A novel gene therapy, focused on the NaV1.7 sodium ion channel, has been developed by researchers, providing a fresh avenue to manage chronic pain by controlling a specific protein interaction.
The location where a protein oversees sodium ion channels has been identified by scientists. By incorporating the channel’s genetic material into a virus, they have been successful in mitigating pain in both cellular and animal experiments.
According to a study published in the Proceedings of the National Academy of Sciences (PNAS), a team of researchers at NYU College of Dentistry’s Pain Research Center has designed a gene therapy that handles chronic pain by indirectly governing a particular sodium ion channel.
This inventive therapy, experimented in cells and animals, is facilitated by the finding of the exact region where a regulatory protein binds to the NaV1.7 sodium ion channel to manage its function.
“We have made significant progress in understanding the fundamental biology of the NaV1.7 sodium ion channel, which can be exploited to offer respite from chronic pain,” commented Rajesh Khanna, director of the NYU Pain Research Center and professor of molecular pathobiology at NYU Dentistry.
Chronic pain is a major public health concern affecting approximately a third of the U.S. population. Scientists are keen on developing more efficient and safer alternatives to opioids for pain treatment.
Sodium ion channels are essential in the creation and transmission of pain as they are vital for neurons to communicate amongst themselves. A specific sodium ion channel called NaV1.7 has emerged as a potential target for pain treatment due to its significance in individuals with rare, genetic pain disorders. Some genetic mutations allow sodium to enter cells in large quantities, causing intense chronic pain, while others that block NaV1.7 lead to total absence of pain.
While attempts to develop treatments to selectively block NaV1.7 have had limited success, Khanna has taken a different approach by aiming to indirectly control it using a protein named CRMP2.
CRMP2 interacts with the sodium ion channel, influencing its activity and regulating the influx of sodium into the channel. By blocking the interaction between Nav1.7 and CRMP2, the influx of sodium can be reduced, thereby decreasing neuronal activity and alleviating pain.
Khanna’s lab has previously developed a molecule that indirectly manages Nav1.7 expression by targeting CRMP2. Although successful in controlling pain in cells and animals, one unresolved question remained: why does CRMP2 only interact with the NaV1.7 sodium ion channel and not the other eight sodium ion channels in the same family?
In their PNAS study, the researchers found a unique region within NaV1.7 where the CRMP2 protein binds to the sodium ion channel to govern its activity. They discovered this region is exclusive to NaV1.7, as CRMP2 did not readily bind to other sodium ion channels.
To inhibit the interaction between CRMP2 and NaV1.7, the team created a peptide from the channel that corresponds to the region where CRMP2 binds to NaV1.7. They incorporated this peptide into an adeno-associated virus to deliver it to neurons and obstruct NaV1.7. Using viruses to deliver genetic material to cells is a promising gene therapy approach, leading to effective treatments for blood disorders, eye diseases, and other rare conditions.
The modified virus was administered to mice experiencing pain, including sensitivity to touch, heat, or cold, as well as peripheral neuropathy caused by chemotherapy. After a week to 10 days, the animals’ pain was alleviated.
“Our findings show the potential of using engineered viruses, containing a small piece of genetic material from a protein we all possess, to effectively manage pain by infecting neurons,” said Khanna.
The team also replicated their findings across different species, including rodents and cells of primates and humans. While further studies are necessary, these initial results are promising for the translation of this therapy into a treatment for humans.
Khanna further stated, “There’s a significant need for new pain treatments, including for cancer patients suffering from chemotherapy-induced neuropathy. Our ultimate goal is to develop a gene therapy that could be used to better manage these painful conditions and enhance patients’ quality of life.”
Table of Contents
Frequently Asked Questions (FAQs) about Gene Therapy for Chronic Pain
What is the new approach to treating chronic pain that researchers have developed?
Researchers have developed a gene therapy that targets the NaV1.7 sodium ion channel. By manipulating a specific protein interaction, this innovative therapy offers a promising new approach to treating chronic pain.
How does this new gene therapy work?
The gene therapy works by indirectly regulating a specific sodium ion channel. Researchers identified a specific region within the NaV1.7 sodium ion channel where a regulatory protein, CRMP2, binds to control its activity. The therapy uses an engineered virus to deliver a peptide, which corresponds to this region, to neurons in order to inhibit NaV1.7 and effectively manage pain.
How significant is this development in the field of pain management?
This development is significant as it represents a major step forward in understanding the biology of the NaV1.7 sodium ion channel and how it can be harnessed to provide relief from chronic pain. It offers a promising alternative to opioid-based treatments.
What were the results of the therapy in the study?
In the study, the therapy was successful in controlling pain in both cells and animals. After the engineered virus was administered to mice experiencing pain, their pain was reversed within a week to 10 days. The researchers were also able to replicate their findings across different species, including rodents and cells of primates and humans.
What are the next steps for this therapy?
While the initial results are promising, more studies are needed to further understand this approach and its potential application in human pain management. The long-term goal is to develop a gene therapy that could be used to better manage painful conditions and improve patients’ quality of life.
More about Gene Therapy for Chronic Pain
- Gene Therapy
- Chronic Pain
- NaV1.7 Sodium Ion Channel
- CRMP2 Protein
- Adeno-Associated Virus
- Proceedings of the National Academy of Sciences (PNAS)
- NYU College of Dentistry’s Pain Research Center
4 comments
Does anyone know when this will be available to the public? Seems promising and I know many people who would benefit. thanks!
Wow, this is incredible! Cant belive what science can do these days. Hope this can give relief to so many suffering from chronic pain.
that’s amazing, especially when u think about all those who are addicted to opioids because of their pain. its about time we have a safer alternative.
This is exactly why i am so interested in biology!! to think we can manipulate our own proteins to help alleviate pain is just… mind blowing.