Well done Silviu and Mesoblast but these results are no surprise. Going by the scant information in the public domain, if Temcell wasn't doing well imo there wouldn't be such growth in sales, it wouldn't be considered an attractive therapy and there wouldn't be the suggestion to study it for cGvHD and prophylaxis. I don't recall what I read most recently was specific to paediatrics.
Also, didn't say the company say earlier this year that in the past couple of years they've had access to improved technology?
It's not uncommon for drugs to do well in clinical trials and poorly in clinical practice. Temcell growth is imo a sign of efficacy and a huge plus point for MSB.
If use is extended to adults and chronic GvHD or eventually prophylaxis, revenue from Remestemcel-L could be substantial.
I've been doing a lot of reading and listening to learn about GvHD from a patient's perspective. Chronic GvHD is a much bigger problem than I realised. The reason I think this is that patients and their carers seem to view years of terrible suffering as the norm.
Given these great results, that MSB already have a product doing well in clinical practice, and in the light of what the FDA has seen fit to ram through this past decade, there should be no delay in approval.
Other Potential Applications of Remestemcel-L
SI recently mentioned 'potentially other applications of the technology'. Chronic GvHD has features of autoimmune disease. GvHD being the 'mother of all inflammation' means there are implications for this product in ANY autoimmune disease.
We have a product that can dial down inflammation without the side effects of steroids. The consensus seems now that MSCs start of a process. SI mentioned a two-step process. I don't know about that exactly, but the notion that the body likes to heal in a certain order struck a chord with me; it goes to the heart of what I've observed and the ambiguous feelings I've had about steroids for a long time.
One of the most interesting questions asked imo was that usual practice is trialling in adults then in paediatrics but we're doing it the other way round. If I recall, the reason was what we took over from Osiris. Well, imo that's turned out to be fortuitous and timely.
MSB have found the worst cases respond best to their stem cells. This has paediatric disease written all over it. In children autoimmune illnesses can come on suddenly like a fire storm and in teenage girls it can be particularly aggressive as it's driven by hormones.
It would be a timely entry because mainstream medicine is waking up to the idea that chronic disease in children (who are designed to get better) might be a wrong premise. Dr Suskind from Seattle CH ( one of the centres MSB has a relationship with) recently published a study on the Specific Carbohydrate Diet in IBD. The diet was devised by paediatrician Dr Haas who found that children who followed it strictly for at least a year symptom-free could return to a "regular diet" and the disease never returned. Wolfgang Lutz MD also used dietary therapy to deliver a cure in over 90% of cases of IBD: 5 papers in peer-reviewed journals, a scholarly work (Dismantling a Myth) and he's written to every Crohn's and Colitis society in the world. Borody's work on FMT (I believe Dr Suskind also has research interest in this area) is essentially coming at it from other end.
If IBD is curable, there are implications for other autoimmune diseases too because of the discovery of the importance of the microbiome, but so far the evidence seems anecdotal. There are numerous studies that show that children with autoimmune disorders such as juvenile ideopathic arthritis have more permeable guts. Methotrexate is widely used in RA and also juvenile cases and this drug is known to increase intestinal permeability.
Medicine is pointing the finger at steroids as the most dangerous drug to take long term but there's evidence even a short course can be harmful. A short course of steroids turned a dreadful case of IBD in my immediate family into a hopeless one.
Can steroids Cause Chronic Illness?
I wondered on this forum if steroids were so fast acting they dampen inflammation in the gut but it still churns away at a low level. Robert Canani MD concluded that exclusive nutritional therapy in children with active Crohn's Disease was 'markedly more effective than steroids in producing healing of mucosal inflammation'. I put this into practice a decade ago with the approval of the treating physician, delivering an absolute cure in a case that was beyond hope.
There's a clear connection between the gut and the skin. While you can't watch the gut heal, you can watch the skin, as in the case of Red Skin Syndrome caused by steroids. I don't think this phenomenon is observed much in mainstream medicine because it's too frightening; you'd step in with a high dose of steroids, and so the cycle would continue. But if you have a trusted physician to go over the peer-reviewed literature (Japan and US) and oversee treatment to ensure it's not life-threatening you can watch the body cure itself of a 'chronic' condition.
The skin clearly wants to do what it wants to do and not be interfered with. It heals in the order mentioned in the literature, as if to a template. The thing I struggle to get my head round is that when it's over there's often (but not always) a complete resolution of the severe atopic eczema; there's an integrity and perfection to the skin, not even the slight roughness you get in remissions from steroid creams. The Daily Mail has been running articles on RSS every few months. One young man from the forum (7000 members) has done a lot more research than I or my relative have and said that before steroids were widely used, eczema didn't used to persist into adulthood, the body eventually overcame it. This sounds plausible to me and it needs looking into.
The biologics were supposed to be steroid sparing but it's these two stalwarts (steroids and methotrexate) that are still holding the fort in autoimmune illnesses. If there's one area you'd want to start phasing them out it's paediatrics imo.
One barrier to adoption in autoimmune diseases was imo still the concern MSCs might turn into cancers. I was disheartened recently to learn this perception was still out there among doctors and wrote that on this forum. Then the partnership with Cartherics was announced. MSB's stem cells being used in research to try to cure cancer! Timely again.
"We understand the mechanism" This is important is it comes from Silviu who is understated. He's been consistently ahead of the game in terms of understanding the mechanism, the need for a durable effect (patients are sick to the back teeth of stuff wearing off and pharma can't get away with this any more), even the pronunciation of 'mesenchymal'. I think he's a phenomenal scientist and an impressive CEO.
I think I can see the groundwork that has been put in place for what MSB is quietly becoming.
All IMO. Please feel free to correct any errors as I'm a layperson
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