Where We Are With Stem Cell Research

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Our bodies are made up of different types of cells, but out of all of them, stem cells are the most remarkable. Stem cells are distinguishable from all other types of cells as they have no specific speciality – they are capable of becoming anything, regardless of their source.

Stem cells can proliferate through cell division, with no limit, and with every new cell created, each cell has the ability to either become a specialised cell – from a red blood cell to a brain cell to an organ or bone cell, and everything in between, or remain an unspecialised stem cell.

Which makes stem cells incredibly unique and vital for all living organisms. In embryos, stem cells are the foundations from which the entire body is created, and in adults, stem cells replace lost, injured or diseased cells.

And it’s because of their ability to create life and to auto-renew that means stem cells have the potential to treat seemingly incurable diseases such as diabetes and cardiovascular disease.

Stem cells could be the difference between someone with a spinal cord injury spending the rest of their life in a wheelchair, or walking again. In short, stem cells have the potential to give life and hope where it seemed there was none.
Different types of stem cells
Embryonic stem cells – these are stem cells, as the name suggests, that come from embryos. Most embryonic stem cells are sourced from fertilised eggs that have been created through in vitro fertilisation (IVF). These eggs have been donated for research.

Non-embryonic (adult) stem cells – these stem cells are found in the body, in all tissues and cells, and can renew themselves to become any cell in that tissue or organ. The role of adult stem cells is to repair and maintain the tissue in which they’re found.

Induced pluripotent stem cells – as the name suggests, these are adult stem cells that have been reprogrammed to become like embryonic stem cells i.e. capable of becoming any type of cell. It’s not yet known what significant clinical differences exist between induced pluripotent stem cells and embryonic stem cells.

Cord blood stem cells – these stem cells are taken from the discarded cord of a newborn baby. Collecting cord blood stem cells poses no risk to the mother or the baby as the cord is harvested for these stem cells once it is no longer needed by the baby. Cord blood is important because it contains haematopoietic stem cells which are incredibly rare and normally found in bone marrow. These cells can make any type of blood cell – red, white and platelets. They’re the cells responsible for our blood production throughout the course of our life.

Amniotic fluid stem cells – stem cells derived from amniotic fluid may not be pluripotent, but there is increasing evidence that stem cells found in amniotic fluid are incredibly valuable, capable of being used in cell therapy and for regenerative medicine.
Why research stem cells
Stem cell research is vital if we hope to find out more about seemingly incurable diseases such as Rheumatoid Arthritis, Huntington’s Disease or HIV, or discover a cure.

By observing stem cells throughout their life cycle, developing from stem cells into the final body part, researchers can learn how certain ailments and conditions develop.

As well as observing the development of stem cells, scientists can grow healthy cells in a lab and use them to replace damaged or diseased ones.

Researchers can even test new drugs on these lab grown stem cells to check for their safety and efficacy, before moving onto human drug trials. In particular, new areas of study have involved looking at the effects of drugs on induced pluripotent stem cells.
3 conditions that can be treated with stem cell therapies
Spinal Cord Injuries
Spinal cord injuries are damage to the spine causing temporary or permanent reduction in its ability to function. Your spinal cord consists of 31 pairs of nerves that travel from your brain down your back, feeding different parts of the body, enabling the brain to give instructions to body parts, from your fingers to your toes, even your heart, bowel and lung function are all controlled by the spinal cord.

Spinal cord injuries can be caused by falls, diseases such as spina bifida, car accidents, sports injuries and much more. Spinal cord injuries can include a loss of muscle usage, being able to feel physical sensations or autonomic function. These symptoms occur below the level of the injury to the spinal cord and are either complete (i.e. complete loss of sensation) or incomplete (partial loss of sensation, with some function remaining).

At present, there is no way to undo damage that has occured to the spinal cord – the spinal cord can not repair itself. Although plenty of work and research is being carried out looking at nerve cell regeneration that aims to improve nerve function following a spinal cord injury. Current treatment involves preventing the condition from worsening.

In January 2019, Japan approved stem cell treatment for spinal cord injuries, which sees mesenchymal stem cells, extracted from the patients’ own bone marrow, injected intravenously, helping them regain some of their lost sensations and even movement.

