Stem Cell Blog
Употребата на матичните клетки од папочна врвца рапидно се зголемува. Пред 10 години крвта од папочна врвца можеше да лекува околу 40 состојби, но денес таа бројка е над 80. Со нетрпение очекуваме нови терапии за болести и нарушувања како што се дијабет, аутизам и мозочен удар, можете да бидете во тек со најновите случувања во регенеративната медицина на нашиот блог за матични клетки.
Researchers are investigating a novel placenta-derived stem cell therapy for multiple sclerosis, a currently incurable degenerative neurological condition. A recent phase 1 clinical trial, with findings published in Scientific Reports, assessed the safety and feasibility of this approach for patients with secondary progressive multiple sclerosis.
What is multiple sclerosis?
Multiple sclerosis is a lifelong condition that affects the brain and central nervous system. It is an autoimmune condition, caused by the immune system mistakenly attacking the myelin sheath which covers and protects the nerves. As a result of the myelin damage, the nerves become less efficient at sending messages to the body. The nerve damage continues to worsen over time, eventually leading to permanent disability.[1][2] Globally, multiple sclerosis affects around 2.3 million people.[3]
There are three main types of multiple sclerosis. In the relapsing-remitting type (RRMS), patients have distinct attacks (relapses) of symptoms, which then fade away partially or completely (remission). In the primary progressive type (PPMS), conversely, there is a gradual worsening of symptoms. Lastly, the secondary progressive type (SPMS) develops after RRMS for many people, and involves a gradual worsening of symptoms with or without active relapses.[4][5]
There is no cure for multiple sclerosis. Currently available treatment focuses on managing symptoms as well as reducing the seriousness and progression of the disease (disease-modifying therapies).[5][1]
Investigating stem cell therapy for multiple sclerosis
Current research is increasingly focusing on trying to find new treatments that would promote myelin regeneration (remyelination), reduce inflammation, and protect the nerves. Mesenchymal stem cells (MSCs) have the potential to address these goals, thanks to their anti-inflammatory, immunomodulatory, regenerative and neuroprotective properties. MSCs derived from the placenta (PL-MSCs), in particular, may have more potent immunosuppressive and immunomodulatory effects compared to other types of MSCs. What’s more, they can be obtained easily and non-invasively.[3]
Researchers posited that PL-MSCs may therefore have a positive impact on patients with SPMS, more specifically on those who no longer respond to conventional treatment (treatment-refractory).
The phase 1 clinical trial involved five patients with treatment-refractory SPMS, and was aimed primarily at determining the safety and tolerability of the PL-MSC treatment over a six-month period. Researchers also looked at exploratory secondary outcomes, including clinical disability, cognitive and psychological assessments, brain imaging (DTI and fMRI), and immunological markers.
What did the study find?
The treatment proved to be safe, with no serious complication occurring. Two patients had a mild headache, but this was resolved with a common painkiller.
Importantly, the results showed sustained improvements in clinical outcomes, with significant reductions in Expanded Disability Status Scale (EDSS) scores, a common measure of MS disability, in the first month after the treatment. By the third month, two participants had a continued reduction in their EDSS scores, while the other three remained stable.
After the treatment, participants also improved in cognitive and psychological tests. Functional MRI analysis suggested significant enhancements in brain connectivity and cognitive function. Blood tests also showed a decrease in the number of B cells, which are immune system cells involved in multiple sclerosis. Inflammatory proteins were found to have decreased, whereas anti-inflammatory protein levels increased.
The potential of placenta stem cells
It is important to keep in mind that this was a small phase 1 trial with a very limited number of participants, no control group and a relatively short follow-up period. The results are quite encouraging, but more research and larger-scale trials are needed to confirm the effectiveness of the treatment.
Nevertheless, studies like these highlight the strong potential of placenta stem cells, as well as other stem cells derived from birth-related tissues, to treat conditions and diseases that are currently considered incurable. Having ready access to a source of these powerful cells could make the difference in the future in terms of treatments that are still being discovered today. To learn more about how to bank your baby’s placenta and umbilical cord for their own future use, fill in the form below to receive your free guide.
References
[1] MS Trust (2022). What is MS? https://mstrust.org.uk/information-support/about-ms/what-is-ms
[4] MS Society (2019). Types of MS. https://www.mssociety.org.uk/about-ms/types-of-ms
[5] NHS (2022). Multiple Sclerosis. https://www.nhs.uk/conditions/multiple-sclerosis/
A review paper, newly published in the prestigious Pediatrics journal, statistically demonstrates that cord blood therapy for cerebral palsy is an effective treatment to improve the motor skills of children suffering from this condition. The paper is an analysis of data from eleven studies on over 400 children with cerebral palsy.
