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

Posted: November 17, 2014 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials | Tags: Acute Myeloid Leukemia, Blood Disorders, cancer, Gail Roboz, Gail Roboz MD, Leukemia, Myelodysplastic Syndrome | Comments Off

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

Posted: November 12, 2014 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials | Tags: Acute Myeloid Leukemia, blood cancer treatment, cancer, hematology, Leukemia | Comments Off

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

Posted: November 6, 2014 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials, Leukemia News, Patient Education, Physician Presentations | Leave a comment

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.

References

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.

http://www.onclive.com/conference-coverage/cfs-2014/Roboz-Discusses-Progress-Challenges-in-AML

Weill Cornell Medical College Selected to Join New MDS Clinical Research Consortium

Posted: June 19, 2012 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials, Laboratory Research, Leukemia News | Tags: Gail Roboz, Gail Roboz MD, MDS, Myelodysplastic Syndrome, Weill Cornell, Weill Cornell Medical College | Comments Off

MDS researchers join forces to advance patient treatments and outcomes

Posted: June 19, 2012 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials, Laboratory Research | Tags: Blood Disorders, Gail Roboz, Gail Roboz MD, MDS, Myelodysplastic Syndrome | Comments Off

NewYork-Presbyterian/Weill Cornell Medical Center is 1 of 6 institutions selected to participate in new MDS clinical research consortium

NEW YORK (June 18, 2012) — NewYork-Presbyterian/Weill Cornell Medical Center is one of six institutions selected to join the newly-founded MDS Clinical Research Consortium. The Consortium’s mission is to significantly advance treatments and patient outcomes for Myelodysplastic Syndromes (MDS) through innovative research and clinical trials.

The five-year, $16 million multi-institution initiative is the first privately funded MDS research consortium in the United States. It is sponsored by the Aplastic Anemia & MDS International Foundation of Rockville, Md., and supported by the Edward P. Evans Foundation. The Consortium’s funding to Weill Cornell Medical College will support MDS research at NewYork-Presbyterian/Weill Cornell Medical Center.

MDS is a cancer of bone marrow stem cells that inhibits the body’s ability to produce healthy blood cells. The disease can be treated and, in some cases, controlled, but currently, the only cure is stem cell transplantation. The new Consortium will help fill a major gap in the United States for MDS-related clinical research by joining dedicated academic medical centers with a high volume of MDS patients, an established database of current and former patients and a significant track record of participation in MDS clinical trials. The collaboration will facilitate evaluation of promising new compounds, epidemiological studies and translational research studies leading to new classifications, treatments and procedures for MDS.

“MDS is an under-recognized disease. Sometimes we don’t know why a patient has developed MDS, but we do know that those who have been exposed to cancer chemotherapy and radiation therapy are at increased risk,” says Dr. Gail J. Roboz, Weill Cornell Medical College’s principal investigator for the Consortium and director of the Leukemia Program at NewYork-Presbyterian/Weill Cornell Medical Center. “This Consortium offers a wonderful opportunity to develop new therapies and also to profile patients using the latest, state-of-the-art technologies so we can start to understand who gets MDS and why.”

Other Consortium partners include the Cleveland Clinic’s Taussig Cancer Institute, Dana-Farber Cancer Institute, MD Anderson Cancer Center, H. Lee Moffitt Cancer Center and Research Institute and the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

“One of the greatest challenges in research of rare diseases like MDS is having enough patients to conduct meaningful clinical trials. No single center can do it alone. This uniquely collaborative effort overcomes that barrier,” said John Huber, Executive Director of the Aplastic Anemia & MDS International Foundation. “To have these six leading MDS research centers working together in this way is unprecedented,” Huber added. The Aplastic Anemia & MDS International Foundation will expand its programs for MDS patients, their families, and caregivers and local physicians who support and complement the aims and purposes of the Consortium.

“We are extremely pleased to work in partnership with AA&MDSIF and these six outstanding Consortium members. This collaborative endeavor reflects Mr. Evans’ desire to support the highest quality MDS research, which will lead to improved treatments for patients and, ultimately, to finding a cure,” said an Evans Foundation trustee.

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View video of Weill Cornell’s principal investigator, Dr. Gail J. Roboz, discussing MDS and the importance of the new MDS Clinical Research Consortium: http://www.youtube.com/watch?v=gBSqofQ1vdY .

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 the Methodist Hospital in Houston. For more information, visit weill.cornell.edu.

New Clinical Trial: Combination Chemotherapy and Dasatinib in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

Posted: March 26, 2012 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials, Leukemia News | Tags: Acute Myeloid Leukemia, AML, blood cancer treatment, chemotherapy, Core Binding Factor Abnormality, dasatinib, Gail Roboz MD, NewYork Presbyterian Hospital, Weill Cornell Medical College | Leave a comment

The Weill Cornell Leukemia Program is now recruiting patients for a new study, “CALGB 10801: A Phase II Study of Induction (Daunorubicin/Cytarabine) and Consolidation (High-Dose Cytarabine) Chemotherapy Plus Dasatinib and Continuation Therapy with Dasatinib Alone in Newly Diagnosed Patients with Core Binding Factor Acute Myeloid Leukemia (AML).”

The physician leading the study at Weill Cornell is Gail Roboz, MD. For more information or to see if you are eligible for the study, please contact Tania Curcio, RN at (212) 746-2571 or email Tania at tjc9003@med.cornell.edu.

Study details:

This is a clinical trial for patients with acute myeloid leukemia (AML) that possesses an abnormal molecular feature (a gene mutation). The purpose of this study is to test the safety and effectiveness of adding the drug dasatinib to a treatment regimen in patients with AML and to determine how well the leukemia responds to the treatment. The study is being done because currently available treatment is not effective in curing patients with this type of leukemia.

There are three parts to the treatment in this study. The first part of the therapy will test the safety and effectiveness of adding dasatinib to the standard combination of chemotherapy drugs used to treat AML that include daunorubicin and cytarabine. The second part of the therapy will test the safety and effectiveness of combining dasatinib with another chemotherapy treatment, consolidation therapy with high-dose cytarabine. Finally, the third part of the therapy will test the effectiveness of the use of dasatinib alone for 12 months during continuation therapy.

Patients will receive therapy for about 18 months on study. After you are finished with the therapy, you will be asked to visit the office for follow-up at least every 2 months for 2 years, then every 3 months for 2 years, then yearly for a maximum of 10 years from when you entered the study.

Key Eligibility

Men and women age 18 and older
Acute myeloid leukemia (AML with Core Binding Factor (CBF) abnormality
No prior chemotherapy for leukemia or myelodysplasia
Detailed eligibility reviewed when you contact the study team

Leukemia Team Presents at ASCO Annual Meeting

Posted: August 3, 2011 | Author: Weill Cornell Leukemia Program | Filed under: Clinical Trials, Leukemia News, Physician Presentations, Uncategorized | Tags: Acute Myeloid Leukemia, AML, Blood Disorders, Ellen Ritchie, Eric Feldman, Gail Roboz, Leukemia, Leukemia Treatment, MD, Myelodysplastic Syndrome, New York Presbyterian, Weill Cornell | Comments Off

The work of Drs. Ritchie, Roboz, Scandura, Gergis, and Feldman, and nurse Tania Curcio was presented at the 2009 American Society of Clinical Oncology Annual Meeting. The presentation focused on a clinical trial treating elderly AML and high-grade MDS patients. To view the presentation, click here.