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

By Julie Grisham,

Monday, June 13, 2016

MSK physician Aimee Crago consults with two colleagues.

A phase II clinical trial for sarcoma has shown that the targeted drug therapy olaratumab extends survival by nearly a year for people with advanced disease. The study’s lead investigator says the results are “unprecedented” for this rare disease, which currently has very limited treatment options.

  • Soft tissue sarcoma is a rare disease that currently has few treatment options.
  • Based on the results of a recent clinical trial conducted at MSK, the FDA has granted olaratumab Breakthrough Therapy status.
  • The drug appears to work particularly well in leiomyosarcoma, a common subtype.
  • MSK is leading a phase III trial to continue studying the drug.

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 the disease, 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
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.


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?

Eduardo, there will be a clinical trial evaluating gemcitabine and docetaxel together with or without olaratumab added. If you are interested in participating in the trial at MSK, you can call 800-525-2225 or go to for more information on making an appointment. Thank you for your comment.

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.

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