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KnuthIsGod

Funded by Vertex Pharmaceuticals, the company that is trying to sell this treatment...

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. "

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> So two of 12 died..

> Another three has significant neutropenia.

I’m not claiming this company is or is not ethical, or that this treatment is or is not safe, but the existing treatment for diabetes type 1 is annoying for patients, but works well.

Because of that, you can’t, ethically, do phase 1 trials on patients that have both a long life expectancy and for which that treatment is an option.

That, in turn, means it isn’t uncommon for patients to die from causes unrelated to the treatment during clinical trials.

As an example, the subject in an earlier study on this kind of treatment (possibly/likely the same medicine; https://diabetesjournals.org/diabetes/article/71/Supplement_...) was “A 64-year-old male with a 40-year history of T1D complicated by impaired awareness of hypoglycemia with 5 severe hypoglycemic events (SHEs) the year before”.

Nothing in this comment deviates from what I would expect of any pharma trial. What conclusion are you trying to bring us to?

How did someone with severe preexisting dementia consent and get approved for the study?

1. It was a preexisting neurodegenerative condition that may have been worsened by the immunosuppression.

2. It is Vertex. They have a colorful history...

“that may have been worsened by the immunosuppression.”

Fear-mongering bullshit. Why are you this upset about a study you don’t understand, about a condition you don’t understand, using parameters you don’t understand? Solely because it was done by the company that developed the drug?

Good god. Just say you hate science. You’ll have good company in HHS, right now.

Diabetes type 1 is quite well manageable if you have a CGM sensor and inject insulin regularly.

But if a person with dementia tends to peel of sensors, gets aggressive when getting injections etc. this might not work. And an unmanaged diabetes can be deadly.

Not sure how these approvals work in that case, but this groups of people might be the first that can benefit from a treatment like this.

Who do you think should fund studies for new medical treatments?

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.

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