Find updates on the work of our researchers here, as well as news about recent advances in Alzheimer's science, funding and awareness.

Tanzi and Armour participants in White House Sponsored Meeting of the Status of Alzheimer’s Research

The status of research in the United States to find a cure for Alzheimer’s disease was the focus of discussion at a White House sponsored event on World Alzheimer’s Day, Tuesday, September 21, 2010.

Cure Alzheimer’s Fund Research Consortium Chair Dr. Rudolph Tanzi and Tim Armour, President of the Cure Alzheimer’s Fund, participated on a scientific panel at the White House event before a select audience of White House senior staff policymakers, leading scientists, advocates and others including Jeff Morby, Chairman and co-founder of Cure Alzheimer’s Fund, and Melody Barnes, Director, White House Domestic Policy Council. The topics covered included the current status of biomarker identification for the disease, current thinking about prevention, the strength of the drug pipeline for Alzheimer’s and possible policy initiatives to accelerate progress toward a cure.

Panelists agreed that more funding from both the public and private sectors needs to be invested in finding a cure and better treatments; and more aggressive efforts at creating public-private partnerships to provide focus for research efforts is crucial.

The clear message to come from the meeting is that Alzheimer’s is having a devastating impact not only on the growing number of patients and their families but on the national budget as well. With the Federal government spending $172 Billion on care for Alzheimer’s patients through Medicare and Medicaid in 2010, and the National Institutes of Health able to invest only $470 million in basic research, the “cure” will be a long time in coming if there is not a rapid change in national priorities.

All agreed that we cannot afford to wait, and the development of effective therapies to prevent or stop Alzheimer’s has to be a national priority, backed by a clear strategy and resources to implement it.


Hobbling Science and Scientists

William Sahlman, the senior associate dean for external relations at Harvard Business School, wrote an excellent op-ed in the Boston Globe yesterday. We couldn’t agree more with his assessment of the dreadful funding environment for scientific research.  His piece helps us understand the difficulties of making productive progress in disease research by taking a look at how research labs work. While this piece was inspired by the recent stem-cell research issue, it is applicable to most research including Alzheimer's.

Read the op-ed in the Boston Globe>

The new γ-secretase modulators

Esther Landhuis covers the topic thoroughly in a good article on Alzforum.

“Despite recent setbacks on the clinical front, the hunt for small molecules that can cleanly tweak γ-secretase to slow Alzheimer disease seems to be alive and well” writes Esther Landhuis on Alzforum about new γ-secretase modulators.

The research published in Sept. 9 Neuron is on the success of gamma secretase modulator (GSM) drugs in Alzheimer’s disease mice from Cure Alzheimer’s researchers Steve Wagner and Rudy Tanzi.

Tanzi writes that the GSMs “could be used like statins are used today to prevent heart disease. If there was pre-symptomatic evidence that amyloid levels were too high in a patient’s brain, a GSM might be taken to lower relevant peptide levels and reduce AD risk. You don’t want to knock out these peptides. They have a purpose. You just want to dial them back to safe levels.”

The Alzforum piece explains the details behind this exciting work.

Read the article: New γ-Secretase Modulators Reduce Aβ42, Avoid Notch

AARP Features Cure Alzheimer's Fund Discovery

Elizabeth Agnvall writes in the September AARP Bulletin: “The controversial new theory gaining traction in the scientific community is that in Alzheimer’s disease the brain is not destroyed by sticky plaques – long held to be the culprit – but by free floating clumps of protein.  .  .(called oligomers).

“Plaques are no longer where the action is” says Sam Gandy . . .

Gandy’s work builds on several years of research that has been moving toward this new theory. And if the theory is correct, the drugs that target plaques – as many of the most promising medications have done in the past few years  - may not help those who have the disease”

This important and potentially paradigm shifting discovery is the direct work of Cure Alzheimer’s Fund’s Oligomer Collaborative. The article quotes CAF Research Consortium Chairman Rudy Tanzi and Research Consortium members Sam Gandy and Sam Sisodia.

