Dr. Roboz on Good Morning America 10/20/15

Dr. Roboz discusses “The Patient’s Playbook,” a new book that will help patients navigate our health care system. Click here to view the video.

AAMDS Boston Patient & Family Conference

Calling all north east patients and family members. AAMDS is holding a conference on Saturday, September 19 in Boston.

For more information please visit the AAMDS website

Weill Cornell Medical College and Cellectis Announce Research Alliance Advancing Drug Discovery and the Translation of Novel Immunotherapies in Leukemia

Collaboration Will Focus on Improving Patient Outcomes in AML Using Targeted Cellular Therapy Developed by Cellectis

June 02, 2015 05:00 PM Eastern Daylight Time


“Cellectis has interesting preclinical data on UCART123 and our alliance will seek to build on these findings to better understand the clinical potential of this therapy. Our patients are anxiously awaiting the start of clinical trials.”

Weill Cornell Medical College and Cellectis have entered into a strategic translational research alliance to accelerate the development of a targeted immunotherapy for patients with acute myelogenous leukemia (AML), a deadly blood cancer. The alliance will foster the development of Cellectis’ lead product candidate in AML, called UCART123.

The collaboration combines Weill Cornell’s broad expertise and resources in translational stem cell science and developmental therapeutics with Cellectis’ work in development and manufacturing of gene edited CAR-T cell product candidates, a special kind of immune cell that includes an antibody-derived receptor. The research will be led by co-principal investigators Dr. Gail J. Roboz, director of the leukemia program and an associate professor of medicine at Weill Cornell, and Dr. Monica Guzman, an assistant professor of pharmacology in medicine at Weill Cornell. Dr. Roboz is an internationally recognized leader in the field of acute leukemia and will design and implement clinical testing of UCART123 in patients with AML. Dr. Guzman is a renowned leukemia stem cell biologist who specializes in preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.

The alliance will seek to accelerate the development of Cellectis’ UCART123 in AML. Cellectis’ proprietary allogeneic CAR T-cell platform utilizes T-cells (immune cells) from healthy donors. The T-cells are engineered with a Chimeric Antigen Receptor (CAR), which enables them to detect specific proteins (antigens) expressed on malignant tumors. Large numbers of allogeneic CAR-modified T-cells are grown in the laboratory and then infused into a patient. The enhanced cells are designed to recognize and attack stem cells harboring the CD123 antigen, which is present on AML blast and stem cells. To enhance safety and minimize toxicity for patients, the company’s gene-editing process features customized control properties that seek to prevent the T cells from inappropriately attacking healthy tissues. Cellectis hopes to develop a cost-effective, “off-the-shelf” allogeneic CAR T-cell product, designed for efficient storage and distribution to patients around the globe.

Cellectis in April opened a new research and development facility in New York City, located in close proximity to the Weill Cornell campus.

“We are pleased to collaborate with Cellectis to develop and advance next-generation treatments for patients with this devastating form of leukemia,” said Dr. Laurie H. Glimcher, the Stephen and Suzanne Weiss Dean of Weill Cornell Medical College. “Cellectis’ proficiency in genome engineering and our complementary expertise in translational research will help us realize our common goal of improving human health in New York and around the globe.”

“CAR-T cells have shown remarkable promise in the treatment of acute lymphoblastic leukemia,” Dr. Roboz said. “Cellectis has interesting preclinical data on UCART123 and our alliance will seek to build on these findings to better understand the clinical potential of this therapy. Our patients are anxiously awaiting the start of clinical trials.”

“Weill Cornell offers unsurpassed expertise in translational research, with a wealth of leading-edge technologies and resources to help advance our pipeline of unique CAR-T product candidates,” said Dr. Mathieu Simon, executive vice president and chief operating officer at Cellectis. “We are excited by the prospect of working with Dr. Roboz, Dr. Guzman and other premier investigators in leukemia stem cell research.”

Weill Cornell’s Office of BioPharma Alliances and Research Collaborations negotiated the three-year alliance. In the program’s pre-clinical phase, Weill Cornell researchers will perform multiple analyses, including data mining of primary AML samples, immune profiling of AML patients and in vitro evaluation of allogeneically derived anti-CD123 CAR-T cells. In the alliance’s second phase, Weill Cornell and Cellectis will jointly develop protocols to facilitate early-phase testing, including phase 1 clinical trials.

