ROAD TO THE CURE UPDATE AUGUST 2014Category: Road to the Cure
One of the main reasons why there are no diagnostics and curative therapies for neurodegenerative disorders such as Parkinson’s disease (PD) is the inability of most drugs (~98% of all drugs) to gain access to the central nervous system (CNS). A physical barrier, known as the blood-brain barrier (BBB), denies entry of a majority of drugs into the brain. Small molecule drugs such as Levodopa that can reach the CNS do not cure or modify the disease. These drugs merely provide short-term symptomatic relief and stop working after sometime. Macromolecule drugs such as antibodies have the potential to cure or modify the brain disorders but they have very poor permeability across the BBB into the CNS. The reported value for the brain uptake of classical antibodies is 0.1%. That is why, in spite of annual research and clinical expenditure in excess of an estimated $40 Billion, classical antibodies have not been found useful to treat brain diseases. These classical antibodies have been used by pharmaceutical companies for clinical trial on Alzheimer’s patients, which unfortunately resulted in a colossal failure during the last couple of years, wiping out multi-billion dollars’ worth of investments in Alzheimer’s projects.
To enhance chances of success, thinking out-of-the-box, ICBI has followed an unconventional, approach right from the beginning when the Company was founded. It has developed a very unique class of antibody mimics comprised of the BBB permeable properties of small organic molecules and the exquisite specificity of classical antibodies. These novel antibody mimics, referred to as SMART Molecules (SMs), have demonstrated BBB uptake in animal models that is 150 times higher than that of classical antibodies. The figure 1 compares the BBB uptake of ICBI’s SMs with the conventional antibodies still being used by the big pharmaceutical companies.
To the best of our knowledge, we have not seen any published data on the BBB uptake of conventional antibodies that is as efficient as shown by ICBI’s SMART Molecules. Whether it is big name universities such as Harvard, Stanford or a big foundation such as Michael J Fox Foundation or a big pharmaceutical company, such exquisite BBB permeable kinetics have not been reported in the literature. It is possible that such data is present somewhere in someone’s files or we may have overlooked it in the literature. However, through normal computer search, ICBI’s scientists have not found such data. Therefore, it is probably an accurate statement that no one has technology as good as ICBI’s technology is for diagnosing and treating neurodegenerative disorders such as Parkinson’s disease.
Figure 1: Blue bar represents the percentage brain uptake of ICBI’s a-Syn-SM in Parkinson’s mouse, red bar in normal mouse. The green bar is the brain uptake reported for classical antibodies in Alzheimer’s mouse [Bard, F, et al, Nature Medicine 6 (8): 916-9 (2000)]
Transition from the Bench to the Bedside:
In order to take its technology to the needy patients, ICBI has to scale up their production, which requires additional staff, equipment and laboratory space. ICBI is looking for support from the community, foundations and institutions to acquire resources for setting up manufacturing facility for large scale production of its therapeutic drugs to finish preclinical studies to file an IND application with the US FDA for approval to begin human trials.