Comments on Spinal Muscular Atrophy Research News: Study offers hope to kids with fatal genetic disea...

Can't access the whole article but does this m...

2011-08-20T12:05:56.058-05:00

Can't access the whole article but does this mean the only thing holding up treatment is money for the IP?...and isn't genzyme already working on another treatment with Isis with ASO's? Yes I know for all of our benefit?????

Prolactin for spinal muscular atrophy
by Kai-Jye Lou, Staff Writer

Ottawa researchers have shown that the peptide hormone prolactin increased survival of mice with spinal muscular atrophy. But developing the findings in humans likely will require obtaining IP rights from Academia Sinica and Sanofi's Genzyme unit.

http://www.nature.com/scibx/journal/v4/n32/full/scibx.2011.896.html

August 18, 2011 04:01 PM Eastern Daylight Time AM...

2011-08-18T20:13:03.220-05:00

August 18, 2011 04:01 PM Eastern Daylight Time
AMRI Receives NIH Contract Award for the Development of Pre-Clinical Drug Candidates to Treat Diseases of the Nervous System

– Five-year Federal Contract Award Valued at Approx. $43 Million –

ALBANY, N.Y.--(BUSINESS WIRE)--AMRI (NASDAQ: AMRI) today announced it received a federal contract award from the National Institutes of Health (NIH)/National Institute of Neurological Disorders and Stroke (NINDS) to provide chemistry and other drug discovery technologies in support of NINDS’ Medicinal Chemistry for Neurotherapeutics Program (MCNP), part of the NIH Blueprint Neurotherapeutics Network. AMRI has been collaborating with NINDS since 2005 on novel treatments for spinal muscular atrophy, a degenerative neuromuscular disease.

http://tinyurl.com/3fv25v6

Not SMA but pretty interesting and might help... ...

2011-08-17T17:08:02.624-05:00

Not SMA but pretty interesting and might help...

Researchers Combine Stem Cell with Artificial Chromosome to Treat Duchenne Muscular Dystrophy in Mice

Because mesoangioblasts have the ability to cross blood vessel walls after intra-arterial delivery, the researchers then injected MyoD-expressing DYS-HAC–corrected mdx mesoangioblasts into the femoral artery of SCID/mdx mice. A few of the cells were detectable in myofibers within 12 hours, and by 36 hours there was clear evidence that the transplanted cells had uniformly dispersed in downstream muscles. Three weeks later clusters of dystrophin- and EGFP-positive fibers were evident, and skeletal muscle-specific dystrophin transcripts and protein were found at about up to 18% of the levels found in control animals.

The treated SCID/mdx mice in addition demonstrated amelioration of histological changes in skeletal muscle. “These results demonstrate the ability of corrected mdx mesoangioblasts to actively participate in muscle regeneration by crossing blood vessel walls after intra-arterial injection,” the authors write.

http://www.genengnews.com/gen-news-highlights/researchers-combine-stem-cell-with-artificial-chromosome-to-treat-duchenne-muscular-dystrophy-in/81245566/

FDA Reaps Fees in Generics Pact The idea is for ...

2011-08-14T19:27:18.043-05:00

FDA Reaps Fees in Generics Pact

The idea is for generic-drug companies to make payments to the agency when they seek product approvals. The FDA in turn would speed up its review of the drug without the need for more tax money.

Executives of generic-drug companies had said in interviews with The Wall Street Journal in February that they supported the creation of such a system, in large part to speed drug approvals that now can stretch to 2½ years.

http://online.wsj.com/article/SB10001424053111904253204576508601246628560.html

So they can bend safety rules when payments are made but not when debilitating diseases rob people of a decent life? Seems reasonable??...

I wonder how many headlines til we get relief? Th...

2011-08-14T13:32:33.817-05:00

I wonder how many headlines til we get relief?

Thursday, 11 August 2011 09:52
New data on ISIS-SMNRx at the 2011 annual SMA Conference

Isis' drug, ISIS-SMNRx is designed to modulate the splicing of the SMN2 gene to significantly increase the production of functional SMN protein. In preclinical studies, researchers observed proof-of-concept data demonstrating that an antisense drug can dramatically improve clinical features of SMA in a severe mouse model of SMA.

Restoring SMN protein in the CNS should provide significant benefit in treating SMA. Isis is currently conducting IND-enabling studies on ISIS-SMNRx and plans to begin clinical testing late in 2011 or early 2012 in patients with SMA.

http://tinyurl.com/3vbt5ge

Regrowing muscle: brave new world of modern medici...

2011-08-11T17:46:35.520-05:00

Regrowing muscle: brave new world of modern medicine

“The whole thing was gone,” Hernandez explained. “You could see the femur.”

Hernandez was one of the first to try a radical therapy to spark his body to regrow the lost tissue and function. Using what’s called extracellular matrix from a pig, his body recruited its own stem cells to regrow muscle, nerves and vessels.

“A few days after the surgery, there was some twitching, some spasming, some tingling that wasn’t there before,” he said. “And then in a few weeks, I started seeing better results in physical therapy.”

