Stem Cell Therapy Brings Hope to Children with Autism

Autism is a condition which affects an estimated 1 in 45 people in the UK. Around 30% of autistic children will never learn to speak, and many children even with early behavioural interventions still struggle to adapt. Although early intervention and behaviour management strategies help, there are no medically approved treatments that improve the core symptoms of autism.

A recent study by Duke University in North Carolina has shown some promising results that point to the possibility of being able to treat autism using stem cells found in a child’s own cord blood.

First-of-its-Kind

The first-of-its-kind study was lead by Dr. Joanne Kurtzberg, one of the lead researchers at the Carolinas Cord Blood Bank, and Dr. Geraldine Dawson director of the Duke Center for Autism and Brain Development. After seeing successful trials using cord blood to treat children with inherited metabolic disorders and cerebral palsy, they saw a great need for further medical advances in the treatment of autism.

The study involved 25 children with autism whose parents had previously banked blood from their umbilical cord at birth.

In the first treatment each child was given an IV infusion of their own cord blood containing 1-2 billions cells. Three times over the course of a year, an evaluation of the child’s brain activity was carried out, and behavioural observations made.

Positive results

After one year, more than two thirds of children showed significant and continued improvements in behaviour as evaluated by their parents and researchers. This included throwing less tantrums, showing less volatile behaviour, and generally being calmer in every day life.

“Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech,” Kurtzberg says. “Many children were able to attend to play and have meaningful communication in a way that they weren’t before. Some children had less repetitive behaviors than they did when they came onto the study.”

Parents of one of the children, Gracie Gregory, were even able to let her go to a mainstream school, something they previously thought impossible.

Positive but not conclusive

Whilst the research is promising, any results need to be treated cautiously.

As a safety study, not a controlled, double-blind study, it cannot yield definitive proof of positive results. The study was open-label, meaning everyone – the doctors and the families – knew that the therapy was being administered. This means that positive results could be attributed to a number of other factors including a natural improvement of behaviour with age, and the parents subconsciously wanting to see and therefore magnifying any improvements.

A larger second double-blind, placebo-controlled trial is now underway which will involved 165 autistic children between 2 to 8 years old. The added placebo control element, and higher number of children involved, will allow the researchers to better assess the effectiveness of the treatment.

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US clinical study seeks to see if stem cells can cure baldness

Four American surgeons are the latest group of many worldwide who are attempting to see how stem cells could be used to combat baldness.

In the five-patient study lead by Kenneth Williams, D.O. of Orange County Hair Restoration, a clinical trial is taking place in which PRP and stem cells are being used in conjunction with each other to treat hair loss. Fat is removed from the abdomen before being emulsified to separate the stem cells. These cells are then mixed with the patient’s concentrated plasma before the mixed formula is injected into the scalp. Williams hopes the results of his study will be publishable in two years’ time. “The study is taking cells that are in our body that help to regenerate or stimulate inactive or dormant hair follicles,” he told news outlets. “That is the theory behind what we’re doing this procedure on.”

Overseas trials already showing promise

The American Hair Loss Association notes that two-thirds of men will experience some thinning by the age of 35, while by the age of 50, roughly 85 per cent will be affected by significant thinning.

It’s perhaps no wonder then that so many scientists around the world are searching for ways to utilise stem cells to cure the condition. Clinical trials in Japan for example are already making significant strides in research; Kyocera Corp and Organ Technologies are conducting regenerative medicine trials in attempts to develop a cure. Led by Takashi Tsuji, the research team has already had some success in regeneration using stem cells, using them to reinvigorate hair follicles in mice. Hormones can impact the natural cycle of hair follicle regeneration, which is powered by stem cells, as can damage caused via trauma. Taking tips from skin restoration, follicular regenerative medicine works by removing small patches of skin and hair follicles from scalp to extract active stem cells. These are then multiplied, processed, and transformed into follicles using what Tsuji has dubbed the primordium method. These transformed cells are then injected into the patient’s scalp. This process differs from current baldness cures as the hair follicles are actually regenerated, as opposed to treatments such as hair transplants, which simply move the hair from one place to another.

