Tuesday, October 15, 2013

MODDERN Cures Act Reintroduced - ALSA.org

MODDERN Cures Act Reintroduced

Ask your Representative to support today!

Rep. Leonard Lance (R-NJ) has reintroduced the MODDERN Cures Act in the House of Representatives. MODDERN Cures (H.R. 3091) is a game-changer when it comes to the search for a treatment. The legislation provides new incentives to pursue ALS drug development and will significantly increase opportunities to find a treatment for the disease. We urge you to contact your Representatives and ask them to co-sponsor the MODDERN Cures Act TODAY!

The MODDERN Cures Act would accelerate the search for a treatment for ALS and other diseases by removing the barriers that limit medical innovation and by providing incentives to develop new treatments and diagnostic tools that can improve, prolong and, ultimately, save lives. Specifically the bill will: encourage research on treatments, which have been set aside in the lab, but hold promise for treating diseases like ALS that have unmet medical needs; remove barriers and provide incentives to develop new diagnostics; and ensure timely and appropriate reimbursement for new tests and treatments so that patients have access to the latest medical technology as soon as possible. A summary of the bill is available here: 
                                                                                       

                                                                      MODDERN CURES ACT
The ALS Association urges Congress to enact the MODDERN Cures Act (H.R. 3091) to accelerate the 
development of new diagnostic tools and a treatment for Lou Gehrig’s Disease. 

ALS is Always Fatal
No treatment: There currently is no effective treatment for ALS that stops, significantly slows or 
reverses the steady progression of the disease. There is no known cause, cure or means of 
prevention. The average life expectancy for a person with ALS is just two to five years from 
diagnosis and approximately half of all people with ALS will die within 14 months. 

Everyone at risk: ALS does not discriminate and can strike anyone at any time. However, military 
veterans are approximately twice as likely to die from ALS as the general public. 

No biomarkers: Biomarkers that predict or diagnose the disease or determine the efficacy of a 
treatment or proposed treatment do not currently exist for ALS. In fact, ALS is a diagnosis of 
exclusion, meaning that physicians reach an ALS diagnosis after first exhausting all other 
possibilities. These facts not only delay access to needed support services that can help improve care 
for people with ALS, but they also delay the development of treatments for the disease. Diagnostic 
tools like biomarkers can speed treatment development by predicting earlier in the development 
process whether and in whom a possible therapy may work. 

Barriers to Treatments, Barriers to Diagnostics
There are a number of significant barriers that have limited and discouraged the development of a 
treatment for ALS. These include:
Risk: It can take more than 15 years and cost over $1 billion to develop a new drug for ALS and the 
chances of successfully developing a new drug are small. About 99% of all compounds screened fail 
during development and never reach patients. This presents an enormous risk, and can discourage 
drug development especially in a rare neurological disease, like ALS, with a limited population.

Patents: Patent protection and the ability to make a treatment available without generic competition 
for a period of time helps to drive drug development and reduce risk. Indeed, patents enable 
manufacturers to recover part of the R&D costs that are necessary to pursue new treatments, 
including those that ultimately do not make it to clinical development. The Orphan Drug Act is an 
example of how patent protection can spur innovation. However, there are hundreds of thousands of 
potential treatments that either have lost patent protection, or have weak patents. These potential 
treatments that could save lives are sitting on the lab shelf and never will be developed simply 
because they lack patent protection. Moreover, the patent system can create disincentives to pursue 
treatments for diseases like ALS where the time it takes to develop a therapy can exceed 15 or more 
years. That’s because the patent clock starts at the beginning of the development process, leaving 
only a limited amount time after approval for a company to recoup its costs before the patent expires.

Regulatory challenges: There is no clear regulatory pathway for the approval of new diagnostics, 
making it difficult to predict risk and success and discouraging diagnostic development and 
personalized medicine.

Delays in coding and reimbursement: It can take a year or longer for a diagnostic to receive a 
reimbursement code. These delays not only discourage development of diagnostics, but also further 
delay patient access to vital diagnostics tools.
The Solution: the MODDERN Cures Act introduced in the House of Representatives by Congressman Leonard Lance (R-NJ), 

Addresses Unmet Medical Needs: Facilitates the Development of Treatments

MODDERN Cures would create a new category of drugs called “dormant therapies,” which are those 
that are deemed to have insufficient patent protection and also show promise for treating diseases like 
ALS for which there are limited or no treatments. The bill would encourage development of dormant 
therapies by providing a 15 year period of data exclusivity, which offers incentives for therapy 
development similar to those afforded by patent protection. However, unlike patents, the period of 
data exclusivity would begin at the time of FDA approval so that manufacturers are not penalized for 
pursuing treatments for diseases like ALS that can take 15 or more years to develop. 
For a rare disease like ALS, MODDERN Cures builds upon the Orphan Drug Act and opens new 
opportunities to find a treatment.

