In a remarkable case, the administration of AMPC on patients with acute myeloid leukaemia (AML)–a debilitating blood disorder—has elicited a recovery in normal white blood cell counts after chemotherapy. This holds tremendous potential for establishing AMPC as a possible option for AML treatments. Currently, the most effective treatments for AML involve a combination of chemotherapy and bone marrow transplants that pose a series of inadequacies. Particularly, bone marrow transplants require a compatible donor to be found at a likelihood of 1 to 10,000. Matches are particularly difficult to find due to complications that could arise from introducing foreign material into a person’s body, a condition known as graft-versus-host-disease (GvHD). GvHD manifests when an imperfect donor-recipient match causes the immune cells from the donor to attack cells of the recipient’s body. Due to this phenomenon, immune suppressants are sometimes given to recipient’s of bone marrow transplants. If a patient is fortunate enough to chance upon a compatible donor, they will have to undergo the bone marrow transplant which also involves an invasive surgery, in addition to risk of GvHD.

Due to the many risks and complications involved in conventional bone marrow transplants, ASC is dedicated to providing a better solution for AML treatments. The presently offered treatment provides a solution that does not require a donor or invasive surgeries, nor does it cause a risk of GvHD due to the tissues being sourced from your own body. The past cases of AMPC treatment on patients with AML has provided a better alternative to bone marrow transplants. Most importantly, AMPC are cultured from a patient’s own blood, thereby circumventing the arduous task of finding a compatible donor, as cells from one’s own body will not be rejected by the immune system. The cells also show a remarkable safety profile as no form of genetic manipulation go into the culture process, eliminating the risk of culture-induced cancer risks. AMPC provides a safer and faster means of AML treatment post-chemotherapy, as the method of treatment only involves an intravenous reinfusion of the product, foregoing invasive bone marrow transplants.

In order to further elucidate AMPC’s effects on AML, ASC is now welcoming patients with AML to participate in a study of the product. This enables the technology to further develop and be researched with the eventual goal of replacing the need for bone marrow transplants.

Trial Details

  • Study Description

    This study aims to elucidate the safety and efficacy effects of autologous peripheral blood-derived miltilineage potential cells on patients diagnosed with acute myeloid leukaemia. The stem cells may help replace the abnormal cells in the body to create healthy bone marrow.

  • Eligibility Criteria

    Inclusion Criteria:

    • Must be unequivocally diagnosed with AML according to WHO classification with accompanying bone marrow biopsy and blood panel results
    • Must have refractory AML, defined as disease unresponsive to initial treatment; or relapsed AML that re-occured after treatment with conventional high dose chemotherapy
    • Must have had prior treatment with chemotherapy at least 30 days prior to day 0 of treatment and have recovered from treatment-related toxicity of chemotherapeutic agents with the exception of persistent diseases
    • Age 20 to 30 years old only
    • Negative serology for HIV1/2, hepatitis B/C, HTLVI/II, and syphilis

    Exclusion Criteria:

    • Received any investigational therapies 4 weeks prior to treatment with this protocol
    • Candidates who received radiotherapy within 4 weeks prior to the treatment of this protocol
    • Candidates who have not recovered from any AE caused by radiotherapy or any agents received 4 weeks earlier
    • Candidates who have had a prior allogeneic stem cell transplant
    • Pregnant or breastfeeding women
    • Candidates positive for HIV1/2, hepatitis B/C, HTLVI/II, and syphilis
  • Primary Outcome Measures

    Overall response rate of peripheral blood-derived Autologous Multi-lineage Potential Cells (AMPC) in refractory or relapsed acute myeloid leukaemia (AML) [Time Frame: at day 5 with 12 month follow up]

    Overall response rate is defined as whether the patient achieves complete remission (CR) or complete remission with incomplete blood count recovery (CRi)

    CR Requirements:

    • Bone marrow aspiration shows less than 5% of abnormal blasts
    • Bone marrow biopsy shows no clusters or blast cell collections
    • Normal values for absolute neutrophil count in peripheral blood exceeds 1,000/microL
    • Platelet count in peripheral blood exceeds 100,000/microL
    • Absence of extramedullary AML

