Oct 27, 2015
Dr. Salvia Jain: Cutaneous T-cell Lymphoma
What would more research funding for rare cancers mean? New treatments? Increased survival? Better outcomes for patients?
Rare cancers pose unique challenges for patients and doctors alike; treatment options are fewer, and funding to investigate new approaches is harder to secure, especially for young physicians. Often, researchers turn to organizations like the Conquer Cancer Foundation for support.
One such researcher is Salvia Sanjay Jain, MD, an Instructor in Medicine in the Division of Malignant Hematology and Bone Marrow Transplantation at Beth Israel Deaconess Medical Center. In 2013, Dr. Jain was awarded a Conquer Cancer Foundation of ASCO Young Investigator Award to explore a new treatment for cutaneous T-cell lymphoma (CTCL), a rare type of cancer that can affect the skin, blood, lymph nodes, and more.
“The prognosis is poor, so there is a great emphasis on trying to identify novel targets and then develop strategies based on those,” she said.
Dr. Jain and her team identified a certain protein, MUC1-C, as prevalent in patients with CTCL and important to the cancer’s growth. They then tested a drug that suppressed this protein and observed the effect on the cancer cells. The results were encouraging. “Essentially, we showed that if you target this protein, you can cause the cancer cells to die,” said Dr. Jain.
The study was limited to mouse models and tissue samples, so Dr. Jain is now hoping to run a clinical trial that replicates the results in human participants. If successful, her work could ultimately mean better outcomes for patients with CTCL.
“When you see patients doing poorly you realize the need to do something novel for them,” Dr. Jain said. “You need to look at things a bit differently and develop strategies to bridge them to a transplant, which may be their only potential curative option.”
Dr. Jain acknowledges the crucial role her grant played in allowing her to do this research. “I don’t think this project would have been possible if I was not funded and did not have this grant. I’m extremely thankful and extremely grateful to the donors—it’s because of them that research in this rare and challenging disease has been made possible.”
She continued, “Awards like this serve as great launching pads for young investigators. Every young investigator needs time. They may be the most promising people, but if they don’t have grants like this to provide them time to perform research, it’s very hard for them to work on exciting projects. Molecular insights into rare neoplasms can help underpin pathways relevant for other cancers as well, hence their investigation is critical.”
Dr. Mark Dickson: Liposarcoma
What if there were better, more effective treatment options for people living with rare types of cancer?
Treatment options for patients with rare cancers are often limited. This is true for liposarcoma, a type of soft tissue sarcoma that affects fat tissues in the human body, often in the thigh or abdomen. Surgery can be effective, but if the cancer returns, choices are few. Chemotherapy is not typically useful in treating the disease.
Mark Andrew Dickson, MD, an Assistant Attending Physician at Memorial Sloan Kettering Cancer Center and Assistant Professor of Medicine at Weill Cornell Medical College, is attempting to tackle this issue by finding a targeted therapy that is more successful at treating liposarcoma. Thanks to a 2011 Conquer Cancer Foundation of ASCO Career Development Award (CDA), Dr. Dickson and his team conducted a clinical trial testing a new drug to treat liposarcoma.
“We tested a targeted drug that blocks a specific protein, called CDK4, that is important to sustain liposarcomas,” he said. The study showed promising results: the drug appeared effective for many, though not all, participants.
Palbociclib, the study drug, has already been approved by the U.S. Food and Drug Administration to treat breast cancer. Further study is still needed before it is approved to treat liposarcoma.
“The initial trial treated about 30 patients. We expanded that to an additional 60 patients, and further papers will be published soon reporting those results. We’re also exploring why CDK4 inhibitors work in some patients with liposarcoma but not in others,” Dr. Dickson said. “The rewarding thing about research is that it allows you to move from the very small to the very big—from observing how, exactly, a drug works in the lab to taking that treatment to a real person with cancer who is looking for something that might help.”
Of his CDA, he said, “It’s hard to overestimate how important and helpful grant support is. The major way most of the promising young translational and clinical investigators get their funding is through grants like those provided by Conquer Cancer Foundation.”
Funding for innovative early-career investigators is crucial to advancing the field of cancer care. “It’s almost mind-boggling to think about how far cancer treatment has come in the last 30 years. Imagine where we will be 30 years from now,” Dr. Dickson said. “All of the progress that’s going to be made is through clinical research. I can’t think of anything more important to support.”