Studies Show Promise for Treatment Advances in Several Types of Sarcoma


Sarcoma, a type of cancer that can affect the bones or the body’s soft tissues — such as muscle, fat, and tendons — is often curable with surgery. But for those tumors that cannot be surgically removed or that come back after surgery, there have been few treatment options. This is in large part because sarcomas usually do not respond to traditional chemotherapy.

Targeted therapies, which work by taking advantage of genetic alterations in tumor cells, hold promise for bringing sarcomas under control. Now two trials of experimental drugs are showing encouraging results in treating certain subtypes of sarcoma.

A Diverse Group of Tumors

“Historically, the challenge of treating sarcoma has been that it’s a very complex group of different diseases,” says Memorial Sloan Kettering medical oncologist and sarcoma expert Mark A. Dickson. “Sarcoma is not one cancer, but more than 50 different subtypes. Trials that have tried to give the same drug for all sarcomas have not been successful.”

To develop targeted therapies, investigators need to determine the molecular changes that cause the disease in each of the different subtypes. Memorial Sloan Kettering is a leader in that effort, with a database that combines our collection of tumor samples from patients with information about those patients, such as age at diagnosis and details about their tumors and treatments.

“Our research is focused on looking for genes that are either mutated or amplified,” says Gary K. Schwartz, Chief of Memorial Sloan Kettering’s Melanoma and Sarcoma Service. “If a gene is amplified and makes many more copies than it should, it can often be a driver for cancer.”

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Targeting Liposarcoma

A recent phase II trial led by Dr. Dickson focused on one of the most common types of sarcoma, called well-differentiated or de-differentiated liposarcoma, a more common type of sarcoma that originates in fat cells. The study was published online April 8 in the Journal of Clinical Oncology.

The trial was of a drug called PD0332991 (palbociclib), which targets a protein called CDK-4. That protein is overexpressed in this type of sarcoma because of a gene amplification and leads to the growth of cancerous cells. All patients in the trial had this particular molecular abnormality.

In the study, 30 patients received PD0332991 for 12 weeks. The investigators found that 70 percent of the patients did not have their disease progress during that time, which greatly exceeded the researchers’ expectations. In addition, some patients had significant shrinking of their tumors.

“As far as we know, this is the first time anyone has done a phase II trial for this subtype of sarcoma, and in that way it’s a pioneering study,” Dr. Dickson says. “Based on our results there has been a lot of interest from doctors around the country who want to get their patients on this drug, and we are expanding the trial so that we can treat more patients.”

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Testing Combination Therapies

Another trial, published in the April issue of Lancet Oncology, was led by Dr. Schwartz. In that study, also a phase II trial, investigators combined an experimental drug called cixutumumab with temsirolimus (Torisel®), a drug already approved for some forms of kidney cancer, to treat several subtypes of bone and soft tissue sarcoma.

Cixutumumab is an antibody that targets a receptor on cancer calls known as IGF-1R. Temsirolimus blocks a cancer-related pathway called mTOR. Both IGF-1R and mTOR are critical for the growth of many sarcoma types.

“You can think of mTOR as the engine of the cancer cell that’s responsible for its survival, and IGF-1R as the ignition,” Dr. Schwartz explains. “The idea is that you use cixutumumab to cap the ignition, so that the key can’t go in the lock. But you are also adding the extra benefit of inactivating the engine itself by using temsirolimus to block mTOR.”

“This is the first multicenter clinical study ever conducted to evaluate this combination,” Dr. Schwartz adds.

The multicenter trial, which included 388 patients, found that the number of patients whose disease did not progress after 12 weeks of treatment was improved compared with other treatments. In addition, some patients with a type of sarcoma called solitary fibrous tumor had stable disease for more than a year. Patients with the subtypes Ewing sarcoma, osteosarcoma, and chondrosarcoma had partial shrinkage of their tumors.

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Moving Forward with New Trials

Dr. Dickson and Dr. Schwartz plan to move their research forward with larger, phase III trials, but challenges remain. Dr. Schwartz notes that it is difficult to find funding from pharmaceutical companies to treat sarcomas, which are considered rare diseases. Recruiting enough patients to participate in a larger trial can also be a roadblock.

“For research on these rare cancers like sarcoma, which affects about 13,000 people per year, funding from organizations like Cycle for Survival is becoming increasingly important,” Dr. Schwartz says. “Money from Cycle for Survival funded much of this research, from the basic research in the laboratory up to and including this phase II trial.”

Both physicians have trials under way for other experimental sarcoma drugs and continue to focus on research looking for new targets.

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Dr. Dickson’s research was funded by a Career Development Award from the Conquer Cancer Foundation, Pfizer, and the National Cancer Institute under grant CA047179. Dr. Schwartz’s research was funded by Cycle for Survival and the National Cancer Institute under grants CA148260 and CA140331.


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I had emergency surgery in Jan 2013 for a very large bifrontal brain tumor. The pathology only came up in 2015 when I had a recurrence. It was then noted that I had a Solitary Fibrous Tumor not a regular meningioma. Needless to say, I had a body scan, (clear) and recently had a craniotomy to remove the tumor. The tumor now had hemangiopericytotic transformation in part of it. I am only seeking more information on these extremely rare tumors. I have two wonderful doctors and trust them. Can you offer anymore information? Thank you.

