Vol. 11 No. 2 An RRP Foundation Publication
2003 Winter/Spring
P.O Box 6643,
Lawrenceville, NJ 08648-0643
________________________________________________________________________________________________________________
Contents
From the Newsletter Coordinator
My apologies for a very tardy, abbreviated newsletter issue. It is quite clear that I very much need help in preparing, editing and coordinating the publication of the RRP Newsletter. In particular, I am asking for a volunteer to take on the lead role of coordinating and publishing future issues. If interested, please contact Bill Stern (bills@rrpf.org).
We plan to get out a full summer issue very close to schedule, but there are some exciting things happening, especially with regard to vaccine therapies for RRP, and we wanted to let you know. We hope you find this newsletter issue to be interesting and helpful. If you have any questions or comments about this issue please contact Bill Stern or the principal newsletter editor:
Chris Neuberger [Cneuberger@eti1.com].
The RRPF announces a new cooperative relationship with the Cody Pate Foundation for RRP, to help promote RRP awareness and assist RRP families in need of financial support. For more details See: http://www.rrpf.org/rrpf/resources/CotyPateFoundation_mission.htm
We are most grateful to all those
individuals, medical professionals and corporations who have
supported the RRPF. Although it is impossible to publish the
names of all that contribute, we extend our sincere thanks to
everyone who has supported our efforts. Future donations from
individuals, professionals or from the business community will be
very much appreciated.
Tax-deductible contributions may be made to:
RRP Foundation
P.O. Box 6643
Lawrenceville, NJ 08648-0643
Do you donate to the United Way through your employer? You can select a "Donor Choice" option, which would allow you to direct a donation to the RRPF as the 501 (c) (3) of your choice. Since the RRP Foundation is a 501(c) (3) foundation, you may specify the RRP Foundation directly by writing in the name and address of the foundation as follows' RRP Foundation, P. O. Box 6643, Lawrenceville, NJ 08648. If you should need to add our Fed. ID number, it is 521798693. Thank you for your support.
From the RRPF home page (www.rrpf.org) or go directly to http://www.rrpf.org/donate.htm
We once again want to acknowledge the generous efforts of Ed and Maura Weiner along with Jason Wakeman and other friends for a very successful "2nd Annual RRP Hockey Night" fundraiser for the RRPF.
We also would like to acknowledge generous donations from Medtronic Corp. and from a number of employees of AXA Financial.
To physicians and nurses: Please distribute copies of this newsletter to your RRP patients. If you are not registered with the RRPF, please do so by completing the Practitioner Questionnaire enclosed.
RRPF Officers, Directors & Advisors
Marlene Stern
President
P.O. Box 6643
Lawrenceville, NJ 08648-0643
(609) 530-1443
marlenelin@aol.com
Bill Stern
Treasurer and Director
P.O. Box 6643
Lawrenceville, NJ 08648-0643
(609) 530-1443
bills@rrpf.org or rrpf@aol.com
Henry Woo, Esq.
Secretary
Medtronic International Inc.
Suite 1602 16/F., Manulife Plaza
The Lee Gardens, 33 Hysan Ave.
