MIS2017: "Top 10" Medical Innovations: 2018



the way the top 10 works is these are to be the top 10 disruptors in healthcare in 2018 so they're the clinic physicians choices for those things that will have a magnitude effect next year so the two major criteria its innovative and it will change care in a major way in the year 2018 the way it's done is a hundred and fifty to two hundred Cleveland Clinic physicians from each of the Institute's from all the Institute's are interviewed for their suggestions of the top 10 for next year in addition 20 to 30 venture capitalists are surveyed and a few people from the media from that list a list of 300 or 400 suggestions a results and those are weaned down if you will to about a hundred and fifty and then we have a double-elimination where 20 physicians will meet for about five hours each and vote on the top ten and another group of twenty will vote on their top ten and those groups are combined and the result is what you'll see here today we invite questions from the audience these are the experts who are representing the top ten they don't have unless I introduced it any financial interest in any of them we will try not to mention any names of devices but we will talk about the changes in therapies and devices I can tell you that on the innovations website our the list of the top ten for the last I think it's 13 years you'll see we missed one in that period of time of making a major change in the next year these top 10 that are selected we will look at how they do over the next three or four years I'm going to first introduce them by calling a number and saying the technology you'll then see a video about that and again I do have the iPad so feel free to ask questions number ten scalp culling reducing chemotherapy hair loss number ten scalp cooling for reducing chemotherapy induced hair loss a cancer diagnosis can be difficult enough to process without the added anxiety of hair loss but there is a new technology looking to eliminate that worry scalp cooling reduces the temperature of the scalp a few degrees immediately before during and after chemotherapy the hair loss prevention system in this study uses cooling fluid to keep the helmet and scalp cold causing cutaneous vasoconstriction also potentially resulting in reduced biochemical activity experts believe this may help reduce cellular uptake of the agents and or decrease susceptibility of the hair follicles to chemotherapy induced damage during the trial fifty point five percent of the patients were able to preserve their hair while none of the forty seven patients in the control group without the cooling cap kept their hair FDA approved in May of 2017 cooling systems are currently being rolled out to hospitals nationwide bringing comfort to more patients that need it most to discuss the top 10 number 10 is dr. Alberto Montero of the Tau sig Cancer Institute hello so I think from a lot of technologies and oncology you're focused and sort of targeted therapies but I think that this is innovative and transformational because from a patient perspective is very meaningful to not lose their hair especially I'm a breast cancer specialist so a lot of my patients that's that's potentially a big issue and what we've seen we participate in the clinical trials is that there are two major types of chemo anthracyclines and non anthracyclines the non anthracyclines the the provincial hair loss was quite substantial you know I would say more than 80% of patients didn't lose their hair with the anthracycline it was a little more challenging how effective is it in increasing and does it increase chemotherapy effectiveness because it pushes the drug towards the tumor it doesn't increase chemotherapy effectiveness because you're just constricting sort of the scalp and really that's not an area where there could be metastases there are some concern about could there be scalp metastases in patients but the this technology has been used in Europe for several years and prior studies haven't shown any sort of increased risk of metastasis in the scalp and have have people looked at cooling other areas all the other areas of the body that aren't involved in the tumor and to push meaning to get cell salvation in those areas and more effective chemotherapy then they've looked at other things for example nail changes with some chemo is a big issue so sometimes they put patients hands on ice to prevent the chemotherapy to go to the nails but that's not going to increase the effectiveness of chemo because chemo is really not targeted so floats around in the circulation and and you'd have to cut off circulation a lot of areas to to have sort of increase the concentration somewhere else thank you again please feel free to send in questions number nine number nine centralized monitoring of hospital patients alarm fatigue is a top technology hazard in hospitals nationwide the steady stream of sounds can desensitize staff to a critical situation studies indicate that nearly forty four percent of inpatient cardiac arrests were not detected appropriately in search of a solution groundbreaking technologies have been emerging technicians providing a constant i off-site use sensors and high-definition cameras to monitor pressure heart and respiratory rates pulse oximetry and more an alert is automatically generated that triggers on-site intervention a recent study analyzing a newly developed central monitoring unit reported a 93 percent survival rate of cardiopulmonary arrests along with sophisticated algorithms and new systems these results are capturing the attention of hospitals around the world increasing the potential to save lives dr. dan Cantillon of the Heart and Vascular Institute will discuss this before he does that I should tell you that we tend to be prejudiced against Cleveland Clinic inventions that is the voting favors everything outside of the Cleveland Clinic but this one was part of Dan's invention at the Cleveland Clinic and so we should disclose that conflict Dan thank you Mike it's a pleasure to be here along with such a distinguished panel and I will say that it really is a great team of people that I work with alongside of to develop this we're very proud of it because as you saw in the video more than 90% of the alarms that are generated in hospitals are not actionable in the sense that they don't provide they don't stimulate bedside caregivers to do something different and so what that does is that creates a lot of noise and then that volume of the noise the true signal which is a faint signal of that patient that requires our attention can sometimes be lost and so what we've found a way to do with this system is we found a way to hone the attention of our care givers to those patients that are in need of their of their assistance and as was described in the video statistics from the American Heart Association show that approximately one in four patients will survive a cardiac arrest in the hospital and in the circumstances when our off-site technicians were able to get out in front of those