“Cellectis believes the CAR-T platform has the potential to transform the way cancer patients are treated. We are confident that our broad, cross-discipline collaboration with Weill Cornell will foster creativity and speed in drug development for the benefit of clinicians and patients living with AML,” said Dr. André Choulika, chief executive officer of Cellectis.

The mission of Weill Cornell’s Office of BioPharma Alliances and Research Collaborations is to proactively generate, structure and market translational research alliances with industry in order to advance promising research projects that have commercial potential. For more information, contact Larry Schlossman at las2041@med.cornell.edu or at 212-746-6909.

About Weill Cornell Medical College

Weill Cornell Medical College, Cornell University’s medical school located in New York City, is committed to excellence in research, teaching, patient care and the advancement of the art and science of medicine, locally, nationally and globally. Physicians and scientists of Weill Cornell Medical College are engaged in cutting-edge research from bench to bedside aimed at unlocking mysteries of the human body in health and sickness and toward developing new treatments and prevention strategies. In its commitment to global health and education, Weill Cornell has a strong presence in places such as Qatar, Tanzania, Haiti, Brazil, Austria and Turkey. Through the historic Weill Cornell Medical College in Qatar, the Medical College is the first in the U.S. to offer its M.D. degree overseas. Weill Cornell is the birthplace of many medical advances—including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson’s disease, and most recently, the world’s first successful use of deep brain stimulation to treat a minimally conscious brain-injured patient. Weill Cornell Medical College is affiliated with NewYork-Presbyterian Hospital, where its faculty provides comprehensive patient care at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The Medical College is also affiliated with Houston Methodist. For more information, visit weill.cornell.edu.

About Cellectis

Cellectis is a preclinical stage biopharmaceutical company focused on developing immunotherapies based on gene edited engineered CAR-T cells (UCART). The company’s mission is to develop a new generation of cancer therapies based on engineered T-cells. Cellectis capitalizes on its 15 years of expertise in genome engineering – based on its flagship TALEN® products and meganucleases and pioneering electroporation PulseAgile technology – to create a new generation of immunotherapies. CAR technologies are designed to target surface antigens expressed on cells. Using its life-science-focused, pioneering genome-engineering technologies, Cellectis’ goal is to create innovative products in multiple fields and with various target markets. Cellectis S.A. is listed on the Nasdaq Global Market (ticker: CLLS) and on the NYSE Alternext market (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com


Media contacts
Weill Cornell Medical College
Sarah Smith, Director of Media Relations
Phone: 646-317-7401
email: sas2072@med.cornell.edu
Jennifer Moore, Director of Communications
Phone: 917-580-1088
email: media@cellectis.com
BMC Communications
Brad Miles
Phone: 646 513-3125
email: bmiles@bmccommunications.com
IR contact
Simon Harnest, VP Finance and Investor Relations
Phone: 646-385-9008
email: simon.harnest@cellectis.com

Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of Azacitidine with or without Birinapant for subjects with Higher Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

The Weill Cornell Leukemia Program has recently opened a new clinical trial for men and women who have been diagnosed with Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL) . The study sponsor is TetraLogics Pharmaceuticals and the principal investigator at Weill Cornell is Dr. Ellen Ritchie. For more information about the study, please call Katherine Hassfurter, RN at (212) 746-4882 or e-mail kah9068@med.cornell.edu.

Key Eligibility

  • Men and women age 18 and older
  • Subjects that have not been treated with hypomethylating agents for MDS or CMMoL
  • Histologically confirmed diagnosis of the following conditions:
    – Myelodysplastic Syndrome (MDS)
    – Chronic Myelomonocytic Leukemia (CMMoL)
  • Life expectancy of at least 3 months
  • ECOG score of 0 or 1
  • Detailed eligibility reviewed when you contact the study team

Study Details

This is a randomized, double-blind, clinical trial for men and women with high risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL). The study is evaluating an experimental drug called Birinapant.

