Therapeutic Targeting of mRNA Splicing in Cancer

Forum Chairs

Olga
Anczukow-Camarda
,
PhD
Associate Professor
The Jackson Laboratory
Andrei
Thomas-Tikhonenko
,
PhD
University of Pennsylvania

Forum Description

This meeting aims to bring together splicing experts, cancer researchers, clinical oncologists, and drug developers from academia and industry to brainstorm about novel ways to target dysregulation of mRNA splicing in human malignancies.

The discovery of highly recurrent mutations in splicing factors (SFs) across a variety of tumor types has provided compelling genetic evidence for a direct causal relationship between splicing dysfunction and cancer. Even in tumors without SF mutations, mRNA splicing patterns are patently aberrant, resulting in profound re-shaping of cancer transcriptomes and proteomes. This in turn affects both disease pathogenesis and therapeutic responses. Consequently, cancers with imprecisely functioning spliceosomes can be successfully targeted by direct spliceosome inhibitors and/or inhibition of enzymes that regulate spliceosome factors via post-translational modifications [reviewed recently in “RNA-binding proteins of COSMIC importance in cancer”, PS Choi, A Thomas-Tikhonenko, J Clin Invest s131 (18), e151627 (2021)]. Some of these known and emerging drugs are currently in clinical trials for various types of cancer, and many more remain to be discovered. Yet there are no dedicated meetings that bring together RNA biochemists, structural biologists, cancer researchers, clinical oncologists, and drug developers. This proposed forum would fill this gap and reveal novel opportunities for clinical translation.

We aim to promote new collaborative projects amongst participants, parlaying their complementary expertise into the design of new therapeutics targeting RNA alterations in human malignancies. 

Forum Summary

Dysregulated RNA splicing is a molecular feature of almost all cancer types, liquid and solid alike. It constitutes a newly recognized tumor vulnerability, which holds the promise of yielding novel therapeutic interventions for cancer patients. However, a number of outstanding problems and challenges remain to be addressed by the cancer and RNA communities in order to move drugs targeting RNA splicing from the bench to the bedside. They could be broken down in four broad categories: 1) Developing high-throughput approaches to interrogate the function of spliced isoforms at scale; 2)  Developing single-cell and spatial approaches to unravel the role of RNA splicing in drug responses and resistance; 3) Defining the splicing landscapes of tumor, stromal and immune compartments, to enable targeting of tumor microenvironment; 4) Developing of in vitro and in vivo model systems that better represent cancer patients. Addressing these goals will enable a priori identification of patients that will respond and benefit from several existing splicing-targeting therapeutics approaches.

Venue & Travel Information

Asilomar Conference Grounds

800 Asilomar Avenue
Pacific Grove, CA 93950

www.visitasilomar.com
831.372.8016

Travel Forms

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

To help reduce transportation costs, the Foundation tries to group travelers on shuttle vans rather than providing transportation for single travelers. The times below are not exclusive but represent times that we hope to have shuttles leave the airport.


The Monterey Regional Airport (MRY) is the preferred airport as it is only 20 minutes from the meeting location.

  • Arrivals to MRY - Thursday around 1 PM, 3 PM and 5 PM
  • Departures from the meeting venue - Sunday around 8 AM, 10 AM, and 12 PM


San Francisco International Airport (SFO) is 2 hours from the meeting location without traffic. The Foundation tries to prioritize International Flights and situations where flying into SFO is significantly cheaper than connecting to MRY. The times below are for guidance only, and depending on the volume of travelers, we may add an additional shuttle to and from the airport.

  • Arrivals to SFO - Thursday around 3 PM
  • Departures from the meeting venue - Sunday around 10 AM
TRAVEL FORMS DUE:
August 21, 2023
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 21, 2023
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

Frederic
Allain
,
PhD
University of North Carolina
Brittany Angarola
Jones
,
PhD
The Jackson Laboratory for Genomic Medicine
Douglas
Black
,
PhD
University of California, Los Angeles
Robert
Bradley
,
PhD
Fred Hutchinson Cancer Research Center
Angela
Brooks
,
PhD
University of California Santa Cruz
Chonghui
Cheng
,
MD, PhD
Baylor College of Medicine
Ana
Conesa
,
PhD
Institute for Integrative Systems Biology
Luisa
Escobar-Hoyos
,
MSc, PhD
Yale School of Medicine
Eduardo
Eyras
,
PhD
Australian National University
Ernesto
Guccione
,
PhD
Mount Sinai Hospital
Sydney
Lu
,
MD, PhD
Stanford University
Karla
Neugebauer
,
PhD
Yale School of Medicine
Priyanka
Sehgal
,
PhD
Children's Hospital of Philadelphia
Zuzana
Tothova
,
MD, PhD
Dana-Farber Cancer Institute
Juan
Valcarcel
,
PhD
Centre for Genomic Regulation
Kevin
Weeks
,
PhD
University of North Carolina

Forum Scholars

Edie Crosse, MD, PhD
Fred Hutchinson Cancer Center
Xiao Song, MD, PhD
Northwestern University