Dr. Gail Roboz on Medscape InDiscussion Podcast

Dr. Gail Roboz recently joined the Medscape InDiscussion podcast, an audio series for healthcare professionals, for an in-depth dialogue regarding acute myeloid leukemia (AML) with colleagues from the MD Anderson Cancer Center in Houston, Texas. 

In episode 1, they break down a subtype of AML called APL, or acute promyelocytic leukemia, including signs and symptoms, as well as the importance of getting the diagnosis right and the proper treatments. 

Listen to the full episode here: https://www.medscape.com/viewarticle/964529

In episode 2, Dr. Roboz leads a discussion on why certain types of AML are classified differently and how treatment may change as a result. Here’s a preview of this podcast conversation in which they review the nuances and implications of these various molecular and genetic classifications in AML subtypes.

Dr. Gail Roboz

“We’re going to talk about good-risk acute myeloid leukemia (AML). I always struggle with this because AML is a bad disease. I worry sometimes as we’re talking to patients and we introduce the words “good” and “favorable”; I think patients sometimes think that means “don’t worry about it, we’ll live forever,” and I don’t think that’s quite right. But I want to launch the conversation by first defining what are we talking about. I think there’s confusion about assigning good or favorable risk just using cytogenetics, which is kind of the old way we used to do things, and then introducing molecular classifications”.

For the full episode, tune in: https://www.medscape.com/viewarticle/964531

Running for Poppy, Racing for a Cure

When Greg Tanenbaum’s beloved grandfather, known as Poppy, was dealt an acute myeloid leukemia (AML) diagnosis and an estimated 48 hours to live in January of 2017, Greg and his family were devastated. Poppy was not only Greg’s grandfather; he was also Greg’s best friend and number one fan, present at all sporting events and family gatherings with a smile on his face and a joke to tell.


Dr. Gail Roboz and the Leukemia Program team at Weill Cornell Medicine and NewYork-Presbyterian Hospital (WCM/NYP) quickly sprang into action, treating Poppy with a therapeutic regimen meant to keep the cancer at bay and preserve quality of life. As a result, Greg and his family got an extra 15 months with Poppy, during which, Poppy refused to let leukemia stop him from doing what he loved.

Despite his fatigue, Poppy never failed to show up to support his 16 grandchildren at their various athletic competitions, even attending a state championship diving meet with his hospice nurse to cheer on one of Greg’s cousins.

“It’s been said that the best gift you can give someone is your time, because you’re giving them something you can never get back,” says Greg. “Poppy made it clear we were worth every second.”

Poppy passed in March of 2018, but not without leaving a powerful impact on his family.


Inspired by Poppy’s gratitude, perseverance and positivity in the face of cancer, Greg, a former college wrestler with little racing experience, decided to run the 2018 New York City Marathon in honor of his grandfather.

In partnership with the Leukemia and Lymphoma Society’s Team in Training fundraising program, Greg headed into the race backed by 160 donors and about $30,000, which he donated to the Leukemia Fighters at WCM/NYP to support the clinical and translational research efforts of Dr. Roboz and the Leukemia Program.

On marathon day, Greg knocked out the first 20 miles at an 8-minute mile pace before sustaining an injury that would make for a grueling 6.2 remainder. But just like his grandfather, Greg refused to quit.

“Running 26.2 miles is time consuming and difficult,” says Greg. “But my ‘why’ was the most incredible person I was so lucky to know and love.”

2017 American Society of Hematology Annual Meeting and Exposition

Our Leukemia Program researchers and physicians made a huge splash at this year’s American Society of Hematology (ASH) Annual Meeting and Exposition, an educational gathering of over 25,000 clinicians and scientists from around the world who are working to conquer blood disease.

Our team was involved in over 40 study abstracts presented at the meeting, helping to advance the overall understanding of leukemia, as well as improve clinical outcomes and quality of life for those affected by the disease. Here are some highlights:

Drs. Pinkal Desai, Duane Hassane and research colleagues discovered a relationship between specific gene mutations and acute myeloid leukemia (AML) risk, enabling prediction of disease up to a decade prior to its development. Their research was selected for inclusion in the upcoming Highlights of ASH Meeting in January 2018.

Acute Myeloid Leukemia (AML) Patients Demonstrate Increased Prevalence of AML-Defining Mutations in Peripheral Blood Years Prior to the Development of Overt Leukemia: A Case-Control Study

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Dr. Sangmin Lee presented research on a drug showing promise in people with refractory myelodysplastic syndrome (MDS).

