Targeted Therapies in Pediatric Cancers

Forum Chairs

Steven G.
Dubois
,
MD
Dana-Farber Cancer Institute
Martine F.
Roussell
,
PhD
St. Jude Children's Research Hospital

Forum Description

Several novel targeted therapies based on rational scientific data hold promise to offer new and effective treatment not only for adult but also pediatric cancers.The new FDA pediatric legislative initiatives has galvanized pharmaceutical companies to extrapolating efficacy from adult data or other data to the pediatric population to streamline pediatric drug development and help to increase the number of approvals for pediatric use. Several clinical trials are ongoing for primary and recurrent and difficult to treat pediatric cancers that hopefully will advance children’s care.

Forum Summary

The Foundation hosted its first forum since the start of the COVID-19 pandemic. For those who were able to gather in person, the meeting took place in Rancho Santa Fe, California. A virtual option was available for those who were unable to travel. The focus of the meeting was Targeted Therapies in Pediatric Cancers. The meeting co-chairs were Steven DuBois (Dana-Farber) and Martine Roussel (St. Jude). Fourteen scientists joined in person and four scientists participated virtually, allowing us to see how a Forbeck Forum might work using a hybrid format.

Several key themes emerged from this forum. First, it is clear that there is an unacceptable delay in getting novel agents into the pediatric clinic. While the RACE Act will help, there remain regulatory issues related to timeline for fulfilling these trials, role of trials in adolescents only to fulfill RACE requirements, and the pressing need for combination studies. Academic and consortium trials need to consider regulatory requirements and provide data that will potentially be “fit for filing” to meet regulatory requirements. Moreover, increasing requirements for companion diagnostics are not in line with clinical practice for broader (rather than more narrow) testing for specific genomic changes. Second, multiple novel agents targeting epigenetic modifiers are in the pediatric clinic and represent opportunities for innovation. Third, there is tremendous interest in cell states as mediators of drug sensitivity and the potential to shift cells into states that are more sensitive. Fourth, a major theme was resistance to targeted therapies, strategies for mitigating resistance including combination approaches, and developing new preclinical models and screens to evaluate novel approaches to overcoming resistance.

Venue & Travel Information

The Inn at Rancho Sante Fe

5951 Linea Del Cielo
Rancho Santa Fe, Ca 92067

theinnatrsf.com
858-756-1131

Travel Forms

Travel forms are due 30 days prior to the start of the meeting to allow enough time to plan transportation.

The San Diego International Airport (SAN) is the preferred airport as it is only 30 minutes from the meeting location.

  • Arrivals - Thursday at 1 PM, 3 PM and 5 PM
  • Departures - Sunday at 10 AM, 12 PM and 2 PM
TRAVEL FORMS DUE:
August 9, 2021
submit travel form

Travel Policy

Please familiarize yourself with our policies and procedures for travel. We truly appreciate you taking the time to participate in this meeting. As you make your plans, please remember that we are a nonprofit organization dependent on donations and volunteers. We do NOT pay for upgrades, change fees, incurred costs resulting from a flight change, transportation to or from your local (home side) airport, meals or other incidentals.

  • Travel Confirmation will be sent out within 1 week of the meeting. This will include a hotel confirmation number, if there is one, and airport transfer details. We have to wait until we receive almost everyone’s travel to book airport transfer. Due to frequent airline changes, we wait until the week of the meeting to send this out.
  • Airport transfer is provided by Foundation staff, volunteers or arranged shuttle at specific times. If you opt to utilize Foundation airport transportation on your travel form, please be patient in receiving this information. We will send it to the week of the meeting.
  • Speaker agenda is not sent out prior to the meeting. It will be provided upon arrival in the meeting packet. We do not tell people when they are speaking because we expect everyone to attend all sessions. Sessions are all day Friday and Saturday.
  • REMINDER: We do not reimburse for home side airport transfer or incidentals while traveling. We will not honor miscellaneous receipts sent for these expenses.
  • Spouses are welcome to come with you at their own cost but are not allowed to attend the meeting. Please no children.

What the Foundation Pays

Accommodations and meals are provided by the foundation during the meeting. Airfare will be covered only if booked through our travel agent. The Foundation will also cover airport transportation on the meeting side at the designated shuttle times. You can select not to utilize Foundation arranged transportation at your own expense when completing the travel form. Once your travel form is received your accommodations and airport transfer will be confirmed. Please let us know of any food allergies or other information we should be aware of on the travel forms.

  • If you would like your airfare covered by the Foundation, you must book with our travel agent. Note we do not cover upgrades, changes, late bookings, etc.
  • Flights must be booked at least 30 days prior to the meeting to confirm your accommodations and airport transfer.
  • As a nonprofit we utilize volunteers and other methods to maximize our efforts (or our donor support) when making accommodations and arranging ground transportation. Ground transportation will be provided upon your arrival either by a foundation volunteer or arranged shuttle. You will be provided airport transportation information the week of the meeting. We do not reimburse for home side airport transfer or incidentals while traveling.

