Tuesday, 12 February 2013

ISCC reports positive preclinical results on its stem cell treatment for Criggler-Najjar syndrome

International Stem Cell Corporation (ISCC), a California based biomedical company, just announced via a press release positive preclinical results on their patented stem cell treatment for Criggler-Najjar syndrome type 1 (CN1). The study was conducted on a CN1 rodent model and it showed that the company's patented stem cell therapy is both efficient and safe. The press release fails to properly cite the study, so we redistribute the company's claims with caution.

Criggler-Najjar syndrome type 1, is an extremely rare, inherited disorder in which the patients lack the UGT1A1 enzyme. This enzyme is responsible for metabolising bilirubin, a chemical produced by the breakdown of red blood cells. It is estimated that the condition affects one individual per 1.200.000 live births.

Ball-and-stick model of the bilirubin molecule 

The syndrome results in unconjugated hyperbilirubinemia, a disorder characterised by severe neurological complications, which when left untreated, can lead to irreversible acute encephalopathy. The press release explains that alloegenic hepatocyte cell transplantation has shown in the past, promising results for the treatment of the condition, adding that further research and thus progress on this treatment is very difficult.

"One of the major factors limiting the clinical advancement of human HT is a shortage of mature, functioning human hepatocytes as well as the limited repopulation capacity of grafted adult cells." A segment from the press release

According to ISCC, their patented treatment based on hepatocyte-like cells (HLCs) presents many advantages over the previously mentioned therapy. First, the HLCs can be cultured in vitro in large numbers and secondly there is strong evidence indicating that the HLC transplantation may have better long-term results.

The press release states that the pre-clinical study showed that the HLC implantation resulted in a substantial decrease and long-term stabilisation of bilirubin levels in the blood serum of the rodents. The HLCs used in the study were derived from human parthenogenetic stem cells (hpSC), a type of stem cell which in turn is derived from unfertilised human eggs.

ISCC promotional video

Dr. Ruslan Semechkin, the company's vice president, says that the next step is to request approval from the FDA to commence phase 1 clinical trials.

You can read the official press release is available here

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  • The company had also announced promising preclinical results for Parkinson's just a few days ago. Click here to read more

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