Planning a ‘Dancing at the Vatican’ screening to celebrate the global Huntington’s disease community’s journey

On February 19, the University of San Diego (USD) will host the world’s third screening of Dancing at the Vatican, the short documentary featuring South American Huntington’s disease-afflicted families’ historic 2017 encounter with Pope Francis at the Vatican.

As I noted in my preview before the July 2019 premiere of this 38-minute film in Los Angeles, Dancing at the Vatican captures key moments of those impoverished, disease-stricken families’ journey to their meeting with the Spanish-speaking Francis, the first Latin American pontiff in the Catholic Church’s 2000-year history. It was extraordinary: some had never ventured beyond their home towns; some even lacked birth certificates.

Now, as both an HD advocate and faculty member in USD’s Department of History, I’m helping organize the upcoming screening, and hope many more people will see it. 

Dancing at the Vatican also will be shown in London on February 5. Showings are also confirmed for Washington, D.C., in March (date and place TBA), and at the Huntington’s Disease Youth Organization conference in Glasgow, Scotland, in May. Screenings are under consideration for South America, too. Ultimately, the film will become available online.

In the words of producer and narrator Charles Sabine – like me, a presymptomatic HD gene carrier – coming together to view Dancing at the Vatican is an occasion of “extraordinary celebration” for the Huntington’s community.

An Emmy-award-winning former NBC-TV foreign correspondent, Sabine helped spearhead “HDdennomore: Pope Francis’ Special Audience with the Huntington’s Disease Community in Solidarity with South America.” Both Sabine's father and brother died from HD.

While Dancing at the Vatican captures what I called in my preview “the underside of the HD world” – families dealing simultaneously with one of humanity’s most devastating diseases and severe poverty and discrimination – it also portrays what Sabine described as “happy tales set against the dark canvas of our disease.”

At HDdennomore, and as the film recalls, Francis became the first world leader to recognize this horrible disease. And he declared that it should be “hidden no more.” 

Pope Francis with HD families in Rome, May 18, 2017 (photo by #HDdennomore)

Faith, reason, and advocacy

At USD, the primary sponsor of the screening is Frances G. Harpst Center for Catholic Thought and Culture (CCTC). Along with other USD units, the CCTC co-sponsored my trip to Rome for #HDdennomore, and also my public presentation on the event (click here to watch).

USD is a Catholic university where “faith and reason are compatible in education,” and it “welcomes students, faculty and staff of every faith tradition,” according to its statement on Catholic identity. Indeed, since my arrival in 1993, I’ve faced no restrictions on my research on abortion in Brazil, and have taught students from many religious backgrounds.

I have explored the nexus between faith and reason/science in this blog, including the in-depth article “God, Huntington’s disease and the meaning of life.”

After CCTC Director Jeffrey Burns, Ph.D., read my preview of Dancing at the Vatican last July, he e-mailed me to ask whether we could bring the film to USD. Sabine readily agreed to the idea; he’ll introduce the film and take questions afterwards.

Ignacio Muñoz-Sanjuán, Ph.D., a leading neuroscientist seeking HD treatments at the Los Angeles office of the nonprofit CHDI Foundation, Inc., also will speak. Dr. Muñoz helped organize #HDdennomore. He co-founded Factor-H, which aids Latin America’s poor HD-affected families. Both Sabine and Muñoz will also meet with students and faculty interested in their respective professional fields.

We selected the February 19 date because Sabine, based in London, will join Muñoz and several hundred researchers from around the globe the next week at the CHDI-sponsored 15th Annual HD Therapeutics Conference in nearby Palm Springs, CA. I will also attend.

In planning the screening, I’ve strengthened the bond between advocacy and academic work that USD values and that I began to establish after exiting the terrible and lonely “HD closet” in 2012 (click here to read more).

Dr. Ignacio Muñoz-Sanjuán entering the Vatican with Dilia Oviedo Guillén, a Colombian woman who lost her husband and five children to HD (photo by #HDdennomore)

A free event, with many sponsors

The screening will take place from 6:30-8:30 p.m. in USD’s Manchester Auditorium (located in Manchester Hall) and will be followed by a reception. The event is free and open to the USD community, the local HD and biomedical communities, and the public. Attendees must register at cctc@sandiego.edu or 619-260-7936.

To fund the event, we have secured support from Ionis Pharmaceuticals, Inc., the developer of the gene-silencing drug currently under study in a historic Phase 3 clinical trial by Roche. (Click here for a recent update on the trial.) Ionis is located in Carlsbad, CA, part of the San Diego-area biotech hub, one of the world’s most important. Ionis’ chief scientific officer and HD team leader, Frank Bennett, Ph.D., donated to #HDdennomore.

In addition, Roche’s U.S. subsidiary Genentech will also sponsor the screening. Headquartered in South San Francisco, CA, Genentech also has a facility in Oceanside, just north of San Diego. 

