Nav: Home

New treatment of acute myeloid leukemia achieves remarkable results in a disease formerly with little hope

March 20, 2019

The prognosis for older patients with acute myeloid leukemia (AML) is poor: very few achieve remission and for those that don't the option is largely palliative.

Every year almost 1000 Australians die of the disease and clinical trials into new therapies for older patients have largely failed.

A new Australian drug trial has achieved a remarkable result, clearing the bone marrow of leukaemia in almost 60% of patients.

The trial was considered so effective that the US Food and Drug Administration approved its use last November for the treatment of AML.

Kaye Oliver, 74, was the first patient in the world enrolled on this trial at the Alfred Hospital in 2015 - the results of which are published today in the Journal of Clinical Oncology.

Given little hope of survival beyond a few months at diagnosis, Kaye remains well and without evidence of the cancer four years later.

Associate Professor Andrew Wei, from the Alfred Hospital and Monash University Clinical School, commenced research in this area almost two decades ago at the Walter and Eliza Hall Institute of Medical Research. He is now the lead clinician/researcher on the international trial of the cancer drug, currently combined with cytarabine to treat older adults with AML.

Taken separately these drugs achieve little, according to Associate Professor Wei. Venetoclax alone led to a 19% response rate in a US trial and cytarabine had a similar result, he said.

"But combining LDAC with venetoclax in older patients led to a 54% response rate, with half the study population surviving longer than 10 months," he added.

The trial tested 82 patients with a median age of 74 years and was conducted in Australia, Europe and the USA.

The current research is supported by another trial in older AML patients, which combined venetoclax with another drug, azacytidine and led to a 71% remission rate with an average life expectancy of almost 17 months.

Based on the early results of these two studies, the Food and Drug Administration in the US approved the use of these combination drug therapies in older people with AML on November 21 last year.

The drug combination acts on a protein prevalent in leukaemia cells called BCL-2 which controls the survival of the cells. Venetoclax acts by effectively switching off the protein and activating a self-destruct program in the cell.

Associate Professor Wei said that a randomised trial of the therapy, where patients on the therapy are compared to those who are not, has recently been completed and the results are awaited to support a submission to the Therapeutic Goods Association in Australia.

The findings are important not just because of the success of the treatment in a disease that, previously, was fatal, but because with an aging population AML is likely to become more prevalent in the future.

"AML arises due to mutations accumulating in the bone marrow over time. It also arises in patients who have previously had chemotherapy. With an expected doubling in the number of over people over 65 in the next 30 years, the need to find more effective treatments for this disease is paramount," Associate Professor Wei said.

"AML research used to be likened to a 'clinical trial graveyard' because trials of new drugs into AML were rarely successful," Associate Professor Wei said.

"It was widely seen as an untreatable and inevitably fatal condition for older patients by most doctors. These two new trials have given real hope to patients who previously had little."
-end-


Monash University

Related Bone Marrow Articles:

3D atlas of the bone marrow -- in single cell resolution
Stem cells located in the bone marrow generate and control the production of blood and immune cells.
Dangerous bone marrow, organ transplant complication explained
Scientists have discovered the molecular mechanism behind how the common cytomegalovirus can wreak havoc on bone marrow and organ transplant patients, according to a paper published in the journal Cell & Host Microbe.
Viagra shows promise for use in bone marrow transplants
Researchers at UC Santa Cruz have demonstrated a new, rapid method to obtain donor stem cells for bone marrow transplants using a combination of Viagra and a second drug called Plerixafor.
Bone marrow may be the missing piece of the fertility puzzle
A woman's bone marrow may determine her ability to start and sustain a pregnancy, report Yale researchers in PLOS Biology.
Cells that make bone marrow also travel to the womb to help pregnancy
Bone marrow-derived cells play a role in changes to the mouse uterus before and during pregnancy, enabling implantation of the embryo and reducing pregnancy loss, according to research published Sept.
Uncovering secrets of bone marrow cells and how they differentiate
Researchers mapped distinct bone marrow niche populations and their differentiation paths for the bone marrow factory that starts from mesenchymal stromal cells and ends with three types of cells -- fat cells, bone-making cells and cartilage-making cells.
Zebrafish help researchers explore alternatives to bone marrow donation
UC San Diego researchers discover new role for epidermal growth factor receptor in blood stem cell development, a crucial key to being able to generate them in the laboratory, and circumvent the need for bone marrow donation.
New material will allow abandoning bone marrow transplantation
Scientists from the National University of Science and Technology 'MISIS' developed nanomaterial, which will be able to restore the internal structure of bones damaged due to osteoporosis and osteomyelitis.
Blood diseases cured with bone marrow transplant
Doubling the low amount of total body radiation delivered to patients undergoing bone marrow transplants with donor cells that are only 'half-matched' increased the rate of engraftment from only about 50 percent to nearly 100 percent, according to a new study by Johns Hopkins researchers.
Vitamin D and immune cells stimulate bone marrow disease
The bone marrow disease myelofibrosis is stimulated by excessive signaling from vitamin D and immune cells known as macrophages, reveals a Japanese research team.
More Bone Marrow News and Bone Marrow Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Making Amends
What makes a true apology? What does it mean to make amends for past mistakes? This hour, TED speakers explore how repairing the wrongs of the past is the first step toward healing for the future. Guests include historian and preservationist Brent Leggs, law professor Martha Minow, librarian Dawn Wacek, and playwright V (formerly Eve Ensler).
Now Playing: Science for the People

#566 Is Your Gut Leaking?
This week we're busting the human gut wide open with Dr. Alessio Fasano from the Center for Celiac Research and Treatment at Massachusetts General Hospital. Join host Anika Hazra for our discussion separating fact from fiction on the controversial topic of leaky gut syndrome. We cover everything from what causes a leaky gut to interpreting the results of a gut microbiome test! Related links: Center for Celiac Research and Treatment website and their YouTube channel
Now Playing: Radiolab

The Flag and the Fury
How do you actually make change in the world? For 126 years, Mississippi has had the Confederate battle flag on their state flag, and they were the last state in the nation where that emblem remained "officially" flying.  A few days ago, that flag came down. A few days before that, it coming down would have seemed impossible. We dive into the story behind this de-flagging: a journey involving a clash of histories, designs, families, and even cheerleading. This show is a collaboration with OSM Audio. Kiese Laymon's memoir Heavy is here. And the Hospitality Flag webpage is here.