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Tag Archive: Research and Trials

  1. Stem cells used to treat leukaemia in toddler Arlo

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    Stem cell donation is vital

    Toddler Arlo Brown was diagnosed with the rare blood cancer myelomonocytic leukaemia in April. Seriously ill, he underwent a stem cell transplant in August. He is still very poorly but according to recent newspaper reports, is showing signs of recovery, although he relies on regular blood transfusions and a tube for his food and medication.

    While his family wait to see if the transplant has worked for little Arlo, a huge fundraising campaign has started to raise awareness of the life changing impact of stem cell and bone marrow donations.

    His parents Paul and Jodie Brown are urging people to find out if they are eligible to become donors through the charities DKMS and Anthony Nolan, and want to raise awareness of how easy it is to donate stem cells.

    Find out more about the work Anthony Nolan does and how to be a donor here

    Precious umbilical cord blood cells are so important

    Why is it so important to consider either banking or donating your baby’s umbilical cord blood?

    All of the blood cells in your body start out as immature cells called hematopoietic cells which are very young, and not fully developed.

    These are the same stem cells that are found in your baby’s umbilical cord blood and they can mature into any type of blood cell, depending on what the body needs when each stem cell is developing.

    Stem cells for transplants come from the bone marrow (from you or someone else); the blood stream (peripheral blood from you or someone else) and umbilical cord blood from newborns.

    The blood of newborn babies usually has large numbers of stem cells. After birth, the blood that’s left behind in the placenta and umbilical cord (known as cord blood) can be taken and stored for later use in a stem cell transplant. Cord blood can be frozen until it is needed.

    A cord blood transplant uses blood that is normally thrown out after a baby is born. If you choose to bank it, a specially trained person – a phlebotomist – will carefully collect this blood.

    Stem cells in umbilical cord blood can be used to treat leukemia

    A study published in the New England Journal of Medicine led by researchers at the Fred Hutchinson Cancer Research Centre found that in patients at high risk of relapse after transplant, cord blood recipients seem to have better outcomes against leukaemia and the related bone marrow myelodysplastic syndrome. 

    The study found that the probability of overall survival after receiving a transplant from a cord-blood donor was at least favourable as those who had received a transplant from an HLA-matched (human leukocyte antigen) unrelated donor, and was considerably higher than those who had received a transplant from an HLA-mismated unrelated donor.

    Stem cells in umbilical cord blood are less developed than in adult stem cells, and they don’t have to be ‘matched’ as stringently to a patient’s human leukocyte antigen, or HLA type. 

    HLA genes are part of everyone’s unique genetic background that determines the likelihood of rejecting donor stem cells.

    Doctors will typically look our for a ten out of ten match of HLA genes between patients and donors, but if such a perfect match doesn’t exist among relatives or unrelated donors, they will often go with an eight out of ten, or nine out of ten match.

    Transplants from such ‘mismatched’ donors may be better than no transplant at all, but, according to studies such as the one described, cord blood transplants may be the best option of all.

    If you are considering banking your baby’s umbilical cord blood please talk to Biovault Family and we can answer all your questions. 

    Email us at, or find us on Facebook and send us a message.

    You can read more about umbilical cord blood and cancers in our Health Hub


    The Anthony Nolan Trust was set up in 1974 by Shirley Nolan’s whose her three-year-old son Anthony was in urgent need of a bone marrow transplant. Shirley set up the world’s first register to match donors with people in desperate need.

    The New England Journal of Medicine

    Parents Guide to Cord Blood 

    To date, there have been over 35,000 cord blood transplants world-wide, and most of them were for leukemias and other blood disorders (Ballen Verter Kurtzberg 2015).

     Parent’s Guide to Cord Blood Foundation at

    Health Day Article describing a Study in 2016
  2. Intravenous Infusion of Umbilical Cord Mesenchymal Stem Cells Maintains and Partially Improves Visual Function in Patients with Advanced Retinitis Pigmentosa

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    Retinitis pigmentosa (RP) is a hereditary retinal degeneration disease with no effective therapeutic approaches.

    Inflammatory and immune disorders are thought to play an important role in the pathogenesis of RP.