Causeway Bay,
Hong Kong
henry.woo@medtronic.com
Chris Neuberger
Director
13001 Burlingame Ave.
Oklahoma City, OK 73120
(405) 749-8499
cneuberger@eti1.com
Susan Woo
Director
101 Repulse Bay Road
Apt. A3/1st floor
Hong Kong
852-2812-7379
Writeushere@aol.com
Scientific Advisory Committee
Thomas R. Broker, PhD, University of Alabama at Birmingham Schools of Medicine & Dentistry
Haskins K. Kashima, MD, Johns Hopkins University School of Medicine
Linda Miller, RN, MSN, Children’s Hospital of Philadelphia
Clark Rosen, MD, University of Pittsburgh Voice Center
Robert J. Ruben, MD, Albert Einstein College of Medicine
Keerti V. Shah, MD, DrPH, Johns Hopkins University School of Hygiene and Public Health
Bettie M. Steinberg, PhD, Long Island Jewish Medical Center
Kathleen Sullivan, RN, Children’s Hospital of Boston
Voice Specialist/Advisor
Julie Bowne, M.S., CCC-SLP
RRP Newsletter Editors
Other RRP Newsletter Contributors
Toni Barringer
Dale Barringer
Caroline Dugger
Randy Sparkman
Marlene Stern
Bill Stern
RRP Reference Service Editor
David Wunrow
RRPF Fundraising Coordinator
Ed Weiner
(703) 691-1922
eweiner@weinerandassociates.com
RRPF Corresponding Secretaries
Jenny Shamblin
Christine-Hartman Davis
The RRPF produces two publications, the RRP Newsletter and the RRP medical reference service. The RRP Newsletter focuses mainly on the human and clinical aspects of recurrent respiratory papillomatosis and in this regard targets a broad readership, including patients/families, attending physicians/nurses, as well as researchers and the general public seeking to stay in touch with RRP from a clinical perspective. The RRP medical reference service serves those in the community seeking a more comprehensive understanding of this disease. Please help us by supporting these publications and other RRP services including patient outreach, support, advocacy and research
Subscription Policy and Suggested Minimum Annual
Donations
RRP Newsletter
RRP Newsletter plus Medical Reference Service
[Note: Back issues of the RRP Newsletter and Medical Reference Service are available on the website.]
RRPF Network News
by Randy Sparkman
The RRPF-sponsored e-mail
distribution list, or "Listserve", is a valuable resource for the RRP
community. It serves patients, family members, caregivers, and health
care providers. Over the past six months, membership has remained
steady at about 185 members. This relatively low volume (~200
messages in the past 6 months) list is hosted on the free
YahooGroups.com list management service. It is gracefully moderated
by RRP patient and community advocate Petra Hall
(email: petra@communique.se) .
Basic subscription information and complete list archives are available on the Internet/World WideWeb at: http://groups.yahoo.com/group/rrpf. You must register with YahooGroups to gain access. The names and e-mail addresses of the subscribers are private and are only exposed if the subscriber includes them in the text of a posted message. The messages may also be generated and received from within your e-mail computer client or can be completely generated and received from the yahoogroups rrpf list web pages. Messages may be received one at a time or in a "daily digest". Anyone within the rrpf community that needs technical assistance with any aspect of the mailing list can send an e-mail to : randy_sparkman@yahoo.com.
Messages on the list primarily focus on treatment options. This includes both surgical and adjunct therapy. Postings to the "Interactive Posting Board" are also, selectively, distributed to the mailing list. Over the past six months, other significant threads have included discussions of bone density sensitivity to DIM, the relationship of allergies to RRP, issues and experiences with insurance coverage, inquiries regarding finding experienced RRP health care providers, strategies for voice rest after surgery, surgical techniques, timing of surgeries, and the strength demonstrated by the little ones that are so often significantly impacted by this disease.
RRP Meetings
An RRP Foundation sponsored meeting was held on September 24, 2002, in conjunction with the annual meeting of the American Academy of Otolaryngology in San Diego.
Presentations were as follows:
"Introduction and RRP Overview" Bill Stern, RRP Foundation
"International RRP ISA Center and the RRP Website"
Michael Green, International RRP ISA Center
"RRP, RRP Treatments and RRP Task Force Issues" Craig Derkay, MD, East Virginia Medical School
"Understanding RRP: embarking on a genetic undertaking"
Farrel Buchinsky, MD, Allegheny General Hospital, Pittsburgh
"Treatment for scarring of the vocal folds" Clark Rosen,
MD, University of Pittsburgh Voice Center
"Update on the cidofovir study at San Diego Children's"
Seth Pransky, MD, San Diego Children's Hospital
"RRP research activities at U.A.B." Brian Wiatrak, MD, Children's Hospital, Univ. of Alabama, Birmingham
A complete meeting report and presentations details can be found at: http://www.rrpf.org/rrpf/meetings/RRP_Focus2002/RRP_FocusSession_2002.htm
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On February 22, 2003, the Cody Pate Foundation for RRP held a meeting in Memphis , TN. The purpose of the meeting was to introduce the organization and to familiarize some of the audience with RRP. In addition to the officers of the Foundation, the meeting was attended by several local RRP families, some local corporate and media representatives, two notable RRP surgeons/clinical researchers, Drs. Nigel Pashley and Jerome Thompson and RRP Foundation director, Bill Stern.
