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Cannabinoid treatment for all EB pain and itch (2024)

Tantangan sing penting kanggo wong sing duwe kabeh jinis EB yaiku nyeri lan gatel seumur hidup. Panaliten iki nyoba mbuktekake manawa iki bisa dikurangi kanthi obat berbasis cannabinoid.

Wong sing nganggo jas putih, mesem, ngadeg ing latar mburi burem biru cahya.

Dr Marieke Bolling nggarap Prof André P. Wolff ing Pusat Penyakit Blisting ing Pusat Kedokteran Universitas, Groningen, Walanda babagan nyeri lan gatel sing dialami wong sing ngalami EB. Amarga sawetara pasien EB ujar manawa obat-obatan berbasis cannabinoid (CBM) mbantu nyuda rasa nyeri lan gatel, proyek iki bakal nggoleki bukti iki ing pasien EB sing njupuk tetes minyak sing ngemot tetrahydrocannabinol (THC) lan cannabidiol (CBD) ing ilat kaping pirang-pirang saben dina sajrone 6 sasi. Bukti yen perawatan iki efektif bakal mbantu ngasilake pedoman kanggo nggunakake luwih akeh kanggo nambani rasa nyeri lan gatel ing EB.

Waca liyane ing blog peneliti kita

 

Babagan pendanaan kita

 

Pimpinan Riset Prof Dr André Wolff lan Dr Marieke Bolling
Lembaga Pusat Penyakit Blisting, Departemen Dermatologi, Pusat Medis Universitas Groningen
Jinis EB Kabeh jinis EB
Keterlibatan pasien Wong diwasa karo EB bakal nyoba obat cannabinoid
Jumlah pendanaan €177,200
Dawane proyek 3 taun (diperpanjang amarga Covid)
Tanggal wiwitan Agustus 2018
ID internal DEBRA Jonkman1

 

Rincian proyek

This trial tested an oil called Transvamix, containing medical cannabis, to see if it reduced pain in EB. Eight adults (men and women over 16 years old) with EBS, JEB or DDEB completed the study by self-administering an oil under their tongue. They didn’t have to stop other pain medication they were taking and could increase their use of the oil over two weeks until it helped, or they noticed side effects. Some were given Transvamix initially, then swapped to an oil that looked, smelled and tasted identical but didn’t contain the medical cannabis (a placebo). Others were given the placebo oil first then swapped to Transvamix. Neither the people taking part in the study nor the researchers knew, at the time, whether they were receiving Transvamix or the placebo and the order was selected randomly for each person (a randomised, double-blind trial).

The trial lasted seven weeks for each person, during which time they completed questionnaires about their EB symptoms and had MRI scans of their brain. The study finished at the end of 2024 and showed that the Transvamix provided some benefits. It reduced neuropathic pain, and half of those people who were taking other pain medication chose to reduce or stop it altogether while using the Transvamix. All reported side effects resolved on their own without medical attention or intervention. The results will be fully analysed and published.

Ing Nopember 2023, peneliti nglaporake manawa dheweke isih rekrut kanggo sinau klinis. Dheweke butuh paling ora wolung wong kanggo melu supaya asile bisa migunani. Saka pitung wong sing melu nganti saiki, luwih saka setengah wis rampung sinau. Dheweke ngarepake ndhaptar wong nomer wolu ing awal taun 2024 lan bakal nganalisa asil sawise wolung wong wis rampung sinau.

Ing April 2023, peneliti nglaporake manawa rekrutmen pasien kanggo panliten iki diwiwiti ing Maret 2022 nanging luwih sithik wong sing cocog tinimbang sing dikarepake amarga scan MRI sing direncanakake. Dheweke ngganti rencana supaya wong sing ora bisa scan MRI bisa melu sinau. Pitu pasien dijangkepi rampung sinau ing pungkasan Agustus 2023.

Peneliti nerbitake protokol uji klinis ing Desember 2022 lan nampa sawetara tanggapan. Dheweke nyaranake nerbitake manuskrip sing padha karo tim riset liyane ing lapangan EB.

