Medicine is full of challenges. Finding and developing useful treatments for rare disorders or uncommon forms of other disorders is one, especially given the cost of FDA clinical trials, first for safety and then for efficacy.

But what if there were a candidate drug whose safety and lack of toxicity has been tested for centuries and longer without a single death? That could cut clinical research at least in half from the beginning. And surprise, we know of one such medicinal agent….

That’s our take of course. It’s somewhat implied by the article here though, so we’ll let it speak for its less exuberant but important self….

“Modern medicine is an increasingly precise undertaking involving thousands of possible diagnoses, many with multiple treatment options.

There is a wide range of disorders that involve crippling anxiety, including post-traumatic stress disorder. There are also many seizure disorders, including conditions like Dravet syndrome and Lennox-Gastaut syndrome, that are extremely difficult to treat.

In the same fashion, there are hundreds of disorders that cause debilitating pain that persists for months, years or even a lifetime, including interstitial cystitis, Complex Regional Pain Syndrome, and trigeminal neuralgia.

Many of these disorders are rare and entirely unfamiliar to non-specialists. But even when the disorder itself is not so rare, its presentation may be rare in terms of severity. Fibromyalgia and osteoarthritis are common enough, but still can be debilitating in some cases.

As a result, research on such disorders is thin and clinical trials are few and far between. For instance, there are no studies of cannabis for small fiber neuropathy and only a handful on cannabis for cluster headaches. When trials do exist, they are easily criticized as being statistically underpowered because of the small number of participants.

Moreover, standard treatments do not necessarily work for everyone. Neuropathic pain sometimes responds well to neuroleptic drugs like gabapentin (Neurontin), but as a recent Cochrane review found, over half of those treated with gabapentin will not have worthwhile pain relief and may experience adverse side effects.”

The article goes to talk about issues relating to agents like opioids and cannabis and to balance out all the complex concerns in play here. Interesting, well-written and worth advocates and patients’ time.

#MMJ #Research #RareDisorders #OrphanDiseases #Pain #UTpol #TRUCE    

See full article – Rare Disorders Require Unusual Care