Immunotherapy Offers New Strategy for Treating Sarcoma

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

Immune T cells surround a cancer cell. Researchers are investigating ways to stimulate these immune cells to fight sarcoma. (Source: Alex Ritter, Jennifer Lippincoll Schwartz and Gillian Griffiths, National Institutes of Health)

Update: This story was originally published on July 28, 2016, and has been updated with new information.

Immunotherapy, which harnesses the power of the immune system to fight disease, has recently shown impressive results in the treatment of multiple cancers. The Food and Drug Administration’s approval of a class of drugs called checkpoint inhibitors has dramatically improved therapeutic options. People with melanoma, lung cancer, kidney cancer, bladder cancer, leukemia, and other cancers may benefit from these drugs.

Researchers at Memorial Sloan Kettering are hoping that this immunotherapy success can be applied to the treatment of sarcomas as well. These rare cancers grow in the body’s connective tissues, including fat, blood vessels, nerves, bones, muscles, and cartilage.

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

Why use immunotherapy as a treatment for sarcoma?

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The immune system can help fight cancer.
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Sarcoma is a devastating disease for which we need more-effective therapies. Sarcoma has more than 50 distinct subtypes, and the threat of metastasis can be high. Depending on the type of sarcoma and its initial size, the disease can spread in some patients who are diagnosed. Men and women whose cancer has spread to other parts of the body often respond to treatment but the duration of benefit can sometimes be short and the side effects can sometimes be tough.

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 of the sarcoma subtypes. Each of these diseases may behave differently and require distinct treatment approaches.

Immunotherapy is an appealing option because it’s designed to empower the immune system to fight many different types of cancer, not just one. My colleague Jedd Wolchok and I worked on immunotherapy treatment for melanoma. He takes the view that it’s often better to treat the person and let their 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 the cells 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 of 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 ipilimumab (Yervoy®), nivolumab (Opdivo®), and pembrolizumab (Keytruda®), which we’ve already seen be effective in multiple cancers.

Ipilimumab targets a protein called CTLA-4. In earlier clinical studies, it seemed to have a very minimal effect when used alone against selected sarcomas. The latter drug, nivolumab, targets a different protein, called PD-1. We were struck by the deep and rapid responses in people with melanoma when ipilimumab was combined with nivolumab. We think combination immunotherapy will benefit more people with sarcoma. Much of our efforts are focused on identifying the best drugs to combine for patients.

I led a national phase II clinical trial testing the combination of these drugs in people with metastatic sarcoma. We found that nivolumab and ipilimumab are effective in certain sarcoma subtypes. These include undifferentiated pleomorphic sarcoma and myxofibrosarcoma (both types of liposarcoma) as well as leiomyosarcoma, and angiosarcoma. The tumor responses appeared to be similar to those seen with standard chemotherapy. In addition, the combination of these two drugs was found to be very safe and tolerable. The findings were published in January 2018 in the journal Lancet Oncology. They support future studies of this drug combination for people with specific metastatic subtypes. We are continuing to focus on identifying sarcoma biomarkers that allow us to predict who will respond well to this type of immunotherapy.

Sarcoma expert Ping Chi meets with research technician Makhzuna Khudoynazarova
Soft Tissue Sarcoma Clinical Trials and Research
Our doctors and researchers are constantly seeking new and improved treatments for soft tissue sarcoma through our program of clinical trials.
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We’re also exploring therapies that combine checkpoint inhibitors with new drugs that boost the immune response in other ways. For example, there is an ongoing effort combining nivolumab with NKTR-214. This drug is a modified form of a protein called interleukin-2, which is made by the immune system. NKTR-214 is designed to trigger other cells in the immune system to attack cancer cells. The goal is to see if this combination will increase the likelihood of an immune response for metastatic or locally advanced sarcoma.

In addition, there’s an upcoming study combining pembrolizumab with a drug called epacadostat that will be conducted in collaboration with medical oncologist Ciara Kelly. Tumors often can produce an enzyme called IDO1 to avoid the immune system. Epacadostat blocks this enzyme to help the immune system hit the tumor with its full force.

