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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my late wife was a patient of Dr Dickson's and passed away last year from Follicular Dendritic Cell Sarcoma. what made her mad was the lack of research and new drugs being introduced to tackle rare cancer types like Sarcoma. so its great to read this article on new treatment options and I hope more research will follow.

Thank you Dr. Schwartz and Dr. Dickson, for your hard work. I am a patient at Memorial Sloan-Kettering Cancer Center, I'm a patient of Dr. Keohan, Dr. Singer and Dr. Bains. Right now I'm NED (14 months) since my last surgery.
My question is, is there any promising treatment or targeted therapy for Synovial sarcoma? I had AIM in 2007 did well shrank my tumor and cleared my caking in my omental wall I was able to have surgery With Dr. Singer. In a had a small recurrence in my left lung in 2010, no chemo just surgery with Dr. Bains. In 2011 I had a larger recurrence bilobial masses again in my left lung this time chemo Ifosfomide and Etopiside after two rounds they scanned but no change at which point surgery was recommended. I had surgery with Dr. Bains in Feb 2012. I was told that the chemo didn't work. I'm scared I know this disease can come back at any time, I have spoken to many synovial sarcoma patients everyone has such different results with there treatments. Please help all sarcoma patient we need something to help win this battle.

Elizabeth, we are investigating many targeted therapies for sarcoma, including some that may work for synovial sarcoma. If you have specific questions, you should speak with Dr. Keohan. Thank you for your comment.

This is a great news. One of my friend in India is having bone sarcoma. He has taken chemo, Surgery is not good in his case and chemo is not affecting. Family is worried. I was workign with MSKCC for 6 years as a postdoc and now back to India. Can I forward his reports and test reports for your expert advise please?
Dr Nirmala Jagadish

My little brother was diagnosed with a rare sarcoma called Desmoplastic small round cell tumor, I was hoping to find any promising treatment for this type of cancer.
your response is appreciated.

My wife and I traveled to MSKCC from the midwest in 2006 to consult with Dr Schwartz regarding her recent surgery for GIST. The hope and peace of mind from our consult with Dr Schwartz was of great comfort to us. She is NED. The gentle bed side manner and manifested competence of Dr Schwartz was an immeasureable blessing to us in this crisis. As a physician myself, I am inspired, as I hope others are, to emulate Dr Schwartz in caring for others.

My patient is under the treatment of Dr Schwartz since 2008 and he is now almost cured of his Soft Tissue Sarcoma. Dr Schwartz has done a terrific job of taking care and comforting my patient with the most modern and latest therapies at his command. We have been traveling all the way from India for his treatment and it was a very fulfilling and rich experience. I wish Dr Schwartz and Dr Dickson ( who saw my patient initially when he was a Resident ) all the success for new therapies for Sarcomas.

Hi, sir. I am a doctor in Brazil, i am urologic. My nephew had diagnosticed with angiosarcoma in periorbital to right. No have mestastatic but had infiltration in soft tissue. How can I send the exams to you or this center for an initial evaluation.

Ronaldo, if you would like to arrange for a records review or to learn more about coming to Memorial Sloan-Kettering as an international patient, please visit the page on our Bobst International Center to learn more about it. Thank you for your comment.

I am having chondrosarcoma in my right shoulder. Undergone surgery three times. Recurrence is there. I am in India. How can I approach you? Tentative expensive for indoor treatment.

I am newly diagnosed with endometrial stromal sarcoma - low grade. I have had surgery but no other treatment so far except progesterone. I hear this can be a tricky cancer/diagnosis and treatment methods seem undefined. How do you treat this cancer at your facility?

Thank you so much for the hard work you are doing
I hope to keep tabs on your work! I just had my first surgery to remove a tumor May 2nd. Pathology came back as a dedifferentiated Liposarcoma,high grade. My team is going to start radiation on me in a couple weeks. I'll be sharing this news with them. Do you/have you worked with the James Research & Cancer Hospital at Ohio State University or Dr. Raphael Pollock at all (he is leaving MD Anderson and coming to The James)

Nico, we forwarded your question to Dr. Schwartz, and he said he and his colleagues work closely with Dr. Pollock. If you would like to come to Memorial Sloan-Kettering for a second opinion, you can call 800-525-2225 or go to Thank you for your comment.