Nganyari dituduhake dening institusi kasebut ing Maret 2022:

UMCG miwiti riset babagan efek minyak ganja obat ing pasien EB

Diagram sing nggambarake inisiatif sing didorong pasien kanggo riset obat-obatan berbasis cannabinoid kanggo nyeri lan gatel ing EB, sing nampilake otak, kulit, lan godhong ganja bebarengan karo upaya PhD sing fokus kanggo ningkatake perbaikan kulit ing JEB.

Prof Dr Andre Wolff yaiku kepala Pusat Nyeri UMCG, dadi ketua Pusat Nyeri Panggul UMCG lan nduweni minat khusus ing (medis) inovasi perawatan pasien sing gegandhengan karo nyeri kronis. Pakaryane nyangkut diagnosis presisi nyeri kanthi metode invasif ing pasien nyeri kronis lan ngenali nyeri neuropatik. Dheweke uga aktif ing bidang ilmu implementasine lan kualitas perawatan.

Dr Marieke Bolling, MD, PhD, minangka ahli dermatologi sing spesialisasine ing EB lan penyakit kulit warisan liyane lan minangka koordinator medis kanggo tim EB multidisiplin ing Pusat Penyakit Blisting ing Pusat Kesehatan Universitas Groningen. Dheweke kasil mbela tesis PhD ing taun 2010 kanthi judhul: Epidermolysis Bullosa Simplex. Pengalaman riset lan pengawasan dheweke bakal dadi penting kanggo sukses, implementasine lan ngrampungake proyek iki.

Dr José Duipmans, wis setya ningkatake perawatan liwat komunikasi saben dina sing aktif karo pasien lan cabang DEBRA NL lokal. Akeh wektu dheweke wis fokus kanggo mangerteni kabutuhan pasien, pangarepan kulawarga lan mangerteni masalah utama sing ditemokake dening bocah-bocah sing manggon karo EB. José bakal melu logistik sing dipusatake pasien ing panliten iki lan menehi titik kontak utama sajrone sinau.

Nicholas Schräder, BSc, wis nggarap EB wiwit 2010 lan wis njupuk peran aktif ing program pendidikan lan pasinaon sing gegandhengan karo EB kalebu pengalaman ing Rumah Sakit Prince of Wales Universitas Cina Hong Kong, Pusat Riset EB Universitas Freiburg Medical Center, Great Ormond Street Rumah Sakit Anak lan Pusat Kesehatan Universitas Groningen. Wiwit 2012, Nicholas wis melu riset ilmiah kanggo EB ing panuntun dhumateng Prof. Marcel Jonkman, kanthi khusus babagan perawatan gejala lan kualitas urip. Nemokake anekdot sing ora kaetung babagan panggunaan ganja medis kanggo macem-macem masalah sing gegandhengan karo EB, lan makarya ing sistem perawatan kesehatan Walanda kanthi pusat EB multidisiplin sing mapan, dheweke bisa nyelidiki implikasi potensial ganja medis, utawa cannabinoid, kanggo wong. nandhang EB.

Prof Dr Marcel Jonkman tilar donya ing Januari 2019. Dheweke misuwur amarga pendekatan inovatif kanggo riset ilmiah lan akademik EB. Karya lan kepemimpinane nyebabake pemahaman sing luwih jero babagan akeh dimensi sing nduwe peran ing patofisiologi EB, ing njero lan njaba klinik.

"Anekdot, crita lan pitakonan saka pasien penting banget kanggo pemahaman saiki babagan toleransi obat-obatan cannabinoid sing digunakake kanggo gejala EB ... Iki wektu kanggo nganalisis sacara ilmiah perawatan anekdot sing nguntungake iki lan ngupayakake protokol perawatan sing bisa ngowahi kualitas obat kasebut kanthi signifikan. urip pasien sing nandhang sangsara suwene urip."