incidents by providing advance warning to our emergency response Rapid Response Teams we saw an astounding 93 percent return of spontaneous circulation which is really a big achievement in this field and we published those results last year and the Journal of the American Medical Association and we view that what we're doing right now is just really as much of an achievement as it's been it's just the beginning of what we can do to enhance our capabilities of hospital care now most people consider hospitals a very noisy place especially at night inhibiting sleep and everything else that relates to that all right are you able to turn down the noise not only the signal-to-noise ratio but the absolute you know sound noise and help people sleep better yeah that's that's a great question Mike so we're looking at as part of a comprehensive alarms management program we want to make the hospital a more quiet and restful place and so we're looking at ways because of our eyes in the sky and our off scent off center sight bunker of reducing the alarms that are occurring at the nursing units so that there are more quiet restful places bedside alarms so that the technicians are communicating directly with the nursing staff when there's a patient in need of their attention and again we're trying to get rid of a lot of that nuisance type alarming noises now we're sending more and more people home early has this been used on a home monitoring system to let you know when the patient has to come back yeah I think if you look at the the spectrum of what's happening now in remote health management leveraging technology at a distance you're starting to see that the spectrum of care from the ICU the non ICU wards and then the outpatient world is starting to blend together where we're using these technologies to really provide better care at a distance so that when people are released from the hospital we're staying connected with them so that's an area that I think you're going to see more growth so stay tuned to there so let me ask this is all in one central place where all eleven or maybe even four floor it and I don't know where Florida and and Abu Dhabi every but we're gonna get those those all those hospitals you know because we can monitor patients in Florida just as easily as we can in you know the east side of town because the technology allows us to really to be right there at the front line and one of the questions asked was is there a failure in what you do how house how many how much time are you online versus offline is there a redundancy so you never miss things well the best way of thinking about it is that it is an off-site center and the technicians are the eyes in the sky the backup sets of eyes it doesn't take the place of the bedside nursing personnel and the bedside management it's really there to serve as another layer of defense to make sure that those important events the things that are really going to be life-changing or threatening situations are caught not only in a timely fashion but upstream so that we can intervene before things get to that crisis moment where it's harder to resuscitate somebody and the question from the audience is is this does this really relate to a design flaw in the alarm systems and the devices themselves yeah we think that I would phrase that differently and say that there's a major area of opportunity in our in our in our nominal technologies that we're using on the nursing wards and have been using really without much major upgrades for the past you know 10 20 years I think those are some areas where we're gonna see more growth in this space where we have better bedside equipment as well number eight number eight enhanced recovery after surgery strict pre and post surgical routines have been considered the best ways to avoid complications but the data indicates otherwise a substantial growth in hospital readmissions has resulted in a Medicare payout of five hundred twenty eight million dollars in 2016 and there is an opioid epidemic involving two million Americans now physicians are ready to overhaul strategy research has indicated that an enhanced recovery after surgery or a Ras methodology that permits patients to eat before surgery limits opioids by prescribing alternate medications and encourages regular walking is reducing blood clots nausea infection and muscle atrophy one program with a group of 9,000 surgical patients has reported a drop in surgical complications by one-third the program has also reduced its opioid prescriptions by 21 percent and one Center has shown that patients going home within or three days of surgery has an extremely low readmission rate of two percent in 2017 collaborations were formed between surgical societies and large healthcare systems to drive funding and education for hospitals looking to implement a Ras protocols sparking a new era of surgical recovery dr. Conor Delaney who's chair of the digestive disease and surgical Institute will discuss this Conor thanks Mike well this is of personal satisfaction for me because we actually wrote the first paper on this 17 years ago at the clinic in a group of colorectal surgery patients and surgeons tend to be very traditional when they manage their patients but we were able to reset the clock a little bit and show that if you allowed people move early after surgery get up and walk around simple things eat earlier and change the way we do their fluids and analgesia after surgery we can have them recover much more quickly and so it now has just blossomed over the last it's taken a long time to get to this stage but it's just blossoming and I just came back from the American College of Surgeons and this is one of the talks of the program and discussing enhanced recovery after surgery and it provides value both by improving quality and by reducing cost so it cost less to look after patients they stay a shorter time in hospitals so we can use the resources more efficiently but they also recover more quickly they've better satisfaction after surgery and they have a lower complication rate so it's a win-win-win now the video alluded to a decrease in opiate use and obviously we have a problem with opiate addiction in the country and a lot of surgical patients get a lot of extra opiate pills what's happened relating to that with this so there's a couple of areas to mention so first it's a really exciting opportunity with enhanced recovery after surgery programs we've redefined the analgesics after surgery such that we give nerve blocks during surgery we get people on high doses of simple medications like acetaminophen and we find out that their pain response changes and they don't need the opioids so we now have up to 30% of patients who are well enough to go home the day after an intestinal resection which is used to be ten days ten years ago and many of those go home taking Tylenol only so just acetaminophen no opioids so it can hugely transform Mike where we go with analgesic you Talese a ssin and then the next phase of that is work we've been doing using pharmacogenetics to see