Six Top Medical Institutions Launch Research Alliance Program to ‘CRUSH MDS’, a Rare Form of Blood Cancer

Joint Effort Expands Experts’ Capacity to Develop Treatments, Find a Cure

crush_mds_logoEx-marine Kevin Chambers had always been a strong and powerfully built man. The retired 66-year-old Vietnam War veteran used to work as a professional bodyguard in New York City, providing personal security for major celebrities like Michael Jackson, James Cagney and Barbra Streisand. Last year, Chambers needed a wheelchair and a walker just to get around. 

“I got sick in 2014 and felt so strange and weak in so many ways,” said Chambers. After being initially diagnosed with severe anemia along with two other conditions, later test results showed he had atypical myelodysplastic syndrome (MDS), a life-threatening bone marrow failure disease. Thanks to his daughter, an editor at ABC’s Good Morning America, Chambers was referred to Dr. Gail Roboz, the specialist who treated the show’s co-anchor Robin Roberts for MDS.

Roboz is with the Weill Medical College of Cornell University, one of the six preeminent institutions that form the MDS Clinical Research Consortium (MDS CRC). The others include the Cleveland Clinic Taussig Cancer Institute, the Dana-Farber Cancer Center in Boston, MD Anderson Cancer Center in Houston, H. Lee Moffitt Cancer Center and Research Institute in Tampa, and the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins in Baltimore.

The MDS CRC was created with a grant from the Edward P. Evans Foundation. Suffering from MDS himself, philanthropist Evans was determined to speed up drug development by minimizing excessive “red tape” in clinical research. The CRC is the first collaboration of its kind, and its investigators lead unique, high-quality clinical and laboratory studies aimed at improving the lives of MDS patients. It recently launched a website with a public initiative called the Clinical Repository to Understand, Study and Heal Myelodysplastic Syndromes, otherwise known as CRUSH!!MDS.

The consortium works to accelerate and amplify the research conducted at these leading cancer centers. The beneficiaries are patients like Kevin Chambers, who Dr. Roboz quickly involved in a clinical trial. With careful monitoring of his blood cell counts and reactions to drugs, she was able to customize his care with precision treatments that were regularly adjusted based on his progress.

One year later, Chambers is walking again and his strength has vastly improved. He used to need a blood transfusion every two weeks. Now his transfusions are five weeks apart. He jokes that when he has enough blood, he doesn’t even need to nap. “I work very closely with Dr. Roboz and, if I don’t follow what she says, she kind of gives me hell by thanking me for my medical opinion.” That toughness combined with constant attention to the clinical data is how the specialists CRUSH MDS. For more information visit crushmds.org.

Press release originally posted on AAMDS March 2, 2016


AAMDS Patient Conferences 2016

Following are conferences conducted by AAMDS afford you the opportunity to meet top experts and fellow patients at a free program near you:

Living with Aplastic Anemia, MDS, and PNH

Washington, D.C.
Saturday, March 19, 2016
8:30a to 4:30p
For location and registration

Cincinnati, OH
Saturday, April 30, 2016
8:30a to 4:30p
For location and registration
*Interactive kids program – art activities to further their understanding, ice cream social

Raleigh, NC
Saturday, July 16, 2016
8:30a to 4:30p
For location and registration

San Diego, CA
Saturday, September 17, 2016
8:30a to 4:30p
For location and registration
*Disease track sessions will be offered in Spanish at this location. For more information and registration, please visit aamds.or/eventos

San Antonio, TX
Saturday, October 8, 2016
8:30a to 4:30p
For location and registration
*Disease track sessions will be offered in Spanish at this location. For more information and registration, please visit aamds.org/eventos

West Palm Beach, FL
Sunday, November 6, 2016
8:30a to 4:30p
For location and registration

Seattle, WA – Welcome to the 6th Biennial Conference on Marrow Failure
Saturday, June 18, 2016
8:30a to 4:30p
For location and registration
*Joint event with the Fred Hutchinson Cancer Research Institute

For questions and more information please visit the AAMDS conference page


Awareness Week March 1 to 7, 2016

Are you or a loved one affected by aplastic anemia, MDS (myelodysplastic syndromes), PNH (paroxysmal nocturnal hemoglobinuria), or PRCA (pure red cell aplasia)?

This special week corresponds with the National Organization for Rare Disorders (NORD)’s Rare Disease Day, which is held on February 29.

For more information please visit AAMDS


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.


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

NEW YORK–(BUSINESS WIRE)–Regulatory News:

“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

Contacts

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


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.


References

  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.

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


Weill Cornell Cancer Center Highlighted in New York Times

cancer-1-articleLarge   An April 21, 2013 article in the New York Times describes the budding field of Precision Medicine.  The article highlights Weill Cornell’s state-of-the-art Cancer Center and how Precision Medicine is being used to treat patients with Leukemia.  To read the full article, click here.


Dr. Gail Roboz featured in Parade Magazine

Parade  Dr. Gail Roboz was interviewed and photographed alongside Good Morning America anchor and her bone marrow transplant doctor on March 31, 2013.  To read the full article, click here.


Targeted Healthcare interviews Dr. Gail Roboz on improving the standard treatment of AML

To view the video, click here.

Targeted Healthdare Interview


Dr. Gail Roboz Appears on Good Morning America

Dr. Gail Roboz was interviewed Tuesday, November 20th about Myelodysplastic Syndrome.  Watch the interview below.