ACUTE MYELOID LEUKEMIA (AML) Archives

Acute myeloid leukemia (AML) is a fast-growing form of cancer of the blood and bone marrow. AML is the most common type of acute leukemia and occurs when the bone marrow begins to make blasts, cells that have not yet completely matured.

Triage Cancer’s Quick Guide to Health Insurance: Employer-Sponsored & Individual Plans

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Triage Cancer’s Quick Guide to Health Insurance: Medicare

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Understanding Clinical Trials: A Jargon Buster Guide

When it comes to cancer treatment you or a loved one may be considering participating in a clinical trial as a treatment option.  Clinical trials are designed to evaluate the safety and effectiveness of a treatment. They may involve researchers administering drugs, taking blood or tissue samples, or checking the progress of patients as they take a treatment according to a study’s protocol.

Learning about clinical trials can be a steep learning curve – not least because the process comes with a lot of new terms, acronyms and jargon.  To help you, I’ve put together this list of the most common terms you will find when you are researching clinical trial information. This is not an exhaustive list but it is a helpful starting point. At the end of this article you will see links to find more information.

Adverse Effects (AE)   

Also called Adverse Events, or Adverse Drug Reaction, AEs are any harmful event experienced by a person while they are having a drug or any other treatment or intervention. In clinical trials, researchers must always report adverse events, regardless of whether or not the event is suspected to be related to or caused by the drug, treatment or intervention.

Arm 

Subsection of people within a study who have a particular intervention.

Bias

Bias is an error that distorts the objectivity of a study. It can arise if a researcher doesn’t adhere to rigorous standards in designing the study, selecting the subjects, administering the treatments, analysing the data, or reporting and interpreting the study results. It can also result from circumstances beyond a researcher’s control, as when there is an uneven distribution of some characteristic between groups as a result of randomization.

Blinding

Blinding is a method of controlling for bias in a study by ensuring that those involved are unable to tell if they are in an intervention or control group so they cannot influence the results. In a single-blind study, patients do not know whether they are receiving the active drug or a placebo. In a double-blind study, neither the patients nor the persons administering the treatments know which patients are receiving the active drug. In a triple-blind study, the patients, clinicians/researchers and the persons evaluating the results do not know which treatment patients had. Whenever blinding is used, there will always be a method in which the treatment can be unblinded in the event that information is required for safety.

Comparator

When a treatment for a specific medical condition already exists, it would be unethical to do a randomized controlled trial that would require some participants to be given an ineffective substitute. In this case, new treatments are tested against the best existing treatment, (i.e. a comparator). The comparator can also be no intervention (for example, best supportive care).

Completed

A trial is considered completed when trial participants are no longer being examined or treated (i.e. no longer in follow-up); the database has been ‘locked’ and records have been archived.

Control

A group of people in a study who do not have the intervention or test being studied. Instead, they may have the standard intervention (sometimes called ‘usual care’) or a dummy intervention (placebo). The results for the control group are compared with those for a group having the intervention being tested. The aim is to check for any differences. The people in the control group should be as similar as possible to those in the intervention group, to make it as easy as possible to detect any effects due to the intervention.

Efficacy

How beneficial a treatment is under ideal conditions (for example, in a laboratory), compared with doing nothing or opting for another type of care. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.

Eligibility Criteria/ Inclusion and Exclusion Criteria

Eligibility criteria ensures patients enrolling in a clinical trial share similar characteristics (e.g. gender, age, medications, disease type and status) so that the results of the study are more likely due to the treatment received rather than other factors.

Follow-up

Observation over a period of time of participants enrolled in a trial to observe changes in health status.

Informed Consent

A process (by means of a written informed consent form) by which a participant voluntarily agrees to take part in a trial, having been informed of the possible benefits, risks and side effects associated with participating in the study.

Intervention

The treatment (e.g., a drug, surgical procedure, or diagnostic test) being researched. The intervention group consists of the study participants that have been randomly assigned to receive the treatment.

Investigator

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator (PI).

Multicentre Trial

A clinical trial conducted according to a single protocol but at more than one site, and therefore, carried out by more than one investigator.

Number needed to treat (NNT)

The average number of patients who need to receive the treatment or other intervention for one of them to get the positive outcome in the time specified.

Outcome Measures

The impact that a test, treatment, or other intervention has on a person, group or population.

Phase I, II, III and IV Studies

Once the safety of a new drug has been demonstrated in tests on animals, it goes through a multi-phase testing process to determine its safety and efficacy in treating human patients. If a drug shows success in one phase, the evaluation moves to the next phase

  • Phase 1 tests a drug on a very small number of healthy volunteers to establish overall safety, identify side effects, and determine the dose levels that are safe and tolerable for humans.
  • Phase II trials test a drug on a small number of people who have the condition the drug is designed to treat. These trials are done to establish what dose range is most effective, and to observe any safety concerns that might arise.
  • Phase III trials test a drug on a large number of people who have the condition the drug is designed to treat. Successful completion of Phase III is the point where the drug is considered ready to be marketed.
  • Phase IV trials can investigate uses of the drug for other conditions, on a broader patient base or for longer term use.