Read the full story in the AARP Bulletin>

New publications by Cure Alzheimer’s Fund Researchers

Two recent publications (in Neuron and Cell) detail important work being done by CAF researchers paralleling CAF-supported research.

The research published today in Neuron is on the success of gamma secretase modulator (GSM) drugs in Alzheimer’s disease mice from Steve Wagner and Rudy Tanzi.

Tanzi writes that the GSMs “could be used like statins are used today to prevent heart disease. If there was pre-symptomatic evidence that amyloid levels were too high in a patient’s brain, a GSM might be taken to lower relevant peptide levels and reduce AD risk. You don’t want to knock out these peptides. They have a purpose. You just want to dial them back to safe levels.”

Wagner says “We’ve shown that a compound can modulate enzyme activity without completely shutting down the enzyme. We think we’ve opened up a new area of drug discovery for pharmaceutical companies and universities. We hope they will pursue some of these compounds to see if they can be used in people.”

Cure Alzheimer’s Fund is supporting the development of novel GSM's, independent of those presented in the paper.

The Cell paper published last week describes the normal role of  the beta amyloid precursor protein (APP) and strongly supports the Prana Biotechnology drug (PBT2) approach that was first developed in Rudy Tanzi’s lab. Tanzi is a Prana co-founder and is co-author on the paper.

Sam Gandy, M.D Ph.D, Mount Sinai School of Medicine, New York, described this report, as “providing a major advance in deciphering the underlying causes of Alzheimer’s disease while at the same time pointing us toward a new and exciting strategy for treating or preventing the disease with a drug such as PBT2, which affects brain metals”.

The evidence published in Cell shows that the source of beta-amyloid, APP, plays a hitherto unknown critical role in exporting iron out of neurons.  If APP fails to carry out this role, iron builds up in the neurons contributing to oxidative stress, neurofibrillary tangle formation and ultimately neuronal cell death.

Read more on the Neuron publication>

Read the paper in Neuron>

Read the paper in Cell>

With Our Venture Capitalist Approach, Cure Alzheimer’s Fund “Dares to Be Great”

The website Xconomy Boston has written a profile on one of our founders, Henry McCance, outlining our entrepreneurial approach to funding innovative researchers. Xconomy looks at how Cure Alzheimer’s Fund translated Henry’s expertise as a venture capitalist to the realm of medical research, which was sorely lacking a bold source of funding.

“McCance recalls researchers saying that the organizations that traditionally dole out the grants are so risk-averse that they don’t want to fund anything that has the slightest potential of failure. ‘We are forced to submit proposals which we almost already know the answer to,’ was their lament, McCance says. “Research that does get funded is akin to 1-yard plunge. There is no organization that is willing to throw us a 30 yard pass down the field.’”

Xconomy explains the ways McCance and other Cure Alzheimer’s Fund founders developed an alternative approach. They targeted visionaries in the field, brought in high-quality leadership, and kept a frugal culture to ensure funding going straight to research. The result – an unprecedented approach to push the boundaries of Alzheimer’s research.

The article, a great introduction to our entrepreneurial approach, can be read in full here.

Do you have any questions on our funding strategy? E-mail us your questions at

Our Researchers in the News

Nature News article highlights Cure Alzheimer’s Fund researchers Stephen Strittmatter, Robert Malinow, and Sam Sisodia discussing conflicting results of Alzheimer's research and how Cure Alzheimer’s consortium hopes to unite researchers and move more quickly toward a cure.

Read the article, Key Alzheimer's findings questioned>

What does the Lilly drug failure tell us?

The recent Lilly Alzheimer’s drug failure, while disappointing, brings up critical issues around what is next in the “drug” pipeline and the validity of the Amyloid hypothesis.  An article on yesterday covers the story well.  Lilly Alzheimer's Setback Threatens Rivals' Prospects.

