What the trial was testing
The trial enrolled 336 patients with leukemia. The study was sponsored by Forma Therapeutics, Inc. and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
41% of patients with relapsed blood cancer responded to this new targeted pill.
The Lancet. Haematology · 2023 · NCT02719574
These findings — that responded to the new pill when other treatments had failed — were published in the The Lancet. Haematology and represent the headline result of the study.
Researchers tracked outcomes across 336 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with leukemia, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This was early-stage testing and olutasidenib is not yet FDA-approved for widespread use. If you have acute myeloid leukemia with an IDH1 mutation and other treatments haven't worked, talk to your doctor about whether this treatment might be available through a clinical trial or if there are similar approved options.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open leukemia trials
CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT
The major morbidities of allogeneic hematopoietic stem cell transplant with non-human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life threatening infections. T depletion of the donor hematopoietic stem cell graft is effective in preventing GVHD, but immune reconstitution is slow, increasing the risk of infections. An addback of donor CD45RA (naive T cells) depleted cells may improve immune reconstitution and help decrease the risk of infections.
CIK Cell Therapy for Relapsed or Refractory Acute B-Lymphoblastic Leukemia: Prognostic Impact on Patients With Early CAR-T Cell Dysfunction
This is a single-center, double-blind, randomized trial. Patients with relapsed or refractory acute B-lymphoblastic leukemia(r/r B-ALL) experiencing early functional exhaustion of CAR-T cells will be randomly allocated into three groups: the control cell group, the CIK treatment group, and the messenger RNA(mRNA)-CIK treatment group. The primary objective of the study is to evaluate the prognostic impact of CIK cell therapy on the early functional exhaustion of CAR-T cells in children and adolescent and young adult (AYA) with r/r B-ALL. The primary endpoint of the study is the event-free survival rate of these patient in the CIK cell therapy group.A total number of 213 subjects will be enrolled.