What is cerebral palsy?
Cerebral palsy is a group of motor disorders that affect movement, posture and coordination. It’s caused by damage to the brain while it is still developing. This happens most often before birth, but sometimes during or immediately after.
The severity of the condition can vary greatly. Symptoms include lack of balance and muscle coordination, muscles that are either too stiff or too floppy, exaggerated reflexes, and issues with walking, eating and speech.[1][2]
There is currently no cure for cerebral palsy. Available treatment includes physiotherapy, occupational therapy, medication and sometimes surgery. This is aimed at helping people with the condition to be as healthy and independent as possible.[3]
What did the analysis find?
According to the analysis results, children who underwent cord blood therapy for cerebral palsy combined with rehabilitation saw a significantly greater improvement in gross motor skills compared to those gained from rehabilitation alone. The improvements reached their peak between 6 and 12 months after the cord blood therapy; the data also indicates that higher cell doses per kg of patient weight result in bigger improvements. This is consistent with the current hypothesis that the treatment works by reducing inflammation in the brain and stimulates tissue repair, leading to improvements in brain connectivity.[4][5]
After six to twelve months, the majority (68%) of children who underwent cord blood therapy for cerebral palsy scored higher on the gross motor function measure scale used for cerebral palsy (GMFM-66) than the entire control group. The analysis did, however, also find that the treatment had better results in younger children, as well as those with less severe cerebral palsy. Overall, the children who saw the best results were those under the age of 5 who had some ability to walk, whether unaided or with support, before the therapy.[4][5]
What does this mean for the treatment of cerebral palsy?
Cerebral palsy remains one of the most common motor disorders in childhood. It can be accurately diagnosed as early as 6 months of age, which is well within the bracket of highest therapy effectiveness identified by the analysis. What’s more, the sooner a child is treated, the better. This is because a single unit of cord blood will result in a higher cell dose per kg when a child is still small, and may even be enough for multiple treatments.[4]
At the present time, cord blood therapy is not approved as a treatment for CP in any country. This is due to it still requiring large-scale, phase 3 trials before it can reach this approval. Therefore, it is only available through clinical trials as well as expanded access or compassionate use programmes.[4]
This lack of approval means that, although there are cord blood units available for use in public banks, it may not be possible to use these for treatment in the immediate without gaining access to a clinical trial. Even then, the clinical trial may require the cord blood used in the treatment to be autologous (the child’s own), or to come from a sibling donor. Expanded access programmes may also have the same requirements.
A child having access to their own banked cord blood, or a sibling’s, could therefore be essential to ensure treatment can happen during the window of opportunity that could achieve the best results. To learn more about how you could preserve this important health resource for your child and your family, fill in the form below to request your free Parents’ Guide to Cord Blood Banking.
References
[2] NHS (2023). Overview – Cerebral Palsy. https://www.nhs.uk/conditions/cerebral-palsy/
[3] NHS (2023). Treatment – Cerebral palsy. https://www.nhs.uk/conditions/cerebral-palsy/treatment/
A new study on cell therapy for cerebral palsy has, for the first time, directly compared the efficacy of two different treatments. Although more research is needed, the study’s results offer valuable insights for future clinical trials tackling this challenging disorder.
The challenges of cell therapy for cerebral palsy
Over the past two decades, cell therapy has emerged as a promising treatment for cerebral palsy. In particular, mononuclear cells from cord blood (UCB-MNCs) and mesenchymal stem cells from cord tissue (UCT-MSCs) have proven to be a safe and effective therapy. Many clinical trials have obtained results showing improvements in patients’ motor function through these cells’ anti-inflammatory, neuroprotective and regenerative properties.
However, efforts to pinpoint the best treatment method have been stymied by significant variations in trial parameters. Differences from study to study can include the type and severity of cerebral palsy, the age of the patients, the cell type, dose and delivery method, whether the study is open-label or blinded, and even the methods and time periods used to monitor improvement. Consequently, no two studies are alike, and results cannot be directly compared.[1][2]
Study methods and goal
The new study, conducted in Iran, is a pooled analysis of the results of two studies, one on UCB-MNTs and one on UCT-MSCs. Both studies were conducted in the same research centre; moreover, to aid in the comparison, the research methodology and other variables were kept the same as much as possible.[3][4][5]
The study patients were aged 4-14 and had spastic cerebral palsy with white matter lesions. After eligibility screening, 108 patients were randomly assigned to the two treatment arms or a control group. Treatment was done via intrathecal (into the spinal fluid) injection, with the control group receiving a sham procedure instead. Researchers then assessed patients’ motor function, quality of life, disability and spasticity after 1, 3, 6 and 12 months.
