Ajan Reginald: The regenerative medicine entrepreneur treating heart disease with stem cells

Cardiovascular disease (CVD),  in all of its forms, is the number one killer in the UK, Europe and the US. More often than not, it has long-term effects, including an enlarged, damaged and less efficient heart muscle which naturally leads to other disabilities and a notably decreased quality of life.

While the affected person certainly bears the burden of such diseases, society as a whole does as well. In a 2006 study, researchers found that CVD cost the UK economy £29.1 billion in 2004, with healthcare costs accounting for 60% of the total. This number is even higher in the US, with healthcare costs associated with CVD just under $300 billion per year.

It’s worth mentioning that CVD encompasses all diseases and conditions that affect the heart or blood vessels. But the focus here is on coronary heart disease, which often results in heart attacks and heart failure.

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How to Pursue a Career in Regenerative Medicine

If you were to take a look at the educational and professional backgrounds of those apart of Celixir’s leadership – including its founders, board of directors and scientific advisory committee – you might be surprised by the variety you’d find.

Professor Sir Martin Evans, Celixir’s President, Chief Scientific Officer and co-founder, attended Christ’s College (a constituent college of the University of Cambridge) where he studied zoology, botany, chemistry and biochemistry. After graduating with a BA, he moved to University College London where he worked as a research assistant and graduated with a PhD. Not only did Sir Martin Evans isolate the first embryonic stem cells, he’s also published over 120 scientific papers and is the recipient of a Nobel Prize.

This, compared with Celixir’s Chief Executive Officer and other co-founder, Ajan Reginald, whose background is in both science and business. Ajan holds four degrees and is an alumni of Harvard Business School, University of Oxford, Kellogg Business School and University of London. After serving as the Global Head of Emerging Technologies for Roche Group Research, he moved on to the Business Development Director at Roche Pharma. Since, he’s helped develop Celixir’s breakthrough technologies including Heartcel and Tendoncel.

Of course, this is all to say that the path towards a career in regenerative medicine is not necessarily a linear one.

The Field is Growing

While – compared to other fields – regenerative medicine is in its infancy, technological advances, ever-increasing funding and high demand have made it grow quickly.

What’s especially exciting, though, isn’t that regenerative medicine is a growing market. Instead, it’s the virtually limitless possibilities in terms of what scientists and researchers can achieve with their work. As the world’s population ages and diseases like Alzheimer’s and Parkinson’s remain untreatable, more and more people are looking towards this multidisciplinary field for answers to a number of medical questions.

Desired Interests, Skills and Expertise

Regenerative medicine attracts people across several different fields including engineering, business, molecular science, healthcare and even robotics. And, because regenerative medicine is focused so heavily on applying current (and creating future) technology to improve quality of life for patients, it’s an especially attractive field for those interested in actually making a difference.

Ajan Reginald CelixirLuckily, and as demonstrated by Ajan Reginald‘s and Sir Martin Evans Celixir backgrounds, the skills learned in all of the fields mentioned above (engineering, business, molecular science, healthcare, robotics) can be applied to regenerative medicine. This is to say that functional expertise and not necessarily general knowledge about regenerative medicine is key. Imaging specialists, immunologists, bioengineers and transplant surgeons are all contributing to the advances being made, even if they don’t consider themselves specialists in regenerative medicine.

But, there are specific programs available at universities all over the world that act as a ‘direct’ gateway to regenerative medicine. That is to say that they focus on a specific segment of regenerative medicine, like tissue engineering.

Breaking Into the Industry

As mentioned, there is no linear path to break into regenerative medicine. But, there are a number of resources that can help.

If you’re a student, professors or other mentors within your university could provide networking opportunities. Alternatively, you could network at jobs fairs or other conferences like International Society for Stem Cell Research or World Congress on Regenerative Medicine. You can also apply to companies or universities directly.

Bear in mind that a career in regenerative can take a number of forms, from a research associate to lecturer to a project manager.

If you’re interested in stem cell research and regenerative medicine, keep up with the latest news on Celixir’s blog or follow Celixir on Twitter or Facebook.

The Potential Impact of AI on Cell Therapy

Just under two years ago, Ajan Reginald, the founder, CEO and thought leader behind Celixir, sat down with IntelligentHQ.com to talk about current challenges and opportunities within the healthcare industry. While Celixir’s own Heartcel was of course discussed as a cutting edge innovation in life-saving, life-altering medicine, artificial intelligence (AI) and its role in healthcare was also debated.

Ajan reginald celixirAt the time of this 40-minute interview, Ajan Reginald was especially excited about AI in that robots could potentially edit genes and inject cells with more precision than humans. For Ajan, breakthroughs in medicine come from increased patient benefit and he certainly saw the many benefits automation would offer. When you consider that the third leading cause of patient death is hospital error, it’s hard to disagree.

