Report Highlights Advances Made at MSK That Are Changing Patients’ Lives

By Julie Grisham,

Wednesday, January 28, 2015


An annual report from an influential cancer group highlights three MSK studies that have advanced cancer research.

Memorial Sloan Kettering is pleased to share the news that the American Society of Clinical Oncology (ASCO) has recognized three of our recent studies as being among those that have the greatest potential to improve patients’ lives. Clinical Cancer Advances 2015, ASCO’s latest report of the most important achievements in clinical cancer research over the past year, highlights some of our accomplishments in the treatment of both common and rare cancers.

These successes are largely due to advances in molecular oncology and immunity science, two areas where MSK continues to play a leading role in taking findings from the laboratory and translating them into new treatments.

Training T Cells to Attack Leukemia

One of the innovative areas of research showcased in the report comes from MSK’s Center for Cell Engineering, led by researcher Michel Sadelain. Along with members of our Leukemia Service, the group is developing a cell-based therapy for adult patients with B cell acute lymphoblastic leukemia (ALL), a rapidly progressing form of blood cancer.

Their approach involves removing T cells, a type of white blood cell, from patients and introducing a new gene into the cells that enables them to recognize the cancer. After the gene is inserted, the T cells are infused back into the patient, where they multiply and cause a variety of immune responses aimed at attacking the cancer cells. This year, in the largest study to date to evaluate this treatment, the team reported that 88 percent of patients responded to the therapy.

“This work was important because it demonstrates that this is a repeatable outcome,” says medical oncologist Renier Brentjens, one of the senior authors of the work, which was published in Science Translational Medicine.

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Hope for a Crippling Joint Disease

New research from MSK investigators that sheds light on a potential therapy for pigmented villonodular synovitis (PVNS) — a rare and destructive joint disorder that affects approximately 600 young and middle-aged adults in the United States each year — is also cited in the ASCO report. A study led by medical oncologist William Tap, Chief of the Sarcoma Medical Oncology Service, suggested that a drug blocking a protein known to promote PVNS could alleviate symptoms and improve quality of life.

The drug, known as PLX3397, inhibits a protein called CSF1 kinase, which is a driving force in the development and growth of advanced PVNS. “By taking this drug that potently inhibits a single genetic process, several patients with advanced PVNS appeared to experience, in a relatively short amount of time, relief from pain and stiffness as well as marked improvement in joint function, all with minimal side effects,” says Dr. Tap, who presented the findings at ASCO’s annual meeting in June.

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Focus on a Rare, Aggressive Ovarian Cancer

As the ASCO report notes, genomic research is leading to promising findings for many rare cancers. One of those discoveries, made by MSK researchers, identified a genetic mutation that appears to cause an aggressive type of ovarian cancer in young women called small cell carcinoma of the ovary, hypercalcemic type (SCCOHT).

The scientists conducted genetic analysis of tumor samples from 12 patients with SCCOHT using a technology called MSK-IMPACT™. All 12 samples had a mutation in a gene called SMARCA4.

The researchers now plan to develop potential therapies and test them on cell lines that carry the SMARCA4 mutation. “Now that we know the target — what we think is the Achilles’ heel — we’ll try to hit it,” says physician-scientist Douglas Levine, who was senior author of the study, published in Nature Genetics.

You can view the full report on ASCO’s website.

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i may not live to see it, but I believe sooner than later, they will figure it out.

I welcome your monthly email . I read everyword of it. I also pass it on to my cousin in Barbados who is a breast cancer survivor. I lost my dad to kidney cancer. Please keep this very informative correspondence up, as I always look forward to receiving it. Do have a lovely day.
God Bless

I am interested in any up coming trials for PVNS ... I currently have it in my Left Knee and I have been taking gleevec for 5 wks now. I have a partial knee replacement in that knee also.... Any information would be helpful thank you Mariann Morales

Dear Mariann, we are sorry to hear about your diagnosis. You may be interested in reading a recent blog post we did about advances in treatment for PVNS:…

Also, we currently have a Phase I clinical trial evaluating a drug called PLX3397 for people with PVNS:

We hope this information is helpful. Thank you for reaching out to us.

Mi hija tiene un carcinoma neuroendocrino en el cuello del utero de 2,5 cm., no hay metástasis, quieren hacer cirugía radical con anexo, retirar ovarios, cuello de utero y utero, ella tiene 32 años y muchas ansias de maternidad justo en el momento. Muy preocupados, porque no aseguran que con quimioterapia tradicional no prosiga. Somos de Argentina y quisiera saber en que lugar del mundo puede haber algún otro tratamiento menos agresivo.

Querida Graciela, sentimos que tu hija ha sido diagnosticada con cáncer. No podemos ofrecer consejos médico a través de nuestro blog. Si ella quiere hacer una cita con uno de nuestros especialistas para discutir sus opciones de tratamiento, por favor communicate con nuestro Centro Inernacional para pacientes extranjeros: El personal del sevicio pueden contestar sus preguntas sobre la posibilidad de preserver su fertilidad ántes de recibir tratamiento (y hablan Español). Esta página tiene más información sobre éste servicio:

Puedes leer más sobre la fertilidad y cáncer en ésta página:

Gracias por su comentario y desamos lo mejor para tu hija.

My question is has there been any new research for stage IV colon-rectal cancer that has metasizied to the liver?

Loretta, thank you for reaching out.

There is currently a clinical trial at MSK regarding liver metastases from colorectal cancer, although it is not a treatment but research into how well ablation of liver metastases (destroying the tissue through a needle inserted through the skin) has worked.

There are several approaches to treating liver metastases. If you are interested in treatment of this condition at Memorial Sloan Kettering, you can learn more here:…

In addition, there are a number of clinical trials for this condition listed on the government's website, which may be of interest to you:…

For further questions about treatment of liver metastases, including recent research, you also can call the National Cancer Institute’s Cancer Information Service at 800-4CANCER (800-422-6237). To learn more about the CIS, including Live Chat help and how to send them an email message, go to

If you would like to make an appointment with a Memorial Sloan Kettering physician, please call our Physician Referral Service at 800-525-2225 or go to Thanks for your comment.

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