if we can predict which patients respond best to different medications so we can personalize their analgesics after surgery so there's a huge further opportunity in the future 17 years ago you started this if I heard correctly why this year so if you look back at the English Navy they found out that lime juice was essential to prevent scurvy it took more than 50 years to bring them and make it mainstream I'm surprised we've actually done it this quickly because surgeons are probably more traditional and slower changing than than many other groups and so it's taken it's taken a long time to get enough evidence because people were worried that readmission rates might increase or complications might increase and what we're actually finding is that readmission rates if anything probably drop and medical complications around surgery drop and so the data now surrounding that are really robust and so this is the time when it's just exploding number seven number seven arsenal of targeted breast cancer therapies breast cancer claims the lives of more than forty thousand women every year but this year researchers are hoping to make a difference for breast cancer patients that are Braca one or bracket two positive there is new hope for targeted therapy already seeing success in ovarian cancer patients while first-line cancer DNA creating proteins are often dismantled by chemotherapy Braca genes have a back-up plan to repair that DNA carried out by poly adp-ribose polymerases or parks in 2017 results from clinical trials indicated breast cancer patients treated with a PARP inhibitor received three more months of progression-free survival than patients who received only chemotherapy her2 positive breast cancer impacts 20% of breast cancer patients and is often resistant to traditional treatment methods targeted therapies however are demonstrating positive results especially when combining two therapies along with chemotherapy showing long-term remissions in a handful of patients experts believe study results are pointing to an increasing survival rate and perhaps the eventual end of chemotherapy for a significant population of breast cancer patients dr. Pauline fun chain of the Towson Cancer Institute will discuss this Pauline so this is really exciting from a cancer genetics point of view we have known about BRCA it was first sequenced in 1994 1995 brca1 and 2 and since then we've known that there's been this subset of breast cancer patients or just a subset of patients who have breast cancer or ovarian cancer that we needed to treat differently so over the last few decades we've focused a lot on prevention on you know considering more intensive screening content in Civ surveillance and you know considering things like double mastectomy and oophorectomy prophylactically what this particular innovation is is really taking the molecular underpinning of a subset of disease so this is precision but this is what we define as precision oncology defining the tumor not by where it came from but by a particular melodic molecular change that can be targeted and when you identify a subset of patients for which you've found that essential essentially the driver and you can target your therapy for that then you're able to improve outcomes in this case double the response rate and actually decrease side effects and the the side effects in the study that was referred to are about 10% lower so this is release that what we where we'd like to head in terms of targeted therapy and I think it's very exciting from a cancer genetics point of view because we've always sort of had these surveillance patterns but not had drugs or classes of drugs that were made for that subset of patients it seems to me that with all this there will be many more protocols many more and will you ever have we then have enough patience to bring new drugs into trials right so this is a visit a fantastic question and very very current because the idea is that as we learn more and more about genomics and cancer we subset these patients more and more and so there's there's a it's actually kind of a double-edged sword here a sword that cuts effectively both ways we're in a disease like breast cancer we tend not to have you know breast prostate lung we tend not to have as much trouble in terms of accruing to trials but you know I do work in melanoma that's you know it's a much smaller subset and what's interesting and watching the melanoma trials progress is that you've got this sort of broad therapy immunotherapy that's sort of rocking the cancer world but you have a very limited set of patients for this sort of flagship therapy and and the idea is that there's more and more technology surrounding this and it's hard to get the patients into you know there's this slew of combination trials so the the I think as a field we're sort of thinking let's be smarter about this let's use genomics use any omics essentially to try to subset these patients so that you can effectively put patients in trials that are that will be the most effective for them have higher higher yield on outcomes and a lower incidence of side-effects and you can start selecting patients so as you subset we're trying to get smarter about putting patients into the right trials and I think that's sort of the the boon and the the quandary for clinical trials so one of the audience members asked a question triggered by one of the things you said or maybe the video said about when will chemotherapy really be eliminated you know the fact that we can even ask that question and entertain it I think is radical but in melanoma chemotherapy is pretty much eliminate and I think that as we move forward with immunotherapy precision therapy the combinations and timing them correctly choosing the right patient at the right time with the right type of therapy we are actually looking at an end I don't think I'm enough of a betting person to put a time on it though number six number six next-generation vaccine platforms the CDC estimates that vaccinations will prevent more than 21 million hospitalizations and 732 thousand deaths among children born in the last 20 years with that realization and the recent outbreaks of Ebola and Zika researchers are making changes new ways of developing shipping storing and vaccinating are being connected to stave off current and future diseases and epidemics using tobacco plants insects and nanoparticle systems companies are finding faster ways to develop influenza vaccines saving millions of dollars in development costs innovators are also perfecting the use of freeze-drying vaccines allowing shipment to areas where they are needed most thinking outside the syringe alternate methods like edible vaccines mucosa li delivered vaccines intranasal vaccines and vaccine chips are also under development and in 2018 an adhesive bandage sized patch for the flu vaccine is expected to be marketed to kids and adults alike these new platforms are in a position to keep individuals and entire nations healthier than ever you're actually I think seeing some of the problems the