Another local company, Origami Therapeutics, Inc., is supporting the event. It also seeks to develop an HD treatment. It was founded by Beth Hoffman, Ph.D., the former president of the San Diego chapter of the Huntington’s Disease Society of America.

Other USD co-sponsors include the International Center, the Enhanced Student Faculty Interaction Fund, the Humanities Center, the above-mentioned Department of History, the Program in Latin American Studies, and the Department of Communication Studies. The College of Arts and Sciences also has lent its support.

Charles Sabine dancing at the Vatican with #HDdennomore participants (photo by #HDdennomore)

‘All of us standing together’

On January 10, I had a long lunch with George Essig, a well-connected veteran radio ad salesman and former HDSA-San Diego president. Essig’s extended family is affected by HD. As I wrote in a 2014 article, Essig “epitomizes the dedication of the unaffected relative.” (Click here to read more.)

In discussing the screening, we noted that it will be a unique event for the San Diego HD community and its supporters. Over the years, most events – such as galas, marathons, and walks – have focused on raising funds and awareness.

Echoing Sabine, I stressed that this event would be a celebration.

We brainstormed on the meaning of “celebration” for the local HD community – and for the many donors Essig has brought into the cause.

Their support had helped HD “become hidden no more,” he said. 

The screening also will be about “the evolution of the cause,” he added. 

With that in mind, Essig said he would tell supporters that he would be “remiss not to invite you to this celebration.”

The Dancing at the Vatican screening will also celebrate the progress in research, which has advanced thanks to the donors and broad collaboration in the HD community, he noted.

Essig summed it up: the Dancing at the Vatican event will be “all of us standing together and saying: I helped bring a cure to an incurable disease, even if it’s just $10 that I gave.”

(Disclosure: I hold a symbolic amount of Ionis shares.)

‘Navigating’ the Huntington’s disease community towards crucial clinical trials

As scientists and drug companies expand the array of potential treatments for Huntington’s disease, the Huntington Study Group (HSG), the world’s largest HD clinical research network, is redoubling its efforts to educate the HD community for current and upcoming clinical trials and train the necessary medical personnel.

A record 700-plus participants focused on these themes at the 26th annual HSG Meeting, titled “HSG 2019: Navigating HD,” November 7-9 at the Hyatt Regency hotel in Sacramento, CA. (Attendance at the HSG 2017 and 2018 meetings was over 600.)

Clinical trials are crucial for demonstrating drug safety and efficacy. The number of HD trials has increased in recent years, bringing hope for better treatment of the devastating symptoms and perhaps even an attack on the root causes. Key trials in progress include GENERATION HD1, run by Roche, and SIGNAL, administered by the HSG and Vaccinex.

“Figuring out how these trials are going to work, what they’re aiming to do, and what an individual patient or family should do to get involved or not get involved has become complicated, to some extent,” Andrew Feigin, M.D., the HSG chair and a professor of neurology at New York University Langone Health, told me in a November 6 interview. “That’s my interpretation of the ‘navigating HD.’ We’re trying to get at some of these novel therapies and clarify where they’re headed, where they stand, how the HSG can get more involved, and figuring out where people can go for the cutting-edge therapies for Huntington’s disease.”

In the conference-opening “HSG State of the Union” presentation by HSG leaders and staff, executive director Shari Kinel, J.D., reported that the event involved 15 countries, 23 companies, 9 advocacy groups, 17 sponsors, and 15 exhibitors. The sponsors included Roche’s American subsidiary Genentech and Vaccinex.

“This incredible showing […] is a sign that the HSG has more partners, more colleagues, more friends than ever who are engaged, dedicated, and committed to seeking treatments that make a difference for those impacted by Huntington’s disease,” Kinel told the audience.

Dr. Feigin affirmed that in the past year, the HSG has doubled its paid staff from four to eight, plus one part-timer, although he declined to reveal the organization’s annual budget. Headquartered in Rochester, NY, the HSG is mainly funded by firms like Vaccinex that it partners with on clinical trials, he explained. Sponsors cover the cost of the annual meeting.

The audience watches a presentation by Dr. Arthur Combs at the "HD Innovators Forum" at the 26th annual HSG Meeting (photo by Gene Veritas, aka Kenneth P. Serbin)

A full-service organization

The HSG was founded in 1993, the year of the discovery of the huntingtin gene. Dr. Feigin described the nonprofit organization as a “full-service” contract research organization that can carry out all aspects of an HD clinical trial.

In her speech, Kinel stated that the HSG member network includes 801 investigators (researchers), trial coordinators, scientists, and HD experts. Around the globe, the organization has credentialed 127 sites for HD trials, and HSG members have worked with more than 21,000 HD-affected individuals, she said.

The HSG also developed the Unified Huntington’s Disease Rating Scale (UHDRS), the primary assessment tool in HD clinical trials. It consists of tests of a person’s movements, cognition, behavior, independence, and functional capacity.

