Targeting Gene Therapy - A Reality?

John D.
Minna
,
MD
UT Southwestern
Inder
Verma
,
PhD
Salk Institute

Forum Description

Everyone has heard of gene therapy, this is going to revolutionize medicine and bring untold benefits to us all. This is the message we read in the press, but what is the truth? Has any gene therapy trial in patients with cancer produced a single response? The data is not very impressive in 1998, but a great deal of work remains and it is very early days. The ideas behind the majority of gene therapies are developed by scientists, and applied by clinicians. A Forum to bring together the believers and the non-believers in the future of gene therapy should allow a full and frank discourse between these parties and hopefully lead to the instigation of trials which can resolve some of the pertinent issues. In addition, the Forum should serve as an ideal opportunity for scientist and clinician to review the problems each encounters in developing novel strategies based on gene therapy. An appreciation of the problems encountered by each group should speed the development of effective trials. The annual Forum is an ideal environment for such interchanges.

Forum Summary

This was a very exciting and stimulating meeting devoted to basic and clinical research advances in gene therapy for the treatment of cancer. Despite the recent unfortunate death due to gene therapy (of a patient without cancer) that has been widely discussed in the lay press the Forbeck meeting gave a very useful perspective on the current state of the art of this field. From the results presented at this meeting it is possible to estimate that around 1,000 people in the world with cancer have received some form of gene therapy. We can also conservatively estimate that 5-15% of these people have derived some benefit from this therapy. A likely guess is that around 10 people have died possibly related to gene therapy (1% rate) and tests of other new therapies often have mortality rates of 5-10%. While we obviously don’t want anyone harmed by gene therapy, these estimates tell us gene therapy is well within the range for possible benefits and side effects encountered in the development of other types of new cancer therapies. Many different tumor types are being tested and these trials are mainly occurring in adults with children’s trials to come later. A variety of approaches were discussed. One is to replace the damaged gene (much like fixing the brakes on ones car). Amazingly we learned you only need to do this in some cells. There is a ‘bystander’ effect where getting the gene into some tumor cells leads to the death of surrounding tumor cells. Also discussed were ways to target genes to tumors and to put genes into tumors to make them more sensitive to standard therapies such as chemotherapy and radiotherapy. There was discussion of putting genes for drug resistance into bone marrow stem cells to allow us to give more chemotherapy. Gene therapy is also being used to stimulate an immune response against cancer cells. This includes making tumors more immunogenic, and also genetically ‘fix’ or engineer killer T cells to more efficiently kill tumor cells. Finally, genetic vaccination with a patient’s own tumor antigen encoded in DNA was presented as an exciting new approach to tumor immunotherapy.

In all of these presentations we realize that ultimately we need to use standard clinical endpoints such as time to progression, survival and the relief of symptoms to monitor the effectiveness of gene therapy. We need to develop collaborations with industry and avoid conflicts of interest in the finances and reporting of the gene therapy results. Most importantly, we as scientists and physicians need to educate both individual patients and their families about the realistic benefits of gene therapy trials, and also the general public, media, congress, political leaders, and patient advocates about this work. It is essential to have truthful, open discussion as was exemplified by the outstanding discussion and new ideas that came out of this year’s Forbeck Symposium on Cancer Gene Therapy.

Venue & Travel Information

Hilton Head Island

Travel Forms

TRAVEL FORMS DUE:
October 4, 1999
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 forum. 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 forum. 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 forum 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 forum.
  • Speaker agenda is not sent out prior to the forum. It will be provided upon arrival in the forum 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 forum. Please no children.

What the Foundation Pays

Accommodations and meals are provided by the foundation during the forum. Airfare will be covered only if booked in accordance with our policy and only up to the amount in which you were approved for. The Foundation will also cover airport transportation on the forum 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.

  • Note we do not cover upgrades, changes, late bookings, etc.
  • Flights must be booked at least 30 days prior to the forum 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 forum. 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:
October 4, 1999
submit abstract

Forum Structure

The structure of the forum 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 2 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 forum is relatively short, so please be familiar with papers received prior to arrival.
  • It is very important that you commit to all sessions of the 2 days of the forum.

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 forum starts.

Arrival Day
1:00 PM Arrivals
6:00 PM Cocktails (opt'l)
7:00 PM Dinner
Forum 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
Forum 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 forum. 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 forum.
  • 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.
  • 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

Karen
Antman
,
MD
Columbia University
Joseph R.
Bertino
,
MD
Memorial Sloan-Kettering Cancer Center
David
Curiel
,
MD
University of Alabama
Albert B.
Deisseroth
,
MD, PhD
Yale University School of Medicine
Glenn
Dranoff
,
MD
Dana-Farber Cancer Research Institute
Eli
Gilboa
,
PhD
Duke University Medical Center
Phil
Greenburg
,
University of Washington
Stephen A.
Johnston
,
PhD
UT Southwestern
Robert L.
Martuza
,
MD
Georgetown University
John D.
Minna
,
MD
UT Southwestern
Drew
Pardoll
,
MD, PhD
Johns Hopkins University
Jack A.
Roth
,
MD
MD Anderson Cancer Center
James M.
Wilson
,
MD, PhD
University of Pennsylvania

Forum Scholars

Hanno Hock, MD, PhD
Massachusetts General Hospital
Owen A. O'Connor, MD, PhD
Columbia University