• "New Hope for our toughest diseases"

As the saying goes, there is good news and there is bad news.

Here is the bad news: Right now, there are an estimated 5.4 million Americans suffering from Alzheimer’s disease, one million suffering from Parkinson’s, over five million who have a disease related to a traumatic brain injury, 265,000 with spinal cord injuries, 30,000 who have ALS (Lou Gehrig’s disease), and 129,000 who have brain cancer. Over 25 million Americans also suffer from diabetes – about 8.3% of the population – and the problem is growing.

After that, it’s hard to believe there is good news, but here it is: R & D breakthroughs are happening in science and biotechnology at a remarkable pace to deal with these diseases. Biotech companies are on the front lines to find cures for life-threatening diseases. There are currently 800 new treatments being developed for cancer by biotech companies, 300 for heart disease, 200 for diabetes and 100 for Alzheimer’s, according to the Biotechnology Industry Organization.

Some companies, such as the one I represent, Neuralstem, Inc., are using state-of-the-art stem cell research to attack diseases such as ALS, brain and spinal cord injuries, and down the road, Parkinson’s and Alzheimer’s.

What we need to do now is accelerate this research and speed up the development of new cures for those in desperate need.

There is new legislation, introduced by Senator Kay Hagen (D-N.C.), to expedite development and access to life-saving medicines by the U.S. Food and Drug Administration (FDA). The Transforming and Regulatory Environment to Accelerate Access to Treatments (TREAT) Act will help unleash and accelerate the biotech promise of developing new cures.

This legislation will enable the FDA to speed up the review and approval process and encourage more investment and innovation to attack diseases that are serious or life-threatening. While not all the diseases mentioned above would be covered by this legislation, the legislation provides a much needed jump start on a new approach at the FDA.

America landed a man on the moon in less than a decade and yet it takes 10-15 years to take a new drug from discovery to treating a patient. Given the pace of scientific change, America is held back and patients are suffering because of the long time lags. Investors and private funders are reluctant to invest with the regulatory barriers placed in their way.

According to James Greenwood, president of the Biotechnology Industry Organization, “Sixty-one percent of venture capitalists now cite FDA regulatory challenges as having the highest impact on their investment decisions, and 40% expect to decrease their investment in the development of new therapies.”

The approval process for many of these treatment efforts and drugs is simply unacceptably slow. The TREAT Act will speed it up and have a positive impact on these new breakthrough technologies.  Congress needs to pass it this year.

Our company, Neuralstem, has seen encouraging signs of a treatment effect transplanting our neural stem cells in the spinal chord of ALS patients. Our trial has included 14 patients thus far. Others have implanted stem cells in the eyes and also seen encouraging early signs for treating various types of macular degeneration.

There are potentially important and specific changes championed by this legislation. Most notable among them is a new “standard” that the FDA may adopt to help speed along approval of new therapies for diseases like ALS. The FDA is encouraged to consider surrogate markers of efficacy; and to allow faster approval. For instance, if Neuralstem were to show that we can keep our patients’ breathing function strong enough to stay off of artificial breathing machines for a significant time, we might not have to wait until the patients actually die, to prove that they lived longer, to offer the therapy.

If a company such as Neuralstem demonstrates the benefit of a treatment according to the new FDA standards proposed in the TREAT Act, the therapy can be offered sooner and it can begin benefitting patients sooner. In a disease like ALS, where on average patients die around 3 years from diagnosis, time is clearly of the essence.

The FDA simply must keep pace with the safe and effective development of new, innovative, scientific breakthroughs that can literally provide hope for those with life-threatening diseases. The old, bureaucratic ways simply won’t work in the 21st century.

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• Stem Cells: Plural Paths to Harnessing Pluripotency

Neuralstem’s Dr. Karl Johe shares insights on conditions needed to be met to activate neural stem cells pharmacologically, in order to be a viable strategy for such indications as major depressive disorder, Alzheimer’s disease, schizophrenia and post-traumatic stress disorder (PTSD), with Bioworld Insight’s science editor.

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• The Pill That Could Cure Depression by Growing Your Brain

If you are depressed, or schizophrenic or have Alzheimer's, scientists say you probably have a shrunken hippocampus. The good news: a drug that just entered human trials promises to re-grow that part of the brain.

It's an entirely new approach to treating clinical depression, which is the first of several diseases scientists at biotech company Neuralstem are hoping to address with their experimental oral drug. Most antidepressants work on brain chemistry, tweaking levels of neurotransmitters including serotonin, norepinephrine, and dopamine. This is the first drug that aims to re-grow patients' atrophied brains.

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Dr. Karl Johe, Neuralstem's CEO, believes that depression is a three-headed beast that affects neurotransmitter levels, neurons, and hippocampus size. And he says their new drug could address all three. He also hopes the drug will reverse the disease to the point that patients could permanently go off the drug.

"If we can show by MRI that we've increased hippocampus volume and at the same time reversed depression symptoms for six months after patients have stopped taking the drug, then we'll have a cure."

That a too-small hippocampus causes depression and other diseases is still technically a theory in humans (though it's been demonstrated in rats and chimps). So if the drug grows hippocampus volume and thereby treats depression, we'll not only have a new treatment, but the study results would be proof that a shriveled hippocampus is at least in part the culprit.

The scientists showed first that the drug worked in the lab: They started with dishes of neural stem cells and added several compounds they thought might instigate growth. Seven showed promise, but they could only afford to develop one, so they chose NSI-189. They then tested it in mice; after taking the drug, the rodents had larger hippocampi.

Thirty-five healthy humans have now taken the drug with no ill effects, so the FDA gave the company the OK to start testing in depressed patients. They'll give the pill to 18 volunteers (six will get a placebo) in three groups, each receiving a progressively larger dose, each over 28 days. They expect this phase, which is mainly to make sure the drugs is safe, to take about six months. If all goes well they hope to proceed to phase two clinical trials later this year, which will test to determine whether the drug is both safe and effective. (After that, a final phase three trial to confirm safety and efficacy will remain before the company can market the drug.)

I couldn't help thinking about those healthy test subjects who took the drug. Will they get super brain powers? The healthy mice that received the drug did grow extra large hippocampi, the seahorse-shaped part of the brain involved with both short and longterm memory and spatial navigation. Johe isn't ruling out the possibility of souped-up brains:

"It's an exciting possibility and we'll definitely be looking out for it."

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Rockville-based Neuralstem has received approval from the Food and Drug Administration to advance to Phase Ib in its ongoing clinical trial to test its neuroregenerative compound NSI-189 for the treatment of major depressive disorder.

• Neuralstem receives FDA nod to advance MDD compound to Phase Ib

The Wall Street Journal, December 28, 2011
The US Food and Drug Administration (FDA) has given approval to Neuralstem to advance its novel neuroregenerative compound, NSI-189, to Phase Ib in its ongoing clinical trial as a treatment of major depressive disorder (MDD).