My husband has Spindle Cell Sarcoma. After 25 rounds of radiation, he had a tumor removed (the tumor burst during removal) from his armpit last July. He then had 15 more rounds of radiation. In December, his scan showed that it has spread to his lungs. He also has a lesion in the shoulder that he had surgery on. He has started a third chemo treatment.....the first 2 kinds did not help. He feels that the treatment he is taking now is not helping either. The pain in his shoulder just gets worse and worse, and the pain medication is not helping much. He coughs all the time. How do you treat this cancer? He is 60 years old.

My granddaughter was just diagnosed with Rhabdomyosarcoma. She is 21 years old. Is this considered a child's desease? Does your center have experience treating this type of cancer? Which doctor should we approach?

Haroula, rhabdomyosarcoma is generally considered a pediatric disease, even when it affects young adults. You can learn more about our expertise in treating the disease here: To make an appointment to speak with one of our doctors in the Department of Pediatrics, you can call 212-639-5954 or go to for more information. Thank you for your comment.

My wife had a 2.5 cm tumor in t1 nerve seath of the left bracial plexus which caused an onset of severe pain along the ulnar nerve. A Pet scan showed no molecular activity. A qualified surgeon operated, initially thinking that the tumor would be benign, removed the tumor in pieces and saved the nerve. Initial frozen pathology on the day of the surgery confirmed it was benign. However the second, more in depth pathology indicated synovial sarcoma (mono ssx2, grade low to intermediate. ) We have had varying opinions. From surgery to remove the nerve ( to achieve good margins), nerve grafts, all types of radiation and chemotherapy in varying doses. The sticking point for us is chemo - it doesn't seem by our research - becuse of the inactivity of the pet scan, the size, the brain blood barrier that chemo to offers much benefit but several doctors are strongly of the opinion that it is nessesary. Does memorial- Sloan have a treatment protocal, specifically on chemo for synovial sarcoma.

Keith, to learn more about how Memorial Sloan-Kettering treats soft tissue sarcomas, include synovial sarcoma, you can go to We also have several clinical trials for sarcoma. If you’d like to speak to someone at Memorial Sloan-Kettering about receiving a second opinion or coming here for treatment, you can call 800-525-2225 or go to for more information. Thank you for your comment.

I lost my Dad last March due to soft tissue sarcoma. He was a patient at MSK. He received the best care I have ever witnessed, which says a lot as I work in the healthcare field. He responded well to 2 different rounds of chemo. The meds attacked the tumors without damaging him. He was happy and active until the day before he died. His cancer was very advanced when it was found. His family doctor did not think he would live 6 weeks. With new chemo, he lived well. If people and the government continue to support research and new treatment options, this horrible form of cancer could become survivable. Thanks to all of you at MSK for what you did for me and mine, and what you continue to do for others.

I am a 32 year old female and I just had a CAT scan on the back of my upper arm and shoulder blade. The radiologost and my primary care doctor think I could have a liposarcoma or lipoma tumor. They are recommding surgical removal. My questions are what type of surgeon should I have remove the tumor, does MSK have doctors who perform this surgery, and should I wait for the biopsy results before making an appointment at MSK if it is cancerous? Thank-you!

My 21 year old son was diagnosed with synovial sarcoma and is currently receiving treatment at Children's Hospital and University of Alabama Comprehensive Cancer Center in Birmingham, AL. The plan is that surgery will follow chemotherapy and radiation to shrink this high grade tumor, which through extensive testing, has been determined to be localized to his right knee and is 6cm. We have been referred to a surgeon at UAB. I need a recommendation for a surgeon in Birmingham and we will also want a second opinion, as I'm told we will be presented with surgical options for knee replacement, other preservation techniques or not. Please advise.