Given that approximately 18 million people suffer with hair loss in Japan, it’s not only Takashi Tsuji and his team who are on working on potential cures. Cosmetic giants Shiseido Co. have plans to release a cure for baldness throughout Japan and other countries in Asia as early as next year.

Leeds scientists make breakthrough with potential brain tumour treatment

A new study has revealed that targeting the RAD51 molecule could potentially help oncologists treat aggressive brain tumours.

Although the molecule helps cells to repair their DNA, the new study has found that targeting the molecule actually increased the effectiveness of radiotherapy to kill glioblastoma. The University of Leeds carried out the study with funding from Cancer Research UK, and the findings were published in Stem Cell Reports.

Glioblastomas are the most common type of brain tumour affecting adults, treatment for which typically has a low rate of success. It is estimated that less than five in every 100 patients with the disease will live beyond five years after their diagnosis.

While the treatment is some years off being suitable for use in clinical trials, these initial results are a step in the right direction, according to Dr. Justine Alford, Cancer Research UK’s senior science information officer. “This promising study in cells and mice may have found a way to cut off the tumour’s fuel supply,” she said, adding that the approach “could one day help treatments target the disease more precisely and effectively.”

What is RAD51?

RAD51 is a gene that assists with the “homologous recombination and repair of DNA,” or in other words, helps cancer cells repair following radiotherapy treatment.

Researchers believed that one of the reasons glioblastoma cells proved so hard to treat is because of the presence of this RAD51, which leads to cells being able to duplicate quickly and make copies that are highly resistant to treatment. Given that this subgroup of troublesome cells – called glioblastoma stem cells (GSCs) – contained such high levels of the RAD51, researchers used an inhibitor drug on mice that would in effect target this molecule. “By targeting RAD51 with an inhibitor we were able to make these GSCs more sensitive to the effects of radiotherapy, helping remove the tumour,” reveals Dr. Susan Short, lead author of the study. “The exact mechanism by which RAD51 becomes increased in cells that survive radiotherapy is not yet known, but our study provides strong evidence that this is the right protein to target in the treatment of this aggressive brain cancer.”

This most recent study therefore brings welcome news to the cancer research community, following one from Cambridge University last year. As reported on by Ajan Reginald, the research presented a clearer picture of how and why cancer develops, potentially taking scientists one step closer to finding a cure.

Could Stem Cells Be Used To Restore Vision?

The eye is one of the most structurally complex parts of the body. Different disorders of the eye develop when one or more of the components stop working. This is why it is so difficult to treat conditions of the eye, and reverse sight loss.

Stem cells may well hold the answer.  By replacing damaged cells with new, healthy, specialised cells, it could be possible to reverse the damage done and restore vision.

Currently the only clinically approved stem cell treatment for the eye is Holoclar®, a treatment which restores vision to patients with damaged corneas by transplanting limbal stem cells into areas where these cells are lacking.

Use in Macular Degeneration

One of the leading causes of vision loss is Macular Degeneration (MD), a painless, age-related eye condition, which causes the person to lose their central vision making vision blurry.

Stem cell therapy has been trialled to treat MD but has so far been unsuccessful due to the replacement cells struggling to integrate into the original tissue.

Researchers may have found a solution to this in the form of an injection of an  immune system protein- Mesencephalic Astrocyte-derived Neurotrophic Factor (MANF) – to assist with the integration.  Research carried out by the Buck Institute showed success in retinal stem cell transplants on congenitally blind mice, and human trials are the next step.

If the protein is successful in human therapy, it could be used to treat early-stage MD in the future.

MANF has previously been studied by other scientists, including  Ajan Rejinald, CEO of Celixir,  for its role in conditions like Parkinson’s disease

Stem Cells Restore Long-Term Vision in Mice Using Regenerative Vision Therapy

Oeil et test de visionStem cell therapies offer great potential for repairing function in a range of degenerative conditions. In a recent study, scientists restored vision in blind mice to support tackling the immune system’s function in denying transplanted cells.

 

Scientists working on the generation and transplantation of retina stem cells have seen success in one experiment – retina stem cells derived from an adult mouse.

