"After all, there's no better way to drive physician acceptance than a full dataset, properly peer reviewed unequivocally showing efficacy and safety"
Your faith in physicians is touching. If this rigorous process has been going on then how do you explain the disgraceful situation we have in CHF, IBD and RA?
Take the biologics for example. They're considered so effective they have apparently 'revolutionised' IBD treatment, spawned biosimilars and enriched pharma.
The problem is that my local research doesn't reflect this revolutionary situation. I asked a surgeon if the biologics had prevented or delayed surgery. I didn't get a direct answer. I found a study in the Journal of gastrointestinal Surgery (July '17) which reviewed private insurance claims from a data base of 58,681 patients comparing pre and post biologic era. The authors concluded: "The use of Infliximab for ulcerative colitis has, as expected, increased since its approval, but so has the risk of surgery. Thus, the introduction of biologic therapy has not decreased the risk of surgery for this patient population."
More concerning is that fewer patients are electing colectomy and surgeons are now operating on a pool of much sicker people, which means death rate is higher. I don't have exact figures for Crohn's Disease, but according to James Marion MD of Mount Sinai despite the biologics, surgery rates for CD remain 'stubbornly high'.
If things aren't great in IBD you can bet they're much worse in RA. I asked a rheumatologist about the microbiome who told me the research had 'just come out'.Actually, it had been out at least a year at the time of asking but perhaps this physican didn't want to face it because the writing would be on the wall for first-line favourite Methotrexate as it increases gut permeability. Could this be the reason why RA gets progressively worse? Dr Mcpherson Brown, who pioneered the use of Minocycline in RA, dryly observed that RA often began as 'a lesser affliction'. And how did something as dangerous as Tofacitinib find its way onto the market?
From what I gather this seems to be the mindset: patients are showing up with diseases; drugs are being prescribed and they're getting worse; the conclusion is that here we have a disease that, by its very nature, gets worse. WTF?
I've been reading and hearing about Incredibly Smart Drugs in the pipeline for five years. Last time I checked in eight months ago the most exciting thing in IBD was poo and considering the fierce opposition to this therapy five years ago, I think it's down to desperation. It's even been delivered through naso-gastric tube. How's that for a dog's breakfast?
Where GvHD concerned, I find it strange that someone as smart as you suggested that a child with grade 4 severe intestinal hemorrhaging could be cured by 'dumb luck'. I came across paper by Najima and Ohashi , hematology division Komagome Hospital, Tokyo. The strange thing is that when I read that very well written and researched paper I became doubtful that the Osiris trial even failed. They say it significantly improved response of liver and gut; it was the skin that showed no difference to placebo but it's gut issues that have the highest death rate.
As a layperson, if I was researching Temcell/Prochymal for a relative or close friend these are the questions I would ask:
There are similarities between IBD and GvHD when the gastrointestinal tract is involved. I know with IBD the skin can take much longer to heal after the gut is fully healed (even a year). Did they need a longer period of observation?
Or it simply that the skin is such a large organ and it's hard for the MSCs to target such widespread inflammation?
Is timing the key thing? JR-031 was administered within 48 hours of diagnosis. Is Temcell growth a sign it's been moved up the line?
You and your fellow critics never seem to mention side effects. This is a monumental issue for patients. In 2012 a doctor told me a large study had shown the biologics were 'safe'. I read the same study myself and that wasn't my take. My conclusion was they were no more dangerous than all the other stuff (actually I put them in third place after steroids and the chemo drugs) If you work with something all the time, your perception of danger is reduced.
I'll do a page on social media for IBD with questions patients can ask their doctors about hard data on biologics. How effective have they actually been? Present some zero risk options and suggest patients ask their doctor to at least look into Prochymal/Temcell. If it can handle severe intestinal bleeding then it's a signifcant improvement on Infliximab. Prochymal phase 3 CD candidate never seems to get a mention but if it's successful, IBD is a large and growing market.
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