    Human umbilical cord mesenchymal stem cells (UCMSCs), with multiple biological functions such as anti-inflammation and immunoregulation, have been applied in different systemic diseases.

    We conducted a phase I/II clinical trial aiming to evaluate the safety and efficacy of intravenous administration of UCMSCs in advanced RP patients. All 32 subjects were intravenously infused with one dose of 108 UCMSCs and were followed up for 12 months. No serious local or systemic adverse effects occurred in the whole follow-up.

    Most patients improved their best corrected visual acuity (BCVA) in the first 3 months. The proportions of patients with improved or maintained BCVA were 96.9%, 95.3%, 93.8%, 95.4%, 90.6%, and 90.6% at the 1st, 2nd, 3rd, 6th, 9th, and 12th month follow-up, respectively. Most of the patients (81.3%) maintained or improved their visual acuities for 12 months.

    The average NEI VFQ-25 questionnaire scores were significantly improved at the third month (P < 0.05). The average visual field sensitivity and flash visual evoked potential showed no significant difference (P = 0.185, P = 0.711).

    Our results indicated that the intravenous infusion of UCMSCs was safe for advanced RP patients. Most of the patients improved or maintained their visual functions in a long term. The life qualities were improved significantly in the first 3 months, suggesting that the intravenous infusion of UCMSCs may be a promising therapeutic approach for advanced RP patients.


    Tongtao Zhao, Qingling Liang, Xiaohong Meng, Ping Duan, Fang Wang, Shiying Li, Yong Liu, and Zheng Qin YinPublished Online:15 Jul 2020

  3. Hope for the treatment of autism with umbilical cord blood

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    Results from a study have revealed encouraging results for the improvement of symptoms of autism in young children following treatment with umbilical cord blood.

    Autism spectrum disorder (ASD) impairs the way a person communicates and the way they perceive and interact with the world around them. The causes of autism are not yet fully understood but research suggests that genetic and environmental factors may account for changes in brain development. 

    At the Duke Centre for Autism and Brain Development Dr Joanne Kurtzberg and other researchers are bringing new hope to the treatment of autism. 

    Cord blood has the ability to cross the blood–brain barrier and differentiate into neurons and other brain cells. There are a few schools of thought on how cord blood can repair brain trauma or neurodegenerative disorders:

    • The transplanted stem cells directly replace dead or dying cells.
    • The transplanted stem cells secret growth factors that indirectly rescue the injured tissue.
    • The transplanted stem cells build a “biobridge” that connects the healthy section of the brain and the damaged section of the brain to facilitate the transport of new neural stem cells to the area in need of repair.

    Duke University Medical Centre have found that 70 per cent of the children in their study who received cord blood had improvement in one or more of the core symptoms of autism.

    180 children aged 2 – 7 years of age with a confirmed diagnosis of ASD were tested to see whether cord blood infusions would improve symptoms of autism.

    The children tested did not have a known genetic cause of ASD, had no other illnesses, were English speaking, had tested negative for Fragile-X, a genetic disorder characterized by mild-to-moderate intellectual disability, and had a negative chromosomal microarray. They were divided into two sections – treatment and placebo. 

    Children received autologous cord blood (their own cord blood) if they had a qualifying unit that contained a minimum of 25 million cells per kg of the child’s weight, based on the pre-cryopreservation count. Children lacking an autologous cord blood unit received a >4/6 HLA matching unrelated donor cord blood unit. Both the autologous and donor cord blood units had to contain a minimum cell dose of 25 m.

    120 children received cord blood (60 autologous and 60 allogeneic) and 60 children received placebo for their first infusion. Children were analyzed for response at 6 months after their first infusion.  Children crossed over at 6 months to a second infusion. Children receiving cord blood for their first infusion received placebo for their second infusion. Children receiving placebo for their first infusion received cord blood for their second infusion. The type of infusion given at baseline and at 6 months was blinded so that no one interacting with or testing the children knew which infusion was given at each time-point.

    The study showed significant improvements in the cord blood group for the subset of children on the study who were 4-7 years of age and were without intellectual disability (NVIQ >70). Improvements were seen in communication (VABS-3 Communication Scale), attention (eye tracking), and increased alpha and beta EEG power. 

