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. Desai – Treatment for Intermediate & High Risk MDS

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


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.

 

 


Leukemia & Lymphoma Society Awards $1.8 Million to Weill Cornell for Translational Research in Blood Cancers

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.


Dr. Roboz Speaks on Genomics in AML

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.

Roboz ecancer.tv