Placebo

A fake (or dummy) treatment given to patients in the control group of a clinical trial.  Placebos are indistinguishable from the actual treatment and used so that the subjects in the control group are unable to tell who is receiving the active drug or treatment. Using placebos prevents bias in judging the effects of the medical intervention being tested.

Population

A group of people with a common link, such as the same medical condition or living in the same area or sharing the same characteristics. The population for a clinical trial is all the people the test or treatment is designed to help.

Protocol

A plan or set of steps that defines how something will be done. Before carrying out a research study, for example, the research protocol sets out what question is to be answered and how information will be collected and analysed.

Randomized Controlled Trial (RCT)

A study in which a number of similar people are randomly assigned to 2 (or more) groups to test a specific drug, treatment or other intervention. One group has the intervention being tested; the other (the comparison or control group) has an alternative intervention, a placebo, or no intervention at all. Participants are assigned to different groups without taking any similarities or differences between them into account. For example, it could involve using a computer-generated random sequence. RCTs are considered the most unbiased way of assessing the outcome of an intervention because each individual has the same chance of having the intervention.

Reliability

The ability to get the same or similar result each time a study is repeated with a different population or group.

Sample

People in a study recruited from part of the study’s target population. If they are recruited in an unbiased way, the results from the sample can be generalised to the target population as a whole.

Subjects

In clinical trials, the people selected to take part are called subjects. The term applies to both those participants receiving the treatment being investigated and to those receiving a placebo or alternate treatment.

Trial Site

The location where trial-related activities are conducted.


References

The Canadian Institutes of Health Research (CIHR)

TROG Cancer Research

ICH.org

NICE

Further Resources

American Society of Clinical Oncology’s Cancer.Net trials site

National Cancer Institute (NCI) Clinical Trials lists open and closed cancer clinical trials sponsored or supported by NCI. 

ClinicalTrials.gov database of privately and publicly funded clinical studies

CenterWatch Clinical Trials Listing

Complete Guide To Mindfulness

Suja JohnkuttyHi there ! I’m Suja Johnkutty, MD a conscientious mom and neurologist . My one simple goal is to provide you honest, practical, simple action steps to experience better relaxation in your life. https://betterrelaxation.com

How Leukemia is Diagnosed

Introduction to Leukemia

Cancer and neoplastic lesions are affecting our lives every day. Nearly 40% of the world’s population is affected by cancer—irrespective of age, gender, and ethnicity. Equally detrimental to cancer’s physical manifestations are the psychological influences. However, medical advancement and new research are helping to to combat this life-threatening disease.

Of all the cancers of the body, the most treacherous is Leukemia. It is a cancer of blood cells. Humans have three kinds of blood cells: red blood cells, white blood cells, and platelets. Leukemia involves the malignant proliferation of white blood cells (WBC).

Our white blood cells are major components of our body’s defense mechanism. They play a vital role in fighting against diseases, whether bacterial, viral or fungal in nature. They originate within the bone marrow, spleen and lymph nodes.

A person suffering from Leukemia has poor white blood cell functioning. WBCs start to divide abnormally eventually outgrowing the normal number of cells.

Leukemia has 4 types:

  1. Acute Myelogenous Leukemia (AML)
  2. Chronic Myelogenous Leukemia (CML)
  3. Acute Lymphocytic Leukemia (ACL)
  4. Chronic Lymphocytic Leukemia (CLL)

1. Acute Myelogenous Leukemia (AML)

Acute Myelogenous Leukemia is a heterogeneous clonal disorder. It is characterized by immature myeloid cells and bone marrow failure. It commonly affects children and adults. Studies have suggested the disease arises from recurrent hematopoietic stem cell genetic alterations.

2. Chronic Myelogenous Leukemia (CML)

Chronic Myeloid Leukemia is a myeloproliferative (slow-growing blood cancer) disorder characterized by the existence of a balanced genetic translocation of chromosomes 22 and 9. It mostly affects adults. CML consists of 3 distinct phases: chronic, accelerated, and blast phases.

The history of patients with CML shows 3-5 years of chronic stage proceeding to a fatal blast phase and then progressing to an accelerated phase.

3. Acute Lymphocytic Leukemia (ALL)

Acute Lymphocytic Leukemia is the second most common Leukemia occurring in adults. Like other Leukemias, ALL’s pathophysiology is also based on chromosomal abnormalities and genetic alterations which happen to take place in differentiation and proliferation of lymphoid precursor cells present in the bone marrow and blood. In adults, the precursors of B- lymphocytes are greater in number than the malignant T- lymphocytes.

4. Chronic Lymphocytic Leukemia (CLL)

Chronic Lymphocytic Leukemia is a tumor of CD5+ B cells that characterizes the deposition of tiny, mature lymphocytes in the blood, bone marrow and lymphoid tissues. Apart from the CD5 cells, other genetic alterations are involved in the pathogenesis of Chronic Lymphocytic Leukemia. Stromal cells, T cells and nurse-like cells in the lymph nodes also predominate.

