2386 East Heritage Way, Suite B
Salt Lake City, UT 84109
(there is a 24 hour voicemail answering service)
Please Contact Us if you have any questions or if we can be of help in any way.
Mary E. Schwartz, Director
Holly A. Evans, Administrative Assistant
Janice N. Schwartz, Patient Advocate
Note: Mary E. Schwartz is a full-time unpaid volunteer. Holly Evans is a full-time paid contract employee. Janice Schwartz is an unpaid volunteer. PC Project also has an IT consultant who is a contract employee.
Board of Trustees
C. David Hansen, MD (Dermatologist)
Roger L. Kaspar, PhD (Scientist and CEO)
F. Dwight Marchant (Bank Executive)
David R. Owens, Esq. (Attorney)
Mary E. Schwartz (PC Project Director)
Janice N. Schwartz (PC Project Patient Advocate)
Lex L. Udy. PhD (Scientist and CEO; retired)
Craig T. Vincent, Esq. (Attorney)
Medical and Scientific Advisory Board (MSAB)
Click on the name for further information on each member.
Sherri Bale, PhD
C. David Hansen, MD
Peter R. Hull, MD
Roger L. Kaspar, PhD
E. Birgitte Lane, PhD
John A. McGrath, MD
W.H. Irwin McLean, DSc, FRSE
Leonard M. Milstone, MD
Edel O'Toole, MD, PhD, FRCPI
Amy Paller, MD
Dennis R. Roop, PhD
Frances J.D. Smith, PhD
Eli Sprecher, MD, PhD
Maurice van Steensel, MD, PhD
Jean Y. Tang, MD, PhD
Budget and Finances
IRS Form 990 and Schedule A 2010
Annual Report 2010
Audited Financial Statement 2010
No paid advertising, banners or links are permitted on this website or used in any way for fundraising.
PC Project serves patients throughout the world and we are glad to link to special sites which assist in sharing information in various languages. No matter where you live, PC Project is here to serve patients, physicians and researchers.
Brazil. Dermatologia.net is an important site whice makes available information in Portuguese about PC
France - Le Coeur au Pied is a charity for PC patients in France which supports PC Project (Sylvie Cierpucha, President)
UK - PC Project UK (organized under the Trust Fund services of the University of Dundee)
All information collected through the website, the IPCC and the IPCRR is confidential and used only under the US HIPPA laws for patient confidentiality and within the database of PC Project. No contact information is released without written consent.
Brief History of PC Project
MORE HISTORY COMING SOON.........
- Created a new PC Brochure for the Combined Federal Campaign (CFC).
- Updated the PC Brochure for Schools, Friends and Donors.
- Eight articles to update information about PC were submitted as the first set of PC articles to be published in an issue of the International Journal of Dermatology (JID). Topics of these articles include Intravival Imaging, IPCRR Data update, qPCR, PC Skin Equivalents, PC Genetics: Discovery of PC Mutations, Case Reports: K16 R127C and related mutations, Small Molecular Screening, Statins, and accell siRNA.
- Articles for a special PC edition Journal of Investigative Dermatology (JID) are being prepared for submission.
- PC Project received notice that it is a BBB Accredited Charity and meets all 20 standards for charity accountability.
- PC Medical brochure translated into Spanish.
- French Patient Support Meeting was held in Roissy, France on Jun 18-19. This meeting was co-sponsored by Le Coeur au Pied and PC Project.
- PC Brochures translated into French.
- Four PC patients participated in the Nashville Dermatologic Society Meeting that was held on June 18th. PC Project arranged for patients to attend and provided custom handouts for this meeting.
- One PC patient participated in SDOCT imaging at Bioptigen in Durham, North Carolina on June 21st.
- PC Project received notice of its qualification for the 2010 Combined Federal Campaign (CFC) CFC #89437.
- IPCC Newsletter Vol 7, No 2 was completed and emailed to IPCC members.
- IPCRR Forms translated into German and received WIRB approval.
- The 2010 IPCC Symposium was held May 3-4 in Atlanta, Georgia prior to the Society of Investigative Dermatology (SID) annual meeting.
- PC Project's Board of Trustees meeting was held May 10th with special guest Dr. W.H. Irwin McLean (University of Dundee.)
- The "Caring for PC" DVD was translated into French.
