Eighth Annual Melvin R. Goodes Prize Symposium

everybody I'm Mark reuth mayor I'm the CEO with the Alzheimer's drug Discovery Foundation if you have my perspective from standing up here clearly the bride side of the house has more guests than the groom side of the house today one two I think as we all know it's going to be a little bit of a later arriving crowd today because it's un week but you know kudos to all of you for getting yourselves here um so good to be back together again and Nancy had pointed out to me almost three years ago to the date in the exact same room the last time we were all together for the goods prize and so much has happened in the last three years in the space of Alzheimer's science and I'm just going to tick off a couple of them significant shifts one we now know you can start seeing signs of Alzheimer's 20 to 30 years before the first symptoms are going to come out two you can now diagnose diagnose Alzheimer's not only with a pet scan which was there three years ago but now there's a blood test and there's literally a plethora of new Diagnostics that are going to be coming through in the next couple of years I scans artificial intelligence that you're going to get off your Apple phone right that can measure all sorts of things from the way you speak to the gate of your walk that are coming and perhaps the biggest shift that's happened because I know when we were together three years ago it would be like you know why would I want to know right why would I want to know I will tell you that question is now different because the statement is now I can know if I want to know okay and the biggest shift in the last three years and you'll hear it today there are now prevention steps you can take that will push out delay the early signs of Alzheimer's okay and you're going to hear all about that today but for you but before all of that um I need to just give a nod to Nancy Goods Mel Goods the goods family that's here today um you're going to hear a lot about that at lunch but I'll just start with the addf its board has decided that this Goods prize is in perpetuity this will go on yeah forever Mel's Vision his business Acumen being the first Pharma executive that brought the first Alzheimer's drug to Market but here's the other shift in the last three years it used to be Nancy the addf goods prize and we have awarded it in past years to very deserving people that are here today and you're going to hear all about from but the science of Alzheimer's has now shifted this thing about plaques and Tangles and amyloids sure it has a part of it but it's one piece and we know it's now a much more complex disease and it has to be attacked through prevention combines therapies Precision medicine it's called the biology of aging and the biology of Aging is now coming Center Stage and this Goods prize award every year picks out the top scientists in diverse clinical trials so it just so happens Nancy that the goods prize award and where the science is moving is the exact same thing and it's going to become like the Nobel Prize okay so really Lots that's gone on in the last three years so first of all thank you Nancy thank you to the goods family it's wonderful to be here with all of you today if you could have a round of applause for them sure I was going to use a phrase earlier but Robbie Brinton uh scrunched up her face at me and said I don't know that that's the best phrase I was going to say before us is the murderous row of clinical scientists and Alzheimer's and she looked at me she said murder is row and for those y'all know it's a phrase from the New York Yankees who when they had a batting lineup that one through nine could all hit home runs so so Robbie what did you say me it's the home run row of everybody but you will hear from each of them um you were going to hear from all the way at the end is Dr Miranda or our 2022 good surprise winner and she will be feted at lunch today but we also have Dr Longo Dr wardis and Dr Britton Dr Rook rook and Dr kivapelto I believe for Dr rook and Dr Kevin pelto you didn't get to do this in person right so this is so this is your party too today which which is good um Justin Cadence I'm going to turn this over to Howard just in a second he will walk through the panel and and lead a presentation for the next 45 minutes or so and then we're going to open it up to q a and there'll be microphones that will come around to each of you for for your questions um and then the last piece of this of which I knew no script uh is to turn this over to Dr Howard Phillip and um when I think of Howard I think of a partner in addf I think of a co-founder of the addf with the Lauder family he's our chief science officer he is 40 years plus Leonard Lauder likes to call him a truffle Hound for scientific ideas to treat and cure Alzheimer's um he is a phenomenal human being he's a good friend Dr Howard Phillip foreign thanks Mark um and thank all of you for coming today and of course I especially want to thank Nancy and and her family the goods family uh for supporting this and creating this award um and getting us to where we are today it's been a real honor and a pleasure to work with Nancy and her family as Mark mentioned we have been giving this award for a number of years now and the theme really is about aging as the leading risk factor we've had over 100 Years of research on aging and it's a real time to translate that knowledge about what we call the biology of Aging that we've learned over all those years into new drugs for Alzheimer's disease and I think as Mark mentioned that basically in one way or another all of the research science that you're going to hear about today is in one way or another related to the biology of aging and many of you have heard about what's been going on with Biogen and some of the other big Pharma companies hopefully bringing one of their anti-amyloid drugs to Market in the next year maybe or something like that and I think you know there is a possibility that this will happen but we know from the data that we've seen so far that even if these drugs get approved and come to Market so people can take them that the effect size the size of the effect of these drugs is marginal or modest at best and so what that means is we've found a way to start treating the disease these drugs may be the first to market after 30 40 Years of research in Alzheimer's we finally have a drug which by the way is a time frame that's right on time so considering that the National Institute on Aging which is the main supporter of Alzheimer's research in the world really started only about 40 years ago and it takes about 35-40 years to go from some basic science knowledge to new drugs I think we're kind of really exciting to be right on time but what you're going to hear about today is not about what Pharma is doing or these new anti-amyloid drugs it's going to be the new drugs that the new Pathways that are being explored and what's really exciting about it is that we've been supporting people on the panel for many years now and all of the talks that you're going to hear today about the drugs in development are all at the stage of clinical development which means that they're closer to patients than they've ever been phase two-way studies some of them getting even further on along to to research and improving that that these novel Pathways really work so it's it it's really I think you're going to hear a great panel today about where we're going and what the foundation is doing so let me get right to work here I'm going to introduce our first panelist who's going to speak about his work Dr Frank Longo Frank is currently the chairman of Neuroscience and neurology at Stanford and he's a founder of a biotech company that addf has been invested in from the very beginning we've been supporting Frank for I think about 20 years or so but he's finally at the point where it's really where the rubber meets the road his his amazing drug is in the clinic and in later stage going into later stage development now and um so with that let me welcome Frank and it's going to talk about the good surprise and his work yes thank you Howard um and appreciate everyone being here and it's a pleasure for me to be here with all of you in my uh scientific uh colleagues and uh very much appreciate being part of the goods program and the support and I'll be happy to describe some of the work that the program has made possible so what we're attacking in our program is what we call this synaptic failure and synaptic degeneration so in Alzheimer's disease and early on this the synapses those are the connections between the neurons their function starts to fail and the neurons can't communicate with each other well and we start having memory loss and the other symptoms and the synapses fail and then they actually degenerate we lose those connections after a while and so the goal is to find ways to protect those synaptic connections to maintain them or even to bring them back and all kinds of things can contribute to their failure amyloid Tau brain inflammation and people debate what are the most important contributors but in the end the bottom line of synapses synaptic connections are degenerating and as they degenerate inside the cell there are degenerative signaling patterns all of our cells have chemical signaling patterns that tell the cell what to do and so in as these synapse they're degenerating for some reason the neurons