New Research Into AML Offers Promising Breakthroughs
- Upon AML diagnosis, doctors try to pinpoint a patient’s genetic subgroup and develop a specialized treatment plan.
- Treatments include chemotherapy, stem cell transplant, and drug therapies that target specific genes and proteins.
- Breakthroughs in combination drug-therapies, including BCL2 protein inhibitors and FLT3 gene inhibitors, are offering dramatically improved responses.
Ephron kept her disease secret for two main reasons, her son later revealed: First, to protect her ability to work. And second, she believed cancer was a “conversation killer” — and nobody loved sparkling dialogue more than Ephron. While she managed a precursor to the disease for several years, when AML emerged in the last six months of her life, she was unable to fight off a final case of pneumonia.
Attacking The Blood Factory
Read MoreAfter Diagnosis: Know Your Subgroup
Crucial to determining the individual treatment path for each patient with AML, is knowing their prognostic subgroup, says Dr. Gail Roboz, director of the leukemia program at Weill Cornell Medical College and NewYork-Presbyterian Hospital, who was Ephron’s oncologist.
After diagnosis, patients should ask their doctor, “which subgroup of AML am I in?” says Dr. Gail Roboz.
Through cytogenetics — a branch of genetics that studies DNA structure — physicians will examine the types of chromosomes within patients in order to identify their AML subgroup. These prognostic subgroups reveal cytogenetic information, molecular genetic information, and gene mutations.
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“In AML, although the majority of patients have normal chromosomes, that is associated with what is termed as an ‘Intermediate prognosis,’ which means that the treatment pathway is going to be different from what we would use for these so-called ‘good prognosis AML subgroups,'” Dr. Roboz says. Treatment plans include chemotherapy, stem cell transplant, and drug therapy.
AML Treatment Options
Some patients can be cured with chemotherapy alone, says Dr.Raoul Tibes, Director, Clinical Leukemia Program, NYU Langone Health. Others may require more aggressive treatment, like a stem-cell transplant. These treatment decisions will be based on the genetic and genomic information, and need to be made early on in the treatment process. "Our goal is always to cure a patient with AML," Dr. Tibes
Dr. Raoul Tibes, Director of the Clinical Leukemia Program at NYU Langone Health tells SurvivorNet that BCL2 inhibitors disable a protein that protects leukemia cells and creating deep and lasting responses in patients with AML.
Patients who are not candidates for chemotherapy and stem-cell transplants often the case, as AML patients tend to be older may opt for lower-intensity treatments, like drug therapies, which may include promising new combination therapies.
Drug Treatment Advances
Dr. Tibes says, cites exciting new developments in drugs that are designed to attack a protective protein — known as BCL2 — found in leukemia cells. These drugs, known as BCL2 inhibitors, target and disable the BCL2 protein, so it can no longer protect the leukemia cells, says Dr. Tibes.
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Another promising category of drugs targets a specific gene — known as FLT3 – which powers leukemia-cell growth. Drugs designed to inhibit the FLT3 gene work to shut it down. "Every physician that treats leukemia is aware of testing patients for the FLT3 gene mutation so, hopefully, in the next months or years, we will see the approval of other FLT3 Inhibitors so physicians have a choice.”
Two currently approved FLT3 inhibitors are Gilteritinib and Midostaurin. For those with newly-diagnosed AML, Midostaurin has been approved for use; while Gilterintinib was recently approved for patients with relapsed and refractory AML.
Combination Drug Therapies
In addition, says Dr. Tibes, ongoing clinical trials of combination drug therapies — that pair Gilterinib with other FLT3 inhibitors — look promising.
In addition to Gilteritinib, Dr. Tibes says the drug Ventecolax, a BCL2 inhibitor, offers another important breakthrough in the treatment of AML. When used in combination with other drugs, such as Decitabine or Azacitidine, Ventecolax offers dramatic improvements in response rates.
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“We almost triple the response rates, which means the absence of leukemia — so we can see of find leukemia in the patient any longer,” says Dr. Tibes. The drugs can be given on an outpatient basis, are well-tolerated, and offer “deep and lasting” response rates, says Dr. Tibes.
While drug therapy does not replace chemotherapy or stem cell transplantation, Dr. Tibes says that combination drug therapy using Ventecolax can offer “deep and lasting” response rates. "I think it will find a quick adoption in clinical practice pretty rapidly," Dr. Tibes says.
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