Causes and risk factors for Leukemia

Although the exact cause of Leukemia is unknown, certain risk factors can contribute to making a person susceptible to it. These include radiation, viruses, exposure to benzene, smoking, genetics, and family history.

1. Ionizing radiations

Exposure to ionizing radiation comes from continuous radiation therapy for treating any pre-existing cancer. Prolonged exposure to X-rays is found mostly in people who work as radiologists and are exposed to persistent radiation. Patients who have received chemotherapy sessions for cancers are also prone to Leukemia. Ionizing radiations damage the DNA and result in the defective genetic makeup of stem cells.

2. Viruses

The Human T-lymphotropic Virus (HTLV-1) has been shown to have an association with Leukemia.

3. Exposure to benzene

Benzene is a toxic solvent used in cleaning chemicals and some hair dyes. Benzene’s toxic effects on the blood and bone marrow include increasing the risk of Acute Myeloid Leukemia (AML), myelodysplastic syndrome, and other hematological malignancies, such as non-Hodgkin’s lymphoma.

4. Smoking

Smoking is not only detrimental for the lungs alone but for the entire body. Although the link between smoking and Leukemia is unclear, studies say it can affect the bone marrow and increase the chances of AML in young adults.

5. Genetic conditions

Chromosomal abnormalities are also responsible for increasing Leukemia susceptibility. Examples include Down syndrome, Klinefelter syndrome, Fanconi anemia, Li-Fraumeni syndrome, Bloom syndrome, Ataxia-telangiectasia, and neurofibromatosis, to name a few.

6. Hereditary

The most common cause of Leukemia is family history. If any family member has had Leukemia it increases the risk for other blood relatives.

Signs and symptoms of Leukemia

The signs and symptoms of Leukemia vary with different forms. They are generally nonspecific and warrant investigations for proper diagnosis.

Acute Myeloid Leukemia (AML)

The signs and symptoms of AML are:

  • Fever
  • Pain in bones and joints
  • Pale skin
  • Easy bruising and contusions
  • Recurrent infections
  • Unusual bleeding, epistaxis, bleeding gums

Chronic Myelogenous Leukemia (CML)

The signs and symptoms of CML are:

  • Fatigue and muscle weakness
  • Shortness of breath
  • Pyrexia
  • Increased sweating mostly during the night
  • Cachexia (weight loss)
  • Abdominal discomfort secondary to spleen enlargement
  • Stomach bloating
  • Itching
  • Pain in joints and bone

Acute Lymphocytic Leukemia (ALL)

The signs and symptoms of ALL are:

  • Joint pain and muscle fatigue
  • Fever
  • Frequent infections
  • Epistaxis (Nose bleed)
  • Lumps felt around the neck, groin and underarms as a result of lymph node swelling.
  • Pale skin
  • Shortness of breath

Chronic Lymphocytic Leukemia (CLL)

The signs and symptoms of CLL are:

  • Nocturnal sweating
  • Fever
  • Recurrent infections
  • Fatigue and constant tiredness
  • Cachexia
  • Loss of appetite
  • Stomach bloating as a result of splenomegaly (enlarged spleen)
  • Shortness of breath
  • Pea-sized swelling or lumps in groin, neck or armpits.

Diagnosis of Leukemia

Early detection can prevent complications. The earlier the diagnosis the easier treatment is. Medical advancement has made diagnosis easier than ever before. Some of the essentials to reach an accurate and precise diagnosis are enlisted below.

History and examination

A proper and detailed history is the key to an ideal diagnosis. It involves asking relevant questions related to the signs and symptoms that can link to the suspected disease.

Your physician might ask the following questions:

  1. How long have you been feeling a fever?
  2. What is the temperature?
  3. Do you feel a loss of appetite?
  4. Have you experienced prolonged bleeding after a cut?
  5. Have you noticed any changes in weight recently?
  6. When did you notice lumps?
  7. Are these lumps felt, painful and movable?
  8. Did you feel the lumps gradually increasing in size?
  9. Do you face difficulty in breathing?
  10. Do you feel you sweat a lot while sleeping?
  11. Are you taking any medications?
  12. Have any of your family members had any diseases?
  13. When did you feel the need to visit the physician?

The answer to the above questions can lead to the next step, investigations.

Investigations for Leukemia

Investigations are the major component involved in diagnosis as they make the suspected disease clear to understand. These include blood tests, radiology and biopsy.

1. Blood tests

  • Complete Blood Count (CBC)
  • White Blood Cell (WBC) differential
  • Blood smear
  • Tumor markers
  • Cerebrospinal fluid (CSF) analysis
  • BCR ABL1
  • Genetic tests for targeted cancer therapy
  • Chromosome analysis

The most commonly used test is the Complete Blood Count (CBC) which shows a clear picture of the abnormal growth of red blood cells, white blood cells and platelets.

