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.


Cancer Care: The Next Generation

Dr. Gail Roboz on ABC talks about “taking a chance on something new.” [go]

 

 

 

 

 


Not just a shot in the dark

Friday, July 1, 2016

In many ways, it’s a science success story: 8-year-old boy with a rare form of brain cancer is treated by one of the world’s leading experts in the disease, who collaborates with a pioneering precision medicine institute to sequence his cancer and create a first-of-its-kind tumor model replica in the lab, allowing for further analysis and treatment testing without risk of harm to the child.

Upon analysis, the physician-scientist discovers a mutation previously not known to be linked to that type of cancer — and it happens to be in the protein that his colleague has spent a career studying. He contacts the colleague to ask if there is a drug to target the protein, and it arrives the next day. Applied to the tumor model, the drug effectively kills 80-90 percent of the diseased cells.

If only the story ended there.

Unfortunately, although the drug has been approved by the FDA, it cannot be used on the young patient because it has never been tested in children, and the pharmaceutical company controlling the drug is not willing to take the risk.

“We now start the gymnastics of trying to get permission from the FDA based on compassionate use,” said Jeffrey Greenfield, M.D., Ph.D. “We’ve done it before, and it takes anywhere from 3-6 months. This boy doesn’t have 3-6 months.”

Greenfield, a neurosurgeon at Weill Cornell Medicine and NewYork-Presbyterian, shared the anecdote at a special event held at Weill Cornell Medicine on June 29, one of 270 across the United States convened by Vice President Joe Biden in tandem with a national summit at Howard University in Washington, DC

Biden invited regional participants to discuss the goals of the “Cancer Moonshot” mission, announced in January by President Barack Obama as a way to accelerate cancer research, foster data sharing and collaboration, and improve patient access to care — all on a five-year timeline.

Greenfield said his story summed up some of the challenges the nation will face in trying to achieve such an ambitious goal.

“The promise of precision medicine, which is enormous and which we have all bought into, doesn’t deliver in this case,” Greenfield said. “We’ve done all the work that we’ve promised to do, and we still have hurdles. The science is great, the medicine is great, but we’ve got to figure out a way to bridge the chasm between academia, pharma and clinic.”

The future is now

Rohan Ramakrishna, M.D., and Gail Roboz, M.D., at the NYC regional Cancer Moonshot Summit at Weill Cornell Medicine
Rohan Ramakrishna, M.D., and Gail Roboz, M.D.. Photo: Ira Fox

Greenfield was joined at the event by more than a dozen other distinguished researchers and physicians, as well as a standing-room only crowd of around 100.

Participants heard that in many ways, the future of medicine is already here. Silvia Formenti, M.D., discussed how she uses radiation therapy to turn patients’ own tumors into internal “vaccines,” and Ching Tung, Ph.D., director of the Molecular Imaging Innovations Institute described new ways of “seeing” cancer.

Neurosurgeon Mark Souweidane, M.D., spoke about the importance of developing new forms of drug delivery and working with industry to be able to integrate research and technology into the operating room. His colleagues Susan Pannullo, M.D., and Michael Kaplitt, M.D., Ph.D., explained stereotactic radiosurgery and the use of ultrasound technology to poke holes in the blood-brain barrier.

“These are ways we can use novel non-invasive technologies that will put us as surgeons out of business, unfortunately, but will help heal the world,” Kaplitt said.

Gail Roboz, M.D., director of the Weill Cornell Leukemia Program, described immunotherapy, and in particular the use of CAR-T cells as an emerging therapy.

“The idea isn’t new, “Roboz said. “What’s new is that we can actually do it, we are able to finally do things that were Jetsons level before.”

“In 2016, we are at an amazing inflection point in cancer therapy,” added neurosurgeon Rohan Ramakrishna, M.D. “It’s one thing to say you want to accomplish big change in five years, it’s another to be able to do that.”

But he added that the time it takes to get discoveries from bench to bedside is still too long. We need to innovate, Ramakrishna said, and we need to incentivize high-risk research.

To read the full story [go]


Dr. Pinkal Desai Discusses a Clinical Trial of SGI-110 for People with MDS/MPN and CMMoL

This clinical study is aimed at men and women with a diagnosis of: Chronic Neutrophilic Leukemia (CNL), Chronic Myelomonocytic Leuekmia (CMML), atypical Chronic Myeloid Leukemia (aCML), Juvenile Myelomonocytic Leukemia (JMML), and Myelodysplastic & Myeloproliferative Neoplasm Unclassifiable (MDS/MPN-U). Click here to learn more or see if you are eligible to participate.


Phase 3 Trial Shows CPX-351 (Vyxeos) Boosts Overall Survival in AML

Frontline treatment with CPX-351 (Vyxeos) significantly boosted overall survival (OS) for older patients with high-risk, secondary acute myeloid leukemia (AML) when compared with the current standard of care, cytarabine and daunorubicin, according to data from a phase III trial released by the drug’s developer, Celator Pharmaceuticals, Inc.
“These findings confirm that Vyxeos provides the first opportunity we’ve had in decades to extend survival for patients with high-risk AML,” added Gail Roboz, MD, Professor of Medicine and Director of the Leukemia Program at the Weill Medical College of Cornell University and the New York-Presbyterian Hospital in New York, said in a statement. “Also, more patients in remission means more who are eligible for potentially curative therapy.” To see the full article and data visit Targeted Oncology

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