Both individual studies were double-blinded. Furthermore, all statistical analysis, both for individual studies and for the final pooled comparison, was performed by a blinded statistician.
The individual studies aimed to confirm that UCB-MNCs and UCT-MSCs are a safe and effective treatment for cerebral palsy. Following that, the pooled study analysis compared the effects of the UCB-MNC treatment to those of the UCT-MSC treatment across the study period.
Study results
Both the UCB-MNC treatment and the UCT-MSC treatment reported positive results over time when compared to the control group. Patients in both groups showed improvement in gross motor function and quality of life, as well as reduction in disability and spasticity.[4][5]
Figure 1. A comparison graph of the two different treatment groups and the control group.[3]
As the figure above shows, researchers found that the UCB-MNC treatment group showed stronger improvements in motor function early on. However, both the UCB-MNC and UCT-MSC groups achieved the same level of improvement at 6 months post treatment. At twelve months after treatment, there was some gradual deterioration of the improvements; however, researchers noted that UCT-MSCs did seem to result in more sustainable changes, with patients seeing less deterioration compared to the UCB-MNC group.[3]
Due to this deterioration, researchers posit that repeated doses at regular intervals may be the best route to continued improvement.[1] They also suggest that future trials should investigate treatments combining both UCB-MNCs and UCT-MSCs, as it may prove more effective than individual ones.[3]
The future of medicine
Researchers stress that this study is only a start, and further comparative trials and research are needed. Although both UCB-MNCs and UCT-MSCs offer positive results, there is still no certainty on which cell type and source will prove most effective treatment. This is true not only for cerebral palsy, but also for other illnesses and diseases for which an effective treatment is still being sought.
This uncertainty highlights the importance of comprehensive stem cell banking. By storing as many stem cell sources as possible, you could equip your baby and family with the broadest range of options for future regenerative therapies.
To find out how you could preserve both cord blood and tissue, along with amnion and placenta, for your baby’s potential future use, fill in the form below to request your free guide.
References
A recent study has found that a stem cell therapy could reduce epileptic symptoms after stroke and help the brain recover. The study was performed at the Gladstone Institute of Neurological Disease in California, USA, using a rat model of stroke.
Stroke basics
A stroke is a medical condition which causes the death of cells in the brain. This, in turn, stops the brain from working properly. There are two main types of stroke: haemorrhagic, caused by bleeding in the brain, and ischemic, caused by the blockage of a blood vessel. Ischemic stroke is by far the most common, accounting for just under 90% of strokes.[1]
Stroke is the second leading cause of death and one of the major causes of disability worldwide.[2] In the UK, it is the single biggest cause of severe disability, causing a range greater than any other condition, including movement issues, visual problems, and speech difficulties.[3]
The goal of the study
The stem cell therapy tested in the study is based on mesenchymal stem cells derived from donor bone marrow. It has already been successful in clinical trials for improving chronic paralysis after traumatic brain injury[4][5]; this led to its approval as a treatment for this issue in Japan[6].
Now, researchers are hoping it could also help reverse brain damage caused by stroke. In particular, they are focusing on brain hyperexcitability, which is a flawed response in the brain that develops in some people after they have had a stroke.
What is brain hyperexcitability?
Put simply, brain hyperexcitability is an increased chance for neurons to activate in response to a specific stimulus. In the case of stroke, this flawed response develops in the brain as it tries to make up for lost functions.
These overly active neurons send out signals that are too frequent or too strong to other regions of the brain. This, in turn, causes serious issues. Brain hyperexcitability can make it difficult to control muscles (spasticity) and can lead to seizures.[7] This means stroke is a leading cause of acquired epilepsy,[8] particularly in people over the age of 35.[9]
Unfortunately, brain hyperexcitability remains not well understood, and there is no treatment available to prevent it[7]. Post-stroke seizures and epilepsy are simply managed, as and when needed, with anti-epileptic drugs.[10]
Study process and findings
Researchers tested the stem cell therapy in a rat model of stroke. A month after rats had a stroke, modified human stem cells were injected into their brains, near the damaged area.
