IMPACT - BUILDING THE FUTURE OF PRECISION MEDICINE AT THE ADAMS CENTER AND GLOBALLY

Transcript

• 00:00I just wanted to,
• 00:02provide sort of a
• 00:05a few slides on
• 00:07where we are and where
• 00:08we're going to in building
• 00:09the future of precision medicine
• 00:11at the Adam Center and
• 00:13and globally.
• 00:14And I'm glad that Luke
• 00:16started us on a on
• 00:17a poetic way because I'm
• 00:19going to continue on a
• 00:20poetic way
• 00:21with,
• 00:22a quote from Eleanor Roosevelt.
• 00:24Surely, it is more intelligent
• 00:26to hope
• 00:28rather than to fear, to
• 00:29try rather than not to
• 00:31try. For one thing, we
• 00:33know beyond all doubt.
• 00:35Nothing has ever been achieved
• 00:37by the person who says
• 00:38it can't be done.
• 00:40You must do the thing
• 00:42you think you cannot do.
• 00:43And this is exactly what
• 00:45we're working on in the
• 00:46Adam Center to do the
• 00:48impossible,
• 00:49to make the impossible possible
• 00:51and develop precision medicines to
• 00:53prevent
• 00:54and slow Parkinson's disease.
• 00:57And, really, everything we do
• 00:58is for our patients, and
• 01:00I want to,
• 01:02look back to two of
• 01:03my patients at Mass General.
• 01:06John
• 01:07has had Parkinson's
• 01:09for eight years, and here
• 01:10we're looking at his cognitive
• 01:12decline
• 01:13over time.
• 01:15After eight years, he was
• 01:16still able to play hundred
• 01:17sixty five rounds of golf
• 01:19a day and play with
• 01:20his grandchildren.
• 01:22His quality of life was
• 01:24fantastic.
• 01:26On the other hand, Kevin,
• 01:28who was a molecular biologist,
• 01:31a similar,
• 01:32disease duration.
• 01:34But one day, his wife,
• 01:35a nurse came home, and
• 01:37she was looking,
• 01:38into the space, staring into
• 01:40space, reaching for things that
• 01:42were not there.
• 01:43He lost his memory. He
• 01:45lost his job. His wife
• 01:46had to give up her
• 01:48job as a nurse to
• 01:49take care of him full
• 01:50time.
• 01:52And so
• 01:54so this is exactly what
• 01:56we
• 01:57want to predict and prevent
• 01:59at the Adam Center. Turns
• 02:01out Kevin had actually two
• 02:03mutations in the GBA gene,
• 02:06and
• 02:07what we want to do
• 02:08is develop GBA
• 02:11directed therapeutics
• 02:12in this case that bring
• 02:14all the Kevins of the
• 02:15world back to the Johns',
• 02:18and to play hundred sixty
• 02:19five rounds of golf
• 02:21a year.
• 02:24So
• 02:25this pathway has been nicely
• 02:28mapped.
• 02:28We we were able to
• 02:30contribute,
• 02:31to genetics and to a
• 02:33biomarkers,
• 02:34portfolio around it,
• 02:36collaborated
• 02:37with,
• 02:38Sanofi, Pablo Sardi on on
• 02:41drug trials, and many companies
• 02:43are attacking these pathways
• 02:46to develop therapeutics,
• 02:48gene replacement,
• 02:49chaperone therapies.
• 02:51So I
• 02:52think, this will
• 02:54the future is really looking
• 02:56promising,
• 02:57for GBA directed precision trials
• 02:59and treatments in the future.
• 03:01And, yeah, and this is
• 03:03GBA,
• 03:04which affects ten percent of
• 03:05Parkinson's patient. We want to
• 03:07do this all over again
• 03:08for all
• 03:09patients with with Parkinson's disease.
• 03:12And I'm really excited that
• 03:13the Adam Center is the
• 03:15only center in the world
• 03:16for,
• 03:17precision medicine for Parkinson's
• 03:19and related
• 03:21brain diseases.
• 03:22And this is really,
• 03:23going to change,
• 03:25the way we do neurology
• 03:27and the way we treat
• 03:28Parkinson's.
• 03:29Currently, all we do is
• 03:31reactive,
• 03:31one size fits all. We
• 03:33want to reimagine health care
• 03:35to make it predictive,
• 03:38preventive, and precise.
• 03:41And,
• 03:42my wife is going to
• 03:43cringe now in in the
• 03:44audience
• 03:46because the inspiration for how
• 03:48to do this is actually
• 03:49coming from Science Fiction from
• 03:50Harry Seldon, who is the
• 03:52hero of the foundation series.
• 03:54Harry Seldon is a mathematician
• 03:56in the galactic empire,
• 03:58and he developed
• 04:00psychohistory,
• 04:01a mathematical
• 04:02algorithm
• 04:03to take centuries
• 04:05of data
• 04:06on civilizations
• 04:07and planets
• 04:09to, based on these billions
• 04:11of data points,
• 04:12predict
• 04:13the outcome of
• 04:14future civilization and make the
• 04:17minimal needed changes to prevent
• 04:19bad things from happening. And
• 04:21that's exactly what we want
• 04:22to do for Parkinson's disease,
• 04:24map all data in, in
• 04:26as many patients as we
• 04:27can longitudinally.
