Pre-interview w/ Dr. M
By David Rice
September 6, 2022
0:00 / 17:43
Pre-interview w/ Dr. M

The type of, leukemia that, Stella had is, not only quite rare. And it only occurs in things is actually, it's rather, it's a new discovery. So we didn't really know about this particular type of leukemia, until really just a few years ago, to a point that.

There was not even a appropriate test to identify. even with sequencing, this was not known. It was not even, really picked up. So when, Casey reached out to me, that, Stella had relapsed after transplant and nothing work working. I actually, I questioned whether, based on her age, And how, she hadn't responded.

So I actually, I questioned whether this could be the type of leukemia we call the CBF or Ram phenotype. And, so we actually went back to the company that originally had sequenced Stella, and that they reported that there was no, fusion CF. I said, can you look at it again, looking specifically for this fusion?

And I went back and looked at, it said, oh yes, here its. So that was actually the first thing we actually were able to, because of our suspicions and cause this was a sort new, glass of AML, if you will. once we, found out that, this is the particularly high risk that. Then the question comes, how do we approach treating Stella?

And this is where there was really there's no, there was no, therapy. There's no established way. All we knew is that with our standard approach, basically none of these patients there very patients and it's only cause they're really. Dealing with graft post disease after transplant, which is, has its own issues. once we've, found that out, then we said we cannot use standard approach with standard chemotherapy and treat this. I know that Casey and, Jed, they were, really very thoughtful folks actually, but we talked to. Pretty much everyone possible, in the country. And, and, based on, really some of the discussions that we had, they, they decided to come to Seattle. one thing is that once, you relapse after bone marrow transplant the disease by the nature of the disease, probably the most intensive therapy that can, so these are highly resistant of refactory, cells. So when they came here, we, tried. several, approaches. One of the things that we did was we actually sent her leukemia cells to be tested against all available drugs.

And no, not only, not actually one of the, surprising finding was that her leukemia not only did not respond to any of that conventional drugs that we usually test for in one case actually even grew better. Presence of it. Antica it's basically was really remarkable. I'd never seen anything like that.

And we also reached outer who, one of the biggest laboratories

at that time, we had looked at what are some of the targets that are expressed on, On Stella's leukemia. And one of the drugs that was expressed, the protein called the plane and, was trying to see any proteins that are expressed that have available drug for. And the frame was something was developed for multiple myeloma and, Actually, I'm sorry, not for melanoma. and, the company that was, had developed that drug for melanoma, they, they agreed to allow us to give that their draw, to Stella. And that was basically. Almost that first in human for AML. So they had tested it in melanoma, but not in AML. So while we were waiting for that, we tried couple of drugs.

We tried the drug called Venetoclax at the time. it was approved in adults, but we had no data. The, so there was a promising block and unfortunately she did not respond to them in, we tried another. Drug, it's called D one inhibitor signal cells that not so unfortunately getting worse to point that she had so much leukemia that.

Liver was completely pulled between our was. And, so finally we were able to deliver, crane. And unfortunately, although, we knew that the target is expressed and we knew that, this drug is supposed. At the very first dose level. We're not even sure if we're delivering enough of that was there.