Scientists Discover a Cure for the Deadliest Strain of Tuberculosis
nytimes.com
nytimes.com
I know someone critically ill who was petitioning Otsuka for access to Delamanid under compassionate use, but Otsuka would only agree to give access if the patient stayed in a hospital for 4-6 months. This is just a crafty way for pharma companies to refuse access since they know the cost of months of hospital stay is not affordable or practical. Luckily in this instance, TB Alliance and others fought with Otsuka to get this patient access to the drug.
In many high-need countries (India/Africa/China etc), desperate doctors frequently just lie to Otsuka about the patient staying in the hospital in order to get access to the drug.
Opening access to Pretomanid is a great advancement!
For brand new drugs that cure critical illnesses, the FDA has a compassionate use program (officially called Expanded Access) which allows drugs that are not fully approved yet to be used immediately by very ill patients. Otsuka requiring months of hospitalization to monitor for a rare side effect was just wrong and against the spirit of compassionate use access IMO. This side-effect is easily and cheaply monitored by regular EKGs at a local primary care doctor or urgent care clinic.
They are putting the use of the drug entirely in the hands of people who might not have the resources or training needed. If the use of the drug resulted in several deaths, it could significantly delay approval or kill the program entirely.
I was tangentially involved in a clinical development program that offered access through compassionate use. The folks who received the drug were truly out of options. As a result several died. Investigation determine the drug was not the cause. That didn’t stop the rumors from swirling and several physicians pulling out of the clinical trials and actively discouraging patients from taking part. Probably extended the development by at least 2 years (it was eventually approved) and as a result many people who could of benefited from it had to wait, which likely resulted in a number of deaths that could have been avoided.
I think they're unethical and morally bankrupt.
I really appreciate you adding your personal perspective. A big problem in TB today is that the “western” world sees it mostly as a “not-my-problem” kind of disease. What people forget is that TB is airborne and that the world is getting smaller. It will start impacting everyone.
TB is everyone’s problem and more investment should be made by the wealthier countries toward it’s care.
It's not a drug discovery, it's an FDA approval of a 3 drug combination treatment of existing molecules. It's not a more deadly strain of tuberculosis, per se (to clarify: as in "more virulent", like H5N1 is compared to normally circulating strains of influenza), but it's the "XDR" designation. This is regular tuberculosis that has developed drug resistance to the prior final lines of treatments.
edit: pretomanid is the newest and most novel of the 3 drug treatment. The FDA release is useful: https://www.fda.gov/news-events/press-announcements/fda-appr...
Clickbaity, yes. Accurate, hard to say.
So if very few people die from this strain (and I don't know the answer to that, but the grandparent comment implies that very few do), then I'd say it is definitely not accurate.
[0] "The World Health Organization estimates that 1.8 billion people—close to one quarter of the world's population—are infected with Mycobacterium tuberculosis (M.tb), the bacteria that causes TB. Last year, 10 million fell ill from TB and 1.6 million died." ... "There is growing resistance to available drugs, which means the disease is becoming more deadly and difficult to treat. There were 558,000 cases of drug resistant TB last year."
[0] - https://www.tballiance.org/why-new-tb-drugs/global-pandemic
EDIT: I say surprisingly, because before recently I was under impression that tb, being bacterial infection, was solved long ago.
"By the end of 2016, XDR-TB has been reported by 123 WHO Member States. Information from countries with reliable data suggests that about 6.2% of MDR-TB cases worldwide have XDR-TB.
In 2016, there were an estimated 490 000 new cases of MDR-TB worldwide. Only a small proportion of the XDR-TB cases among them are detected given that many low and lower middle-income countries still lack sufficient diagnostic capacity to test for resistance to second-line drugs and thus detect XDR-TB."
In a study in South Africa, MDR-TB had a case fatality rate of about 9% in HIV- individuals, and 20% in HIV+ individuals.
If so, the headline is more or less accurate.
If not, please explain.
The report estimates around 40% mortality for MDR-TB and 60% mortality for XDR-TB.
This compares with https://www.who.int/gho/tb/epidemic/cases_deaths/en/ which gives about ~13% mortality for all TB cases world wide.
So, I think the headline is accurate about that. However, we could cure (at least for some definition of "cure") XDR-TB before; it just took an incredible amount of time with some terrible drugs and some individuals still relapsed. Pa could be a game changer, though. I'll give it a "somewhat justifiably clickbaity" rating. ;)
I suppose you could also argue it's "not a cure" because in the small trail performed it only cures 90%.
Finally, I don't know what you're getting at with "not a more deadly strain". XDR TB is "eXtensively Drug Resistant" which makes it also the most fatal form of TB. There are a lot of things which left untreated can kill you. I assume it is normal for fatality rates of a strain of disease to be stated as the post-treatment fatality rate.
Pretomanid was developed by the TB Alliance, and is targeted to be offered at a cost of $1 per dose. Back in 2000, apparently they raised about ~$150 million for drug development [1], I'm curious how much more they raised/spent since then to get to this point.
Bedaquiline was developed by Janssen, it's not clear at what cost. They have a tiered pricing structure for a 6-month course ($900/$3,000/$30,000) but 3rd world cost has recently come down to $360. [2]
Linezolid was developed by Pfizer, off patent since 2015. Pfizer doesn't seem to limit the cost in South Africa to any reasonable extent, charging between $60 - $150 per dose depending on who is buying it. However at least generics have come online in pill form for ~$7/dose [3]
It sure would be nice if the development costs from Janssen and Pfizer were public, since it makes it basically impossible to compare, but the development model for pretomanid, and the innovative funding of the trials through TB Alliance to me demonstrates -- at least for diseases which are prevalent enough to build these alliances and obtain the donations -- a truly superior approach to providing treatment options for the world at prices that the world can afford.
Congratulations to everyone involved for reaching such an impressive outcome, and particularly for doing it in an ethically responsible and economical approach. XDR TB is not currently extremely prevalent (seems like < 5% of all TB, but growing), but this is the first new TB drug approved in apparently in 40 years, and changes a 60% fatal diagnosis into a 90% curable disease, with a much simpler treatment protocol.
[1] - https://www.tballiance.org/news/public-private-partnership-a...
[2] - http://www.treatmentactiongroup.org/sites/default/files/real...
[3] - http://www.treatmentactiongroup.org/tb/linezolid-factsheet
https://www.theguardian.com/society/2019/aug/12/chlamydia-va...
If this particular patient asks about this study, and I won't be at all surprised if they do because this is a major article in a major paper and they're pretty well informed about their condition, I would probably tell them that while it looks very compelling I don't want to do this incorrectly and right now the best evidence is the WHO guideline as currently followed. Without going into detail, this patient is already on two of the three meds as it is.
Tuberculosis germs burrow deep into the lungs and barricade themselves inside clumps of dead cells. Breaking those nodules apart and killing all the bacteria inside requires taking drugs for months.