Gelar Grant:

Substantiating efektifitas saka lenga adhedhasar phyto-cannabinoid sublingual kanggo perawatan saka pain lan pruritus (gatel) ing epidermolysis bullosa.

Panaliten sing dikontrol eksploratif prospektif babagan cannabinoid kanggo nambani nyeri kronis ing epidermolisis bullosa.

Transvamix (10% THC / 5% CBD) kanggo ngobati nyeri kronis ing epidermolysis bullosa: Sinau crossover intervensi acak, kontrol plasebo lan pindho wuta.

Wong sing nandhang epidermolisis bullosa (EB) ngadhepi gejala sing terus-terusan, ngrusak, kayata nyeri lan gatel. Laporan anekdot saka pasien EB nuduhake manawa obat-obatan berbasis cannabinoid (CBM) efektif kanggo ngontrol gejala.

Saben uwong duwe sistem molekul lan reseptor endokannabinoid sing nduwe peran ing macem-macem fungsi awak, kalebu tandha nyeri lan gatel - nggawe otak ngerti gejala kasebut.

Bukti kanggo macem-macem penyakit sing lara lan gatel, uga pengalaman pasien EB, nuduhake manawa CBM, sing diprodhuksi ing njaba awak (kayata saka tanduran Ganja) bisa tekan tingkat gejala sing padha karo obat-obatan utama. Asring luwih saka siji obat digunakake kanggo tujuan siji (contone kanggo nyeri, opiat & anti-inflamasi digunakake) sing bisa nyebabake efek samping sing ora dikarepake utawa ora perlu. Laporan anekdot anyar saka pasien EB ing saindenging jagad, kanthi rinci babagan pengalaman karo CBM, sing diresepake lan dipikolehi dhewe, wis akeh banget, lan njaluk riset ilmiah kanggo netepake safety lan efektifitas CBM. CBM kasedhiya ing akeh negara, kalebu Walanda, nanging kawruh lan pedoman khusus EB kurang.
Mula, nalika nyinaoni perawatan potensial iki, proyek iki duwe tujuan kanggo ngerteni apa pungkasane bisa nambah kualitas urip ing pasien EB.

Sacara global, peneliti saiki nliti macem-macem formulasi CBM lan formulir administrasi kanggo macem-macem penyakit. Ing Walanda, kemajuan anyar wis nyebabake standarisasi tanduran ganja kanggo panggunaan terapeutik dening pasien Walanda. Babagan pilihan CBM kanggo panliten iki, "phytocannabinoids" (cannabinoid adhedhasar tanduran) diekstrak saka tanduran lan digabungake menyang lenga sing diwenehake minangka tetesan ing ilat (sublingual). Produk pungkasan yaiku minyak CBM kelas farmasi, lan saiki disebarake kanggo pasien ing Walanda sing wis entuk resep saka dokter sing wis kadhaptar.

Panggunaan CBM adhedhasar tanduran ora gawe ketagihan fisik, lan ora ana hubungane karo komplikasi sing ngancam nyawa utawa ngrusak. Amarga saiki ora cukup informasi babagan efek jangka panjang panggunaan cannabinoid ing sistem saraf sing berkembang ing bocah-bocah, panliten iki mung kanggo pasien sing umure luwih saka 18 taun. Kriteria inklusi spesifik bakal nganggep kondisi kejiwaan sing wis ana amarga bisa kena pengaruh nggunakake CBM.

Panaliten kasebut bakal nyelidiki yen perawatan tambahan karo lenga sublingual iki bisa ningkatake kualitas urip wong diwasa kanthi EB nandhang lara lan / utawa gatel. Efek saka lenga CBM bisa tahan 4-8 jam lan mulane kudu dijupuk kaping 4 saben dina kanggo njaga tingkat getih. Patients bakal laporan pain, gatel lan owah-owahan ing kualitas urip saben wulan liwat 6 sasi. Kualitas urip, nyeri lan gatel bakal diukur kanthi nggunakake kuesioner pasien utawa pangukuran asil sing dilaporake pasien. Sawetara iki bakal digunakake kanggo uga ngenali pangukuran sing paling sensitif marang owah-owahan ing pasien EB.
Mungkasi obat saiki ora dibutuhake minangka peserta sinau, sajrone riset, tim riset bakal ngawasi owah-owahan panggunaan obat saben pasien (kayata nyuda panggunaan opiate), lan ing pungkasan mriksa apa hubungane. karo nggunakake lenga CBM.

