making bread safe for celiacs - immusant
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Making bread safe for celiacsBy Alix Stuart | G L O BE C O RRES PO N DEN T MA RC H 1 1 , 2 0 1 3
JIM DA V IS/GLOBE STA FF
Dr. Alessio Fasano, Victoria Kennedy, Dr. Ronald Kleinman, and Dr. Peter Slavin have their
picture taken by a Mass. General staff photographer at the Museum of Medical History and
Innovation.
When Leslie Williams, a former pharmaceutical executive, agreed to meet a visiting
professor from Australia in Boston for a lecture, she thought it would be a routine lunch
in her role as a business mentor.
But the meeting, three years ago at the Boston Cambridge Marriott, turned into an
intense five-hour discussion as Dr. Robert Anderson explained how his research into
celiac disease promised to render the destructive disorder obsolete.
An autoimmune disease triggered by gluten proteins in wheat, barley, and rye, celiac
disease affects some 3 million Americans. Untreated, it can destroy digestive tract tissue
and can lead to anemia, osteoporosis, infertility, neurological dysfunction, or even
Business
Dr. Robert Anderson’s research is
zeroing in on a potential vaccine
against celiac disease.
cancer.
Currently, the only solution is to avoid gluten altogether. That means not eating
standard versions of bread, pasta, and pizza, or anything else that contains even traces
of wheat, including soy sauce and some candy, such as Twizzlers.
But as Anderson explained that afternoon to
Williams, his research was zeroing in on a vaccine
to cure celiac disease.
The science “struck me as quite special and
possibly game-changing,” Williams recalled.
She agreed to work with Anderson, and in short
order Williams lined up seed capital from an angel
investor and then went to Australia to unravel
legal agreements around Anderson’s research and his company. Within the year,
ImmusanT was formed, with Williams as chief executive and Anderson as chief scientific
officer. By its first anniversary, the firm had $20 million in venture funding.
ImmusanT is headquartered in the biotech boomtown of Kendall Square in Cambridge
and is conducting clinical trials for its vaccine, NexVax2, under “fast-track” designation
from the Food and Drug Administration for diseases for which no comparable therapies
exist.
“If it works, you’ll see the entire paradigm of treatment for celiac changed,” said Sundar
Kodiyalam, managing director for the venture investor Vatera Healthcare and an
ImmusanT board member. His firm was so enamored of the science that it invested
before the company had persuasive clinical data.
Beyond ImmusanT, Boston has become a locus for research into celiac disease.
Massachusetts General Hospital scored a coup when it recently convinced a leading
researcher, Dr. Alessio Fasano, to head its new celiac treatment and research center.
“Our mission is to make life normal for people with celiac disease,” Fasano said at a
ceremony marking the opening of the Mass. General center in February.
With similar research units at Beth Israel Deaconess Medical Center and Children’s
Hospital Boston, the city now has “a critical mass of expertise” in celiac disease, said Dr.
Ronald Kleinman, physician in chief of Mass. General’s pediatric unit.
“I’m not sure that I see miracles happening” with the research underway now, said Lee
Graham, chairwoman of Healthy Villi, a 900-member support group for celiac sufferers
in New England. “But the gathering that’s happening in Boston is terrific, and
tremendously encouraging to us.”
Formerly at the University of Maryland, Fasano in 2003 published a landmark analysis
in which he determined that celiac disease affects many more people than previously
thought: about 1 out of 100 people. Up to that point, the scientific wisdom was that
celiac was relatively rare, and that a gluten-free diet worked as a sufficient “cure.”
But Fasano and others have since shown that some patients who avoid gluten continue
to suffer gastric distress, leading to the conclusion that diet alone is not enough.
Not surprisingly, with the market for gluten-free foods at $4.2 billion, ImmunsanT has
some company in the race for a solution.
One rival is Alba Therapeutics, a Maryland company that Fasano helped start in 2005.
(He is no longer involved in the company, though he owns some stock.) The other is
Alvine Pharmaceuticals, of San Carlos, Calif., spawned from research at Stanford
University.
Both companies are working on pill-based therapies to counteract the unintentional
consumption of small amounts of gluten; complements to the gluten-free diet rather
than replacements. And both are preparing for Phase 2b clinical trials to determine if
their medicines work, and at what doses.
Alba’s compound targets zonulin, a protein that is believed to contribute to “leaks” in the
gut that allow gluten to infiltrate the digestive system. Cephalon Inc., now owned by
Teva Pharmaceuticals, has a $7 million option to buy Alba if its drug proves effective.
Alvine’s therapy involves an enzyme that decomposes gluten into harmless particles
before it reaches the gut. Patients in its most recent trials who consumed gluten for six
weeks while taking the Alvine compound showed little or no damage to their intestines,
with some even showing improved conditions.
ImmusanT’s drug is at a much earlier stage of development. At the time he lunched
with Williams, Anderson was a professor at the Walter and Eliza Hall Institute in
Melbourne, Australia. He had a start-up, Nexpep Pty Ltd., and was in need of funding to
continue developing a celiac vaccine.
Anderson said he was struck by how many patients struggled with a gluten-free diet,
which can be less healthy than typical diets.
“Having a treatment that would allow full recovery and return to normal diet would be
life-changing for patients, and may motivate more patients to be checked for celiac
disease,” he said.
Williams, meanwhile, had worked at Merck, GlaxoSmithKline, and other drug makers
and was chief executive of Ventaira Pharmaceuticals when it was sold to Battelle in
2007.
In his research, Anderson had latched on to a key catalyst: the three gluten peptides
that are believed to be at root of the reaction patients suffer from gluten. NexVax2
essentially tries to reeducate the immune system to tolerate those peptides. Initial
study results indicate that ImmunsanT has identified the correct peptides, and that
Nexvax2 is safe to take — two important steps.
Still, there are many questions. For one thing, ImmunsanT’s early volunteers
maintained gluten-free diets during the study, so its not clear how well the vaccine
works in the presence of gluten. And it will work only for an as-yet undefined subset of
celiac sufferers.
Even so, Fasano, who has no connection to the company, calls the concept behind the
ImmunsanT’s vaccine “the holy grail” that would allow patients to eat regular bread,
pasta, and other gluten-rich foods.
Though ImmusanT and the other firms are small, Williams and others in the field said
the pharmaceutical industry has a keen interest in their research.
Moreover, because celiac disease is currently the most well understood autoimmune
disorder, many scientists believe the research could serve as a springboard to drugs for
larger markets.
“It’s not just about curing celiac,” Fasano said. “It’s about treating MS and diabetes, and
all these other autoimmune conditions, and that is where industry really takes an
interest.”
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