    In most of the measures, there were no advantages of allogeneic or autologous cord blood identified. But on the Clinical Global Impression-Improvement scale, only children receiving allogeneic cord blood showed improvement, compared to placebo.

    It is very important to note that children receiving allogeneic cord blood were given a higher total dose of cells compared to the children receiving autologous cord blood. Thus, it was not possible to know whether this ‘advantage’ of allogeneic cord blood was due to increased dose or the donor cell source.

    The study has concluded that there were encouraging results for the sub-group of children ages 4-7 with NVIQ >70. Significant improvements were seen in communication, eye tracking and EEG Bran scans.

  4. Stem cells hold huge promise for the treatment of Parkinson’s disease

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    Stem cells offer the greatest potential in any area of medicine to treat some of humanity’s most devastating and debilitating conditions.  These include; Parkinson’s disease; spinal cord injury; heart attack; diabetes; blindness; multiple sclerosis; Alzheimer’s; stroke; organ transplantation; arthritis and osteoporosis; bone, tendon and cartilage damage; and sports injuries.

    This year Black Sabbath frontman’s Ossie Osbourne’s daughter reported he was ‘feeling better’ and had seen his Parkinson’s symptoms improve since undergoing experimental stem cell treatment for the neurodegenerative condition.

    “Seeing, after one treatment of stem cell, what has happened and the progress that he’s made is mind-blowing,” said Kelly. “He wants to get up. He wants to do things. He wants to be a part of the world again. He’s walking better. He’s talking better. His symptoms are lessening. He is building the muscle strength back that he needs.”

    Ozzy was diagnosed in 2003 with Parkin 2 – a very rare genetic form of Parkinson’s.

    Parkinson’s is a progressive neurological condition. This means that it causes problems in the brain and gets worse over time. 

    It develops when cells in the brain stop working properly and are lost over time. These brain cells produce a chemical called dopamine.

    Symptoms start to appear when the brain can’t make enough dopamine to control movement properly.

    There are 3 main symptoms – tremor (shaking), slowness of movement and rigidity (muscle stiffness) – but there are many other symptoms too.

    The number of people diagnosed with Parkinson’s in the UK is about 145,000. But there are more than 1 million people in the UK who are affected, either by living with Parkinson’s, or as a friend, colleague, or family member of someone who is.

    Stem cells hold tremendous promise for regenerative medicine due to their unique ability to self-renew and divide into specialised cells. This means that when the body needs more stem cells, they can replicate to produce more specialised cells that can replace damaged ones.

    They can be derived from several sources including umbilical cord blood, embryonic origins or reprogrammed from adult cell types.

    While no stem cell-based treatment has been proven safe and effective for the treatment of Parkinson’s, stem cells do hold huge promise, which is why pioneering research is happening around the world to develop treatments.

    Read more about stem cell research for Parkinson’s disease here.

  5. Umbilical cord cells to be used in breakthrough treatment for Covid-19

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    Blood derived from umbilical cord cells are being used to treat patients with serious forms of coronavirus.

    In a world first, discovered by Australian researchers, patients at a Melbourne Hospital will be infused with the treatment to check its safety.

    The trial will recruit up to 24 patients with moderate to severe pneumonia, which develops in serious cases of Covid-19, and it is expected to be finished before the end of the year.

    The treatment could prevent the progression of pneumonia.

    Speaking to the, study co-leader Professor Graham Jenkin, Monash University,  said: “It’s not going to cure coronavirus and its not like a vaccine that will prevent it, but it’s particularly designed for patients who progress from the very mld form [of the virus] to the very dangerous form that causes people to have to go to hospital.”

    A good example of this kind of case is Prime Minister Boris Johnson who was hospitalised after his condition worsened. Mr Johnson made a full recovery, but this is not always the case, and Mr Jenkins comments that if patients are treated as soon as they arrive at hospital, before their condition progressed to Acute Respiratory Distress Syndrome (ARDS),  it could prevent deaths.

    ARDS develops from cytokine stork syndrome, an over-reaction of the body’s immune system to the virus caused by localised over production of inflammatory factors.

