An existing drug for liver disease may be effective against Alzheimer's.
This condition is characterized by progressive memory loss, difficulties with problem-solving, and disorientation, among other symptoms.
The current treatments for Alzheimer's focus on slowing down the progression of some of these symptoms and managing the condition's impact on a person's behavior and psychological state.
For this reason, new research is always on the lookout for more targeted treatments focusing on the mechanisms that drive Alzheimer's at cellular level.
In a new study, investigators from the University of Sheffield in the United Kingdom found that a drug that has so far been used to treat liver disease could actually also help tackle Alzheimer's disease.
The researchers report their findings in a paper published in the Journal of Molecular Biology.
Drug boosts mitochondrial function
Previous research suggested that the liver disease drug ursodeoxycholic acid (UDCA) has potential in halting the progression of Parkinson's disease. That study found that UDCA was able to improve the functioning of mitochondria in certain individuals diagnosed with Parkisnon's.
Mitochondria provide cells with the energy they need to grow and divide. A helpful parallel would be to think of mitochondria as cells' "powerhouses" or "batteries."
In Alzheimer's disease, mitochondrial dysfunction is often a factor, and these changes appear to occur even before toxic protein plaques begin to amass in the brain.
This makes mitochondrial dysfunction a great therapeutic target, so the researchers decided to investigate whether UDCA would be able to tackle this problem in people with Alzheimer's.
Using tissue collected from different patients with Alzheimer's disease, the researchers who conducted the recent study confirmed that the existing drug did improve mitochondrial function.
"For the first time in actual Alzheimer's patient tissue," explains lead author Dr. Heather Mortiboys, "this study has shown that the drug UDCA acid can boost the performance of the cells' batteries, the mitochondria."
How does UDCA impact mitochondria?
The authors also noticed that the drug improves mitochondrial function by "correcting" the shape of the affected mitochondria.
UDCA does this by redistributing a protein called "Dynamin-related protein 1" (Drp1). Drp1 plays a key role in supporting the healthy dynamics of mitochondria, and the scientists believe that it may ultimately protect against neurodegeneration.
"We also found that the drug, which is already in clinical use for liver disease, acts by changing the shape of the batteries which could tell us more about how other drugs can be beneficial in Alzheimer's," explains Dr. Mortiboys.
"Most importantly," she adds, "we found the drug to be active in cells from people with the most common type of the devastating disease — sporadic Alzheimer's — which could mean it has potential for thousands of patients."
It is 'vital' to consider 'many angles'
Since UDCA is already in use as liver disease medication, the researchers believe this may help speed up the advent of trials testing its effectiveness and safety in Alzheimer's therapy.
"As the drug is already in clinical use for liver disease; this speeds up the potential time it could take to get this drug to the clinic for patients," says Dr. Mortiboys.
Dr. Sara Imarisio — head of research at Alzheimer's Research U.K., who funded the new study — explains that, since Alzheimer's is such a widespread condition, it is of paramount importance to find better ways of treating it.
"[Since there have been] no new dementia drugs in over 15 years, it's vital we continue to approach Alzheimer's from as many angles as possible. Through innovative research we are building a clearer picture of the complexities of the disease and how it develops in the brain."
Dr. Sara Imarisio
Dr. Imarisio encourages scientists to continue to delve into the implications of the findings obtained through the recent study.
"This work suggests a potential new way to target Alzheimer's but needs further exploration before we can know whether this drug used for a liver condition is safe or effective for people with Alzheimer's disease," she notes.