Most recently, in collaboration with Dr. Kelly, we conducted a clinical trial utilizing TVEC, an attenuated herpes virus injected directly into the sarcoma tumor, in combination with pembrolizumab. This trial just completed patient accrual, and we are currently analyzing the results.

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

We have been collaborating with a company to engineer T cells to fight synovial sarcoma. This type of sarcoma has a specific protein called NY-ESO-1 that is not on other cells. Because of that we are able to target the cancer cells selectively. The treatment is being tested in a pilot study at MSK. The trial involves removing T cells from a person with synovial sarcoma, engineering the cells to recognize the NY-ESO-1 protein, and then giving them back to the person in large numbers.

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Learn five reasons why joining a clinical trial at MSK might be right for you.
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As of November 2017, 37 patients have received these modified T cells in multiple groups with variations to the treatment approach. Overall, the response rate has been promising. There are ongoing efforts to optimize this treatment approach and determine the best strategy moving forward. We are now in the process of leading a similar effort for people with myxoid liposarcoma. Their tumors also express NY-ESO-1. This trial is actively enrolling patients.

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

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What is the biggest 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. The cells all look different under a microscope and have different mutations. 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. 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 people with sarcoma.


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Dear Mahir, we’re sorry to hear about your sister’s recurrence. The immunotherapies that are being used for sarcomas are currently only available in clinical trials. If your sister would like to participate in a trial in India, we recommend she speak to her doctor there. If she is interested in coming back to MSK, she can contact our International Center at or go to for more information, or reach out to her surgeon from MSK for more information. Thank you for your comment, and best wishes to you and your family.

My 49yr old Navy veteran husband is recovering from his 3rd surgery in as many years for well differentiated retroperitoneal liposarcoma. The 1st surgery removed a 14lb tumor, the 2nd surgery he lost his right kidney with the tumor attached and this 3rd surgery two tumors were removed but is very healthy otherwise. Are there any immunotherapy trials that would benefit him with this type of sarcoma? We currently reside in Jacksonville, Florida. Thank you for all the research you continue to work on for these cancer cures!

Dear Laura, we’re sorry to hear about your husband’s diagnosis and recurrences. If you are interested in finding out about trials at MSK and coming to New York for a consultation, you can call 800-525-2225 or go to for more information on making an appointment. If you are not able to travel to New York, you may want to consult the National Cancer Institute’s list of designated cancer centers to find one that is closer to you. These centers are focused on research and may have the kind of trials you are looking for. You can find the list at

Thank you for your comment, and best wishes to you and your family.

Do you know of any immunotherapy trials for the sarcoma subtype nerve sheaf tumor? thank you.

Dear Adele, we don’t have any immunotherapy trials specifically for this subtype, but you may quality for one of our trials, depending on the characteristics of your tumor. If you would like to make an appointment to learn more, you can call 800-525-2225 or go to for more information on making an appointment. Thank you for your comment, and best wishes to you.

Has any immunotherapy research been done for malignant nerve sheath tumors?My sister had a very large sarcoma removed from the center of her chest which was successfully removed. She did have some positive margins for sarcoma and was treated with proton radiation.We are trying to be proactive with any new treatments so she does not have a re occurrence. We would appreciate any thoughts you have. Thank you

Dear Maryann, we’re very sorry to hear about your sister’s diagnosis. MSK does not have any immunotherapy trials specifically for this tumor, but your sister may qualify for a trial based on the mutations on her tumor. If she would like to learn more, she can call 800-525-2225 or go to for more information. Thank you for your comment, and best wishes to you and your family.

I'm a 55-yo male. I've been diagnosed with a mixoid lipo sarcoma, dumbell shaped at my sacrum, around the nerve sheathes with a growth in the spinal canal. Most of the treatments I see or have heard of include a large resection, which removes not just the bone, but also nerves, likely affecting bladder, bowel and sexual functions. For background, I had the same type of tumor successfully resected from my right thigh in 2003, and have been perfectly healthy since. I live on the West Coast and have been diagnosed by the Kaiser Permanente health group.

Dear Michael, we’re very sorry to hear about your diagnosis. If you are interested in coming to MSK for a consultation, you can call 800-525-2225 or go to for more information on making an appointment. If you are not able to travel to New York, you may wish to consult the National Cancer Institute’s list of designated cancer centers to find one in your area. You can find that list at Thank you for your comment, and best wishes to you.