But, despite the obvious benefits, there were still a number of hurdles that had to be overcome, specifically in terms of data privacy and encryption. Scientists and researchers would have to work hard to develop technology that would ensure that the right patient got the right medicine in the right dose at the right time.

The question is, have the necessary advancements been made over the last 21 months?

On Our Way to AI-Dependent Healthcare

While healthcare providers and tech companies have been investing billions into testing AI-powered tools, the scientific and medical communities are still struggling to find solutions to data and privacy concerns. But, that doesn’t mean there haven’t been significant advancements with massive potential in terms of robotic surgery and image analysis.


AI-Assisted Surgery

Not only does AI-assisted surgery have an estimated value of $40 billion according to a report from Accenture, but, given robots ability to analyse data from pre-op medical records, they could be used to guide surgeons’ instruments during surgery, leading to a 21% reduction in patients’ hospital stays. This is clearly beneficial for the patient. Of course, less time spent in the hospital equates to reduced costs for insurance companies and hospitals, too.

AI-assisted surgery has also been proven to be more effective, with five times fewer complications compared to surgeons operating alone according to a study published in The Spine Journal.

Today, robots are being used in both eye surgeries and heart surgeries. The Da Vinci, the most advanced surgical robot, successfully operated on a human eye seven months ago. As Ajan predicted, the robot was able to perform complex procedures with greater control than conventional approaches.  

Image Analysis

Up until June of 2018, image analysis was incredibly time consuming. That’s because humans were doing the analysing and it could take two hours or more to see a change in 3D medical scans.

Now, thanks to an MIT-led research team, there is a machine-learning algorithm that can analyse 3D scans up to 1,000 times faster than was previously possible. The changes are essentially studied in real time, allowing surgeons to react more quickly during operations.

AI is also expected to help improve the next generation of radiology tools. Instead of collecting tissue samples through biopsies which has the potential to cause an infection in the patient, the AI-powered tool would show images with very close registration. Currently, these tools are just being piloted.

The Future of AI in Healthcare

AI in healthcare is in its infancy. But, it’s clear that AI tools and systems can help treat patients faster and with more precision than is possible for humans. While we’re still waiting to utilise robots in providing cell therapies, we’re sure to see advancements in how AI processes and interprets data to make the application and administration of medicine easier.

For more stem cell and regenerative medicine news, keep up with Celixir’s blog.

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Hematopoietic Stem Cells: What Are They and What is Their Function?

Most of us understand the importance of blood cells. Red blood cells carry oxygen throughout the body while white blood cells fight infection and help us develop immunity to diseases. But, what about the stem cells that turn into our blood cells?

Scientists, researchers, and doctors have been studying hematopoietic stem cells (HSCs) – the stem cells that form blood and immune cells – for over 60 years, starting after the bombings of Hiroshima and Nagasaki in 1945. They’re now routinely used to treat patients with cancer after chemotherapy.

What Is A Hematopoietic Stem Cell?

Celixir Stem CellsThe hematopoietic system – the system responsible for the production of the bodies’ cellular components – relies on the presence of HSCs.

In fact, HSCs are the only source for the continued production of red blood cells, platelets, white blood cells, and all other cells in the system. When you consider the fact that the average human requires around 100 billion new hematopoietic cells each day, you realise how vital the role of HSCs is in each of our bodies.

Identifiable Traits of Hematopoietic Stem Cells

Because HSCs behave like normal white blood cells, scientists have spent a considerable amount of time identifying key properties and characteristics of the stem cells.

Studies on mice laid the groundwork for our current understanding, and we now know that a HSC has four important properties: it can renew itself, it can differentiate to a variety of other specialised cells, it can mobilise out of bone marrow into circulating blood and it can undergo programmed cell death, called apoptosis.

We also know that there are several different sources of HSCs, including bone marrow, peripheral blood, umbilical cord blood, fetal hematopoietic system and embryonic stem cells and germ cells.

Bone marrow has been used as a source of HSCs for over 40 years  But, peripheral blood is now the preferred source for medical treatments and, as umbilical cord blood banks are receiving more and more support around the world, umbilical cord blood is being considered a more viable option for patients as well. The final two sources – the fetal hematopoietic system and embryonic stem cells – are used for clinical purposes only.

Clinical Uses and Current Applications

Today, tens of thousands of transplants are performed annually around the world.

Medically, HSCs are used to treat patients with acute myeloid leukemia, chronic myeloid leukemia, acute lymphatic leukemia, aplastic anemia, and other primary immune deficiencies and metabolic diseases. In treating cancer patients, HSCs are transplanted after chemo- or irridation therapy to regenerate the hematopoietic system. In most cases, this is achieved in just 2-4 weeks.

