Xiaoan’s history: cured by his own cord blood 19 years later

In December 2021, Xiaoan, who had just turned 18, suddenly developed symptoms such as ulcers and blood spots. After a series of examinations, Xiaoan was diagnosed with aplastic anemia. Aplastic Anemia is an auto-immune condition where over-reactive T-cells in the blood have been triggered to attack the body’s own bone marrow. Xiaoan also had “PNH clones” in the blood (paroxysmal nocturnal hemoglobinuria), which is a hallmark of severe aplastic anemia. This severe attack can lead to complete bone marrow failure and death. While seeking treatment in many domestic hospitals, Xiaoan’s parents learned that: At present, umbilical cord blood hematopoietic stem cell transplantation is one of the effective methods for treating aplastic anemia, and the cure rate of cord blood transplants with the patient’s own autologous cord blood is high. This gave Xiaoan’s family, who had been shrouded in the haze of the disease, a new light of hope, because as early as 2003, when the child was born, Xiaoan’s parents had stored umbilical cord blood hematopoietic stem cells for the child in the Tianjin Cord Blood Bank.

After Xiaoan’s treatment plan for autologous umbilical cord blood transplantation was determined, the hospital and Xiaoan’s parents contacted Tianjin Cord Blood Bank as soon as possible. Upon receiving the news, the staff of the cord blood bank immediately devoted themselves to the preparations for the release of the cord blood. Re-examination by professional technicians showed that the umbilical cord blood hematopoietic stem cells that had been “sleeping” for 19 years were in good condition, and all indicators met the release standards. In December 2022, Xiaoan was transferred to Beijing Lu Daopei Hospital, a well-known specialized hospital for blood diseases in the country, and was officially prepared to receive a stem cell transplant. With the efforts of the staff, the precious cord blood was successfully delivered to the transplantation hospital. December 25, 2022 is of great significance to Xiaoan’s family. After the all-out treatment by Director Xiong Min of Beijing Lu Daopei Hospital, Xiaoan underwent reinfusion of umbilical cord blood hematopoietic stem cells on the same day, and this precious seed of life returned to the owner smoothly. About a month later, Xiaoan was released from intensive care, and all physical indicators recovered well. He was discharged from the hospital in February of 2023 and returned to normal life.

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Stem Cell Transplants May Safely Slow MS Progression, Suggests Study

A new study has suggested that stem cell transplants using a patient’s own stem cells to “reset” their immune system can safely slow the progression of relapsing–remitting multiple sclerosis (MS) and should be considered as the standard-of-care for severe disease. The research is published in The Journal of Neurology Neurosurgery & Psychiatry.

Identifying more treatments to benefit MS patients

MS is an inflammatory disease affecting almost 3 million people worldwide. It is thought to be an autoimmune disorder – in which the body’s immune system attacks its own tissues – and affects the central nervous system, attacking the protective myelin sheath that covers nerve fibers, leading them to deteriorate.

The disease has many symptoms, such as fatigue, difficulty walking and vision problems. But not everyone with MS is affected in the same way. The most common presentation is relapsing–remitting MS (RRMS), characterized by flare-ups of the disease followed by periods of recovery. However, RRMS over time can progress to secondary progressive MS (SPMS), which is more severe.

MS has no cure, but disease-modifying treatments (DMTs) can be used to help curb inflammation and delay relapses. However, a treatment called autologous hematopoietic stem cell transplantation (aHSCT) – commonly used to treat blood cancers – was first used to treat MS in the 1990s. aHSCT essentially “resets” a patient’s immune system, first wiping it out with chemotherapy and then rebuilding it using their own blood stem cells harvested before treatment. This is thought to eradicate the self-reactive immune system and rebuild one with better control over disease-causing cells.

“Another name for aHSCT is high-dose chemotherapy with stem cell support, which perhaps better describes the procedure,” said the study’s senior author Dr. Joachim Burman, an adjunct senior lecturer at Uppsala University, speaking to Technology Networks.

aHSCT was recognized in Sweden as a treatment for MS in 2016 but has yet to be implemented in clinical guidelines in many countries. In the current study, Burman and colleagues analyzed data from a Swedish MS registry, investigating the safety and efficacy of aHSCT when used in routine healthcare settings – which are more representative of the general population – rather than clinical trials.