We live in Pa so I'm not sure where this hospital is located, but my son has Ewings sarcoma it started out as a PNET tumor and has now spread over the yrs. He was almost 16 when he got diagnosed and is now 24. We asked about the ammuneotherapy and Pittsburgh children's hospital told us they don't do that, and we were told last week there is nothing else they can do for him. It's now in his hips , pelvis and spine. . Is there anyway you can help my son. He has a 2 yr old little boy, and my heart is breaking for now only my son but for my grandson as well.

Hi Team, Have you treated any Desmoid fibromatosis patients, How many of them have been completely tumor free now and what were the treatments given.
My wife has undergone 2 surgeries this year, One in 02.2017 and another 09.2017. Still we have traces of tumor left in NECK. wondering what will be the right steps next ?

Dear Raviraj, we’re sorry to hear about your wife’s diagnosis. MSK has extensive experience treating desmoid fibromatosis. It may be useful for you to use our nomogram (prediction tool) for desmoid fibromatosis. You can find it here:

If your wife would like to come to MSK for a consultation, you can call 800-525-2225 or go to for more information. Thank you for your comment, and best wishes to you and your wife.

My spouse, 28-yrs has been diagnosed with Ewing's Sarcoma tumor in left chest area/left lung. We have had combination of chemotherapy of VDC at 21-days cycle, great news is that latest PET scan shows tumor has been reduced by 75%, but still there are hilar nodes showing metabolic activity in chest. We have stained slides from biopsy, but unfortunately, we don't have any block left to perform FISH. Is there any option to go into deeper diagnosis to treat further? as we want to know if we shall perform surgery or shall continue with chemotherapy or may be immunotherapy.

My Grandon was diagnosed with Epithelioid Sarcoma August of this year. It appeared first in his right hand and has traveled up his arm into upper arm and armpit lymph node and possibly to lungs. Do you know of any clinical trials involving this type of Sarcoma?

Very impressed with the work your doing. My niece who is 19 years old was diagnosed with osteosarcoma in her pelvis bone in June. Just underwent 3 rounds of heavy chemo and repeat scans show tumor has grown and spread to spine. She lives in Ireland and is well enough to travel, still walking ok and has complete control or her bowel/ bladder. Cant find a doctor or hospital in Europe to provide any options or treatments or trials. She wants to come to US to see if any treatment vs trials are availabe, I work as a nurse here in NY and availbe to help and support her. Ireland is doing major fund raising for her treatment and she now has raised over 100,000 for treatment, Her doctors have given her weeks vs months to live. Please if any trial is available please contact me. I have tried to make contact on website and phone but not getting any response. Maybe no trials or treatments are available ??

Dear Ger, we’re very sorry to hear about your niece. You can contact our International Center at 212-639-4900 or to learn more about what would be involved in having her come to MSK from Ireland. Thank you for your comment, and best wishes to you and your family.

My son 25 yrs is patient of ewing sarcoma/lung cancer since the last 4.5 yrs.How hw can enter in immunotherapy clinical trial.terms and conditions etc for the comment

My husband, 47 years old, was diagnosed with undifferentiated pleomorphic sarcoma, high grade, March 2017. A 7cm was removed from his left ventricle. (Origin of tumor is the heart) After 2 types
Of chemo it has grown back larger extending into the pulmonary trunk. It has metastasized-to his lungs. He is currently treating at Dana Farber. Are there any clinical trials targeting his specific canacer?

Dear Liza, we’re very sorry to hear about your husband’s diagnosis. MSK does not have any trials specifically for this type of sarcoma, but depending on the mutations that are present in his tumors he may be able to participate in a basket trial. If you would like to learn more you can call 800-525-2225 or go to for more information on making an appointment. Thank you for your comment, and best wishes to you and your family.

