New Targeted Drug Therapy for Advanced Sarcoma Extends Survival by Nearly a Year

MSK physician Aimee Crago consults with two colleagues.

MSK is leading the trials of olaratumab, a promising targeted therapy for treatment of advanced soft tissue sarcoma.

Editor’s note: On April 25, 2019, Eli Lilly, the company that makes olaratumab (Lartruvo®), announced that it was withdrawing the drug from the market. This move came after results from a phase III clinical trial found the drug did not improve survival for people with soft tissue sarcoma. The company said it was establishing a program that would allow people who have benefited from the drug to continue to have access to it.

Original post: A new targeted drug therapy is being shown to extend the lives of people with advanced soft tissue sarcoma by almost a year. Data from a multicenter phase II clinical trial demonstrating these results were published June 9 in The Lancet.

Based on the encouraging outcome, the US Food and Drug Administration has granted the drug, called olaratumab, Breakthrough Therapy status. This designation speeds up the development of drugs that are considered particularly promising. An international phase III trial is already under way, with MSK as the lead center.

“Soft tissue sarcoma is a rare disease with limited treatment options,” says Memorial Sloan Kettering medical oncologist William Tap, the study’s lead author. “The fact that this drug improves overall survival this much is unprecedented. It’s off the charts from anything we’ve previously seen.”

Targeted Drug Therapy for an Uncommon Disease

Soft tissue sarcomas affect the body’s soft tissues, including muscle, fat, tendons, nerves, and connective tissues. There are many subtypes of sarcoma, all of which differ in where they originate and also how they behave. The American Cancer Society predicts that more than 12,000 people in the United States will be diagnosed with a sarcoma this year. When the disease is caught at an early stage, it often can be completely removed with surgery. But once it has spread, it’s very difficult to control.

The current study included 133 patients with a variety of sarcoma subtypes who were randomized to receive either olaratumab combined with the chemotherapy drug doxorubicin or doxorubicin alone, which is a current standard treatment. All of the patients had advanced disease that had spread beyond the original location of the tumor. Some had previously been treated with other drugs and others were newly diagnosed.

[This drug] is off the charts from anything we've previously seen.
William D. Tap medical oncologist

The average survival of patients who got the combination was 26.5 months, compared with 14.7 months for patients who got standard treatment, an improvement of 11.8 months.

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A Sarcoma Treatment Option that Works for Many Subtypes

Olaratumab is a targeted antibody drug that binds to and blocks a receptor called PDGF-alpha that’s found on the surface of certain cancer cells and in the tumor microenvironment (the area surrounding the tumor).

The most frequent subtype represented in this trial was leiomyosarcoma (a cancer of smooth muscle cells that is a more common type of sarcoma). The drug appeared to work particularly well in this disease. “Something else that was remarkable was that it worked so well in patients who had already received prior therapies,” Dr. Tap says. “The first therapy a person receives for a new diagnosis often has the best chance of controlling their disease, so the fact that we saw such a benefit in patients who had received prior therapy was pretty astounding.”

Olaratumab showed benefit in combination with doxorubicin. According to Dr. Tap, “Olaratumab may be working in more than one way, including by possibly acting on the tumor microenvironment. How the drug works in sarcoma is the focus of several ongoing basic science and clinical research programs.”

Researchers are conducting laboratory studies to learn more about how olaratumab works.

The side effects experienced by patients in the trial were mostly those that result from doxorubicin. There was a slight increase in a condition called neutropenia — low white blood cell counts that can increase the risk of infections. But Dr. Tap says those increased rates did not correspond to patients needing to be hospitalized.

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This study was funded by Eli Lilly, which developed the drug.


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I need your help. I am in advanced stages and cannot get an out of network authorization from IL Medicaid to be seen at your hospital. Please let me in one of your studies!

Dennis, we’re sorry to hear you’re going through this. Please contact our Physician Referral Service at 800-525-2225. They’ll be able to speak with you about clinical trials at MSK and can answer questions about insurance coverage as well. You can go to for more information on making an appointment. Thank you for your comment.

Is Olaratumab only effective when used with Doxorubicin or is it effective on it's own, as a single agent?

Diane, olaratumab does not work as a single agent on its own, but only in combination with chemotherapy. Thank you for your comment.

now that otaratumab has worked in combination with doxorubicin in extending life, is there plans of using your new drub with another Chemotherpy Drug anytime soon? I have used doxorubicin and would not qualify for this trial because of this.

Warren, according to Dr. Tap, combination studies with other chemotherapy drugs are being planned. A study with gemcitabine and docetaxel and olaratumab will open soon. You may want to periodically check on to monitor when that study is open. Thank you for your comment.

Can this treatment be found in UK? Or only USA

Steve, according to Dr. Tap, there is a trial planned for the Royal Marsden Hospital in London, to be led by Robin Jones. You can contact the hospital for more information. Thank you for your comment.