Phase II Clinical Study of the Clinical Efficacy and Safety of Tosedostat in Patients with Myelodysplastic Syndromes (MDS) after Failure of Hypomethylating Agent-Based Therapy

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Two Weill Cornell Medicine and NewYork-Presbyterian hematology/oncology fellows made contributions to acute myeloid leukemia (AML) research. Dr. Ghaith Abu-Zeinah compared the efficacy and side effects of plasma and cryoprecipitate given to AML patients to treat and prevent bleeding, and Dr. Jorge Monge explored ethnic disparities in medicine by studying the difference in mutation rates between Hispanic and Non-Hispanic AML patients.

A Single Institutional Experience Comparing Cryoprecipitate to Plasma in Patients with Disseminated Intravascular Coagulopathy (DIC) from Acute Myeloid Leukemia (AML)

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Mutational Frequency in Hispanic and Non-Hispanic Patients with Acute Myeloid Leukemia (AML): Updated Analysis of 227 Patients

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Franco Izzo, PhD, had the highest-scoring abstract in the post doctoral fellow category and was chosen for the ASH Outstanding Abstract Achievement Award.

Single-Cell Methylome and Transcriptome High-Resolution Maps Define the Effects of Epigenetic Modifiers on the Landscape of Hematopoietic Differentiation

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Drs. Gail Roboz and Monica Guzman were also tapped as special session speakers for their expertise in care and research. Dr. Roboz discussed adapting treatment to individual AML patients as part of the Friday Satellite Symposia, and Dr. Guzman led a trainee program teaching others how to set up a laboratory research program.

We are so proud of our Leukemia Program’s leadership at ASH and of the team’s relentless work to make life better for leukemia patients and their families.

Emergency Preparedness for People with Leukemia

In the past few weeks, devastating hurricanes and earthquakes have forced people out of their homes and away from their cancer care facilities, highlighting a need for better education and preparedness surrounding the medical consequences of natural disasters. Emergency situations such as a hurricane, earthquake, blizzard, flood, or blackout, are unpreventable and can drive a city into disarray in a matter of hours – but the more preemptive thinking and planning that people do prior to a catastrophic event, the better equipped they will be to respond. This is especially true for people with leukemia, who must be particularly cautious during such times, as they are often more susceptible to infection or injury.

Follow these tips to help minimize the harm that a natural disaster or public emergency can cause to your personal health.

Wishing everyone a safe fall and winter season!

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FDA Approves CAR T-Cell Therapy for Kids and Young Adults with Acute Lymphoblastic Leukemia

On August 30, 2017, the United States Food and Drug Administration (FDA) approved the cell-based gene therapy Kymriah for treatment of children and young adults with a certain form of acute lymphoblastic leukemia (ALL), the most common childhood cancer in America. The approval greenlights the first gene therapy to be made available in the United States.

Each dose of Kymriah is customized to the individual patient by way of an emerging form of immunotherapy called chimeric antigen receptor (CAR) T-cell therapy. T-cells are extracted from the patient’s blood and genetically modified in the laboratory to produce chimeric antigen receptors, surface-level proteins that enable the T-cells to recognize and fight leukemia cells that possess the antigen CD19. The newly engineered T-cells are then infused back into the patient’s body. The goal of Kymriah and other forms of immunotherapy is to target and attack the cancer cells that they are programmed to destroy.

This historic approval follows clinical trials demonstrating durable safety and efficacy in children and young adults up to age 25 with relapsed or refractory B-cell precursor ALL.

Weill Cornell Medicine and NewYork-Presbyterian Hosptial has ongoing clinical trials evaluating CAR T-cell therapy in adults with certain forms of leukemia. To learn more, visit: https://jcto.weill.cornell.edu/.

FDA Approves Two Treatments for AML

The United States Food and Drug Administration (FDA) has approved CPX-351, a combination of chemotherapy drugs daunorubicin and cytarabine also known as Vyxeos, for treatment of two types of high-risk acute myeloid leukemia (AML).

Clinical trial participants with newly diagnosed therapy-related AML (t-AML) and those with AML accompanied by myelodysplasia-related changes (AML-MRC) demonstrated increased life expectancy when treated with CPX-351, as compared to those treated with separate administrations of daunorubicin and cytarabine.

The Weill Cornell Medicine and NewYork-Presbyterian Leukemia Program, in collaboration with our Joint Clinical Trials Office, participated in the expanded access protocol for CPX-351, and we continue to use the drug across our various studies.

We were also among the sites for the clinical trial that led to another of this week’s FDA approvals: Idhifa, a targeted therapy for relapsed or refractory AML patients with the genetic mutation isocitrate dehydrogenase-2 (IDH2). After treatment with Idhifa, 34 percent of the 157 trial participants who required blood or platelet transfusions at the start of the study no longer required transfusions.