The Future

Current clinical trials are looking at gene therapy, vehicles for gene delivery and other gene-editing strategies. There are several promising HSC gene therapies in the early phases of clinical trials, including treatments and products for sickle cell disease , X-linked forms of SCID, and Wiskott-Alrich Syndrome.

A clinical trial  that’s sponsored by the National Heart, Lung and Blood Institute in Maryland is currently recruiting pregnant women to examine the best ways to collect, process and store umbilical cord blood. For babies born with sickle cell disease, the blood collected from the cord and placenta will be stored indefinitely for use in gene therapy treatments later in life. For those babies born without sickle cell disease, the cord blood will be stored for up to 3 years and may possibly be used to treat living or future siblings who have, or may be born, with the disease.

In an attempt to treat both SCID and Wisckott-Alrich Syndrome, lentiviral genes are being used as vectors in Phase I/II trials. So far, there’s been with initial success in treating SCID in both older and younger patients. Likewise, in treating Wiskott-Alrich Syndrome, lentiviral therapy has proven both safe and effective.

Like all regenerative therapies, there are considerable obstacles to overcome and it takes time to research, develop, test, and regulate new products and methods. Nonetheless, over the last five decades, we’ve seen incredible advancements in HSC therapies that have helped doctors and patients treat and even cure several different disorders.

The hope, of course, is that successful clinical trials will lead to approvals by regulatory agencies, as was the case recently for Celixir’s own IND for Heartcl.  Eventually, researchers hope that these therapies will be adopted on a large scale by healthcare systems.

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Stem Cell Research Around the World

Around the world, different countries – and entire continents – are working separately but collectively to develop innovative and life-changing treatments through stem cell research. The field is growing quickly as clinical trials prove that stem cell therapies have the potential to treat or reduce symptoms of diseases, conditions and disabilities that have, up until this point, been untreatable.


In China, stem cells from baby teeth are being used to regrow damaged dental tissue. In Canada, neural stem cells are being used to restore motor impairments caused by cerebral palsy. But, it’s important to recognise that there isn’t one set of laws that govern the globe and some countries are extremely limited by the laws and policies that are in place.

Here’s a look at stem cell research around the world.


As a continent, Africa is lagging behind others in terms of research and clinical trials related to stem cells with 80% of Mesenchymal stem cell clinical trials occurring in Asia, Europe and North America and just 2.5% occurring in Africa. But, given legislative issues and religious beliefs, this isn’t surprising.

In South Africa, regulatory legislation has existed since 2013 and is mostly concerned with the use of embryo derived stem cells, induced pluripotent stem cells and adult stem cells. But, the legislation isn’t strictly implemented and is often completely overlooked, especially for medical scientists involved in stem cell tourism.

In Egypt, experimental stem cell research (including cloning) is generally accepted whereas, in Tunisia, it’s prohibited, along with research of embryonic stem cells. But Tunisia is the exception, not the rule. In most other African Arab countries, there isn’t clear legislation that controls stem cell research, and governing bodies rely on established religious and ethical beliefs. Without concrete rules and government support, the growth of stem cell research is stifled, and Africa as a whole isn’t able to contribute as much to the field.

Nonetheless, scientists and researchers are still working together at workshops like the one held in Stellenbosch, South Africa. Here, attendees discussed initiatives for developing stem cell therapies for diseases like cancer, diabetes and other non-communicable diseases that have overtaken HIV as the major cause of death in Africa


Stem cell research in the second largest country in Asia – China – has progressed significantly since the official policy on stem cells was announced in 2015. In fact, stem cell research has become a focal point for the government’s plan for developing the life sciences and biomedical sectors and there’s been an extraordinary amount of money put forth to equip laborites with the latest technology. It’s made an impact. If you look at international publication trends, between 2006 and 2010, China’s stem cell research output jumped from 176 articles to 677.

Elsewhere in Asia, the Indiana Council of Medical Research (ICMR) recently issued a revised draft of the National Guidelines for Stem Cell Research after noting the need for rigorous clinical trials and regulatory processes in developing safe and effective stem cell therapies. As a result, research on in-vitro cultures of intact human embryos beyond 14 days of fertilisation and research on xenogenic cells, xenogenic-human hybrids, modified human embryos, and germ-line stem cells have been banned.

Countries like Japan and Singapore are both seen as leaders in stem cell therapies and, though they might not have the outputs of China – are internationally recognized for the work they continue to do in the field.


Spain, Italy, Germany and – of course – the United Kingdom are all contributing to stem cell research in a big way. After scientists successfully cloned a sheep in Scotland back in 1996, Britain has been Europe’s leader.  

Nonetheless, Italy and Germany both have some of Europe’s most restrictive legislation regarding stem cell research. Those two countries aren’t alone. Croatia, Lithuania, and Slovakia also have very restrictive policies. The rest of Europe is (for the most part) quite permissive, with Belgium, Sweden and the UK having the least restrictive policies and Austria, Ireland, Luxembourg and Poland having no legislation about hESCs at all.

