FDA Gives Orphan Drug Status to Ketamine for CRPS

Complex Regional Pain Syndrome (CRPS) is one of the most challenging chronic pain syndromes to treat. There are currently no effective, long-term treatments that work for most patients. In response to new research results indicating ketamine helps many patients with CRPS, the FDA has approved PharmaTher Holdings’ request for ketamine’s orphan drug designation. The Toronto-based pharmaceutical company has also been granted orphan drug status for ketamine to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease).

“Receiving our second FDA orphan drug designation with ketamine for CRPS continues our momentum in building a specialty ketamine-based product pipeline for not only mental health disorders but also for rare and near-rare conditions present in neurological, pain, and inflammatory disorders,” explained Fabio Chianelli, CEO of PharmaTher.

Like opioids, ketamine works on the brain’s pain receptors without the troubling addiction and overuse concerns surrounding many opioids. Currently, ketamine is FDA-approved only for treating depression and use as anesthesia. However, in intravenous infusions, ketamine can be used for complex pain conditions, including CRPS.

The orphan drug status removes some of the research restrictions for drugs used to treat rare or uncommon conditions so that treatments are brought to market sooner, and more companies are willing to investigate alternative uses for established medications. Orphan drug incentives can include tax credits, exclusive marketing rights, and waivers on FDA filing fees, which can run into millions of dollars.

PharmaTher hopes to begin Phase 2 clinical trials for treating CRPS with ketamine in 2022. They have also started clinical studies focused on treating Parkinson’s disease with ketamine. In the future, they hope to develop ways to treat Type 2 diabetes and obesity with ketamine as well. Stringent guidelines will be in place, as ketamine can induce hallucinations and visual problems in some patients.

Potential Vaccine for Rheumatoid Arthritis Discovered at University of Toledo

While researching a specific protein and its link to immune system problems, researchers at the University of Toledo discovered that the protein (14-3-3) might help, rather than hinder, the fight against rheumatoid arthritis (RA). Earlier studies indicated that the protein could trigger RA and other autoimmune diseases, resulting in inflammation, pain, and loss of bone density.

Researchers were startled to find that 14-3-3 zeta proteins seem to prevent rather than aggravate arthritis. When the protein was removed from laboratory animals using gene editing, arthritis onset was early and severe. In response, researchers developed a vaccine containing the purified protein. Animals injected with the vaccine produced a long-lasting response that seems to protect lab animals from rheumatoid arthritis. Preliminary results also indicate that vaccinated rodents developed improved bone density and collagen production.

“Much to our happy surprise, the rheumatoid arthritis totally disappeared in animals that received a vaccine. Sometimes there is no better way than serendipity. We happened to hit a wrong result, but it turned out to be the best result. Those kinds of scientific discoveries are very important in this field,” noted Ritu Chakravarti, Ph.D., lead author of the research and assistant professor at the University of Toledo College of Medicine. The findings were published in Proceedings of the National Academy of Sciences.

Ultrathin Brain Electrodes May Be Effective in Treating Chronic Pain

Researchers at Lund University in Sweden are excited about the results seen in rats when using a new type of targeted brain stimulation to alleviate chronic, severe pain. The new treatment uses extremely thin microelectrodes to stimulate specific areas of the brain related to pain perception. The treatment can be tailored to each patient’s particular needs and doesn’t have the side effects often experienced with traditional analgesics.

“The electrodes are very soft and extremely gentle on the brain. They are used to specifically activate the brain’s pain control centers without simultaneously activating the nerve cell circuits that produce side effects. The method involves implanting a cluster of ultra-thin electrodes and then selecting a sub-group of the electrodes that provide pure pain relief but no side effects. This procedure enables extremely precise and personalized stimulation treatment that was shown to work for every individual,” explained Professor of Neurophysiology Jens Schouenborg.

The tissue-friendly electrodes are gelatin-based and are surgically implanted in the brain with extreme precision. The electrodes stimulate the pain control centers in the brain, blocking only the transfer of pain signals. In theory, this stimulation may successfully treat any type of pain transmitted via the spinal cord, which accounts for the majority of chronic pain issues.

Matilde Forni, first author of the new study, noted, “We have achieved an almost total blockade of pain without affecting any other sensory system or motor skill, which is a major breakthrough in pain research. Our results show that it is actually possible to develop powerful and side-effect-free pain relief, something that has been a major challenge up to now.”

This Week’s Pain Fact: The cost of treating chronic pain is increasing at an astronomical rate. By 2027, the market for pain management drugs worldwide is expected to reach over $91 billion, with North America being the largest market. Running close behind will be Canada, Mexico, and Europe. By 2023, the demand for pain-relieving drugs is expected to reach nearly $6 billion.