Could Stem Cell Therapy Help With Autism?

Human stem celsl in biomedical scientific laboratory.

Back in April of 2017, 25 autistic children participated in a study at Duke University in North Carolina. The study – the first of its kind – aimed to treat the children’s’ autism by transfusing the blood from their own umbilical cord. This blood contained rare stem cells and, after the transfusions, two-thirds of the participants showed improvements in their symptoms.

At the time, skeptics – and even the researchers who created the study – were hesitant to announce the findings as a potential treatment for the disorder. Regardless, it was certainly a much-needed medical advancement as The Center for Disease Control and Prevention estimates that 1 in 68 children suffers from a disorder on the autistic spectrum.

Earlier this month, The Marcus Center for Cellular Cures was established at Duke, where the research began. The new Marcus Center is focused on clinical trials to develop and evaluate cellular and tissue-based therapies, learning to harness the body’s own mechanisms for cellular repair and manufacturing and delivering cell tissues and biomaterials to patients in need. In particular, they’re focused on cures for MS, strokes and – of course – autism.

Geraldine Dawson, a PhD, professor of Psychiatry and Behavioral Sciences and director of the Duke Center for Autism and Brain Development was named co-associate director of the center. She noted that “There currently are no FDA-approved biomedical treatments for autism. Our goal is to develop effective treatments that can significantly improve outcomes for individuals with autism and other developmental disorders.”

Their goal is admirable and has the potential to help hundreds of thousands of people around the world.

As mentioned, 1 in 68 children in America suffers from a disorder on the autistic spectrum. Unfortunately, according to a study conducted by Spectrumnews.org, there isn’t very much reliable information regarding its prevalence in other countries. Regardless, it’s widely considered an epidemic and its consequences weigh heavily both on the children and their parents.

Those suffering with Autism Spectrum Disorder (ASD) have deficits in social skills, have trouble with speech and non-verbal communication and engage in repetitive behaviours. Often, they’ll suffer with debilitating anxiety and, according to Focusforhealth.org, 30 percent of autistic children never speak a word, 20 percent have epilepsy, and – in the most serious cases – children are so frustrated that they self-harm.

After Duke’s 2017 study, CNN reported that Gracie Gregory, a 7-year-old who participated, dramatically improved and her parents reported that the changes were monumental. Her disorder went from taking up 75 percent of her day to just 10 percent.

Duke isn’t alone in their progressive research and because of their initial study, scientists and researchers all over the world have developed their own studies and the results are promising.

At the University of Texas Health Science Center in San Antonio, three scientists carried out a study in a rodent model of autism based on ‘an urgent need for new therapeutic strategies’.

In their study which published in Nature, they sought to restore interneuron function within the GABAergic neurotransmitter system. They used a dual-reporter embryonic stem cell line to generate enriched populations of PV-positive interneurons. These interneurons were then transplanted into the medial prefrontal cortices’ of rodents.  The transplants effectively alleviated deficits in social interaction, helped in cognitive flexibility and reduced the core symptoms of autism.

Likewise, research done at the Hospital for Sick Children and the University of Toronto determined that brain stem cells – in collaboration with the environment they live in – actually build brain circuits during development.

Dr. Freda Miller, a lead in the research, said “Neural stem cells are like “parent” cells that generate their children, the neurons and glia that build brain circuits, in a precisely controlled fashion in response to signals from their environment. These signals ensure that there are enough stem cells to build the brain, to make the correct amounts of neurons and glial cells at the right time and place in the developing brain, and that some stem cells persist into adulthood where they can participate in brain repair. If we can understand what these signals are, and how stem cells respond under normal circumstances, then that information will not only allow us to understand what happens in neurodevelopmental disorders such as autism spectrum disorder but will also provide us with the information we need to activate stem cells in the mature brain to promote repair”.

Worldwide, scientists are asking big, important questions in order to better understand autism. The continued support of stem cell research has helped give these scientists the freedom to explore uncharted territories and is bringing them closer to finding effective treatments and potentially even a cure. Continue to read our blog for further updates.

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Functioning Kidney Tissue Produced from Stem Cells

For the first time in the history of medical science, scientists from the University of Manchester have been able to produce functioning human kidney tissue inside a living organism. For the medical community and for ailing patients, this means more effective treatments for kidney disease.

The study, which was published in the journal Stem Cell Reports, was led by Professors and researchers Sue Kimber and Adrian Wolf.