A patient who has been already treated with doxorrubicin, trabectedin,pazopanib and others chemotherapy can take olaratumab?

Will this work for DSRCT of the epididymis?

April, we sent your question to Dr. Tap, who responded that he and his team are interested in looking into the combination of chemotherapy and olaratumab in patients with DRSCT [desmoplastic small cell round tumor]. But he said it is hard to know in advance if it would help and if so how much. There is currently a clinical trial of gemcitabine and docetaxel with or without olaratumab, and he would consider this option in patients with DRSCT who have had standard chemotherapy. If you’d 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.

I am a patient of advanced soft tissue Sarcoma, precisely Synovial Sarcoma. I want to thank Dr. Tap for such a nice article. I want to know if gemcitabine/ Docetaxel can be administered with olaratumab to patients with chronic wounds as I am having one. Thank you in advance

Arnab, we sent your question to Dr. Tap, who responded that it would be difficult to say without more information — eligibility for the clinical trial would be based on if it was safe to administer the chemotherapy with the presence of a chronic wound. The trial has strict criteria in this regard. Your would have to be evaluated by a center in which the trial is open if you would like to be considered. If you’re interested in coming to MSK, you can call 800-525-2225 or go to for more information on making an appointment. If you’re interested in being treated elsewhere you can got to to find a trial that is in a better location for you. Thank you for your comment.

Really fascinating! I'm a 23 year childhood cancer survivor of stage III rhabdomyosarcoma. I love reading studies like this and the hope for the future!

I have had two Leiomyosarcomas and have had successful surgical removal. The first time was 20 years ago and after surgery I was treated with adromycin and cisplatten, a nightmare of side effects, but I went 11 years before it came back. I had another huge surgery and then gemcetabine. Then I went 4 more years and then had surgery at MSK with Dr. Sam Yoon, who told me that this was called a PEComa. That was two years ago. Each time they felt they got it all. This year it metasticised to my lung. Very small, and robotically removed. Also a PEComa. Now here I am, praying I remain in remission. My question for you is it hopeful that this new drug for soft tissue sarcomas will be improved to the point of stopping these growths forever? It must be your goal, but is it reasonable to be optimistic?

Ellen, because this drug is still in experimental trials, it’s too early to predict what its long-term benefit will be. Thank you for sharing your story and for your comment.

We leiomyosarcoma patients are SO in need of treatments! Please don't forget about us and THANK YOU MSK and Lily for all you are doing! Often the rare diseases are overlooked in deference to drugs that will make a lot of money. The LMS patients need drugs to fight this horrible disease and I just want to thank you!!

Can this drug be used with gemcidibine? My moms doctor is Dr. Hensley shouldn't we be told about this? Maybe my mom will be a candidate.

Michelle, according to Dr. Tap there are trials planned that would look at gemcitabine and docetaxel together with olaratumab. It’s important to know that olaratumab is still experimental and only being tested in a small number of patients as part of clinical trials. If you mother is interested in participating in a trial we recommend she speak with Dr. Hensley about whether this appropriate for her situation. Thank you for your comment.

Extremely impressed hearing and meeting Dr Tap at Leiomyosarcoma Retreat in April. On Yondelis 16 months, mets remain stable in spine, liver, lungs and iliac. P53 mutation. Gem/tax in 2011 and 2014. Two new scalp mets biopsied in June. Any chance of Olaratamaub + Trabectiden combo? I think I need to see Dr Tap.

Dear Daryl, if you would like to make an appointment for a consultation with Dr. Tap, please call our Physician Referral Service at 800-525-2225. Thank you for reaching out to us.

Any possibility olaratumab will be combined with Trabectidin? Pls tell Dr. Tap that "The Unsinkable Daryl Brown" from Baltimore is asking. Thanks!

Hi Daryl, Dr. Tap said that numerous combinations are being discussed and there is much interest in combining olaratumab with trabectedin, but he is not aware of any trials yet. Thank you for your comment.

Great information & hope for the future . I am a 32 years survivor treated at Msk with surgery & radiation Brachy therapy . So far so good thanks to God & Msk I am alive & . in future if I should again Need the services again I will be calling.

Dear Maria, we are glad to hear you felt well-cared for by your MSK care team. Thanks for sharing your experience on our blog!

I had leiomysarcoma/retro peritoneal surgically removed with tot historectomty in Dec.18/2015. chem with Gen/Tax.It has now spread to lung,spine and pelvis. I'm a young healthy 77yr old Fe. I need a new drug! Help Christina Eckert

Hi Christina, MSK currently has two trials open for uterine sarcoma. You can view them here: If you’re interested in making 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.