Regenerative medicine pioneer Dr. Stephen Badylak explained the biologic implant as the glue that holds the cells together, a scaffolding with a plan.

“And what the plan involves is using your own stem cells,” Badylak, of the McGowan Institute for Regenerative Medicine at the University of Pittsburgh, stated. “It recruits them to the site and then tells those cells what to do.”

http://www.kens5.com/news/Regrowing-muscle-brave-new-world-of-modern-medicine-127475558.html

...and yet we wait ?

GSK moves to develop gene therapy for ‘bubble boy ...

2011-08-08T22:20:32.287-05:00

GSK moves to develop gene therapy for ‘bubble boy disease’

Adenosine Deaminase Deficiency-Severe Combined Immune Deficiency, or ADA-SCID, is often fatal in children. London-based GSK, which has its U.S. headquarters in Research Triangle Park, North Carolina, licensed an investigational gene therapy for the disease from the San Raffaele Telethon Institute for Gene Therapy in a 2010 deal worth more than $14 million up front to the Italian institute. Now GSK has signed a two-year, $7.7 million deal with Italian biotechnology company MolMed (MILAN:MLM) for that company to develop a production process for the gene therapy.

ADA-SCID is caused by the alteration of a single gene.

http://www.medcitynews.com/2011/08/gsk-moves-to-develop-gene-therapy-for-bubble-boy-disease/print/

Other diseases seem to be digging into clinical tr...

2011-08-06T11:04:13.817-05:00

Other diseases seem to be digging into clinical trials very quickly and offering hope to patients.

Parkinson's Patients Hear From The Scientists

If work goes as planned, the money will help advance the Loring lab's research to the point where it's ready for testing in humans, at which point approval and, they hope, grants from regulators will be needed. Since the project bridges work from the research to clinical side, both are involved. Loring heads the project's research side. Melissa Houser, M.D., director of the Parkinson's Disease and Movement Disorder Center at Scripps Clinic, heads the clinical side.

The stem cells will be produced from human skin cells of patients, transformed into IPS cells, then grown into the proper kind of cells. In the case of Parkinson's patients, these will be dopamine-making neurons. If all goes well, these replacement cells will be transplanted into the patients, making the missing dopamine, and relieving or curing their symptoms.Because the transplants will be autologous, immune reaction is not expected to be a problem.

Read more: http://www.nctimes.com/article_78f4fe9a-bfbb-11e0-9b09-001cc4c002e0.html#ixzz1UGVfs6Lw

Tex Guv Perry Gets Stem Cell Treatment: what if ES...

2011-08-04T23:42:16.251-05:00

Tex Guv Perry Gets Stem Cell Treatment: what if ES cells were his only hope?

Many of us are still scratching our heads as to how the treatments can be offered at all if not FDA approved. It was interesting to read a quote from the Tribune article that suggests that the FDA is trying to regulate these procedures and clinics that offer it but that the issue is in court:

“The FDA, which is in litigation over its authority to regulate new stem cell clinics, has not approved the use of adult stem cells for anything other than bone marrow transplants”

So my question is what would Guv Perry have done if his doctors had told him that for his condition that adult stem cells would not work and that his only hope was ES cells? Perry has a track record of opposing ES cell research, but facing say death or lifelong disability, what would he have done if ES cells were his only hope?

http://www.ipscell.com/2011/08/tex-guv-perry-gets-stem-cell-treatment-what-if-es-cells-were-his-only-hope/#comments

pretty interesting....

Drug testing advancements..... This year the foun...

2011-08-01T10:35:50.404-05:00

Drug testing advancements.....

This year the foundation will spend $18 million -- a $6.4 million increase over last year -- on stem cell and neuroscience research, including grants to early-career investigators.

“This is disruptive medical research, like the discovery of penicillin,” said Eggan, standing near a row of blue hydrangea bushes at the home of Jodie and John Eastman on Lily Pond Lane.

The red-haired 2006 MacArthur Fellow is developing a stem cell bank that will allow drugs to be tested in the laboratory.

http://www.bloomberg.com/news/2011-08-01/hamptons-scene-harvard-stem-cell-researcher-eggan-courts-patricof-wien.html

For those interested in learning.... Uploaded by C...

2011-07-30T20:28:54.767-05:00

For those interested in learning....
Uploaded by CIRMTV on Jul 29, 2011
Geron's Embryonic Stem Cell Clinical Trial for Spinal Cord Injury

Dr. Jane Lebkowski, executive vice president and chief scientific officer at Geron Corporation, gave the keynote address for the 2011 CIRM Bridges Trainee Meeting. Last year, Geron began the first clinical trial in the world using human embryonic stem cells. CIRM recently approved a $25 million loan to Geron to assist in this trial. The embryonic stem cells used in the trial are being tested as a potential therapy for spinal cord injury. Lebkowski reviewed the work leading up to the trial and gave the latest update on its progress.

http://www.youtube.com/watch?v=c46ANweHGNw&feature=youtube_gdata_player

ALS but interesting none the less.. 'Inhibito...

2011-07-30T17:29:04.070-05:00

ALS but interesting none the less..

