4/12/2023
Stem cell-based treatment controls blood sugar in people with Type 1 diabetes
November 27, 2023
University of British Columbia
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An innovative stem cell-based treatment for Type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results. The therapy aims to replace the insulin-producing beta cells that people with Type 1 diabetes lack. Dubbed VC-02, the small medical implant contains millions of lab-grown pancreatic islet cells, including beta cells, that originate from a line of pluripotent stem cells.
An innovative stem cell-based treatment for Type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results from the University of British Columbia (UBC) and Vancouver Coastal Health (VCH).
"This is a significant step toward a functional cure for Type 1 diabetes," said Dr. David Thompson, principal investigator at the Vancouver trial site, clinical professor of endocrinology at UBC and director of the Vancouver General Hospital Diabetes Centre. "For the first time, a stem cell-based device can reduce the amount of insulin required for some trial participants with Type 1 diabetes. With further refinement of this approach, it's only a matter of time until we have a therapy that can eliminate the need for daily insulin injections entirely."
The findings, published today in Nature Biotechnology, arise from a multicenter clinical trial for an experimental cell therapy developed by U.S. biotechnology company ViaCyte (acquired by Vertex Pharmaceuticals) that is being clinically tested in Canada.
The therapy aims to replace the insulin-producing beta cells that people with Type 1 diabetes lack. Dubbed VC-02, the small medical implant contains millions of lab-grown pancreatic islet cells, including beta cells, that originate from a line of pluripotent stem cells.
The devices -- approximately the size of a Band-Aid and no thicker than a credit card -- are implanted just beneath a patient's skin where it is hoped they will provide a steady, long-term regulated supply of self-sustaining insulin.
"Each device is like a miniature insulin-producing factory," said co-author Dr. Timothy Kieffer, a professor within the departments of surgery and cellular and physiological sciences at UBC, and past chief scientific officer of ViaCyte. "The pancreatic islet cells, grown from stem cells, are packaged into the device to essentially recreate the blood sugar-regulating functions of a healthy pancreas. This may have tremendous benefits over transplant of scarcely available donor-derived cells, given that we can create a virtually limitless supply."
The clinical trial was conducted at Vancouver General Hospital, with additional sites in Belgium and the U.S. Ten participants, each of whom had no detectable insulin production at the start of the study, underwent surgery to receive up to 10 device implants each.
Six months later, three participants showed significant markers of insulin production and maintained those levels throughout the remainder of the year-long study. These participants spent more time in an optimal blood glucose range and reduced their intake of externally administered insulin.
One participant, in particular, showed remarkable improvement, with time spent in the target blood glucose range increasing from 55 to 85 per cent, and a 44-per-cent reduction in their daily insulin administration.
The results are the latest in a series of clinical trials funded by Canada's Stem Cell Network and conducted by the UBC-VCH team.
"The Stem Cell Network is delighted to support this clinical trial and we're pleased to see the promising results," said Cate Murray, president and CEO of the SCN. "Moving toward a functional cure for diabetes will require a coordinated and collaborative effort. It takes excellent science by top researchers in world-leading institutions, funders, like SCN, that de-risk research, and innovative biotech companies that can manufacture and scale the technology. SCN is proud to play its part and we look forward to what's next in game-changing diabetes research."
The trials aim to accelerate the development and clinical testing of novel stem-cell based therapies for the 300,000 Canadians living with Type 1 diabetes. Diabetes is estimated to cost the Canadian health-care system $29 billion annually.
Previously, in a 2021 study in Cell Stem Cell, the researchers were the first to show that the approach could produce insulin in the human body. The latest trial sought to significantly increase the amount of insulin produced by leveraging two-to-three times more devices per participant, alongside an updated device design with small perforations to allow for blood vessel ingrowth -- a feature aimed at improving survival of the lab-grown cells.
In another ongoing trial, the UBC-VCH team is investigating whether a version of the device containing cells that have been genetically engineered to evade the immune system, using CRISPR gene-editing technology, could eliminate the need for participants to take immunosuppressant drugs alongside the treatment.