Birinapant is being studied as a potential new treatment for MDS or CML. Birinapant removes certain chemicals (proteins) in a cancer cell which leads to the death of cancer cells. From laboratory and animal studies, birinapant is more likely to cause the death of cancer cells than normal cells. Studies combining birinapant with chemotherapy in the human cancer cell laboratory models showed that the addition of birinapant to chemotherapy can result in further blocking of cancer growth, or overcoming cancer resistance to chemotherapy.  In previous research studies, birinapant has shown to be well-tolerated when given alone or in combination with other chemotherapy drugs.

The purpose of this study is to to compare the safety and efficacy (how well it works) of azacitidine plus an investigational drug (birinapant) versus azacitidine plus a placebo (an inactive substance), in patients with higher-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL).

Dose Escalation and Cohort Expansion Study of TEN-010 in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome

The Weill Cornell Leukemia Program has recently opened a new clinical trial for men and women who have been diagnosed with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). The study sponsor is Tensha Therapeutics, Inc. and the principal investigator at Weill Cornell is Dr. Gail Roboz. For more information about the study, please call Tania Curcio, RN at (212) 746-2571 or e-mail tjc9003@med.cornell.edu.

Key Eligibility

  • Men and women age 18 and older with a confirmed diagnosis of AML or MDS
  • Previously treated with at least one prior therapy
  • Subjects with a history of allogeneic (from another person) stem cell transplant are eligible for study participation
  • Life expectancy of at least 2 months
  • Detailed eligibility reviewed when you contact the study team

Study Details

This is a Phase 1, non-randomized, open-label, multi-center study that utilizes the investigational study drug TEN-010. TEN-010 belongs to a group of drugs called bromodomain inhibitors.  Bromodomains are found in cancer cells and bromodomain inhibitors may have promise as a therapy for patients who have cancer.  Currently, there are no bromodomain inhibitors approved by the FDA for humans. Research in the laboratory has shown that TEN 010 kills cancer cells in different types of both blood cancers.

The study is conducted in two parts; Part A and Part B. In Part A, escalating doses of TEN-010 will be administered to patients to evaluate safety and side effects that may limit the amount of TEN-010 given to patients. One of the goals of Part A is to establish the maximum tolerated dose (MTD) of TEN-010. Part B is an expansion study in which patients are treated at the MTD of TEN-010 to identify safety, tolerability, and how well the disease responds to treatment with TEN-010.

All subjects participating in this study will receive the study drug TEN-010 once daily. Subjects will be assigned to one of three different dose levels ranging from 0.06 mg/kg to 0.24 mg/kg .

New Clinical Trial: Randomized, Open Label, Phase 2 Study of Selinexor (KPT-330) vs Physician’s Choice in Patients Greater Than or Equal to 60 Years Old with Relapsed/Refractory Acute Myeloid Leukemia (AML) who are Ineligible for Intensive Chemotherapy and/or Transplant

The Weill Cornell Leukemia Program has recently opened a new clinical trial for men and women who have been diagnosed with Acute Myeloid Leukemia (AML). The study sponsor is Karyopharm Therapeutics and the principal investigator at Weill Cornell is Dr. Gail Roboz. For more information about the study, please call Tania Curcio, RN at (212) 746-2571 or e-mail tjc9003@med.cornell.edu.

Key Eligibility

  • Men and women age 60 and older with a confirmed diagnosis of AML
  • Previously treated with at least one prior therapy
  • Have not undergone and currently  ineligible for stem cell transplant and/or intensive chemotherapy
  • Have not been diagnosed with Acute Promyelotic Leukemia (AML M3),  Chronic Myeloid Leukemia (CML), and Central Nervous System Leukemia
  • Detailed eligibility reviewed when you contact the study team

Study Details

This randomized, open label study has been designed to assess whether Selinexor (KPT-330) can improve the overall survival in patients with relapsed or refractory AML who are not candidates for intensive chemotherapy. Selinexor (KPT-330) works by trapping “tumor suppressing proteins” within the cell and thus causing the cancer cells to die or stop growing.  The study drug has previously been tested in humans to define a safe dose to be administered. Selinexor is currently being tested in other clinical trials in patients with advanced cancers. This study will examine the effects of Selinexor on AML and the side effects that may occur as a result of treatment. It will also compare the effect of Selinexor with the effect of other existing treatments for AML that your physician can recommend.