have a degenerative signaling pattern of chemical signaling pattern inside them so our goal is to prevent that degenerative pattern now one way to control the signaling in the cells with a proteins called growth factors growth factors are proteins that have very powerful effects in telling a cell what to do so for example a growth factor can sell it tell a cell to divide and if it's doing that inappropriately that cell becomes cancerous so this is very powerful biology and those those growth factors work on what we call receptors on the surface of the cell and those receptors control the signaling inside the cell in the case of neurons so-called nerve cells in the brain we have growth factors two very famous One one's called nerve grow growth factor and the other one is called brain derived growth factor or bdnf brain derived neurotrophic factor and so these are very powerful protein growth factors that can that can control what the neuron survival and the survival of its synaptic connections they're important for forming the brain but we can't use them as drugs directly unfortunately because these proteins these growth factor proteins won't get through the blood-brain barrier that's a barrier that protects the brain so while growth factors can be important for diseases outside of the brain for example there's a growth factor called epogen that we use if we have anemia it can stimulate red blood cells so growth factors have been proven to be very successful in medicine but not for brain problems [Music] great time for a fire alarm yeah [Music] set it off again yeah there we go well it's good they're checking that so so our the goal of our program then was to create a new a brand new class of drugs that would stimulate these receptors on the neurons to prevent the synapses from degenerating so our goal was to create drugs that do what nerve growth factor does or do what bdnf does uh to to protect our synapses so in our first program we managed to create the first what we call small molecule drugs that can get inside the brain that do what nerve growth factor does and the discoverer of nerve growth factor Dr Rita Levy multicini and Italian neuroscientist one of my heroes she actually won the Nobel Prize in medicine in 1986 for her Discovery because she thought and everybody agreed that someday the way these growth factors work could create drugs someday and she said someday somebody has to come along and create a drug based on this discovery so luckily with the addf and goodies program Goods program support we've been able to do that so so with the our small molecule drugs that mimic what nerve growth factor does we discovered that they work very well in mice many different kinds of Alzheimer mice and we have been able to go through the move them into human trials into phase one human trials accessing safety and demonstrating that they're able to the drug is able to get in the brain and we recently completed a phase 2A trial in patients with mild to moderate Alzheimer's disease and we're so excited to find out that the the patients and mild to moderate Alzheimer's disease is fairly Advanced Alzheimer's and many people think it's too late to treat at that point but we went late on purpose um and we're excited to learn that um the spinal fluid testing shows that our drug treatment is decreasing the degeneration of those synapse connections um and this is the um the first drug shown to decrease two of the key measures of those synaptic degeneration so it's real exciting that this drug is is decreasing the degeneration of synapses in the brain as shown by two different measures and it's the first time a drug has shown that by both of these measures in our second program that the the goods funding is made possible we we've managed to create small molecule drugs that do what brain bdnf does this is a very powerful thing and we've managed to see it work successfully in different kinds of Alzheimer's mice and we're excited that it prevents degeneration caused by both amyloid and Tau and and so we think it will be very powerful as well and we've done the work that will make it possible now to begin to move it into human studies and so the goal now would be to do a human trial with this bdnf type of drug and bdnf promotes learning it promotes plasticity of the synapses and it may be one of the factors that exercise uses for brain health and perhaps me able to talk she knows more about exercise than I do so we're excited about that program too and we appreciate the support of the goods program and making it possible so thank you concerned let me just say that we're going to ask each of the speakers to present to you please think write down your questions and we'll have a question and answer after all the speakers have presented their work so if there's any pressing questions please keep them in mind and we'll get back to them okay that's wonderful thank you our next speaker is Dr Waterson from Northwestern University he's so Frank Frank's so in the car just to put things in context so Frank's uh drug is is a broad spectrum type of drug neural protection uh regardless of one of the great things about it is no matter how the neurons get injured Frank's drug could potentially save those neurons and if it works and we're so far the data has been really exciting um then it could have a widespread application for all sorts of neurodegenerative diseases not just Alzheimer's so we're we're really excited it could really be a breakthrough drug this idea of having a neuroprotective drug has been in people's in scientists mind for many many decades and this is the first time I think that we're getting close Dr waterson's work that was funded by an India for many years addresses one of the Hallmarks of the biology of aging and that's inflammation and Marty is currently the John Searle professor of molecular biology and biochemistry at Northwestern school of medicine and a founder of a biotech company that we've also invested in like pharmatrophics a company called neurokine so Marty please thank you interesting um we're actually studying stress Pathways so I think I was I was tempted to stick you know Frank's arm here and bleed him clear quickly by the way I think the Yankees analogy is um very appropriate having grown up near the steinbrenners and talked to them about how they identified these Stars they made a point of looking early they actually had people going on and watching Little League games and Pony League games and identifying and even in other countries so uh I think the ADF in terms of identifying people the rest of these panels is much more outstanding than me is really at an inflection point in their careers where the NIH had provided a lot of support actually did a key investment that allowed us to take high risk Pathways and pursue the therapeutic applications of our research and following Frank's description of the environment of the synapses we actually study one particular aspect of that the brain inflammation in fact we focus very much on one class of small molecules that are up regulated but you also need them for homeostasis balance are called cytokines you've probably heard a lot recently about cytokine storms so in acute injuries certain other types of situations you have a rapid increase and that causes the synapses to be injured or malfunctioned but normally you have a fluctuation the cells in the brain are called glia and so the neurons tell the glia I'm happy don't worry about it the glia gives some factors that help out with that and they go back and forth but when there is a gradual progression and a chronic neurodegenerative disorder the neurons are doing these very subtle signals being sent out and the pathways are being disturbed as Frank described and that sets off these increasing age-dependent susceptibilities that one can pick up early so what we're doing we're actually monitoring some of the molecules or there have been cerebral spinal fluid markers which have now been correlated in the last year with blood levels so that greatly enhances our ability to study a larger number of patients at multiple sites and monitor that gradual increase over a period of time we're also looking at the susceptibility of certain populations certain groups of people who would be susceptible and the small molecule drugs we've developed for this get into the brain they bring down it we do bring down to a homeostasis level we call it to where it's under control the analogy here would be something such as maybe amlodipine which probably most of the males in this room are taking okay it's basically bringing down control of a pathway it also has to do in terms of the lipid controlling drugs which Mr goodis was very famous for in pioneering that so it's the same concept it's but it's more focused on the homeostasis so that is this Progressive disease and identifiers alerting you the fact that you're more susceptible to that you would be able to have a safe small molecule that can be taken orally once a day that would attenuate that risk and I want to appreciate express my appreciation too for the goodest family for this catalytic funding as I call it and also the addf for the great network of scientists they've stimulated working on these various aspects of brain pathophysiology and again thank you very much and so so mark thank you Marty yes okay so as I mentioned Marty's uh therapeutic program addressed to some of the uh dysregulated inflammation