Dr. Michael Hayden, MB, ChB, PhD, FRCP(C), FRSC, C.M., O.B.C
Dr. Michael R. Hayden, MB, ChB, PhD, FRCP(C), FRSC, C.M., O.B.C is the Canada Research Chair in Human Genetics and Molecular Medicine, University Killam Professor in the Department of Molecular Genetics and University of British Columbia Senior Scientist at the Center of Molecular Medicine and Therapeutics. He and his team are dedicated to finding a treatment for HD. They are investigating biomarkers for assessing neuroprotection in the brain as a measure of treatment efficacy.
RESEARCH: Therapeutics aimed at lowering mutant HTT (mHTT) in the central nervous system (CNS) prevent regional forebrain atrophy and improve HD-like behavioural phenotypes in preclinical studies and are currently in clinical development for HD. Neurofilament light chain (NfL), a neuron-specific component of the cytoskeleton, is released from the CNS into the CSF following neuronal damage. In HD, concentrations of NfL in biofluids correlate with brain atrophy and disease progression. However, it is unknown how levels of NFL would be affected when mutant HTT (mHTT) levels are reduced in the CNS. We therefore hypothesized that lowering mHTT in the CNS may stabilize NfL concentrations in biofluids by improving neuronal health in the CNS. Using the YAC128 mouse model of HD, we are investigating the effect of mHTT lowering in CNS using an antisense oligonucleotide on NfL concentrations in biofluids.
2020 Recipients
Dr. Michael Hayden, MB, ChB, PhD, FRCP(C), FRSC, C.M., O.B.C
Dr. Michael R. Hayden, MB, ChB, PhD, FRCP(C), FRSC, C.M., O.B.C is the Canada Research Chair in Human Genetics and Molecular Medicine, University Killam Professor in the Department of Molecular Genetics and University of British Columbia Senior Scientist at the Center of Molecular Medicine and Therapeutics. He and his team are dedicated to finding a treatment for HD. They are investigating biomarkers for assessing neuroprotection in the brain as a measure of treatment efficacy.
RESEARCH: Therapeutics aimed at lowering mutant HTT (mHTT) in the central nervous system (CNS) prevent regional forebrain atrophy and improve HD-like behavioural phenotypes in preclinical studies and are currently in clinical development for HD. Neurofilament light chain (NfL), a neuron-specific component of the cytoskeleton, is released from the CNS into the CSF following neuronal damage. In HD, concentrations of NfL in biofluids correlate with brain atrophy and disease progression. However, it is unknown how levels of NFL would be affected when mutant HTT (mHTT) levels are reduced in the CNS. We therefore hypothesized that lowering mHTT in the CNS may stabilize NfL concentrations in biofluids by improving neuronal health in the CNS. Using the YAC128 mouse model of HD, we are investigating the effect of mHTT lowering in CNS using an antisense oligonucleotide on NfL concentrations in biofluids.
Dr. Jodi McBride, PhD
Dr. Jodi McBride of Oregon National Primate Research Center in Beaverton, or is working to develop therapeutics to slow the progression of HD in the rhesus macaque monkey model. She and her team are working to correlate biomarkers to imaging modalities. It is hopeful that biomarkers of neurodegeneration and neuroinflammation will be established to evaluate therapeutics.
Wenzhen Duan, M.D., Ph.D, | Professor of Psychiatry & Neuroscience | Director, Laboratory of Translational Neurobiology
Wenzhen Duan, M.D., Ph.D, Associate Professor of Psychiatry and Director of Laboratory of Translational Neurobiology at John Hopkins University. Wenzhen and her team are investigating the possibility of converting astrocytes to functional new neurons as a therapeutic cell replacement therapy.
RESEARCH:
JHU Neurobiology | JHU Neuroscience | JHU CMM graduate program
Wenzhen Duan, M.D., Ph.D, | Professor of Psychiatry & Neuroscience | Director, Laboratory of Translational Neurobiology
Wenzhen Duan, M.D., Ph.D, Associate Professor of Psychiatry and Director of Laboratory of Translational Neurobiology at John Hopkins University. Wenzhen and her team are investigating the possibility of converting astrocytes to functional new neurons as a therapeutic cell replacement therapy.
RESEARCH:
JHU Neurobiology | JHU Neuroscience | JHU CMM graduate program
Summerfield Health Care Center
Summerfield Healthcare Center in Cloverdale, Indiana is one of only 4 facilities nationwide that care specifically and exclusively for patients with Huntington’s Disease. Each year the foundation grants the facility funds to benefit patient care and comfort. This year $3,120.00 was awarded to purchase a restraint-free, fall reduction chair that provides safety, as well as comfort, for residents in the advanced stages of the disease.
Courtney Treharn-Gatza MSW, LSW
Courtney has been with the Indiana University HD clinic since 2015. In her role as the clinic social worker, she provides support to patients and their families during their journey through HD. This often comes in the form of emotional support, guidance toward available resources, and multiple aspects of clinic coordination. In 2019, she also began working with the Enroll HD study and helps coordinate and conduct visits with participants at IU. In addition to providing clinic resources, Courtney also serves as a resource to families who may be impacted by HD but not receiving care through IU. She works with families across the state to identify local supports, provide education, and assist in referrals for predictive testing for individuals who live at-risk. She also assists in facilitating, developing, and supporting local support groups. When COVID-19 isn’t disrupting social events, she also organizes the annual Indiana HD Symposium which connects our local HD community with local resources and experts in the field.
Courtney Treharn-Gatza MSW, LSW
Courtney has been with the Indiana University HD clinic since 2015. In her role as the clinic social worker, she provides support to patients and their families during their journey through HD. This often comes in the form of emotional support, guidance toward available resources, and multiple aspects of clinic coordination. In 2019, she also began working with the Enroll HD study and helps coordinate and conduct visits with participants at IU. In addition to providing clinic resources, Courtney also serves as a resource to families who may be impacted by HD but not receiving care through IU. She works with families across the state to identify local supports, provide education, and assist in referrals for predictive testing for individuals who live at-risk. She also assists in facilitating, developing, and supporting local support groups. When COVID-19 isn’t disrupting social events, she also organizes the annual Indiana HD Symposium which connects our local HD community with local resources and experts in the field.
Learn More
What is Huntington’s Disease?
Huntington’s Disease is a degenerative brain disease that strikes in mid-life, usually between the ages of 30-40. People lose their ability to walk, talk, and even feed themselves. Even though people may live for 10-20 years with this disease, their quality of life is taken from them.
Who Was Bev Hartig?
In 1998, about one year after getting married, Bev received a paralyzing phone call that her birth father had been diagnosed with HD. This meant every sibling had a 50% chance of inheriting this disease. She found out that she also carried the gene and would suffer the same tragic fate.