The team then measured electrical activity in the brain to determine the effectiveness of the therapy. Furthermore, they also analysed the structure of the brain and blood cells in detail, so they could study the changes wrought by the stroke and by the therapy.
They found that the stroke had caused hyperexcitability in the rats’ brains. The stem cell therapy, however, had reversed this, returning the brain to normal function. Furthermore, a number of proteins and cells that are important for brain function had also increased.
Additionally, by comparing rats which had received the therapy to control rats, researchers identified molecules in the blood that had changed after the stroke. They further saw that these same molecules were restored to normal by the therapy. Additional analysis found that a week after the transplant, few stem cells remained in the rats’ brain; however, the effects were long-lasting.
Hope for future treatment
This therapy is in the very early stages of research; it remains to be determined whether the reversal of brain hyperexcitability would lead to a reduction of symptoms in actual patients.
Still, studies like these highlight the tremendous regenerative potential of stem cells, and the hope they can offer for the treatment of illnesses and injuries that today are considered incurable.
Today, stroke occurs more than 100,000 times per year in the UK – about once every five minutes. Advances in immediate treatment have meant that the number of patients dying from stroke continues to decrease. Unfortunately, however, rehabilitation hasn’t kept pace, and the number of people living with severe disabilities after stroke continues to increase.[3]
Stem cell therapies for post-stroke disability could prove to be truly lifechanging.
Some of the most potent stem cells that could be used in regenerative therapy come your baby’s umbilical cord and placenta. Both of these are normally thrown away at birth – but they could instead be stored for your baby’s future use. If you want to find out more about this rich source of stem cells, and learn how you could preserve it, fill out the form below to download our free parent’s guide.
References
The results reported from Phase II of the first FDA-approved trial for the treatment of multiple sclerosis (MS) using stem cells are highly promising, suggesting that stem cells could be a viable treatment option to help improve the lives of those with the condition. [1]
What is the MS trial?
The Phase II trial, which was carried out by The Tisch MS Research Center of New York, constitutes the next stage in the development of the groundbreaking research signalled by the results from Phase I, the first FDA-approved trial to explore the injection of stem cells for MS.
The Phase II trial involved 54 patients split between a test group and a control group in a randomised, double-blind, placebo-controlled study.
Members of each group received 6 injections of either autologous (meaning their own) mesenchymal stem cell-derived neural progenitors (MSC-NPs) or saline placebo every two months.
These injections were administered into the cerebrospinal fluid of multiple sclerosis patients.
The study utilised a cross-over model by which patients who received the stem cell injections in year 1 were then given the saline placebo in year 2 and vice versa.
How does the stem cell treatment work?
Building on their findings from Phase I, researchers identified MSC-NPs for the treatment having ascertained that they promote tissue regeneration and immunomodulatory effects, in addition to being safe and well tolerated. [2]
MSC-NPs are a subpopulation of mesenchymal stem cells that can upregulate growth factors like hepatocyte growth factor (HGF), and minimise ectopic differentiation – essentially, the abnormal differentiation of cells.
By injecting them into the cerebrospinal fluid – a process called intrathecal (IE) injection – researchers found in an experimental autoimmune encephalomyelitis (EAE – an accepted way of modelling the effects of MS) mouse model that MSC-NPs were associated with increased spinal cord myelination, neurological recovery and reduced immune infiltration into the central nervous system. [3]
What were the results?
The results of the Phase II trial demonstrated huge promise for the use of stem cells in treating MS.
Patients requiring walking assistance saw significant improvements in both a timed 25-foot walk test and a 6 minute walking test than those in the control group.
Additionally, treatment recipients demonstrated improved bladder function, with 69% showing improvements in post-void residual volume.
Amongst the other findings were indicators that these stem cells could be helping to restore neuronal cells and reverse cognitive decline in patients with less advanced disease progression.
Furthermore, the stem cell treatment occasioned notable biomarker changes in cerebro-spinal fluid, especially with regards to the decreased levels of CCL2, a protein associated with inflammation, and the increased levels of MMP9, an indicator of the increased presence of reparative cells. [4]
Overall these findings are extremely promising, indicating that stem cells could be used in future treatments that may help to reverse many of the most debilitating aspects of multiple sclerosis, like disability.
The next stage in the study will be for researchers to investigate the effects of increasing the stem cell dosage.
What does this mean for cord blood banking?
This trial is just the latest example of the regenerative potential stem cells have to help treat life-changing conditions like MS.
As exemplified by this study, mesenchymal stem cells in particular, with their immunomodulatory and tissue repair properties, are especially promising.