The Cody Pate Foundation president, Lynette Pate, described the organization and the mission, which has a primary dedication to financially assisting RRP families, particularly with travel expenses (for more details please see: http://www.rrpf.org/rrpf/resources/CotyPateFoundation_mission.htm)
Dr. Nigel Pashley, from Children’s Hospital in Denver, provided a brief overview of RRP treatments and made the case for adjunct treatments in addition to surgery for RRP. Dr. Pashley’s main focus was his Mumps vaccine protocol (see: http://www.rrpf.org/rrpf/therapies/PashleyMumps.htm) and in particular RRP treatments using the tri-valent MMR vaccine which has replaced the mono-valent Mumps, which is generally unavailable. Preliminary results are positive based on a sample of 12 RRP patients with 3 to 12 month follow-up. Using an objective scoring system to assess the extent of respiratory papilloma involvement (lower number meaning less papilloma), pre-treatment scores ranged from 19-28 with post treatment improving to 1-8, and 9 of 12 being in "remission" from 3-20 weeks after treatments.
Dr. Jerome Thompson, from the University of Tennessee, will be trying Dr, Pashley’s Mumps/MMR vaccine protocol at U of T, first in off-label compassionate use, but eventually will seek to develop the setting for a double-blind crossover trial.
There was a meeting of the RRP Task Force on Saturday May 3, 2003 in conjunction with the Spring otolaryngology meetings being held in Nashville, Tennessee.
Several current and proposed clinical projects were discussed as follows:
The American Society for Pediatric Otolaryngology (ASPO) has a web-based member survey on RRP management, mortality and experience with adjuvant modalities. Some of the findings are:
Despite the many letters and emails written by members of the RRP community and supportive remarks from Dr. Julie Gerberding, Director of CDC, the RRP Task Force and RRP Registry remain unfunded at this time. The RRP Foundation will continue to do whatever it can in support of these important RRP efforts.
Science & Research Activities
Positive results from Phase II Clinical Research Study in Pediatric RRP using the HspE7 Vaccine for HPV
[The following article is based on a press release and subsequent conference call. More details may be found at: www.stressgen.com]
On 10 June 2003, Stressgen Biotechnologies Corporation announced that results from its Phase II trial with HspE7 showed a statistically significant reduction in the number of surgeries for a group of 27 moderate to severe RRP patients.
Top line data from this trial involved the interval between surgeries following treatment with HspE7 compared to the pretreatment surgical interval, the primary endpoint of the trial. In the study population as a whole, there was a mean increase of 78.6 percent in the interval between surgeries (p=0.015) following treatment with HspE7 compared to the pretreatment surgical interval, resulting in fewer surgeries for these patients.
Longer-term follow-up continues in this trial out to 60 weeks beyond the initiation of treatment with HspE7. Preliminary projections of the number of surgeries per year have been made. Among the patients estimated to have a reduction of at least one surgery per year as compared with the pretreatment period, it is projected that more than 50 surgeries will have been avoided during the year after treatment.
HspE7 was given to 27 patients age from 2-18 through a 500 mcg subcutaneous injection administered three times over a 60-day period. Prior to study initiation, all of the children required frequent surgeries under anesthesia in order to reduce the size of warts to unblock their airways. Approximately one-half of the children enrolled in this trial had severe disease defined by a clinical score considered by experts to correspond to severe disease. In addition, seven of the children in this study were known to have had papillomas in the lungs, which is the most serious manifestation of RRP. By both of these measures, the children in this study presented a difficult challenge for HspE7 treatment.