Panaliten iki dianggep minangka prospektif, label terbuka, bukti studi konsep - nguji obat anyar ing sawetara pasien cilik, ing ngendi saben wong njupuk obat anyar kanggo ndeleng apa keuntungan sing bisa diidentifikasi. Mula, nalika nyinaoni perawatan potensial iki, proyek iki duwe tujuan kanggo ngerteni apa pungkasane bisa nambah kualitas urip ing pasien EB.

Pengalaman positif sing dituduhake dening dokter lan pasien karo EB karo cannabinoid sing digunakake minangka agen terapeutik, wis nyumbang banget kanggo pemahaman kita. Panaliten iki bakal miwiti proses nganalisis anekdot kasebut kanthi ilmiah lan ngupayakake protokol perawatan lan pedoman adhedhasar bukti sing bisa ngowahi kualitas urip pasien EB kanthi signifikan. Amarga panliten iki kalebu panggunaan minyak CBM sing diwenehake kanthi sublingual, asil lan kesimpulan bakal cocog karo formulasi lan formulir administrasi CBM iki, amarga formulasi lan formulir administrasi liyane diproses dening awak manungsa liwat mekanisme sing beda-beda.

 

Panaliten sing dikontrol eksploratif prospektif babagan cannabinoid kanggo nambani nyeri kronis ing epidermolisis bullosa.

Transvamix (10% THC / 5% CBD) kanggo ngobati nyeri kronis ing epidermolysis bullosa: Sinau crossover intervensi acak, kontrol plasebo lan pindho wuta.

Karya persiapan lan regulasi kanggo uji klinis wis rampung. Saliyane persetujuan etika, logistik kanggo koordinasi obat sinau, pencitraan radiografi lan formulir laporan kasus online (database) wis disetel lan disetujoni. Tanggal 15-03-2022 rekrutmen kanggo partisipasi wis diwiwiti. Periode inklusi sing dikarepake yaiku 6 sasi. Penutupan sing dikarepake ditindakake kanthi analisis data yaiku Q4 2022.

Tim riset nemoni wektu tundha sing signifikan amarga proses vetting etika. Iki nyebabake metodologi uji klinis sing dirumusake maneh kanggo ningkatake kualitas lan kekuwatan sinau - kanthi khusus babagan parameter data obyektif (fMRI-nggunakake, kontrol plasebo, desain cross-over). Kajaba iku, kabeh masalah ing pandemik COVID, lan peraturan akibate, nyedhiyakake penundaan tambahan. Kaya sing kasebut ing ndhuwur, owah-owahan metodologi nyuda beban partisipasi, lan ngidini koleksi lan analisis asil sing kuat. Wiwit disetujoni kanggo miwiti kegiatan riset, perhatian media saya tambah, DEBRA-UK kacathet minangka distributor hibah. (Saka laporan kemajuan 2022).

Pambuka

The C4EB study concluded at the end of 2024. This project was initiated by our research team at the University Medical Centre of Groningen, with a focus on research that improves daily life. The team included a diverse group of experts: José Duipmans (Nurse Practitioner), Marieke Bolling (Dermatologist), André Wolff (Pain Specialist), Roy Stewart (Epidemiologist), Karin Vermeulen (Methodologist), Peter Söros (Neurologist/Neurophysiologist), and Nicholas Schräder (Physician/PhD Researcher).

