The Innovation of AML Therapy: A Look From the 1970s to 2000

If you or one of your loved ones has been affected by acute myeloid leukemia, you might be curious about how treatments have evolved over the years. Let's take a journey from the 1970s to the year 2000 to see how treatments began for patients with AML.

Limited Options From 1970-2000

Treating AML between the 1970s and early 2000s was extremely difficult due to the lack of treatment options available. Very few advancements during these 30 years were made outside the use of traditional “7+3” chemotherapy and the initial approval of an immunotherapy called gemtuzumab ozogamicin, otherwise known as Mylotarg. 

The Start of 7+3

In the 1970s, there was limited understanding of AML and because of this, the mainstay of treatment was a standard chemotherapy regimen that was given to patients regardless of their type of AML. “7+3” refers to a combination of two chemotherapy drugs, a 7-day infusion of cytarabine and a 3-day infusion of daunorubicin. The daunorubicin is given during the first 3 days and the cytarabine is given for the entire 7 days. This regimen was established through a series of clinical trials aimed at refining dosages and schedules to optimize the effects of the two drugs while being mindful of toxicity. 

Unfortunately, these chemotherapy medicines not only target a person’s AML cells, but also their healthy cells which causes side effects such as nausea, hair loss, and serious infections. The use of these drugs makes it challenging for doctors to find the right balance between killing the cancer cells and keeping the patient safe, thus supporting a major need to develop less toxic and better-tolerated AML therapies. 

While 7+3 is still an anchor treatment in AML to this day for many people, doctors have learned a lot about how to manage the toxicities of this treatment and who this treatment benefits the most. "Lower intensity" regimens have also been developed and utilized for those patients who are thought to not be able to tolerate 7+3. 

The First Allogeneic Stem Cell Transplant

The first successful allogeneic stem cell transplant was performed in 1968. Transplantation was further explored during the 1970s, but the procedure remained in its early stages of development and was not as commonly performed as it is today. During this time, transplantation was usually reserved for cases where other treatments had failed, and finding suitable donors was a major challenge. 

Advances for APL in the 1990s

In the 1990s, things slowly started to change for people with a specific type of AML called acute promyelocytic leukemia (APL) which is characterized by a genetic abnormality that causes immature blood cells to accumulate in the bone marrow. APL accounts for 5-10% of AML cases.

A drug called all trans retinoic acid (ATRA) was developed based on a new understanding of the underlying genetic and molecular mechanisms causing APL. Scientists discovered that the abnormal gene responsible for APL, called the PML-RARA fusion gene, could be targeted by ATRA. ATRA works by helping these immature cells mature into normal cells, thereby preventing the accumulation of harmful cells in the bone marrow. On April 5th, 1995, ATRA was granted approval by the U.S. Food and Drug Administration (FDA), a day that marked a pivotal moment where research at the molecular level translated into a targeted therapy that transformed the prognosis for APL patients. This success story illustrates the power of combining genetic knowledge with medical research to create treatments that precisely address the root causes of diseases like AML, a concept that will become much more widely researched in the years to come.

AML in the 2000s

By the time we reached the year 2000, outcomes were slightly less grim. Doctors had begun research on drugs called "targeted therapies" that aimed at the specific molecules causing AML to grow. Unlike chemotherapy, these treatments go after the bad cells while leaving the good ones alone. 

A drug called gemtuzumab ozogamicin or Mylotarg was initially FDA-approved in 2000 but as you will read in the next article, this drug has faced a rocky road. 

The 1970-2000 AML Treatment Journey

The journey from the 1970s to 2000 shows us that drug development for AML was quite the opposite of what it is today. A review of the past reveals that there were no real advancements for non-APL patients for over 20 years. Luckily this has changed, and over the next few days, we will discuss all of the newer developments that are providing cures, hope and extending the lives of those with AML.

Invitation to Attend the Healthtree 2.0 Launch Event on October 23

AML innovation didn't stop in the 2000s, and it's not stopping today! HealthTree is excited about the AML innovation happening today that's accelerating toward a cure. You can be part of this acceleration and innovation! 

Join us for an exciting journey through the history of blood cancer care and the launch of HealthTree 2.0, a revolutionary platform designed to empower AML patients and caregivers.

We invite you to participate in this momentous occasion and make it even more special by hosting a virtual watch party with your friends, family, or support group.

Let's come together to celebrate the progress we've made in AML treatment and explore the promising future that lies ahead.

Register for our virtual launch today!