- PC Project participated in the NIAMS Coalition annual conference call on May 20th.
- Four PC Patients participated in the Stanford University Practice of Medicine Course on May 28th. PC Project arranged for patients to attend and provided custom handouts for this course.
- PC Project updated the PC brochures.
- PC Project sent invitations for physicians to join the IPCC Physicians Network. This network will help with educational outreach, development of best practices guidelines, consultations, publications and clinical studies.
- PC Kids Club web meeting was held April 17th.
PC Project attended the American Academy of Dermatology (AAD) annual meeting in Miami, FL. During the AAD a Mini PSM for patients was held on Mar 4 with doctors, Sancy Leachman (University of Utah) and Takashi Hashimoto (Kurume University School of Medicine.) PC Project also attended the Coalition of Skin Diseases (CSD) annual meeting that was held on Mar 8th.
- PC Project attended and hosted a dinner at the first GO Delivery! Meeting held at Stanford University in Stanford, CA on March 24-26.
- IPCRR Addendum #3 received WIRB approval.
- IPCC Clinical Studies web meeting was held Feb 3rd. Topics included PC genetic findings update by Frances Smith (University of Dundee), Statin Clinical Study (Peter Hull, MD, Royal University Hospital Division of Dermatology, Saskatoon, Canada.
- The second PC Kids Club web meeting was held on February 13th.
- Received certificate that Utah's Governor, Gary Richard Herbert, declared Feb 28th, 2010 as Rare Disease Day in Utah.
- IPCC Newsletter Vol 7, No 1 was completed and emailed to IPCC members.
- Received notification of extension of the PC & Statin Study with an additional low dose of oral retinoid.
- A Patient Advisory Board (PAB) meeting was held on Jan 7th.
- Completed Doing Business As (DBA) applications and received certificates for Pachyonychia Congenita Fund aka PC Fund aka Pachyonychia Congenita Project aka PC Project.
- PC Project sent letter to Governor of Utah to set Feb 28th as rare disease day.
- IPCC Newsletter Vol 6, No 4 was emailed to IPCC members.
- PC Project's Board of Trustees Meeting was held December 4th.
- PC Kids Club first web meeting was held December 12th.
- PC project applied for the 2010 Combined Federal Campaign (CFC).
- PC Project attended the NIAMS Coalition Meeting in Washington, DC on Nov 3rd.
- The IPCC Clinical Studies working group held a web meeting on Nov 4th. Topics included PC genetic findings update by Frances Smith (University of Dundee), siRNA Clinical Trial summary by Dr. Sancy Leachman (University of Utah) and an outline of the GO! Delivery Grant (ARRA funding) by Roger Kaspar (TransDerm, Inc.)
- PC Project participated in a web meeting for the Coalition of Skin Diseases (CSD) on Oct 28th.
- Bill and Melinda Gates Foundation announced that a grant was awarded to TransDerm for development of microneedles for delivery to skin.
- A notice was received on Oct 20th of acceptance for publication by Gene Therapy (a leading journal) of the data on the TD101 Clinical Trial for siRNA treatment of a PC patient.
- PC Project qualified for the 2009 Combined Federal Campaign (CFC) CFC #89437.
- A mini gathering and dinner for PCers in NYC with Roger Kaspar (TransDerm Inc.) was held on Oct 26th.
- Received IRB approval for a topical ibuprofen study but the company selling the product had received warning letters to stop selling the product from the FDA so we are looking for another treatment to reduce pain that will be FDA-approved.
- The New Blog feature on the Patient Home Page lets PCers comment to each other as well as on different PC topics. The feature was first demonstrated at the 2009 SLC Patient Support Meeting.
- The 2009 Patient Support Meeting was held September 24-26 in Salt Lake City, Utah. See Patient Support Meeting Reports page for more information.
- Received notification that the GO Delivery! Grant was awarded on September 28, 2009. This will greatly benefit research to develop effective delivery of nucleic acids (siRNA) to skin! This is a huge achievement for the scientists who are working so hard to bring therapies to PCers and other skin disorders.
