Transcript Supporting the Discoveries of Tomorrow

Basic Research
Target Identification
Drug Screening
“Proof-of-Principle”
Testing in Animals
Translational Research
“Preclinical Drug
Development”
Clinical Research
Including Trials
Sanjay Bidichandani, MBBS, PhD
Vice President for Research
MDA’s Research Portfolio
Total
~300 grants
~$100 M
Annually
~100 new grants
~$40 M
•
•
•
•
Medical Advisory Committee
Scientific Advisory Committee
Translational Res Advisory Committee
Ad hoc peer-review
MDA’s Research Portfolio
Muscular Dystrophies
Duchenne
Becker
Limb-Girdle
Facioscapulohumeral
Congenital
Oculopharyngeal
Distal
Emery-Dreifuss
Myotonic dystrophy
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Spinal Muscular Atrophy
Spinal Bulbar Muscular Atrophy
Inflammatory Myopathies
Polymyositis
Dermatomyositis
Inclusion Body Myositis
Neuromuscular Junction Diseases
Myasthenia Gravis
Lambert-Eaton Syndrome
Congenital Myasthenic Syndromes
Endocrine Myopathies
Hyperthyroid Myopathy
Hyopthyroid Myopathy
Diseases of Peripheral Nerve
Charcot-Marie-Tooth Disease
Friedreich Ataxia
Dejerine-Sottas Disease
Metabolic Myopathies
Phosphorylase Deficiency
Acid Maltase Disease
Phosphofructokinase Deficiency
Debrancher Enzyme Deficiency
Mitochondrial Myopathy
Carnitine Deficiency
Carnitine Palmityl Transferase
Deficiency
Phosphoglycerate Kinase
Deficiency
Phosphoglycerate Mutase
Deficiency
Lactate Dehydrogenase Deficiency
Myoadenylate Deaminase
Deficiency
Other Myopathies
Myotonia Congenita
Paramyotonia Congenita
Central Core Disease
Nemaline Myopathy
Myotubular Myopathy
Periodic Paralysis
MDA’s Research Portfolio
Basic Research
Basic Research
Program
Drug Screening
Target Identification
Basic Research
Program
“Proof-of-Principle”
Testing in Animals
Translational Research
“Preclinical Drug
Development”
Translational / Basic
Research Program
Translational
Clinical Research
Including Trials
Translational / other
$60.8
$14.2
$3.7
$4.8
Active projects (million)
$7.2
MDA’s Research Portfolio
45
Percent of NIH Spending
40
**
35
30
25
20
15
10
5
0
FY10 / non-ARRA
MDA VENTURE PHILANTHROPY
Turning Research into Treatments
MDA’s Drug Development Program
Translational research
2 yrs
Target
ID
1yr
1.5-2yrs
Hit
ID
Lead
Optim
.
1 yr
Can
1 yr 2 yr
PreIND
P1
2-3 yrs
P2
P3
Reg
MDA
Risk
Academia
Cost
Biotech
Big Pharma
MVP projects by stage of development
9
8
No. projects
7
6
5
4
3
2
1
0
animal PoC
preclinical
Phase 1 or 2a
Stage (projects may overlap)
Phase 2
MDA’s Recent Advances In Therapy Development
Repligen
SMA
Repligen
Friedreich ataxia
Repligen
Friedreich ataxia
Askelepios
Duchenne / Becker
ReveraGen
Duchenne / Becker
Duchenne
PTC
LGMD2d
Mendell
Miller
ALS (SOD1)
PoC
PreC
I/IIa
IIb
…and that’s a good thing…
“It’s a great
time to be a
mouse with a
neuromuscular
disease!”
John Porter, Ph.D. (NIH / NINDS)
Challenges
How to safely and effectively extend
these successes to humans?
Who is going to fill the void between
biotechs funded by foundations & fed
- and - big pharma?
Smart partnership….
Finding Treatments: Smart Partnership
• Leveraging through MDA’s drug development program
• Federal govt & other non-profits
• Partnership with Pharma and Biotech companies
• Venture capitalists
Enhancing Research through
Communication & Collaboration
MDA Symposium Series - 2012
MDA Neuron Symposium
Role of glia in motor neuron degeneration in ALS
MDA Muscle Symposium
Newborn screening for Duchenne muscular dystrophy
MDA Translational Symposia
Encouraging technology transfer from academia
Gene therapy for neuromuscular disease (with AFM)
Getting a head start…
Clinics & Clinical Research Networks
MDA Clinic
MDA ALS Clinic
Duchenne research network
ALS research network
Investment in Training Researchers….
Currently = 40 Trainees (~$7 million)
Historically = 377 trainees (~ $48 million)
• Basic researchers (DG)
• Clinical researchers (CRTG)
MVP: Program Review
Zero trainees in translational projects!
Bridge to Industry (B2I)
• B2I postdoctoral fellowships
 Training future NMD translational researchers
Academia
Industry
Future Muscle Disease Researchers