I have been battling Stage 4 Pleomorphic Rhabdomyosarcoma for over 4 1/2 years. After multiple metastasis from thigh to lungs (repeatedly in lungs), chest wall, diaphragm, pericardium, brain and finally liver. I have had 3 areas radiated including entire chest, brain and thigh (primary site), 5 surgeries and multiple lines of unsuccessful chemotherapy... When it went to my liver is when I received Nivolumab on compassionate grounds in February of 2017. I was pretty sure my life was coming to an end. Miraculously, my cancer finally showed "marked response" with tumors literally disappearing. My lungs were clear and have remained so since my scans in June, my cancer saturated liver showed stability, with masses decreasing in size or just vanishing... Never in a million years had I imagined that this was a possibility. My question with this being such a new drug, is this, is there an expectation that the cancer will eventually gain the ability to "outsmart" the treatment like it has with chemotherapies? Is it too early to consider the long term effects? Can you be on it forever? I have a form of cancer that most doctors will never have the ability to look at. Are sarcoma patients too few and far to really understand the effects that this may have on us? Since I have been sick, I have attempted multiple times to be a part of studies, with rejection being the common result. Is this because what I have does not merit research because of its rarity in the population?

We’re very sorry to hear that you’ve been through so much, but glad to hear that your cancer is responding to nivolumab. You are correct that research on rare cancers historically has been hard to do, because of the small number of patients with these cancer types. This is one reason why a new type of trials called basket trials are so exciting. These studies enroll patients to receive drugs that target particular mutations regardless of where in the body the tumor started. In this way, patients with rare tumors have increasing opportunities to participate in research studies.

In addition, at MSK, we have researchers who are studying genes linked to rhabdomyosarcoma in the lab, with the hopes of eventually developing treatments that are specific to these tumors.

If you are interested in learning about participating in a basket trial at MSK, you can call 800-525-2225 or go to for more information on making an appointment. Thank you for your comment, and best wishes to you.

My brother age 26 has been just diagnosed with Ewings Sarcoma .Do you have immunotherapy options/trials for Ewings sarcoma or any other modality treatment other than the conventional chemo /radiation and surgery options

My wife is having lieomayosarcoma from last 3 years and now metastasis to lungs, liver and retropertonium . 3 years back when it was found in retroperitonium then it was surgically removed followed by radiation. But then it spread within 1 year and then she has doxorubicin as chemo. Then she take pazopanib for 1 year which stabilize it for some time but not for long. Now she is having chemotherapy with docetexol and gemcitidine.

Is there any trial you can suggest or immunotherapy agent to control this lieomayosarcoma from spreading.

New Zealand

Dear Lukesh, we are very sorry to hear about your wife’s cancer. If she is interested in coming to MSK for a clinical trial, you can contact our International Center at or go to for more information.

You may want to also ask her doctor about clinical trials in New Zealand, or go to to look for them there. Thank you for your comment and best wishes to both of you.

My wife has a small blue cell sarcoma that has not been identified. Duke treated the cancer a Erwings. The cancer spread to the lungs and we just found out it has spead to her brain. She has 2 tumors in the vision section of her brain and 1 is bleeding. Duke sent her home to die and says there is nothing more to be done. She also has a blood clot in the lungs which prevents brain surgery. Is there any treatment options you can offer? Trials? Anything?

Thank you Dr. Kelly. My brother has LMS mets to liver spleen and spine. Went thru chemo effect. Today 2.1.2018 he is starting the Yervoy/Opdivo combo..your recent input has made this possible. Praying for containment.

My father-in-law had squamous cell skin cancer which invaded his sinus and resulted in a 13 hour surgery. He was clear of cancer for a year, but then developed an area with spindle cells that was eventually diagnosed as sarcomatoid squamous cell cancer. Most, but not all, was removed by recent surgery. Is there any immunotherapy clinical trial for which he may be eligible?
Thank you

Dear Teri, we’re very sorry to hear that your father-in-law’s cancer has come back. If he is interested in coming to MSK to find out about participating in a trial here, he can call 800-525-2225 or go to for more information on making an appointment. If he is not in the NYC area and not able to come here for treatment, we recommend he discuss the possibility of receiving immunotherapy with his current care team. Thank you for your comment, and best wishes to you and your family.