- “Achieving successful delivery of nucleic acids to skin: 6th Annual Meeting of the International Pachyonychia Congenita Consortium” was published in the Journal of Investigative Dermatology (by Kaspar RL, McLean WH, Schwartz ME)
- The IPCC Clinical Studies working group held a web meeting on August 5th. Topics included PC genetic findings with several case studies by Frances Smith (University of Dundee) and IPCC Updated Data (over 200 PC patients with genetic findings) by C. David Hansen (University of Utah.)
- PC Project's Board of Trustees Meeting was held on August 21st.
- IPCC Newsletter Vol 6, No 3 was emailed to IPCC members.
- Received notice that the PC & Statin Study is being extended for 6 more months.
- PC Project released a new "Caring for PC" DVD" and distributed to patients with a special Patient Newsletter.
- PC Project participated in the NIAMS Coalition Conference call on 23rd of July.
- The matching fund program is being continued by sponsor for another year. For every $1 donated, PC Project receives and additional $2.
- The IPCC Newsletter Vol 6, No 2 emailed to IPCC members.
- The 2009 IPCC Delivery Symposium was held May 4-5 in Montreal, Québec. The symposium was co-sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Office of Rare Diseases Research (ORDR), PC Project, Agilent Technologies, Avontec, Orfagen, and TransDerm Inc.
- A Grand Opportunity (GO) Grant called GO Delivery! was filed by scientists working on PC.
- PC Project's Board of Trustees meeting was held on May 1st.
- PC Project will sponsor additional IPCC Interactive Opportunities including the Clinical Studies working group's quarterly web meeting which will be held on the first Wednesday in August, November and February. Information will be presented on various studies that are underway. Also, PC Project will continue to sponsor the monthly IPCC Genetics web meeting that is held the first Wednesday of every month.
- IPCC Newsletter Vol 6, No 1 was completed and emailed to IPCC members.
- PC Project attended the Coalition of Skin Diseases (CSD) annual meeting on March 9th in San Francisco, California.
- A mini PSM was held March 10-11 in San Francisco, California following the Annual American Academy of Dermatology (AAD) meeting.
- PC Project was awarded a NIAMS Grant for IPCC Symposium to be held May 4-6 in Montreal, Quebec.
- New Archive/Index feature was added to the website.
- IPCRR forms translated into Portuguese and received WIRB approval.
- PAB Web meeting was held on February 7th.
- A mini PSM dinner was held February 8th in Calgary, Alberta.
- PC Project participated in the Coalition of Skin Diseases (CSD) Conference call.
- An audited financial statement was prepared for PC Project for 2008 and has been posted on the website with the tax return and annual report (see above under Budget and Finances.)
- PC Project filed an application for recognition with the Better Business Bureau Wise Giving Alliance and with the Combined Federal Campaign. The CFC is to allow PC Project to receive direct payroll donations from federal employees.
- C. David Hansen, MD is working with PC Proejct to prepare IPCRR data for publication. We've learned a lot about PC through the IPCRR registry and we now need to share this information with physicians, scientists and patients around the world in a professional and recognized manner. Dr. Hansen is a great asset to help us accomplish this goal.
- Sponsor funds $2-for-$1 donation matching program.
- New translational tool available on the PC Website.
- IPCRR Retinoid Questionnaire Addendum #2 received WIRB approval.
- IRB approval was received for a PC study using statins with Dr. Peter Hull
- The Board of Trustees meeting was held
- An R-13 meeting grant was awarded for the “Delivery to Skin” meeting for May 2009 by the Office of Rare Diseases
- A team of physicians and scientists are reviewing the data on PC and retinoid use for a future publication
- $2-for-$1 donation match program continued
- The International PC Research Registry received continuing study renewal; graphs are posted on the website to indicate information gathered
- An important publication regarding the clinical trial for TD101 (siRNA for PC) was submitted for publication
- PC Bulletins were begun to provide regular updates to features on the PC website and other PC news
- PC Webinar series initiated
- The completely re-designed website at www.pachyonychia.org was launched on 1 Aug 2008
- Monthly conference calls for physicians and scientists was instigated for consultation regarding genetic testing status
- The IPCC Newsletter solicited authors to publish data now available in the IPCRR database
- The 2008 PSM was held in Pitlochry, Scotland on July 15-17th. There were 59 PCers from more than a dozen countries with about 161 people total in attendance.