2. Radiology

The excessive proliferation of the cells in the bone marrow leads to marrow expansion and invasion of the cortex which, in later stages, can be seen by radiographic studies. A simple X-ray can reveal any spot bone changes. In some circumstances, a CT-scan may be needed to extensively study the disease and prognosis.

X-ray findings may include:

  • Osteolytic lesions; most commonly seen in Acute Lymphocytic Leukemia seen in small and flat bones, metaphysis of long bones.
  • Metaphyseal bands (classical Leukemia lines)
  • Bone destruction
  • Some pathological fractures
  • Radiological lesions, in later cases, are seen in the form of vertebral collapse, osteolysis of bones and avascular osteonecrosis.

3. Bone marrow biopsy

This is the gold standard investigation to diagnose Leukemia. This invasive procedure is done after the suspicion of Leukemia or when the blood test reports point to a Leukemic picture.

The procedure involves the removal of a small sample of bone marrow from the hipbone. A long, thin is the needle is used to extract the bone marrow. Once the sample has been taken it is sent to the laboratory where the histopathologists study the tissue microscopically.

Prior to examining the histopathologist or the lab technician prepares a slide. During the process, the specimen is then cut into thin slices. The sectioned structure is dyed using different dyes. The dye discriminates against the parts of the cells. The section is then placed on a glass slide and then covered with a slip on the top to keep the specimen intact. The slide is now ready to be placed under the microscope.

The samples examined under the microscope are then studied based on the type of cells, how the cells are arranged, whether the cells are normal or abnormal etc.

The microscopic findings may reveal:

  • Acute Myeloid Leukemia

Increase in bone marrow cellularity, consisting of granulocytic or monocytic forms a number of erythroid precursors

  • Acute Lymphocytic Leukemia

Hypercellular bone marrow with multiple tightly packed lymphoblasts that have undetectable cytoplasm, round irregular shape, divided nuclei, and dispersed chromatin. The bone marrow has B and T lymphoblasts with indistinguishable morphology along with necrosis in some areas.

Treatment for Leukemia

Treating Leukemia challenges all medical practitioners. Its success and failure solely depend on the extent of the disease and how far has it spread within the body.

Following are treatment options that can help fight Leukemia.

1. Chemotherapy

Chemotherapy is the use of anticancer drugs to kill or halt the proliferation of cancer cells. Generally, chemotherapy is administered orally or intravenously. In some patients, the chemotherapeutic agent is given intrathecally, i.e., injected into the CSF (cerebrospinal fluid) that bathes the brain and spinal cord. This is done after performing a lumbar puncture and injecting chemotherapeutic drugs, such as methotrexate. The course is usually repeated every three weeks.

2. Radiotherapy

Compared to chemotherapy that attacks all the cells of the body, including the healthy ones, radiotherapy is a localized treatment regimen. High ionizing-energy radiation emits to destroy cells that have an increased proliferation rate. Radiotherapy can either be given to cure the disease (therapeutic) or to improve the signs and symptoms encountered during the disease course (palliative).

3. Stem cell or bone marrow transplantation

Transplants are widely used in management and treatment of the disease. Bone marrow is transplanted from a patients’ family member, or another person who bears the same type of bone marrow, into the diseased person. The survival rates vary with different factors but the cost and affordability remain the major concern in this treatment modality.

4. Immune therapy

Immune therapy is another set of treatments that has some promising result in managing Leukemia. The immune therapy works by promoting the immune cells of the body to fight against cancer cells. One of the successful regimens in immune therapy is Gleevec, commonly given in Chronic Myeloid Leukemia. CML patients live a long, symptomless life with the daily oral administration of this drug.

Complications with Leukemia

Though Leukemia is curable, treatments may give rise to certain complications–basically the body’s response to the treatment given. The complications mostly arise from chemo and radiation.

They can include:

  • Anemia
  • Skin rashes
  • Altered taste sensations
  • Soreness of the mouth and throat
  • Liver dysfunction
  • Hair loss
  • Diarrhea
  • Fatigue
  • Bleeding
  • Fertility problems
  • Nausea and vomiting
  • Neurological effects
  • Impaired sexual activity

Relapse of the disease may also occur after some years.

Conclusion

The prognosis of Leukemia depends on how far has the disease has spread but the medical advancement has brought in new regimens that can now treat Leukemia at any stage. A person suffering from Leukemia and undergoing its treatment should be dealt with love, care, and pampering

How to help prevent Leukemia

Informational campaigns and awareness programs help people learn about the risk factors of Leukemia. Family members of Leukemia patients should undergo blood screenings to see if they have been affected. A good diet can help improve health status. Limiting use of benzene-infused chemicals can also make the disease less susceptible. Ceasing tobacco smoking can also help keep Leukemia at bay.

