Immunotherapy Offers New Strategy for Treating Sarcoma

By Jim Stallard,

Thursday, July 28, 2016

Enlarged microscopic image of blue-green immune cells surrounding one blue cancer cell.

Soft tissue sarcoma, a diverse group of cancers that arise in the body’s connective tissue, is difficult to treat after it has spread. MSK clinicians are investigating the use of immunotherapy to treat this disease. The main approaches involve checkpoint inhibitors and adoptive T cell therapy, which have shown effectiveness against several other cancers. A few clinical trials have already started at MSK and many more are planned to open soon.

Immunotherapy, which harnesses the power of the immune system to fight disease, has recently shown impressive results in the treatment of melanomalung cancer, kidney cancer, bladder cancer, and leukemia. The recent FDA approval of a class of drugs called checkpoint inhibitors has dramatically improved therapeutic options for many patients.

Researchers at Memorial Sloan Kettering are hoping to transfer this immunotherapy success to the treatment of sarcomas, rare cancers that grow in the tissue that connects and supports the organs and other structures in the body.

We spoke with MSK medical oncologist Sandra D’Angelo about the current state of research, including which clinical trials at MSK are under way or planned to open soon.

What’s the rationale for using immunotherapy as a treatment for sarcoma?

Sarcoma is a devastating disease for which we need more effective therapies. It has more than 50 distinct subtypes, and the threat of metastasis is high, affecting up to half of people who are diagnosed. Men and women whose cancer has spread to other parts of the body rarely respond to treatment and have a median survival of just ten to 15 months.

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With conventional treatments such as chemotherapy, or even newer targeted therapies, we try to treat the tumor. But it will likely prove difficult to develop a single therapy that would work across all the different sarcoma subtypes because each of these diseases may behave differently and require distinct treatment approaches.

Immunotherapy is an appealing potential option because it’s designed to empower the immune system to fight many different types of cancer, not just one. My colleague Jedd Wolchok, with whom I worked on immunotherapy treatment for melanoma, takes the view that it’s often better to treat the patient and let the patient’s own body treat the tumor.

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What types of immunotherapy are now being investigated for sarcoma?

The main approaches involve checkpoint inhibitors and adoptive T cell therapy. Checkpoint inhibitors are drugs that block specific proteins on the surface of immune T cells. This releases a natural brake on the immune system, allowing it to attack the cancer. Adoptive T cell therapy involves removing T cells from patients and modifying them in a way that enables them to recognize and attack specific molecules on the surface of cancer cells. MSK has led the way in using both these approaches to treat cancer.

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How is checkpoint inhibitor–based immunotherapy being tested against sarcoma at MSK?

The checkpoint inhibitors we are investigating include the drugs nivolumab and ipilimumab, which we’ve already seen be effective in multiple cancers. Earlier clinical studies showed that ipilimumab — which targets a protein called CTLA-4 — seemed to have very minimal effect when used alone against selected sarcomas.

We think combination immunotherapy strategies will benefit more people. We were struck by the deep and rapid responses in melanoma patients when ipilimumab was combined with nivolumab, which targets a different protein called PD-1.

I am leading a national phase II clinical trial testing nivolumab with or without ipilimumab in patients with metastatic sarcoma. This trial has completed enrolling patients and we are now analyzing the data, which we hope to present soon. We will try to determine which patients are responding — and why — and use this information to plan future trials.

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What about the use of adoptive T cell therapy for sarcoma?

We are collaborating with a company to engineer T cells to fight synovial sarcoma, one sarcoma type. Because synovial sarcoma has a specific protein called NY-ESO-1 that is not on other cells, we are able to target the cancer cells selectively. The National Institutes of Health spearheaded this clinical trial, initially in pediatric patients. However, MSK has played a major role in understanding how to extend this therapy to adult synovial sarcoma patients, and we have been able to treat most of the adult patients on this study.

We are now in the process of leading a similar effort for patients with myxoid liposarcoma, whose tumors also express NY-ESO-1.  That clinical trial will be opening within the next few months.

Beyond this specific approach, we are working with MSK investigator Michel Sadelain in a type of adoptive T cell therapy called chimeric antigen receptor [CAR] T cell therapy, which has demonstrated remarkable results in patients with chemotherapy-resistant leukemia. We are trying to develop CAR T cells that will target a protein expressed on the surface of many sarcomas. That’s a big project that we’ve been working on for two years, and we hope to launch a clinical trial testing this approach in the near future.

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What is the biggest remaining challenge for using immunotherapy against sarcoma?

The major hurdle is identifying the right strategies for specific subtypes. It’s difficult to know what will work in a particular sarcoma because they all differ in histology — how the cells look under a microscope — and which mutations they carry. There are ongoing efforts to identify sarcoma biomarkers that can help us predict whether a therapy will be effective.

The important point is that there is great potential and hope for immunotherapy to have some effectiveness against sarcoma because both checkpoint inhibitors and CAR T cell therapy have demonstrated success in other cancer types. We hope to continue to figure out ways to extend this benefit to sarcoma patients. We are looking forward to opening up numerous clinical trials here in the next months, keeping MSK at the forefront of sarcoma immunotherapy.

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Is there any clinical trial to test immunotheraphy in patients with leiomyosarcoma metastatic?