that occurs in the brain that is associated with cognitive decline and progression of the disease our next speaker is Dr Robbie Britton a colleague I've known for a very long time and she's been also working on a neuroprotective strategy for protecting neurons within the Aging brain from degeneration and death but her Focus has been very much on the hormonal side of developing Therapeutics her lead drug is a drug called alloprignanolone which is a derivative of of progesterone which is obviously along the lines of the hormonal balance in women with estrogens and progesterone and she's been at this for a long time but finally in the clinic with her drug she's started a biotech company and she's also currently the inaugural director of the center for Innovation and brain science at the University of Arizona where she also serves as a professor of pharmacology and Neurology in the College of Medicine so probably please thank you Howard and thank you all for coming and joining us this morning and I think something that we've all heard from addf is that hope is on the horizon and I hope from after you hear us this morning you will agree with me that hope has arrived and for our work we started this 25 years ago and um one of the things that was really fantastic was that addf had two approaches one bold the other is precision medicine for Alzheimer's before Precision medicine was a term and when I came to addf and told them this bold idea that what if the brain could actually regenerate what if the Alzheimer's brain wasn't Gone Forever what if we could actually regenerate that brain and we turn to a discovery that I made and part of that was aloe pregnant alone this molecule aloe a-l-l-o for short I made the serendipitous Discovery in the laboratory that when I looked through the microscope after I treated nerve cells with aloe and there were more of them and what was very exciting about this is that we've now moved past understanding how aloe works and through multiple tests of safety and actually all of you have experienced the regenerative effect of aloe already actually it's the generative effect all of you have a brain and all of you went through a burst in brain growth during the third trimester of pregnancy when the brain undergoes a four-fold increase in size when that brain is developing generating new neurons generating new connections and so we are woo it's one minute we are actually leveraging what Mother Nature has developed that is safe an effective in females and males to regenerate the Alzheimer's brain and I have to say that one of the great contributions Beyond being bold and supporting our our research addf has had is through the goods prize because even though we have a very exciting possibility to regenerate the brain it's not a novel molecule like Frank's like Marty's it's one that Mother Nature has tested and it works so what we've been able to do with the goods prize is to move forward with patenting formulations of this molecule to deliver that molecule to the brain and lastly what I would say end on is that we have very encouraging data from our phase 1B to a clinical trial suggesting that indeed the Alzheimer's brain can generate new nerve cells and new connections so thank you addf and the goods prize thank you thank you Robbie and I I just want to say what Robbie mentioned about developing the drugs well here's a natural hormone that we all have in our bodies um and if we want to develop a drug what we really have to do is somehow find a commercial pathway through intellectual property so the drug could ultimately be approved by the FDA paid for by the insurance companies and be made available to patients so in other words the the problem is we could have a lot of science going on the science could be published in a scientific literature but it ain't getting to patients unless there's an intellectual property strategy which could be something like reformulation and we've worked with Robbie and talked over several years I would say about what that strategy is and you know Robbie can take the L pregnant alone and give it to people but she needs a formulation that can be patented and then if the drug Works can be sold and so we're talking about a patch and other formulations that would make this desirable and and bring the print her drug to Market which I hope will happen in the next few years and again another example also of someone who's been at this for about 20 years 25 25 years on this one project and you know it it just takes a long time and a lot of money to develop a drug it's it's uh and and in Academia it's particularly I think hard to academics don't have the resources that big Pharma has but academics can take risks that big Pharma won't take and that's I think our sweet spot so thank you Robbie our next speaker is Dr Jerry Rook from Vanderbilt University Jerry is currently a research instructor in the department of pharmacology at Vanderbilt but also a very novel chemist I would say and pharmacologist and her drug is actually developed to enhance cognition and maybe reduce amyloid and so on through What's called the M1 receptor so we're proud to have you as a good surprise winner and listen to your talk thank uh I want to thank everyone for being here today I always love these events and unfortunately we haven't been able to do it for a few years but I'm very happy to be here uh definitely want to thank the uh tonight thank Nancy and the goods family as well as Howard and the Alzheimer's drug Discovery foundation for your continued support your efforts really do make a very big difference um both in the our understanding of the disease of Alzheimer's as well as our pursuit to find new treatment strategies so today I just want to talk to you a little bit about my projects both the one that Howard mentioned as well as a new one that I've started um and due to the goods prize and so Howard would referenced a small molecule that we developed called the M1 positive allosteric modular or M1 Pam and so many of you know or have had family members who have taken acetylcholinesterase Inhibitors it's one of the standards of care for early treatment of Alzheimer's disease unfortunately it's accompanied by a lot of really negative adverse side effects it's a very what we call Dirty drug in that it it hits multiple targets throughout the body not just the ones that are important for learning and memory in the brain and so what we did is we we were teased out you know which which of these targets really are important in the brain and how can we selectively turn on those targets to try to enhance the cognitive impairments that occur in the Alzheimer's brain and so we spent several years with the help of the Alzheimer's drug Discovery Foundation discovering new model new molecules so we have several novel molecules that we've demonstrated are very selective for this M1 receptor in the brain um they're very um they're very selective they're very potent they have cured Alzheimer's in mice which we all try to do and so far uh have have a really nice safety profile so we're able to avoid all of those negative adverse effects that are observed with a non-selective activation of these acetylcholine receptors throughout the body and right now I'm happy to say that we're currently in phase one clinical trials the program's going very well we've partnered this program partly with all the help of addf we were able to advance the program to a stage that got partnered with Acadia Pharmaceuticals and so now they're conducting our clinical trials for us and the second program I'd like to talk about is is in its much earlier stages and so with a Goods award we've been able to start a new drug Discovery program it's really hard to get started with programs because there's not very many mechanisms to support this type of work so we were really fortunate and this program it really focuses on a different chemical in the brain the first one was acetylcholine this one is glutamate and so glutamate is a chemical in the brain that's also essential for learning and memory and um what we know or what we have evidence of is that this chemical is is over active or there's too much of it in the brain in early stages or we're talking before symptoms of Alzheimer's disease and so what we're focusing on is to try to inhibit some of that activity so that we can so that we can stop this over activity that causes the death of these neurons and the death or the disconnection of the synapses so we've developed a lot of really novel compounds that Target the in Blue 5 receptor in the drain it's a negative allosteric modular so instead of increasing the activity it tones it down so far we have some really nice data these are all pre-clinical models it hasn't made it into the clinic yet but what's really nice about this program is that we have a pet radio like in so we all talk about biomarkers and how important they are in drug Discovery research and for this program we actually do have a really nice biomarker and it's going to help us you know translate what we see in all of our play clinical models into our clinical trials so the programs are going really well again I'm happy that you were all here and that I was able to share our programs with you and thank you to the goods family and to Howard and the adtf thanks thanks Jerry and I in the spirit of trying to give a big picture point of view um what what Jerry is doing in terms of developing new chemistry