 

References

Acute Lymphoblastic Leukemia

Leukemia: an overview for primary care

Acute Myeloid Leukemia: diagnosis and management based on current molecular genetics approach

Treatment of acute myeloid leukemia

Clonal hematopoiesis and preleukemia-genetics, biology, and clinical implications

No free rides: management of toxicities of novel immunotherapies in ALL, including financial

Adult Acute Myeloid Leukemia Treatment

Acute Lymphocytic Leukemia

Genetics and prognosis of ALL in children vs adults

Etiology of leukemia. A review

Symptoms of adult chronic and acute leukaemia before diagnosis: large primary care case-control studies using electronic records

Acute lymphoblastic leukemia: a comprehensive review and 2017 update

Acute myeloid leukaemia: optimal management and recent developments

Overall Health and Mindfulness Improves Treatment Response: An Expert Explains

Overall Health and Mindfulness Improves Treatment Response: An Expert Explains from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee shares the benefits of meditation and yoga and explains how mindfulness can affect your overall health.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

How about this one? A positive attitude and mindfulness can improve treatment response.

Dr. Lee:

Absolutely. Absolutely. Treatment for leukemia can be tough. Some of the treatment involves intense chemotherapy. Treatment for leukemia can involve stem cell transplant. And a key important aspect of treatment is being healthy and being optimistic about treatment, because a lot of treatment can have side effects, and side effects can be not as apparent if you are physically more active, and in a good state. So, I think that having a positive outlook is very, very important.

Patricia:

Quality of life issues are difficult for some people. How do you talk with your patients about their quality of life, and staying healthy during their treatment?

Dr. Lee:

So, quality of life is absolutely important. I mean, the whole point of treating leukemia and any other treatment is not only to address the leukemia, but also have good quality of life. So, when discussing treatment options, you always have to balance the quality of life and side effects versus potential benefits. So, that’s always on our mind when discussing potential treatment options, and how it impacts the quality of life. Throughout the treatment process, we always tell our patients that being active, and having a good quality of life, and having good nutrition, is absolutely important, because that’s a key aspect of treatment for leukemia.

Patricia:

What about meditation and yoga for coping with anxiety around cancer diagnosis and treatment? Mindfulness.

Dr. Lee:

Absolutely, absolutely. Those can help. Especially having leukemia, it’s very life-changing, so a typical way that patients are diagnosed with acute leukemia is patients live a normal life, and then they develop, all of a sudden, abnormalities. And they’re diagnosed with acute leukemia, and it can be very sudden. And it can be very difficult. So, that can understandably make patients have anxiety, and other issues.

And I believe that meditation, and yoga, and other exercises can absolutely help cope with this.

Patricia:

And there’s tons of resources for meditation and yoga out there, that are reliable.

Dr. Lee:

Yes. Yeah.

Patricia:

Yeah. Should patients regard yoga and meditation as part of their treatment, as part of their self-care, during this process?

Dr. Lee:

Absolutely, absolutely, if the patients are into meditation and yoga. Meditation is very harmless, and it can absolutely help in terms of guiding their mind through their treatment journey. Yoga is good if you’re physically able to do it. So, one caution is that, if you’re not someone who does yoga normally, then you should start off slow, and not push yourself as aggressively.

Does Cannabis Oil Have a Role in Cancer Treatment?

Does Cannabis Oil Have a Role in Cancer Treatment? from Patient Empowerment Network on Vimeo.

Is it just a trend or could cannabis oil truly have a role in cancer care and treatment? Dr. Sangmin Lee share his perspective.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

The use of cannabis oil is becoming prevalent. Does this have a role in cancer care and treatment?

Dr. Lee:

Absolutely. So, we use it for a lot of side effect management. So, cannabis can be helpful, in terms of appetite and nausea, for example. So, we often use it in conjunction to manage some of the side effects that patients can have throughout their treatment.

You should consult with your medical team, and of course, I should say that laws differ state by state, so it doesn’t apply to every state. But when it’s available, it can be a valuable addition.

Patricia:

Sure. Discuss that with your physician.

Sugar Feeds Cancer: Fact or Fiction?

Sugar Feeds Cancer: Fact or Fiction? from Patient Empowerment Network on Vimeo.

Does sugar feed cancer? Dr. Sangmin Lee addresses the rumored connection between sugar and cancer.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

Okay, a little more fact or fiction, here. This is what we’ve heard from patients who have AML about cures, okay? Sugar feeds cancer, and severely restricting my diet will treat my AML.

Dr. Lee:

That’s not proven so far. There are some laboratory studies, especially with keto diets, showing some promise, maybe. But then it hasn’t been proven in humans, yet. The most important thing about AML treatment is actually nutrition. As patients go through AML treatment, it’s very important to stay healthy, and part of that is nutrition.

So, starvation, in general, is not recommended, because nutrition is so important, in terms of being able to undergo the treatment, as well as treatment visits, and everything. So, we recommend that nutrition is very important.

Are Clinical Trial Participants Monitored More Closely?

Are Clinical Trial Participants Monitored More Closely? from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee discusses the monitoring of clinical trial participants and the measures taken for patient safety.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

How about this next one? I am monitored more closely in a clinical trial.