'Inhibitor' Molecule Helps ALS Mice be Stronger, Live Longer

Upon examination, the research team observed that, compared to their untreated counterparts, the TSA-treated mice:
had better survival of motor neurons (muscle-controlling nerve cells that are lost in ALS);
demonstrated less degeneration of the long nerve-cell fibers called axons, through which neurons transmit and receive signals;
experienced less muscle atrophy;
had less of the scarring that forms in the wake of motor neuron death (a process called gliosis); and
exhibited reduced numbers of ALS-associated neural "disconnects," called denervations, in neuromuscular junctions. (Neuromuscular junctions are composed of a special type of tissue through which signals between nerve cells and muscles pass.)

http://alsn.mda.org/news/inhibitor-molecule-helps-als-mice-be-stronger-live-longer

27-Jul-2011 Increasing SMN protein may be therapeu...

2011-07-29T20:48:21.139-05:00

27-Jul-2011
Increasing SMN protein may be therapeutic even after SMA disease onset

Using a new mouse model, these investigators discovered that restoring levels of SMN protein after disease onset extends lifespan and improves motor function. These results raise the possibility that an SMN-mediated treatment may be effective in patients with SMA even if administered at later stages of the disease.

http://www.smafoundation.org/news-114-increasing-smn-protein-may-be-therapeutic-even-after-sma-disease-onset.html

A case in point can be found in studies of motor n...

2011-07-29T20:46:47.018-05:00

A case in point can be found in studies of motor neuron degeneration being carried out in the laboratory of Dr. Dongxian Zhang, associate professor at Sanford-Burnham. The death of motor neurons in the spinal cord is responsible for lethal diseases such as spinal muscular atrophy and amyotropic lateral sclerosis (Lou Gehrig’s disease), neither of which is treatable or curable.

Dr. Zhang and his team eventually realized that this stretch of DNA might actually hold the recipe for a novel protein after all, and named the hypothetical protein SIP (short for Syvn-interacting protein). Their next step was to create a new mutant mouse in which only SIP was missing, leaving MNR untouched. The SIP-deficient mice once again experienced motor neuron death, which in this case could be rescued by re-introducing SIP into the mouse.

At this stage, SIP is still a protein without a known function. Dr. Zhang’s group is now carrying out experiments to determine the importance of SIP in motor neuron physiology and to understand why its loss causes motor neuron degeneration. They are finding that human SIP contains mutations that may be associated with the disease distal motor neuropathy.

http://beaker.sanfordburnham.org/?p=5762#more-5762

Are they already using this on MS? MULTIPLE SCLER...

2011-07-27T14:24:31.481-05:00

Are they already using this on MS?

MULTIPLE SCLEROSIS: NTx®-488

Scientific investigations by Dr. Samuel Weiss from the University of Calgary have characterized two potentially important therapeutic effects of prolactin on the central nervous system (“CNS”). In these published studies prolactin has been shown to act as both a neurogenic agent to increase the number of progenitor cells that mature into neurons and as an agent that promotes oligodendrocyte production and remyelination of the brain and spinal cord.

SCT was recently granted two key United States patents and one Australian patent for the use of prolactin in neurologic diseases based on the demonstrated insights into the effect of prolactin by Dr. Samuel Weiss. Moreover, the publication of those studies in high impact journals strongly support and validate the concept that prolactin may represent a potential new therapeutic platform for the treatment of white matter injury, and an impetus for a clinical program aimed at treating patients with MS.

http://www.stemcellthera.com/Technology.aspx?section=488

I'm not sure why it needs to cost $5 million o...

2011-07-27T10:08:32.377-05:00

I'm not sure why it needs to cost $5 million or need be a long wait since prenatal mothers use it?

Commentary
Of SMN in mice and men: a therapeutic opportunity

Kathryn J. Swoboda

Department of Neurology, University of Utah, Salt Lake City, Utah, USA.

Address correspondence to: Kathryn J. Swoboda, Pediatric Motor Disorders Research Program, Department of Neurology, 30 N. 1900 E. SOM3R149, Salt Lake City, Utah 84132, USA. Phone: 801.585.9717; Fax: 801.587.9346; E-mail: swoboda@genetics.utah.edu.

First published July 25, 2011

In the animal model of severe SMA, the ability of subcutaneously delivered PRL to upregulate SMN in brain and spinal cord resulted in a marked improvement in motor function, but only a modest improvement in survival (24). What makes this story potentially more compelling than that of other candidates with similar capacity for SMN upregulation is that this effect may prove more robust in humans than in mice because of the presence of a higher number of STAT5-binding sites in the human SMN2 promoter. In addition, PRL crosses the blood-brain barrier, and since recombinant PRL has been previously tested and proven safe in mothers of preterm infants to augment lactation, the immediate potential therapeutic implications are evident as we await other SMN-targeted therapies to navigate the hurdles of the clinical trials pipeline (26). As such, PRL joins a short list of candidates potentially worthy of further testing in SMA patients in the near term.

http://www.jci.org/articles/view/58752?key=ac37ad7fc13d2aa6c04e