Removes Barriers for Developing New Diagnostics and Personalized Medicine
MODDERN Cures would ensure prompt coding and reimbursement for new diagnostic tests by 
defining improvements to the process for determining payment rates and developing a system for 
assigning temporary HCPCS codes to new tests until a permanent code is established. HCPCS codes 
are necessary to ensure reimbursement, coverage and patient access to new diagnostics.
The bill would encourage the development of companion diagnostics that accurately predict for 
whom a medicine will work by prolonging the period before which generics may enter the market by 
an additional 6 to 12 months.

MODDERN Cures would establish a council to develop educational tools and programs to promote a 
common understanding of the terminology and definitions related to the development and use of 
diagnostics.  In these ways, the MODDERN Cures Act will speed the development of new treatments and diagnostic tools that can improve and extend the lives of people living with Lou Gehrig’s Disease. MODDERN CURES ACT

The ALS Association urges Congress to enact the MODDERN Cures Act (H.R. 3091) to accelerate the 
development of new diagnostic tools and a treatment for Lou Gehrig’s Disease. 

Drug restores cells that are missing in MS patients

http://arstechnica.com/science/2013/10/drug-restores-cells-that-are-missing-in-ms-patients/

Multiple sclerosis (MS) is an autoimmune disease in which the immune system destroys the myelin sheaths surrounding neurons. These myelin sheaths are often likened to the insulation surrounding electrical wires; they enable neurons to transmit electrical impulses specifically and efficiently.
Cells called oligodendrocytes make myelin and do so throughout adulthood. People with MS have plenty of oligodendrocyte precursor cells (OPCs), and these cells are able to migrate to sites where myelination is required. The problem is that these precursor cells fail to mature properly into myelin producing cells, and this failure promotes disease progression.
Many current approved therapies for MS are immunosuppressants, but researchers at The Scripps Research Institute tried another tack. They screened 100,000 (or so) structurally diverse molecules to try to find some that would induce OPCs to mature. The researchers identified compounds that could make rodent OPCs mature into oligodendrocytes in a dish.
A thyroid hormone can do this, but it “has several physiological effects that make it unattractive as a therapeutic agent for MS.” Other compounds that work “have limited therapeutic potential due to off-target activities, toxicity, poor brain exposure, and/or demonstrated lack of in vivo efficacy.” But these compounds provided valuable positive controls in the experiment, showing that it was possible to pick out these useful compounds.
One of the most effective inducers of OPC maturation they found is a drug called benztropine, which is already available in oral form as an approved treatment for Parkinson’s disease. Once the researchers homed in on benztropine, they confirmed that the oligodendrocytes it had coaxed into maturity could in fact make myelin when in a dish with neurons. The team then analyzed benztropine’s activity in a mouse model of MS, finding that it diminished the clinical severity of the acute and remission phases of the disease while pretty much eliminating the relapse phase. In this model system, benztropine worked at least as well as the immunosuppressive drugs now in use to treat MS.
Benztropine isn't ideal. Its use is “associated with dose-dependent adverse neurological side effects”—cognitive changes, blurred vision, anorexia, and psychosis. So the authors decided to see if it could be used at a lower dose when combined with other therapies.
Fingolimod is an immunosuppressant that has reduced the relapse rate in relapsing-remitting multiple sclerosis by over half. But its use can lead to a dose-dependent bradycardia—it slows the heart rate. So in the researchers' mouse model, a combination of suboptimal doses of this with benztropine yielded a decrease in clinical severity. The combination of drugs was as good in terms of results as the standard therapeutic dose of Fingolimod.
Since the side effects are dose dependent, reducing the dose of each drug without sacrificing efficacy is a big deal. The authors hope that remyelination enhancers like benztropine might one day become good clinical options to treat MS, and they are now examining other hits from their initial screen to find some more.

Tuesday, October 08, 2013

Comedians In Cars Getting Coffee

This is a brilliant and funny show hosted by Jerry Seinfeld.  Each episode is only 15 minutes and worth it.  Enjoy.

http://comediansincarsgettingcoffee.com/