    The study’s co-leader is Dr Atul Malhotra, a clinician scientist at Monash Health and Monash University, who said when the virus enters the immune system it triggers the body’s immune response to attack the virus, resulting in localised inflammation. 

    This can result in hyper-inflammation, causing serious harm to affected organs, causing multi-organ failure and, if untreated, the cytokine storm syndrome, which is usually fatal.

    Increasing evidence suggests that cells from umbilical cord blood are anti-inflammatory, immunosuppressive and lodge in the lung—the primary site of the SARS CoV-2 infection – when given intravenously.

    Mr Malhotra said: “Our aim in this COVID trial is to prevent the hyper immune reaction leading to a cytokine storm before it progresses to acute respiratory distress syndrome.”

    This is the first trial to use cord blood-derived cells for Covid-19 related pneumonia.

  6. New Cell Therapy Trial Launches for Patients with Severe COVID-19

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    Researchers at the University of Miami Miller School of Medicine have received approval from the United States Food and Drug Administration to start a small clinical trial using stem cells against COVID-19.

    The trial will evaluate the use of umbilical cord-derived mesenchymal stem cells to address the symptoms of life-threatening lung inflammation suffered by people with the severe form of the disease caused by Coronavirus.

    Dr. Camillo Ricordi, director of the Diabetes Research Institute and Cell Transplant Centre at the school and the principal investigator on the trial, said the cells derived from one umbilical cord, which are typically discarded after a woman gives birth, could generate enough stem cells to treat over 10,000 patients.

    Stem cells have the capability of becoming specialized cells that sometimes repair damage, and the type taken from umbilical cords — known as mesenchymal stem cells — are adaptable to a number of uses.

     “There is no time to waste,” said Dr. Ricordi. “Patients who die from COVID-19 have a median time of just 10 days between first symptoms and death. In severe cases oxygen levels in the bloodstream drop, and the inability to breathe pushes patients toward their end very quickly; any intervention that might prevent that trajectory would be highly desirable.”

    Ricordi is particularly optimistic about that potential for the type of life-threatening lung condition that can accompany COVID-19, because IV infusions of the stem cells are known to naturally flow to the lungs.

    Beyond that, Ricordi said the mesenchymal stem cells are known to have anti-inflammatory qualities that help calm so-called “cytokine storms,” when a patient’s immune system is kicked into overdrive and does more damage than the virus itself.

    “It’s practically injecting an army of cells that can fight some of the most severe complications of the virus infection,” Ricordi told the Miami Herald.

    The 24-patient trial will begin this week in Miami, with half of the patients receiving the stem cell therapy and the other half serving as the control group. Those patients will not receive stem cell infusions but will still be administered advanced medical treatment, Ricordi said.

    As an established diabetes researcher, Ricordi has already developed notable treatments with stem cell therapy, including one trial that allowed patients with Type 1 diabetes to avoid insulin after receiving stem cell transplants.

    After seeing small but promising studies in China and Israel, Ricordi said he is “very optimistic” because he works with those researchers.

    The China study looked at 10 patients in Beijing from January 23 to February 16, observing the effects of the treatment for two weeks after stem cell injections in seven of those patients. For all seven, the researchers found that lung function and other symptoms significantly improved two to four days after the treatment.

    Ashok K. Shetty, associate director of the Institute for Regenerative Medicine at Texas A&M University College of Medicine and co-editor of the the Aging and Disease medical journal, said in an editorial that the Chinese study showed promise.

    “This study demonstrated that intravenous infusion of MSCs is a safe and effective approach for treating patients with COVID-19 pneumonia, including elderly patients displaying severe pneumonia,” Shetty said.

    Ricordi said UM already has the manufacturing capability in place to treat the patients in the trial, and that could be expanded quickly. He added that if the trial is expanded, he believes the team would have the ability to treat all of the COVID-19 patients at UM and its partner Jackson Health System, Miami’s public hospital.

    The doctors should be able to tell if that expansion is coming soon.

    “This is not a study you have to follow up with in six months, because the results are immediate,” Ricordi said. “In one week, you know: Is it working or not?”

