DALE SCHENK is a rock star of science. He is the man behind blockbuster multiple sclerosis drug Tysabri, the inventor of a vaccination approach to Alzheimer’s and has a treatment for Parkinson’s in development.
“We’re brought up thinking scientists wear white coats in a lab and are not real people,” says Schenk, chief scientific officer of Irish biotech company Elan. “But we’re ordinary people just like everybody else.”
Schenk was admitted to the “Rock Stars of Science” hall of fame in 2009, a US campaign to make science hip. Its motto is: refuse to accept ‘no cure’ as an answer. Through the treatments he is developing for some of the world’s most intractable diseases, Schenk’s research has rocked patient’s lives and Elan’s share price alike.
A biologist with a PhD in physiology and pharmacology, Schenk was director of neurobiology firm Athena Neurosciences when it was acquired by Elan in 1998 under former chief executive Donal Geaney. Athena’s research has fuelled Elan’s product pipeline since. But taking a drug from lab to bedside is not simple.
Schenk has been working on a treatment for Alzheimer’s since the 1980s, but the first double-blind, placebo controlled studies of Bapineuzumab on several thousand volunteers is not due to be completed until next year. It’s hoped the data will support the approval of the drug for treating patients with mild to moderate forms of the disease.
Did he think it would take this long?
“Probably not,” he says. “In every step of our progress, nothing’s been there when we got there. We’ve had to create each step of the field along the way. We’ve had to move the field and help the field mature through this process.”
Schenk’s research team was the first to develop a mouse model for the disease, estimated to affect up to 35 million people worldwide. The progressive neurological condition destroys nerve cells, making it difficult to remember, reason and use language.
“With Alzheimer’s, you get something in the brain called plaques, made up of small protein fragments that self-associate and form this glue-like gunk all over the brain,” he explains.
Alongside Bapineuzumab, which he calls Bapi, Elan is also developing a vaccination for the disease, something he says was initially seen as a wild card.
“Having developed the mouse model, we listed 30 different experiments we wanted to do. This vaccination was one, but we all agreed it had such low likelihood of working that we’d put it as number 30, the last one.”
Mice with the disease were stimulated to generate antibodies against the proteins that cause the plaque. The hope was that some of the antibodies would get into the brain to clear out the plaque. They did.
“It was so dramatic, the animals that had been vaccinated had nothing in their brains, so we assumed we’d mixed them up in the cages and were looking at the wrong animals,” says Schenk. “What happens is that when an antibody binds to a plaque, your immune system knows that’s a bad thing [and to] go in and remove it. These antibodies are acting like flags to tag the plaques and cause clearance.”
Both Bapi and the vaccination use antibodies to clear the plaque. In Bapi, the antibodies are given but in the vaccination, the body is stimulated to produce them.
Now in phase three clinical trials, Bapi is closest to market, with US regulatory approval likely to be sought in the next three years.
Schenk credits the team of 200 scientists who worked with him with the success. “It’s easy to think of these things – it’s hard to pull them off,” he says.
Although created by Elan, a series of labyrinthine deals sees ownership of some of its Alzheimer’s work somewhat splintered. In September 2009, Janssen Alzheimer Immunotherapy, a subsidiary of Johnson Johnson, acquired all of the assets and rights of Elan related to its Alzheimer’s Immunotherapy Program (AIP), rights Elan had shared with Wyeth.
Elan however holds a 49.9 per cent equity interest in Janssen Alzheimer Immunotherapy, and will be entitled to a share of the profits and certain royalty payments upon the commercialisation of AIP products.
Janssen Alzheimer Immunotherapy is continuing Alzheimer’s activities with Pfizer, which acquired Wyeth in 2009. The move is consistent with a trend of bigger pharma companies licensing and partnering with smaller biotechs to access their discoveries.
So does Elan consider itself in the big pharma league? Schenk prefers to see the company as a growing biotech firm.
Having sold its drug formulation and manufacturing unit earlier this year, Elan will now focus its efforts on creating new products.
“We aim to remain infrastructure light and will focus on our core competence of neuroscience drug discovery and development.”
Schenk says positive results from animal models are the biggest hurdle to overcome in pharmaceutical innovation. “That’s the biggest Eureka moment because we have a lot of ideas. Most of the ideas don’t work and to actually work well in an animal model is very, very, very rare,” he says.
Translating a discovery into a drug involves expanding the cast to regulatory, safety and manufacturing experts to produce something that can be tested in human clinical trials.
While such trials can help to indicate whether or not the treatment works, nothing is ever crystal clear.
Elan’s multiple sclerosis drug Tysabri, which it co-markets with US-based Biogen, is a case in point. Launched in 2004, it was taken off the market in 2005 when three patients, carriers of a common virus called the JC virus, developed a rare brain infection called PML. It was cleared to return to market in 2006 and now about 60,000 MS sufferers are taking Tysabri. Some 124 carriers of the virus have gone on to develop PML, with 23 dying from the complication.
Schenk has just developed a blood test for the JC virus which, he says, will help those with multiple sclerosis to judge their risk of having an adverse reaction to Tysabri.
For many, however, the benefits outweigh the risks. “Since the test has been out there, of those who have taken it and test positive , most stay on Tysabri,” says Schenk. “They have said ‘I understand the risk but the therapy is so important to me, I want to take that risk’.”