again from the point of view of getting to commercial goals and being bringing her drugs to Market in in her case and broadly speaking the new intellectual property comes from the chemistry uh and so we don't have to worry about things like formulation necessarily or other things because the real solid uh intellectual property comes from inventing these new molecules which is really an amazing thing because you know you have these people that are called medicinal chemists that see molecules in their mind and they they that they're amazing people if you ever want to talk to somebody who's creative and incredible um you know they can take like a receptor like Jerry was talking about the M1 receptor and I know you all don't know what an M1 receptor is but imagine that there's this really important receptor you can put it up on a screen Frank did this 20 some years ago and it took like a year to do it and now I guess you can do it in about a week or so with new computational power um but it's you know it's it's like taking this this molecule and then designing it in maybe in software for example and then creating you know creating the molecules in in powder form and then being able to test them and it's it's a long process but once a new molecule proves that it's effective the intellectual property is there and the access to patients for an indication becomes possible I think that's important thank you our next speaker is a world leader probably the leading figure in the world right now in prevention and I know many if not most of you have questions about prevention and how we can prevent Alzheimer's disease and even cognitive decline with aging and Dr Kevin pelto is a professor of geriatric medicine at The karolinska Institute in Stockholm her original studies were done in Finland where she's from where she conducted one of the first randomized clinical trials of prevention to see if and try to prove that prevention really works and of course mayor was able to do that and of the the power of her study in Finland called the finger study is only is best illustrated by the fact that her study is now being replicated as a prevention approach to the disease in more than 45 countries around the world where each country wants to prove that these this strategy works for for their population the Chinese cotillions whoever whoever it might be and what we've been doing with Mia which she'll tell us about is sort of the fingers the name of the trial's finger she'll tell us about finger 2.0 where we're adding the leading anti-aging drug which is metformin to her prevention efforts so may I please thank thank you and really nice to be here and see you all and hear all these fascinating achievements is really wonderful so I also want to thank first Nancy at the code is prize and addf for the support without that our work would not have been possible so thank you for that so first I would like to give a very short update about our work focusing on prevention and here we are moving towards Precision prevention and combination Therapies so prevention how I see it is really one of the key elements if we might want to manage the global dementia epidemic we are getting more and more cases with demands and prevention can really be the key and luckily there is so much we can do already today the latest estimates are saying that at least 40 percent of all Dementors are related to vascular metabolic lifestyle environmental factors and could be prevented or at least postponed and the list is getting longer it's not anymore this normal lifestyle factors but we also have factors like hearing loss social isolation even air pollution has been part of this list and we are studying new risk factors like stress sleep disturbances and of course covet pandemic has been also affecting many of these risk factors so there is a great prevention potential well the challenge has been how to translate this epidemiological and experimental findings to successful clinical trials and as we hurt our finger trial was the first large randomized control trial to so that it is possible to prevent cognitive impairment if you put together all these different risk factors so you need to have the multi-domain intervention now we call it the finger model because it's easy to remember it's like 105 fingers healthy Palace diet physical activity cognitive training social activities and relaxation and taking care of all vascular and metabolic risk factors what are the mechanisms we don't know yet but I'm grateful for the support from addf that we can use the new blood-based biomarkers to study the mechanisms and actually pdnf could be one of those that's my hypothesis then the next step we are upgrading finger to finger 2.0 and that's even more individualized model so that we can take into account the risk profiles and genetics and combine that with the possible disease modifying repurposed drug and we selected metformin the diabetes medication because there are so many links between diabetes and Alzheimer's and Metformin is very interesting drug because it's widely available maybe many of you or someone is using that I know is widely used and is linked with the key pathological features in Alzheimer's disease like inflammation oxidative stress synoptic function amyloid and tough so we want to test this this combination maybe in the future we can add to this combination some of the new trucks we have been hearing now here finally just mentioning the world white fingers what we heard about we have 45 countries testing optimizing and adapting the female model in different cultures and in different settings I'm sure we will learn a lot what is the best way to do the interventions and we are also using prospective harmonization so it's easier to compare the results we will get Peak data and I'm thinking all the opportunities we have for data-driven analysis AI based analysis making new discoveries and findings so I'm very optimistic for the future we can do so much already now and even more interview so thank you so much for being uh I'm sure we'll have a lot to discuss on prevention when we get to the Q a meeting as we're going to comment other than you know you've really revolutionized the field by the work that you've done um and made it real I think is is really what's happened um and now it's my real privilege to introduce our winner of this year's good surprise award and a very creative scientist and someone who's work we're really excited about Dr Miranda Orr who's a assistant professor of geriatric medicine right now at Wake Forest and if I can kind of context it we've talked a lot about the biology of Aging um and all of the speakers that you've heard from have one pathway in that biology and I would say that as we age that we in all of our organs we develop what are called senescent cells and I'll let Miranda I don't want to steal her Thunder talk about it but the big picture is that we're taking a field called zeroscience or gerontology which has been around for probably a hundred years or more and for the first time really translating geoscience and gerontology into new Therapeutics and the addf has proposed this several years ago and and what Miranda is doing is actually developing an entirely new class of drugs called cenolytics which are anti-aging drugs based on very fundamental biology to get rid of Aging cells that are refusing to die and are highly inflammatory and are known to be in the Alzheimer brain so it's really at the very front of where we are with the biology of aging and it's it's sort of what we call promiscuous in the sense that this drug can have widespread effects so let me turn that over to Miranda for her presentation today thank you so much thank you Nancy and the goods family for this amazing award and thanks Howard and the addf for supporting me in my research and I have to say a huge thanks to all of my co-panelists here I I say this all the time I'm really standing on the shoulders of giants these are leaders in the field this is the who's who panel in Alzheimer's disease and to be a part of this club you've got me forever so don't don't ever kick me out sticking with this analogy of murders row and being the new kid on the Block what am I doing I am murdering cells and I get asked all the time do you really think this is a great idea do you want to kill cells in a disease where cells are dying and the answer is we're going to find out so as Howard mentioned as we get older all of our tissues in our body we accumulate stressed and damaged cells just by everything that we do in our life and these stressed and damaged cells is particular kind called senescent cells they're also sometimes called toxic zombies and I'll tell you why they don't die they're not able to Die the only way that they can get out of our system is by the immune system and so they send out messages telling our immune system come get me I'm damaged I need to get out but if the immune system doesn't hear this message these cells continue to spit out these molecules that are very inflammatory and they stick around and they cause disease and my lab found that these cells accumulate in Alzheimer's disease and if we use drugs that Target these cells and let them die uh we saw improvements in brain structure and function in mice of course we've all probably cured mice everybody on this panel has cured mice and so now the big test is how will this impact people with Alzheimer's disease so we found evidence