    To that end, Ricordi said UM researchers and doctors are already preparing to expand the trial to more patients.

    “We are already doing cell production anticipating this,” he said. “We are planning for success, but of course we have to see how it does with our patients.”

    Article source:

  7. Covid 19? What it means for mums-to-be

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    None of us has seen anything like the coronavirus pandemic before. We are facing the worst public health crisis in a generation, and the virus is quickly affecting just about every aspect of people’s lives, including the birth of our children.

    Understandably, many mums-to-be are asking if the Covid19 can be passed to the baby in utero. The virus and protocols are so new that limited information is available but there has been some encouraging news for pregnant women who may be exposed to the SARS-CoV-2 virus that causes the disease.

    According to a new observational study on women with COVID-19 in their third trimester of pregnancy, the virus does not appear to travel across the placenta from mother to cause infection of the fetus.

    In China, where the outbreak began, 38 documented cases of pregnant women in China diagnosed with COVID-19 were observed.

     All women, between the ages of 26 and 40, were in their third trimester when they developed the infection. All women affected delivered via C-section. Thirty hours after birth, a newborn from a coronavirus-infected mother also tested positive for the virus.

    To confirm if the virus was transmitted before, during or after birth, physicians and researchers looked at samples of amniotic fluid, umbilical cord blood, breast milk and samples from the newborn’s throat. It was concluded a mother does not pass the virus to the baby in utero. Physicians suspect the baby was infected after touching the mother.

    Significantly, as of the observational study’s publication date, there were no confirmed cases of prenatal transmission of SARS-CoV-2 from mother to infant. 

    In those cases where amniotic fluid, placentas, umbilical cord blood and throat swabs of newborns were tested, all were negative for the virus despite some cases of pre- and post-birth complications. Fortunately, in the 38 women evaluated in this article, COVID-19 does not appear to cause maternal death which is reassuring at this moment in time for mums-to-be.

    These practical tips are very important to follow in order to reduce risk of infection:

    • Avoid touching your eyes, nose, and mouth.

    • Cover your cough or sneeze with a tissue, then throw the tissue in the trash.

    • Clean and disinfect frequently touched objects and surfaces using a regular household cleaning spray or wipes.

    • Wash your hands often with soap and water for at least 20 seconds. If soap and water are not readily available, use an alcohol-based hand sanitizer with at least 60% alcohol.

    • Vaccinations – Stay up to date on vaccinations, including the influenza vaccine.

    • Avoid close contact with people who are sick. Maintain at least six feet of distance from anyone exhibiting obvious symptoms.

    • Stay home when you are sick.


  8. Important Trials Underway Using Stem Cell Technology to Try and Find a Cure for Coronavirus

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    Could stem cell technology be used in the treatment of people critically ill with the coronavirus?

    Science and Technology Daily has reported that Chinese researchers are currently studying this.

    Four Covid-19 patients who received stem cell treatment while in a serious condition have been discharged from hospital in China after recovering, Science and Technology Daily has reported.

    The treatment used mesenchymal stem cells which are the adult stem cells traditionally found in the bone marrow and other tissues including cord blood, peripheral blood, fallopian tube, and fetal liver and lung.

    Why stem cells?

    When most people think about stem cells treating disease they think of a stem cell transplant.

    In a stem cell transplant, stem cells are first specialised into the necessary adult cell type. Then, those mature cells replace tissue that is damaged by disease or injury. This type of treatment could be used to:

    • Replace neurons damaged by spinal cord injury, stroke, Alzheimer’s disease, Parkinson’s disease or other neurological problems;

    • Produce insulin that could treat people with diabetes or cartilage to repair damage caused by arthritis; or

    • Replace virtually any tissue or organ that is injured or diseased.

    But stem cell-based therapies can do much more including the treatment of Novel Coronavirus Severe Pneumonia, as revealed on

    Mesenchymal stem cells have the ability to:

    • Self-renew and multiply
    • Maintaining the potential to develop into other types of cells. 
    • Become cells of the blood, heart, lungs or other body parts.
    • Perform a strong secretory function, promoting the formation of new blood vessels, cell proliferation and differentiation
    • Inhibit inflammatory response, experts say.

