I met Tacie Fox—a trustee of the Fox Family Foundation, which funds research on Huntington disease (HD)—last November at the Huntington Study Group (HSG) 2018 conference in Denver, Colorado. There, she told me about her family’s history with HD. Characterized by worry, suffering, and even death, Tacie’s story was devastating, but it was also hopeful. She expressed immense gratitude and admiration for the researchers who are working around the clock to ﬁnd treatments and a cure for HD, and she had enormous faith that the results of this work would prevent her own two teenaged children from becoming yet another sad chapter in her family’s narrative.
The clock is ticking for people like Tacie and her already diagnosed family members, who struggle to be patient, even as their lives unravel around them. But HD researchers and clinicians, fully aware of the imperativeness and urgency of their tasks, continue to plow ahead. In fact, sitting in the conference sessions and listening to these men and women speak I was impressed by just how seriously they take their jobs. In the words of Tacie Fox, “For them, it’s not just a job and a career; it’s a life mission.”
Since the conference, two important breakthroughs have occurred in the ﬁght against HD. First, IONIS Pharmaceuticals, Inc., announced that its drug IONIS-HTTRx successfully and safely lowers the toxic mutant huntingtin protein (mHTT) in people with HD. The company now is planning a larger trial to test whether IONIS-HTTRx slows disease progression. Experts all agree that this is an unprecedented step toward ﬁnding an effective treatment for HD.
Second, Spark Therapeutics received approval by the Food and Drug Administration in December for its gene therapy Luxturna, which treats retinal dystrophy, a rare inherited form of blindness. The treatment actually restores vision to people who were born blind. With the approval of Luxturna by the FDA, there’s little doubt that a gene therapy will soon be available for HD.
Both of these recent developments are described in more detail in this issue of HD Insights. In addition, we’ve included an interview with Tacie Fox, with the goal of helping you—our readers—feel even more connected to your friends and colleagues in the HD community.
As the new deputy editor of HD Insights, I ﬁnd myself becoming entrenched in this community, along with the scientists, clinicians, communicators, patient advocates, and most importantly patients and families, who comprise it. It’s an incredible feeling, to join ranks with these people, each of whom contributes his or her own positive, hopeful, and determined energy toward ﬁnding a cure.
As always, if you have suggestions for future content, we would love to hear them. We also would like to post any jobs you may have available. Contact me directly at email@example.com or submit your jobs and stories ideas online at http://huntingtonstudygroup.org/hd-insights/.
Send a message to the author: