Collaboration takes aim at therapy for early-onset dementia

[caption id="attachment_25043" align="alignright" width="293" caption="Senior Research Associate Karen Poksay works in the Dale Bredesen Laboratory of the Buck Institute, where a new look at Alzheimer’s is the focus of a Buck, BioMarin collaboration."][/caption]

NOVATO – The Buck Institute for Age Research and BioMarin Pharmaceutical are collaborating on what could be a breakthrough therapy for a most feared disease.

Familial, or early-onset, Alzheimer’s can affect people as young as 30, 40 or 50, well below the usual onset of the neurological condition, which is usually developed by people in their 70s or 80s.

“As devastating as Familial Alzheimer’s is, there are no therapies being researched for it because the patient population is relatively tiny,” said Dr. Gordon Vehar, vice president of research for BioMarin.

A report issued by the Alzheimer's Association in March 2007 puts the number of people with AD who are younger than 65 at around 200,000 in the U.S.

“Ironically, most all the research on Alzheimer’s is being done with mice that have been bred to develop the Familial type, even though the target is the much larger Alzheimer’s population,” said Dr. Vehar.

That makes it a perfect fit for BioMarin, which focuses on rare genetic diseases that affect small populations.

BioMarin has agreed to fund a joint two-year research collaboration, the first of its kind, with the Buck Institute, which focuses on the diseases and conditions of aging.

The work is being done in the Dale Bredesen Laboratory, headed by Dr. Bredesen, who holds views on Alzheimer’s that run counter to the traditional way of thinking.

Typically, Alzheimer’s research views the condition as a disease of toxicity, characterized in part by an overabundance of the amyloid Beta (AB) peptide.

But studies of mice in the Bredesen Lab have shown that AB has a physiological function. It acts like a switch to balance trophic activity in the brain. Alzheimer’s represents one end of the spectrum, where people become very, very good at forgetting and failing to form memories.

“We have found a trophic factor, a protein naturally produced in the brain, that both reduces and competes with AB and enhances the right kind of signaling. We believe it could be used to restore the balance of trophic activity and reverse the memory loss function,” said Dr. Bredesen.

A successful therapy has not been developed using the traditional way of looking at Alzheimer’s, he pointed out.

“It’s time to try something new, and I think we have it here. BioMarin is the perfect development partner for us because their therapies are protein-based and their scientists are experts on producing proteins for human use,” said Dr. Bredesen.

BioMarin is also very familiar with the clinical trial process, he added.

According to Dr. Vehar, if the early research should successfully lead to a therapy, BioMarin would take it through the FDA approval process and market it. Such a drug would receive orphan status from the FDA because of its small target population, speeding the process considerably.

“That’s very far off. Now we’re in the earliest stages of research. But Dr. Bredesen’s discoveries are quite exciting,” he said.