"We envision a future where people with Type 1 diabetes are able to live their lives free from daily insulin injections and free from immune-suppressing drugs," said Dr. Thompson. "That future is now within reach, and Canada is leading the way in efforts to bring these novel treatments to patients."
Meta-Analysis of Cord Blood for Cerebral Palsy November 2023
Megan Finch-Edmondson, PhD, on behalf of the study team Megan Finch-Edmondson, PhD, lead author Introduction to cerebral palsy: Cerebral palsy is an umbrella term that describes a group of disorders affecting a person’s movement and posture. Cerebral palsy is a life-long condition that arises from injury to the brain during development, usually before, during or shortly after birth. Cerebral palsy is a highly variable condition. Symptoms may include difficulties in walking, balance and motor control, eating, swallowing, speech or coordination of eye movements. Cerebral palsy can vary in the way it affects an individual’s movement (type), the part of the body affected (topography) and by how severe the symptoms are (severity), which can be measured using the Gross Motor Function Classification System (GMFCS). In addition, people with cerebral palsy may also experience other impairments or co-occurring conditions such as epilepsy, behaviour disorders, or vision or hearing impairments. What treatments exist? Numerous interventions exist that help people with cerebral palsy to develop and maintain their functional skills as well as reduce the symptoms of cerebral palsy. These include various rehabilitative and medical interventions such as physical, occupational, and speech therapies, as well as botox injections, oral or intrathecal baclofen and even surgery to manage muscle stiffness and contractures. Currently there are no available treatments that directly target the underlying brain injury to improve function and quality of life. Why cell therapies? There is significant interest in investigating cell therapies such as umbilical cord blood as a treatment for cerebral palsy. This is because umbilical cord blood contains a variety of stem and progenitor cells that have been shown to be beneficial following a brain injury in laboratory research. These benefits include reducing inflammation and cell death, and promoting repair following a brain injury, mainly via release of cellular factors (“trophic mechanisms”). This may help to reduce the size or severity of an injury, and/or improve the connections within the brain. Cord blood for cerebral palsy: The earliest reported infusions of umbilical cord blood for cerebral palsy date back nearly 20 years. This was included in a paper published by Sun et al. in 2010 in which 140 children with cerebral palsy received autologous (their own) umbilical cord blood from March 2004 to December 20093. Since then, numerous studies have been published, with a 2021 review finding nearly 800 individuals with cerebral palsy had been treated with umbilical cord blood across all phases of clinical studies, including six medium/large randomised placebo-controlled trials. A 2016 systematic review concluded that cord blood treatment is safe and more effective than rehabilitation alone in improving gross motor function. Τhe study also suggested that umbilical cord blood treatment in children with cerebral palsy improved gross motor skills more than controls, and that higher cell dose and repeated treatments are likely beneficial, with data presented for the 6-month follow up timepoint.