committee had in that a lot of the new technologies are combinations of things dr. Steve Gordon who's chair of infectious disease I think now over the respiratory Institute will discuss the vaccine platforms well thank you Michael and it's a pleasure to be here so as an infectious disease clinician I would much rather not treat and prevent and is I think people know I think vaccinations globally and historically have been the most cost effective medicine that we've had so this goes back to Jenner with the smallpox vaccination and now of course I would look at if you look at big pharmacy now vaccines used to be kind of a afterthought but now it's viewed as actually high-tech and actually high-value in most Big Pharma have big vaccine platforms in part of this I think is obviously the prevention aspect all of us grew up or at least I'm old enough where we had a lot of immunizations but we we didn't we had things missing off the plate and now we're at the point where actually we're being able to target two different populations if we look at Zika you have to have a different strategy for women in pregnant as opposed to people who may be going to those areas and non-immune and so we're getting more things on the menu the other thing I would say is we can't say the elimination word in vaccine in vaccine we can talk about disease elimination we can go back obviously to da Henderson that's Ohio's Lakewood owned and the successful smallpox eradication in veterinary medicine we've eliminated rinderpest polio still remains elusive that also has a Cleveland connection there's still three countries where there is endemic transmission but that is within reach and then of course all of you if you look around about half of you probably will not get immunized for influenza this year despite the fact anyone over six months of age should that's an opportunity in terms of delivery we can ask how many of you have been vaccinate see I think this is an intelligent crew and finally I will say as you know in talking about cancer we have anti-cancer vaccines from hell from hepatitis B vaccination to HPV not just for cervical but for human papilloma virus and so I think the field is is obviously extremely exciting we still have some challenges as I said but it's going to be extremely exciting and I think as we move forward and we have some questions that both the young and the old in our group so this is a wonderful set the first one someone knows that there's mumps recurrences at universities and they wonder how often are they going to have to get the mumps vaccine that's interesting so well you know mumps is part of the MMR mumps as many people might remember from their parents was one of the more common causes of acquired infertility in males it wasn't just the the cheeks that gets swollen there has been resurgence in terms of outbreaks most of these are being tracked to the fact that some some people come in from other countries haven't been immunized in the immunogenicity in terms of our aging population in adolescents has actually waned and that is causing a reassessment from the ACIP about booster immunization for MMR in that college or high school population and so when you say caused a re-evaluation what's the net result of that reevaluation the next the next result will be obviously outside of outbreak situations in terms of immunizations but reassessment of potentially booster adding in a booster immunization for adolescents well that comes from a question of someone who's 70 and said how many booster shots will I have to get how many realizations should I be getting lining up for well I think if you're 70 where we're targeting is mostly pertussis because a lot of grandparents handle their grand the infant babies it's not that we're worried about coughing the elderly its transmission to that infant and protecting that infant will we have no effective immunization the other thing for people getting up to my age is we talk about zoster vaccine and there'll be a new zoster vaccine that's inactivated that will probably be released in ready to use before the end of the year number five number five the emergence of distance health extending the healthcare environment to the patient's home has been a goal for decades after years of trials experiments and modest growth distance health technologies are about to become a widely accepted standard of care why now experts point to the preponderance of connectivity 80% of Americans now on smartphones and nearly 75% have broadband service innovators from many disciplines have been removing demographic logistical financial regulatory and technological barriers today more than 90 percent of health care delivery systems have or are building a telehealth program distance health technologies are also extending far beyond the common two-way video platforms over 19 million patients are projected to use remote monitoring devices that feed regular information to their doctors in 2018 platforms are also being built that enable more interaction between patients and clinicians around the world with momentum building experts believe that the emergence and acceleration of distance health technologies and services are assured in 2018 dr. John Schaefer from the ortho and Rheumatology Institute will discuss this well thanks very much Mike and it's a pleasure to be here we all remember a number of us remember that 50-plus years ago the introduction of the videophone at the 1964 World's Fair the technology has finally arrived it's here it's being applied to healthcare and now we actually have it as you saw in our hand and thanks to dr. Kant Ellen's description we're applying these technologies to many clinical situations providing medical advice and assistance to our patients at anytime and from anywhere using these distance health technologies has finally reached a critical momentum so the obvious question is I guess in relationship to Connors statement it took 50 years this has taken 50 years why do we think why do you think next year's the year that this will breakthrough you know that's a great question and if we look at the introduction of technology innovation succeeds when there's utilization utilization requires engagement engagement in turn requires awareness and technologies now many of us have our children to thank for introducing technologies to us that's how we find them communicate with them you're not going to the payphone anymore if you're in college and so we now have a mandate to remove the geographic barriers to care which are tying distance and expense and at the same time this is all about our patients and their continuum of care how do we apply these technologies we're finally at a point where we can start to do that so if we optimize the patient's health and their care and engage them and all these modalities these applications these interventions and we'd have to include a coaching in that this is where we're actually going to hit stride this year and applying them and also measuring having the metrics measuring those metrics and proving that we have the