A Retrospective Analysis of Safety and Efficacy of Wharton’s Jelly Stem Cell Administration in Children with Spina Bifida

Boruczkowski, Dariusz; Zdolińska-Malinowska, Izabela

doi:10.1007/s12015-019-09902-6

Cited by 8 publications

(11 citation statements)

References 87 publications

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“…These experiments were conducted by other teams using the same medicinal product delivered by the same manufacturer. Previous papers describing results obtained in other neurological indications revealed a dose‐dependent therapeutic effect with the minimal effective dose corresponding to at least three administrations 41,44 . The first administration was always intravenous for safety reasons; the following injections were administered intravenously or intrathecally.…”

Section: Methodsmentioning

confidence: 99%

“…The dose, scheme, and route of administration was selected on the basis of papers reporting positive effects of WJ-MSC administration in another neurological diseases. 41,44 These experiments were conducted by other teams using the same medicinal product delivered by the same manufacturer. Previous T A B L E 1 Characteristics of the cells administered to the patients papers describing results obtained in other neurological indications revealed a dose-dependent therapeutic effect with the minimal effective dose corresponding to at least three administrations.…”

Section: Treatment Administrationmentioning

confidence: 99%

“…Previous T A B L E 1 Characteristics of the cells administered to the patients papers describing results obtained in other neurological indications revealed a dose-dependent therapeutic effect with the minimal effective dose corresponding to at least three administrations. 41,44 The first administration was always intravenous for safety reasons; the following injections were administered intravenously or intrathecally. The rational basis of intrathecal administration is the presence of dystrophin in myelin-forming Schwann cells 45 and additive effect of cotransplantation of myoblasts and Schwann cells.…”

Section: Treatment Administrationmentioning

confidence: 99%

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The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

Świątkowska-Flis

Zdolińska-Malinowska

Sługocka

et al. 2021

Stem Cells Translational Medicine

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Muscular dystrophies are genetically determined progressive diseases with no causerelated treatment and limited supportive treatment. Although stem cells cannot resolve the underlying genetic conditions, their wide-ranging therapeutic properties may ameliorate the consequences of the involved mutations (oxidative stress, inflammation, mitochondrial dysfunction, necrosis). In this study, we administered advanced therapy medicinal product containing umbilical cord-derived mesenchymal stem cells (UC-MSCs)

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Section: Methodsmentioning

confidence: 99%

Section: Treatment Administrationmentioning

confidence: 99%

Section: Treatment Administrationmentioning

confidence: 99%

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The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

Świątkowska-Flis

Zdolińska-Malinowska

Sługocka

et al. 2021

Stem Cells Translational Medicine

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“…Boruczkowski and Zdolińska-Malinowska [27] reported that transplanting allogeneic mesenchymal stem cells obtained from Wharton's jelly of umbilical cords to children with cerebral palsy improved their daily quality of life. They were also effective in patients with spina bifida: improving their motor functions, micturition/ defecation control, cognitive functions, and quality of life [28].…”

Section: Achievements and Progress In Clinical Neurorestorative Therapies 41 Cell Therapymentioning

confidence: 99%

The 2019 yearbook of Neurorestoratology

Huang¹,

Chen

Mao³

et al. 2020

Journal of Neurorestoratology

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Time is infinite movement in constant motion. We are glad to see that Neurorestoratology, a new discipline, has grown into a rich field involving many global researchers in recent years. In this 2019 yearbook of Neurorestoratology, we introduce the most recent advances and achievements in this field, including findings on the pathogenesis of neurological diseases, neurorestorative mechanisms, and clinical therapeutic achievements globally. Many patients have benefited from treatments involving cell therapies, neurostimulation/neuromodulation, brain–computer interface, neurorestorative surgery or pharmacy, and many others. Clinical physicians can refer to this yearbook with the latest knowledge and apply it to clinical practice.

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“…The aim of our study was to evaluate the effect of compassionate use of Wharton’s jelly-derived mesenchymal stem cells (WJ-MSC) in terms of progression and survival in ALS patients. This was done in the form of a medical therapeutic experiment (described elsewhere [ 10 ]) with retrospective case-control analyses.…”

Section: Introductionmentioning

confidence: 99%

Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study

Barczewska

Maksymowicz

Zdolińska-Malinowska

et al. 2020

Stem Cell Rev and Rep

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Objective Amyotrophic lateral sclerosis (ALS) is still incurable. Although different therapies can affect the health and survival of patients. Our aim is to evaluate the effect of umbilical mesenchymal stem cells administrated intrathecally to patients with amyotrophic lateral sclerosis on disability development and survival. Methods This case-control study involved 67 patients treated with Wharton’s jelly mesenchymal stem cells (WJ-MSC). The treated patients were paired with 67 reference patients from the PRO-ACT database which contains patient records from 23 ALS clinical studies (phase 2/3). Patients in the treatment and reference groups were fully matched in terms of race, sex, onset of symptoms (bulbar/spinal), FT9 disease stage at the beginning of therapy and concomitant amyotrophic lateral sclerosis medications. Progression rates prior to treatment varied within a range of ± 0.5 points. All patients received three intrathecal injections of Wharton’s jelly-derived mesenchymal stem cells every two months at a dose of 30 × 10 6 cells. Patients were assessed using the ALSFRS-R scale. Survival times were followed-up until March 2020. Results Median survival time increased two-fold in all groups. In terms of progression, three response types measured in ALSFRS-R were observed: decreased progression rate ( n = 21, 31.3%), no change in progression rate ( n = 33, 49.3%) and increased progression rate ( n = 13, 19.4%). Risk-benefit ratios were favorable in all groups. No serious adverse drug reactions were observed. Interpretation Wharton’s jelly-derived mesenchymal stem cells therapy is safe and effective in some ALS patients, regardless of the clinical features and demographic factors excluding sex. The female sex and a good therapeutic response to the first administration are significant predictors of efficacy following further administrations. Graphical Abstract Medical therapeutic experiment with retrospective case-control analyses Electronic supplementary material The online version of this article (10.1007/s12015-020-10016-7) contains supplementary material, which is available to authorized users.

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A Retrospective Analysis of Safety and Efficacy of Wharton’s Jelly Stem Cell Administration in Children with Spina Bifida

Cited by 8 publications

References 87 publications

The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

The 2019 yearbook of Neurorestoratology

Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study

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