Pachyonychia Congenita Fund

Fighting For a Cure. Connecting & Helping Patients. Empowering Research

aka PC Project   |   Holladay, UT   |  www.pachyonychia.org

Mission

PC Project was first recognized as a public charity in the USA in 2004. The goal: Find a cure for Pachyonychia Congenita. As we understand more about PC through larger numbers of patient participants, we realize the cure isn't hiding out there somewhere. In fact, we won't 'find' the answer -- we will have to 'develop' the drug or treatment for PC. We've change the word from 'find' (which is somewhat passive) to 'fighting' for a cure which much better describes the effort required to develop a new treatment. At PC Project, we are fighting for a cure, connecting and helping patients and empowering research. And, we are successful because of the participation from the patient community. Thank you for joining with us as we move forward to success in these areas.

Ruling year info

2004

Executive Director

Mrs. Janice N. Schwartz

Main address

P.O. Box 17850

Holladay, UT 84117 USA

Show more contact info

EIN

68-0567493

NTEE code info

Alliance/Advocacy Organizations (E01)

Alliance/Advocacy Organizations (G01)

Alliance/Advocacy Organizations (H01)

IRS filing requirement

This organization is required to file an IRS Form 990 or 990-EZ.

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Communication

Blog

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Pachyonychia Congenita (PC) is a chronically debilitating ultra-rare autosomal dominant skin disorder. It is caused by a mutation in one of five keratin genes: KRT6A, KRT6B, KRT6C, KRT16, or KRT17. The most common physical symptoms are painful calluses and blisters on the soles of the feet and palms of the hands (palmoplantar keratoderma). Other common features of PC include nail dystrophy (thickened nails), multiple cysts (also extremely painful), oral leukokeratosis (white plaques on the tongue) and follicular hyperkeratosis (sensitive bumps all over the body). The most striking feature of PC is the extremely painful and debilitating plantar keratoderma which severely affects patients’ mobility. PC patients often crawl and use walking aids such as crutches or a wheelchair. Currently, there is no cure or specific treatment for PC. Patients worldwide live in isolation, with no hope and many have not been diagnosed correctly.

Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

PC Research

PC Project supports research at many centers around the world and sponsors an effective collaboration of more than 50 leading researchers. The success of these programs will benefit PC as well as other related skin disorders affecting thousands of people.

Population(s) Served

Since 2004, Pachyonychia Congenita Project has maintained the International Pachyonychia Congenita Research Registry (IPCRR). The patient registry is WIRB approved and now has over 700 participants with PC and similar disorders. All IPCRR services are free. IPCRR services provided to individual patients include -
• An IRB-approved Questionnaire which allows patients to report and record information about their condition.
• Optional consultation with a board-certified dermatologist to validate the information on the Questionnaire, discuss personal medical questions, consult on care (one hour minimum each patient).
• Free Genetic testing to identify the specific keratin mutation.
• A report of the genetic testing results with diagrams, explanations, copies of articles, etc. additional counseling by a physician is also provided as needed.
• After the report, patients have access to on-going services including physician consultations with members of the PC Project MSAB or physician panel, physician referral, website access to special materials and other support.

Population(s) Served

Formed in 2004, the International Pachyonychia Congenita Consortium (IPCC), an international organization consisting of over 150 scientists and clinicians who collaborate on PC related research efforts, genetic discoveries and patient care. The IPCC welcomes all scientists, physicians and industry interested in collaborative efforts to develop and deliver effective treatments for PC and related disorders. Hosts the International (IPCC) Symposium, an annual international research meeting that brings together scientists and physicians for collaboration on developing and delivering effective treatments for PC and related disorder. PC Medical and Scientific Advisory Board and PC Project Steering Committee, a smaller group of professionals who guide PC Project concerning specific research projects and assisting patients. PC Physician Network, open to all who treat or are interested in treating patients with Pachyonychia Congenita.