clinical the patient and the fiscal outcomes that will demonstrate the value of the technologies you're generating a whole mess of questions John so one of them is and this is what a lot of us worry about how will telehealth not overburden clinicians well that's why we have to look at the efficacy it's got to be of efficacious for the patient it has to be embedded in the continuum of care I've said for years if you have a disorganized clinician and give them technology of any sort you're gonna have a more disorganized clinician so how do we organize the continuum of care the clinicians activities so that this becomes part and parcel of what we do and how do we make everything more efficient why am i pulling back a post-op knee arthroplasty patient to come see me face to face when I can save my time their time frankly make it safer by avoiding a road traffic accident and see them on the screen and that has now become our standard and it's becoming the standard in many practices that's only one small piece we've got to go back to the beginning of the continuum for any particular episode of care and say how can we use the technologies to make it better safer more efficacious so the next question I'm going to combine two questions if I can is do you believe the distant health appointments will eliminate pre and post surgical checkups totally and the second part of that is if you take the continuum what percent of care is now done by distance health and what will it be say in five years nothing happens in a linear fashion and certainly if we want to wind back to the video phone at the World's Fair and where we've gone through the years we've seen happy things happen in exponential spurts I think we're at a critical exponential spurt now where I would dare say in 10 years 50% of our care could be delivered through remote technologies and we'll all care pre and post-op be done that way you know I think we've got to prove the patient clinical fiscal efficiencies and efficacies that will help accelerate it will go exponentially but you know if you want to quote alchek II when he was Northwest orient Airlines he was asked a similar question you know was the airline going to go away and granted we're now seeing the after 50 years the 747 is being sunsetted but I'll check I said nothing will ever replace a handshake and frankly if you're a patient nothing will ever replace that iterative conversation you have face to face which is different at this point but eventually what we have a telepresence model that could replace it I don't think we're quite there yet but I think things will come so maybe in 20 years this whole panel could be virtual number I don't know how to respond to that quite yet number four number four the unprecedented reduction of LDL cholesterol low-density lipoprotein or LDL cholesterol is known as bad cholesterol for a good reason it brings about fatty deposits that can clog arteries causing carotid artery disease or coronary heart disease the number one killer of Americans approved by the FDA in 1986 statins aimed to block the hmg-coa reductase enzyme the liver uses to make cholesterol but there is a large subgroup of patients who need more help pcsk9 inhibitors formerly in our 2015 top ten is the newest class of cholesterol-lowering drugs that block the enzyme from allowing the cleanup of LDL particles in the extracellular fluid these new drugs are lowering cholesterol to unprecedented levels and when pcsk9 is taken along with a statin LDL levels are being reduced by 75% studies are recommending lowering LDL to ultra low levels opening up the door to clinicians to treat their patients more aggressively than ever before dr. Leslie Cho of the Heart and Vascular Institute will discuss this thank you Mike it's my pleasure to be here with you today to talk about LDL reduction since we're approaching lunch hour we can maybe have some kind of an interesting conversation what do you think is the LDL level of a baby when they're born Mike I don't know my wife is a pediatrician as you know and we have a firm rule that we don't discuss medicine okay well that's so the LDL level for a newborn infant is ten ten to twenty and as you know the greatest level of mental development occurs during that time and but yet we have been very scared as clinician to drive down LDL to this very very low level we used to actually take patients off of statin therapy if their LDL became anything less than 35 but now we know what these pcsk9 try drugs that not only is it mentally safe that patients can take it without any neural cognitive deficits you can have unprecedented LDL reduction and with corresponding to that a reduction in cardiovascular events this is sort of a revolutionary thought process in cardiology also in 2017 another very interesting development is the is the small interfering RNA therapy to lower LDL these are pcsk9 inhibitors as well but they are given twice a year instead of every two weeks or every month like the pcsk9 inhibitors so we will continue to see these new class of agents these very very low LDL reduction and then finally you know everyone has always talked about is it LDL or is it inflammation that makes statins so beneficial for our patients with cardiovascular disease and we know now in the in the light of the cantos trial that it's not only LDL reduction which is what we see in pcsk9 but just a pure inflammatory reduction that we see with these new anti-inflammatory drugs that are benefiting our patients with cardiovascular so the obvious questions are will we be putting the essentially putting these in the drinking water or whatever way we can give them thin-film instead of injections whatever will we be doing that routinely that is it's used for secondary prevention as I understand it to get that LDL below 30 or whatever Willie we start to do that on everyone in over the age of 50 I think that would be you know too much these are cost prohibitive for everyone but I think what we've learned is it's not only the LDL reduction but it is the duration of that LD low LDL that benefits patients and so I think as a society we have to think about what is the acceptable LDL and how low to get it for the general population we know from lots of epidemiological studies that populations that have very very low LDL continue to have incredibly low cardiovascular event rate in general so I will L dia will statin be a over the counter drug some day most likely so the message for all of us is keep exercising keep doing more dietary and then the anti-inflammatories and add statins and now so that's everybody when will we add the the PK P so the pcsk9 currently the way there are worded is I mean I think clearly there is benefit with pcsk9 inhibitors in people with genetic cholesterol defects so people with homozygous fh or heterozygous fh in my mind there is no doubt that pcsk9 is really help what we've also learned from these pcsk9 drug is we used to think so if you are homozygous fh we used to think that your LDL receptor because of fh is familiar for anemia sorry sorry if that your