A New Start in Latin America

This site is just the first; by the end of 2014, Enroll-HD should include 19 sites in eight nations across the Spanish- and Portuguese-speaking nations of the Americas, eventually involving more than 3,000 people affected by HD, their family members and caregivers. With this launch, Enroll-HD now becomes a truly global effort, soon to include participants in five of the world’s six inhabited continents.

Launching a multinational study of HD is new for the region, which has not previously had a network of clinical research sites as existed in North America and Europe. For Latin Americans affected by HD, the study offers a route to participation in international advances in research and clinical care. When new therapies are tested in clinical trials in coming years, study participants are likely to be among the first to have the choice to join. “We want to have the same chances for our patients as the Americans or Europeans,” says Micheli.  “If we want to be involved, we have to be ready.”

A wave of sites

Enroll-HD will expand gradually across Latin America during the course of this year and next. Argentina plans two additional sites, both in Buenos Aires. Chile and Peru are expected to be next to join, followed by Brazil before the end of 2013, then Ecuador and Venezuela. Locations in Colombia and Mexico will be added in the following year.

Brazil alone, with its population of nearly 200 million, aims to recruit as many people to the study as the rest of the Latin American region combined, says Francisco Cardoso, MD, professor of neurology at the Federal University of Minas Gerais of Belo Horizonte, Brazil. At his clinic, which will be an Enroll-HD site, people welcome the idea.  There is enthusiasm that, finally, something coordinated is being done in the quest for a cure for HD,” he says. “In the past, by the time these patients received a diagnosis they felt pretty depressed by the realization that they could find almost no information on the condition itself, and even less about investigation.” By instituting a clinical network, improving communication, and tracking how well people do in various centers, Enroll-HD also aims to help improve the quality of care across Latin America.

The sites for Enroll-HD were established through the Red Latinoamericana de Huntington (RLAH), a multinational network founded in 2010 to link specialists across Latin America to promote research and share expertise. RLAH now includes about 30 clinician-researchers and has additional contacts with about 250 other interested specialists in neurology, geneticsand psychiatry. Micheli is currently president of RLAH, and Cardoso is vice president.

In a sense this is a rebirth of HD research in Latin America. It was because of the families in villages around Lake Maracaibo in Venezuela that the gene for the disease was first identified 20 years ago. That part of Venezuela has the highest known concentration of people with HD in the world, and clinical samples from the families there made it possible to find the gene mutation that causes HD.

Many nations, many rules

For a global study like Enroll-HD, data has to be collected and organized in exactly the same way at all sites so that information gathered on one continent can be combined with and compared to that from participants on the other side of the world.

Getting a multinational clinical study off the ground also means meeting all the laws and regulations of each country, which is not trivial. Each nation’s regulatory authorities and various bioethics committees must be satisfied that Enroll-HD is appropriately designed and follows strict ethical guidelines. In some countries the governmental ministry of health must directly approve the study protocol. Licenses must be arranged in order to export blood to the central biorepository lab in Milan, Italy, and to import clinical supplies to researchers. Fulfilling these obligations requires between six and nine months per nation.

Argentina also has a special requirement: review by the government’s National Directorate for the Protection of Personal Data. In the past, some clinical studies have recruited rural indigenous people without adequate privacy protection and without ensuring that participants truly understood what they were agreeing to. In Argentina, the protection of personal data is now a constitutional right. A law passed in 2000 also protects confidentiality and requires that people enrolling in a clinical study receive information appropriate to their level of education.

Despite these tough regulations, the directorate officially approved Enroll-HD at the end of 2012 without any objections. “We’re proud of that,” says Claudia Perandones, MD, PhD, a clinical geneticist with the National Administration of Laboratories and Institutes of Health in Argentina who is also the scientific director of RLAH, and sits on the scientific and publication review committee for Enroll-HD.

Launching Enroll-HD in Latin America involves some practical hurdles. Argentina is dealing with a prolonged economic and social crisis. Sites like the one in Buenos Aires draw many different types of people with different needs, from wealthy city dwellers to poor rural people with limited education. But Latin America also offers unique opportunities. Echoing Cardoso’s sense of optimism, Perandones points out that at a scientific conference about movement disorders last fall in central Argentina some people traveled all the way from Venezuela, more than 4,000 km (2,500 miles) away, to hear about Enroll-HD. “We have a lot of excitement here about the start of Enroll-HD,” she says. “It means that there’s hope.”
This story was originally published in the February 2013 issue of Enroll!