- A new 3-year grant was awarded to Dr. Frances Smith to continue the genetic testing for PC patients
- The 2008 IPCC Symposium was held in Hefei City, China on May 18-20th. One day was a Patient Support Meeting with 11 PCers and the other days included scientific presentations and discussion on moving PC research forward. Following the meetings, IPCC members traveled together to White Mountain.
- On May 13, 2008 in Kyoto, Japan, IPCC members hosted a ½ day meeting in conjunction with the SID/IID annual meeting to provide an update of PC research. in Kyoto, Japan on May 13th.
- PC Project staff attended Skin Disease Research Development Day on April 9-11 th hosted by the AAD and CSD.
- Representatives from PC Project met in Philadelphia on April 8th to discuss plans for a PSM. A small dinner was enjoyed with eight PCers from the Philadelphia area.
- Received approval from the US Internal Revenue Service confirming PC Project as a 501(c)3 public charity.
- PC Project staff finished preparing and mailed a 1” thick and over 150 pages “Facts and Circumstances” report for the US Internal Revenue Service to establish permanent status for PC Project as a public charity.
- A clinical trial approved by the US FDA began. The purpose of the trial is to test the safety of an siRNA developed to target a specific PC mutation. The trial will be for 14 weeks, plus a 3-month ‘wash out’ period.
Oct - Nov 2007
- completed the rodent toxicity study; filed the FDA IND application for Phase1B clinical trial.
- Holly Evans is our full time assistant for PC Project.
- IPCC Members attended a ‘western adventure’ focus meeting to evaluate the various pathways for development of PC treatments.
- The 2007 PSM was held in Park City, Utah on June 12-14th. There were 46 PCers and about 130 people total in attendance.
- The IPCC annual meeting was held in Los Angeles, California on May 9 in conjunction with the SID annual meeting. A dinner was held where 10 PCers and interested physicians and scientists from the SID met.
- The annual AAD meeting was held in Washington DC on Feb 2-6 th. PC Project was asked to provide a presentation on our progress in developing treatments for PC.
- The Philip Mazzo Scholarship fund was established to provide travel assistance for families to attend PC Patient Support Meetings. Mr. Mazzo is the grandfather of Nicole Smith, a PCer.
- A meeting of the PC Fund Board will be held November 1 at the PC Project offices in Salt Lake City.
- PC Project was a co-sponsor for the 2nd Annual Meeting of the Oligonucleotide Therapeutics Society New York City. Drs. Leachman, Kaspar, Hickerson and Mary Schwartz attended the meeting.
- The revised IPCRR questionnaire and consent form were approved and will be available in the next few weeks.
- On October 25 the toxocology studies for the siRNA inhibitor developed by TransDerm (see June 2006 below) began and will continue for 28 injections. The FDA requires these studies and very complex testing to assure safety of the drug.
- The PC biopsies taken in May 2006 were successfully grafted on mice with collaborators in Spain. Roger Kaspar visited the lab in Spain and conducted tests on the human skin which had been grown on the mice.
- Roger Kaspar was invited and gave a presentation on siRNA inhibitors for PC at the DEbra meeting in Dublin, Ireland.
- A final US Patent has been filed for the PC siRNA lead inhibitors (the preliminary patent was filed about 11 months ago).
- The MSAB held three conference calls and decisions were made regarding the IPCC meetings in 2007 and 2008, as well as a special focus group meeting following the 2007 Patient Support Meeting.
- The quarterly IPCC Newsletter was sent to approximately 80 members of the IPCC
- A special patient Newsletter was mailed to all PC patients/families who are registered with PC Project
- PC Project has funded a research grant for Dr. Sancy Leachman of the University of Utah so that she can devote time to prepare for pachyonychia congenita clinical trials. The application to the FDA and an application for a grant from the OOPD will be under Dr. Leachman's direction. Dr. Pho will serve as the PC Research Fellow. Dr. Pho completed her M.D. degree in May 2006 at the University of Utah and will assist Dr. Leachman in the laboratory work necessary to establish efficacy and measurements for PC treatments.
- The revised IPCRR questionnaire has been completed (after two years of effort!) and will be submitted for IRB approval. Now less than half as long, the Questionnaire is designed to be both shorter and more precise. The 100+ PC patients who have participated in the registry and completed the 'long-form' will not need to re-do the questionnaire as we have carefully worked to coordinate the responses in our database. We hope all who have participated will know their responses and participation in the registry has led the research and shaped the direction of the work being accomplished for PC.