LDL scepters were defective but even if you have LDL receptors that are defective they are these pcsk9 drugs which prevent LDL receptors from breaking down can still help patients with FH so it's really I think that's one of the sort of a key concept we've learned with pcsk9 inhibitors so genetic cholesterol problem patients in my mind pcs canines are very helpful people who have statin intolerance people who can't take cholesterol-lowering medicine because they have muscle aches or they have some type of joint aches or whatnot with cholesterol-lowering medicine they benefit from cholesterol these pcsk9 drug if we look at registry about five to ten percent of patients who are on statins have statin intolerance so that's a huge amount of populations that we're talking about and then finally people who can take cholesterol medicine but they're not at goal so those patients benefit from pcsk9 number thank you number three number three gene therapy for inherited retinal diseases gene therapy for rpe65 mediated inherited retinal diseases may be approved as early as 2018 examples of the rpe65 mediated inherited retinal diseases or IR DS include some forms of Leber congenital amaurosis and retinitis pigmentosa Leber congenital amaurosis causes severe vision loss in children retinitis pigmentosa results in progressive vision loss and even blindness in adults scientists and companies have spent decades perfecting the use of vectors used to deliver specific genes to specific cells phase three trial results showed improvement in functional vision between intervention and control participants at one year the FDA has awarded orphan drug status to rpe65 gene therapy this could lead to more gene therapies getting orphan drug and breakthrough status the is now clearer than it has ever been for restoring vision for patients with eye RDS and bringing gene therapy into the mainstream dr. Alexandra ratchet Sakaya from our Cole eye Institute will discuss this thank you so much and it's a pleasure to be here with you today so I think when we talk about gene therapy eyes opthamologist I'm extremely excited but it's really affecting all areas of Medicine not just their area of ophthalmology just in the past year there has been two approvals by FDA of gene therapy for some types of cancer one happened in August and one happened just a couple of days ago so we are hoping that in ophthalmology we're going to have our first gene therapy approval hopefully in the end of this year or beginning of 2018 and it's it's really important for us because what happens is we have a lot of patients who suffer from genetic conditions and at this point these patients all we can do for them is give them their diagnosis based on their genetic profile and then we see this patient slowly lose their vision and go blind and the best we can offer is low vision therapy so introduction of gene therapy will allow us to give hope to this patient to restore vision that would otherwise be lost and there as the video has mentioned there has been a phase three trial that just was published in August and based on those results we're looking forward to the approval we heard from Anna Abram I think was from the FDA that there are now 500 IMD's for gene therapy so this is going to be taking off but the question is can you help someone who's totally blind and when will the big problems like Alzheimer's be tackled well I was going to fix it tomorrow but so it is you absolutely right there are so many applications to FDA to for gene therapy and it just highlights how important it is and how important these early approvals are so we can make sure that based on the post approval studies these therapies are effective and that they continue to be safe that's what we saw in the in the face three trial now to answer your question about the patient who is completely blind can we bring their vision back the gene therapy by itself is not going to do it at this point so what the gene therapy does is let's say in this trial there's a patient who has a genetic condition so both of the genes one inherited from the mother one from the father are affected and so the patient can produce the protein that's important in the visual cycle and that's why this time they become blind so gene therapy is going to introduce the new gene that's going to allow the functional protein to be present that will allow to preserve vision and hopefully prevent progressive loss of vision it won't be able to reverse at this point that damage has been done now to talk about patients who who have lost more vision than that there's so many exciting things that we have going on in ophthalmology we have retinal implants that we actually use in patients who are completely blind from retinitis pigmentosa and some other exciting things that I think are going to be coming around in the future stem cells stem stem cell therapies and also what I'm very excited about is something called optogenetics where you take a cell in the retina that has other function and you modify it to function like the cells that are diseased so we would use ganglion cells for instance to function as photoreceptors so I think this future toptenz exactly number two number two neuromodulation to treat obstructive sleep apnea more than 21 million Americans suffer from obstructive sleep apnea and are not seeking treatment for many continuous positive airway pressure or CPAP devices provide the relief needed but for a subset of patients it brings to mind anything but rest these masks often represent noise bulk and irritation 40% of patients refuse to wear them prompting innovators to search for a less intrusive way to treat obstructive sleep apnea the result neuromodulation controlled by a remote or wearable patch these systems include a breathing sensor and a stimulation lead powered by a small battery the implant inserted during a minimally invasive surgical procedure uses stimulation to the tongue and throat to keep the airway muscles open throughout the night this technology is slated to take the sleep market by storm in 2018 bringing a better night's sleep to more patients dr. tina waters of our Sleep Disorders Center in the neurologic Institute will discuss this before we go on there is a potential conflict with a Cleveland Clinic device not yet approved but there is that potential but tell us about what's available now sure absolutely pleasure to be here I mean who here doesn't enjoy a great night's sleep I mean right that's what we all have in common and sleep is important for all disease prevention and unfortunately as the video showed you know sleep disorders are on the rise especially with obstructive sleep apnea and where a lot of our problems lies with the treatment especially those who have moderate to severe degrees of obstructive sleep apnea and CPAP is the gold standard treatment but it can be cumbersome for a lot of individuals and as a result a lot of people go without treatment and then that therefore increases their risks of having a lot of other underlying diseases present coronary artery disease