 

Making certain that transplanted cells can survive long enough to work is one of the most common encounters in developing stem cell therapies. But now researchers have reported one of the first examples in improving longevity of functionally integrated stem cells and hampering the immune response that triggers the rejection of transplanted cells.

 

The good news? This incredible discovery holds great promise for bringing back function in a whole host of degenerative conditions, however, one of the strategic difficulties is how to make sure the cells survive in the body long enough to work.

 

By transplanting photoreceptors originating from human stem cells, researchers from the Buck Institute could demonstrate long-term vision restoration in mice by stopping the immune response that prompted patients to reject transplanted cells.

 

One of the crucial topics that needs to be tackled to enhance stem cell regeneration therapy productivity is immune system rejection, as published in the Cell Stem Cell.

 

These promising results support a means to developing scientific therapies, mainly for bringing back human vision through enabling photoreceptors emanating from human stem cells to amalgamate and develop in the eye.

 

As declared by Buck faculty and senior author Deepak Lamba, PhD, MBBS, “This turned into a nice story of long-term restoration of vision in completely blind mice. We show that these mice can now perceive light as far out as nine months following injection of these cells.”

 

Specialised neurons in the retina, photoreceptors alter light into signals that the brain translates as sight. A decline in these cells is a typical cut-off point in progressive eye diseases.

 

While human embryonic stem cells can offer a possible foundation for photoreceptor replacement, researchers hadn’t been able to demonstrate longstanding sustained vision restoration, even though Lamba’s previous work indicated that photoreceptors originating from stem cells could function in mice.

 

According to Lamba, one of the main controversies in this field is whether or not the transplanted photoreceptors merely perish or are vigorously eliminated by the immune system – the eye, together with the brain, had long been thought to be “privileged” in that the cells of the immune system didn’t monitor those locations.

 

The next step in Lamba’s research was to have the group carefully inspect the extent to which immune rejection adds to unsatisfactory results in stem cell therapies for the eye, and to ascertain if they could stumble upon a solution to the problem.

 

Supposing that rejection was happening and that it could be controlled, transplanted photoreceptors, they discussed, sprang from stem cells that may well have time to join into the visual system and start communicating information to the brain.

 

Using a specific mouse strain that was healthy but lacking in a specific immune cell receptor, the team discovered the mouse was unable to reject transplanted foreign cells. Named immunodeficient IL2 receptor gamma (IL2rl) null mice, these creatures lack the IL2ry receptor that humans also have as part of a functional immune system.

 

According to the publication’s lead author, Jie Zhu, PhD, a postdoctoral researcher who started in Lamba’s lab three years ago, “This mouse strain is a great model for this research because they are otherwise healthy and normal, including in their vision, so it allows us to conduct studies focused on cell integration.”

 

The mice used in the team’s research proved that without the rejection process, there was a 10-fold rise in living human embryonic stem cell-derived donor retinal cells that matured and integrated into the retina.

 

Having witnessed a momentous, long-term improvement and having established that transplanted cells could integrate, the next stage was to investigate if the cells actually worked.

 

So the team then transplanted the stem cell-derived photoreceptors into a different strain of mouse, known as CRX null, which is genetically blind. The team calculated the pupils’ reaction to light and observed the brains’ visual reaction centres to illustrate that signals from the eye were moving to the correct parts of the brain.

 

Even nine months to a year following photoreceptor transplantation, the team discovered that eyes were responding to light and relaying sight messages to the brain.

 

Lamba confirmed, “That finding gives us a lot of hope for patients, that we can create some sort of advantage for these stem cell therapies so it won’t be just a transient response when these cells are put in, but a sustained vision for a long time. Even though the retina is often considered to be ‘immune privileged,’ we have found that we can’t ignore cell rejection when trying to transplant stem cells into the eye.”

 

Dedicated to the scientific applications of human stem cells, Dr. Lamba’s lab has a specific interest in recovering vision that has been jeopardised by progressive eye diseases, like macular degeneration. At present, Zhu and Lamba claim they’re improving the current work. One angle is to utilise already-approved drugs to counteract rejection for organ transplant that affect the same receptor.