Hi, what if you have a cancer that is so rare/difficult to pin down as to diagnosis, that no one can figure out the exact type it is? There are few trials that are truly "all comer" trials - most are geared to specific more common diagnoses. And the ones that are "all comers" often require the more common diagnoses for the expansion phase. So the number of trials available is rather small. Can they be admitted to one of the "more common diagnosis" trials upon a select review, perhaps?

Dear Bill, people with rare cancers may be able to benefit from what are called basket studies or basket trials, depending on what mutations are found in their tumors. You can learn more about this approach here:…

At MSK, people are assigned to basket trials based on the mutations that are found in their tumors using a test called MSK-IMPACT.

If you or someone you know is interested in a consultation to learn more about this, the number to call is 800-525-2225. You can go to for more information on making an appointment. Thank you for your comment, and best wishes to you.

Hi, I am from Australia, my work friends 14-year-old son has Ewing's Sarcoma, had chemo and amputated leg last year, sadly he has relapsed, and I think will not do Chemo again. Are you aware of any trials taking place in Australia? If not, we might try to raise funds for him to have treatment in the USA. Thanks, Don.

Dear Don, we’re very sorry to hear about this. We recommend you go to This is an international database for all kinds of trials. Thank you for your comment.

My daughter has been diagnosed with endometrial stromal sarcoma .. she is currently being treated with chemotherapy - Gemcitabine Docetaxel - with significant tumour shrinkage. would she be considered for immunotherapy treatments? we live in New Zealand with no other options offered for her.

Dear Robyn, we’re very sorry to hear about your daughter’s diagnosis. There are no immunotherapies that are currently available specifically for this cancer, but depending on the mutations that are found in her tumors, she may benefit from treatment with the immunotherapy drug pembrolizumab. There may also be clinical trials for her cancer available in New Zealand. We recommend you discuss this with her current care team. You may also want to seek out a second opinion at an academic medical center. In addition, you can go to to look for any available clinical trials in the part of the country where you live. Thank you for your comment, and best wishes to both of you.

I have oligometastatic undifferentiated pleomorphic Sarcoma and I am completing arm C of the ipilimumab/nivolumab neoadjuvant trial with neoadjuvant radiation prior to resection of a locally recurrent primary. I’ve had the oligometastatic disease in my chest respected with no near term recurrence. At the end of this I will hopefully have a window of undetectable disease where further immunotherapy might still be of significant enduring benefit. Do you have any basket trials that might fit my situation?

Desperately seeking information on Rhabdomyosarcoma. My 25 year old daughter was diagnosed stage 4 early November 2017, please advise. Thank you.

Dear Mona, we’re very sorry to hear about your daugther’s diagnosis. If you would like to learn more about rhabdomyosarcoma and MSK’s approach to treating it, you can go here:

To make an appointment for a consultation with one of our experts, including to learn about clinical trials, you can call 800-525-2225 or go to for more information. Thank you for your comment, and best wishes to you and your family.

Hi Guys, awesome information thank you. I have very recently had a preliminary diagnosis of Spindle cell sarcoma in the retroperitoneal cavity just under left kidney (primary - with no signs of metastases) awaiting sub-typing. In 2011/2012 i had Burkitts Lymphoma and subsequent CODOX-M chemotherapy. I reside in Australia, I am looking at complimentary ( in not alternative) treatment for my current condition. I would love to converse with anyone who can point me in the right direction. Thanks again for great information. regards Mark

Dear Mark, we’re sorry to hear about your diagnosis. We recommend that you check with the major cancer centers in Australia to found out if any of them have centers for complementary or integrative medicine. Thank you for your comment, and best wishes to you.

Are there effective options for any of the new drugs in the Denver area?

Dear Mary, if you are not able to travel to New York City for treatment, we recommend that you seek out treatment at a National Cancer Institute-designated cancer center. You can find a list here:

If you are interested in learning more about clinical trials in your area, you may also want to go to

Thank you for your comment and best wishes to you.

Has this drug been tested on well-difference liposarcoma?

There are a number of different treatments mentioned in this story, so we’re not sure which drug you’re referring to. If you or someone you know has been diagnosed and would like to arrange a consultation with an expert at MSK to find out about our approach to treating liposarcoma, you can call 800-525-2225 or go to for more information. Thank you for your comment.