Comments on Spinal Muscular Atrophy Research News: SMA breakthrough 'could have implications for a nu...

The World Stem Cell Summit taking place this week ...

2011-10-06T09:12:06.929-05:00

The World Stem Cell Summit taking place this week in Pasadena, California, and this year's keynote came from Andy Grove, who founded Intel Corporation.

Unconstrained thinking: the link between computer chips and clinical trials

In his talk on Monday, Grove had two key themes. The first related to the way in which biotechnology research is conducted, and his strong belief in the imperative of collaborative translational research programs. He even went so far as to suggest that all health research should be conducted in hospital-based research institutes, so that patient needs and clinical practice drive the innovation process. While the need for physical co-location could be debated, the focus on collaborative translational research has been a core value of entities such as Canada's Stem Cell Network for the past 10 years (it’s our belief that the rapid advances we have catalyzed in Canada flow from the establishment and support of multidisciplinary research teams that included stem cell biologists, bioengineers, and clinicians).

The second theme related to his frustration with the current regulatory approval process of multi-phase trials prior to a treatment being approved by the FDA / Health Canada and other national regulators. As Grove pointed out, the problem with this process is that it can take 5-10 years to complete, which may be beyond your life expectancy if you have one of the many devastating diseases stem cell research is targeting. Furthermore, not everybody can enlist in a trial -- the "exclusion" criteria (i.e. the necessary characteristics of patients that can be enlisted in the trial to ensure it is statistically valid and methodologically sound) can be quite narrow, leaving nowhere for patients to go (except perhaps to one of the many unregulated “clinics” -- see other blog articles). The frustration of patients is completely understandable, especially if the treatment is eventually validated, and could have helped them had they been allowed in the trial.

http://scnblog.typepad.com/scnblog/2011/10/unconstrained-thinking-the-link-between-computer-chips-and-clinical-trials-stem-cell-research.html

Systemic Antisense Injections Rescue Severe SMA Mi...

2011-10-05T15:37:50.104-05:00

Systemic Antisense Injections Rescue Severe SMA Mice

Experiments in SMA mice suggest that antisense treatment is most
effective when it reaches all body tissues, not just the central
nervous system
http://quest.mda.org/news/ASO1027-injections-help-sma-mice

Don Reed: Why we need new stem cell therapies and ...

2011-10-04T16:59:08.004-05:00

Don Reed: Why we need new stem cell therapies and how you can help

http://www.youtube.com/watch?v=pxILgNvZpMk

Oct. 4, 2011, 10:51 a.m. EDT Repligen Provides Q2...

2011-10-04T14:52:58.855-05:00

Oct. 4, 2011, 10:51 a.m. EDT
Repligen Provides Q2 FY2012 Corporate Update

Orphan CNS Drugs

We are developing RG3039 for Spinal Muscular Atrophy (SMA) and RG2833 for Friedreich's ataxia (FA) which are serious and debilitating neurodegenerative diseases typically diagnosed in childhood. SMA and FA are characterized by a defect in a single gene which results in diminished production of a key protein. During the quarter we enrolled 24 subjects in a Phase 1 clinical study of RG3039 in healthy volunteers. To date there have been no serious adverse events in any subject. We also filed an application in Italy to initiate a Phase 1 study of RG2833 in patients with Friedreich's ataxia from which we expect a response this quarter. We are currently engaged in discussions with numerous companies to explore whether a partnership would accelerate the global development and ultimately maximize the commercial potential of these programs. There are approximately 20,000 patients in the U.S. and Europe with SMA and 15,000 patients with FA worldwide, a combined market opportunity greater than $1 billion.

http://www.marketwatch.com/story/repligen-provides-q2-fy2012-corporate-update-2011-10-04?

There was also an intriguing talk by former CEO of...

2011-10-04T11:11:37.336-05:00

There was also an intriguing talk by former CEO of Intel, Andy Grove.

Grove’s central argument today was that the way we as a country handle the development of new drugs is in serious need of a transformation, a real transformation. He argued that the FDA should go back to what he called its original mission of approving new drugs that are proven safe, and leave the tests of efficacy to a process that is post-FDA.

http://www.ipscell.com/2011/10/inspiring-first-day-of-world-stem-cell-summit-my-thoughts/

Time moves fast.... Roman Reed: What all patient ...

2011-10-04T11:07:43.337-05:00

Time moves fast....

Roman Reed: What all patient advocates at the World Stem Cell Summit should know

http://www.youtube.com/watch?v=WQ9_7pkHns4

@Matt ..please explain...and if its obvious why ha...

2011-10-02T17:16:54.320-05:00

@Matt ..please explain...and if its obvious why hasn't any one moved forward to a cure with the data?

I don't want to sound unimpressed with this fi...

2011-10-01T17:29:39.234-05:00

I don't want to sound unimpressed with this finding but I could have told you this!!!! Stating the obvious is not progress??????!!!!!!!!???

Article In Regenerative Medicine Explains Potentia...

2011-09-29T14:36:52.957-05:00

Article In Regenerative Medicine Explains Potential of Stem Cell Therapies for ALS and SMA.
September 28, 2011.
Authors Eva Feldman, Nicholas Boulis, and colleagues present supporting evidence for the utilization of stem cell technology in the treatment of amyotrophic lateral sclerosis and spinal muscular atrophy in the Journal Regenerative Medicine. They also discuss the first-in-human Phase I trial currently underway examining the safety and feasibility of intraspinal stem cell injections in amyotrophic lateral sclerosis patients as a foundation for translating stem cell therapies for various neurological diseases. NeuralStem Inc. released interim data on the trial in September 2011.
http://www.fsma.org/Research/News/index.cfm?ID=5955&TYPE=1158

Patient, doctors encouraged by ALS trial (CNN) -- ...

2011-09-28T16:29:47.524-05:00

Patient, doctors encouraged by ALS trial
(CNN) -- A little more than two years ago, Ted Harada felt his left leg weakening, and he found himself quickly running out of breath. Doctors first thought he had asthma, but in May 2010 they told him he probably had ALS, or Lou Gehrig's disease.

In August 2010, doctors confirmed Harada, then 38, had the fatal disease, and he knew it was progressing.

"Every month they [doctors] could see deterioration," Harada said.

ALS patients lose muscle function in the lungs until they can no longer breathe. Most people with ALS die from respiratory failure within three to five years of first symptoms, according to the National Institute of Health. The disease causes nerves to wither and the lungs to stop working. About 10% of ALS patients live 10 years or longer.

Harada joined a clinical trial at Emory University in Atlanta, where doctors were injecting neural stem cells -- the precursors to nerve cells -- into the lower spinal cord of ALS patients.

Before the procedure, Harada walked with a cane and would get winded just by walking to the mailbox. He had to quit his job as a manager for a shredding company. He was so tired he couldn't play with his three children. He was too weak to pick up his youngest child. He couldn't even open a Ziploc bag.

Harada hoped the treatment would help, but he didn't expect it to. However, two weeks after getting the stem cell injections in March, he says he started to feel better.

"It's been nothing short of miraculous," he says. "I cannot begin to explain the difference it has made."

He hasn't touched his cane in months, he says, and his breathing has improved.

http://www.cnn.com/2011/09/28/health/early-als-trial-results-encouraging/