Potential subjects will be enrolled in of two treatment groups:
Treatment group 1: In group 1, KPT-330 will be given orally (by mouth) twice weekly
Treatment group 2: In group 2, your physician will choose one of the following AML treatments that are currently available:

  • Best supportive care (BSC) including blood product transfusions, antimicrobial drugs, growth factors as needed, and hydroxyurea
  • BSC + low dose Ara-C given twice a day by subcutaneous injection
  • BSC + hypomethylating agent azacitidine given by subcutaneous injection or decitabine administered intravenously

Selinexor will be given orally twice weekly (Monday and Wednesday or Tuesday and Thursday) at a dose of 60-120 mg

Dr. Gail Roboz Discusses Challenges and Progress in Acute Myeloid Leukemia

When Gail J. Roboz, MD, took the stage Wednesday to give her talk on what’s ahead in the treatment of acute myeloid leukemia (AML), she admitted feeling a little jealousy toward her colleagues in the lymphoid diseases.

“AML continues to languish at the bottom of the survival curve. The lymphoid diseases are just doing so much better,” said Roboz, associate professor of Medicine and director of the Leukemia Program at the Weill Medical College of Cornell University and the NewYork-Presbyterian Hospital.

That is not to say, however, that research into myeloid diseases is “completely languishing,” Roboz stressed in her presentation at the 2014 Chemotherapy Foundation Symposium. Real progress has been achieved in understanding AML’s biology, and new targeted agents are being explored to improve outcomes.

For example, Roboz noted, mutations in FLT-3 (FMS-like tyrosine kinase 3) are associated with highly proliferative leukemia and adverse outcomes, while mutations in NPM1 (nucleophosmin 1) and biallelic mutations in CEBPA (CCAAT enhancer-binding protein a) have significantly more favorable survival.

“Although the mechanism of action of AML is much better understood, it’s not simple, and that’s the problem,” Roboz stressed.

Another challenge in treating patients with AML—which Roboz noted results in 10,000 deaths of the approximately 13,000 cases diagnosed each year—is whether more cases will be diagnosed, as patients survive other cancers. “We know that it’s associated with chemo and radiation exposure,” as well as other known environmental risk factors, genetic abnormalities, and benign and hematologic diseases also associated with AML.

Improving on Standard of Care
Although the current cytarabine-based 7+3 regimen remains the standard of care, “we do understand our weapon a little better, and this has certainly resulted in some survival benefit,” said Roboz, adding that this “much-worked-on regimen can be given to much older patients.”

Roboz, who will be leading an AML education session at the American Society of Hematology Annual Meeting in San Francisco next month, reviewed successive efforts by the German AML Study Group “to make chemo better,” through variations on (and additions to) the 7+3 dosing regimen, but these have led to what she described as “superimposable curves.”

“Is it in fact a triumph of hope over experience to add things on to 7+3?” This is a useful question, she elaborated, because “is it that we’re adding new things that aren’t new enough or are we adding them in the wrong place? It’s certainly concerning that all of these efforts over all of these years led to superimposable graphs.”

Other agents are pending, said Roboz, including clofarabine which, she said, “definitely works in AML, but we can’t quite get it right to be where it needs to be an approved drug for AML. We’re anxiously awaiting whether it can ‘beat’ 7+3,” she said.

A phase II study of CPX-351,1 which, Roboz explained, “is taking 7+3 and trying to make it better. This is a formulation that holds cytarabine and daunorubicin in a fixed 5:1 ratio, and we’re waiting to see whether what looked like a benefit in overall survival in a very difficult-to-treat population of secondary AML patients will hold up in a randomized trial, and whether taking the best regimen that we have and making the formulation better will get the job done.”

Roboz also hopes to have data available soon from the multicenter Alliance trial, looking at decitabine versus decitabine plus bortezomib in a 10-day schedule.

Looking ahead, said Roboz, “We have epigenetics, we have targeted therapies, personalized medicine. We must be on the way to improved therapeutic options.”

“Hope springs eternal. We want these agents to work and to synergize with our ‘best regimens,’” she said.


  1. 1. Lancet JE, Cortes JE, Hogge DE, et al. Phase 2 trial of CPX-351, a fixed 5:1 molar ratio of cytarabine/daunorubicin, vs cytarabine/daunorubicin in older adults with untreated AML [published online March 31, 2014]. Blood.



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