that these cells are in the brains of people with Alzheimer's disease and with the support of addf we we launched a phase two multi-site clinical trial where we're testing this and with the goods prize we are using a really cutting edge novel approach to take a look at what's happening in these cells this is like if you open up the front door of the cell and walk in not only can you see what's in the room but you can see what designer made these things in the room and why this cell decided to put the lamp in this room and why a cell decided to put a lamp in a different room and with this new information not only are we understanding what's happening in these cells but we think we're going to be able to develop some new drugs and and further this whole drug development process and in making connections with everybody on my panel so these cells are inflammatory so we heard from Marty this is a huge aspect of aging and Alzheimer's disease we know that these cells aren't very good neighbors and so if we can get rid of them I would love to start a trial with Robbie and you know once these bad guys are gone now we actually give the brain a chance to rejuvenate with growth growth factors prevention there is actually evidence that exercise can reduce the number of senescent cells in your body so some of these healthy aging interventions or preventative methods are are directly relevant to the aging process so I'm really excited and I Echo what all of my panelists have said where I think that we're on an inflection we're in this inflection point where there is hope I think the addf for being bold in supporting this and I think everyone here for supporting us and and I'm just so grateful to be part of this team [Applause] so now that you've heard everyone I I hope you can remember your questions um and please we have a microphone so if you just want to raise your hand if you have a question um we'll get you a microphone and you can ask any of the panelists some questions uh Sharon yeah non-medical question but this panel was so compelling uh can you tell us if I I know it's being filmed when it will be available on our website so that we can direct other people to it and maybe uh hear it again ourselves thank you uh the answer was by Monday from our CEO so Carol Carolyn you're talking about inflammation and there used to be data that I saw that NSAIDs and some statins that also have the effect of lowering inflammation in the brain possibly and might be preventative for Alzheimer's do you believe in that and should we be taking statins or Advil or something for our brains well um that's a very good question the departments pardon the sports analogy but we need lots of shots on goal okay and these are existing ones and and some of the drugs you're talking about um the statins especially they're part of a age-dependent susceptibility and they do have an indirect effect so there's they're um term is pleiotropic okay they have multiple effects so what we're we're actually leveraging that prior work and finding these particular spots we can have laser guided compounds that will hit them because their primary movers in that response and that's how you're going to get to Precision medicine so even if our drugs don't make it to Market because it didn't have the right appeal to Industry and even if we have good intellectual property during the clinical trials by having these guided drugs or the specificity that you've heard about you can better interpret the clinical trials and know how to come back recursively and improve those in the Next Generation so it's a very good question those are the Pioneer drugs that we got hints from in fact Howard was one of the early people to identify this trend himself so we're sort of the second generation if you will I I think you know you asked about Advil for example in the non-steroidals and and back 20 30 years ago there was epidemiological work that if people who took these non-steroids had 20 30 40 50 reduction in Risk but of course the epidemiological studies are um not conclusive they just show associations and when Pfizer did the um there's their study with um siloxobic I forget how you say that but you know drugs like Advil Merck did a did a study of vioxx which eventually but when you talk about inflammation it's so complicated there's so many Pathways and maybe the non-steroidals are not the right pathway for it it's reducing one element of inflammation what Marty's doing is looking at these cells that we know cause inflammation in the Aging brain and Alzheimer's called microglia and trying to inhibit Pathways within those cells so these are like think of them as anti-inflammatories that are very specific to the brain and to Alzheimer's disease these very specific Pathways so when you've seen one anti-inflammatory you've seen one anti-inflammatory I also think one more to add from a big picture is that when the clinical trials of the non-steroidals like Motrin and Advil were done we didn't really know what we were doing we didn't have biomarkers these were done in a day when clinical trials weren't nearly as sophisticated as they are today and so the questions that Marty can ask today of his anti-inflammatory are much different than what could have been asked even possible we don't even know going back 20 years when those trials were done who the patients were that went into those trials so I think we have to kind of either do them over or forget them at this point but it's it's an it's a great question and I think that's what our foundation is doing we're looking at very specific Pathways of inflammation and trying to find drugs that might be helpful he's up here yeah and just to add that we did also a pharmacology related with the real demands of it as you said it has been not so easy to translate it to clinical trials so is what type of drug and the time window when to have it in the finger trial we have preliminary evidence that those who had higher inflammatory inflammatory profile to start with seem to get better response and we are now studying the wide range of influentary markers it's very complex like you mentioned but I think I said we are learning a lot and moving to this more Precision medicine yes sir we have a question over here it's hard to keep track of the order murder is Roe every every everybody needs a coach and put together murder is row so again I think that the good family to be congratulated in the Alzheimer's Foundation to be congratulated but let me say this that great breakthroughs are made by great coaches and one of the issues that Alzheimer's victims have is they don't have great coaches to coach them through this so you're spending all this time and there are so many different things and what I have heard from all of you is it's going to be a multiple approach five fingers plus yeah now the reality is if we do not actually have those coaches trained there's a big missing element of what you're doing and I'd like to know what you think about that how are you going to train the doctors the professionals the neurologists they need to know they need to actually have a way to analyze take your science and so therefore you don't have to go to quacks here and there because they all at the end only take a small part and do not know what you are doing which is so important so I thank you all you know I don't want to dominate but I feel very strongly about this so I'm going to answer your question um I I've been doing this over 40 years I remember when I started people when I opened my lab over at Rockefeller University down the block here um and people would ask me what do you do and I said I do research on Alzheimer's disease and they look at me cross-eyed and say old-timer's disease I never heard of that I mean literally no one ever heard of it when most of us went to graduate school or medical school I know when I went to medical school in the early 70s Alzheimer's disease was not in my textbooks okay I'd never heard of it I graduated med school in 74. I didn't the first time I heard of Alzheimer's disease was around 1980 when I happened to be well I won't go into that but what I'm saying is that the the thing you're bringing up is very important we've been trying to educate doctors for example about how you make a diagnosis and how you treat and how you manage patients with Alzheimer's disease for 40 years and I do think there's been tremendous progress in that in the care we have practice guidelines we have drugs in the market that are modestly effective but they're in some patients they're they're useful but the second thing besides education is you're right that the caregiving element of this is so burdensome uh and how do we support caregivers how do we train caregivers and that field has really come along also there's a lot of home care agencies for example that can get caregivers support for for spouses you know you have a couple where both people are 85 and the husband's taking care of a wife who's completely demented I mean this is a horror show this was my father so you know that we have we have a ways to go but at the same time time in my professional lifetime I think the world has changed dramatically in terms of clinical care early diagnosis and and so on I think you raise an important point we have a ways to go but it's really been an amazing change and one of the things I think that is important here we've talked a bit about biomarkers and I wanted to ask Mia actually