- A new brochure "For Schools" was prepared and mailed to families with school-age children.
- The 2006 Patient support was hosted by Drs. W.H. Irwin McLean and Frances J.D. Smith on July18-20 in Dundee, Scotland. There were 31 PC patients in attendance, 27 with known mutations. Patients were from 11 countries -- Brazil (1), England (11), Denmark (1), Finland (1), France (4), Ireland (1), Netherlands (1), Scotland (3), Sweden (1), USA (4), and Wales (1). Of these patients, there were 15 attending a PC meeting for the first time and 16 prior attendees. A total of 24 patients participated in some associated research efforts, including sampling of hair and calluses from the feet. A favorite feature of this meeting was the tour of the McLean research laboratory at the University of Dundee.
- Dr. Mark Eliason begins his 4-year residency in Dermatology at the University of Utah. Mark has accomplished so much for PC and been a very integral part of PC Project working closely with us on our grant applications, the retinoid survey, the severity and pain scale development, the photography protocols and many other important projects. We will miss him greatly.
- Rachel Miller is our part time clerical assistant for PC Project.
- One of the first PC grants awarded was for development of a mouse which has the human PC gene. In June the first chimeric PC mouse pups were born at Baylor. There are still many steps needed to see if these mice will reproduce and pass this human gene on to their offspring so that PC can be induced. This will be very important for testing prior to human clinical trials.
- TransDerm, our biotech partner, received notice of Orphan Drug Status for their lead siRNA inhibitor for pachyonychia congenita. This is a major step on the pathway to drug approval through the FDA.
- Roger Kaspar, Sancy Leachman and Mary Schwartz met with Steve Hutcherson a professional consultant who is assisting in planning and preparing for submission to FDA for clinical trial approval. Steve also arranged for discussions with Bert Spilker and for contacts with companies to conduct the toxicology studies in rodents for the lead siRNA inhibitors.
- The response to the Retinoid Supplement has been outstanding and preliminary results were mailed to all who responded. Dr. Eliason is finalizing the information for publication and available on the PC website. The results show that PC patients have not found retinoid treatment to be satisfactory or beneficial.
- We have contacted specialists to assist us in thermal photography and other techniques which can be used to measure changes after treatment for PC hyperkeratosis.
- The third IPCC Symposium was held May 2-3, 2006 in Philadelphia, PA as an ancillary meeting to the SID. PC Project had a booth and several posters at the SID meeting.
- Following the IPCC meeting, an Action Plan has been developed for the coming months and the MSAB are working to achieve these goals.
- At the two year anniversary for the IPCRR (PC research registry) more than 300 PC patients have been identified worldwide and more than 100 are participants in the registry.
- Dr. Birgit Lane joined the PC Medical and Scientific Advisory Board (PC MSAB).
- Dr. Sancy Leachman conducted tests on a PC patient (and on her own foot!) using saline and dye injections to determine how much and how to inject siRNA’s into human skin and calluses. Again, these steps are in preparation for later clinical trials.
- Biopsies were provided by two PC patients and sent to collaborators in Spain to produce synthetic human skin which will be important as PC therapy moves forward. Also, a second biopsy was sent to Dr. Leonard Milstone (Yale) to develop immortalized K6a cells. Other scientists have and are developing stabilized and immortalized PC cells lines for K16, K6b and K17. All of these are needed for the testing necessary prior to clinical trials.
- The CORT applicants were notified that their research grant application was scored and was 11the out of 30. It appears unlikely the grant will be funded. Also, word was received that a grant submitted by TransDerm also received a score that will not be funded. This grant score was surprising and upsetting in that the rationale for the score seemed to be based on (a) an incorrect assumption that retinoids are an effective therapy for PC and (b) some confusion between TransDerm's grant and another grant. We will not let these scores be a discouragement to our progress.
- Transderm, Inc. demonstrated that the siRNA successfully inhibit the specific mutant gene in tests in mouse paws.
- PC Project is sponsoring a trial in which Dr. Sancy Leachman began an 'off label' trial for one PC patient using an approved drug which may have some effect on K16. It will be some months before results are known.
