Substantiating the effectiveness of a sublingual phyto-cannabinoid based oil for the treatment of pain and pruritus (itch) in epidermolysis bullosa.Ongoing
|Project lead||Nicholas Schräder and Professor André P. Wolff|
|Organisation||University Medical Center Groningen, Groningen|
|Partner organizations & collaborators||Initiated by Professor Marcel Jonkman|
|Project budget||EUR 177,200|
|Start date / Duration||01. Aug 2018 / 36 months|
|Funder(s) / Co-Funder(s)||DEBRA UK, MSAP/EBEP Reviewed|
|Research area||Symptom prevention & relief|
Short lay summary
People with Epidermolysis Bullosa (EB) face constant, debilitating symptoms, such as pain and itch. Anecdotal reports from EB patients suggest that cannabinoid-based medicines (CBMs) are effective for symptom control.
Everybody has an endocannabinoid system of molecules and receptors that play a role in the various functions of the body, including the signalling of pain and itch – making the brain aware of those symptoms.
Evidence for numerous painful and itchy diseases, as well as EB patient experiences, suggest that CBMs, produced outside of the body (such as from the Cannabis plant) reach comparable levels of symptom relief to mainstream medicines. Often more than one medicine is used for a single goal (e.g. for pain, opiates & anti-inflammatories are used) which may lead to unwanted or unnecessary side effects. Recent anecdotal reports from patients with EB around the world, detailing their experience with CBMs, both prescribed and self-obtained, have been overwhelming, and call for scientific research to assess the safety and effectiveness of CBMs. CBMs are available in a growing number of countries, including the Netherlands, however EB specific knowledge and guidelines are lacking.
Therefore, in researching this potential treatment, this project aims to gain an insight as to whether it can ultimately improve the quality of life in patients with EB.
Globally, researchers are currently looking into various CBM formulations and administration forms for numerous diseases. In the Netherlands recent advances have led to the standardisation of cannabis plants for therapeutic use by Dutch patients. With regard to the CBM choice for this study, these “phytocannabinoids” (plant-based cannabinoids) are extracted from the plants and incorporated into an oil which is administered as droplets under the tongue (sublingually). The final product is a pharmaceutical - grade CBM oil, and is currently distributed to patients in the Netherlands who have obtained a prescription from their registered physician.
The use of plant-based CBMs is not physically addictive, and has not been associated with life threatening or debilitating complications. As there is currently not enough information about the long term effects of cannabinoid use on the developing nervous system in children, this study is only for patients over 18 years old. Specific inclusion criteria will take into account pre-existing psychiatric conditions as these can be affected by CBM use.
The research will investigate if additional treatment with this sublingual oil can improve the quality of life of adults with EB suffering from pain and/or itch. The effects of the CBM oil can last 4-8 hours and will therefore need to be taken 4 times daily to maintain blood levels. Patients will report pain, itch and changes in their quality of life monthly over 6 months. Quality of life, pain and itch will be measured by the use of patient questionnaires or patient reported outcome measurements. A number of these will be used to also identify which measurements are most sensitive to changes in patients with EB.
Stopping current medication is not required as a study participant, during the course of the research, the research team will monitor the change in medication-use of each patient (such as a reduction in opiate use), and at the end scrutinize whether this correlates with CBM oil use.
This study is considered a prospective, open label, proof of concept study – testing a new medicine in a small number of patients, where everyone takes the new medicine to see what benefits can be identified. Therefore, in researching this potential treatment, this project aims to gain an insight as to whether it can ultimately improve the quality of life in patients with EB.
Positive experiences shared by clinicians and patients with EB with cannabinoids used as therapeutic agents, have significantly contributed to our understanding. This study will start the process of scientifically analysing these anecdotes and work towards a treatment protocol and evidence based guidelines which may significantly alter the quality of life of EB patients. As this study involves the use of a sublingually administered CBM oil, the results and conclusions will be largely relevant to this CBM formulation and administration form, as other formulations and administration forms are processed by the human body through different mechanisms.