    How can umbilical cord mesenchymal stem cells treat Covid-19?

    Coronaviruses are a group of viruses that usually cause mild illnesses, such as the common cold. However, certain types of coronavirus can infect the lower airway, causing serious illnesses like pneumonia or bronchitis. Most people get infected with coronaviruses at some point in their lives and the majority of these infections are harmless.

    Stem cell therapy has also been used in treating H7N9 avian flu and showed good results.

    A research team from the fifth medical centre of the Chinese PLA General Hospital has been conducting clinical research on the safety and effectiveness of mesenchymal stem cell therapy in treating Covid-19 patients, with the research focusing on improving the condition of critically ill Covid-19 patients.

    When the patients are in a severe condition, the cause of deterioration and even death is the “inflammatory storm”, when the human immune system is over-activated by the infection.

    That will damage the lungs and impair breathing and some severe patients may have respiratory failure.

    Lung inflammation and injury are the focus of the treatment of severe patients, and damage to lung tissue can be fatal.

    Stem cell therapy is expected to help repair the damage.

    Currently, three kinds of stem cells – mesenchymal, lung and embryonic stem cells – are used in treatments. Researchers usually inject stem cell products into the lungs.

    Stem cells can improve the immune microenvironment in the lungs and reduce the risk of pulmonary failure caused by inflammation.


    New Scientist –

    Strait Times – (2020) – Mesenchymal Stem Cell Treatment for Pneumonia Patients Infected With 2019 Novel Coronavirus (2020)- Study of Human Umbilical Cord Mesenchymal Stem Cells in the Treatment of Novel Coronavirus Severe Pneumonia
  9. Alzheimer’s Disease: umbilical stem cells can protect neurons

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    A new paper published in Stem Cell Research & Therapy reveals that stem cells from the umbilical cord can protect neurons in the human brain. This adds to a growing body of evidence showing that umbilical stem cells have the potential to treating and preventing neurological injury and diseases such as Huntington’s, Parkinson’s and Alzheimer’s.

    Umbilical cord stem cells for neurological problems

    Many studies have now shown that Mesenchymal Stem Cells (MSCs) have the potential to treat injuries and diseases that affect the central nervous system. These include:

    • Spinal cord injuries
    • corneal injuries
    • optic nerve injuries
    • neurodegenerative diseases such as, Huntington’s, Parkinson’s, and Alzheimer’s diseases.

    How are umbilical stem cells collected?

    The MSCs used in this study were extracted from Wharton’s Jelly, a cushioning substance found in the umbilical cord. Both cord blood and cord tissue can be collected after birth without disruption to the birthing process or post-natal care.

    Cord tissue can be frozen and cryogenically stored until the stem cells it contains are needed. The cord is collected post-partum, allowing the mother to have a natural third stage. The process of cord tissue collection does not require early clamping, so parents can optimise the health of their child by practising delayed cord clamping as advised by NICE and WHO and store their child’s stem cells, potentially protecting them from future illness or injury.

    Umbilical cord stem cells for Alzheimer’s Disease

    This latest study tested the scientists’ hypothesis that MSCs could protect neurons from damage sustained in Alzheimer’s Disease (AD).

    Alzheimer’s disease (AD) is responsible for 50–70% of dementia cases in the elderly, and effective treatments are still not available (Stem Cell Research & Therapy).

    Mesenchymal stem cells (MSCs) are potential therapies for a number of neurological disorders, including AD. MSCs from the placenta have been shown to reduce memory impairment and stem cells from cord blood have also been shown to promote neuronal differentiation, prevent cell death and improve memory.

    Neuroprotection by MSCs appears to stimulate neurogenesis and/or combat inflammation, a concept known as secretome. In this study, scientists investigated the neuroprotective potential in an in vitro model of Alzheimer’s.

    The team’s findings suggest that stem cells from the umbilical cord are able to protect neurons from oxidative stress and synapse damage. The writers predict that these MSCs may “contribute to the treatment of AD and other neurodegenerative disorders.”

    Find out more about cord tissue and cord blood collection and storage for your family’s future.