I've been following GTBP for some time now and have taken a keen interest in their research and the ability to improve the condition of patients who otherwise had run out of therapy options. My father had suffered from acute Leukemia at one point and had to go through extremely invasive chemotherapy, which is why I took an interest in this company specifically. The TriKET therapy provides what thus far, has been a no toxicity alternative, with studies ongoing and results pending.
Research Update
TAMPA, FL / ACCESSWIRE / November 12, 2020 / GT Biopharma, Inc. (OTCQB:GTBP) (GTBP.PA) an immuno-oncology company focused on innovative therapies based on the Company's proprietary NK cell engager (TriKET) technology is pleased to announce its abstract "GTB-3550 TriKET for the Treatment of High-Risk Myelodysplastic Syndromes (MDS) and Refractory/Relapsed Acute Myeloid Leukemia (AML) Safely Drives Natural Killer (NK) Cell Proliferation At Initial Dose Cohorts"
Relapsed/refractory AML and MDS present a clinical challenge. Despite FDA approval of multiple new targeted agents, many patients lack actionable mutations and have exhausted conventional chemotherapeutic options.
Patients with CD33+ malignancies (primary induction failure or relapsed AML with failure of one reinduction attempt or high risk MDS progressed on two lines of therapy) age 18 and older are eligible (NCT03214666). The primary endpoint is to identify the maximum tolerated dose (MTD) of GTB-3550 TriKE. Correlative objectives include the number, phenotype, activation status and function of NK cells and T cells.
To date, 7 patients have been enrolled, two at 5 mcg/kg/day, two at 10 mcg/kg/day, two at 25 mcg/kg/day, and one at 50 mcg/kg/day. Six patients (5-25 mcg/kg/day) have completed therapy to date, with one patient (50mcg/kg/day) entering their third week of therapy. The first patient at 5 mcg/kg/day had stable disease after course 1 of GTB-3550 therapy, and the first patient at 25 mcg/kg/day saw AML blast levels decrease from 18% to 12% by morphological analysis after course 1 of GTB-3550 therapy.
Of particular note, the previous reported MTD of continuous infusion rhIL-15 was 2 mcg/kg/day (Waldmann, TA et al, Clin Cancer Res. (2019) 25:4945-54) was associated with fevers, tachycardia and constitutional symptoms. Validating our pre-clinical data, patients treated with GTB-3550 TriKE displayed no signs of clinical immune activation or serious adverse events (SAEs) at the 5, 10 or 25 mcg/kg/day dose cohorts.
What is Acute Myeloid Leukemia?
AML is a cancer affecting blood and bone marrow, which "acute" versions developing aggressively, where bone marrow produces abnormal white blood cells which crowd out healthy cells: https://rarediseases.info.nih.gov/diseases/12757/acute-myeloid-leukemia
AML is one of the most common types of leukemia among adults over the age of 40, with roughly 20,000 cases annually: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5030376/
What is Myelodysplastic Syndrome?
MDS is a similar group of myeloid disorders characterized by peripheral blood cytopenias and risk of transforming into AML: https://pubmed.ncbi.nlm.nih.gov/29214694/
Essentially, MDS is a class of myeloid disorders, which consist of slow-growing blood cancers in which bone marrow makes too many abnormal red blood cells, white blood cells or platelets, which accumulate in the blood: https://rarediseases.info.nih.gov/diseases/9319/chronic-myeloproliferative-disorders
Myeloid cells are "differentiated descendants" from stem cells in the bone marrow, consisting of granulocytes and monocytes which are cells called upon during a pathogen invasion as part of the body's immune response: https://pubmed.ncbi.nlm.nih.gov/15147715/
*Disclosure: Writer owns stock in GTBP