https://parentsguidecordblood.org/en/news/meta-analysis-cord-blood-cerebral-palsy
How Long Can Cord Blood Be Stored? October 2023
The cryogenic storage of cord blood was pioneered by Dr. Hal Broxmeyer at the Indiana University School of Medicine. He preserved the cord blood for the world’s first cord blood transplant in 1988: the cord blood from newborn Alison Farrow was frozen and shipped to Paris to treat her brother Matt Farrow. Over the decades since then, Broxmeyer’s laboratory has demonstrated that cryopreserved cord blood is still viable after 5, 10, 15, and 23.5 years in frozen storage. Dr. Hal Broxmeyer passed away in 2021, but his laboratory team has continued his work and in summer 2023 they published a demonstration that cryopreserved cord blood is still viable after 27 years in frozen storage. The existing research shows that when cord blood is properly cryopreserved, it does not seem to age at all. In Nov. 2018, Australia’s BMDI public cord blood bank announced that they had successfully transplanted a leukemia patient with a donor cord blood unit that had been in storage for 20 years. In May 2023, it was reported at the 10th Cord Blood Conference of the China Maternal and Child Health Association that a young adult in China had been cured of aplastic anemia with their own cord blood that had been in storage for 19 years. Before cord blood banking was invented, scientists were already cryopreserving cells for the purpose of animal husbandry, where it is desirable to save semen from high value studs. The record for the oldest cryopreserved sperm to yield live births is held by sheep that were conceived with semen that had been frozen at Australia’s Sydney Institute of Agriculture for 50 years. Their laboratory reported that the live birth rate from the half-century-old sperm was equivalent to sperm that had been in storage for one year. The human record for live births from cryopreserved sperm is held by twins that were conceived through in vitro fertilization using semen that had be stored for 40 years. To conclude, researchers agree that properly cryopreserved stem cells are effectively immortal. To learn more about cord blood banking, visit Parent's Guide to Cord Blood Foundation at https://parentsguidecordblood.org/en/news/how-long-can-cord-blood-be-stored
Cured by his own cord blood 19 years later October 2023
The following story was reported in May 2023 at the 10th cord blood conference of the China Maternal and Child Health Association. The patient has been given the pseudonym “Xiao An”. In Chinese, “Xiao” is a gender-neutral name that is often translated as “dawn”, “little”, and “reverent of the elderly.” "I didn't expect that the umbilical cord blood saved for the child would actually save the child's life 19 years later!" said the father of Xiaoan, an aplastic anemia patient, excitedly. In 2021, 18-year-old Xiao An suddenly suffered from aplastic anemia. Fortunately, the cord blood stored in Tianjin Umbilical Cord Blood Hematopoietic Stem Cell Bank under Zhongyuan Concorde saved his life and he is recovering well. This is by far the longest storage of autologous cord blood used for therapy in China, creating a new record for self-storage and self-use of cord blood in China. Every time Xiaoan's parents recalled their decision to preserve their cord blood, they felt extremely grateful. When Xiaoan was born in 2003, domestic awareness of umbilical cord blood was still in its infancy. With the mentality of "preparation, multiple guarantees, and multiple peace of mind", they stored the umbilical cord blood for their children. It was this seed of life from Xiaoan himself that brought the fire of life back to him 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. 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 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. Xiao An is unfortunate, but also "lucky". Talking about the child's illness and treatment experience, Xiao An's parents are very grateful for storing the umbilical cord blood for the child. Umbilical cord blood is the blood collected from the umbilical vein after the baby is born and the umbilical cord is severed. It is rich in blood-forming “hematopoietic” stem cells and is of great help in the treatment of blood diseases. At present, with the continuous development of medical technology, umbilical cord blood has clinical application value in more than 80 diseases of the blood system and the metabolism. The popularization of umbilical cord blood storage technology will provide health protection for more families. In Xiaoan's treatment case, autologous cord blood played a positive role. At the same time, the mature storage technology and safety guarantee of Tianjin Cord Blood Bank have also been unanimously recognized by clinical experts. The autologous transplant successfully rekindled life across 19 years of time and space, and also guarded a healthy and happy future for a family. Disclaimer: In most countries, it is the policy of the medical community to try to find an unrelated donor for a patient with Aplastic Anemia. This is the case in the United States and in India. However, if an unrelated donor is not available, and if the Aplastic Anemia is considered to be “acquired” (triggered by a virus, and not due to inborn genetics), then doctors may restore the immune system that the patient had at birth by transplanting the patient’s own cord blood stem cells. Another example of this therapy is the story of Henrique in Portugal. References Tianjin Cord Blood Bank. Nineteen years like a day, holding up the "seed of life". Concord News. To learn more about cord blood banking, visit Parent's Guide to Cord Blood Foundation at https://parentsguidecordblood.org/en/news/cured-his-own-cord-blood-19-years-later
Does young blood hold the fountain of youth? Can transfusions of blood from younger donors reverse or slow down the aging process in an older recipient?
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