Using stem cells to treat cancer

There are many scam artists around nowadays proclaiming the benefits of their particular unproven stem cell therapy, for anything from curing cancer to making paralysed people walk again. It’s not surprising really; stem cells are a pool of cells in every organ that are almost eternally youthful and can regenerate themselves and all other cells in the organ. They sound almost magical. However, last year the FDA (the US Food and Drug Administration) had to move to crack down on these clinics, citing the of lack of evidence that any of them work and a number of serious complications reported following treatment. Complications including patients in Florida dying, a woman developing bone fragments in eyelids following a stem cell facelift, and another developing nasal tissue in her spine after a doctor promised to cure her paralysis with stem cells.

It is a field ripe for abuse partly because it is one with so much potential. Stem cells do have fascinating possible applications, and there is a lot of research going in to them at the moment. Unfortunately, most exposure people have with them is in science fiction or alternative medicine. Which is why it was very interesting to see a study published last week that underlined how much real potential this field of research has. The study used mice instead of humans, so is still at an early stage, but is very promising nonetheless. Continue reading

What happens when we don’t publish clinical trials

The last blog I posted emphasised the importance of publishing all clinical trials. The story of Lorcainide is a stark warning of what happens when we don’t.

In 1980 a cardiologist in Nottingham named Alan Cowley carried out a small clinical trial of a drug called Lorcainide. It was known at the time that heart attacks could cause irregular heartbeats in patients (known as arrhythmia), and these arrhythmias often lead to early death. Lorcainide had been shown to suppress arrhythmia, so it made sense that patients who came to hospital with a heart attack should be treated with the drug. Cowley and his colleagues carried out a small trial with 95 patients, and tested them to see whether they were getting more or fewer arrhythmias. The drug worked, lowering the frequency of serious arrhythmia.

The doctors noticed something else however. Of the 48 patients on the drug, 9 had died, compared to only 1 patient on the placebo. This was a very small trial, so the doctors weren’t overly alarmed. It’s not surprising that 10 patients died in the study; these are patients who are presenting with heart attacks after all. It was just worrying that there was such an imbalance between the groups. The doctors chalked it up to bad luck, and viewed their trial as a success.

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Problems with clinical trials

Clinical trials are at the heart of our progress in medicine. If we have a new therapy, clinical trials tell us whether it is better than the current one. They measure outcomes, but also look out for side effects and unexpected consequences of taking the therapy. They are absolutely essential to our progress, and it is vital that they are carried out properly and transparently. Continue reading

The placebo effect

The recent decision by NICE to no longer recommend acupuncture for lower back pain got me thinking about the placebo effect. It is a bizarre phenomenon: any treatment (regardless of whether it is a real treatment or not) will improve symptoms in some people simply because the recipient believes that it will work. So if we give someone a placebo (a sugar pill for example) and tell them that it can work for their illness, a proportion of patients will feel better. There are so many interesting things about the placebo effect it’s difficult to know where to start.
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fig15_killertSometimes it pays to think differently about a problem. Who would think that treating a cancer without actually targeting the cancer could be a viable strategy? But this is exactly what the latest major breakthrough in cancer therapy has shown.

The breakthrough is in a group of drugs called “Immune checkpoint blockers”. They have already provided remarkable results in the treatment of two of the most common and dangerous cancer types (melanoma skin cancer and lung cancer), and they are being tested in other tumour types with promising results so far. Interestingly, these efficient and powerful chemotherapies do not kill tumour cells directly. Instead, they are cleverly designed to boost the body’s own defences against the tumours. Continue reading

Gene therapy for cystic fibrosis

Cystic fibrosis is a terrible disease, which often results in fatal lung failure by the age of 30. It is the most common genetic life-shortening disease in the Caucasian population, and it is caused by mutations in one single gene. The first gene therapies were tested in clinical trials in the early nineties. It felt like the cure was round the corner. However, more than 20 years later, we are still trying to find the best way of restoring the function of the damaged gene. Last month, the scientific journal Lancet respiratory Medicine published a study where a new gene therapy was tested on cystic fibrosis patients. This therapy was reported by several news outlets as a potential cure for cystic fibrosis by 2020. However, the truth is that the therapy failed to detect a relevant clinical benefit for the patients.

Why the lack of progress?

Read more about where we stand with gene therapy for cystic fibrosis in the blog post I wrote as a guest author for !