The article points to the weaknesses in Lilly’s trial. First, there is still no consensus that the target of their trial is the best solution. Analyst Les Funtleyder from Miller Tabak & Co. notes:

“The problem is that amyloid is still not the unanimous choice for what causes Alzheimer’s. If you don’t understand how the disease starts and operates, it’s going to be hard to find a cure or a treatment. That’s what we are up against.”

Furthermore, Rudy Tanzi, Cure Alzheimer’s Fund Research Consortium chairman, notes that the way in which Abeta was targeted by the Lilly drug was not optimal.

“the failure of the Lilly drug was due to the poor performance of the drug they used rather than the entire approach of controlling beta-amyloid. Gamma secretase has dozens of proteins it must cleave to have a healthy brain and a healthy body. You can’t hit that enzyme with a sledgehammer because it’s vital and has lot of different functions.”

While the drug failure is frustrating, we weren’t terribly surprised. See Tanzi’s write-up titled Novel Therapies for Alzheimer's Disease Based on Early Onset Genes which was prepared as Supplemental Information for Senate Special Committee on Aging Hearing in the Summer of 2008. He wrote:

LY450139(the Lilly drug) is a gamma secretase inhibitor (GSI) targeted at blocking the activity of gamma-secretase, an enzyme necessary for the production of A-beta. While this particular drug has done quite well recently proceeding all the way to phase III clinical trials, this class of drug has generally been shrouded with potential safety concerns. This is because the enzyme, gamma-secretase, is normally needed to process many other proteins beyond the APP. For example, gamma-secretase is required to processes the essential protein called Notch. When this event is blocked, the result can be skin cancer. Yet, the fact that Lilly’s candidate has made it all the way through to phase III trials would attest to this inhibitor’s safety.

That Dr. Tanzi was skeptical of the Lilly drug over two years ago should be proof enough that the current research strategy is too narrow-minded in its approach. A successful drug for AD will require one that 1. Is safe; 2. Gets into the brain; and 3. Has sufficient potency. With the hope of achieving this goal, Cure Alzheimer’s Fund is supporting research at UCSD and MGH on drugs called gamma secretase “modulators”, which lower amyloid beta protein levels without the safety issues of the gamma secretase inhibitor that just failed at Lilly.

This failure reaffirms the rising importance of better understanding the triggers behind Alzheimer’s. As Tanzi also says in the Bloomberg article,

“If the existing drugs don’t work, advances in genetics are giving researchers a growing list of targets to slow or prevent Alzheimer’s disease”

And this is our goal at Cure Alzheimer’s Fund. More research is needed to make sure we are driving toward a cure. We hope the Lilly trial sends a message to all those effected by this disease: better understanding of the cause will lead to better chances of finding a cure.


Great Recognition

MassGeneral Magazine’s August issue profiles the creation of Cure Alzheimers Fund and highlights the success of our venture capital approach. The article details the innovative work being done by Dr. Tanzi, such as the Alzheimer’s Genome Project, thanks to the bold investments of Cure Alzheimer’s.

In the years when the Alzheimer’s scientific community was focused on better understanding and exploiting the four known genes implicated in the disease, Dr. Tanzi was one of few researchers advocating for finding others. By design, CAF’s founders — who had spent their careers in venture capital, banking and real estate — had established that the organization would do away with the red tape and reams of paperwork required for most grant proposals. And that modus operandi has resonated with the thousands of other donors who give to CAF.

To read the rest of the article, click here.

For a more in-depth look at the Alzheimer’s Genome Project, read MassGeneral Magazine’s article, “Unforgettable Breakthroughs in Alzheimer’s,” in the same issue.

Sharing of Data Leads to Progress on Alzheimer’s

Congratulations to the collaborators and researchers of ADNI (Alzheimer’s Disease Neuroimaging Initiative). The New York Times reports the good progress of ADNI to “find biomarkers that show the progression of Alzheimer’s disease in the human brain”.

Cure Alzheimer’s Fund is a proud contributor of this effort. Read about our funding>

Read the story in the New York Times>