9 Things to Know About Stem Cell Therapy and Treatments

Stem cell research and treatments represent exciting advancements in medicine. This innovation and our patients’ needs drive everything that we do. All of our efforts are focused on bringing life-saving medicines to the market.

With more than 20 tissue-specific regenerative medicines in our pipeline, we want the public to understand what stem cells are, their potential in treating a range of formerly untreatable conditions, and why their use in medicine has been considered controversial in the past.

  1. Different Stem Cells Serve Different Purposes

There are four major types of stem cells:

Embryonic Stem Cells: ES Cells are cells derived from early stage pre-implantation embryos. In order to harvest these stem cells, an embryo has to be fertilized in a laboratory as opposed to inside the female body. ES cells are pluripotent, meaning they can divide into more stem cells and can become other types of cells in the body.

Adult Stem Cells (Tissue-Specific Stem Cells): Adult stem cells (found in both children and in adults) come from developed organs and tissue. They can self-renew indefinitely to replenish dying cells and to regenerate damaged tissue but they have limited differentiations..

Induced Pluripotent Stem Cells (iPSCs): Scientists have recently discovered how to reprogram adult stem cells to act more like pluripotent, embryonic stem cells. They have the potential to produce new cells for any organ or tissue in the body and can be made from someone’s own skin, potentially preventing rejection from the immune system.

Cord Blood Stem Cells: After childbirth, stem cells can be harvested from the umbilical cord and frozen for future use. These stem cells can produce all other cells found in blood, including cells of the immune system. This makes them especially useful in treating blood diseases like Leukemia.

  1. Regenerative Medicine and Tissue Engineering Are Different

Tissue engineering is an interdisciplinary field which applies the principles of engineering and science to develop biological substitutes that restore, maintain, or improve tissue function. The key word here is develop. Tissue engineering has the capacity to build biologic materials.

Regenerative medicine doesn’t require the production or growth of biological substitutes. Rather, regenerative medicine is any therapy aimed at restoring function.

  1. Stem Cells Could be Used to Treat Everything from Heart Disease to Menopause

Because stem cells can differentiate themselves into a range of adult cells, they can potentially treat any disease or condition that causes and/or is perpetuated by the destruction of cells and tissues.

At Celixir, we’ve focused our efforts on how iMP cells (Integral Membrane Protein) can treat patients with heart disease. Around the world, scientists have found stem cells to be useful in treating Parkinson’s Disease, Diabetes, Leukemia and even menopause. Scientists are also closer than ever to finding a cure for Human Immunodeficiency Virus (HIV) because of extensive stem cell research.

  1. Stem Cells Could Regenerate Tissues, Bones and Cartilage

Cell-based bone and cartilage replacement is an evolving therapy that could help amputees, those have lost limbs in an accident, and those with autoimmune diseases that attack and destroy cartilage tissues in the body. While many animals have the ability to regenerate or replace lost or damaged appendages, humans, unfortunately, do not.

But, back in 2017, scientists from the University of New South Wales (UNSW) transplanted multipotent stem cells into areas of damaged tissue. Controlled repair of tissue seemed to be observed. Human trials are expected to begin this year.

  1. There is Controversy Surrounding Stem Cell Research and Treatment

The controversy surrounding stem cell research and treatments is of a moral nature rather than scientific. Namely, opponents believe that harvesting embryonic stem cells and using fertilized embryos is unethical as they believe first stage embryos should have the same rights as fully developed humans.

On the other side of the argument, supporters believe that embryos aren’t yet humans. Donor couples whose eggs and sperm were used to create the embryo give their consent in putting forth their embryo to be used in valuable, potentially life-changing scientific research.

Now, with iPSCs, there’s less of a need for human embryos in research which has alleviated some concern for opponents.

  1. Embryonic Stem Cells Were First Identified Less Than 40 Years Ago

Scientists only just discovered how to harvest embryonic stem cells from mice in 1981. Just 17 years later, scientists created a method to do the same with human embryos, effectively growing embryonic cells in laboratories. In 2006, a team of scientists in Japan successfully reprogrammed adult cells to create iPSCs, putting to rest some of the ethical and moral debates surrounding stem cell research. Today, clinical trials using stem cells are being approved around the world, including Celixir’s own Heartcel in Europe and the US.

  1. Stem Cells Have Been Proven to Work

As more and more trials are being approved, it’s becoming increasingly evident that stem cell therapies are effective in treating a range of diseases and conditions. While, as mentioned, there has been controversy surrounding stem cell therapies on a moral basis, the success of trials in both animals and humans suggests that stem cells do work. New, exciting research from Universities and biotech companies around the world is being published weekly. For a summary of 11 stem cell studies set to revolutionize healthcare, click here.

While you can keep up with the latest stem cell and regenerative medicine news through various medical and science journals and niche publications, Celixir often retweets relevant news on Twitter.

  1. You Can Preserve Your Child’s Stem Cells

Stem cell banks can store stem cells derived from amniotic fluid or umbilical cords for future use. It’s easy to collect, with no risk for the mother or the child, and can be used to treat over 80 diseases. Cord blood can treat both children and adults, although adults need two cord blood samples compared to just the one that children need.

