Maybe I’m an idiot, but I added to Kite on the drop yesterday. These are all people who would die in 6 months without the treatment. The treatment apparently works for them. However, one out of 300, who was especially sick, died sooner with the treatment. Wow! Would that change your decision to have the treatment if you were dying? Would it change your doctor’s decision to give it to you?
As for ZIOP, it makes their position so much stronger! They are the only ones who have a rheostat switch. They can turn off the t-cells that can get out of control and cause the cerebral edema. Here’s an explanation paraphrased from the conference call:
“The activated T-cells release cytokine proteins, and the sudden activation of too many T-cells at once can lead to a serious side effect called cytokine release syndrome. Ziopharm’s exclusive license to use the RheoSwitch technology may have the solution to this problem. The Veledimex pill is developed on the RheoSwitch platform to act as a switch to control gene expression. When orally administered, Veledimex in combination with Ad-RTS-IL-12 “starts” the gene expression, and it can be “stopped” by withholding the pill”.
I also added to Ziop yesterday. It was down about 7% too in a knee-jerk reaction. Anyone who understands the issues should have suspected that, if anything, this makes their position stronger.
Car-T treatment isn’t going away because one dying patient out of 300 got cerebral edema! Are you kidding? These companies have selected to work with patients who have no other hope on purpose, and the treatment works. I mean, Glioblastoma!!! NOTHING worked for glioblastoma before this. You have it, boom you’re dead in six months, after awful surgeries and chemotherapies.
“Overreaction” to one death is a huge understatement. These 300 were all going to die without treatment.
“Overreaction” to one death is a huge understatement. These 300 patients were ALL going to die without treatment.
If you need proof, read the conference call. They learned about it in late April. They obviously didn’t consider it a material event that had to be disclosed before the quarterly report. There were plenty of insiders and friends of insiders and doctors and nurses who knew about it. There was NO selloff from people-in-the-know in the last two weeks (the price rose a dollar or two over the two weeks). Nothing was paused. They are still about 2% fatal reactions in a population which had a 100% fatal prognosis. They are speeding ahead with launch plans.
I posted that after reading the conference call transcript. I’m keeping my position and may add a little as you have. Still confident they are moving in the right direction especially with their new combo safety cohort investigation adding the IL-6 inhibitor and Keppra to see if they can prevent some neurological events.
If we panicked after 1 death in 300 patients for all our meds we sure wouldn’t have many treatments available… but that’s just my humble opinion.
What I don’t get is why medicine in general (not just in extreme cases like this) doesn’t look more into what characteristics of patients relate to better response to certain treatments and worse response to others. They might even have been able to tell that the patient that died was too poor a candidate for this experimental treatment.
" What I don’t get is why medicine in general (not just in extreme cases like this) doesn’t look more into what characteristics of patients relate to better response to certain treatments and worse response to others. They might even have been able to tell that the patient that died was too poor a candidate for this experimental treatment."
Ed they do but sometimes it is only in retrospect that you see a pattern emerging and sometimes you need to see a technology or test or marker be born before you can utilize it. An example is patients with colorectal cancer who for years used a drug called Erbitux. It was only later that testing for KRAS mutations became available showing that KRAS mutated patients would not respond to therapy.
In addition the FDA stipulates that only patients with a Performance Status of 0 or 1 (essentially almost normal minimally symptomatic patients) can actually participate in a trial and so a sick weak patient will be excluded from participation precisely because side effects are unknown.
What I don’t get is why medicine in general (not just in extreme cases like this) doesn’t look more into what characteristics of patients relate to better response to certain treatments and worse response to others. They might even have been able to tell that the patient that died was too poor a candidate for this experimental treatment.
There are always certain selection criteria for treatments. There are hundreds of grading systems and scoring systems which predict better or worse responses to various treatment. For less common treatments, it’s hard to find these characteristics because the sample size is too small to draw any meaningful conclusions and there are too many variables to adequately control.