Including AYA Patients in Cancer Clinical Trials


MSK physicians and researchers design and conduct clinical trials that include adolescent and young adult (AYA) cancer patients, ensuring they are not lost between the typical siloes of adult and pediatric cancer trials. As a result, more AYA patients benefit from an increasing number of opportunities to access experimental therapies that may improve their outcomes.

Legacy of Inclusion

Medical oncologist Alexander Drilon is Chief of MSK’s Early Drug Development Service. He specializes in lung cancer and early-phase clinical trials

Medical oncologist Alexander Drilon is Chief of MSK’s Early Drug Development Service. He specializes in lung cancer and early-phase clinical trials

Medical oncologist Alexander Drilon, MD, helped design three multicenter, open-label, single-arm clinical trials of larotrectinib for patients with TRK-fusion positive solid tumors. The three studies include a phase I study for adults, (1) a phase I/II study for children, (2) and a phase II basket study for adolescents and adults. (3)

Larotrectinib demonstrated rapid, durable antitumor activity in all age groups, with an overall response rate of 75 percent and 71 percent of responses ongoing and 55 percent of patients remaining progression-free at one year. (4) Based on these results, the US Food and Drug Administration (FDA) granted accelerated approval to larotrectinib in 2018 for adult and pediatric patients. (5) This was the first approval for a targeted therapy at the same time for children and adults with TRK fusions and the second tissue-agnostic approval for cancer treatment. (5)

Dr. Drilon also helped design clinical trials for selpercatinib, a targeted drug for patients with RET fusion-positive non-small cell lung cancers and thyroid cancers. He partnered with the drug manufacturer to ask the FDA to include patients as young as 12. The FDA granted accelerated approval to selpercatinib May 2020 (6) based on trial results showing substantial and durable antitumor activity with an objective response rate of 64 percent over a median duration of 17.5 months and an 85 percent response rate among previously untreated patients. Selpercatinib also demonstrated a promising intracranial response rate, an important factor given the high lifetime risk of brain metastases. (7)

Current Study Underway

Pediatric oncologist Emily Slotkin is a specialist in pediatric sarcomas

Pediatric oncologist Emily Slotkin is a specialist in pediatric sarcomas

Pediatric oncologist Emily Slotkin, MD, is leading the first clinical trial investigating the safety and efficacy of the experimental drug prexasertib in 30 MSK patients older than 12 months with relapsed or refractory desmoplastic small round cell tumor and rhabdomyosarcoma. Prexasertib is a checkpoint kinase inhibitor that interferes with DNA repair mechanisms and promotes apoptosis. This novel dose-escalation study is testing the safety and efficacy of prexasertib when given in combination with standard drugs irinotecan and temozolomide. It opened in 2019 for pediatric, AYA, and adult patients at the same time, allowing all sarcoma specialists at MSK to provide their patients with an opportunity to participate. (8)

Cancer specialists at MSK’s new Lisa and Scott Stuart Center for Adolescent and Young Adult Cancers are focused on developing more trials for AYA patients. At the same time, they will make it easier for AYA patients to learn about clinical studies and enroll, regardless of whether their primary physician cares for adults or children. Recognizing that AYA patients place a premium on convenience, MSK is now offering more trials at regional sites and investigating ways to move some parts of trial administration to the virtual space, including electronic consent to participation.

Learn more about how MSK manages AYA patients, including uniting experts across 16 specialties such as medicine, surgery, social work, sexual health, and survivorship, all focused on improving outcomes for cancer patients ages 15 to 39 and addressing the unique challenges they face.

Refer a Patient
Call our dedicated clinician access number at 646-677-7440 or click the link below, and one of our care advisors will assist you with your referral needs.
  1. A Study to Test the Safety of the Investigational Drug Larotrectinib in Adults That May Treat Cancer.
  2. A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children (SCOUT).
  3. A Study to Test the Effect of the Drug Larotrectinib in Adults and Children With NTRK-fusion Positive Solid Tumors (NAVIGATE).
  4. Drilon A, Laetsch TW, Kummar S, et al. Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children. N Engl J Med. 2018;378(8):731-739.
  5. FDA approves larotrectinib for solid tumors with NTRK gene fusions. November 26, 2018.
  6. FDA approves selpercatinib for lung and thyroid cancers with RET gene mutations or fusions. May 8, 2020.
  7. Drilon A, Oxnard GR, Tan DSW, et al. Efficacy of Selpercatinib in RET Fusion-Positive Non-Small-Cell Lung Cancer. N Engl J Med. 2020;383(9):813-824.
  8. A Study of the Drugs Prexasertib, Irinotecan, and Temozolomide in People with Desmoplastic Small Round Cell Tumor and Rhabdomyosarcoma.