Eduardo, thank you for reaching out. Memorial Sloan Kettering does not currently have any clinical trials testing immunotherapy for leiomyosarcoma. However, you can read a recent story about the latest research on this form of sarcoma here:

These are the clinical trials for leiomyosarcoma that are ongoing at MSK:…

There is also one trial listed on the government's website (…

If you or a loved one would like to make an appointment at MSK for a consultation, please call our Physician Referral Service at
800-525-2225 or go to

I joined a nivolumab clinical trial in the fall of 2014 and was on it for 1 full year. The end result was that tracked tumors decreased by close to 50%. I was off treatment for 8 months then when the cancer began to grow again (slowly) I was put back on treatment and am responding once again. I owe my life to MSK and their immunotherapy clinical trials. So grateful.

Dear Luis, we are glad to know that you have felt well-cared for by your MSK team and that your cancer is responding to treatment with immunotherapy. Thank you for sharing your kind words on our blog. We wish you all our best!

I am so encouraged that this double checkpoint inhibitor trial for sarcoma patients is finally starting! 8 years ago, I was diagnosed synovial sarcoma. Even though I had 2 recurrences and metastatic disease, I went through great lengths to harness my immune system against cancer (NY-ESO-1 peptide vaccine, dendritic vaccine, NK cell vaccine, Coley's Toxins, cryoablation). I am a biology researcher, and the science convinced me that the immune system is capable of curing cancer. I have been cancer-free for the past 3 years. I am so thankful that immunotherapy treatments, such as this trial, are readily available to sarcoma patients now. I hope sarcoma patients will take advantage of this incredible treatment opportunity and emerge cancer-free.

Metastatic chordoma of the spine... are there any somewhat promising treatments or trials coming down the pipeline outside of what's already been established as first, second, line treatments for this disease? The CAR T cell therapy sounds exciting. Thanks for what MSKCC and it's team are working toward.


Firstly thank you for your research into such a terrible illness, it fills us with confidence that you are exploring alternative therapies which is not typical chemotherapy.

My partner who is just 25 was diagnosed with Renal Dedifferentied Liposarcoma 9 months ago and under went a full nephrectomy. She was not offered any other treatment after this.

Yesterday she was suddenly in severe abdominal pain and was taken into hospital. After a range of CT scans the doctors confirmed two more growths near where the kidney was removed, nearer the colon. They have also confirmed the presence of 7 lumps in the lungs.

We are planning on having the abdominal masses removed straight away but for obvious reasons we want to try all we can to fight the lung growths.

Immunotherapy is something which we have hope in and was wondering if there is any suitable trials or treatments you can offer there to give us a chance of beating this.

We currently live in Singapore but are more than happy to travel there if you have any suggestions or input.

Once again thank you, and really do hope you can help us in this time of need.

Kind Regards,


Danny, thank you for reaching out. We are sorry to hear about your partner's situation.

If she would like to speak to someone about coming to Memorial Sloan Kettering for a consultation or possible clinical trial (or sending her records for review), I encourage her to contact our International Center by calling 1­ 212­-639­-4900 or going to The email address is

Have you treated anyone with fibromyxoid sarcoma or know of any trials for that specific sarcoma?

It is hopeful and promising to see that there are a variety of treatment options offered at MSK. My father just learned that he has sarcoma, but it is has still not been determined which specific kind. How quickly can one typically get an appointment with a specialist at MSK?


Dear Alanna, we are sorry to hear about your father's diagnosis. If you are interested in making an appointment for him to see one of our specialists, please call our Physician Referral Service at 800-525-2225. Wait times vary and depend on a number of factors, including how long it takes to get his relevant medical records sent to us, but our staff there can answer your questions as to when the first available appointment would be. Thank you for reaching out to us.

Thank you to MSK for doing this fantastic research to find a cure for such a horrific and rare form of cancer. My mother is currently battling late stage, aggressive Synovial Sarcoma, and I was wondering if there are any trials she could become involved in, or whom I could contact to register her for future opportunities? Thank you.

I was diagnosed with leiomyosarcoma in October 2014. The tumor is totally enclosed in the inferior vena cava. I have been on 3 different types of cancer. Gemzar reduced the tumor in half during the first 1-3 'drips" but the tumor then stabilized and I took a break (per doctors' orders). When next checked 2 months later tumor was growing again. Back on Gemzar but tumor continued to grow until original size. New chemo is keeping it at bay right now. My name is on list for clinical trial at MD Anderson.

Any advice, reactions, suggestions?

Dear Ann, we are sorry to hear about your diagnosis. You may be interested in reading a recent blog post we published on what's new in this area of research: We also offer clinical trials for people with leiomyosarcoma, which are listed here:

If you have any questions about these studies or would like to make an appointment with one of our specialists for a consultation about possible next steps in your care, please call our Physician Referral Service at 800-525-2225. Thanks for reaching out to us.

My father was diagnosed with leiomyosarcoma (LMS) about 7 years ago and had surgery to remove slow growing tumors. However, about 2 years ago, the disease has metastasized and he is not responding to any conventional treatment or the new chemo that was recently approved. Are there any trials that have proven successful? We are out of state - do you have partnerships with hospitals in Chicago?

Dear Julie, we are sorry to hear about your father's diagnosis.

Unfortunately, we don't have any alliances with hospitals in Chicago, but if you are interested in finding a local comprehensive cancer center to see if they offer a trial that's appropriate for your dad, you may search for one on the National Cancer Institute's website: Robert H. Lurie Comprehensive Cancer Center at Northwestern University and The University of Chicago Comprehensive Cancer Center are both listed as comprehensive cancer centers.

You might also look for clinical trials in your area on this national database maintained by the National Institutes of Health:

In case you are interested, MSK does have a couple of open trials evaluating new treatments for people with leiomyosarcoma, which can be found here:…. If you have any questions about these studies or would like to make an appointment with one of our specialists to discuss possible next steps in your father's care, please call our Physician Referral Service at 800-525-2225.

Thank you for reaching out to us.

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