Dr. Desai – Treatment for Intermediate & High Risk MDS

Dr. Desai discusses approved treatments for intermediate and high risk MDS. For more information visit crushmds.org.


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


Are You a Patient Taking Vidaza or Dacogen?

Seeking Research Volunteers

Predicting Response To Your Myelodysplastic Syndrome (MDS) Treatment

Azacitidine (Vidaza®) and decitabine (Dacogen®) are FDA-approved drugs for the treatment of MDS. While these drugs help many patients with MDS, sometimes patients who initially respond to these drugs eventually lose their response. Why? Why do the drugs stop working? MDS-CRC investigators are trying to answer this question. Through CRUSH!!MDS, we are recruiting patients who have not responded or lost their initial response to azacitidine or decitabine. Patients will be able to have blood drawn at the time of a routine visit to their local doctor and we will arrange for the blood to be delivered to Weill Cornell Medical College, at no cost to the patient. At Weill Cornell, the blood will be analyzed in the laboratory of Dr. Joseph Scandura, M.D.

For more information about the study and the CRUSH!!MDS initiative, please visit our website.

 

 


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


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


Dr. Gail Roboz reviews existing and evolving approaches to the treatment of patients with AML for Medscape Education

Medscape AML Presentation  To view the entire presentation and slideshow, click here.