because we do have blood tests on the market and we know from our biomarker research from the blood test for example of the brain scans that Alzheimer's disease we can pick it up and and recognize it in the brain or in the blood 20 years before people get symptoms so I'm wondering how you envision this new world of diagnostic tests that are on the market that our foundation has helped to bring to Market there's a blood test on the market in 49 states now the only state that doesn't have it is New York because we have a very complicated regulatory environment but you know what about this issue from the point of view of prevention if people come in when when they're 50 and they're worried because mom had it would you how do you think about getting that blood test and how would getting that blood test influence a prevention strategy very important role because it's easier of course to use them and it gives us an important thing like that this is a brain disorder there is some marker you can have it as you say there are still some doctors may think that dementia Alzheimer is part of normal aging is not still the knowledge is not yet there but we test with plus blood tests and other tests I think it gives it gets much easier to get the understanding there is something that can be used to support the diagnosis and it's there so how I see it blood tests could be an important part in the diagnosis if you have memory problems or family history you could have it maybe it's not giving the whole story but it's the first step and that can help you to see these persons should do more examinations if I have a profile that there is a risk for example amyloid or Tau maybe then starting with the lifestyle factors and then adding the new medications when I think it will surely help a lot in my my clinic in Stockholm we use the CSF the the lumbar puncture is more invasive it's not always so easy maybe for patients to think that as the first step of course blood test is is absolutely much much better so I think that can help to know if you have an increased risk and then tailor the interventions in the fields and one more thing that we are learning more about Alzheimer maybe it's not one disease maybe they are more subtypes four or five and in the future we may have more tailored interventions and treatment for these subtypes a little bit like cancer treatment is now it's not only one treatment but we are tailoring the treatments taking into account the biomarkers any other questions the audience I guess I'm going for going for exam and I'd like to know well I I guess I I don't think I don't mean to dominate but I do I use it in the clinic um so it's a company that we invested in called c2n out of Washington University in St Louis and the name of the test is called persivity a d p r e c i v i t e y a d Alzheimer's disad and a regular they're still rolling out the test to to doctors offices it requires some special tubes and so on it's pretty easy to get and for anyone who wants to get it it can be done but they're still in the phase of kind of rolling it out into the marketplace and it's not widespread use but you can get it so if you ask your doctor about it your doctor can call the company the company will come to the doctor's office and help to get the blood done okay you're a very good friend of many many years I'm just wondering the the aloe molecule what is the drug called that you are testing that you're in Phase 1b2 trial what is it called so we're in thank you for asking and um you know it's interesting you ask that question about what's the drug name yesterday where we're just having a discussion with my colleagues in new Therapeutics our company what are we actually going to call it and I'd like to call it the regen but we'll see whether that works but we don't have a drug name yet and we're in phase two clinical trials [Music] will begin recruiting for those Trials of that trial in January of 2023. so we're very optimistic and hopefully we will speed through this phase two and keep on going and we have some we have plans that have really we've thought about how we can quickly Advance development of this very safe therapy through the FDA through breakthrough status fast track status other strategies that we can use to Fast Track This to those who need it the most okay we will wait for that to come out and then to our first Speaker you had your your growth factor drug what was that name of that drug sure yeah it's a it's [Music] lm11a-31. so it's not a great an lm11a-31 and early on you know when you're designing what we call small molecule drugs here we're dealing with hundreds of them so that's how they end up with names like that and then we narrow down this is the one that emerged as the best one and so at this point like like what Robbie's talking about we need to come up with a name for we have some candidate names we're not quite there but we'll have a a better name for uh soon and then you have a drug that your focusing on well this this one drug the first one I mentioned lm11a-31 it protects neurons and their connections against amyloid and Tau and inflammation all three the bdnf drug is also protected against amyloid and Tau both well the growth factor it imitates is called bdnf brain derived neurotrophic Factor bdnf if you Google it you'll see all kinds of neat studies how it promotes what we call plasticity these synapses working and again there's another chemical name for the drug that mimics that um yeah we could give you more of the information yeah but it's interesting when my husband and I went to Dr Phillip for the first time he said the unusual he said you know 40 years ago had a person come in and he could remember the first name and then my father said my husband said Rowena and he said yes he said that was the first patient he had that started him really getting involved and interested and here my husband 45 years later he's coming in and showing how the pattern continues and we're very hopeful that something here might be able to delay and hold off because he's in very early stage and thank you Nancy for getting me to Dr Phillip and to all these other experts [Applause] let me just say it's amazing that yes Rowena was was one of the first patients who admitted to the Rockefeller hospital for research when we first started back in 1981 and I remember her very well and it was amazing to now then maybe unfortunate I guess it's announced to see her son right in in the clinic also with Alzheimer's and obviously there's a family history there but um just I'll let Robbie tell the story but one of the things we tried to do in in science is bring you know clinicians to the researchers and the researchers to the clinicians and so we invited Robbie's group at Rockefeller when Robbie was there as a as a fellow at the time into the hospital on rounds one day so that they they were doing research in a lab on Alzheimer's disease but they had never met a patient with it and so we I invited them to to rounds we went around on rounds talked about it and then Robbie I'll let you tell the rest of the story you went into Rowena's room she became your inspiration and I'll let you tell the story I'm getting a little teary because Dr ansbacher Rowena ansbacher is responsible for the Epiphany and the transformation of my career to understand why the brain develops Alzheimer's disease and um and learning that two-thirds of all people with Alzheimer's disease are women that it was as Howard said it it for me it was transformative I would spend hours speaking you know listening to Dr ansbacher describe her life as an adlerian psychologist and you know the feuds between young and Freud and Jung and Adler and and we would walk down the Esplanade of the Rockefeller University and one night I after we'd spent some time together I took her back to her hospital room I bit her good night and I closed the door and I waited 30 seconds and I knocked and entered and I said Dr ansbacher do you remember me and she was so lovely she said I'm so sorry should I and it was in that moment that I both learned about Alzheimer's and I also recognized that this was my life's journey to not only understand why the brain develops Alzheimer's but to cure it so thank you [Applause] I'm crying I can't speak but um I uh the questions I had were just answered so thank you for asking them but I wanted to thank all of you so very much for your important and Brilliant work and also Howard thank you for bringing everyone together to share all this with us who can't speak scientific ease and somehow we understand everything you're saying so thank you so much [Applause] thank you everybody it was really brilliant to listen to all of you um I'm curious to know if have if any of you have studied the difference between um Alzheimer's in families and Alzheimer's that is not familial individuals get it but it doesn't go on to the next Generation if there's been any study about that yeah yeah um well maybe I can turn it over to Frank would you like to answer it yeah so you're asking about the whether there are differences between Alzheimer's it runs in families or and versus the what's more common the later onset that doesn't necessarily run in the family um yeah The Field's been fascinating so the the Alzheimer's it runs and families we call it early onset the dementia starts in the 40s or 50s it's generally caused by one of a few genes that each child has a 50 chance of inheriting and most of