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.

FDA Approves Mayo Clinic’s Automated Bioreactor

The Food and Drug Administration (FDA) has approved a new platform developed by the Mayo Clinic’s Center for Regenerative Medicine that can mass produce stem cells in a way never done before. This is one of the first automated methods approved in the United States. Before this momentous approval, patient’s had to wait sometimes months for medical treatment that involved the creation of multiple stem cells. Now, stem cells are being manufactured in the billions in just a few days.

To understand the far-reaching effects of the FDA’s approval of the Mayo Clinic’s automated bioreactor, we must first look to understand the history of stem cell research and production.

Stem cells are – essentially – shape-shifters. They can develop and differentiate into other cells and repair and regenerate damaged tissue. Because of this, scientists and researchers are looking to stem cells to help treat a variety of conditions, from Parkinson’s and Alzheimer’s diseases, to spinal cord injuries, to Diabetes.

For a long time, the production and harvesting of stems cells has been a very labor-intensive process. Before the automated system, hundreds of hours of around-the-clock work over the course of several months only equated to the cultivation of enough cells for a few patients.

But it’s not just the speed of production that’s revolutionary. Before the Mayo Clinic’s automated bioreactor – which took over four years to develop – scientists needed stem cells from the patients themselves. Now, stem cells from other healthy individuals can be used in treating ailing patients.

“This may make treatments possible in cases where the patient’s own cells are not viable as therapy,” said Abba Zubair, M.D., Ph.D., medical director of Transfusion Medicine and the Human Cell Therapy Laboratory on the Florida campus. “In addition, because the cells can be produced in days instead of months, it may also make treatments available on short notice when they’re needed for acute care.”

The Mayo Clinic – a non-profit organization out of Jacksonville, Florida – was founded in 1889 and since then has been dedicated to finding solutions to transform medicine and surgery. With the automated bioreactor, they’ve succeeded in doing just that. So far, the scope of possibilities for stem cell research is limitless as it’s not just current patients that could benefit from the recent development. Given that stem cells can now be produced in the billions, they can rigorously test other possible treatments using stem cells.

“Although Mayo Clinic has been poised to scale up regenerative clinical trials, to date we did not have the capacity to support them. With this new technology, we now can develop phase II trials enrolling larger numbers of patients to fully test the efficacy of cell-based therapies, ” said Zubair.

They plan to use this new stem cell platform to advance therapies in degenerative diseases that, as of yet, have no cure.

Stems cells have already been proven to be vital in repairing tissue, skin, and bone. With the new, more efficient technology, the Mayo Clinic is looking to study and treat diseases like Arthritis that currently affects over 350 million people worldwide.

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.

Enhanced Culture System Allows Scientists to Quickly Derive Embryonic Stem Cells From Cows

Ever since embryonic stem (ES) cells were derived from mice in 1981, the scientific community has been looking to do the same with bovine ES cells. Now, 37 years after the cells were cultured from mice and 20 years after the cells were cultured from humans, they’ve finally captured and sustained the cells in their primitive state from a cow. In a study published in the journal Proceedings of the National Academy of Sciences, scientists at the University of California, Davis, detail how they were were able to enhance culture systems and derive stem cells with almost complete accuracy in just 3-4 weeks, a relatively quick turnaround time.

Access to these cells – which are able to develop into more than one mature cell or tissue type from muscle to bone to skin – could mean healthier, more productive livestock and could also give scientists and researchers an opportunity to model human diseases.the

ES cells are easily shaped and moulded and have a potentially unlimited capacity for self-renewal. This means that they’re extremely valuable in regenerative medicine and tissue replacement. In livestock and cattle, they offer the potential to create a sort of Super Cow that produces more milk and better meat, emits less methane, has more muscle, that adapts more easily to a warmer climate, and that is more resistant to diseases.

“In two and a half years, you could have a cow that would have taken you about 25 years to achieve. It will be like the cow of the future. It’s why we’re so excited about this,” author of the study Pablo Ross, an associate professor in the Department of Animal Science at UC Davis’ College of Agricultural and Environmental Sciences, told Science Magazine.

