Page 7 - Fall 2020 CMTA Report
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family with CMT, thrilled to
the feeling of finally getting to
be around people who under- CMT1A, CMT1B, CMT4B
stood what life with CMT is
like. Prior to Camp Footprint,
she said, her six-year CMT jour- PROJECTS APPROVED
ney was something she had to
navigate alone: “In some ways, it
was like everyone’s angsty THE CMTA BOARD OF DIRECTORS approved $360,000 in new grants
teenage belief was true—quite to top CMT scientists at its September meeting. Covering work on
literally, no one around me types 1A, 1B and 4B, the grants represent the CMTA’s commitment to
understands.” At virtual Camp finding treatments for the community.
Footprint, by contrast, “Day
after day, the screen lit up with The projects are aimed at improving understanding of disease
over 100 beautiful faces who mechanisms, discovering new therapeutic targets and preparing for
know exactly what it’s like and and moving into clinical trials.
can understand those pieces of For more than three decades, the CMTA has funded basic, clinical
me that I’ve carried alone with- and translational research, investing more than $16 million through
out having to say a word. The STAR in the last decade alone to develop therapies for the community.
joy in that togetherness, and the The new projects include:
sense of peace that came with
seeing other happy, thriving
people with CMT across all age
groups, was such a gift. I cannot CMT1A Project: The board awarded Dr. John Svaren,
wait to experience it in person University of Wisconsin Madison, $60,000 to explore
one day!” approaches for lowering the expression of the gene PMP22
Returning counselor Erin in animal models. If successful, this approach—using an FDA
Weierbach, 34, said the moments approved drug and similar compounds—could lead to an
that made the strongest impres- accelerated treatment for patients.
sion on her came when the 52
counselors least expected it: “In
between organized activities and CMT1A Project: The board awarded Dr. Luigi Puglielli,
planned engagement, campers University of Wisconsin Madison, $89,889 to explore a
connected through opportunities potential new target for treating CMT1A. He will conduct
to ask questions, getting to know genetic, cellular and pharmacologic tests to validate this target.
each other and share stories.
Bonds formed in front of us as CMT1B Project: As part of that $89,889 grant,
young people celebrated each Dr. Puglielli will also explore a potential new target for
other’s differences, similarities treating CMT1B, conducting genetic and pharmacologic
and shared experiences and our tests to validate this target.
tribe grew by many.”
Berger found it unsurprising
that a group of campers and staff CMT4B1/B2 Project: The board awarded $201,000
used to fighting through CMT’s to Dr. Alessandra Bolino, Ospedale San Raffaele in Milan,
daily challenges refused to let Italy, to explore treating CMT4B1 and CMT4B2 with an
COVID-19 get in the way of
their week in the Tribe of the FDA-approved drug. In addition, Dr. Bolino’s team will
Funky Feet. And, while he hopes conduct studies with CMT4B1 and 4B2 patients to better
that campers and counselors will understand the natural progression of the disease and help
be able meet in person next year, prepare for clinical trials in the future.
he says that whatever happens,
“Camp Footprint will keep
moving forward—one footstep
at a time.” h
FALL 2020 THE CMTA REPORT 7