those genes are related to generating amyloid in the brain so those are thought to be really be amyloid as a main cause whereas the late onset Alzheimer's which is the more common that's 98 is more of the late onset that likely has multiple genes not just one gene there they're more likely different causes contributing to the degeneration I mean amyloid might be one but then that's where we worry about Tau and inflammation vascular issues all kinds of things stress so the big difference is the later onset more common is probably multiple factors and that's why each of us are working on so many different areas right and the early onset straight genetic is thought to be amyloid because the mutations are in genes related to producing amyloid but unfortunately just recently you know big trial was done with their family in Colombia that has the you know family genetic type Alzheimer's and the trial was done using amyloid-based Therapeutics you know antibodies that clear amyloid out of the brain so people thought wow if there's one group of people this amyloid antibody treatment should work on it's his family in Colombia that have the familial kind and unfortunately it looks like not much of an effect so I you know there's always more things to try but I think again it's another message for us that these alternative approaches these non-amyloid approaches I think are going to be what we need an amyloid approach might contribute in certain people but it I think it's another sign it won't be the complete answer so that's the the difference between the genetic and the the later onset Frank could you comment on neighbor E4 as a risk factor sure um Howard's asking about apoe4 that's another Gene so there's a gene uh called apoe4 and it makes a protein that carries cholesterol around the body and that Gene comes in three different kinds type 2 type 3 and type four and those of us inherited the Type 4 at higher risk for Alzheimer's doesn't mean we'll get it we're just higher risk and so if you do a 23 and me for example you can find out which form of the apoe gene you have and I have many patients coming to my clinic where families will say should I get on 23 I mean should I find out if I have the four type four high-risk Gene I generally advise caution on that I used to run a genetic counseling clinic and I find that you have to ask well what would I do with that information how would it affect my life how will it affect my interaction actions with my significant other or my kids or my family because it can change those Dynamics and uh why would I say you can't put that Genie back in the bottle once you find out you have this high risk Gene you can't unlearn that right and so people say well if I find out I have that the high risk Gene I'll exercise more I say well maybe we should exercise anyway if we can right and so you know but or I've had patients do genetic testing and have issues with their job their their disability insurance they're all kinds or it being in the medical record so if you're you happen to be forgetful about something the kids will say okay there's it's starting right now and it might just be normal forgetfulness right so I always like to think very carefully about the genetic testing but that's the apoe4 gene so if I could just add about 20 percent of people have an aprily 14 either one or two of them about five percent of people have two of them and they're maybe 15 times the general puppy population risk for getting Alzheimer's disease and people that have the double four also tend to get it about 10 years earlier so if I see a patient who's onset of let's say Dementia or cognitive impairment to an Alzheimer's is at age 65 instead of 75 then I'm thinking about apoe um and uh I think Frank is isn't making an important point about how you manage that information once it's gotten I should say that although 20 of people have an April before 60 of people with Alzheimer's disease have an apoe4 um and so one of the things that I think that April before Gene illustrates is how hard it is to develop a drug because we've known about the apoe4 risk and the April 54 Gene for almost 30 years now it was discovered about 30 years ago and we haven't been able to drug it and all kinds of atdf is funded this it's sort of like a low-hanging fruit we know this is a major genetic risk factor um and there's a very complicated biology around it it's much more than just cholesterol and we've tried different ways with investigators to see how it could be fixed and we came across one program here in in the city at Wild Cornell Dr Ron Crystal who heads the chair he's chairman of the Department of genetics there and we said you know this is a genetic disorder we should treat it with gene therapy and what Ron is an expert in is taking proteins that people need to fix whatever disease they have and put it into a non-infectious virus and that virus makes the protein of interest and can be injected into people safely and used to treat disease and there's already FDA approved diseases using this viral Vector so what Ron did as a broad approach sort of regardless of the pathways that apoe4 affects which are many as we know from epidemiological studies that apoe 2 offsets the risk of apoe4 and so the idea was could we give apoe 2 in a virus injected into the brain the brain would make the apoe2 and in making the apoe2 it would offset the risk of apoe4 we've helped to spin out a company from Wild Cornell called lexio which is now in phase one with this gene therapy if it works it'll have a huge impact I think because it's such a strong risk factor and role in the disease and we have some preliminary data so far from The Trial that the viral Vector injected seems safe and and maybe working let me put it that way so it's really an exciting illustration of genetics and gene therapy and Alzheimer's disease and I could imagine a day where the same day if you're age 50 and we have a world where we have effective Therapeutics we can answer this question which I get all of us get all the time you know why should I bother getting a diagnosis if there's nothing you can do and I hope that one thing if you take away more than anything else and I also get very angry about this is there's a lot you can do there's a lot that needs to be done for prevention for enrolling in clinical trials for getting involved and helping us to enter this new world which is rapidly coming upon us I think where we're going to have drugs and I do think that getting an early diagnosis is critically important to this disease and so um I think I think that's my main message is that if you look at if there's something called the value of knowing in health economics what is the value of knowing about a diagnosis and there was there was a survey that was done of people in the community and kind of dispelled the myth that people don't want to know because about 85 percent of people in that survey when asked if they wanted to know about April E4 they said yes they want to know their risk factors they want to know about their genetic risk factors but but as Frank said I think it's not so much in getting the test it's managing the information once you get that test which is true for all genetic diseases it's not that different so we're really making Headway I think the question is if you have two or three does it mean you won't get Alzheimer's it doesn't mean that at all right no but but when we when we talk about your risk profile it's an important risk factor to take into consideration so for example even the persivity test that's on the market now it not only measures your beta amyloid status and your Tau status but it also it will well it will measure the town status but it also measures your 8.4 to go into an algorithm that determines your individual risk so of course okay um please metformin oh well I'll turn that over to me a bit so about the myth for me efficacy yes I mean there are a lot of studies both epidemiological experimental and even smaller clinical trials so in that metforminot those persons who are using Metformin seem to have a lower risk for dementia and Alzheimer's disease there was some data based on acne that persons with mild cognitive impairment are metformin seem to have better cognition and even better Alzheimer's biomarker profile so there are a lot of evidence that metformin is protective but it has been never tested in a larger randomized trial and that's what we want to do in the mid finger trial it's 600 persons and we want to see what's the optimal dose we don't know if it's 500 if it's 1 000 if it's 2 000 and back to the Apple e we also want to have at least 50 percent employee for carriers maybe in the future then we can know that if you have employee four you may need smaller or higher dose so I would say yes we have some evidence that metformin is good but we don't know exactly yet when to start and what's the optimal dose for the brain okay so uh everybody stay right there we're going to do one last wrap it around we're going to start with you Frank we're going to end with you Miranda and don't worry Howard you still get the last word I promise dude we'd love to ask your panelists and I think on behalf of everybody here we've heard so much today and uh hope is here and um Frank would like to start with you and again for each of the members of the panel what are you most excited about in Alzheimer's science right now yeah what am I most excited about I'm excited about