In order to enhance culture systems to sustain the ES cells, scientists at the Salk Institute in San Diego, California, had to expose ES cells to a new culture medium, a substance (sometimes a solid, sometimes a liquid, and sometimes a semi-solid) that’s designed to support the growth of microorganisms and cells. In this case, scientists used a protein to encourage cell growth and another molecule that hinders cells from separating or evolving.

“They used an accelerator and a brake at the same time,” George Seidel, a cattle rancher and a reproductive physiologist at Colorado State University in Fort Collins, told Science Daily.

In order for the enhanced culture systems to eventually lead to genetically superior cows, scientists will first have to augment these ES cells into the cattle’s gametes, or sperm and egg cells. The result would be endless genetic combinations, a sort of controlled evolution and accelerated natural selection. Of course, given that the evolution is taking place in a lab, each ‘generation’ would progress without any animals actually being born.

Ross maintains that “It could accelerate genetic progress by orders of magnitude”.

But it’s not just farmers and consumers that could benefit. The cows’ cells could help create larger models for studying human disease, something that mice simply couldn’t aid in due to their size. The science has also proved effective in deriving and sustaining cells from sheep. On scientists’ radars now: dogs.

Researchers find new way to kill cancer stem cells

For the scientific community, looking for ways to combat cancer continues to be a challenge, albeit one that has experienced a number of breakthroughs in recent years and even months.

cells

Researchers in Penn State recently found that grape-based compounds can kill colon cancer stem cells, after conducting petri-dish trials and trials on mine. In Salford, Manchester, researchers found that a combination of vitamin C and antibiotics can knock out cancer stem cells. Now just last week, researchers in Canada’s McMaster University have identified a unique feature of cancer stem cells, which could potentially play a vital role in the development of more targeted cancer treatments.

How existing drugs can kill deadly cancer stem cells

In a study published in Cell Chemical Biology, research reveals that an existing series of drugs has proved effective in killing off cancer stem cells.

It is thought that these stem cells play in part in the recurrence of cancer following treatment, and so using these drugs may be able to help patients stay cancer free. These drugs are thought to be able to attack these cancer cells thanks to the presence of a protein called Sam68. According to Mick Bhatia, the study’s principal investigator and scientific director of the McMaster Stem Cell and Cancer Research Institute, the findings are helping the team uncover how stem cells function in cancerous human tumours. “The drugs helped us to understand the biology,” he writes. “We’ve worked backwards, employing a series of drugs used in the clinic to understand a new way that cancer stem cells can be killed.”

It is the hope of Bhatia that this breakthrough will enable those being treated for cancer to receive more targeted, relevant therapy. While patients undergoing treatment for breast cancer currently receive targeted treatments depending on the type of disease, therapies for cancers for example do not. “In the case of breast cancer, other researchers have found new ways to make existing drugs more effective by only giving them to people who were likely to benefit based on their specific traits and using drugs that target these traits,” said Bhatia.

Stem cells help recovery from prostate surgery

It is relatively common for men recovering from prostate surgery to experience erectile dysfunction during recovery. Research shows that up to 80% of men have difficulty having sex in the months following the operation.

erectile

A clinical trial is pointing to the possibility of stem cells being used to help treat erectile dysfunction in these cases.

In the first-phase of clinical trials, eight out of 15 men who were unable to have an erection after their prostate surgery, had sex six months after one-time treatment of stem cells.

The procedure involves removing fat cells from a patient’s abdomen via liposuction. After a specialised treatment, these are transformed into all-purpose stem cells.

The stem cells are then injected into the penis, where they begin to change in to nerve and muscle cells, as well as the endothelial cells that line blood vessels.

The 12 month follow up showed that the success of the treatment was ongoing.

 “As far as we know, this is the first time that a human study with a 12-month follow up shows that the treatment is lasting and safe,” said Lars Lund, a professor at Odense University hospital in Denmark.

 “That is much better than taking a pill every time you want to have intercourse,” he said.

The study has been so successful, that the next stage, a double- blind randomised trial has been approved. This study will include a placebo group

Only men recovering from prostate cancer and able to control their bladders will be enrolled in the new experiments, Lund explained.