"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. "
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”.
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.
“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.
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.
>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".
As written above: This might be different for people with dementia or other issues that are not in a mental state to manage their diabetes.
From what I understand from the study the aim is to show that mass producing islet cells from stem cells is possible. Previously those where extracted from pancreases from dead people.
Having cells extracted from your own body has the advantage that there are a lot of they are not rejected by the immune system.
The reason the immune suppression is still needed is the cause of type 1 diabetes: It is a auto immune disease where the body attacks its own islet cells.
But this a specific immune reaction which could be easier to prevent than the generic rejection of cells from a different body. But this is not what this approach is trying to do for now.
This study just wants to show: This approach of creating islet cells work and it is worth trying to do a bigger more expensive study that can produce statistically relevant results.
"Curing" type 1 diabetis is still years off and that requires the immune issue to be solved as well.
> This study just wants to show: This approach of creating islet cells work and it is worth trying to do a bigger more expensive study that can produce statistically relevant results.
It is incredible to me what a hard time people are having understanding this extremely obvious fact.
Knowing this is a possible path to a cure, even if it’s currently undesirable for the majority of patients, is an important step.
> Two deaths occurred — one caused by cryptococcal meningitis and one by severe dementia with agitation owing to the progression of preexisting neurocognitive impairment.
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. "