North America

As the global stem cell market grows, with an expected value of $270.5 Billion by 2025, North America’s market share also grows and is expected to be worth $167.33 by 2025. And, with 33 of the 50 people listed in ‘Most Influential People on Stem Cells Today’ being from North America, it’s clear that the United States and Canada are very involved in research for the stem cell therapeutics market.

There have never been any federal laws in the United States that have banned stem cell research, but there have been restrictions on funding and use. Although, certain restrictions were removed in 2009. In removing the restrictions, Former President Barack Obama had some foresight when he said ‘We will lift the ban on federal funding for promising embryonic stem cell research. We will vigorously support scientists who pursue this research. And we will aim for America to lead the world in the discoveries it one day may yield.’

Nonetheless, stem cell research is still a politically charged issue in the United States and different states have different bans and restrictions.

Canada is contributing as well and, because of a $4 million investment by Canada’s Stem Cell Network in April of 2018, 24 projects involving stem cell research have been planned, involving 95 scientists across the country.

While the field isn’t growing at the same pace around the world, it’s clear that there is a global trend toward more appropriate restrictions and increased funding for stem cell research. For scientists and researchers and patients struggling with diseases ranging from cancer to diabetes, the progress is promising. It’s a global effort and, together, we have the potential to treat and even cure some of the most widespread diseases.

Celixir Stem Cells

How Regenerative Medicine is Helping Treat Cardiovascular Disease

The Impact of Cardiovascular Disease

Cardiovascular disease (CVD) – in all of its forms – is the number one killer in the UK, Europe and the US. More often than not, it has long-term effects, including an enlarged, damaged and less efficient heart muscle which naturally leads to other disabilities and a notably decreased quality of life.

While the affected person certainly bears the burden of such diseases, society as a whole does as well. In a 2006 study, researchers found that CVD cost the UK economy £29.1 billion in 2004, with healthcare costs accounting for 60% of the total. This number is even higher in the US, with healthcare costs associated with CVD just under $300 billion per year.

Current Methods for Treating Cardiovascular Disease

It’s worth mentioning that CVD encompasses all diseases and conditions that affect the heart or blood vessels. Here, we’ll focus on coronary heart disease, which often results in heart attacks and heart failure.

Coronary Heart Disease

According to the NHS, patients suffering from coronary heart disease can be prescribed a combination of medications that help to reduce blood pressure, lower cholesterol or widen the arteries or blood vessels. Unfortunately, many medications have negative side effects and, given that they must be taken long-term, can be costly. Blocked arteries could require interventional procedures, including bypass grafts, angioplasty and even transplants. Success rates depend on numerous factors, including age and lifestyle and, in the case of transplants, there’s understandably a higher demand than there is supply.

What About Prevention?

Currently, prevention of CVD is directed at lifestyle changes. The Mayo Clinic recommends a healthy diet, exercise and stress management for heart health. Of course, their number one recommendation is to stop smoking. But what about medical prevention? It’s clear that there’s a need for preventative methods that will limit ischemic injury and regenerate tissue that’s been damaged before the patient suffers a heart attack, heart failure, or another life-threatening condition.

Regenerative Medicine and the Future of Treatment

Scientists around the world are working tirelessly to turn research into effective treatments and, in the past decade, we’ve witnessed a surge of scientific enthusiasm for regenerative medicine. And it’s well and truly a group effort as it requires scientists and clinicians with different expertise, from cardiology to cell biology to engineering.

California’s Stem Cell Agency  have awarded over $202 million to researchers looking into heart disease, in particular how to create stem cells that can replace the damaged heart muscle and restore the heart’s ability to efficiently pump blood around the body. Other researchers are focusing more on tissue engineering technologies by building artificial scaffolds in the lab, loading them with stem cells and placing them in the heart with the goal of stimulating the recovery of the muscle.

In terms of prevention, Cardiology News reported Dr. Andre Terzic of The Mayo Clinic believes that regenerative medicine will protect against chronic disease and help match healthspan with life span in aging patients.

Of course, Celixir is developing its own life-saving therapies. Heartcel,  an immunomodulatory progenitor (iMP) cell therapy for the treatment of adult heart failure, has been approved for clinical trials in both the UK and the US. EU Phase II trials were completed back in 2016 with overwhelmingly positive results. Most notably, 100% of patients were free from any major adverse cardiac event (MACE), 30% of patients experienced improved heart function and 50% of patients experienced improvements in their quality of life.

Over the next several years, we should see more and more regenerative therapies leaving the research pipeline to be used in clinical environments and, in time, we can hope that deaths and healthcare costs associated with CVD will decline thanks to new treatments.


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

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.