drug resistant hypertension uncontrolled diabetes so it's really important to target that population and neuromodulation for obstructive sleep apnea is is an answer for some of those individuals what happens with obstructive sleep apnea is your airway muscles lose their ability to maintain the airway in an open state they kind of get floppy so to speak and the the treatment of the neural modulation is a way that you can stimulate the nerves that are controlling those floppy ear way muscles so they can stiffen up and the airway can open so that the patient can breathe appropriately and get that great night's rest will see paps ever be retired totally oh my goodness well we know that CPAP spin-around since 1981 for obstructive sleep apnea so it's not that long ago but I would still see somewhat of a role for them and have there been many complications related to the implant of the devices thus far no there's a few companies that are out there there's one currently that's only commercially available some that are on the launchpad and of those patients implanted thus far no serious complications number one number one hybrid closed-loop insulin delivery system nighttime brings about the most fear in parents of children with type 1 diabetes relinquishing all monitoring control and relying on the unpredictable pancreas millions of type 1 diabetes sufferers rely on the use of an insulin pump providing an alternative to multiple daily injections however that open loop system requires the patient to determine how much insulin the pump injects using information from a continuous glucose monitoring device or CGM now there is a revolutionary breakthrough removing the patient from the equation in 2016 the FDA approved the first hybrid closed-loop insulin delivery system being hailed as the world's first artificial pancreas the new technology enables direct communication between the CGM device and the pump making type 1 diabetes management easier than ever studies show it stabilizes blood glucose at an unprecedented level dropping a1c levels a half percent hybrid closed-loop technology is predicted to have a huge impact on the diabetes treatment market and is a big step towards developing a fully automated insulin delivery system dr. Jim Young who has many functions at the clinic but is also chair of the endocrine and metabolism Institute who's been on the panel I think all ten years or eleven years that I've done it has finally made it into the number one chair Jim why will this change everything for diabetics thanks Mike it it's nice to be top dog finally here but you know there are 30 million or so diabetics in the United States and that's about nine 10% of the population and type 1 diabetes probably another 10 percent of that so 3 million or so it's a huge problem creating macro vascular and micro vascular disease heart attacks stroke micro vascular disease kidney failure small vessel disease in the brain associated with Alzheimer's and memory plays a huge problem and trying to control diabetes is a challenge for many patients so we've had insulin pumps for several years we've had monitoring devices where patients have to prick their fingers and we've had ways to try to drive that hemoglobin a1c down a measurement of hyperglycemia we believe that tighter control will translate into better outcomes and extremely importantly is it's challenging for a patient to manage their own diabetes multiple finger pricks programming the device and so a closed system where you have a glucometer a glucose monitor a continuous glucose monitoring device that can tell eMeter data to the pump and then information that a scene in the glucose measurements can be plugged into a proprietary algorithm that can regulate how much insulin is given that any so having a closed-loop system that infuses a drug is a dramatic change we've been trying to develop closed-loop systems for many different things and this really is an example of the first and best to come on board it's not quite an artificial pancreas because the pancreas doesn't just regulate insulin it has other functions that are unrelated to diabetes but it is the closest to an artificial pancreas to managing diabetes that we have now you told me that this got approval last year but it got approval so fast from the FDA that the company wasn't ready to launch it correct a big question with these closed loop systems is safety and efficacy the sponsor and manufacturer of the device got the letter of approval on October 28 so almost exactly a year ago today and it was after just about a hundred days of evaluation by the FDA and I can tell you from having many interactions with the FDA that's unheard of and so a high bar for getting over the demonstration of efficacy and safety was met so it took some time for the sponsor to ramp up and they became commercially available just this past June so with that number of diabetics with the advantages that we have in the population that it's approved for type 1 diabetics who are 14 years of age or older and using more than eight units of insulin a day right now we're there and I think it's going to be an explosive technology that's going to get out there big and fast and you alluded to the fact that there are complications of diabetes macro and micro vascular disease heart attack strokes memory loss kidney failure amputations etc will this decrease those substantially that's our hope we still don't completely understand the pathophysiology and the biologic problems that are associated with diabetes I I'm a cardiologist so I look at diabetes as a vascular disease which drives a lot of the complications of it if our hypothesis is correct the hypothesis being getting the hemoglobin a1c down to normal levels and conclude controlling the glucose this should definitely translate into improvements in mortality and morbidity in the diabetic population so two questions that the audience is asked the whole mess of questions on a lot of your choices that I haven't been able to get to from a time standpoint this one is closed loop systems are available in electronics for a lot of years why'd it take so long and the second one related to that is how often does the device need to be refilled with insulin so the device can be refilled in insulin over a several day period of time the sensor is placed every seven days or so the device is calibrated on a once a day basis with one or two finger sticks it may be up to four which is perhaps half or a third of the number of calibrations that have to be done why did it take so darn long well fear of failure because if you deliver too much insulin or too little insulin you can create a major problem so the issue was when do we go to the moon we can't fail we're through with this panel I want to thank all of you for the questions I want to thank the panelists but I especially want to thank Shannon sure Meg Fagan Matz anchor and person we couldn't do this without Susan Burnett who and I want you to give them a thank you for helping do all the questions and answer thank the panelists as well thank you