Incredibly, the umbilical cord and placenta are one of the richest sources of these stem cells, but are routinely thrown away after birth.
By saving these for your baby, you could be giving them the key to future therapies for everything from cancer and stroke to diabetes and heart disease.
The potential offered by stem cells is vast, but you only get one chance to save baby’s stem cells, and that’s the day they’re born.
To find out more about cord blood banking and how it could protect your family’s health, fill out the form below to request a free Welcome Pack.
References
[1] FDA-Approved Phase II Stem Cell Treatment Trial Shows Significant and Diverse Improvements for Multiple Sclerosis (MS) Patients. Tisch MS Research Centre of New York. https://www.tischms.org/phase-ii-analysis-results
[2] Harris, Violaine K. et al. (2018) Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis. eBioMedicine, Volume 29, 23 – 30. https://doi.org/10.1016/j.ebiom.2018.02.002
[3] Harris, Violaine K. et al. (2012) Clinical and pathological effects of intrathecal injection of mesenchymal stem cell-derived neural progenitors in an experimental model of multiple sclerosis. Journal of the Neurological Sciences, Volume 313, Issue 1, 167 – 177. https://doi.org/10.1016/j.jns.2011.08.036
[4] Harris, V.K., Stark, J., Williams, A. et al. (2024) Efficacy of intrathecal mesenchymal stem cell-neural progenitor therapy in progressive MS: results from a phase II, randomized, placebo-controlled clinical trial. Stem Cell Res Ther 15, 151 (2024). https://doi.org/10.1186/s13287-024-03765-6
A new therapy, recently granted a UK Innovation Passport Designation, could help to improve treatment outcomes for Krabbe disease patients post-stem cell transplant.
What is Krabbe disease?
Krabbe disease is a rare genetic disorder affecting around 1 in every 100,000 births and is caused by a deficiency of the enzyme galactocerebrosidase (GALC), leading to the breakdown of the myelin in the nervous system.
The most common variant of Krabbe disease, Infant Krabbe Disease, affects children under the age of 1, and can cause muscle stiffness, seizures, and developmental delays. It is often fatal if untreated.
Approximately 85% of Krabbe disease cases are the infantile subtypes. [1]
What are the current treatment options for Krabbe disease?
Currently, the only effective treatment option for Krabbe disease is a transplant of haematopoietic stem cells (HSCs), which are found in bone marrow, peripheral blood, and umbilical cord blood. [2]
By transplanting HSCs from a donor to a Krabbe disease patient, the patient’s unhealthy cells lacking in the GALC enzyme are eventually replaced by healthy red blood cells, white blood cells and platelets derived from HSCs.
These healthy blood cells can then work to populate the brain with GALC enzyme activity, reducing the breakdown of myelin and thereby stabilise cognitive function. [3]
Stem cell transplants using haematopoietic stem cells from cord blood, specifically, have been shown to be highly effective in improving neurological outcomes if the transplant is performed before the development of symptoms. [4]
While HSCT remains the only viable treatment option for Krabbe disease, it is not a cure.
It also does not combat the peripheral neuropathy occasioned by Krabbe disease, a condition affecting the nerves beyond the brain and spinal cord, leading to decline in motor function. [5]
What is the new gene therapy and how does it improve stem cell transplant outcomes?
Developed by Forge Biologics, the FBX-101 therapy works by delivering a copy of the GALC gene to cells in the nervous system, which improves myelination (the process by which the myelin sheath forms) and, crucially, motor function.
It is designed to be administered intravenously after the current standard of care, a haematopoietic stem cell transplant.
In its early phase trial, REKLAIM, FBX-101 was shown to improve motor function in all five of the patients who underwent treatment.
Building on these promising results, the Innovation Passport designation means that FBX-101 will be able to enter the Innovative Licensing and Access Pathway (ILAP), which accelerates both market and regulatory access in the UK. [6]
Why is newborn screening for Krabbe disease so important?
Newborn screening for Krabbe disease is crucial because the condition progresses rapidly, especially in its infantile form, and early intervention is the key to preventing severe neurological damage.
Ideally, the patient would receive a transplant within 30 days in order to have the best chance of improved neurological and transplant outcomes. [7]
Symptoms of Krabbe disease often appear within the first few months of life, and once they start, the deterioration of the nervous system is fast and irreversible.
By the time symptoms are noticeable, significant damage has already occurred, limiting the effectiveness of available treatments.