This HspE7 study, Stressgen’s first for its investigational immunotherapeutic therapy for RRP, had two objectives. The first objective &emdash; standard for a Phase II trial &emdash; was to assess the clinical effectiveness of HspE7 in children with RRP, through the primary endpoint of the length of post-treatment inter-surgical interval compared to median pretreatment inter-surgical interval. The first objective was achieved and leads to a Phase III confirming trial. The second objective involved the establishment of a very high hurdle of a doubling of the first inter-surgical interval, which, if achieved, might have further accelerated the clinical program. The second objective was almost made, thereby providing further support for a Phase III RRP program. Based on these results and the recommendations of the expert panel that reviewed the data, Stressgen is planning to develop a Phase III RRP program moving towards a goal of a Biologics License Application submission to the U.S. Food and Drug Administration (FDA) for this indication.
Stressgen has entered into a collaborative agreement with Roche for the co-development and global commercialization of HspE7. Roche is producing commercial grade HspE7 to be used in the proposed Phase III RRP trial. The Phase III trial is anticipated to start during the second half of 2004.
HspE7 is produced by Stressgen Biotechnologies Corporation
www.stressgen.com Canadian Office: #350 - 4243 Glanford Avenue Victoria, BC Canada V8Z 4B9 Phone: (250) 744-2811 Toll Free: (800) 661-4978 Fax: (250) 744-2877
Principal Executive Office:
10241 Wateridge Circle, C200
San Diego, CA 92121 USA
Telephone: (858) 202-4900
Fax: (858) 450-6849
Merck and some other companies are also developing, primarily prophylactic, HPV vaccines, the objective being to target a large population that are likely to be exposed to genital HPV. We are following this work with much interest. Since there has been therapeutic success using Mumps and MMR vaccines designed to be preventative, it is felt that some of these HPV vaccines that target HPV 6 & 11, could be at least as effective.
Look for more on HPV vaccines in the Summer 2003 RRP Newsletter issue.
Impact of RRP on the Quality of Life of children with RRP
The RRP Foundation is pleased to announce that it has funded investigators Jonathan P. Lindman, MD, Brian Wiatrak, MD, FACS, FAAP and Linda S. Lewis, MSN, RN, CNS, of Childrens Hospital in Birmingham, AL to study the impact of RRP on the quality of life of children.
The purpose of this project is to utilize the PedsQLTM 4.0 Generic questionnaire to evaluate the impact of RRP on the quality of life of children affected by this disease. This is a reliable, validated questionnaire that has been used for similar studies with other diseases and is designed to measure the 3 core dimensions of health (physical, social and emotional) as delineated by the World Health Organization, as well as role (school) functioning. It will be administered to children (and their parents) enrolled in this study.
Genetic Patterns may Determine Response to I3C/DIM
The RRP Foundation is proposing to support and collaborate in a study to understand why some people respond to I3C/DIM and some others do not.
Researchers at the North Shore-Long Island Jewish Research Institute, including principal investigator Dr. Leslie Goodwin and Dr. Karen Auborn as one of the consultants, plan to begin a study to determine why some persons respond to treatment with indole-3-carbinol or diindolylmethane (I3C/DIM) while others appear to be refractory. The likely reason results from differences in the genetics of individuals because I3C/DIM change the expression of more than 100 genes. New technology exists that allows researchers to readily determine about 10,000 known variations in genes called polymorphisms, and the Institute is a testing site for this technology. This technology will continue to expand.
Because many persons with RRP have taken I3C/DIM, the RRP community could be a tremendous help in this endeavor by providing samples which would be a buccal swab (a cotton swab that the person swishes around in his/her mouth). Ultimately, results from this project could pre-determine whether persons with RRP will or will not benefit from I3C/DIM. As the RRP support group is well informed, I3C/DIM is a relatively benign treatment. However, not every person responds to this therapy.
Currently, consideration for funding and an application to the Institutional Review Board at the Institute are in progress. Details are being developed to keep patient’s identity anonymous, set up a data base and a consent form that is acceptable to the patient advocate, the General Clinical Research Core and the Institutional Review Board. The RRP Foundation will be a resource for this project. Persons, willing to participate in this research project should contact Bill Stern at the RRP Foundation. There should be no cost or risk to any person. Buccal swabs do not require a visit to the doctor. We will keep the RRP community informed regarding the status of this research project.