RNA therapy has shown real promise against psoriasis in its first human trial


A phase 1 trial involving a new type of RNA therapy has shown that the treatment could be used to fight psoriasis, a debilitating skin condition that affects nearly 3 percent of the world’s population.

At the American Chemical Society (ACS) meeting in Philadelphia last week, researchers announced that AST-005, a type of RNA therapy, is safe to use in humans, and were optimistic about the drug’s dose-dependent response in psoriasis patients.

Psoriasis is an auto-immune disease, triggered when the body creates too much of a normally healthy protein, tumour necrosis factor-α (TNF-α). The immune system attacks this protein, causing red, itchy, and scaly skin patches.

Right now, there is no cure, and very limited treatments, but RNA could be the key to controlling it.

In every one of your cells, RNA acts as a messenger between your DNA and protein. Although DNA stays in the nucleus at all times, RNA moves around the cell, directing the creation of various proteins.

One of the ways scientists have been able to limit protein creation – such as the overproduction of TNF- α – is by destroying RNA genes using a relatively new technique called RNA interference, or RNAi. RNAi enters the cell, destroys the regular RNA, and less protein is created.

While we’ve been able to use RNAi in animal models pretty effectively, as Robert Service explains at Science Magazine, this has been difficult to get right in humans:

“The trouble is that traditional antisense RNA drugs [synthetic RNAi] usually don’t work. To date, only two antisense RNA therapies have been approved in the United States, despite decades of effort and dozens of clinical trials.

Among other problems, most introduced RNA snippets get chopped up before they reach their target by enzymes that patrol the cell for foreign material.”

But there’s renewed hope that this technique could work in humans, with the development of a new type of RNAi, called spherical nucleic acid (SNA).

Developed by researchers at US-based company, Exicure, AST-005 is a gel made up of SNAs, which has been shown in the past to lower the amount of TNF-α in animal trials. And unlike previous synthetic RNAis, the SNA’s structure is not chopped up by enzymes in the cell.

Back in April, researchers from Exicure announced treatment of its first patients for a phase 1 clinical trial for the AST-005 gel, which they hoped would lower the amount of TNF-α protein produced by the corresponding gene, and therefore limit patients’ symptoms.

“This clinical trial will enable our team to study safety and tolerability of AST-005 while demonstrating that the SNA technology can be used to treat diseases locally using a nucleic acid therapy. We are excited to bring this approach to patients in need,” said David Giljohann, CEO at Exicure, when the trial was first announced.

The results are looking promising. At the ASC meeting, one of the team, Chad Mirkin, explained that AST-005 has been found to be safe in humans, and shows a dose-dependent response to TNF-α.

This means that although there is more work to do in finding the correct dose, the researchers are hoping that a treatment could be on the way for those suffering psoriasis.

The researchers didn’t go into much detail, as it is still very early days yet. The initial observations have only been discussed at the ASC meeting, there has been no paper published in a peer reviewed journal, so unfortunately we can’t explain much more about the trial, or get too excited just yet.

But if the treatment continues to show promise in this and other follow-up trials, it’s just the beginning for similar SNA therapies, with the potential for more new drugs based on this technique to target cancer-causing genes, and a number of auto-immune diseases.

Although this is just an initial observation, we’re looking forward to seeing the final results.

First Ever Human Trial Finds Magic Mushrooms Beat Severe Depression.


Get ready world, “magic” mushrooms, which contain the psychoactive compound psilocybin, may soon become the standard go-to for reversing what the World Health Organization says is the number one cause of disability on the planet: depression.

A brand new first of its kind study published in The Lancet reports that psilocybin mushrooms were able to lift the severe depression of all twelve human volunteer participants, even though they had been struggling with the disease for an average of over seventeen years and despite that fact that none of the subjects had found relief with multiple rounds of standard anti-depressant medication.

“This is the first time that psilocybin has been investigated as a potential treatment for major depression,” says lead study author Dr Robin Carhart-Harris of the Imperial College London, where the study took place. “Treatment-resistant depression is common, disabling and extremely difficult to treat. New treatments are urgently needed, and our study shows that psilocybin is a promising area of future research.”

Via: The Lancet

Via: The Lancet

What is most remarkable about the study is that the depression symptoms lifted considerably following just a single treatment dose of psilocybin for every participant in the study, and for a majority of them the antidepressant effects of the mushrooms were still in effect three months after the dosing.

Amazingly, five of the original twelve severely depressed patients were in complete remission from depression three months after the study took place, even though they were following no other treatment plan.

“Previous animal and human brain imaging studies have suggested that psilocybin may have effects similar to other antidepressant treatments,” says Professor David Nutt, who co-authored the study. “Psilocybin targets the serotonin receptors in the brain, just as most antidepressants do, but it has a very different chemical structure to currently available antidepressants and acts faster than traditional antidepressants.”

Depression is usually treated with selective serotonin re-uptake inhibitors (SSRIs), which not only have a long list of negative side effects associated with them, including dizziness, insomnia, headaches, and even lower birth weights in infants, but need to be taken on a daily basis as well.

Psilocybin mushrooms, on the other hand, are entirely natural and do not need to be taken every day in order for one to experience their profound anti-depressive properties. They can be consumed when needed, and their benefit can last for weeks, months, or even years after each session.

“The key observation that might eventually justify the use of a drug like psilocybin in treatment-resistant depression is demonstration of sustained benefit in patients who previously have experienced years of symptoms despite conventional treatments, which makes longer-term outcomes particularly important,” says Professor Philip Cowen, a clinical scientist at the University of Oxford, in a linked comment on the study.

The truth is that a fast-acting and completely natural single dose anti-depressant that actually has higher remission rates than any other current treatment available could totally revolutionize the way depression is currently handled in mainstream medicine. Mother Nature has proven herself superior to chemical cocktails once again.

The only thing standing in the way is the law of course, as psilocybin mushrooms are still classified under Schedule I, despite this study, other similar findings, and their long history of medicinal and sacred use in indigenous cultures.