"Along with its gene editing therapy Casgevy, Vertex is offering fertility preservation support for its patients—a program that the HHS claims violates anti-kickback statutes."
"Vertex sued the HHS in July 2024 after the department's Office of the Inspector General (OIG) decided not to issue a favorable opinion on the company's fertility support for Casgevy patients. "
Scaevolus · 2h ago
Who do you think should fund studies for new medical treatments?
diffeomorphism · 32m ago
The point is that the reporting has financial ties to the entity funding the studies. Looking at the disclosures form, this seems unproblematic in this case.
In general: funding for studies and funding for critical review should overlap as little as possible.
tiahura · 3h ago
How did someone with severe preexisting dementia consent and get approved for the study?
KnuthIsGod · 3h ago
1. It was a preexisting neurodegenerative condition that may have been worsened by the immunosuppression.
2. It is Vertex. They have a colorful history...
mikequinlan · 5h ago
Yes, cured but…
>But patients in the trial had to stay on drugs to prevent the immune system from destroying the new cells. Suppressing the immune system, he said, increases the risk of infections and, over the long term, can increase the risk of cancer.
>“The argument is this immunosuppression is not as dangerous as what we typically use for kidneys, hearts and lungs, but we won’t know that definitely for many years,” Dr. Hirsch said.
>Patients may have to take the immunosuppressant drugs for the rest of their lives, the Vertex spokeswoman said.
I'm not diabetic, but the trading "existing Type-1 management" for "lifelong immunosuppression" seems bananas. Usually the alternative to immunosuppression is "dying", not "manually and/or pump administered insulin".
No comments yet
etaioinshrdlu · 3h ago
And one of the patients in the study died of meningitis...
gsf_emergency_2 · 3h ago
>allogeneic
The hope is that patient-derived cells will eventually be possible (whether the margins are attractive or not)
gnabgib · 5h ago
Article title: Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes
n=14, 2 died during the study, 10 had insulin independence (the paper does not say cured).
Funding conflict: Funded by Vertex Pharmaceuticals (the makers of the cell–derived islet-cell therapy)
andyfleming · 3h ago
> Two deaths occurred — one caused by cryptococcal meningitis and one by severe dementia with agitation owing to the progression of preexisting neurocognitive impairment.
bix6 · 3h ago
There are better options in development. Immunosuppressants are not something that most T1Ds I know are willing to take.
crawsome · 3h ago
"Cured" seems to be words chosen by OP.
idiotsecant · 2h ago
This sounds like some honest to goodness Nazi mad scientist stuff.
reify · 3h ago
Cured? highly unlikely.
14 participants, 2 died, 10 out of 12 cured.
I thought this was going to be about the wonders of the ketogenic diet on diabetes, not another dangerous fantasy drug.
If the islet cells persist then it's effectively cured. If new cells have to be injected regularly it's still likely to be a very effective treatment.
spacedcowboy · 2h ago
Perhaps, but generally we hope the cure to be better than the affliction. Type 1 is fairly easily managed with insulin pumps and CGM for a closed loop system that almost approximates a functional pancreas.
Taking immuno-suppressants, which often have painful or nauseous side-effects, as well as the elevated health risks of reducing your immune system doesn’t sound like it’s an actual improvement - at least IMHO.
Vertex Pharmaceuticals early investors stand to benefit.
Patients not so much...
"Two deaths occurred — one caused by cryptococcal meningitis and one by severe dementia with agitation"
"Neutropenia was the most common serious adverse event, occurring in 3 participants. "
So two of 12 died..
Another three has significant neutropenia.
Vertex have a bit of a track record:
https://www.biospace.com/policy/hhs-says-vertex-is-grasping-...
"Along with its gene editing therapy Casgevy, Vertex is offering fertility preservation support for its patients—a program that the HHS claims violates anti-kickback statutes."
"Vertex sued the HHS in July 2024 after the department's Office of the Inspector General (OIG) decided not to issue a favorable opinion on the company's fertility support for Casgevy patients. "
In general: funding for studies and funding for critical review should overlap as little as possible.
2. It is Vertex. They have a colorful history...
>But patients in the trial had to stay on drugs to prevent the immune system from destroying the new cells. Suppressing the immune system, he said, increases the risk of infections and, over the long term, can increase the risk of cancer.
>“The argument is this immunosuppression is not as dangerous as what we typically use for kidneys, hearts and lungs, but we won’t know that definitely for many years,” Dr. Hirsch said.
>Patients may have to take the immunosuppressant drugs for the rest of their lives, the Vertex spokeswoman said.
https://www.nytimes.com/2025/06/20/health/diabetes-cure-insu...
No comments yet
The hope is that patient-derived cells will eventually be possible (whether the margins are attractive or not)
n=14, 2 died during the study, 10 had insulin independence (the paper does not say cured).
Funding conflict: Funded by Vertex Pharmaceuticals (the makers of the cell–derived islet-cell therapy)
14 participants, 2 died, 10 out of 12 cured.
I thought this was going to be about the wonders of the ketogenic diet on diabetes, not another dangerous fantasy drug.
https://pmc.ncbi.nlm.nih.gov/articles/PMC10448543/
Taking immuno-suppressants, which often have painful or nauseous side-effects, as well as the elevated health risks of reducing your immune system doesn’t sound like it’s an actual improvement - at least IMHO.