A stem cell trial that is currently underway is a phase 1 clinical trial researching treatment for trauma induced spinal cord injuries. The FDA has already approved phases 2A and 2B for this trial, which will see randomise control crossover trials.

In the current trial, participants will receive intrathecal injections of adipose derived mesenchymal stem cells (cells that can be used to reduce the likelihood of cell death caused by the body’s inflammatory response to injury). If successful, the treatment would drastically improve patients’ lives, negating the need for them to undergo further surgery or require permanently implantable devices.

While it’s known that stem cell treatment can be incredibly effective in treating spinal cord injuries, it’s the how, when and why stem cell treatment works in spinal cord injuries that needs further investigation.

Type-1 Diabetes

Approximately 10% of diabetes sufferers worldwide have type-1 diabetes. This equates to roughly 42.5 million people living with the disease globally. In the UK, type-1 diabetes sufferers number 400,000, with just under 10% of those being children.

For some sufferers of type-1 diabetes, they can manage the disease through lifestyle changes, but for most, they require daily insulin injections to keep their blood glucose levels in check. Type-1 diabetes, unlike type-2, can’t be prevented. And worst of all, it can occur at any age.

Not knowing what the root cause of type-1 diabetes was has plagued scientists for years, but now they understand that the destruction of beta islet cells inside the pancreas causes the disease.

And without knowing exactly what causes type-1 diabetes, has prevented scientists from finding a cure.

At present, patients with type-1 diabetes have to take immunosuppressant drugs to prevent the body from attacking the beta cells and simply replacing diseased islet cells with healthy ones (islet cell transplant) isn’t a cure. If left unchecked, diabetes can cause kidney failure, blindness, even heart disease and death.

Stem cell research has opened up a range of diabetes specific research including growing insulin producing beta cells in the lab, enabling better availability of the beta cells to be used in future research.

There is plenty of research trying to uncover a cure for diabetes currently, including encapsulation research, immunotherapy research, and regeneration research. There have even been successful experiments in mice showing that induced pluripotent stem derived beta cells can reverse the damage caused by diabetes.

So who knows, maybe a cure for diabetes is just round the corner.
Heart Disease
With the recent announcement that eminent cardiac physician scientist Professor Bernard Gersh has been appointed to Celixir’s Scientific Advisory Board, heart disease needs to be included on the list of conditions that could be treated with stem cells.

In the summer of 2019, researchers in Cambridge announced that they could treat heart failure with stem cells. This news was revelatory as 25 million people around the world are living with heart failure and cardiovascular disease is the leading cause of death globally, accounting for over 17 million lives lost yearly.

At present there are a number of treatments available to manage heart disease such as medications including Beta blockers and ACE inhibitors, but most cases eventually require surgical treatments such as coronary artery bypass, valve repair or even a pacemaker being fitted. And while these treatments can help manage heart failure, they can’t stem the progression of the disease, nor address its root cause, which is the loss of functioning heart muscle cells. Once these cells are damaged or destroyed, they can’t be replaced.

Celixir has developed its own cardiac regenerative medicine, Heartcel™, to treat moderate to severe heart failure. Adult heart failure trials are being undertaken at Imperial College London’s Royal Brompton currently, and are due to be completed in 2020.
Celixir – research into regenerative medicine
We’ve already touched on one such area of research that Celixir is working on currently, Heartcel™, but this isn’t their only avenue of stem cell research.

In just over 10 years, the team at Celixir have developed over 20 tissue specific regenerative medicines, focussing in particular on stem cell therapies and gene based therapies. Potentially finding the cure for incurable diseases, or better the existing management of untreatable diseases.

Their ongoing innovative work is currently looking into developing potential cancer gene therapies, Tendoncel – a platelet lysate-based therapy for tendon injuries, and Myocardion – a progenitor cells of mesodermal lineage (PML) therapy for mild to moderate heart failure.

Celixir is a member of the Bio Industry Association

See also

Ajan Reginald with Nobel Laureate Sir Martin Evans in Celixir

Celixir announces US FDA approval of the IND application for cell therapy Heartcel

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