• 04:30And we are we have
• 04:31set up platforms
• 04:32to do this at the
• 04:33level of brain cells,
• 04:35deep cohorts, and real world
• 04:37data, and we're measuring,
• 04:39genetics, transcriptomics,
• 04:41omics across all of these
• 04:43platforms.
• 04:45And with with this
• 04:48with this, dataset
• 04:50and linked the linked biospecimens
• 04:52that we have and the
• 04:54linked
• 04:54personal stem cell avatars
• 04:57will be able to,
• 04:59identify
• 05:00disease drivers in each patients.
• 05:02For example, GBA,
• 05:04go to our freezers to
• 05:05take out the cryopreserved
• 05:07white blood cells
• 05:08and make them into personal
• 05:10stem cells for clinical trials
• 05:12in a dish, work with
• 05:13pharmaceutical
• 05:14companies as well as across
• 05:16Yale to develop precision
• 05:18medicines,
• 05:19and then recontact
• 05:21the patients
• 05:22to,
• 05:23do smart proof of concept,
• 05:26clinical trials right here in
• 05:28the movement disorders clinic with
• 05:29doctor Vives and and other
• 05:31colleagues.
• 05:32And, so
• 05:34we are,
• 05:37our goal is or we're
• 05:38inviting we want all
• 05:41scientists, physicians, and engineers
• 05:43across Yale
• 05:45to, work together
• 05:47with a shared passion to
• 05:48solve Parkinson's disease.
• 05:50And to do this, we
• 05:52are,
• 05:54setting up,
• 05:56six platforms
• 05:58to,
• 05:59leverage the ingenuity of academic
• 06:02labs and combine it with
• 06:04the efficiency
• 06:05of a a biotech
• 06:06drug development
• 06:09platform to, bring
• 06:11new targets and new drugs
• 06:14to early,
• 06:15stage clinical trials.
• 06:17We
• 06:18already have a genomics platform,
• 06:21at work.
• 06:22We have,
• 06:23the beginnings of an AI
• 06:25platform. We're still,
• 06:27recruiting on building this app
• 06:28to use AI to predict
• 06:31the disease,
• 06:32to develop better drugs, to
• 06:34do drug repurposing,
• 06:36and to,
• 06:38prognosticate.
• 06:40We are,
• 06:42we are glad that Don
• 06:44Hui,
• 06:45Lin joined us. He's a
• 06:47specialist in antisense oligonucleate,
• 06:50therapeutics. Are you here, Donohue?
• 06:52There you are. From the
• 06:53Peron Institute,
• 06:54and he's working on ASOs
• 06:56for Parkinson's genes.
• 06:58And we're working with,
• 07:01Don Gellert and Kevin Hartshitz
• 07:04on medicinal chemistry of some
• 07:06of the repurposed drugs, but
• 07:07this is also one of
• 07:08the goals. So the light
• 07:10blue are the platforms that
• 07:11where we have sort of
• 07:12a a seed, but we're
• 07:13hoping to build it up
• 07:15over the years.
• 07:19The fourth platform,
• 07:21personal drug tiles with stem
• 07:23cells,
• 07:24in a dish and with
• 07:26fluclay
• 07:27on on a chip.
• 07:29The Yale Harvard biomarker study
• 07:32where we have more than
• 07:33four hundred thousand biosamples,
• 07:35four four thousand patients enrolled,
• 07:37thousand currently being enrolled by
• 07:39twenty six,
• 07:41that allow to link data
• 07:43to biosamples
• 07:45and to patients.
• 07:46And,
• 07:48most importantly,
• 07:49we want
• 07:51to take with great urgency,
• 07:54compounds, new drugs into early
• 07:57stage proof of concept trials,
• 07:59working with
• 08:01clinical division of movement disorders
• 08:03and and Jesse Sederbaum.
• 08:06And so we are thrilled
• 08:08to have so many labs
• 08:10working,
• 08:12in the center within walls
• 08:14and
• 08:14and without walls
• 08:16and with wonderful,
• 08:18collaborations
• 08:19developing with, departments of neuroscience,
• 08:22neurology,
• 08:22bits,
• 08:24radiology,
• 08:25and and genetics.
• 08:28And
• 08:29Yale
• 08:30has a powerful ecosystem
• 08:32for neuroscience
• 08:34that we can leverage to
• 08:36make this happen.
• 08:38Over the last years,
• 08:40Yale was, I think, the
• 08:42only,
• 08:44place in the world that
• 08:45got four ASAP,
• 08:47grants with altogether fifty four
• 08:49million dollars,
• 08:51from the ASAP Foundation. So
• 08:52thank you ASAP Foundation. That's
• 08:55really great. And we also,
• 08:58last year, were awarded the
• 09:00APDA Center
• 09:01for Advanced Research,
• 09:04which is is really,
• 09:06a very
• 09:07great honor.
• 09:09Yale
• 09:11was leading
• 09:12the path to bring the
• 09:14first disease modifying treatment for
• 09:17Alzheimer's disease to FDA approval,
• 09:20and has a very deep
• 09:22bench of genetics
• 09:24and,
• 09:25medical genetics.
• 09:26So we are looking forward
• 09:29to
• 09:30to work with with our
• 09:32friends across the campus.
• 09:34And so with that,
• 09:36I'm looking forward to working
• 09:38with you to build a
• 09:39better future for our patient.