ROAD TO THE CURE UPDATE AUGUST 2013 · Parkinson's Resource Organization


Category: Road to the Cure

1. First in BBB Patents:  On July 11, 2013 the United States Patent and Trademark Office published the FIRST AND ONLY patent entitled Blood-Brain-Barrier Permeable Peptide Compositions.

2. Reproduction of Mutant Alpha-Synuclein Transgenic Mice:  The San Diego Biotech Company (SDBC) has acquired mutant (A53T) alpha-synuclein transgenic (Tg) mice to breed a large colony of mice.  Mutant alpha-synuclein transgenic mice are poor breeders.  Usually, fewer and unhealthy pups are born. While a normal female mouse can give birth to 7-10 healthy pups, the mutant transgenic mouse gives birth to only 3-6 pups, some of which do not survive.  In order to circumvent a breeding problem, a healthy female mouse will be put together with the male carrier.  We need this mutant strain to:

  • Test recognition of mutant alpha-synuclein by SMART™ Molecules (SMs)
  • Develop PET imaging based early diagnosis of Parkinson’s disease
  • Test therapeutic efficacy of SMs

3. Generation of Wild Type Alpha-Synuclein Transgenic Mice:  The SDBC has generated 60 wild type alpha-synuclein transgenic mice, which are now about two and a one-half months old.  These mice have to grow to the age of about 9-10 months before they will be used in PET imaging and therapeutic efficacy studies in collaboration with Van Andel Institute, Michigan.

4. Integrity of Blood-Brain Barrier (BBB) In collaboration with UCSD Radiology Department:  SDBC scientists are about to conduct an experiment to answer an important question related to the integrity of the BBB.  Is the BBB compromised in Tg mice?  It has been suggested by some scientists (from Pfizer) that the reason for BBB permeation of SDBC’s picobodies, now known as SMART™ Molecules, is that the tight junctions of the endothelial layer of cells forming the BBB is broken or compromised in Tg mice.  Intelligent this thought may seem, the possibility of compromised BBB is far-fetched to the scientists of SDBC.  The scientists feel that without the intact BBB, the interior milieu of the central nervous system (CNS) of transgenic mice (and patients) would be flooded by humoral neurotransmitters and blood elements that would cause serious injury to the CNS, resulting even in death. Nevertheless, it is important to conduct experiments to put this issue to rest whether or not the BBB is intact in Tg mice.  The results obtained may also be applicable to patients.

5. Mutant-LRRK2 Project:  We reported last month that our attempt to isolate and purify mutant LRRK2 SMART™ Molecules (SM) using a small segment of 2725 amino-acid along mutant LRRK2 protein did not produce an active SM.  By the term active SM we mean a mutant-LRRK2 picobody that inhibits the kinase activity of the LRRK2 protein.  The mutation at position, G2019S, makes LRRK2 very wild in terms of its ability for self-phosphorylation.  Phosphorylated LRRK2 overwhelms the neuron which adversely affects motor and cognitive functions.  That is why, the SDBC is still looking for about 20 mg (Estimated cost ~ $204K) of mutant LRRK2 protein to generate active mutant LRRK2 SM.

Would you or someone you know, like to have your name associated with the potential cure of Parkinson’s disease?  Just an idea! Ask about it if you decide to step up to the plate and purchase the LRRK2 protein for the SDBC.

This science needs funding to bring it to fruition.  Being a part of this historic, humanitarian effort requires an investment.  Contact Jo Rosen who will introduce you to the right parties for making your investment possible.  The sooner the funds are raised, the sooner human clinical trials can begin.

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Updated: August 16, 2017