Gail Roboz, MD from Weill Cornell Medicine discusses minimal residual disease (MRD) found in acute myeloid leukemia (AML) patients. According to Dr Roboz the biology of the remaining leukemia cells may not be similar to the bulk disease that was eliminated with initial therapy. Currently there are efforts to characterize and quantify the remaining cells, with the hopes to determine whether existing or novel treatments can be used to lower their number to below the threshold level required for stem cell transplants. Furthermore, stem cell transplants are dramatically less effective if there is minimal residual disease detected so any therapy to reduce these cells may confer an advantage. Recorded at the 2016 Annual Meeting of the British Society of Haematology (BSH) and International Society of Hematology (ISH), in Glasgow, Scotland.
Original story posted to Video Journal of Hematological Oncology [go]
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]
Dr. Gail Roboz discusses the latest research, clinical trials and breakthroughs to be presented at the upcoming American Society of Clinical Oncology (ASCO) annual meetingPosted: June 3, 2016
Dr. Gail J. Roboz appeared as a guest on Fox 24’s News Talk Central to discuss the latest research, clinical trials and breakthroughs to be presented at the upcoming American Society of Clinical Oncology (ASCO) annual meeting. Please view the WGXA-FOX 24 segment
“One of the most important things coming out of this meeting is that there is more collaboration between people and there’s more collaboration between pharmaceutical companies to bring new drugs and new technologies together,” notes Dr. Roboz.
Ellen Ritchie, M.D., assistant professor of clinical medicine, joins OncLive in a discussion on the current best practices in the management of myelodysplastic syndrome and how the latest research is likely to impact the field.
“I think it’s very important when we talk to patients about MDS that we talk about the broad spectrum of disease and that we don’t label it as cancer necessarily. Because I think that it can almost be as detrimental to their quality of life with the disease afterward, by labeling it as such, than the treatments and supportive care that we have to give them.” – Dr. Ellen Ritchie
To learn more about our CRUSH!!MDS program and to participate in a clinical trial [go]
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.
When Jody Winsick-Soluri was diagnosed with acute lymphoblastic leukemia (ALL), she found out she had a chromosomal abnormality, the Philadelphia chromosome, which made her prospects bleak.
“I was bleeding out; they said I might only have 24 hours to live,” Winsick-Soluri remembers. Now, after many rounds of chemotherapy, total body irradiation, two bone marrow transplants and seven years, Winsick Soluri takes a targeted drug — Sprycel (dasatinib) — that blocks a protein leukemia cells need to proliferate. “Now, I’m four-and-a-half years out from the last transplant,” the New Jersey mother of four says. “More people with ALL are staying alive a lot longer.”
See the original article posted on March 16, 2016