Abstracts

Abstracts are due 30 days prior to the start of the meeting to allow enough time to prepare the meeting book.

The abstracts should be only one or two paragraphs outlining the theme of your presentation and should reflect the objective and spirit of the meeting (see above). Abstracts will be circulated about one week before the meeting. The meeting organizer will start requesting them a month before the meeting.

abstracts DUE:
August 9, 2021
submit abstract

Meeting Structure

The meeting structure has been developed over years of experience.

  • Participants have approximately 45 minutes, depending on the number of participants, for their presentation and discussion. The presentation is meant as a conversation start and should last about twenty minutes briefly covering background information and areas that are new or need further input. This should be structured in such a way as to lead to a lively discussion. Participants are encouraged to interrupt to ask questions or start discussions.
  • A MAXIMUM of 5 slide equivalents per presentation is allowed (Power point slides should not contain more than one graph or gel per slide and no more than 5 bullet points to stress the points being made by the presenter.) We appreciate cooperation with the spirit of this guideline. Handouts are welcome but should be distributed before sessions.
  • Everyone is expected to actively participate in every session and discussions.
  • The time spent at the meeting is relatively short, so please be familiar with papers received prior to the meeting.
  • It is very important that you commit to all sessions of the 2 days of meetings.

Forbeck Scholars Participation

Scholars are selected for each Forbeck Forum. These are outstanding junior clinical or post-doctoral fellows selected based on the quality and relevance of science.

  • Scholars present for 30-45 minutes, depending on the number of participants
  • The same presentation rules apply for scholars
  • After the Forum you are selected to attend, you will attend three years of Scholar Retreats held in Lake Geneva, WI. If you attend a Fall Forum, you will attend the Spring Retreat. If you attend a Spring Forum you will attend a Fall Retreat.
  • Scholars are selected by the Foundation Scientific Advisory Board and peer reviewers selected from past Forbeck Scholars.

General Program

The outline below illustrates a typical program schedule. You will receive a complete schedule, including speaking times, the Thursday the meeting starts.

Arrival Day
1:00 PM Arrivals
6:00 PM Cocktails (opt'l)
7:00 PM Dinner
Meeting Day 1
7:00 AM Breakfast
8:00 AM Scientific Sessions
12:00 PM Lunch
1:30 PM Scientific Sessions
6:00 PM Cocktails & Dinner
Meeting Day 2
7:00 AM Breakfast
8:00 AM Scientific Sessions
12:00 PM Lunch
1:30 PM Scientific Sessions
6:00 PM Cocktails & Dinner
Departure Day
7:00 AM Breakfast
8:00 AM Departures

Frequently Asked Questions

Below are some of our most Frequently Asked Questions. If you have something new to ask, please feel free to contact us.

  • Travel Confirmation will be sent out within 1 week of the meeting. This will include a hotel confirmation number, if there is one, and airport transfer details. We have to wait until we receive almost everyone’s travel to book airport transfer. Due to frequent airline changes, we wait until the week of the meeting to send this out.
  • Airport transfer is provided by Foundation staff, volunteers or arranged shuttle at specific times. If you opt to utilize Foundation airport transportation on your travel form, please be patient in receiving this information. We will send it to the week of the meeting.
  • Speaker agenda is not sent out prior to the meeting. It will be provided upon arrival in the meeting packet. We do not tell people when they are speaking because we expect everyone to attend all sessions. Sessions are all day Friday and Saturday.
  • Frequently airport transfer is provided by volunteers. Please be patient on receiving this information. Airport transfer will be sent out prior to arrival.
  • REMINDER: We do not reimburse for home side airport transfer or incidentals while traveling. We will not honor miscellaneous receipts sent for these expenses.

Forum Participants

Michael
Cox
,
PharmD, MHSc, BCOP
Loxo Oncology
Timothy
Cripe
,
MD, PhD, FAAP, CPI
Nationwide Children's Hospital, Ohio
Maryam
Fouladi
,
MD, MSc, FRCPC
Cincinnati Children's Hospital
Birgit
Geoerger
,
MD
Gustave Roussy, Paris, France
E. Anders
Kolb
,
MD
Nemours Center for Cancer and Blood Disorders
Elizabeth
Lawlor
,
MD, PhD
Seattle Children's Hospital
Charles
Mullighan
,
MD
St. Jude Children's Hospital
Ari
Shilatifard
,
PhD
Northwestern University
Kevan
Shokat
,
PhD
UCSF
Kim
Stegmaier
,
MD
Dana-Farber Cancer Institute
Sarah
Tasian
,
MD
Children's Hospital of Philadelphia

Forum Scholars

Jessie Brown, PhD
Columbia University
Adam David Durbin, MD, PhD
Dana Farber Cancer Institute
Loretta Li, MD
Ann & Robert H. Lurie Children's Hospital of Chicago