News

August 2017
RVCL Center Family Meeting and Video

We thank everyone who attended our half day family meeting held Saturday August 26, 2017. This educational meeting summarized new research studies, provided updated information about the recently begun clinical therapeutic trial, and offered advice about genetic testing, prenatal diagnosis and alternative reproductive strategies.

You can watch each presentation that was made at the family meeting by clicking the link provided below and choosing each presentation you wish to watch. Here is a summary of the presenters, their topics and approximate length of presentation:

#1  John Atkinson, MD, Professor of Medicine and Director of the RVCL Research Center at Washington University School of Medicine (WUSM) provides a welcome and introduction (25 min)

#2  Andria Ford, MD, MSCI,  Associate Professor in Neurology (WUSM), describes  neuroimaging assessments (20 min)

#3  M. Gilbert Grand, MD, Professor of Ophthalmology describes eye testing and results (24 min)

#4  Kathy Liszewski, Assistant Professor of Medicine and RVCL Research Coordinator (WUMS), provides an update on the Clinical Trials (13 min)

#5  Madonna Bogacki, RVCL Administrator (WUMS), describes how the RVCL clinical trials are paid for and patient financial resources (13 min)

#6  First Question & Answer Session (18 min)

#7  Jonathan Miner, MD,  PhD, Assistant Professor of Medicine (WUSM), summarizes his RVCL research of developing a mouse model of RVCL (17 min)

#8  Nan Yan, PhD, Associate Professor at University of Texas Southwestern Medical Center, describes his RVCL research (18 min)

#9 Sandy Morse, MD, Chief Virology Section, National Institutes of Health, summarizes his RVCL research (8 min)

#10 Stacey Barton, MSW, LCSW, discusses genetic testing (9 min)

#11  Diana Gray, MD, Professor of Obstetrics and Gynecology (WUMS), advises about prenatal screening and diagnosis (17 min)

#12 Emily Jungheim, MD, MSCI, Associate Professor Fertility & Reproductive Medicine (WUSM) describes assisted reproductive technologies (9 min)

#13 Final Question & Answer Period (7 min)

Click below to access videos:

8-26-2017 RVCL Family Meeting Video

 

December 2016
First clinical trials begun

The first clinical trial utilizing Aclarubicin for treatment of RVCL commenced December 5, 2016 at Washington University School of Medicine. Two patients initially received infusions of Aclarubicin for four consecutive days with blood work and other testing prior to and at the conclusion of the infusion. The study will be conducted for up to two years with patients participating in monthly cycles of five consecutive days per week (Day 1 for testing to verify the patient can participate followed by four days of infusions).

This clinical trial followed nearly a year of preparations including approval by the Food & Drug Administration for our Investigational New Drug application (IND # 131473, approved 8/2/16) entitled, “A Pilot Study of Aclarubicin for the Treatment of Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL).”

For more information about the trials, contact Madonna Bogacki at 314-362-8391 or mbogacki@wustl.edu

For more information:  Read about specific goals and protocols here »

January 2016
Robert Clark of Clayco donates to Washington University for study of wife’s fatal disease

January 4, 2016

Robert Clark and his partners at Clayco have pledged $4.1 million to Washington University for research into the rare disease cerebroretinal vasculopathy. Clark’s wife Ellen died five years ago at age 50 from the disease that affects blood vessels in the brain.

Continue reading on the St. Louis Post-Dispatch website »


$4.1 million Clayco Foundation gift aids research into rare disease

January 4, 2016

“This generous gift by longtime supporter Bob Clark officially establishes Washington University as the world’s leader in CRV research and related diseases,” said David H. Perlmutter, MD, the School of Medicine’s new dean and executive vice chancellor for medical affairs.

Continue reading on the School of Medicine website »


Doctor researches rare diseases

February 2015  THE RHEUMATOLOGIST

John P. Atkinson, MD, is a great detective. But instead of solving murder mysteries, he unravels the mysteries behind diseases—diagnosing them, understanding their origin or cause, and identifying effective treatments.

Continue reading on The Rheumatologist website »


Caregiver inspires crusade: Patient spearheads fundraising efforts for research into rare disease

February 2011

For 20 years, Kim Morey wondered and worried if she had inherited the devastating disease that killed her father at age 49. But she continued with her life, getting married and having three children, in her hometown of Bentonville, Ark.

Continue reading in Outlook Magazine »


Feature photo: In RVCL, blood vessels deteriorate. As seen in this picture of the retina of an RVCL patient, white areas show normal blood vessel growth, while the darkened area shows where blood vessels have “dropped out.”