Dr. Lee:

In some cases, it’s true. Clinical trials do have certain monitoring visits, in terms of doctor’s visits, laboratory tests, and physical exams.

The purpose of that is to make sure that it is safe. So, the purpose of monitoring closely, in a lot of cases, is for the patient’s safety. We are testing drugs in a lot of clinical trials, for which the complete safety profile, as well as efficacy profile, is not known. So, the purpose of closer monitoring is to make sure whatever we’re doing is safe, and if there are any unexpected side effects, then it allows us to address the side effects, as well. So, it’s mainly for patients’ safety.

Will Clinical Trials Cost You? The Facts.

Will Clinical Trials Cost You? The Facts. from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee reviews the financial impact associated with clinical trials, including a discussion of what expenses are covered for participants.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

All right, how about this one: I may have unexpected costs if I join a clinical trial.

Dr. Lee:

So, typically, that’s actually, usually not true, because how it works is that the clinical trial drugs, and that there may be extra procedures or visits associated with clinical trials.

And what usually happens is that the sponsor of the clinical trial provides the cost of the drug, intervention, and anything extra that are required for the clinical trial. So, in the end, the cost of participating in a clinical trial should not be any more than receiving standard care treatment.

In some rare cases, there may be stipends associated with the clinical trial, especially with travel. So, if you participate in a clinical trial, and you live far away, then you should ask to see if there is any stipends available, especially for travel.

The Truth About Clinical Trials in AML

The Truth About Clinical Trials in AML from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee reviews common misconceptions about clinical trials and shares examples of how these studies are changing the landscape of AML treatment.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:                      

I’ll tell you a few things that we’ve heard from AML patients, and you tell me if this is fact or fiction. Okay, Dr. Lee?

Dr. Lee:

Yeah.

Patricia:

Clinical trials are the last resort treatment option.

Dr. Lee:

That’s actually not true in a lot of cases, because, yes, there are a lot of clinical trials after you have tried all of the standard therapy. But then, standard therapy in AML, like any other condition, are not perfect. So, there are many clinical trials where, even if you’re diagnosed with new AML, because standard care is not perfect, there are trials to try to improve upon the standard of care.

So, there may be clinical trials when you’re first diagnosed with AML, as well.

Patricia:

Yeah. How about this one: I feel like a guinea pig.

Dr. Lee:

Well, the clinical trial is to test drugs in humans. So, in a way, you are a test subject. But then, you have to remember that all of the drugs that we are testing have a rationale.

They all show promise, in terms of laboratory testing to kill leukemia cells in the test tube. And the problem is that, just because they are killed leukemia cells in the test tube, or in an animal model, doesn’t actually mean that it works in humans, or we know the safety profile. So, we need to do these testings to demonstrate that these drugs, which seem promising, actually work in humans.

Patricia:

Right. Well, then, that’s a good segue to this thing we’ve heard: Treatments being studied today may be the future standard of care.

Dr. Lee:

That is absolutely true, because all of the new developments that have come out, including Venetoclax, or IDH inhibitors, or other inhibitors, that are approved today, came through the clinical trial process. One example I like to include is a patient of mine, who, five years ago, had very, very aggressive leukemia, and she happened to have an IDH2 mutation.

It was four or five years ago. And she has a very refractory, aggressive leukemia, and it was life-threatening. And she had an IDH2 mutation. And we enrolled her in a clinical trial involving ivosidenib, which was in clinical trial at the time.

Ever since then, she became – she went into remission, and she has a normal blood count. And, to this day, she’s on this medicine, which is now approved, and she remains healthy with a normal blood count, in remission. So, yes. Clinical trials do include promising drugs, and if they show really good efficacy and promise, they will become standard of care down the road.

The advantage of clinical trial is that you may get early access to drugs that may become standard care down the road. So, it’s a way to get early access to potentially promising drugs.

Patricia:                      

How do you counsel your patients about joining clinical trials? What are you thinking about when you’re talking with them?

Dr. Lee:                       

So, in terms of clinical trials, we all look at clinical trials, and they exist for a reason, because we think that an intervention or drug can do better than standard of care. So, how I approach it is that, depending on the situation, if we can improve upon what is available, or if there are no other options, then it definitely is a great option to improve upon what would otherwise be standard.

Patricia:                      

I’ve got one more. Once I enroll, I am locked into the trial, and I can’t change course.

Dr. Lee: 

Absolutely not true. So, clinical trial participation is always voluntary. So, if you sign a clinical – So, what happens is you typically sign a consent to participate in a clinical trial.

And if you change your mind at any time, you can decide not to participate in a clinical trial. It’s not a binding agreement, so you can decide not to participate at any time.

Patricia:

Great. And that’s obviously a decision you should make with your healthcare provider before withdrawing.

Dr. Lee: 

Oh, absolutely. Absolutely, absolutely. But you should always remember that just because you sign up for a clinical trial, it’s not a binding requirement to stay on it.

