First Stem Cell Transplant in Myelination Disorder Trial, StemCells, Inc (NASDAQ: STEM)
STEM announced its proprietary HuCNS-SC® human neural stem cells have been used to treat the first patient enrolled in its Phase I clinical trial in Pelizaeus-Merzbacher Disease (PMD), a myelination disorder that afflicts male children. The stem cells were administered at the University of California (UCSF) Children’s Hospital by direct injection into the brain of a patient with connatal PMD and the most severe form of the disease. This marks the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder, and the second clinical trial involving the use of HuCNS-SC cells in a neurodegenerative disease.
Myelin is the substance that surrounds and insulates nerve cells’ communications fibers (also known as axons). Without sufficient myelination, these fibers are unable to properly transmit nerve impulses, leading to a progressive loss of neurological function. Multiple sclerosis, transverse myelitis and certain types of cerebral palsy are more commonly known myelination disorders that also affect the central nervous system. Patients with PMD are born with a defective gene, which leads to insufficient myelin in the brain. Those with the most severe form of the disease, connatal PMD, lose the ability to walk and talk and eventually die, often before the age of 10. Currently, there are no effective treatments for PMD.
ReNeuron (LON: RENE) has been … finally … given full regulatory approval by the UK Gene Therapy Advisory Committee to initiate a stem cell trial for ischemic stroke after a 5 years approval cycle.
ASTM continues its cardiovascular clinical programs and expects to aggressively to advance these programs in 2010. Net loss was $4.575 M or $.03 per share. Total revenues were $16 K. Total costs and expenses were $4.6 M. R&D expenses increased to $3.283 M (reflecting continued expansion of clinical development activities and share based compensation expense). G&A expenses decreased to $1.316 M (due to an offset of stock compensation expense). At 12/31/09, the company had $14.7 M in cash. Post the completion of the public offering 1/10 ($12.4 M in net proceeds), ASTM now has $25.5 M in cash. The burn rate should average $1.4 M per month. Timing is everything, at this share price ($0.18 with a Mtg Cap of $31.49), the new CEO’s vision will be better articulated and focused!
Aastrom has approved a 1 for 8 reverse stock split of the common stock effective on 2/18/10. The reverse stock split should help ASTM’ shares reflect the value created through their clinical programs and might help attract more institutional ownership of the stock. The reverse stock split is intended to increase the per share trading price of ASTM’s common stock to satisfy the $1.00 minimum bid price requirement for continued listing on NASDAQ. Following the reverse stock split, ASTM expects to have approximately 28.3 M shares of common stock outstanding.
ISCO.OB is focused on using hpSC to treat severe diseases of the eye, the nervous system and the liver where cell therapy has been proven … clinically … yet is limited by the availability of safe immune-matched human cells. ISCO.OB is about to demonstrate the therapeutic applicability and potential immune rejection advantages of hpSC lines relative to other stem cell classes. Its new production facility for clinical-grade stem cell products has passed final building inspection consisting of separate suites to further develop and produce different cell types from the their human parthenogenic stem cell (hpSC) technology. These parthenogenetic stem cells have the medical advantage of allowing immune-matching to large segments of the population and the ethical advantage of not requiring the use of human embryos.
Stem cell surgery options and procedures continue to evolve at a rapid rate and companies such as Cytori Therapeutics are making preparations to serve more medical centers and cosmetic surgery facilities with stem cell orders in the oncoming years. Most recently, Cytori has received an order for a StemSource Cell Bank from Cosmetic Surgery Seishin in Japan. The order request is for the patented Cell Bank which includes a complete StemSource processing system, equipment, software, a database, and protocols needed to freeze and preserve patient’s adipose-derived stem cells. This StemSource Cell Bank allows doctors to use adult regenerative cells that are extracted directly from the patient’s adipose tissue, and use them in procedures such as stem cell breast augmentation, skin resurfacing procedures, and facial plastic surgery procedures that demand rapid regeneration of cells and skin tissue.
Access Pharma (OTC: ACCP.OB) is an emerging bio-pharmaceutical company which is focusing on the development of a late-stage, diversified oncology pipeline in addition to a treatment called MuGard that is cleared for marketing in the U.S., Europe, and other key global markets for a common side effect of some cancer treatments known as mucositis (painful sores in the mouth and GI mucosal lining).
The turmoil in the last 2-3 weeks markets makes it hard sometimes to focus GERN’s short-term share price appreciation. An expanding indication for GRNOPC1 is good news as the FDA placed a clinical hold on the early-stage trial of this compound. The outstanding warrant exchange accounting for approximately 3/4 of all warrants strengthened the balance sheet with GERN selling additional shares of common to investors at a premium for gross proceeds of $10 M, generating a 2.7 M shares lock-up provision with the new block of shares being purchased and issuance of a call option to the investors to purchase an additional $5 M of common stock. Adding to the these events, the GRN163L compound is being tested on human cells and on rodents showing very striking results; attacking not only the bulk of the tumor cells, but also the rare cancer stem cells that are believed to be responsible for most of a cancer’s growth.