ARTICLE IN BRIEF
Researchers discuss the impact that Pfizer's decision to cut funding for Alzheimer's and Parkinson's diseases research can have on current and future investigators.
Pfizer, one of the largest pharmaceutical companies in the world, announced in January that it would end all funding for research in Parkinson's disease and Alzheimer's disease, taking researchers by surprise. In interviews with Neurology Today, neurologists who participate in large study groups in both disorders said they were worried about a domino effect in the pharmaceutical industry, and what message it sent to patients and their families. But they also said the field would adjust and research would move forward.
“I think this is discouraging to the Parkinson's community, because it's not viewed as a cut-and-dry business decision; it's seen as a statement that research into this disease is worthless and hopeless,” said Allison Willis, MD, MS, an assistant professor of neurology and epidemiology at the University of Pennsylvania.
“What I tell my patients is that I don't want you to give up being hopeful and we need to think of what we can learn from the data that went into that decision.”
Pfizer made the announcement on January 7. In a statement, Mikael Dolsten, MD, PhD, president of Pfizer Worldwide Research and Development, wrote that the decision was based on science, not cost, and that the research efforts “were simply not making the progress necessary to translate into truly transformational therapies for patients.”
The company ended all early development programs in neuroscience, including pre-clinical, phase 1 and phase 2 studies. Neda Wadhwa, a spokeswoman for the company said in an email to Neurology Today that the company is identifying opportunities to out-license or co-fund neuroscience programs.
“This was a difficult decision and it is not lost on us that there is a tremendous need for new therapies in this therapeutic area,” Wadhwa wrote. “We recognize the important and ongoing need for research in this area. This is why we are creating a dedicated venture fund that will seek out investment opportunities in biotech companies that are conducting promising neuroscience research.”
Pfizer will continue to commit resources to late-stage development programs for Lyrica (pregabalin) for diabetic nerve pain, and for tanezumab, a drug being tested in osteoarthritis pain, as well as back and cancer pain. It also said it would continue funding research on drugs for rare neuromuscular and neurological diseases.
IMPACT ON RESEARCH PIPELINE
Neurologists told Neurology Today they were concerned that seeing large pharmaceutical companies withdraw from neuroscience trials would have a detrimental effect on new researchers.
Jennifer G. Goldman, MD, MS, FAAN, associate professor of neurological sciences, section of Parkinson's disease and movement disorders at the Rush University Medical Center, said Pfizer has not had success with Alzheimer's drugs, pointing out the 2012 failed phase 3 trial of bapineuzumab, which attacked the amyloid-beta protein. (Pfizer partnered with Johnson & Johnson and Elan Pharmaceuticals in that trial.)
Dr. Goldman said that there have been several compounds that have failed in clinical trials, dashing high hopes for the five million Americans with Alzheimer's and their loved ones. She said it's a research area that's “on the edge of making history” for biomarkers and understanding mechanisms but there is a great need to translate these into successful clinical therapeutics.
“This development is very disheartening and sobering,” Dr. Goldman said. “It may mean that people will need to go into other avenues to do their research and clinical trials for Alzheimer's and Parkinson's, and look to where there is more development and funding.”
Michael W. Weiner, MD, is the principal investigator of the Alzheimer's Disease Neuroimaging Initiative (ADNI) at the University of California, San Francisco, which has received $200 million in National Institutes of Health funding since its inception in 2004. He said Pfizer's move would discourage some companies from investing into Alzheimer's discoveries, but that there is a “huge role” for discovery in university environments and small companies that don't have the resources for a phase 2 or phase 3 trial, which costs millions of dollars to run.
“It's a high-risk, high-gain situation,” Dr. Weiner said. “There's going to be a huge financial reward for whomever comes up with a successful treatment, but it's going to take some clever science and good business in deciding which way to go.”
There's a bit of luck here too in the way the whole drug discovery system works in the world — we're just seeing this play out on this small field.”
IMPACT ON OTHER FUNDERS
James Beck, PhD, the Parkinson's Foundation chief scientific officer, said they knew Pfizer was struggling in the neuroscience area but that the complete exit was unprecedented.
‘What really concerns us is what other companies are going to be doing in light of this decision,” Dr. Beck said.
In an interview published January 16 in The Financial Times, John LaMattina, PhD, a former head of research and development at Pfizer, shared the same worries as Dr. Beck.
“I think others will pull out,” said Mr. LaMattina, adding that the US government agency for medical research, the National Institutes of Health, might have to step in to plug the funding gap as private companies vacate the space.
Dr. Beck said he was concerned about the exorbitant cost to run clinical trials for Alzheimer's and Parkinson's disease, and that small companies, which often start the research and license the compounds, would not be able to run a trial and market a product.
“What I do know is that this underscores the need for federal funding and the need of our foundation and others to ensure that we have that kind of basic research funding,” Dr. Beck said. He noted that the Foundation just announced the enrollment of the 10,000th Parkinson's patient in the largest clinical study of the disease. The study, launched in 2009, includes 29 clinics in five countries.
A SHIFT IN FOCUS
But John Nutt, MD, FAAN, co-founder and director emeritus of the Oregon Health and Science Parkinson Center and Movement Disorders Program, said he saw the announcement as a shift in the focus of the research. There are good ideas about Parkinson's disease and Alzheimer's disease, he said, but the focus is changing from targeting amyloid plaque to thinking more about the role of tau proteins that may open up new avenues of funding and discovery.
“Neuroscience is not an area that's scientifically inactive right now, it's still very active and I think that will keep scientists involved,” he said. “Pfizer's move is unfortunate, but maybe they simply didn't have enough good ideas to convince their senior management that they had something worthwhile going on.”
He pointed to the 2010 Parkinson's Progression Markers Initiative supported by the Michael J. Fox Foundation, which aims to find biomarkers for the disease.
“I think a lot of work is taking place in Parkinson's and dementia, and within 10 years, we're going to see therapies on the market or coming on the market that will alter the progression of the disease. I think a lot of scientists believe that, and I don't think Pfizer's departure from the field is going to turn down that enthusiasm.”
Kirk R. Daffner, MD, director of the Center for Brain/Mind Medicine and chief of the division of cognitive and behavioral neurology at Brigham and Women's Hospital in Boston, and the J. David and Virginia Wimberly Professor of Neurology at Harvard Medical School, said pharmaceutical companies are beholden to stockholders, and that considering the recent track record and cost of running clinical trials, he was not surprised by Pfizer's actions. Still, he said, the decision was extremely disappointing, given the enormous burden of Alzheimer's disease.
But he still felt “cautiously optimistic” that researchers were making progress and, more than that, have had an opportunity to learn from failed trials.
He thought patients and their families were more likely to ask about the possibility of being treated with a “promising” experimental drug they had read about than focus on Pfizer's lack of interest, and he himself was excited about Alzheimer's prevention trials for at-risk adults with biomarker evidence of the disease. These prevention trials are taking place in multiple centers, he noted, and they are not only focused on looking at biomarkers, but using biomarkers to select participants in intervention trials.
“People have to be upbeat in our business,” he said. “It's important to maintain hope and find ways to mitigate disappointment because we have to keep moving.”