Vol 17, No.2 Mar-Apr 2022
The Power of One
With a condition as rare as PC, the efforts of each person add up quickly. In this newsletter, we feature stories that reflect the difference one person can make. A patient publicly shared his PC story and raised awareness in his country, a volunteer helped create a new PC flyer that was passed out to hundreds of dermatologists, an advocate called US patients to tell them about the current clinical trial. Additionally, each person who joins the IPCRR, participates in a PC study, or creates a fundraiser makes a huge difference.
Never think because you are just one person that your contributions won’t matter. They do. And they are powerful. The work and services of PC Project would not be possible without the help of each of one of you!
New PC Brochure
One of the challenges PC patients have is trying to help others understand PC and it’s impact on their lives. With the help of Rutu Patel, a volunteer, we now have a new brochure that succinctly explains PC and the services of PC Project. Hundreds of these brochures were given to dermatologists in March at the American Academy of Dermatology (AAD) Annual Meeting.
You can download the complete document here and use it to help educate family, friends, medical professionals, teachers, employers, and others about PC. You can even share the brochure for PC Awareness month in June!
VAPAUS Phase 3 Clinical Trial
The study site in the UK is nearly ready to receive patients for the Phase 3 trial. Those in the UK who filled out the interest form will be contacted within the next few weeks. (We know UK patients have been patiently waiting for the site to open!)
Meanwhile, trial enrollment is ongoing in the US. If you live in the US and have PC type K6a, K6b, K6c, or K16, are over 18 years old, and did not have the medicine from the last trial applied to your feet, we still need you!
If you participate in the trial, you will have your 4 doctor’s visits at one of the following sites:
- Portland, OR (OHSU)
- New Brighton, MN (Clinical Research Center)
- Orange Park, FL (Park Avenue Dermatology)
- Austin, TX (DermResearch, Inc.)
- Palo Alto, CA (Stanford)
- New Haven, CT (Yale University)
- Salt Lake City, UT (University of Utah)
- London, England for UK patients
The study sponsor (not PC Project) will pay for all travel costs for you and a companion for the 4 office visits needed over an 8-month period. You will also receive a small stipend for each visit. If you are interested in learning more, please fill out this form or visit pachyonychia.org/vapaus/
Many thanks to PC Advocate, Roseann McGrath, who volunteered her time to call PC patients in the greater New York area. Roseann invited them to come to New York City to evaluate if the trial would be a good fit for them. All US patients were also extended the same invitation by Roseann at a Town Hall meeting.
On Saturday, April 23, a group of 11 PC patients along with their loved ones, met individually with Dr. Steven Kempers from the Minnesota trial site in New York City. Roseann and her husband, Michael, warmly welcomed patients and hosted a luncheon for all who came.
Although this event was not sponsored by PC Project, we appreciate Roseann’s involvement as an official PC Project Advocate to ensure our PC community members were well cared for. We also sincerely thank the patients who attended, and thank ALL patients in the study at various sites. Your service is a benefit to the entire PC community!
PCer Shares His Story
For Rare Disease Day 2022, PC advocate Tom Baker, shared his PC experiences with a reporter who wrote a news article to raise awareness about the ultra-rare PC. Publicly letting others know you have PC can be difficult, but Tom courageously taught others about living with this challenging condition.
You can read the full article on Wales Online or at pachyonychia.org/pcfeetarticle-tom/
We admit to smiling when the article warned of a “graphic image” only to find the photo was simply of Tom’s cute PC feet!
If you want to write your experiences about living with PC with our community, please send them to us at info@pachyonychia.org and we will include them in a future newsletter!
PC Town Hall
Dr. David Hansen, Dr. Steven Kempers, and Roseann McGrath joined PC Project for our Town Hall on April 5th, with a special emphasis on the VAPAUS Phase 3 Clinical Trial, an update on PC Project, and a question-and-answer session.
You can watch the recorded Town Hall at youtube.com/watch?v=gW2uoxvUmwE&t=1103s
We gratefully acknowledge those who give their time and talents behind the scenes to prepare for these meetings. Here are a few of their smiling faces:
Neurovascular Structures in PC
The Journal of American Academy of Dermatology (JAAD) recently published a paper based on data provided by participants in the International PC Research Registry (IPCRR) about a little-known feature of PC that affects 62% of
patients: Genotype-phenotype correlations of neurovascular structures on the feet in patients with pachyonychia congenita: A cross-sectional study
Written primarily by Xiang Li Tan and her mentor Professor Edel O’Toole, the article was made possible by PC patients who join the registry and continue to share their confidential data for research. This and all other publications about PC can be found by searching in the PC Research Articles on the PC website at https:// www.pachyonychia.org/research-articles/
Dermatological Research Medal awarded to PC Expert Edel O’Toole
Edel O’Toole receiving her award from Daniele Bergamaschi,
president of the British Society of Investigative Dermatology.
Congratulations to Professor Edel O’Toole, a key member of PC Project’s scientific and medical team, for earning the British Society for Investigative Dermatology Medal for Contributions to Dermatological Research.
Facebook Fundraisers
Prof. O’Toole regularly contributes her time and expertise to PC Project. She will soon lead the UK site for the VAPAUS study and was recently awarded a large grant from the Leo Foundation to study steatocystoma multiplex which PC-K17 patients especially struggle with. We are honored to have someone of Dr. O’Toole’s caliber and compassion working for PC patients.
PC Project at the AAD
PC Project attended the American Academy of Dermatology (AAD) Meeting
in March to raise PC awareness and spread news about the current clinical trial to dermatologists.
We also collaborated with pharmaceutical representatives and worked with the Coalition of Skin Diseases leaders on advancing patient voices in
the dermatology world.
Coalition of Skin Diseases (CSD) leaders and patient advocates at AAD meeting 2022
IPCC Symposium Preparations
PC Project is busy getting ready for our annual meeting which gathers members of the International Pachyonych- ia Congenita Consortium (IPCC) of scientists, physicians, and pharmaceutical representatives to present and col- laborate regarding PC-related research and drug devel- opment. Because while we continue to help patients, finding treatments and ultimately a cure for PC is a critical part of PC Project’s mission!
