I hope MUSC is connecting with biotechs regarding Kallikrein serine protease inhibitors to explore their potential in alleviating hEDS symptoms caused by pathological Kallikrein variants, such as pain, inflammation, and swelling. Incidentally, the biotech company behind Orladeyo® (berotralstat) is located in North Carolina. Berotralstat is the first oral plasma kallikrein inhibitor that is FDA and EMA approved for the prevention of HAE attacks. Your pioneering paper mentions that; "Additionally, in certain pathological conditions such as hereditary angioedema (HAE), dysregulation of the interlinked kallikrein system and the complement pathway can lead to excessive inflammation and tissue swelling 17, features observed in hEDS patients." With this in mind, I sent BioCryst, the company behind berotralstat, a link to your pre-print paper today. Who knows, fingers crossed, Berotralstat may alleviate hEDS symptoms linked to pathological Kallikrein variants. Since it is already an approved medication and on the market, it may help patients in countries where off-label prescribing is not regulated by strict reimbursement rules (it is an expensive medication at $18k per month). Should there be a benefit, such countries would be ideal as hosts for a wider trial.
Novartis also has several patents for Kallikrein serine protease inhibitors, according to a friend who conducted a check. I long for the day when Big Pharma sees hEDS patients as cash-cows, much in the way there are now Janus kinase (JAK) inhibitors used to treat Rheumatoid Arthritis, it is likely that some hEDS patients will need Kallikrein serine protease inhibitors.
Great summary, thank you for taking the time!
I hope MUSC is connecting with biotechs regarding Kallikrein serine protease inhibitors to explore their potential in alleviating hEDS symptoms caused by pathological Kallikrein variants, such as pain, inflammation, and swelling. Incidentally, the biotech company behind Orladeyo® (berotralstat) is located in North Carolina. Berotralstat is the first oral plasma kallikrein inhibitor that is FDA and EMA approved for the prevention of HAE attacks. Your pioneering paper mentions that; "Additionally, in certain pathological conditions such as hereditary angioedema (HAE), dysregulation of the interlinked kallikrein system and the complement pathway can lead to excessive inflammation and tissue swelling 17, features observed in hEDS patients." With this in mind, I sent BioCryst, the company behind berotralstat, a link to your pre-print paper today. Who knows, fingers crossed, Berotralstat may alleviate hEDS symptoms linked to pathological Kallikrein variants. Since it is already an approved medication and on the market, it may help patients in countries where off-label prescribing is not regulated by strict reimbursement rules (it is an expensive medication at $18k per month). Should there be a benefit, such countries would be ideal as hosts for a wider trial.
Novartis also has several patents for Kallikrein serine protease inhibitors, according to a friend who conducted a check. I long for the day when Big Pharma sees hEDS patients as cash-cows, much in the way there are now Janus kinase (JAK) inhibitors used to treat Rheumatoid Arthritis, it is likely that some hEDS patients will need Kallikrein serine protease inhibitors.
This makes it so much easier to understand. Thanks for taking so much time to interpret the preprint.
Thanks so much for this!