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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Dear Debra, we’re very sorry to hear about your son’s diagnosis. We recommend that you check with, a database of clinical trials for various diseases that are being conducted all over the world, including in Australia. You may also want to consult with MSK’s International Center. They can be reached at [email protected], or you can go go for more information. Thank you for your comment, and best wishes to you and your family.

Can the Olartumab be given with Doxil instead of Doxorubicin?

Dear Karina, MSK is not currently looking at this combination, but you can go to to see if there are trials at other hospitals that are. Thank you for your comment.

I was a participant in the double blind study using doxorubicin & olaratumba . When will the results become available?
My oncologist at the University of Michigan now has me on the palative care chemo trabectatin . Can I assume that I did not receive the olaratumba during the study?

Dear Patricia, we recommend you discuss these questions with the healthcare team that oversaw your care during the trial. Thank you for your comment, and best wishes to you.

My husband was diagnosed 07/17 with Leiomyosarcoma, 26 radiation treatments, full pelvic exenteration in Dec. Feb/18 Mets to brain, surgery to remove, Mar, infection removed another tumour, scan showed Mets to lungs. June whole head radiation, now on round 5 of Olaratumab/Doxorubicin. We are in NB, Canada. Presently only patient on Olaratumab, last lung scan showed improvement. Very rare in our Province, looking for prognosis. Current brain tumour has shown shrinkage with radiation. Thank you

Dear Sonja, we’re sorry to hear about your husband’s diagnosis. If you would like to arrange for a consultation with one of our experts, including speaking with someone about having his records reviewed, please contact our International Center at [email protected]. You can go to for more information. Thank you for your comment, and best wishes to you both.

Hello I'm from australia and my husband was diagnosed with dedefferentiated chondrosarcoma which has now progressed to lung metastisis have these drugs been tested with this subtype of sarcoma. And if so has there been any positive outcome so far. thankyou

Dear Georgia, we’re very sorry to hear about your husband’s diagnosis. We recommend that you consult with an academic medical center in Australia. If your husband is interested in participating in a clinical trial, you can go to to look for trials for his type of sarcoma. Thank you for your comment and best wishes to both of you.

My Mother 59 Yrs, has been diagnosed with Metastatic PEcoma, with primary at Uterus and metastatis at left lung and Brain in March. The cancer was identified through brain surgery in March'19. She was given mTor inhibitor ( Everolimus 5 Mg OD) since 1st May'19 for 35 days after which she has major stomach ache and vomiting. Based on CT report doctors have now informed that disease has progressed into liver, right lung and intestine.
They have told there is no approved Chemo for this diseases. There is no disease progression in Brain as of now.

Any medicine we can try now ?

Dear Mohit, we’re very sorry to hear about your mother’s diagnosis. If you would like to speak with someone at MSK about what her treatment options are, you can arrange a consultation by calling 800-525-2225 or you can make an appointment online. Thank you for your comment and best wishes to you and your mother.

Any new clinical trials for Leiomyosarcoma? Recently heard of Alpha Dart Radiation Therapy at Sloan but not for Leiomyosarcoma. Sarcomas are so vicious and I hope you will consider trial using Alpha Dart in Sarcomas. My husband has very large primary attached to outside of stomach with two mets to liver. Need more trials for LMS.

Dear Lori, we’re very sorry to hear about your husband’s diagnosis. If he is interested in arranging a consultation to learn about clinical trials at MSK, you can make an appointment online or call 800-525-2225. Thank you for your comment and best wishes to both of you.