We are proud to be among the first medical centers offering novel treatment options like CPX-351 and Idhifa to our patients and look forward to continued prompt delivery of therapies that may improve life expectancy and quality of life for those affected by leukemia.

Independence from Cancer on July 4th

Manhattan fireworks showThis Fourth of July holiday, we’re not only celebrating the red, white and blue that honors the independence and freedom of our country, but also freedom from cancer and the cancer “blues.” Feeling this sense of freedom may mean that you’re cancer-free or that you’re unwilling to let a cancer diagnosis define you.

To be cancer-free means that tests show no evidence of any cancer remaining in the body, a term coined “complete remission.” In some cases, it is possible to complete treatment but still have some evidence of the cancer. This is called “partial remission.”

At Weill Cornell Medicine and NewYork-Presbyterian Hospital, many of our patients and their families experience a wide range of emotions during and after cancer treatment. Often, freedom from cancer is both something to celebrate and something that comes with an air of caution. That’s because the joy of being cancer-free may be accompanied by fear that the cancer may return.

Fighting cancer is a tremendously emotional experience, so take comfort in the fact that you’re not alone. For patients dealing with any cancer diagnosis, Weill Cornell Medicine and NewYork-Presbyterian offer a patient support group on the first and third Thursday of every month from 12-1p.m. For more information about this safe and supportive place for those dealing with any cancer diagnosis, please contact Susan Marchal at (212) 746–9039.

Have a wonderful holiday!

This article originally appeared on “What’s New In GU?,” a blog from the NewYork-Presbyterian/Weill Cornell Genitourinary (GU) Oncology Program. 

Newly-Opened Clinical Trial for People with Acute Myeloid Leukemia

Dr. Gail J. Roboz is the principal investigator for a new immunotherapy clinical trial at Weill Cornell Medicine and NewYork-Presbyterian for people with acute myeloid leukemia (AML). The clinical trial is evaluating engineered CAR-T cells, UCART123, that have been specially engineered in the laboratory to be able to target a molecule on the surface of leukemia cells called CD123. It is hoped that the T cells will be able to destroy the CD123 positive leukemia cells and lead to remission in selected patients with AML. 

Contact us or click here to learn more: http://bit.ly/2rpQso8

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Dr. Gail Roboz to Lead Clinical Trial in AML for Novel Immunotherapy, UCART123

In the summer of 2015, Weill Cornell Medical College and Cellectis, a French pharmaceutical company, announced a translational research collaboration for a new allogeneic chimeric antigen receptor T cell therapy called UCART123. This “off-the-shelf” product is a directed immunotherapy for patients with acute myeloid leukemia (AML), an aggressive blood cancer. T cells are harvested from healthy donors and engineered to target the CD123 antigen, which is found on AML blast and stem cells, as well as tumor cells in another aggressive malignancy, blastic plasmacytoid dendritic cell neoplasm (BPDCN).

The clinical trial of UCART123 in AML is being led by principal investigator Dr. Gail J. Roboz, Professor of Medicine and Director of the Leukemia Program at Weill Cornell Medicine and NewYork-Presbyterian. Translational scientific studies for the trial are being conducted in the laboratory of Dr. Monica Guzman, Associate Professor of Pharmacology in Medicine, also at Weill Cornell. Clinical research on UCART123 in BPDCN will be led by Dr. Naveen Pemmaraju and Dr. Hagop Kantarjian at the MD Anderson Cancer Center in Houston, Texas.

Stay tuned for more updates regarding the new phase 1 clinical trial for UCART123 to be initiated here at the Leukemia Program in the coming months.

New Classifications for Myelodysplastic Syndromes (MDS)

Dr. Ellen Ritchie recently participated in an OncLive discussion on the latest modifications to the World Health Organization (WHO) classification of Myelodysplastic Syndromes (MDS). WHO classification is the standard diagnostic system utilized by medical institutions worldwide, including here at Weill Cornell Medicine. Recent advances in our understanding of the biological course of MDS have warranted revision to its WHO classification, which was last updated in 2008. In particular, mutational and cytogenetic analyses have to led to refinement of diagnostic terms for MDS. These modifications include a distinction between single versus multilineage dysplasia and elimination of the term “cytopenia.”

The OncLive discussion centered on implications of the new classification on the prognosis and treatment of MDS. While the WHO classification is just one of many factors to consider when evaluating the prognosis of the disorder, the panelists agree that the new modifications will make it easier to determine an appropriate course of treatment for their patients. To learn more, click here or watch the video below.