Does The Cure Alzheimer's Fund Accept The Answer?
We believe nosotros directly receive got a real comprehensive agreement of the causes of Alzheimer’s disease
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| Jeff Morby, Chariman Cure Alzheimer's Fund |
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Several weeks ago, the Cure Alzheimer’s Fund (CAF) Executive Committee together with staff met amongst the members of our Research Consortium inwards Chicago.
The purpose of the coming together was to review the progress of the dissimilar researchers together with to brainstorm amongst them, every bit nosotros produce every year, close what modifications nosotros should brand to our roadmap to receive got wages of our findings to appointment together with to decide query targets for the coming yr – always amongst the objective of accelerating our progress inwards finding a cure/preventative for Alzheimer’s.
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This yr the coming together amongst the researchers was dissimilar from yesteryear meetings, because nosotros believe nosotros receive got reached an of import inflection point:
First, every bit a upshot of our query to date,
we believe nosotros directly receive got a real comprehensive agreement of the causes of Alzheimer’s disease.
We together with our researchers receive got jointly agreed upon a standardized model of the many causes of Alzheimer’s illness stemming from our research, together with nosotros receive got linked those causes to specific genes. This is a tremendous breakthrough because it gives us a clear pathway amongst which to focus our query – the genes together with the causes receive got directly been linked!
Second, nosotros directly receive got acquired the consummate dataset amongst which nosotros tin dismiss decide amongst precision where inside the entire human genome occur “functional variants.”
These are variations inwards the deoxyribonucleic acid of whatsoever cistron of involvement which modification poly peptide functions every bit compared to the functions of normal proteins.
We are specifically searching for functional variants inwards the genetic subset of the 100+ Alzheimer’s genes that directly receive got been discovered together with which would move predicted to straight affect opportunity for disease.
The novel genomic dataset has been created yesteryear us every bit a upshot of ii achievements:
1) The completion of our $5.4 1000000 Alzheimer’s Disease Whole Genome Sequencing Project (WGSP) of 1,500 Alzheimer’s families, combined amongst 2) the evolution of state-of-the-art algorithms together with software (the biometrics) to permit us to efficiently analyze the “big data” generated yesteryear the WGSP.
The WGSP was financed solely yesteryear Cure Alzheimer’s Fund, together with the biometrics projection was supported both yesteryear Cure Alzheimer’s Fund together with a $4 1000000 grant to Dr. Tanzi from the National Institute of Mental Health.
We are the outset query grouping to generate a comprehensive familial Alzheimer’s illness database capable of linking cistron defects straight amongst encephalon malfunction for virtually whatsoever cistron of interest.
We believe this is currently the most comprehensive database of Alzheimer’s genetics inwards the world.
Third, nosotros receive got 3 primal potential cures inwards advance states of development.
One, the Gamma Secretase Modulator (GSM) project, which nosotros funded initially together with directly are continuing to fund together with refine amongst the NIH (which is contributing millions of dollars to the project), should move moving into human trials over the coming year.
Two other drug evolution projects, ane revolving around the Alzheimer’s cistron known every bit CD33 together with the other involving ACAT inhibitors (which inhibit the production of amyloid), are also good underway. The CD33 gene, which nosotros were the outset to seat v years agone inwards our outset whole genome scan, is extremely powerful inwards damage of impact, together with nosotros receive got been working to empathise its “mechanisms of action” (that is, empathise how it works) – this nosotros directly receive got accomplished! The CD33 genetic variant is protective against Alzheimer’s together with has provided valuable clues for identifying therapies that would mimic that fashion of protection.
Fourth, nosotros are planning a large release of novel query projects to move directed yesteryear a broad multifariousness of outstanding scientists aimed at agreement together with finding cures for every ane of the novel Alzheimer’s genes shown inwards the attached chart, amongst the APOE cistron together with tau tangles every bit transcend priority targets.
Our progress on all of these efforts is existence greatly accelerated by our ability, using our novel tools together with datasets to seat the precise variations inwards our deoxyribonucleic acid that explicate how our novel Alzheimer’s genes affect opportunity for disease.
So what is the “inflection point” I mentioned above?
It is the betoken at which nosotros are in conclusion inwards a seat to believe that nosotros receive got all the information nosotros directly withdraw to honor a cure.
That information is inwards the shape of a comprehensive model of AD pathology; an advanced database together with algorithms to pinpoint variants amongst precision together with link them to their deleterious manifestations; a rich prior history of experimentation (over 100 query papers inwards the best publications), which provides us amongst the background to intelligently translate information identified amongst our novel tools; together with a world-class grouping of AD researchers, totally knowledgeable together with committed to working inwards a collaborative agency to rid this planet of Alzheimer’s disease.
But it volition toll coin to receive got total wages of our unique together with privileged position inwards agreement the link betwixt Alzheimer’s genetics together with encephalon pathology, directly provided to us yesteryear our highly successful whole genome sequencing efforts.
The founding families together with directors both contribute to query funding every bit good every bit pay all of the operating costs of the foundation together with thence that all charitable contributions larn 100% into research.
We buy the farm along to brand that commitment - putting our coin together with efforts “where our mouths are.”
We together with our researchers are real excited close the hope offered yesteryear this novel phase of our development, together with nosotros hope y'all are too.
If y'all receive got already made your annual appeal contribution, I give thank y'all you. If y'all receive got non yet, delight assistance us inwards this quest.
Jeff Morby
Chairman, Cure Alzheimer’s Fund
Editor Note: all emphasis together with bolding are mine.
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