The team were able to grow kidney glomeruli from embryonic stem cells in a laboratory. Kidney glomeruli serve an important function in the filtration of blood and subsequent production of urine. In fact, these microscopic parts of the kidney serve within a network as the first stage in the filtering process. These tiny capillaries grew inside of a plastic culture dish containing a nutrient broth full of molecules to promote kidney development.

Once the kidney glomeruli grew and matured, they were combined with what researchers are calling ‘a gel like substance’. It acted as natural connective tissue which formed a tiny clump and was injected into mice.

The result: mini, human-like kidneys.

Three months after the initial injection, the structures that actually produce urine as a process of removing waste from the blood had formed. The structures, called nephrons, contained most of the parts present in human nephrons and tiny human blood vessels had formed which were nourishing the new kidney structures. But, these new kidneys aren’t yet fully functioning. They lack an essential artery that pumps more blood into them.

Nonetheless, the system was successfully filtering, producing and excreting urine.

“We have proved beyond any doubt these structures function as kidney cells by filtering blood and producing urine – though we can’t yet say what percentage of function exists,” said Professor Kimber.

It’s a stunning advancement in biological and medical science but there is still a ways to go before scientists will be able to grow functioning kidneys to replace failing kidneys in patients. Where human kidneys have about one million glomeruli in their kidneys, this mouse-grown structure contained only a few hundred.

The study was supported by The University of Manchester’s School of Biological Sciences, Manchester Regenerative Medicine Network (MaRM) and Kidneys for Life and funded by The Medical Research Council and Kidney Research UK. It’s clear why: There are over 100,000 people on the kidney transplant waiting list. The waiting list has doubled in size over the last 10 years and people are waiting 5-10 years for a transplant. While hundreds of thousands of people are organ donors, less than 1 percent of deaths offer organs that can be used.

But it’s not just people on transplant waiting lists that are suffering. When you include dialysis, the number grows exponentially.

“Worldwide, 2 million people are being treated with dialysis or transplantation for kidney failure, and sadly another 2 million die each year, unable to access these treatments,” Woolf said in a press release.

With such a growing need for kidneys and very limited availability, it’s paramount that scientists work to find an alternative. This is a great first step and could potentially save the lives of millions.

“… We are tremendously excited by this discovery — we feel it is a big research milestone which may one day help patients,” Woolf said.

Stem Cells Capable of Regenerating Lung Tissue

Over the last several months, studies and trials from around the world have found new and groundbreaking ways to repair lung functions in both humans and mice. In the latest study, researchers from the Perelman School of Medicine at the University of Pennsylvania report identifying a lung stem cell that repairs the organ’s gas exchange compartment.

The findings will be essential in repairing slow to regenerate lung tissue that has been damaged by respiratory ailments like severe influenza, pneumonia, cystic fibrosis and bronchitis to name a few. The European Respiratory Society reports that an estimated one billion people are currently suffering from chronic respiratory conditions.

The team – led by Edward E. Morrisey, PhD, a professor of Cell and Developmental Biology, director of the Penn Center for Pulmonary Biology and scientific director of the Institute for Regenerative Medicine – published their findings in February in Nature.

The lung is often considered one of the most complex organs and in order to find treatments, the team first had to seek to understand its function and complex structure.

“One of the most important places to better understand lung regeneration is in the alveoli, the tiny niches within the lung where oxygen is taken up by the blood and carbon dioxide is exhaled,” Morrisey said. “To better understand these delicate structures, we have been mapping the different types of cells within the alveoli. Understanding cell-cell interactions should help us discover new players and molecular pathways to target for future therapies.”

One of the cell types that line the alveoli are called epithelial cells which are vital in regenerating damaged tissue and restoring gas exchange (breathing) after an injury or illness. Organs like the intestine turn over the entire epithelial lining every five days. But, like we’ve said, lung tissue is slow to regenerate.

In studying epithelial cells in general, the team found and studied an alveolar epithelial progenitor (AEP) lineage which acts as a wetting agent and keeps the lungs from collapsing.

By studying mouse AEPs, the team was able to identify a conserved cell surface protein called TM4SF1. TM4SF1 was used to isolate AEPs from the human lungs which were then used to generate three-dimensional lung organoids.

“From our organoid culture system, we were able to show that AEPs are an evolutionarily conserved alveolar progenitor that represents a new target for human lung regeneration strategies,” Morrisey said.

These studies, while in their beginning stages, provide much-needed insight into how the lung regenerates. By exploring molecular pathways, they may be able to promote AEP function, design drugs that activate signaling within the lung and promote lung regeneration.

