ROAD TO THE CURE UPDATE JANUARY 2015

"Patients may not care about detecting the pathogen … because they know they have the disease … and all they want is … to function normally.” Supported, in part, by a Michael J. Fox Foundation Grant.

As a part of human clinical trials, each drug is subjected to a bio-distribution study (a method of tracking where compounds of interest travel) to understand what happens to it after it is administered into a patient. A bio-distribution study is a study by which pharmacologists understand how the drug is distributed among various organs in the body. In the case of the SDBC drugs (SMART Molecules, SMs), the goal was also to study brain uptake in addition to uptake by various organs. Their collaborators in Canada recently successfully completed the brain uptake and biodistribution study of α-synuclein-SMART Molecule in young (4–5 month old) Parkinson’s-like transgenic (Tg) and normal non-transgenic (non-Tg) mice. A total of 72 mice were used in this study. This was a major undertaking requiring the use of large quantities of their SMART Molecule.

The results are spectacular. At 2 hours post injection, even the brain of normal, non-Tg, mice was saturated with the SMART Molecule. These experiments further reconfirm the earlier findings of a world renowned UCSD scientist that the SDBC SMART Molecules are capable of very efficiently crossing the insurmountable Blood Brain Barrier (refer to last month’s PRO Newsworthy Notes) and entering the central nervous system of animals regardless whether or not they have the neurodegenerative disease. Classical antibodies which our own bodies make or are made in animals, such as mice, have very poor brain uptake due to the presence of the blood-brain barrier (BBB), which so far had crippled the development of non-invasive diagnostic tests and therapeutics for neurodegenerative diseases. Scientific pundits theorize that the SDBC SMART Molecules cross the blood-brain barrier (BBB) of Tg mice because the BBB in animals afflicted with Alzheimer’s (AD) or Parkinson’s disease (PD) is compromised (leaky). Non-Tg mice are normal mice with an intact BBB. The Blood Brain Barrier permeability of the SDBC’s SMART Molecules in normal mice nullifies the theory of BBB permeability due to a compromised BBB. Thus, it appears that SMART Molecules are very unique, revolutionary molecules for diagnosing and treating brain disorders such as AD and PD.

Unprecedented Imaging of Parkinson’s Pathogen in the Brain of Live Mice

Live visualization of pathogenic proteins in the brain of Parkinson’s (PD) and Alzheimer’s (AD) patients has been a big problem due to the presence of the blood-brain barrier (BBB), a nature made physical barrier that allows the entrance of essential nutrients into the brain while blocking substances it senses as invaders/foreign such as viruses, bacteria, parasites, chemicals including most of the pharmaceuticals. If there is no technology to detect the pathogen (anything that causes a disease), physicians cannot monitor the effect of any therapy on the pathogen involved in causing the brain disease. Treating patients with drugs without knowing what it is doing to the cause of the disease has been an approach with no effect on modifying (halting or curing) the neuro-degenerative diseases so far. This is exactly the reason for the current status of therapy for brain diseases: no improvement in curing and/or halting the disease process and progression except providing a short-term symptomatic relief. The SDBC is determined to change the old paradigm by its revolutionary Blood Brain Barrier permeable technology.

Admittedly, patients may not care about detecting the pathogen or what its levels are because they know they have the disease and all they want is therapeutic intervention to make them function normally. The SDBC’s technology will allow diagnosis, monitoring the effect of therapy and treating the brain diseases simultaneously. This type of Point-of Care will be most beneficial to the patient because it will guide the physicians whether to increase or lower the dose or change the drug altogether without wasting months or years of time to find out if the patient is responding to the therapy.

What Does the Reproducibility of these Blood Brain Barrier Permeable Experiments in Different Hands across the Globe Mean?

The reproducibility of this BBB technology is in the hands of two reputable groups, one at UCSD and the other at Center for Molecular Imaging Sherbrook, Canada, which means that the SMART Molecule technology is robust. It is very close to developing a diagnostic and a therapeutic product for treating all kinds of brain diseases because SMART Molecules have the following unique characteristics: Ability to get into the central nervous system behind the blood-brain barrier, a barrier that has been an insurmountable barrier in the history of mankind; Real time images prove that the SMART Molecule is not only capable of getting into the central nervous system but can also grab onto the pathogen that is responsible for causing Parkinson’s disease; Binding of a SMART Molecule to a pathogen is a process that is known to neutralize the disease causing pathogen. Thus, these SMART Molecules are potentially very powerful therapeutic drugs; Since the brain signal continued to rise for 8-9 days the SMART Molecules need to be given only once a week for effective therapy.  

National and International Recognition of ICBI (SDBC) Technology

In the past 12 months, ICBI’s technology has steadily gained attention in national and international scientific circles. They were invited by and Dr. Cartier gave a symposia at the following key national and international events:

• Keystone Symposium, March, 2, 2013
• CNS Disease World Summit, San Francisco, Sept. 18-19, 2014
• Translational Imaging in AD and PD Diseases Symposium at McGill University, Montreal, September 22, 2014
• 2nd International Conference on Alzheimer’s Disease & Dementia on September 23-25, 2014
• Valencia, Spain (Dementia – 2014) Neuroimaging Symposium at the Michael J. Fox Foundation, New York, Oct 28, 2014
• Society for Neurosciences, Washington, DC, November 15, 2014

Plans for 2015-2016

The SDBC plans to test its SMART Molecules on 3 primates by June 30, 2015. Scale up production of SMART Molecules, finish all pharmacological studies, submit IND (The U.S. Food & Drug Administration’s Investigational New Drug (IND) program which gives a pharmaceutical company permission to ship an experimental drug across state lines (usually to clinical investigators) before a marketing application for the drug has been approved) in order to perform Phase-1 clinical trials by early to mid-2016.

In April, 2014, PRO proudly introduced the ICBI scientists and their incomparable history making work on the #PARKINSON’S ROAD TO THE CURE. This remarkable science continues as they help you understand the never before possible, “real time”, imaging of the proteins causing Parkinson’s in the brain of live mice. We invite you to contact us or them if you want information about supporting or investing in The Parkinson’s Road to the Cure. We would be proud to make this important introduction and we continue to salute our scientists as they forge ahead!