Dear Eve, we’re sorry to hear about your son’s diagnosis. Unfortunately, we cannot provide medical or treatment advice on our blog. If you would like to seek a second opinion from one of our doctors, you can go to to learn more or call 800-525-2225 during the week to speak to one of our Physician Referral specialists. You may also wish to contact the National Cancer Institute at 1-800-4-CANCER or learn more from the NCI website at Thank you for your comment.

My mother has undergone a surgery (Well differentiated Retriperitonial Liposarcoma) 10 days back here in India, Doctors got succeed in removing the complete Tumor....would you please help me with the details of further treatment and info, Thank you so much.

My niece has been diagnosed with stage 4 Epithelioid Angiocaroma of the peritoneum after going into urgent care for stomach pains the day before Thanksgiving. She is only 19 and basically a healthy active young adult. She is now under the care of UCLA Med Center which we hope is best for her but given the rarity of this cancer we are hoping to get additional consultation or collaboration with her doctors at UCLA. How can we request this or get MSKCC interest/involvement?

My uncle is diagnosed with dedifferentiated retroperitonial liposarcoma 6months back in india.he had undergone 2 surgeries 1 for abdomen which they removed 21 inches tissue and other for kidney. He was responding properly to 2 cycles of chemo but after the recent one he was collapsed and organs didnt work for some time but by gods grace he is fine now. Sarcoma in abdomen is 16 inches now can you please suggest us with what could be the further treatment.

Dear Jacks, unfortunately we cannot answer personal medical questions on this blog. But if your uncle is in India and would like to learn about coming to Memorial Sloan-Kettering or in having his records reviewed he (or you) can contact our International Center at 1-212-639-4900 or go to Thank you for your comment.

My friend has just last week been diagnosed with follicula dendritic cell sarcoma.the doctors here in australia have never treated anyone with it. We are desperatly looking for learnings and treatments which can help are you able to give us a contact so,that our doctors can speak to,you about the best treatments.
Yours sincerely.

I have been dealing with Leiomyosarcoma for 26 years. At one point , i went 7 years and 10 months without any tumors. I have recently been diagnosed by Dr. Singer , with yet another tumor. This will be my 9th surgery (5 at MSKCC with Dr. Brennan and Singer) and my second time for chemo. Dr. Dickson is wonderful . He is so kind and knowledgeable and really listens to your concerns. The whole staff is great. If you must go somewhere , go to MSKCC. My first 3 tumors went undiagnosed and finally someone said "go to Sloan". The rest is history. In between treatment , I live a normal life. I am a wife (of almost 50 years) a mom, grand mom, retired RN and teacher. I pursue many hobbies and Sloan has kept me living and loving life. I am a poster "child" for their care---26 years ago I was told to prepare for the worst---now I face this new challenge hoping again for the best with the help of my team.

what are the treatments or your thoughts on ewing sarcoma after having surgery then it returning to the same spots only to be in different spots though. They were originally in the right lung and growing into the heart but now are growing around the heart and in the other lung. We have sent our information to the hospital regarding this issue from the hospital we are currently at ( Fox Chase Cancer Center) my wifes name is Marnie Houck
all of the information on here looks very promising with the trials.

My brother was recently diagnosed with undifferentiated pleomorphic sarcoma. A roughly 3 cm mass was removed from his inner thigh in Dec 2013. He underwent 4 weeks of radiation treatment in March and had the region excised with wide margins in June. Chest CTs and thigh MRIs done before and after the radiation were negative as was the tissue removed in June. My question is should the follow-up CTs be done with PET scans? My brother is being treated in Canada and is told that PET scans are not appropriate for this type of cancer. My impression is that PET scans are done routinely in the US for following up on many, if not most, cancer types. Also, could you let us know about any new treatments or clinical trials for undifferentiated pleomorphic sarcoma. The only treatment for recurrence of this kind of sarcoma that we are aware of is doxorubicin and that drug is apparently not very effective. Thank you.