I am taking Votrient, but am not tolerating it well. I just read about Dr. William Tap and the phase 3 trial of olaratumab.Any chance of that drug helping me? Thank you, Kate G. Reiner

Dear Kate, we’re sorry to hear that you’re having side effects. Olaratumab is now FDA approved for use in combination with doxorubicin, so you don’t necessarily need to be in a trial to take it. It is still being evaluated in trials in combinations with other drugs. We recommend you discuss with your MSK oncologist whether this drug may work for you. Thank you for your comment, and best wishes to you.

My mother is a patient at MSK and was diagnosed with stage 3 uterine leiomyosarcoma. She has had her tumor removed already with hysterectomy. Would these drugs be a possibility for her? Thank you.

Dear Beth, it’s possible that your mother may benefit from this drug. We recommend that she discuss it with her MSK team. Thank you for your comment, and best wishes to you and your family.

Hi MSK, may i ask on the current status of the olaratumab drug? The article was published in 2016 where it's in clinical trial phase II. How is the progress since then? I am an overseas patient, Stage 4 LMS and I am interested in enrolling for further clinical trials,

Olaratumab was approved by the FDA for use in the United States in October 2016. It was approved to be given in combination with the chemotherapy drug doxorubicin. There are trials ongoing to test it in combination with other chemotherapy drugs. You will need to check the status of its approval in the country that you are in. Thank you for your comment, and best wishes to you.

Hello. Has Olaratumab been tested on people who have Aveolar Rhabdomyosarcoma? If so, has it been proven to be beneficial along with standard treatment of VAC?

Dear Kim, according to Dr. Tap, it hasn’t been tested with the combination of VAC yet. He said there is an early clinical trial in pediatric malignancies combining it with multiple combinations, so this may be an option. Thank you for your comment, and best wishes to you.

Hi why cant you have olaratumab if you had doxurubician for lms sarcoma

Dear Pauline, olaratumab is currently FDA approved for use in combination with doxorubicin. If the patient can no longer receive doxorubicin, he or she would not be a candidate for this treatment. However, olaratumab is being tested in clinical trials with other chemotherapy drugs. If you or someone you know is interested in making an appointment for a consultation, the number to call is 800-525-2225. You can go to for more information. Thank you for your comment.

I am a pacient of advanced liposarcoma. I've been in this process for 5 years now, I would like to know everything related to this treatment such as the cost and the viability of it working for my case. If you currently have any experimental treatment that you are working on I would be more than welcome to try it.

I have advanced stage Leiomyosarcoma right now it's in my pelvic area it's 10 cm and limp nodes in my stomach is bigger my last treatment didn't work and every were I called they don't except Humana hmo or I'm not in network could you help me please..I want to live

Dear Vickie, we’re very sorry to hear about your diagnosis. Please call our Patient Access Service at 800-525-2225. They will be able to answer your questions about coming to MSK for a consultation or treatment, including questions about insurance coverage. Thank you for your comment, and best wishes to you.

HI my husband Frank is a patient at MSK currently for the past 3 years with LMS He's receiving Doxil right now could he benefit by adding this new drug to the doxil??? Please let us know we will be up in city on the 21st. Thank you

Dear Janet, we recommend that you discuss this with your husband’s doctor when you are at his appointment next week. Thank you for your comment, and best wishes to both of you.

I keep hearing that Sarcomas are so rare, yet it seems like many people have it and yet there is nothing worth while to treat it with. As much money that is donate to the Cancer Association they have yet to come up with a cure. But, man can travel in space!

Hello are these trials available to someone diagnosed with uterine leiomyosaromca with Mets to spine? 3yr survivor previously treated with gemcitabine and docetaxel since disease has spread to spine physicians are saying there is more they can do. Is Olaratumab responsive to metastasized sarcomas?

Dear Debra, we’re sorry to hear that your cancer has spread. Because olaratumab is now approved by the FDA, you don’t need to be part of a clinical trial to receive it. We recommend you discuss with your oncologist whether this drug may be right for you. If you’re interested in making an appointment for a consultation at MSK, you can call 800-525-2225 or go to for more information. Thank you for your comment, and best wishes to you.

Have any studies been done to look at efficacy of immunotherapies on metastatic clear cell sarcoma (untreated as of yet other than initial resection)? And would running a MSI or MMR test be worthwhile to assess potential application of Keytruda?

Dear Peter, there have been no trials looking at immunotherapy specifically at this subtype of sarcoma. We recommend that you discuss with your doctor whether testing for MSI or MMR might be appropriate. If you would like to arrange for a consultation 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.

Hello , are there any positive advances in the treatment of synovial sarcoma my son has metastatic high grade but in the 12 months since diagnosis has had good response to 6 months doxi and ifos then 3 months later a 12 rounds of radiation then 3 months later he will start Pazopanib in 2 weeks, we live in Australia and want to know if there’s trial in the US he may qualify for or success with past trials , Thankyou