Newborn screening allows for early diagnosis before symptoms develop, enabling early intervention through HSCT, which is at its most effective if administered before significant damage to the nervous system. [8]
Could cord blood banking help?
Umbilical cord blood is a vital source of haematopoietic stem cells which can differentiate into various kinds of blood cells. These cells are crucial in the treatment of Krabbe disease, but rely on finding a donor match in order for a transplant to be successful.
If Krabbe disease runs in your family, saving cord blood for every child is probably a worthwhile investment.
If your baby does end up developing Krabbe disease, having their sibling’s stem cells in storage could make the difference in being able to access a life-saving transplant as a sibling has a 75% chance of being a partial donor match.
For more information about how cord blood banking could help with the treatment of Krabbe disease, visit our Krabbe Disease and Stem Cells page, here.
If either you or a family member or someone you know is expecting, why not download our free Welcome Pack to learn more about the benefits of cord blood banking. Simply fill out the form below.
References
[1] Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar; Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood 2021; 137 (13): 1719–1730. doi: https://doi.org/10.1182/blood.2020005477
[2] Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar; Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood 2021; 137 (13): 1719–1730. doi: https://doi.org/10.1182/blood.2020005477
[3] (2022, August 13). Krabbe Disease (Globoid Cell Leukodystrophy). Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/6039-krabbe-disease-globoid-cell-leukodystrophy
[4] Wright, M. D., Poe, M. D., DeRenzo, A., Haldal, S., & Escolar, M. L. (2017). Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology, 89(13), 1365–1372. https://doi.org/10.1212/WNL.0000000000004418
[5] Beltran-Quintero, M.L., Bascou, N.A., Poe, M.D. et al. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis 14, 46 (2019). https://doi.org/10.1186/s13023-019-1018-4
[6] (2024, March 19). Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation. Forge Biologics. https://www.forgebiologics.com/forge-biologics-novel-aav-gene-therapy-fbx-101-for-patients-with-krabbe-disease-is-granted-uks-innovation-passport-designation/
[7] Page, K. M., Ream, M. A., Rangarajan, H. G., Galindo, R., Mian, A. Y., Ho, M. L., Provenzale, J., Gustafson, K. E., Rubin, J., Shenoy, S., & Kurtzberg, J. (2022). Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease. Blood advances, 6(9), 2947–2956. https://doi.org/10.1182/bloodadvances.2021006094
[8] Page, K. M., Ream, M. A., Rangarajan, H. G., Galindo, R., Mian, A. Y., Ho, M. L., Provenzale, J., Gustafson, K. E., Rubin, J., Shenoy, S., & Kurtzberg, J. (2022). Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease. Blood advances, 6(9), 2947–2956. https://doi.org/10.1182/bloodadvances.2021006094
There’s exciting news from the University of Galway, where researchers are developing a new technique that they hope will improve the viability of stem cell treatments for Parkinson’s disease.
The pioneering method, which involves the use of a substance called hydrogel, could revolutionise the way stem cell treatments for Parkinson’s disease are carried out, making them more effective and less prone to failure.
Parkinson’s disease is a neurological condition affecting around 150,000 people in the UK. [1]
The condition, which is caused by a lack of dopamine in the brain, manifests in severe ways with symptoms including tremors, muscle rigidity and slowness of movement. [2]
Unfortunately, Parkinson’s is degenerative, meaning that it gets worse over time. It can make people who live with the condition more vulnerable to poor health and disability, which can end up having fatal repercussions in some cases.
The degeneration of nerve cells in the brain is what results in the underproduction of dopamine. The aim of recent stem cell research has been to find a way of repairing and replacing these cells through the use of induced stem cells.
These induced stem cells are harvested from different areas of the body, such as skin, and then reprogrammed to become the type of cells necessary for brain repair.
However, these cells require transplantation at a very early stage of their development into brain cells and once transplanted, many of them do not end up converting.
What researchers at the University of Galway have discovered is that by transplanting these induced stem cells in a collagen hydrogel, effectively a water-based scaffold, significantly improves the chances of the stem cells both surviving and then differentiating into the cells necessary for therapy. [3]
With funding from the Michael J. Fox Foundation for Parkinson’s Research (MJFF), the study’s findings were published in the Journal of Neural Engineering and have been met with widespread acclaim.
The research is ongoing, but the team at the University of Galway and MJFF hope that this new transplantation technique will significantly improve outcomes for sufferers of Parkinson’s disease.
If you want to learn more about how you could give your baby access to future stem cell therapies, download our FREE Welcome Pack below.
References
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