“We are very excited at this novel finding,” said James P. Kiley, PhD, director of the Division of Lung Diseases at the National Heart, Lung, and Blood Institute, which supported the study.

“Basic studies are fundamental stepping stones to advance our understanding of lung regeneration. Furthermore, the NHLBI support of investigators from basic to translational science helps promote collaborations that bring the field closer to regenerative strategies for both acute and chronic lung diseases.”

With access to more than 300 lungs through the lung transplant program, the team plans to dive deeper into their understanding of influenza-damaged lung tissue in particular. Such research naturally lends itself to the understanding of other respiratory ailments and has the potential to save the lives of thousands around the world.

Struggling With Menopause? New Stem Cell Treatments Can Help

After stem cells from bone marrow were injected into the ovaries of 33 women, the scientific community is hopeful that the new treatment may be able to help reverse the effects of early menopause and even allow women to continue having children naturally. The trial focused on women with premature ovarian failure (POF) and after just six months of treatment, they began having periods again.

To understand the far-reaching effects of the new treatment, we must first understand the extent to which menopause and POF can affect women.

When occurring after the age of 40, menopause is a normal condition. It marks the end of a woman’s reproductive years as her body stops releasing an egg every month. Unfortunately, while natural, it can induce several uncomfortable changes in the body including slowed metabolism, weight gain, mood changes, hot flashes, thinning hair and dry skin.

When menopause occurs before the age of 40, it’s considered premature ovarian failure (also known as primary ovarian insufficiency). Women affected by POF can become infertile and develop dementia, depression, anxiety, heart disease and osteoporosis.

Until now, there were no fertility treatments that could help the 1 in every 100 women suffering with POF. Some doctors prescribe hormonal replacements to treat other symptoms of POF but even then, their chances of having children are reduced by half and the injection regimes are difficult to keep up with. Other women seek egg donors but, because of religious, cultural, ethnic, or monetary reasons, many women aren’t able to.

The aim of the pioneering stem cell research by US scientists was to ‘‘support improvement in quality of life and reverse infertility’ and so far, it looks like they’ve done just that. Their findings were presented at the Society of Reproductive Investigation in San Diego, California in early March and while the study is ongoing, the results are cause for celebration.

Women who received stem cell injections experienced increased oestrogen levels, symptoms from hot flashes and insomnia decreased, and after six months, their periods began again. What’s more, no complications or safety issues have been reported making it an extremely viable option for women going forward.

Dr Kate Maclaran and Dr Marie Gerval of the Daisy Network charity agree, saying: ‘This study offers hope for women with POI that in the future, they may be able to conceive naturally or have fertility treatment using their own eggs.’

Dr Christos Coutifaris, president of the American Society for Reproductive Medicine (who was not involved in the study) shares the optimism of those who were involved, saying ‘‘These preliminary findings are exciting. If these observations are validated under further experimental protocols, their implications for female fertility and reproductive hormonal function may prove extremely significant.’

Through the injection of stem cells derived from bone marrow, ovarian function can be stimulated, allowing the return of ovulation and normal hormone levels as well as the possibility of pregnancy. All of the women in the study are currently trying to get pregnant and, once the research has been fully carried out, the option to use stem cell therapy as a treatment for infertility across the board will be explored.

This isn’t the first study exploring the use of stem cells in treating infertility. Back in 2009, scientists in China showed that is was possible to isolate stem cells in mice, store them, and then transplant them back into sterile females to enable them to give birth. But, in this most recent study from US researchers, patients are able to reactivate their own ovaries.

The scientific community and women everywhere are looking forward to final results from the study which will show if the women will, in fact, get pregnant as a result of stem cell injections.

Sheep-Human Hydbrids Pave the Way for Organ Transplants

In late-February, scientists and researchers from Stanford University in California announced that they have successfully grown sheep embryos containing human cells. The announcement came during the annual meeting of the American Association for the Advancement in Austin, Texas and while animal rights activists have raised concerns, the ground-breaking research means that soon, supply for organ transplants might finally meet demand.

The process is called interspecies blastocyst complementation. The approach requires genetically disabling the development of a specific organ in a host embryo and introducing human cells with chimera (animal-human hybrid) formation potential.

Through the research, scientists at Stanford have found that human pluripotent stem cells (hPSCs) can integrate and differentiate in livestock species. The takeaway: soon, transplantable human tissues and organs could potentially be grown in engineered animals. But scientists are quick to say that there isn’t a concrete timeline.