the quality of the trials we're able to do because of the biomarkers I mean I think it we could more efficiently now come up with any of the new treatments any of us are developing really assess our our patients more accurately figure out who might benefit from this and then measure more quickly and with less cost if we're if we're affecting our Target so that's been a big missing thing in over the Decades of previous Alzheimer's trials we would give these new drugs and not really know if we're doing what we wanted to do other than waiting to measure the cognition which is really hard to measure in an exact way but with the new biomarkers now in a month or three months or six months whether it's an Imaging or blood biomarker we can know more quickly in a smaller trial are we affecting the mechanism or not and if we're affecting it we can prioritize that approach if we're not we're saying we missed on that let's do something you know so I think that's gonna that's accelerating the field and I'm I'm excited about that I concur with Frank um I think the big innovation and the excitement is better design trials they're being approached as actually testing clinical hypotheses and this is where the Precision medicine comes in so not only do we have the array of biomarkers to look at the the profile of the responses which is very important because it's a complex disease but also in the early trials to make a point that Cummings who is not here has made is that the early trials since we have these laser-guided type Therapeutics Precision medicine we in the early trials try to actually have a cohort of these people to find out if they do respond or not so you have a very quick go or no-go decision about the investment in the trials so I would concur um and really think more deeply around the Precision approach Precision medicine approach to alzheimer's which addf and Howard championed before Precision medicine was a term and the ability now to identify those individuals who would be appropriate for this therapeutic strategy we we know that Alzheimer's disease is a progressive degenerative disease so one size is not going to fit all and it's not going to fit all for all time right so if we know the the etiology of that disease for example was family history apoe4 driven traumatic brain injury driven type 2 diabetes driven we can now begin to really identify the right people for the right therapeutic at the right time and that's what I'm so excited about and that again great admiration for Howard and addf who understood the necessity for precision medicine decades ago and that's why there's not a single therapeutic approach that is duplicated we're all unique and I would say that hope has arrived right here right now I think I'll expand a little bit she pretty much just said what I was thinking but I think what's really exciting for me is just the diversity of the research that is out there there for decades most people were focused only on amyloid or Tau and Howard really you know piloted all of this in just expanding our approaches to try to treat it I think we've convinced ourselves that there's not one single cause of Alzheimer's disease so there's not going to be one Silver Bullet treatment and so having this really diverse wide we're at the of research out there is really advancing the field much faster than it had been in previous decades fully agreed I mean this book we heard today I'm very very excited so it's exactly the thing using the Precision medicine in randomized control trials and having this broad portfolio of different mechanisms I think that can really accelerate the development and I'm also thinking that um we'd say normally that we are 15 20 years behind cancer but with all this developed we can make it quicker I'm optimistic that if we put together all the knowledge so it can happen even in a more fast way and I think that's needed and finally the implementation someone asked that that if we could do even more there because there is so much knowledge available starting to use that even more I think would be very important I disagree with everyone no no I'm kidding I agree with everyone and I keep changing my answer because everybody can get ads on it but I think definitely Precision medicine understanding that there are subtypes of Alzheimer's disease there's so much data that we are we are developing in the lab now to understand this so we'll have more molecular targets and you know the a beta trials have maybe gotten a bad rap but I think we all agree that there's going to be combination therapies and so perhaps our Therapies in addition to some of these a beta therapies or Tower therapies is going to be in the future and what I'm personally excited about is that targeting aging is not on The Fringe anymore as Howard said it's front and center and we're doing it and so I think there's a lot of Hope power well I want to thank first the panelists and and I I hope you get just a small feeling because we have over 30 clinical trials going on so this is just six clinical trials and all of this fascinating work uh which is novel and Innovative and new drug targets having little to do with the mainstream of what Farmers doing for example is represented here today and these are all programs that started at the bench they started in the lab they're all in the clinic they're in our sweet spot which is what's called phase 2A which is what are called exploratory studies where addf can be funding can be catalytic to get things going to the next phase which are the larger phase to be in phase three studies that start getting into a regulatory Pathway to ultimately get FDA approval so we can bring these drugs to Market and because these drugs that you're hearing about today are in Phase 2A well we say that it can take 35 years to go from a basic science Discovery to a a drug and it generally takes like on average 10 to 12 to 15 years to begin a drug program and ultimately bring it to Market what you're hearing today is that these are all well along the road I mean we've been funding Robbie and Marty and Frank for 15 20 years and they've been at it I mean these are the heroes you know devoting their lives as Robbie said to and I'm sure Jerry and certainly me and Miranda but this takes a long time a lot of effort it's very risky 90 over 90 percent of drug development programs fail but every time we do an experiment we learn something and I think the great thing about where we are today again from my point of view and many of us is this is a completely different situation than we were in 30 40 years ago we I like to say that we know enough as much about how Alzheimer's disease happens today as we know about cancer and large disease and other illness chronic illnesses of Aging but because of this time shift which we haven't gotten into we're just in the phase of developing the first drugs to Market that are disease modifying and it's just gonna you're gonna see an explosion in the next few years of really amazing progress I think um so I want to thank all of you for being here today with us for joining us in this effort and um for coming to this and I want to thank the panel again um and we can't do this alone Thomas this is enormous effort and so we really appreciate you working with us and I hope you'll enjoy lunch too I hope the food is good and the company is good thank you and yeah now before anybody gets up I want to acknowledge there are two other good surprise winners that are not here Dr Michaela Gallagher and Jack Dr Jeff Cummings who had um prior commitments and couldn't be here with the goods family today I bring that up because there's a Jeff and there's a Michaela there's a boy and a girl and I just want to point out that on our team there are three boys in one two three four five girls so yeah okay so that's one at and we're damn proud of it right I also I'm standing here because Howard just brought up something that I want to make sure we point out Pioneers in these three seats who had crazy somebody said bold crazy ideas that their labs and the traditional academics were like what and Howard and the addf came along then as we drift this way we have a Next Generation who's come in who were not held back by these crazy ideas because of the Pioneers we said there's something to this biology of Aging approach I say this because I also say this to the goods family this is how science works and you've been transforming to all of this so that's one two since we keep talking about murder as role I just have to point something out as you might have seen when the fire alarms were going off our staff were running all over the place and trying to get him to turn the fire alarms off of course as they do because they're excellent but what the staff found out is that those Firearms going off because it's un week and there were dignitaries coming through the hotel so they had to put test systems on and I inquired about the dignitaries presidents Prime Ministers but it came back they said oh it's Murderers Row is in the house so we have to do all these tests right that we do terrific lunch will be served so if you just go out the back door you'll be directed to where lunch is and once again for the panel and for Howard and for the goods family thank you [Applause]

Channel: Alzheimer's Drug Discovery Foundation

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Id: OmoH3IU7x0I

Length: 88min 25sec (5305 seconds)

Published: Fri Sep 30 2022