Patricia:                      

Okay, okay. And let’s talk for just one moment, if you have a second again, about why patients – why it’s important for patients to participate in clinical trials.

Dr. Lee:                       

Why it’s important? It’s because the drugs we test could become the standard care in a few years. And you might have early access to a promising drug that may change treatment of AML. One prime example is Venetoclax. Venetoclax, when it was in clinical trial, was very promising, but before we started treatment, we had no idea how well it was going to work.

So, the patients receiving Venetoclax obviously benefitted from it, and they had early access to a drug that would have become standard of care a few years down the road.

Is It Safe? Breaking Down the Clinical Trial Process

Is It Safe? Breaking Down the Clinical Trial Process from Patient Empowerment Network on Vimeo.

The idea of a clinical trial can be intimidating and confusing for many patients. Dr. Sangmin Lee explains the phases of clinical trials, including the safety protocols in place to protect patients.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

What is the process for getting medicine to patients during clinical trials?

Dr. Lee:

So, clinical trials are basically what’s needed to prove that drugs work. So, a lot of times, we test drugs in a test tube in AML cell lines, and they show great promise. But just because a drug works in a test tube setting, doesn’t actually mean that it will work in humans, because human bodies are much more complicated. So, we need to test promising drugs in humans to make sure they are safe and effective.

And that’s what the purpose of clinical trials are. Once they demonstrate safety and efficacy, then a drug then gets to be approved, and is available commercially. So, that’s the purpose of clinical trials.

To be involved in clinical trials, what it involves is, basically, you have to meet a sort of criteria, called eligibility, because different clinical trials have different criteria for selection. So, we have to look into that. And then, once you fit an eligibility or selection criteria, then you typically undergo certain diagnostic tests to enroll on a clinical study. And then, you get whatever drug or intervention that is designed to test in that setting.

So, there are numerous steps to actually enroll in a clinical study.

Patricia:

And like you mentioned, there’s a long way between rat studies and human trials. What are the phases of clinical trials?

Dr. Lee:

So, there are three phases for clinical trials, commonly. There’s phase one, and phase two, and phase three. Phase one is the earliest part of the clinical trial process. So, goal of a phase one study is to make sure a drug is safe in a human. So, phase one studies are usually the first time that you are testing the drug in humans, and the main purpose is to demonstrate that it’s safe. So, typically, in a phase one study, typically, you test a drug at a lower dose or dose levels to demonstrate safety. What it means is that you’re enrolling a few patients at a time.

Once a drug is proven to be safe, then you move on to phase two, which is basically testing the drug in more patients. And the purpose of phase two is to get a preliminary assessment of how effective a treatment would be.

So, typically, a phase two study involves many more patients in that setting. And then, if a phase two study shows that a drug is very promising, then the drug may move on to phase three, where, basically, in phrase three, you are comparing one intervention or a drug compared to the standard of care. And, typically, in a phase three setting, a computer decides randomly which intervention you get, whether it’s an intervention or new drug versus standard of care. And standard of care may include either placebo or chemotherapy intervention, that is standard of care. So, it’s not always placebo in phase three.

AML Treatment Advances: What’s New for YOU?

AML Treatment Advances: What’s New for YOU? from Patient Empowerment Network on Vimeo.

 AML specialist and researcher, Dr. Sangmin Lee, breaks down the recent advances in AML treatment and how targeted therapies are improving patient care.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

Dr. Lee, thanks so much for joining us, again. Can you please give us an overview of the field of AML research?

Dr. Lee:

So, AML research is evolving very rapidly, and there’s a lot of promising new drugs that have come out in AML. As with any other cancers, we’re getting more sophisticated in characterizing AML in terms of molecular mutations, and characterizing AML stem cells, so –

The field is moving very rapidly in that regard. There have been a number of promising and effective drugs that have been approved in the last few years, as well. For example, Venetoclax has been a game changer in treatment of AML, especially in the elderly population. And there are several targeted agents that have been FDA approved in the recent years, as well. So, definitely since about three to five years ago, there have been new drugs that have come out for AML that is very exciting for treatment of AML.

Patricia:

Let’s talk a little bit about genetic testing. How is that changing the landscape for AML patients?

Dr. Lee:

So, genetic testing has become standard in AML patients, in terms of – at their diagnosis and relapse. And part of that is, we can use that information to guide prognosis, how well or not well a patient is expected to do.

But more importantly, there are actually drugs that can target specific mutations. For example, there are new drugs that target a mutation called IDH1 and 2 that have been approved recently for patients with AML, as well as new drugs that target mutation called FLT-3, or FLT3 mutation, as well. So, genetic testing has become standard, not only to tell you prognostic information, but also used in therapy for AML patients.

Patricia:

You mentioned a few treatments that were advancing. What other therapies are showing promise for AML?

Dr. Lee:

So, there are a number of treatments that are ongoing. Venetoclax has been game-changing, and now, although Venetoclax has improved outlook, in terms of AML treatment, compared to conventional therapy, there’s still resistance to Venetoclax and the response is not durable.

