Page 7 - 2021 Winter CMTA Report

CMTA Board Approves
book series; Against All Odds:
Black Women in Medicine; and
Grants to Jackson Lab to
Master Builders of the Modern
World: Reimagining the Face
Study Two Inhibitors
of STEM (science, technology,
engineering and math).
Crystal is also the founder
and CEO of URU The Right to he CMTA Board of
Be, Inc., a non-profit content Directors approved two
production company that research grants to Robert
fosters communication and Burgess, PhD, at the Jack-
understanding among diverse T son Laboratory in 2020.
people by discovering and The first grant of $45,000
applying tangible multimedia will be used to explore HDAC6
solutions at the intersection of (histone deacetylase 6)
the arts, humanities, science inhibitors, a potential therapeu-
and technology. Crystal’s goal is tic approach for multiple forms
to move all stakeholders toward of CMT. The many genes associ-
a more equitable and humane ated with CMT make it unlikely
world. She also conceived, that a single treatment will work
designed and launched the for all forms of the disease, but Dr. Robert Burgess, PhD
innovative Changing the Face of preclinical studies with HDAC6
STEM national educational ini- inhibitors, which have been With the second grant of
tiative in 2015. The culmination shown to reduce motor and sen- $110,000, Dr. Burgess will study
of this initiative was a 2017 sory deficits, have demonstrated whether inhibiting SARM1 is of
congressional forum in Wash- promising results in several therapeutic benefit in multiple
ington D.C., in conjunction with mouse models of CMT. Based on forms of CMT. SARM1 (sterile
the National Academy of Sci- these promising results, scien- alpha and toll interleukin recep-
ence, attended by more than tists believe that HDAC6 tor motif containing protein 1)
500 STEM professionals, poli- inhibitors might be beneficial in was identified in fruit flies as
cymakers, educators, students treating a wide array of neurode- being actively involved in axon
and members of the media. generative conditions including degeneration and similar effects
Today, Crystal uses a demyelinating (Type were shown in SARM1
wheelchair and a ventilator, 1 and 4) and axonal Preclinical knockout mice.
relying on friends and health (Type 2) CMTs. models of The hypothesis is
aides to help her eat, bathe and Dr. Burgess, a HDAC6… have that inhibition of
get in and out of bed. It takes member of the demonstrated SARM1 may be bene-
her 2.5 hours to get from bed to CMTA’s Scientific promising results ficial in some, but not
wheelchair in the morning. Advisory Board, will in several mouse all, forms of CMT.
That’s a challenge, she says, but use mouse models of models of CMT. The results obtained
the bigger challenges come in several forms of CMT in these five CMT
the way CMT affects her ability to determine which types may be models (1A, 1X, 2E, 2D, 2S) will
to hang out with friends, go to candidates for treatment with be definitive and publishable and
her family’s homes and work as HDAC6 inhibitors and whether are not part of a larger effort on
an artist. HDAC6 inhibitors may be of SARM1 in the Burgess lab. How-
Nonetheless, she says, therapeutic benefit across a vari- ever, these results are important
“CMT doesn’t dominate me. ety of CMT types. The latter will for the field and relevant to drug
It’s just like any other obstacle. help inform whether patients development efforts that are
You just have to figure out how with genetically undiagnosed already underway. Furthermore,
to get around it.” Quoting cases of CMT are likely to bene- though this proposal is a pilot of
Henry Ford, she added, “Obsta- fit from this therapeutic modest scale, the results will
cles are those frightful things strategy, or whether only select inform future research on just
you see when you take your forms of CMT may respond to how generally applicable SARM1
eyes off your goals.” k this treatment. inhibitors may be in CMT. k

WINTER 2021 THE CMTA REPORT 7