CAR-T Therapy Trial for Relapsed/Refractory Acute Myeloid Leukemia

A new clinical trial is now open and actively recruiting participants for patients with acute myeloid leukemia (AML) whose disease has returned after previous treatments (relapsed) or no longer responds to standard therapies (refractory).

This trial focuses on a new treatment called CB-012, a type of allogeneic chimeric antigen receptor (CAR-T) cell therapy specifically designed to target a protein on AML cells called C-type lectin-like molecule-1 (CLL-1). 

What is CB-012?

CB-012 is an allogeneic CAR-T cell therapy, meaning that the T cells are from a donor and once ready, infused into the patient. These CAR-T cells are genetically modified using CRISPR technology to target CLL-1, a protein present in AML cells, helping the immune system to specifically recognize and attack cancer cells.

CAR-T cell therapies have been highly successful in treating other blood cancers, but their application in AML has been limited due to the lack of suitable targets. Many common targets are also found on healthy blood cell-producing stem cells (HSCs), increasing the risk of damaging important cells. CLL-1 can be a safer and more effective target because it is present on AML blasts and leukemic stem cells but is not in healthy stem cells, minimizing the risk of undesired adverse effects. 

Another key advantage of allogeneic CAR-T therapy CB-012 is that it allows for the creation of a universal CAR-T product, which can be manufactured in advance and stored for future use. In contrast, most CAR-T therapies available are autologous, meaning that they are made from a patient's own cells, which implies a longer process. Allogeneic CAR-T therapy has the potential to shorten treatment timelines and increase availability for patients.

The AMpLify Trial

The phase 1 AMpLify trial (NCT06128044) aims to assess if CB-012 is safe and if it works for patients who have experienced relapse or have not responded to previous therapies (refractory).

How is CB-012 Tested in the Trial?

The trial will measure several important outcomes, including the rate of side effects related to dose within the first 28 days of receiving CB-012 and the overall response rate (ORR) over the course of 12 months.

1. Part A: Dose Escalation – This phase will evaluate in a small group of patients different doses of the treatment to identify the one that is most effective and safe.different doses of the treatment in a small group of patients to identify the 

Patients in the trial will receive cyclophosphamide and fludarabine before therapy to prepare their bodies for the CAR-T cells.

2. Part B: Dose Expansion – After determining the ideal dose, this phase will test CD-012 in a bigger group of patients. 

Who can participate in the trial? 

To qualify for the trial, patients must have received between one and three previous lines of treatment for AML. This trial can be a great alternative for those who no longer have standard treatment options available.

FDA Fast-Track Designation for CB-012

CB-012 has received Fast Track designation from the U.S. Food and Drug Administration (FDA), allowing a faster review process. This way, as soon as the clinical trials show that CD-012 is safe and effective, it will be available faster for patients with relapsed or refractory AML. 

What are the Advantages of the New CB-012 Therapy?

• Enhancing a targeted approach: CB-012 focuses on the CLL-1 protein, a target associated with AML cells, meaning it is a more specific target to only AML cells.

• CRISPR-edited: allows more precise genetic modifications to the CAR-T cells.

• Less side effects: These edits eliminate the risk of graft-versus-host disease, enhance anti-tumor activity, and help the CAR-T cells have a longer-lasting response.

• Allogeneic option: Since CB-012 is derived from donor cells, it offers the potential for faster treatment, as it would be readily available and doesn’t need to wait to obtain the patient’s own immune cells.

• FDA Fast-Track: This designation helps accelerate the development of CB-012, potentially bringing it to patients sooner if early results prove that patients are responding to the therapy and that it is safe.

If you or someone you know is living with relapsed or refractory AML and has tried unsuccessful standard treatment options, participation in the AMpLify trial may offer access to this promising investigational therapy. Talk to your healthcare team to find out if you are eligible for this trial. 

If you are interested in finding more clinical trials, keeping track of your labs in one place, and discovering treatment options, you can create a free HealthTree Cure Hub account and use all our resources today. 

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Sources: 

• https://clinicaltrials.gov/study/NCT06128044 
• https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.TPS6586 
• https://www.cancernetwork.com/view/cb-012-earns-fda-fast-track-status-in-r-r-acute-myeloid-leukemia