimed at applying an innovative method of treatment which is not a standard therapy for a given disease. A medical therapeutic experiment is often the only alternative for patients who have not yet been treated for cause-and-effect treatment, or a given treatment didn’t bring the desired therapeutic effects.
The main and overarching goal of the medical therapeutic experiment is to improve the patient’s health. These activities must always be supported by the consent appropriate of the Bioethical Commission operating by the Medical Chamber. The documentation submitted to the Bioethical Commission must include the justification and the purpose of initiating the therapeutic procedure with a detailed description of the disease, the exact scheme of the experiment, including the dosage, the amount of infusion and the place of administration, the frequency of controls, the number of patients participating in the treatment along with the criteria for experiment and informed consent of the patient or patients to carry out this type of therapeutic activity.
Klara Medical Center – Polish Center for Cell Therapy and Immotherapy with its headquarters in Częstochowa, Poland is a leading research and treatment center in Poland, conducting therapeutic treatments using mesenchymal stem cells from Wharton jelly (WJ-MSC) in the field of neurology and ophthalmology since 2014. So far, medical therapeutic experiments have been successfully carried out in the following neurological diseases: cerebellar-cerebellar ataxia, autism, severe psychomotor developmental delay, muscular dystrophies (various types), quadriplegic paralysis, psychomotor delay, epilepsy, meningo-muscular hernia, spinal muscular dystrophy type 1, multiple sclerosis, amyotrophic lateral sclerosis, significant developmental disorder, myelomeningocele, motor neuron disease, Devica syndrome and spinal cord secrecy. Klara Medical Center – Polish Center for Cell Therapy and Immotherapy also has experience in the treatment of the following ophthalmic diseases using WJ-MSC: optic nerve neuropathy, bilateral optic nerve atrophy, optic nerve hypoplasia, demyelinating optic nerve damage, retinal pigmentosa degeneration, Stargardt’s disease, cone-prosthetic dystrophy, glaucoma and Behcet’s disease.
The risk associated with the initiation of therapy is negligible, because in the current scientific literature there were no significant side effects associated with administration of WJ-MSC to patients. The whole procedure of the medical therapeutic experiment is designed to minimize the possibility of side effects and to obtain the optimal therapeutic effect.
Mesenchymal stem cells are obtained for the medical therapeutic experiment (MSC-WJ) in the Laboratory of Cellular Engineering [permission of the Chief Pharmaceutical Inspector No. GIF-IW-400/WTC0436/01/41/UA2/14 dated May 7th 2014 ] NZOZ laboratory “Polish Stem Cell Bank” (PBKM). Allogeneic mesenchymal stem cells originate from healthy donors after prior separate medical history (regarding possible medical burdens of the donor’s parents and grandparents) and obtaining the written consent of biological parents to collect umbilical cord tissue and exclude transmission of infectious diseases on the basis of venous blood tests.
The acquisition procedure has been used many times before for cell preparation for scientific and research purposes. In a medical therapeutic experiment, human embryonic stem cells are not used.
Preparation of biological material
In the Laboratory of Cellular Engineering, tissue fragments are isolated from the umbilical cord, which are then subjected to in vitro culture, in conditions consistent with the above-mentioned Chief Pharmaceutical Inspector decision – allowing to obtain mesenchymal cells from them.
Before administering the cells, tests are carried out confirming that the obtained cells have the desired parameters – testing the surface antigen characteristic of mesenchymal cells, testing the number and viability of cells, microbial cultures and other control tests.
Accurate diagnosis and appropriate, effective therapy in combination with medical and diagnostic equipment meeting the highest world standards, allows us to guarantee comprehensive care for our patients. In our daily work, we value safety, professionalism, commitment, kindness, responsibility for the health of others. The values that we follow and the goals we set for ourselves make the quality of our services meet the expectations of patients who are looking for professional medical care. We are aware of the progress that medicine achieves every day, therefore, in the interests of health, comfort and safety of our patients, we constantly strive for perfection.