A “significant advantage” over DMTs

The researchers analyzed data from 174 RRMS patients treated using aHSCT prior to 2020 – the average age was 31 years and 64% were women.

In the first three years after the procedure, 20 patients (11%) received DMTs – however, nearly three-quarters of patients showed no evidence of disease activity after 5 years and almost two-thirds showed no activity after 10 years.

Looking at the level of disability among patients, of the 149 patients who displayed disability prior to treatment, 54% improved, 37% remained stable and 9% worsened.

“Another important finding is that the treatment effect is durable. Ten years after the procedure only 35% had evidence of disease activity and only 11% needed to restart some other treatment. It is likely that some of the patients will never need treatment for MS again,” Burman added.

The safety of the therapy is also a considerable concern, as it is perceived as a high-risk procedure – five patients required treatment in intensive care, but none died as a result of treatment. Infections were common – 61 contracted a bacterial infection within 100 days of the procedure and 23 experienced a viral infection, with 3 patients developing shingles. The most common side effect observed in 68% of patients was febrile neutropenia – a high fever and low white blood cell count.

“Patients treated in routine healthcare come in all flavors and are more complicated than patients in clinical trials, which are generally more homogenous,” Burman explained. “Prior to this study, one concern had been that the number of adverse events would be higher in routine care. Reassuringly, there were very few serious adverse events and no treatment-related mortality, so I think it is fair to say that this procedure can be performed quite safely.”

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Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing

Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi anemia, glucose-6-phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The available management strategies for these disorders are still unsatisfactory and do not eliminate the main causes. As genetic aberrations are the main causes of all forms of hereditary anemia, the optimal approach involves repairing the defective gene, possibly through the transplantation of normal hematopoietic stem cells (HSCs) from a normal matching donor or through gene therapy approaches (either in vivo or ex vivo) to correct the patient’s HSCs. To clearly illustrate the importance of cell and gene therapy in hereditary anemia, this paper provides a review of the genetic aberration, epidemiology, clinical features, current management, and cell and gene therapy endeavors related to SCD, thalassemia, Fanconi anemia, and G6PDD. Moreover, we expound the future research direction of HSC derivation from induced pluripotent stem cells (iPSCs), strategies to edit HSCs, gene therapy risk mitigation, and their clinical perspectives. In conclusion, gene-corrected hematopoietic stem cell transplantation has promising outcomes for SCD, Fanconi anemia, and thalassemia, and it may overcome the limitation of the source of allogenic bone marrow transplantation.

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Salvador’s story: using stem cells to treat autism

Salvador, a five-year-old Portuguese boy who was diagnosed with autism spectrum disorder, recently underwent treatment using stem cells from his own umbilical cord blood with the aim of improving his condition. The procedure was carried out in August 2022, at Duke University Hospital, in the United States of America (USA), within the scope of the Expanded Access Protocol (EAP) led by Prof. Joanne Kurtzberg, internationally renowned pioneer in the use of umbilical cord blood. It is estimated that, in Portugal, 1 in 1000 children of school age lives with autism spectrum disorder1. (Editor’s Note: At the end of 2022 the Expanded Access program stopped enrolling children with autism.)

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Stem cell therapy for neurological disorders

Neurological disease encompasses a diverse group of disorders of the central and peripheral nervous systems, which collectively are the leading cause of disease burden globally. The scope of treatment options for neurological disease is limited, and drug approval rates for improved treatments remain poor when compared with other therapeutic areas.

Stem cell therapy provides hope for many patients, but should be tempered with the realisation that the scientific and medical communities are still to fully unravel the complexities of stem cell biology, and to provide satisfactory data that support the rational, evidence-based application of these cells from a therapeutic perspective. We provide an overview of the application of stem cells in neurological disease, starting with basic principles, and extending these to describe the clinical trial landscape and progress made over the last decade. Many forms of stem cell therapy exist, including the use of neural, haematopoietic and mesenchymal stem cells.