Population(s) Served

Each year, PC Project hosts an annual Patient Support Meeting where PC patients and their families come together to learn from leading physicians and scientists who provide information and training on how to care for and live with this isolating, painful disease. These meetings are not only educational but emotional and even life-changing for PC patients who have never met another person with PC. In addition to adult sessions, meetings include sessions for teenagers and children who learn about the science behind PC, how to deal with pain, social issues, difficult questions, bullying, and how to stay focused on their positive strengths and characteristics.

Furthermore, PC Patient Support Meetings serve as an opportunity for doctors and scientists to study and learn directly from PC patients. Various studies and focus groups conducted at Patient Support Meetings over the years have resulted in greater knowledge for clinicians and researchers due to the benefit of having a gathering of rare patients to learn from. The hope is that if an effective treatment is found for PC, it may benefit those suffering from other related keratin skin disorders.

The first PC patient support meeting for patients within the UK and other parts of Europe was held in Dundee, Scotland in 2004. Patient Support Meetings have continued in the United Kingdom/Europe every two years with meetings held in the USA in the intervening years. The benefit to PC patients and their caregivers as well as attending researchers and physicians makes this meeting invaluable to the overall PC community.

Additional feedback from PC patients who attended a Patient Support Meeting (PSM):

“At my first PSM meeting, for the first time I looked into the eyes of another person with the same disease, saw them move like me, and use the same tricks I use to get through the day. This gave me an immense feeling of no longer being alone and it's a wonderful feeling. I would say that I've never been as happy and confident before the PC PSM experience.”

“Attending the PSM is the one best thing you can do to help the way you psychologically handle your PC. It has finally allowed me to be able to talk openly about my PC.”

“It's amazing, comforting and helpful to be able to relate to other people with the same skin condition.”

“I have hope again.”

Population(s) Served

Patients are supported worldwide through PC Project and the International PC Research Registry.

Population(s) Served

Patients are reached through the website, social media platforms, news briefs and direct emails.

Population(s) Served

Where we work

Our results

SOURCE: Self-reported by organization

How does this organization measure their results? It's a hard question but an important one.

Number of clients participating in support groups

This metric is no longer tracked.
Totals By Year

Number of patients enrolled in the IPCRR (Registry)

This metric is no longer tracked.
Totals By Year
Related Program

International PC Research Registry

Number of people engaged in social media platforms

This metric is no longer tracked.
Totals By Year

Number of published PC related articles

This metric is no longer tracked.
Totals By Year
Related Program

International PC Consortium

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

PC Project’s mission is three-fold:

1. Help and support PC patients and their caregivers as they navigate life with an extremely painful disease. This includes educating people who affect PC patients, including the clinicians who serve them, school officials, workplace leaders and others.
2. Empower and facilitate PC related research through basic science, translational studies and clinical applications.
3. Partner with industry stakeholders interested in making a real difference in the lives of a mostly forgotten population

1. Enrolling patients in the International Pachyonychia Congenita Research Registry (IPCRR). Once enrolled, patients receive both a clinical and genetic diagnosis of PC, often knowing for the first time in their lives what disease they truly have. This is empowering for patients. Once it’s determined a patient has PC, on-going support is given through the PC community, including Patient Support Meetings, PC Patient Advocacy help, access to clinicians who are experts in PC, doctor’s letters and disability help, monthly PC News Briefs and a private social media group.

2. International Pachyonychia Congenita Consortium (IPCC), comprised of medical and scientific professionals around the world who collaborate in order to further meaningful research for PC. This group is supported by regular IPCC symposiums and quarterly newsletters. This group also utilizes the Research Registry for publications, studies and information about PC.

3. Partnering with industry stakeholders, by collaborating, sharing resources, and utilizing information from the Patient Registry.

1. Our worldwide International PC Research Registry can be accessed through out PC website. The website also has a plethora of information about PC, targeted for patients, family members and professionals, including a bibliography of all publications related to PC.