    CRi Requirements:

    • All parameters of CR except platelet recovery or neutrophil recovery
    • Incomplete recovery—platelet count is less than 100,000/microL or neutrophil count less than 1,000/microL in peripheral blood
  • Secondary Outcome Measures

    Summary of treatment-emergent adverse events (AE) 60 days after treatment (with 12-month follow up)

    AE is defined as any unintended or undesirable experience that occur during the course of the clinical investigation regardless of whether they are considered to be drug-related

    To determine the pharmacologic effect of AMPC on leukaemic cells [Time Frame: from day 5 to 35]

  • Detailed Description

    Study Drug Administration:

    On day -7 prior to the intervention, medical histories of possible candidates will be examined for suitability for AMPC treatment

    On day -5, the patient will undergo a pathology test for the following test items:

    • Electrolyte/liver function test (E/LFT)
    • Full blood count (FBC)
    • Hepatitis B/C
    • Human T lymphocytic virus type I and II (HTLV-I/II)
    • HIV1/2
    • Syphilis serology
    • Mycoplasma serology

    On day 0, staff will provide candidates with a consent form and agreement for the administration of AMPC. Peripheral blood will then be collected, ranging from 250mL to 400mL depending on candidate fitness. The blood is collected into a sterile blood bag and sealed. Subsequent processes will be conducted in the blood bag within a  closed-system to minimise contamination risks.

    On day 0 to day 3, the collected blood will be sent to the laboratory for stem cell culture, and a sample of the collected blood will be sent to a third-party laboratory for contamination testing of the following parameters:

    • Bacterial endotoxin
    • Total viable aerobic count
    • Total viable count
    • Microbial growth
    • Mycoplasma real-time PCR test

    On day 4, the biotest results will be released and must reflect the safety of the sample taken before the cultured stem cells may be released for treatment

    On day 5, candidates will receive an infusion of the cultured stem cells. Prior to the infusion, the treating doctor will first conduct an allergy skin test to determine suitability for reinfusion. The cultured stem cells are then reinfused intravenously into the candidate in a process that could take up to 2 hours

    The candidates participation will take place on day 0 and day 5 for peripheral blood collection and stem cell reinfusion respectively; with 12 month follow up after treatment

    • 3 days post-treatment follow-up: full blood count test
    • 10 days post-treatment follow-up: full blood count test
    • 1 month post-treatment follow-up: full blood count test with bone marrow aspiration and biopsy
    • 3 month post-treatment follow-up: full blood count test with bone marrow aspiration and biopsy
    • 6 month post-treatment follow-up: full blood count test with bone marrow aspiration and biopsy
    • 12 month post-treatment follow-up: full blood count test with bone marrow aspiration and biopsy
  • Study Arms

    Experimental: Pilot study

    Sample size: 3

    To determine the effects of AMPC AML by monitoring the overall response rate and to observe in the long-term any manifestation of AE

    Experimental: Phase II of private study

    Sample size: 17

    To determine the effects of AMPC on AML by monitoring the overall response rate and to observe in the long-term any manifestation of AE

  • Study Type

    Interventional

  • Lead Organisation

    Autologous Stem Cell Treatment Pty Ltd

  • Cost

    All costs for treatment, product processing, hospital, and doctors fees are fully funded by ASC.

    Note that patients will still require to cover their own travel and accommodation expenses.

Apply for Trial

  • Eligible

    If you meet the above eligibility criteria, please feel free to contact us with the relevant medical records and physician notes.

  • Non-Eligible or Unsure

    If you do not fit the above criteria, or are unsure of eligibility, do also contact us by leaving your contact information and our team will get back to you as soon as we can. Alternatively, you may subscribe to the email list to stay up to date regarding the trial.

  • Subscribe for Trial Update

    Subscribe to the email list to stay up to date regarding the trial.

    Keep me updated on trial progress