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


Category: Road to the Cure

Making it work on less than optimal funding, the SDBC continues to astonish the world with results and scientific experiments they carry out on their ROAD TO CURE PARKINSON’S. Last month the SDBC reports:

Genotyping of LRRK2 Pups ~ Genotyping of pups bred from LRRK2 breeding pairs was conducted using polymerase chain reaction (PCR) technology to determine which of the pups acquired LRRK2 allele (forms of the same gene). Data reveals that about 50% of the pups acquired the allele. At the moment we have 36 LRRK2 pups, which will be ready for experimentation in about six months.

Genotyping of α-Synuclein Transgenic Pups ~ Experiments are underway to determine which of the pups from α-synuclein breeding pairs carry the gene. We are trying to generate 96 transgenic mice for experiments proposed in the MJFF grant. Our goal is to answer several questions as quickly as possible before α-synuclein SMART™ Molecules can be tested as a drug in patients:

What is the biodistribution of α-synuclein- SMART™ Molecules in the body?

What percentage of SMART™ Molecules ends up in the brain relative to serum concentration?

Does a higher dose result in delivering higher amounts of SMART™ Molecules across the blood-brain barrier into the central nervous system? Some of the big named institutions and foundations have told us that if we can demonstrate higher transport of SMART™ Molecules into the central nervous system with a higher dose, our discovery will classify as the best discovery of the 21st century.

Are α-Synuclein-SMART™ Molecules safe in the body?

At the time the blood-brain barrier study was conducted at UCSD, neither did we have access to all the needed Transgenic mice nor did we have an adequate supply of α-Synuclein-SMART™ Molecules. Also, at the time, our main focus was to unequivocally prove that SMART™ Molecules overcome the hurdles of the blood-brain barrier impermeability, which had paralyzed research efforts for the development of diagnostics and therapeutics for neurodegenerative disorders. Only after successfully breaching of the blood-brain barrier, did we start our fund raising campaign to take this technology from the Bench to the Bedside.

Mutant-LLRK2 SMART™ Molecule ~ Last month we reported that the SDBC has isolated and purified a small quantity of mutant LRRK2 SMART™ Molecules using a small segment of the full length mutant

LRRK2 protein. Unfortunately, the small segment of peptide failed to yield an active form of mutant LRRK2- SMART™ Molecule (antagonist) with an acceptable biological activity. Since full length protein is enormously expensive, the scientists tried a less expensive approach to isolate and affinity purify mutant LRRK2 SMART™ molecule but the isolated SMART™ Molecule did not have the requisite activity. Thus, it has become obvious that in order to isolate mutant LRRK2 antagonist, the SDBC must acquire full length protein.

α-Synuclein Project ~ Efforts are underway to generate the needed quantities of α-Synuclein SMART™ Molecules for mice studies in collaboration with Van Andel Institute and UCSD. We have obtained 4 mg of the SMART™ Molecules but need at least 16 mg more before the experiments can begin with 96 transgenic mice.

Feedback from Big Pharmaceutical Companies ~

During May–June, we had lengthy discussions with two of the largest pharmaceutical companies. One of the companies had found us of its own; we had reached out for the second one. Both of the companies advised us to answer all questions listed under paragraph 2 and get back to them. Thus, we are under the radar of these big pharmaceutical companies and may have the opportunity to partner with them in the future. Nevertheless, we know that when these companies are ready to partner with us based on the results of the questions outlined under paragraph 2 that our work will be much more valuable because conducting the science that answers these questions is a multimillion dollar endeavor. We share their excitement that we are on the right track continuing our Road to the Cure and we continue to look for financial partners to get us there sooner.

Parkinson’s Resource Organization is appreciative to have been the first Parkinson’s Organization to have been introduced to this life-changing science. This science needs funding to bring it to fruition.

Being a part of this historic, humanitarian effort requires an investment. If you have Parkinson’s it may be an investment into your life, if you don’t have Parkinson’s it may be an investment in humanity. Contact Jo Rosen who will introduce you to the right parties for making your investment possible. The only obstacle to getting to the human side of this science is funding. The sooner the funds are raised the sooner human clinical trials can begin. If complete funding had happened a year ago January this science would probably be, or have been, in human clinical trials. If funding happens a year from now, you might expect clinical trials two years from now. The sooner it’s completely funded, the sooner it will be in human clinical trials.

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Updated: August 16, 2017