16 Comments

  1. Pink Elephant Solutions said:

    Register medical device as per Indian regulatory body i.e. CDSCO(Central Drugs Standard Control Organization). https://morulaa.com/ CDSCO Guidelines, CDSCO Approval

    June 28, 2019
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  2. cooking expert said:

    Best Digital Health Trend News https://www.digitalhealthtrend.com/

    June 28, 2019
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  3. Kathryn Carter said:

    Gee, the ads were far more interesting than these burning old men. I'm sure they're knowledgeable, but the presentation really leaves something to be desired.

    June 28, 2019
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  4. Alina Woods said:

    Visit and get yourself registered for the healthcare conference: https://healthcare.euroscicon.com/registration

    June 28, 2019
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  5. Thomas said:

    cancer cure ??????

    June 28, 2019
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  6. Angie Absten said:

    Statins have never done any good but much harm. Like all chemical health.

    June 28, 2019
    Reply
  7. Angie Absten said:

    Vaccines are assault & sabotage not helpful. See VAXXED. truth about vaccines cancer & GMOs.

    June 28, 2019
    Reply
  8. Angie Absten said:

    BRCA is such a small percentage. Damn all chemo & chemical health.

    June 28, 2019
    Reply
  9. Angie Absten said:

    IV vitamin C for infections.

    June 28, 2019
    Reply
  10. Angie Absten said:

    Herbal intervention so much better & safer both analgesic and blood thinner non constipating Tylenol good for liver& kidney toxicity.

    June 28, 2019
    Reply
  11. Angie Absten said:

    Too many surgeries actually due to toxicity & nutritional limits. Replace what is missing no more cutting.

    June 28, 2019
    Reply
  12. Angie Absten said:

    Pharmacy & vaccines like all chemicals are disease promotion. Sorry for our children.

    June 28, 2019
    Reply
  13. Angie Absten said:

    We need vitamins & minerals lost to pollution, liquid better absorbed . NO MORE DAMN DRUGS.

    June 28, 2019
    Reply
  14. Angie Absten said:

    Chemical health is criminal due to extreme pollution toxicity with resultant malnutrition. Vaccines are producing more bacteria & virus so sabotage not helpful.

    June 28, 2019
    Reply
  15. Akeem RW Ross said:

    Mmm mmm best holla at me mmm mmm talk this Residual .

    June 28, 2019
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  16. Obaid Rashid said:

    Hopeful and informative. Much more exciting for medical students.

    June 28, 2019
    Reply

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