So, there is research looking at resistance mechanisms to Venetoclax, for example. The other exciting field is, there are some advances in immunotherapy, with clinical trials underway. Like in other malignancies, there are clinical trials involving CAR T-cell, or other ways of engaging your own T cell immune system to approach and attack the AML.

AML Research: What’s New in Treatment?

AML Research: What’s New in Treatment? from Patient Empowerment Network on Vimeo.

 AML expert, Dr. Jessica Altman, discusses the future of AML research, and new learnings that continue to improve current treatment approaches.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

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Transcript:

Patricia:            

Are there any new treatments on the horizon that you can talk about, Dr. Altman?

Dr. Altman: 

Absolutely. So, I love to talk about new therapies in AML. Until the last couple of years – it had been 40 years since we approved a sustained treatment in the marketplace in AML. We had been treating the disease the same. And over the last couple of years there have been a growth of therapies. We’re now trying to sort out exactly when we’re using one over another. We also have clinical trials where we’re combining novel therapies for adults with either newly diagnosed disease or relapsed and refractory disease. 

We are in an era of looking out at antibody therapy in AML – that’s one of the new waves of treatment. We are still exploring targeting therapies in the sense of inhibition of FLT3, IDH, and other mutations. So, it’s an era where there’s lots of excitement, and I’m hopeful for our patients.

Patricia:     

Yeah. Tell me what makes you most hopeful about the future of research in this area, and treatment?

Dr. Altman: 

So, I think that’s a great question. I think the fact that we now – the deeper the understanding we have of the biology of the AML, why AML happens, what mutations drive the disease, and then how to target those mutations with individual therapies is what excites me the most. So, our basic science research has exploded, and that occurs at a very quick pace, and that’s allowing us to develop therapies at a much faster rate than I would have anticipated before.

Patricia:

What a wonderful way to end our chat. Thank you so much, Dr. Altman, for taking the time to join us today.

Dr. Altman: 

It’s a pleasure to be here. Thank you so much.

Misconceptions in Clinical Trials: What’s Fact and What’s Fiction?

Misconceptions in Clinical Trials: What’s Fact and What’s Fiction? from Patient Empowerment Network on Vimeo.

AML expert, Dr. Jessica Altman, addresses common misconceptions patients have about clinical trials regarding treatments, regulations, and standards of care.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

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Transcript:

Patricia:            

What about clinical trials? What common misconceptions do patients have about enrolling in trials?

Dr. Altman: 

So, I think the misconceptions regarding clinical trials can be very masked. And I think it really depends on the intent of a clinical trial and the phase of the clinical trial. I think that a well-designed clinical trial is almost always the right choice for a patient with acute leukemia at any stage in their therapy. 

That is a bias as a clinical trialist. I think it’s the right bias, but it is still my bias. I think patients frequently worry that they’re being treated as a guinea pig, or they’re not getting an appropriate treatment. What I can tell you is the clinical trials that we and my colleagues across the country and across the world participate in are clinical trials where the patients are getting at least what we consider a standard of care for that phase of their disease, and they may be getting something in addition to that or something that is slightly different, but expected to have a similar response rate. 

We have this phrase in clinical trials, something called equipoise, that if there’s a randomization between options that we need to feel, as the practitioner and as the clinical trialist, that each option is at least as good as the other.  

Patricia:

That kind of goes back to the vetting of treatments before they go to a clinical trial. Tell me a little bit about history. How can we make patients feel more comfortable?

Dr. Altman: 

I want to make sure that I understand the question.

Patricia:

So, how thoroughly are treatments vetted before they go to a clinical trial?

Dr. Altman: 

Great. So, the way that agents get into early phase clinical trials and then later phase studies are these are compounds that have been studied in the laboratory, then studied in small animals, then larger animals. And then, frequently, a drug is started in a patient with relapsed and refractory Acute Myeloid Leukemia and found to be safe – that’s what we call a Phase I study. 

Once we know the right dose and the associated side effects from an early phase clinical trial, later phase studies – i.e. Phase II, where the goal is to determine the efficacy and response rate is conducted. And then, if that appears and looks like it’s promising, a larger, randomized, three-phase study is frequently conducted, where we compare a standard of care to the new approach. 

Patricia:

So, patients should be comfortable that the clinical trial that they’re going through has been thoroughly vetted, has gone through multiple stages before human trials occur?

Dr. Altman: 

That is accurate in terms of compounds get through animal studies, and then depending on the way that the trial is being connected, will then be studied in patients either with relapsed or refractory disease or very high-risk disease. But it’s also very important to mention that these pharmaceutical companies and physicians are not making these decisions alone. 

The clinical trials are all reviewed by scientific review committees through the cancer centers, which are other investigators making sure that everything appears appropriate. In addition, there are institutional review boards at every university whose goal it is to keep patients and research subjects in well-done clinical trials safe. That is their primary goal. And the IRBs – institutional review boards – are very involved with making sure that clinical trials are appropriate and that the conduct of clinical trials is appropriate.