‘It could take five years or it could take 10 years but I think eventually we will be able to do this.’ project lead Dr Hiro Nakuachi, a professor of genetics at Stanford, told the American Association for the Advancement of Science conference.

These findings only represent the beginning of a long road towards meeting this goal. By cell count, only about one in 10,000 cells (or less) in the sheep embryos are human. While it is still 99 percent sheep (and one percent human like you and me), it’s still worth celebrating the successful introduction of human cells.

This isn’t the first experiment of its kind. Back in 2016, researchers from the University of California, Davis successfully combined hPSCs cells with pig DNA inside a pig embryo. Over the course of the 28-day study, the human stem cells showed signs of rooting and growing into a transplantable human pancreas.

Likewise, in 2017, another Stanford team proved that a rat-grown pancreas could successfully be transplanted into a mouse with diabetes. The recipients of the pancreas only needed days of immunosuppressive therapy as opposed to life-long treatment to prevent rejection of the organ and the mice were actually cured of their diabetes.

What’s more, less than two years ago, the US government said it would approve funding on animal-hybrid experiments for the sake of organ transplantation, only to later retract their statement because of complaints from animal rights groups.

While it’s easy to understand arguments from such groups, it’s also important to understand why there is so much interest in chimeras for the sake of organ transplants. It’s possible for organs to grow to adult size within just nine months in these surrogate animals, meaning scientists could have found a much-needed solution for terminally ill patients in need of organs.

“We need to explore all possible alternatives to provide organs to ailing people.” said a member of the team and reproductive biologist Pablo Ross from the University of California, Davis.

In the US, someone is added to the organ donor list once every 10 minutes. There are currently around 76,000 people in the US and 6,500 in the UK on organ transplant lists. 32 people are dying everyday waiting for a transplant. With tens of thousands of people around the world in desperate need, it’s crucial that scientists get the funding they need to find solutions that will save them.

Stem Cell Therapy Becoming More Effective Treatment for Meningitis

Meningitis – a very serious disease if not treated quickly – affects upwards of one million people around the world every year according to the Confederation of Meningitis Organisations. What’s more, it’s difficult to diagnose and it most commonly affects babies, toddlers, children and teenagers.  Current treatments don’t guarantee recovery and the repercussions are life-altering, including late-onset learning difficulties, hearing loss, and general developmental delays. While scientists around the world are working tirelessly to develop and test bone marrow transplants for widespread, life-threatening diseases like cancer, scientists in Germany have been leading the way in allogeneic stem cell transplantation to treat meningitis.

Meningitis can be viral, bacterial or fungal. Bacterial is the most severe. Unfortunately, because it’s so difficult to recognize in its early stages, many children and adults are diagnosed too late and face brain damage and even death. Because common symptoms of meningitis (fever, stiff neck, drowsiness, nausea) resemble common symptoms of dozens of other, less harmful diseases, they might not be taken seriously.

In children, the symptoms are even more difficult to recognize as all signs point to a generally fussy baby rather than a sick one. New mother’s likely won’t rush to hospital because their child is especially irritable, tired, or crying, but all three are known symptoms, specifically in toddlers.

Most often, meningitis is treated one of three ways. In each case, though, doctors will usually start with broad-spectrum antibiotics. They’ll likely even prescribe the antibiotics before the test results come back as a preemptive measure. Patients can also be given a lumbar puncture (spinal tap) as quick and definitive (albeit invasive) alternative to blood tests and x-rays. In a lumbar puncture, cerebrospinal fluid (CSF) is collected and in patients with meningitis, the CSF will show low blood sugar, increased white blood cells, and increased levels of protein.

Patients who are confirmed to have meningitis and who aren’t stabilized with the initial course of antibiotics are often hospitalized and treated with injected antibiotics. But even that isn’t enough often times. In the US alone, 10-15 percent of those diagnosed with meningitis won’t survive and of those that do survive, 10 percent will have lingering symptoms like seizures and stroke.

Doctors in Germany were the first to use allogeneic stem cell transplantation. At a children’s hospital in Halles, Germany, a 19-year old was successfully treated, the infection was controlled, and nearly a full neurological recovery was made. It’s since been dubbed the future of meningitis treatment. In allogeneic stem cell transplantation, stem cells are collected from a matching donor, transplanted into the ailing patient, and the stem cells go to work suppressing the disease and restoring the patient’s immune system. This process is different from autologous stem cell transplants which use the stem cells from the patient’s own body. Allogenic stem cells transplants are used around the world to treat cancers such as lymphoma, myeloma, leukemia as well as other diseases of the bone marrow or immune system.

