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
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.
“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]
Six Top Medical Institutions Launch Research Alliance Program to ‘CRUSH MDS’, a Rare Form of Blood CancerPosted: March 9, 2016
Joint Effort Expands Experts’ Capacity to Develop Treatments, Find a Cure
Ex-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
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.
Leukemia & Lymphoma Society Awards $1.8 Million to Weill Cornell for Translational Research in Blood CancersPosted: August 31, 2012
New Translational Research Grants Awarded to Accelerate Promising Blood Cancer Research Discoveries from the Laboratory to the Patients’ Bedside. Read the press release here.
For more information on the Leukemia and Lymphoma Society, click here.
For information on the Guzman Lab and the research of Monica Guzman, PhD, click here.
For information on the laboratory of Dr. Duane Hassane, click here.
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.
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.
Dr. Gail Roboz spoke with ecancertv at ASH 2011 in San Diego about the major genomic research on acute myeloid leukaemia. There has been a lot of recent success on identifying mutations and abnormalities in AML; however, Prof Roboz believes that the discovery period with genomic research is coming to an end and a move towards clinical trials and targeted therapies need to be developed. The largest development has been the role of stems cell in research and how to target the cells that are left over after chemotherapy.