cascade of processes resulting in cell death , cognitive decline and eventual dementia .
For more than two decades , Mark Tuszynski , MD , PhD ’ 91 , UC San Diego professor of neurosciences , and colleagues have been investigating the potential of gene therapy for AD in a different way .
In 2001 , Tuszynski conducted a novel phase I clinical trial to assess whether injecting nerve growth factor into the brains of patients
“ Across the spectrum , from basic science to clinical care , our scientists and physicians have grappled with the challenge like few other institutions . We won ’ t stop until we , in collaboration with others , can claim true and complete success .”
— Chancellor Pradeep K . Khosla diagnosed with AD might slow or reverse neuronal degeneration . That seminal work led to last year ’ s announcement of a new , first-inhumans clinical trial to assess the safety and efficacy of delivering a specific protein called brain-derived neurotrophic factor ( BDNF ) into the brains of patients with AD or Mild Cognitive Impairment . BDNF is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote the growth and differentiation of new neurons and synapses . BDNF is particularly important in regions of the brain susceptible to AD degeneration .
“ It has required decades of focused work to get to this point , but we ’ ve found in animal studies that delivering BDNF to the parts of the brain that are affected earliest in Alzheimer ’ s disease results in reversal of loss of neuronal connections and protects against ongoing cell loss ,” says Tuszynski , who also serves as director of the Translational Neuroscience Institute at UC San Diego School of Medicine .
Repurposed Drugs The search for brand-new treatments for AD has yet to produce a major success story . Consequently , many researchers have turned to looking anew for potential remedies already in their armamentarium .
The second effort funded by the Epstein gift is dubbed Powder for Pennies , or P4P . It will ask whether drugs already approved or developed for other conditions may also benefit persons with AD . The recent case of bumetanide provides an example . This treatment is an oral diuretic or “ water pill ” used to ease congestive heart failure by reducing fluid retention , but bumetanide has also been found to reverse abnormal expression of a gene already identified as a strong risk factor for sporadic or late-onset AD .
Thus , an older drug might be brought through clinical trials quite quickly and , if successful , could provide an affordable and widely available new treatment possibility for AD .
The P4P program will also develop and implement early-phase “ SMART ” clinical trials with an expedited assessment of the potential of existing drugs and compounds . It will further leverage ADCS ’ s existing network of labs and collaborators and their deep expertise in conducting early trials of candidate treatments . Once compounds of promise are identified , they could be sent to USC for larger-scale phase III trials .
Finding a Cure “ It ’ s instructive to step back and ponder how UC San Diego is frequently front and center for the most seminal discoveries in Alzheimer ’ s disease ,” says James Brewer , MD , PhD , chair of the Department of Neurosciences and director of the ADRC . “ Not all of these discoveries were acclaimed at the time as groundbreaking or fundamentally changed thinking , but over time , they endured scientific scrutiny , were built upon by others , and finally became widely recognized as breakthroughs critical for advancing the fight .”
Brewer is cognizant of the long road ahead , however . It ’ s a fight with no end in sight . But he is also optimistic that the work here , supported by philanthropy , will make a difference .
“ I have no doubt the path to a cure passes through UC San Diego .”
— Scott LaFee is director of communications and media relations for UC San Diego Health .
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