After the success of the 19-year old in Germany, doctors in Germany are keen to help foreign patients. German Medicine Net, created back in 2001 as an answer to the UK’s waiting list problem, co-operates with renowned institutions for stem cell therapy. Meningitis is regarded as a condition that can be considered for treatment. As research and clinical trials continue, the future of medicine – especially in treating meningitis – truly lies in stem cells.

Stem Cell Research Bringing Doctors Closer than ever to HIV Cure

After 30 years and thanks to extensive stem cell research, scientists are closer than ever before to finding a cure for Human Immunodeficiency Virus, or HIV. Led by Dr. Scott Kitchen, an associate professor of hematology and oncology at UCLA’s David Geffen School of Medicine, the group of US scientists from California, Maine, and Washington have successfully engineered blood-forming stem cells that can carry genes capable of detecting and destroying HIV-infected cells.

But it’s not just that the stem cells were able to destroy the HIV-infected cells, they persisted in doing so for over two years without any negative effects. This equates to long-term immunity and the potential to completely eradicate the disease which, after 1981, quickly became the world’s leading infectious killer.

Kitchen received just over $1.7 million from California’s Stem Cell Agency to carry out his research. California has a special interest in the research as the state ranks second in the United States in cases of HIV. Over 170,000 people are infected, incurring healthcare costs which are being billed to the state. The total has continued to rise and now equates to over $1.8 billion per year.

California’s Stem Cell Agency maintains that “A curative treatment is a high priority. A stem cell based therapy offers promise for this goal, by providing an inexhaustible source of protected, HIV specific immune cells that would provide constant surveillance and potential eradication of the virus in the body.”

In the grant details, Kitchen identifies the potential impact of his research:

“The study will allow a potentially curative treatment for HIV infection, which currently doesn’t exist. This will eliminate the need to administer antiviral medication for a lifetime.”

According to his study published in the journal PLOS Pathogens, Kitchen’s curative treatment involves the use of a ‘optimized’ chimeric antigen receptor (CAR) gene that interferes with interactions between HIV and CD4 cells (white blood cells).When a part of the CAR molecule binds to HIV, it’s instructed to kill the HIV-infected cell. These CAR proteins proved highly effective as they killed HIV-infected cells throughout the lymphoid tissues and gastrointestinal tract, two major sites in HIV replication.

If Kitchen and his team are able to effectively kill off infected cells, they have the potential to save millions of those currently infected with HIV across the globe and can also prevent the virus from advancing into Acquired Immunodeficiency Syndrome, or AIDS. In both cases, the immune system is completely broken down. T-cells, which normally fight and prevent all kinds of bacteria and viruses in the body, are weakened and depleted allowing common and usually treatable infections to become deadly.

Throughout the 80’s and early 90’s, long before stem cell research, the number of people carrying HIV continued to climb as it continued to spread and in 1995, complications from AIDS became the leading cause of death for adults aged 25-44. Shortly thereafter, in 1997, the first truly effective treatment was developed. Highly active antiretroviral therapy (HAART) became the standard and there was a 47% decline in death rates.

By the early 2000’s, the World Health Organization set a goal to treat 3 million people and by 2010 there were 20 different treatment options available.  5.25 million people had treatment and over 1 million more were set to start treatment soon.

While these numbers are a massive improvement and the FDA (Food and Drug Administration) is continuing to approve and regulate HIV medical products, the disease is being slowed rather than halted. According to UNAIDS, over 35 million people are still currently living with HIV/AIDS.

Back in 2011, Kitchen co-authored a study about stem cell research in the treatment of HIV/AIDS in the journal Current Opinion in HIV and AIDS. In it, he said that stem cell-based strategies for treating HIV were “a novel approach toward reconstituting the ravaged immune system with the ultimate aim of clearing the virus from the body.”

Since then, he’s continued to reach higher towards that ultimate aim.

Stem cell treatments utilize patients’ own cells for testing on humans and stem cell advances provide the very necessary opportunity for large clinical trials. It is Kitchen’s hope – and it’s safe to assume the worlds’ hope – that stem cell innovation can one day effectively eliminate the disease, therefore preventing its spread, saving billions of dollars in healthcare costs, and – most importantly – saving lives.