- Dr. Frances J.D. Smith, the PC Career Development Awardee is the visiting research for six weeks at Transderm, Inc. working on collaborative development of various siRNA sequences to show efficacy of this approach.
- On April 28, Dr. Smith visited Salt Lake City with Roger Kaspar and she was the guest lecuturer at the University of Utah Department of Dermatology.
- PC Project representatives attended the Obstacles to Translation Conference – March 1-2 in San Francisco. PC Project hosted a dinner after the Obstacles Conference for members of PC Project’s MSAB and other distinguished guests.
- A meeting of the MSAB board was held March 3 in San Francisco after Obstacles conference. Some immediate goals set included locating companies who can manufacture siRNA to the quality needed (GMP) for use in humans. Two outstanding companies are Dharmacon which has supplied most of the research siRNA. At the end of March, Roger Kaspar, Mary Schwartz and Sancy Leachman met with Greg McParland of Girindis to discuss their options for GMP quality siRNA.
- Notification was sent to several hundred dermatology applicants announcing the PC Research Fellowship.
- A Supplement to the Questionnaire was approved by the IRB and sent to those patients who had completed the Questionnaire.
- Dr. Sancy Leachman conducts a clinical trial with Janice Schwartz using Tazorac as a topical application. Although the treatment is not really expected to work, the trial is useful to set up protocols for future clinical trials.
- PC Fellow Dr. Mark Elliason developed a severity scale and pain scale which may also be useful for future clinical trials.
- PC Project is accepted as a member of the Coalition of Skin Disease (CSD) which is an umbrella support group of organizations devoted to skin disease. The CSD works closely with the American Association of Dermatology (AAD) and coordinates the displays at the AAD annual meeting.
- The Journal of Investigative Dermatology: 2004 PC Symposium Proceedings issue was completed after months of preparation and published this month. A complimentary copy was mailed to each patient who is part of the IPCRR. Copies were also mailed to each author and sub-author, to all members of the IPCC, and to other physicians who have been listed by PC patients.
- The American Society of Human Genetics (ASHG) conference was held in Salt Lake City, UT on October 26-29. PC Project had a booth. Also, a poster presentation by Roger Kaspar and Robyn Hickerson demonstrated sequence-specific siRNA effectively inhibiting mutant PC cells.
- PC Project hosted a dinner meeting for a select group of conference attendees who have a specific interest in skin and keratin disorders.
- A series of informative lectures were given at various locations in Orem and Salt Lake City by Roger Kaspar outlining the exciting developments in siRNA therapy for PC.
- The CORT grant was submitted to the NIH/NIAMS for consideration.
- A provisional patent filed by TransDerm for PC therapy
- A marvelous and successful Patient Support Meeting took place in Niagara Falls August 25-27, 2005 with more than 100 in attendance including 36 PCers with their family members and 12 outstanding clinicians and scientists. Just before the meeting PC Project received approval to conduct sample collection at the meeting and this activity was well supported by the attendees. Sample collection included digital photography, confocal photography, tape strip collection, skin shards and hair samples.
- Planning for the upcoming Patient Support Meeting in Niagara Falls continues. PC Project has applied for a NIH-NIAMS grant to support the 2006 IPCC Scientific Meeting.
- Additional staff are assisting with PC activities. In addition to Dr. Mark Eliason, there are several administrative staff working on the CORT grant application at the University of Utah. The CORT is a major NIH grant over five years which may be very important in moving our research forward to clinical trials.
- Initial contacts have been made with the FDA to being the process of seeking approval for an siRNA therapy developed to inhibit keratin genes and which appears effective in very early stage cell culture testing..
- July 1, 2005 marked the incorporation of TransDerm, Inc. headed by Roger Kaspar, founder and CEO. TransDerm has formed a partnership with PC Project and will actively pursue siRNA therapy for PC.
- The PC Project Medical and Scientific Advisorty Board (MSAB) set PC Project Goals for 2005-2006 based on the input from the IPCC meeting in May.
- Dr. Mark Eliason, MD - is the PC Project Research Fellow for 2005-2006, Dr. Eliason will begin his Dermatology residency in July 2006 at the University of Utah. He has completed a one year fellowship in Dermatology in addition to his other medical studies.
- Matt James - is our summer intern from the University of Florida. Matt will be working on non-governmental grant applications and fund raising.