Christopher, we are sorry to hear about your brother’s diagnosis. We recommend that you speak with his treating oncologist about what type of follow-up tests he should be having. If you are interested in finding a clinical trial for your brother, you might want to go to This database, maintained by the US National Institutes of Health, has information about clinical trials all over the world. If your brother is interested in coming to MSK from Canada, you can contact our Bobst International Center at [email protected] or go to for more information. Thank you for your comment.

My mother was recently diagnosed of Leiomyosarcoma of the uterus in India. It was found out free she underwent a total hysterectomy. There were no metastatis in the CT and the oncologist has started her on GemTax chemotherapy.. is there anything else that we could do?

Dear Shilpa, unfortunately we can’t provide personal medical advice on our blog. If you’d like to get a second opinion for your mother from a Memorial Sloan Kettering doctor, you can call 800-525-2225. If you are presently in India, then you may be able to arrange a medical records review by mail through our center for international patients. Here’s more info on our international center: Thanks again for contacting us, and we wish you and your mother all the best.

Husband diagnosed last year with Epithelioid Sarcoma. Primary tumor around 3cm left hip/thigh area-"Whoops" procedure. Clear margins after resection. Now found in lungs-just had rt lung wedge resection. Other nodules on left lung. MSKCC Dr. DeAngelis said chemo, radiation not affect this subtype. Our oncologist wants to try it on lung. Would my husband be better served by getting second opinion? Are there new therapies to treat this type? Thank you for your time!

Dear Susan, thanks so much for reaching out. Unfortunately, we cannot give personal medical advice on our blog. What we can suggest is that you call one our appointment specialists to discuss getting a second opinion. The number is 800-525-2225, or you can read more here: When you call, you might also ask for advice on any clinical trials (studies of new experimental therapies) that might be appropriate for your husband. Thank you again, and we wish you both all the best.

My brother is 56 year old,was diagnosed with dedeffereniated liposarcoma Rt Anterior thigh 6 month ago.operated then recurre near the same area. We do not have an expert who can deal with sarcoma medically and we can not come to United States
Can you suggest any hospitals in Europe or any expert in sarcomas in Europe
Or Asia
Or Middle East
Mr. Kamal Mohamed

Dear Kamal, we are sorry to hear about your brother’s diagnosis. Our International Center may be able to suggest hospitals in your area that can treat this type of cancer. You may email them at [email protected]. You may also be interested to know that our physicians do medical records review by mail. Here is a link with more information:…
Thank you for your comment.

My son who is now 30 years old had in all three surgeries so far. Last year in Feb, he was diagnosed with Retriperetonial Mass which upon biopsy was found to be sarcoma. The mass was surgically removed after 8 weeks of radiation. After six month when he underwent full body scan they found a subcentimeter nodule in left lung. Just because he had history, the thoracic surgeon suggested to get it out by surgery. The biopsy diagnosed spindle cell. By the time he recovered from this surgery, in the next scan which was after three months, they found one more very small nodule, the Oncologist said the only option is surgery as this will not have any effect of radiation or chemo to which we also cuncrred after reading whole lot on internet. He is now recovered but we afraid it may come back. Is there any way you can help my son? Also is there any other treatment other then surgery? Can we get appointment with Dr. Gary Schwartz?

Dear Nick, thanks for writing us. You can read more about the types of treatment we offer for sarcoma here:…. But we’re not able to make treatment recommendations for individuals on our blog. If you or your son would like to speak to an appointment specialist the number is 800-525-2225, or you can go here to learn more about making an appointment: Dr. Schwartz is no longer at MSK, but you can see our full list of sarcoma experts here:…. Thanks again for reaching out to us.

I have attempted to call the patient referral line multiple times to set an appointment. A father in my son's school has a Soft Tissue Sarcoma and is in dire need of a second opinion, as it has come back with mets. My husband was treated at Sloan Kettering and it is the best! I am told he can't get a second opinion here due to currently taking chemo (which is experiential chemo out of his own pocket). Do you have any suggestions on how I can make a second opinion with your experts happen?

Dear Alissa, we would like to help the patient. Can he give us a call during regular business hours at 646-497-9067? Please tell him to ask for Sue and explain that his friend left a comment on MSK’s blog. Thanks so much for your question.