The C4EB study aimed to investigate whether a cannabinoid-based medicine, Transvamix (medical cannabis in the form of an oil applied under the tongue), could help reduce pain in patients with Epidermolysis Bullosa (EB). The study drug was prepared by a research pharmacy and contained two cannabinoids: THC and CBD. Over recent years, research has highlighted the potential benefits of cannabinoids for managing pain and itch in various conditions, including EB. While patients often report positive effects, it is crucial to study these medications under controlled conditions to understand how they work and how effective they are.

Since this study involved a medical treatment, strict criteria were in place for who could participate and which assessments they would undergo. Participants had to be adults aged 16 or older, and they needed to experience pain as a symptom of EB at the time of inclusion. The study lasted 7 weeks for each participant, during which they received both Transvamix and a placebo oil (which looked, smelled, and tasted identical to the study drug). To ensure reliable results, neither the participants nor the researchers knew the order in which Transvamix or placebo was given.

Participants gradually increased their dosage over two weeks until they either experienced relief from their symptoms or noticed side effects. In addition to completing surveys about pain and how they coped with it, participants also underwent MRI scans. These scans helped researchers observe how the brain processes pain signals, similar to creating a “video recording” of brain activity. Throughout the study, participants were allowed to continue their usual pain medications and adjust their doses as needed.

 

results

A total of 9 patients with EB participated in the study. Unfortunately, one participant dropped out early, though this was unrelated to Transvamix or the placebo. The remaining participants represented various types of EB, including EB Simplex, Junctional EB, and Dominant Dystrophic EB.

The primary focus of the study was a pain survey that categorized pain into four types. For three of these types—continuous pain, intermittent pain, and affective pain (emotional processing of pain)—there was no significant difference in relief between the placebo and Transvamix. However, for neuropathic pain (pain linked to nerve dysfunction), participants reported more relief when using Transvamix compared to the placebo.

Additionally, among participants who were taking pain medication during the study, half were able to reduce their dosage or stop their medication altogether while using Transvamix. Importantly, both participants and researchers reported any side effects (adverse events) that occurred during the study, whether related to the placebo or Transvamix. All reported side effects were considered mild or moderate and resolved on their own, typically within a few hours, without requiring medical attention or intervention.

 

Data to be Processed

The MRI data collected during the study requires complex calculations and detailed analysis, which is still underway. Over the next few weeks, our team will finalize the results and prepare a comprehensive summary for publication in a scientific journal, allowing EB and non-EB researchers around the world to learn from our findings.

 

Serat

This study is the first to formally evaluate the use of cannabinoid-based medicines to manage pain in patients with EB. While the results may not be considered groundbreaking, they provide valuable insights. Transvamix, when used in a controlled and safe environment, showed potential benefits for some patients with EB, particularly in managing neuropathic pain. The reduction in the use of other pain medications and the mild nature of reported side effects further support the potential of cannabinoid-based treatments for EB-related pain.

 

Prospek Future

Through this study, we hope to inspire other researchers and clinicians to undertake similar investigations, both in clinical practice and research. Every patient with EB is unique, and there are many promising avenues for exploring cannabinoid-based treatments. These include variations in cannabinoids (CBD vs. THC), forms of administration (oil, cream, vapor), and addressing other challenges faced by patients, such as itch, chronic wounds, and scarring. Additionally, this study’s methodology—using a small group of participants and incorporating a placebo—provides a promising framework for future research. Our team is currently working on the COSEB project (Core Outcome Set for EB), which aims to standardize the tools and measurements used in EB research. We are happy to answer any questions you might have about this research. Please bear with us as we prepare to publish the complete data as soon as possible. Please see the links below, news articles referring to our work.

Links:

UMCG starts scientific research into cannabis oil for chronic pain in Epidermolysis Bullosa November 2021

The effectiveness of cannabis oil in pain management for EB-patients

UMCG miwiti riset babagan efek minyak ganja obat ing pasien EB Maret 2022

UMCG starts research into medicinal cannabis for Epidermolysis Bullosa Maret 2022

UMCG starts research into the effects of medicinal cannabis oil Maret 2022

(From 2024 final progress report.)