- Kim Baker - our clerical assistant began working with PC Project in January and will be with us through the end of the year.
- Annual meeting of the IPCC in St. Louis, MO. More than 30 leadingclinicians and scientists attended the meeting. The presentations were excellent, the dynamics amazing, and the collaboration and ideas were powerful. A special award was given to Qian Wang of Standord University for her work on PC this past year. The CDA Award was presented to Frances JD Smith.
- The PC patient registry (IPCRR) has identified approximately 200 PC patients.
- PC Bibliography completed including locating 487 articles, creating pdf files, obtaining more than 80 translations; Bibliography available on web with search features; PC website structure re-designed and updated.
- Dr. Sancy Leachman, Mary Schwartz and Jan Schwartz met with Dr. Alan Moshell of NIAMS (NIH) to discuss options for funding for research on pachyonychia congenita.
- Three new publications were prepared (1) For Medical Professionals and Scientists (2) For Patients and (3) For Fund Raising or general use Completed agreements for licensing and other arrangements regarding biotech issues for delivery.
- Initial negotiations to establish a biotech entity to focus on delivery challenges and incorporated TransDerm as a PC biotech firm.
- Developed concept of three focus areas (1) Basic and Translational Research (2) Clinical Research and (3) Patient Support; began discussions on means to succeed in delivery of therapeutics to skin.
- Sixteen PC patients and 15 family members attended a PC Patient Support Meeting in Dundee, Scotland hosted by Irwin McLean and Frances Smith
- PC Project hosted ten PC patients and 5 family members at the FIRST Conference in Kansas City, MO; PC Project Board of Trustees organized; PC Project recognized as a Public Charity by US IRS.
- The response to the Patient Message Board prompted us to purchase new software and a new and improved PMB is now available on the website.
- IRB approval received for the International PC Research Registry (IPCRR). PC Project announced eight research grants were awarded. A Patient Support Meeting was held in Salt Lake City. The meeting included 4 PC patients and two dermatologists and questions submitted by PC patients worldwide were discussed. The meeting was videotaped and is available to view on the website.
- Grant proposals were received and reviewed for funding. In this first year, we want to explore as many possibilities as possible so that we can find the most promising research to lead to clinical trials.
- We submitted our application for IRB approval for research registry. This was a major effort for this entire month in preparing and developing the protocol, database, questionnaire, consent and other requirements.
- The first Pachyonychia Congenita Symposium was held in Park City, UT. The International PC Consortium (IPCC) was organized with 23 physicians and scientists.
- Following the Symposium, we reset our goals to allow funding of up to 3 Seed Grants for 2004. We will aim for selection of a Career Development Award (CDA) research grant recipient to follow in 2005. We held planning meetings and complete inital planning for Registry including drafting of questionnaire and initial database considerations; drafting proposal for IRB.
- Also, this month the PC Patient Message Board is up and running on the website as a double-protected site for PC patients and family members to exchange messages.
- A photo albume was added and the first PC images were added to website. A few bibliography links were added to website. PC brochures were printed for distribution at the AAD conference. We have now made contact with several PC patients through the website.
- Bibliography (draft) was posted to the website. A brochure for medical professionals and scientists was prepared. We held our first meeting with the Institutional Review Board (IRB) regarding our proposed research registry. PC Project offices were set up and a protected database established for communications and protected data. Also, an article was submitted to F.I.R.S.T.
- Pachyonychia Congenita Fund (PC Fund) aka PC Project was incorporated as a US public charity under 501(c)(3).
- First PC Project website posted to internet including grant award information. Citations for a PC bibliography were identified. Filed with the U.S. Internal Revenue Service (IRS) for status as a public charity
- Dr. Ralph Bradley referred us to Dr. Sancy Leachman at the University of Utah. She is a dermatologist who specializes in skin cancer genetics. Met with Dr Leachman and established objectives for the PC Project including the need to make PC known among leading researcher and thought leaders so that all possibilities can be explored and the best options selected for success.
- Established inital funding for PC Project in a charitable gift account.
- Schwartz family sent email requests to institutions and researchers, but received no response. We conducted research to identify rare disease organizations. We also contacted an attorney regarding organization of a charity. Then we saw the article “Saving Ryan” in Reader's Digest and we set the goal to seek a cure for PC.