Cord Blood banks now exist in every developing country and within most developing nations, with approximately 500 operating worldwide. Today, the question isn’t so much should you store the cord blood of your offspring for future use, but whether to support public vs. private cord blood banks.

  1. It Takes Time for Treatments to be Approved

Recently, regulatory bodies like the FDA have released new guidelines to ensure the delivery of safe and effective regenerative medicine advanced therapies (RMATs). As it stands, stem cell-derived products that are minimally altered and that are used for the same purpose in both donor and host do not need premarket approval. Products that do not fall under this umbrella, though, are regulated as drugs, biologics or devices. In this case, the drug must be tested on animals before the company can submit an application to the FDA. The FDA then reviews the application to assure that the proposed studies/clinical trials do not place human subjects at unreasonable risk of harm.

From there, the drug is approved for Phase 1 testing with 20-80 healthy volunteers. Phase 1 emphasizes safety. Phase 2 then involves hundreds of patients with a focus on effectiveness. Afterward, the FDA and sponsors discuss how large-scale studies in Phase 3 will begin. Phase 3 involves thousands of patients and studies different populations, dosages, and the use of the drug combined with other drugs. After another review meeting with the FDA and sponsors, the company submits an NDA, formally asking for approval for marketing in the US. The FDA has 60 days to review the application. In June of this year, the FDA approved Celixir’s Investigational New Drug application (IND) for Heartcel, an exciting and significant regulatory milestone that will allow Celixir to conduct potentially pivotal trials with Hearcel.

To keep up with stem cell and regenerative medicine news, read our blog or follow us on Facebook and Twitter @CelixirLtd

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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.

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.

New Trial Suggests Stem Cells Could Heal Patients’ Brains Post-Stroke

Thanks to researchers from the University of Georgia’s Regenerative Bioscience Center and startup ArunA Biomedical, stroke victims have a renewed sense of optimism when it comes to one day fully recovering. The new treatment, called AB126, uses stem cells to decrease brain damage and increase the brain’s natural healing tendencies. As of late-February, clinical trials have proven effective on both mice and pigs. Scientists are now looking to begin tests on humans in 2019.

The treatment will mean a second chance for hundreds of thousands of people. According to the Stroke Association, one person suffers from a stroke every two seconds and strokes are the fourth leading cause of death in the UK. Before we understand the future of treatment, we must first look to understand why strokes occur.

Ischaemic strokes – which account for 85 percent of all strokes – are caused by a blockage that cuts off blood supply to the brain. Haemorrhagic strokes – which account for the other 15 percent of all strokes – are caused by blood vessels bursting within or on the surface of the brain. In either case, because the brain is affected, the whole body bears the burden. Mobility in both arms and legs is decreased, patients will likely suffer from pain and headaches, and they will also have trouble speaking, understanding, reading, writing, and controlling their bowels.

The consequences of strokes are devastating and far-reaching, impacting nearly every aspect of day-to-day life for survivors. Currently, the best treatment for these victims is tPA (tissue plasminogen activator). This FDA approved IV works by dissolving the blood clot and improving blood flow. While – yes – it has been proven to reverse side effects, it’s only effective if administered within three hours of the stroke. Doctors estimate that only 5 percent of patients are able to make that very limited window.

Unless patients are able to seek treatment within three hours of their stroke, their options are limited to rehabilitation. Recovery depends on the severity of the stroke’s complications.

With AB216, the window is still slightly limited. Researchers are currently administering the treatment no more than 6 days after the patient suffered from the stroke. Of course, this is quite a substantial increase in time from three hours as is the case with tPA.

The study, which was published in the journal Translational Stroke Research, details how extracellular vesicle fluid filled structures called exosomes are used to decrease the amount of brain tissue lost in the injury. These exosomes – which are present in eukaryotic fluids (blood, urine, etc.) – are especially useful in that they can carry multiple doses of treatment and are small enough that they’re able to cross barriers that other cells can’t.

When tested on mice, MRI scans showed close to 35% decrease in size of injury and a 50% decrease reduction in brain tissue loss. This is the first time such results have been seen in exosome stroke treatment studies.

ArunA is already producing AB126 exosomes to meet post-trial demands, with an eye to maintain consistency while still keeping the cost of production low. As apart of their trials, researchers intend to test the effects of the new treatment on traumatic brain and spinal cord injuries as well as epilepsy.

This exciting development is the third in a series that started in 2014 in London. There, researchers from Imperial College Healthcare NHS Trust and Imperial College London, used stem cells from bone marrow in the rapid treatment of strokes. It was the first of its kind published in the UK and the results were encouraging. A particular set of CD34+ stem cells – known to help with the production of blood cells and blood vessels – was used. Four out of five patients were able to live independently six months after suffering a severe stroke that historically leaves only four percent of victims alive.

In 2016, scientists at Stanford University used mesenchymal stem cells, which can mature into multiple types of specialized cells, to restore brain function. The trial involved 18 stroke victims and after depositing the stems cells directly into their brains, one woman made a near full recovery as she regained the function in her legs and learned how to walk again.

Given the progress that has been made over the last several years, stroke victims have a lot to look forward to in 2019 when human trials begin for AB126.

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.