In May 2016, doctors at Burjeel Hospital in Abu Dhabi treated two patients with a pioneering new therapy. The treatment, known as autologous stem cell therapy, involves extracting stem cells from the patient’s bone marrow and then injecting those cells back into the body. It has been touted as holding hope for a wide array of conditions.
The Burjeel patients had progressive amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that leads to increasing muscular weakness and eventual death. The disease is regarded as incurable, with most sufferers dying within five years of diagnosis. This means any improvement – or even a temporary stalling of the patient’s decline – can be classed as a major success.
“One of the patients had this procedure performed before, in Vienna, and proceeded here for two other sessions,” says Dr Norbert W Dreier, oncology and haematology consultant at Burjeel Hospital. “She has been clinically stable during this time. She is able to communicate with her family and is mobilised with a wheelchair. Of course, we are not expecting a cure for such a patient, but years of stabilisation are already a success and were not expected.”
It’s an encouraging result for an experimental treatment. Although stem cell therapy itself has been used in oncology for over three decades, this particular procedure is not yet the standard of care.
Point of difference
“Normally, stem cells are used after high-dose chemotherapy to restore the blood-building system in malignant diseases,” says Dreier. “This treatment is different and is based on the idea that stem cells have the potential to differentiate various body tissues if in the right environment. It has been used for some years now to improve the status of some patients who otherwise have no standard medical options.”
In the context of cancer treatment, stem cell therapy aims to replenish the healthy cells killed during chemotherapy. Essentially, cytotoxic agents cannot discriminate between rapidly growing tumour cells and the hematopoietic stem cells in the bone marrow, meaning the patient may need a transplant if their blood cells are to be restored to normal. While this form of stem cell therapy is widely practised, it has a high mortality rate, killing around 38% of recipients. As a result, it is only used for patients who are already grappling with life-threatening conditions.
The real hope for stem cell therapy is that it might one day be used safely across a panoply of indications, ranging from the very minor, such as hair loss, to major conditions, such as heart disease. The Burjeel patients could be a sign of things to come, heralding a world of widely available regenerative medicine. While many types of stem cell treatments are being investigated, they can be bracketed into two categories in general: allogeneic and autologous. In allogeneic transplants, the cells come from a donor, but in autologous transplants, they come from the patients themselves. These two treatments may have different risks and benefits, as well as raising different concerns.
Allogeneic transplants are not currently performed in the UAE, owing to a limited regulatory framework and various infrastructure hurdles. Autologous treatments, however, are more likely to be approved, because they are widely seen as safe and non-contentious.
“As a haematologist, having done only autologous stem cell transplants in malignant diseases until now, I believe that autologous stem cells will have potential in other indications, and do not have the problems of rejection and ethical restrictions,” says Dreier.
Potent process
In a typical procedure, high-yielding mononuclear stem cells are harvested from the patient’s bone marrow, along with bone-marrow-derived plasma. Once the cells have been processed, they can be stored for later use – as is the case with cancer treatments – or administered immediately using the point-of-care technique, as was the case at Burjeel. After a short time, the stem cells multiply and produce the specific cells needed for the damaged organ to repair itself. While there are different types of stem cells in the body, they are generally multipotent, meaning they can give rise to all the different cell variants within an organ.
“In the patients at Burjeel, the bone-marrow stem cells were harvested and given back in the same procedure in the operating room under full anaesthesia,” says Dreier. “This meant minimal manipulating steps and no storage was needed. There is less risk of any adverse reaction and no risk of rejection.”
Following a treatment of this nature, patients require a couple of days in hospital to recover. Aside from that, the treatment is low-impact: the cells are recognised by the body, there is no risk of tumours forming in future, and improvements can be seen almost immediately.
“The only risk is due to the procedure, as you have to puncture the bone marrow and return the cells after processing into the desired tissue,” says Dreier. “So, although there is a risk of bleeding, infection, haematoma and pain, these are minor risks and manageable side-effects.”
Future promise
If the treatment comes to be used more broadly, it could hold promise for neurological diseases, coronary heart disease, strokes, chronic wounds, ischemia and more. In all these conditions, the common denominator is simply damaged tissue – the body is missing a population of important cells that could potentially be restored through stem cell transplants.
In the Middle East, the importance of such a treatment speaks for itself. Non-communicable diseases are on the rise throughout the region; in particular, heart disease and diabetes. According to WHO, around 34.5% of Emiratis are obese and 70.6% are overweight, correlating with a number of metabolic conditions.
In the case of heart disease, stem cells are injected directly into the heart through a coronary catheter, followed by bone-marrow-derived plasma. This regenerates the damaged coronary tissue. One 2012 paper showed that, two years after receiving stem cell therapy, an entire group of patients showed improvement in heart function.
With diabetes, there are various potential uses, such as the treatment of vascular disease and non-healing diabetic foot ulcers. Over the past few years, researchers from Tufts University in the US have shown that skin cells extracted from foot ulcers can be reprogrammed to a pluripotent state, thereby providing a store of autologous stem cells.
Meanwhile, a pilot study from Mansoura University in Egypt has found that patients treated with stem cells showed greater reduction in ulcer size than a control group. “At present, there are more indications available, especially wound care, decubiti [bed sores], diabetic feet, coronary heart disease and multiple sclerosis,” says Dreier. “Wound care, especially, is showing promising results and the application is relatively easy.”
If such treatments turn out to live up to their hype, the impacts could be significant. According to Professor Georg Kobinia, a pioneering Austrian surgeon who also works at Burjeel Hospital, the trajectory is likely to be towards increasing adoption and normalisation.
“Stem cell therapy is a new method, so it’s normal that people are afraid of what’s going to happen, despite the fact there have been more than 6,000 treatments without complications,” he said in 2013. “I like to compare this with the start of artificial insemination, when people were afraid of what kind of kids we’d have – would they be little ETs with green ears? Well, they are perfectly normal kids and that has become a normal procedure. I’m sure that in ten years’ time, stem cell therapy will be a completely normal procedure for everybody.”
Unsurprisingly, the market for autologous stem cell treatments is growing fast. According to a report by Transparency Market Research, the global market will climb from $3.8 billion in 2015 to $23.7 billion by 2025, with a compound annual growth rate of 21.9%.
Caution needed
However, it is worth pointing out that most of these treatments are at an early stage of development. While the research seems promising, many reports on their effectiveness remain purely anecdotal and are not backed up by large-scale randomised controlled trials. Recently, three women in the US were blinded by an experimental stem cell treatment for macular degeneration. Cases of this kind are rare but they do reinforce the importance of caution.
In the UAE, stem cell treatments are tightly regulated, a situation Dreier values. “At present, we only have restricted access to these procedures, as each and every treatment needs the approval of a health authority,” he says. “This, of course, is important and will mean such treatments are not available from institutions with less experience.”
He adds that since this is not the standard of care, stem cell treatments are not covered by patients’ health insurance. “More documentation and studies are needed to get it approved for standard treatment,” he said. “So, deciding on this approach requires a discussion with the patient and their relatives.”
Nonetheless, given the success of stem cell treatments to date, he is highly optimistic about the prospects.
“I do think that, within the next few years, we will use this kind of treatment more for indications where standard medicine has nothing to offer,” he says.