 

Zhu proclaims that: “Using an antibody against this specific receptor means that the immune system might not need to be suppressed more generally, which can be very toxic.”

 

Lamba maintains that: “We can also potentially identify other small molecules or recombinant proteins to reduce this interleukin 2 receptor gamma activity in the body — even eye-specific immune responses — that might reduce cell rejection.” She goes on to say that: “Of course it is not validated yet, but now that we have a target, that is the future of how we can apply this work to humans.”

Stem Cells Ease the Pain of Spinal Cord Injuries in Mice

A recent study at the University of California, San Francisco has found that neural stem cells can help alleviate the nerve pain and bladder issues caused by spinal injuries.

Patients who suffer spinal injuries often have to deal with numbness, loss of bladder control, and associated side effects of their paralysis. In part, this is due to overactive spinal cord circuits, which can cause pain and decrease quality of life.

In this particular study, a spinal injury was induced in mice, and two weeks later the stem cell treatments were tested. Instead of focusing on the site of the injury, researchers focused on areas where the spinal circuits were at their most active. These cells dispersed, and were integrated into the spinal cord. Because embryonic stem cells from humans rather than mice were used, this allowed them to see how the cells may act if the study is extended to human subjects.

By using a special paper in their cage lining, scientists were able to see where and how often the mice had urinated. After the treatment, the mice had fewer large spots, showing greater control over their bladders. They also showed signs of being in less pain, and exhibited less scratching behaviour – a chronic itch can be a side effect of spinal injuries.

Many patients with spinal injuries rely on a cocktail of drugs, from painkillers to antidepressants, as well as medicines to help them control their bladder. Unfortunately, each of these drugs comes with its own side effect, and they cannot always be relied on in the long term.

In the last few months, there have been several stories from across the world of stem cell treatments. Ajan Reginald, CEO of Celixir, compiled some of the highest profile updates in a recent blog post, and this included the story of a quadriplegic who had regained movement in his arms. While many of these stories focus on recovery from paralysis, the team at University of California, San Francisco are focused more on improving quality of life for spinal injury patients, and looking for alternatives to the usual prescription painkillers.

New Research Provides Insight into How Cancers Develop

Cancer cells - 3d RenderingA study carried out by Cancer Research UK has shown that cancers need a ‘perfect storm’ of conditions to be able to develop.

Carried out at the Cambridge Institute, this research gives a clearer picture than ever before of why and how cancers develop, and why some organs are more likely to develop the disease. This research could prove invaluable in learning how to prevent and best treat many different kinds of cancer.

The researched focused on the role of stem cells, which replicate to repair damage, or create other cells that the body needs. Certain stem cells can end up with random mistakes in their DNA, or certain environmental factors can increase the likelihood of these mistakes. This includes things like smoking, drinking, and obesity – all things that we’ve long know increase the risk of cancer.

When damaged stem cells are ‘sleeping’, no cancer develops, so the stem cells with DNA mistakes aren’t able to cause cancer alone. The problem begins when these cells with DNA mistakes start to replicate, to repair some sort of damage or wear and tear. The ‘faulty’ stem cells then develop into a cancer.

For a patient to develop a cancer, there has to be a ‘perfect storm’ of factors at play. There has to be something in the body that needs to be healed, plus the stem cells with DNA mistakes to begin replicating. That’s why certain areas where the stem cells are most active, such as the colon, are common sites for cancer.

One scientific debate that this study aims to resolve is whether cancer is just down to bad luck, or whether environmental and lifestyle factors have a greater proportion of blame. The study showed that cancer requires three separate things in order to grow; tissue damage, stem cell DNA mutations, and the activation of these mutated stem cells.

Some other findings in the study included the fact that DNA mistakes in stem cells build up as you get older, which accounts for the risk of cancer being higher as you age.

To carry out the study, researchers used mice that had modified cells which produced a fluorescent green protein when ‘switched on’. This allowed them to track what happened to the cells in various organs and at different stages of their lives. For example, when the mice had damaged livers, researchers were able to see the cells divide rapidly and tumours formed.

By being able to replicate how cancers are formed, this could open up the potential of cancer research, and mean that preventative medicines and new treatments could be coming to the market.