CD64 CAR T Cell Therapy for Leukemia and MDS

DS
Overseen ByDerek Schatz
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: University of Colorado, Denver
Must be taking: Hypomethylating agents, Venetoclax
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CD64 CAR T cells, a type of immunotherapy, for individuals with certain difficult-to-treat blood cancers, such as acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). The trial aims to determine the treatment's safety and optimal dosage. Interested patients should have AML or MDS that has recurred or not responded to other treatments and must have tried at least one prior treatment. Participants must also have cancer cells with a specific marker (CD64) and be willing to consider potential follow-up treatment if necessary. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Is there any evidence suggesting that CD64 CAR T cell therapy is likely to be safe for humans?

Research shows that CD64 CAR T cell therapy is being tested for safety in treating relapsed or hard-to-treat acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). Previous studies have examined how patients tolerate these modified immune cells. Although detailed safety data from these studies is not yet available, the Phase 1 trial indicates that researchers are still in the early stages of testing safety in humans. Phase 1 trials primarily focus on safety to determine the right dose without causing harmful side effects.

Earlier research has shown that CAR T cell therapies can cause side effects like fever and low blood pressure. However, each new version of CAR T cells, such as the CD64 CAR T, differs, so their side effects might also differ. Prospective participants should know that the main goal is to learn about the safety and optimal dose of this new treatment.12345

Why do researchers think this study treatment might be promising?

Researchers are excited about CD64 CAR T Cell Therapy because it offers a novel approach to treating leukemia and high-risk myelodysplastic syndromes (HR-MDS). Unlike traditional treatments, which often rely on chemotherapy and stem cell transplants, this therapy uses engineered T cells that are designed to specifically target cancer cells expressing the CD64 antigen. This targeted mechanism may improve effectiveness and reduce damage to healthy cells, potentially leading to better outcomes with fewer side effects. The ability of CAR T cells to directly engage and destroy cancer cells represents a promising advance in treatment options for patients with relapsed or refractory disease.

What evidence suggests that CD64 CAR T Cells might be an effective treatment for leukemia and MDS?

Research shows that a new treatment, CD64 CAR T cells, holds promise for fighting leukemia and myelodysplastic syndromes (MDS). In studies with mice, these cells successfully targeted and reduced the growth of harmful leukemia cells, indicating potential effectiveness against acute myeloid leukemia (AML). Participants in this trial with relapsed and/or refractory AML or high-risk MDS will receive CD64 CAR T cells after lymphodepleting chemotherapy. Early results suggest that these CAR T cells can remain active in the body for a long time, which is important for lasting treatment effects. While research in humans continues, this approach offers hope for treating these challenging conditions.12346

Who Is on the Research Team?

MA

Mathew Angelos, MD, PhD

Principal Investigator

University of Colorado, Denver

Are You a Good Fit for This Trial?

Adults over 18 with relapsed or refractory Acute Myeloid Leukemia (AML) or high-risk Myelodysplastic Syndromes (MDS), who have tried at least one therapy including Venetoclax, and whose cancer cells show CD64 expression. Patients must not have a very high white blood cell count or active graft-versus-host disease.

Inclusion Criteria

I am 18 years old or older.
I have been diagnosed with MDS or AML.
My white blood cell count is low enough for apheresis, possibly with hydroxyurea help.
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Lymphodepletion

Participants receive lymphodepleting chemotherapy prior to CD64 CAR T-cell infusion

1 week

Treatment

Participants receive CD64 CAR T-cell infusion and are monitored for safety and efficacy

6 weeks
Weekly visits for monitoring

Follow-up

Participants are monitored for progression-free survival and overall survival

Up to 12 months post-infusion

What Are the Treatments Tested in This Trial?

Interventions

  • CD64 CAR T Cells

Trial Overview

The trial is testing 'CD64 CAR T' cells, which are the patient's own immune cells modified to target leukemia. It's an early-phase study to find out the safest dose and see how well these cells work against AML/MDS that hasn't responded to other treatments.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Relapsed and/or Refractory AML and HR-MDSExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials
1,842
Recruited
3,028,000+

Citations

CD64 CAR T Cell Therapy in Adults With Relapsed and/or ...

This is a Phase 1, open label, dose-escalation study to evaluate the safety, expansion, persistence, and preliminary clinical activity of ...

Is There a Future for CAR-T Therapy in Acute Myeloid ...

Therefore, it has the potential to achieve similar outcomes in R/R AML. However, its effectiveness is hindered by the difficulty of identifying ...

A phase 1 study of anti-CD64 CAR T cells in patients ...

We now propose to demonstrate safety and effectiveness of CART64 in a phase 1, first-in-human, clinical trial in patients with advanced AML and ...

Cellular and immunotherapies for myelodysplastic ...

In this review article, we outline the current landscape of immune and cell therapy-based approaches for patients with myelodysplastic syndromes (MDS).

Paper: A Novel CD64 CAR-T Therapy for the Treatment of ...

We further show that CD64 CAR-T cells target m-LSCs and reduce the engraftment potential of primary AML cells in mice with a nearly 9-fold ...

3416 CD64 CAR-T Therapy Targets Venetoclax-Resistant ...

Acute Myeloid Leukemia (AML) continues to have a poor prognosis despite therapeutic advances. Venetoclax-based therapy induces remissions in ...