Dear Ellen, we are sorry to hear about your health issues. Soft tissue sarcoma that arises in the blood vessels is called angiosarcoma, hemangioendothelioma, hemangiopericytoma, or solitary fibrous tumor. For more information about these types of sarcomas, please visit…. We hope this is helpful. Thank you for reaching out to us.

My brother was diagnosed with sarcoma. He had a tumor on his inner thigh that the doctors removed, after they removed the tumor when they tested it to see if it was cancerous or not it came back positive that it was cancer. He was home for six weeks and got an infection on his inner thigh where the surgery took place. He went back to his doctor and they opened him up again and cleaned the infection out but then found another tumor that was also cancerous. After they found they tumor they decided to use a sponge to close up the wound and the doctor told him that there is nothing more they can do for him but he has to go to the Moffitt Cancer Center in Tampa,Florida. They now told him that he has six to eight months to live. They want to give him chemotherapy treatments to slow the process down I would like for him to come to Sloan Kettering to get a second opinion. I would like to know if he has coverage in florida would he be able to use it in Sloan Kettering in New York. Thank you for your time

Dear Amelia, we are sorry to hear about your brother’s diagnosis. If he would like to make an appointment for a consultation with one of our specialists, please call our Physician Referral Service at 800-525-2225. They can also answer your insurance questions. For more information about insurance coverage at MSK, please visit Thank you for reaching out to us.

My 23 year old niec was diagnosed with epitheliod sarcoma. Was operated in Japan,then followed through with Proton Therapy,was then put on chemotherapy .
Currently she is looking for any clinical trial or a doctor with a different view on how to threat this.
Please advice.

Dear Lucy, we are sorry to hear about your niece’s diagnosis. For information about our open clinical trials for people with soft tissue sarcoma, please visit…. If she would like to make an appointment for a consultation, please call our Physician Referral Service at 800-525-2225. If she lives outside of the United States and would like to arrange for either a medical records review by mail or an in-person consultation with one of our specialists, please contact our International Center at (or for more information visit Thank you for reaching out to us.

Our 10 year old son has been diagnosed with undifferentiated round cell sarcoma with CIC-DUX4 mutation. The tumor is in the right 12th rib. He is currently undergoing chemo with surgical removal of the rib in the near future, followed by more chemo and radiation. Are there any immunotherapy options or supplemental treatments that will improve his chances of a full recovery? Can you please help?

Hi Mridu, MSK does have some trials open for pediatric sarcoma, although these are for various targeted therapies, not for immunotherapy. If you’d like to learn more, you can call our Physician Referral Service at 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.

Is there any trial or promising Chemotherapy for Radiation therapy induced Malignant Peripheral Nerve Sheath tumor. My niece was diagnosed at stage 2B and chemo is not a promising option.

Dear Debra, we are sorry to hear about your niece’s diagnosis. This is an active area of research at MSK - you may be interested in reading one of our recent blog posts describing advances we have made to control these types of rare tumors:….

To browse through our open clinical trials investigating new treatments for people with soft tissue sarcoma, please visit…. If you have any questions about these studies or would like to make an appointment for your niece, please call our Physician Referral Service at 800-525-2225. Thank you for reaching out to us.

My husband is currently in the trial combining imantinib with MEK162 for GIST would love to talk with anyone else in this trial

Dear Tammie, we are sorry to hear about your husband’s diagnosis. He may be interested in participating in one of our support groups for people with sarcoma. We offer an online group that gives participants the opportunity to discuss issues that may arise through the course of their treatment, to share their personal experiences, and to provide practical and emotional support for one another. To learn more and to register, please visit Thank you for reaching out to us.

My Dad, 57 yo, diagnosed Pleomorphic Sarcoma (MFH) Sept 2015 on his right back femur and operated. After that he got radiated 30x until Des 2015. Then the doctor found metastatic on his lung and he got chemo 6x with doxorubicin until June 2016. When he got PET CT on Oct 2016, the sarcoma come back on back femur above the previous one and the lung sarcoma still there and metas to os sacrum lv 2. He already got chemo 4x with ifosamid, pacxitaxel & mesna. But on CT scan last week the sarcomas are getting bigger. What can we do? Do you have recommended doctor & therapy for my dad? We confuse because in Indonesia there isn't sarcoma center. Can you help us to get treatment there? Thank you

Multiple tumors were found from the MRI on the left leg calf muscles of My close relative. The doctors are calling it Pseudomyogenic hemangioendothelioma. What kind of treatment is recommended ? Will the person need a surgery?

I have a dear friend who was just diagnosed with Spindle. He's been on kidney transplant rejection drugs for years. It is thought that it might be connected. Do you have any information or place I can read up on that if there is any? We are so scared.

Dear Kelly, we’re very sorry to hear about your friend’s diagnosis. For more information, we recommend that you contact the National Cancer Institute’s Cancer Information Service at 800-4CANCER. Thank you for your comment, and best wishes to you and your friend.