2. The IRB approved Patient Registry is overseen by medical doctors and a professional geneticist. Free genetic testing is offered for patients and access to clinicians who understand this rare disease is given to patients or their caregivers. The Registry is also utilized by researchers, resulting in over 100 publications regarding PC. The Registry continues to dispel myths and establish truth about what PC really is and how it affects patients.

3. A Medical and Scientific Advisory Board (MSAB) and an even smaller, Steering Committee, comprised of scientists and physicians who not only understand the complexities of PC but who genuinely care about PC patients. These groups guide the work of PC Project and generously volunteer their time.

4. A small, but dedicated staff who passionately cares about PC patients, a team of PC patient advocates, a consortium of scientists and clinicians who freely donate their time to this cause, a working PC Project Board of Trustees, and probably most importantly, an engaged and patient population which supports the cause. For example, in 2008, over 200 PC patients participated in person and remotely in an Externally-led Patient Focused Drug Development meeting before 42 FDA officials. This meeting allowed PC patients to share their voices with key FDA officials who make critical decisions regarding treatments for PC. A direct result of this meeting was a Fast Track Designation by the FDA for a treatment for PC patients.

Established a safe community where PC patients can be supported and support one another with this isolating, debilitating disease which many feel embarrassed about. This is done through the pachyonychia congenita website at www.pachyonychia.org, access to clinicians who understand PC, social media platforms, including a private Facebook chat, 17 Patient Support Meetings held annually at various locations, monthly patient news briefs, patient advocacy program, numerous phones calls and emails to assist patients.

Built an IRB approved International PC Research Registry that has over 1900 patients. By offering free genetic testing, patients have a genetic diagnosis which is empowering when many have lived their entire lives not knowing or understanding their disease. This Registry also allows researchers to have real data about PC, and a body of willing patients available for studies. PC Project will continue to grow this registry with approximately two new patients per week.

Facilitated a scientific community dedicated to PC related research. Hosted 15 annual scientific meetings, supported the IPCC members who published over 100 articles relevant to PC in scientific journals, sponsored over 20 clinical studies and two phase 1b clinical trials, including the first ever phase 1b siRNA trial for skin in a human. In collaboration with the scientific community, PC Project gives researchers access to de-identified data from the PC patient registry.

Collaborated with industry partners interested in furthering research into meaningful treatments for PC patients, including a biopharmaceutical company preparing for a 2/3 phase clinical trial.

Hosted an Externally-led Patient Focused Drug Development Meeting where 42 FDA officials heard the voices of over 200 PC Patients who participated in person and remotely and gave testimonials of living with PC and managing PC.

In addition to its programs, qualified staff members will continue field thousands of emails and calls from doctors, scientists, current patients and newly diagnosed PC patients in order to answer questions, share information, and provide support. It will also continue to send letters of support for disability accommodations.

Most importantly, PC Project will continue its mission of helping patients, encouraging PC research and working with industry stakeholders until a cure is found for PC, or at the very least, meaningful treatments are available to patients which will stop the life-affecting pain and increase mobility and quality of life. Until the day comes, PC Project is dedicated to the daily work to achieve its mission.

Financials

Pachyonychia Congenita Fund
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Operations

The people, governance practices, and partners that make the organization tick.

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Pachyonychia Congenita Fund

Board of directors
as of 06/18/2019
SOURCE: Self-reported by organization
Board chair

Mr. Jack Padovano

C. David Hansen

University of Utah

Jack Padovano

IPM Advancement

Janice Schwartz

Barbara Feinstein

USAID

Jason Hunter

BDO

James Rittle

United Airlines

Board leadership practices

SOURCE: Self-reported by organization

GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.

  • Board orientation and education
    Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes
  • CEO oversight
    Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes
  • Ethics and transparency
    Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes
  • Board composition
    Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes
  • Board performance
    Has the board conducted a formal, written self-assessment of its performance within the past three years? Yes