Cell therapies derived from differentiated embryonic stem cells and induced pluripotent stem cells are also starting to feature prominently. Over 200 clinical studies applying various stem cell approaches to treat neurological disease have been registered to date (Clinicaltrials.gov), the majority of which are for multiple sclerosis, stroke and spinal cord injuries. In total, we identified 17 neurological indications in clinical stage development. Few studies have progressed into large, pivotal investigations with randomised clinical trial designs. Results from such studies will be essential for approval and application as mainstream treatments in the future.

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Fetal Mesenchymal Stem Cells in Cancer Therapy

There is compelling evidence that mesenchymal stem cells (MSCs) can be utilized as delivery vehicles for cancer therapeutics. During the last decade, bone marrow MSCs have been used as delivery vehicles for the local production of therapeutic proteins in multiple tumor types, taking advantage of their innate tropism to the tumor site and their low immunogenicity.

More recently, MSCs have been isolated from fetal tissues during gestation or after birth. Fetal MSCs derived from amniotic fluid, amniotic membrane, umbilical cord matrix (Wharton’s jelly) and umbilical cord blood are more advantageous than adult MSCs, as they can be isolated noninvasively in large numbers without the ethical reservations associated with embryo research.

Several studies have documented that fetal MSCs harbor a therapeutic potential in cancer treatment, as they can home to the tumor site and reduce tumor burden. This natural tumor tropism together with their low immunogenicity renders fetal MSCs as powerful therapeutic tools in gene therapy-based cancer therapeutic schemes. This review summarizes various approaches where the tumor-homing capacity of fetal MSCs has been employed for the localized delivery of anti-tumor therapeutic agents.

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Clinical Application of Adult Stem Cells for Therapy for Cardiac Disease

Cardiovascular disease is a major cause of death worldwide. Different medical and surgical therapeutic options are well established, but a significant number of patients are not amenable to standard therapeutic options. Cell-based therapies after clinical application have shown different results in recent years. Here, we are giving a comprehensive overview on major available clinical data regarding cell therapy.

Cell-based therapies and tissue engineering provide new promising platforms to develop upcoming therapeutic options. Initial clinical trials were able to generate promising results. A variety of different stem cell types have been used for the clinical application. Different adult cardiac stem cells and progenitor cells, including mesenchymal, CD34+ and CD133+ autologous human bone marrow–derived stem cells (BMCs), human myoblasts, and peripheral blood–derived stem and progenitor cells (PBSCs) have been used for the therapy for end-stage heart failure. Future experiments will show the importance of novel cell populations and clarify the mechanism causing cell therapy–mediated observed effects.

Several clinical trials have reported on sole therapy, as well as combined application of autologous adult stem cells with conventional revascularization. The reported promising findings encourage further research in the field of the translational research.

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The Personalized Stem Cells That Could One Day Treat Parkinson’s and Heart Failure

Could an injection of lab-cultured brain cells, created from a person’s own cells, reverse symptoms of Parkinson’s disease? That’s an idea that Aspen Neuroscience Inc., a startup based in San Diego, plans to test in human trials later this year.

In patients with Parkinson’s, neurons die and lose the ability to make the chemical dopamine, leading to erratic, uncontrollable movements. Aspen Neuroscience will test if the newly injected cells can mature into dopamine producers, stopping the debilitating symptoms of this incurable disease, says Damien McDevitt, the company’s chief executive officer. Tests in animals have shown promise, the company says.

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Umbilical blood stem cell transplant puts woman in HIV remission

A transplant of stem cells from the umbilical cord has resulted in a mixed-race woman going into remission for HIV for the first time.

The woman, known as the New York patient, has been clear of detectable HIV since 2017, after she received HIV-resistant stem cells that had been harvested from umbilical cord blood to treat her leukaemia. Stem cells are produced by bone marrow and can turn into different types of blood cells.

Several people have previously gone into remission from HIV after receiving stem cells from adult donors who carry two copies of a naturally occurring mutation of the CCR5 gene. This delta 32 mutation prevents the virus from entering and infecting healthy cells.

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