The “HSG State of the Union” presentation outlined the HSG’s mission, accomplishments, clinical trials, educational activities, efforts to improve patient care, and plans for the future.

You can watch the presentation in the video below. Click here for my video album of the event, which included a variety of presentations on patient care, clinical trial techniques and measurements, new scientific findings, and innovations in drug and clinical trial development.

Seeking a better drug to treat chorea

Prior to the main conference, the HSG held organizational meetings for KINECT-HD, a Phase 3 clinical trial by the HSG and San Diego-based drug developer Neurocrine Biosciences to test the efficacy of valbenazine to treat chorea, the involuntary movements typical in HD. 

The HSG ran the successful clinical trials of two other drugs for chorea, Xenazine and Austedo, the only HD-specific medicines to receive approval from the U.S. Food and Drug Administration (FDA). On November 14, it issued a press release announcing the start of the 18-week trial, which seeks to enroll HD-affected individuals with chorea at 55 sites in the U.S. and Canada.

In 2017, valbenazine was approved by the FDA with the name Ingrezza for the treatment of tardive dyskinesia, an irreversible involuntary movement disorder. This status allowed Neurocrine and the HSG to take it directly into a Phase 3 trial for HD.

Like Xenazine and Austedo, valbenazine is a VMAT2 inhibitor. Xenazine requires three daily doses, and Austedo two. 

“The upside thing of valbenazine is that it’s a drug that can be dosed once daily,” said Dietrich Haubenberger, M.D., the Neurocrine medical director, in a presentation forming part of the “HD Research Round-Up” at the close of the scientific sessions on November 8.

Wearable sensors and the search for biomarkers

In the quest for HD treatments, researchers hunt for new biomarkers, that is, signs of the disease and the effect of remedies. Biomarkers are especially critical in brain-related diseases, because doctors cannot do biopsies on the organ.

With a key innovation, KINECT-HD will also look for biomarkers. It will be the HSG’s first trial in which participants use wearable sensors – for continuous monitoring of their movements and other biological functions, even at home. Researchers hope this more detailed monitoring will provide both a better understanding of chorea and valbenazine’s impact on it.

Called BioStamp nPoint, the sensors were designed by MC10, Inc., and cleared for use by the FDA. MC10 is based in Lexington, MA.

MC10 chief medical officer Arthur Combs, M.D., described the system at the conference’s “HD Innovators Forum.”

“It weighs less than eight grams [0.28 oz.],” Dr. Combs said, explaining that the sensor can be placed anywhere on the body and worn even during showers and swimming. “It’s like putting on a Band-Aid.”

MC10 developed 44 algorithms for the system to help measure trial participants’ data. In addition to chorea, BioStamp nPoint will help investigators observe individuals’ gait, heart rate, sleep, posture, and other bodily functions, Dr. Combs added.

In one previous study, “patients with symptomatic Huntington’s disease spent 50 percent of their day” lying down, he explained. That may be a response to exhaustion or the risk of falling, he said. Thus, the BioStamp nPoint system could help determine whether lying down is a “marker” for the disease, and whether less time at rest is a sign of drug efficacy, he said. It also accounts for the uniqueness of each patients, he added.

To obtain continuous data in GENERATION HD1, Roche developed an HD Digital Monitoring Platform, with participants wearing a smartwatch and using a smartphone.

You can watch Dr. Combs’ presentation in the video below.

The latest clinical trial news

In addition to Neurocrine, other firms reported on their clinical trials during the “HD Research Round-Up”: Voyager Therapeutics, uniQure, Wave Life Sciences, Vaccinex, and Roche.

The Roche GENERATION HD1 update of the company’s historic Phase 3 clinical trial of the drug RG6042 was one of the most anticipated. A gene-silencing drug, RG6042 is aimed at the roots of HD and caused a stunning improvement in the health of HD-affected mice. On October 14, Roche announced that it was expanding the number of trial participants from 660 to 801 and adding China to the nearly 20 countries in the study.

The announcement noted that recruitment in the U.S. had “exceeded expectations” and was now complete. Expanding the number of volunteers and adding China will allow for more abundant data and the study of a more diverse population, Roche said.

Enrollment for the Roche HD program has been “absolutely electric,” with over 800 individuals already in 2019 in GENERATION HD1 and related HD studies, said Scott Schobel, M.D., M.S., Roche’s associate group medical director and clinical science leader for RG6042 (click here to watch Dr. Schobel’s presentation). If the trial is successful, Roche will apply for drug approval from the FDA and regulatory agencies in other countries.

On November 9, HSG held a “Family Day” for the HD community, with presentations by advocates like me, presentations by scientists, and an update on GENERATION HD1.

In upcoming articles, I will report on Family Day and more of the scientific and clinical developments discussed at the meeting.

Disclosure: my travel expenses were covered by the HSG and the Department of History of the University of San Diego.