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July 2010 In this Issue:
 This month, the MRF achieved two very important milestones: we completed matching a $10 million challenge grant, and we were awarded our second patent! Completing the challenge grant was no small feat in an economic downturn that has seriously impacted philanthropic giving. I can only say that those who so generously donated to help us complete the match with a gift or pledge saw the value that the MRF offers not only to MS patients, but also to all patients who are waiting for new medicines. Completing this challenge brings us to a total of $42 million raised since 2004. And it couldn’t have come at a better time. The costs of validating and translating the basic science from our university labs are high—the cost per target could be as much as $1 million. So the only rate-limiting step for us to move multiple targets forward are the funds to do so. We now have an exceptional internal research and drug discovery team in place and the necessary funds to move forward in the short term. All of this while maintaining our administrative costs below 20%. I am grateful to each of you who have shown your commitment to and faith in the MRF by making a contribution and by asking others to do the same. The issuing of our second patent is also cause for celebration. It has always been part and parcel of the ARC (TM) model to protect the discoveries coming out of our university labs. Without this protection, engaging pharmaceutical industry partners would be extraordinarily difficult, if not impossible. Based on the early interest of more than one pharmaceutical company in the high-quality of our science, we are optimistic that our patent portfolio will be in great demand by those with an interest in developing new medicines for myelin repair. I see these milestones as evidence that we remain on the right track toward our first Phase 1 myelin repair clinical trial by 2014. Thank you again for making this possible. Scott Johnson Translational Medicine and Dr. Tassie Collins, MRF's Newest Team Member
Though she may be new to the MRF, Dr. Tassie Collins, the MRF’s new Director of Translational Medicine, is in no way new to translational medicine, biotechnology or the pharmaceutical industry. In fact, she brings more than 14 years of experience as a leader of drug discovery and development teams in the fields of inflammatory diseases and immune disorders, including multiple sclerosis. She has previously served as Scientific Director at Amgen and led three small molecule drug discovery teams through IND-enabling development and brought them into clinical studies. Dr. Collins brings the MRF invaluable insights into the process of translational medicine and the difficulties that lie in bringing a drug to market. She holds a Ph.D. in Immunology from Harvard University.
“It’s very exciting for me to join the MRF team as the Director of Translational Medicine,” said Dr. Collins. “Not only because the MRF has an impressive track record, with respect to the discoveries that have come out of their discovery biology labs but also because I have first-hand experience with how hard it is to implement a complex, elegant assay if all you have is a protocol from the literature. “I know in this position I will be able to help move the incredibly valuable myelin repair targets discovered in the MRF labs from academia to industry where they will hopefully find their way through the drug discovery and development process and onto the market as a therapy for MS,” continued Dr. Collins. “I am delighted to be of service to the MRF and also to all who suffer from MS.” What do we mean by translational medicine? To help readers understand the scope of Dr. Collins’ responsibilities, we have prepared the chart below that tracks the process of taking discovery biology--basic discoveries made in our funded laboratories--and ”translating” them into a therapy that can be safely used in human patients. The MRF is the only non-profit disease research organization that is funding and managing the process of translational medicine for targets identified in its own laboratories. Preparing basic scientific discoveries in this way is the fastest way this new knowledge can be developed and commercialized into new treatments. From Basic Discovery to Patient Treatments
Spotlight on Research: Role of Micro Molecule miR-219 Discovered by MRF Research Team Because of the role that oligodendrocytes (OLs) play in myelination of the central nervous system (CNS), the MRF research team at the Stanford University of Medicine decided to dive into the microscopic reasons why OLs differentiate (to change from a oligodendrocyte precursor cell into an adult cell) and myelinate. What they resurfaced with was a tiny piece of matter called miR-219. The “mi” of miR-219 stands for micro, the “R” for RNA and 219 is the number given to this specific microRNA to distinguish it from the rest of the miRNAs that are contained in the flotsam and jetsam of a cell. miRNAs are small noncoding RNA molecules and it was this piece of genetic material, specifically miR-219, that the research team, under the direction of Dr. Ben Barres, concluded would instigate OL differentiation in oligodendrocyte precursor cells (OPCs) and partially rescue the differentiation deficit caused by the loss of mature miRNA production in OLs in mice. Now, knowing that miRNA is required to promote normal OL differentiation, the research team can utilize this information as they try to develop a therapy for MS via myelin repair. By decoding the smallest parts of genes in mice, scientists will eventually figure out how to use the same code to repair damage done by diseases like MS in humans.
The picture above is a graph from the miR-219 paper published by Ben Barres and the Stanford research team. It shows how fqRT-PCR was used to determine expression levels of miR-219, -138, and -338 in acutely purified P5 or P17 neurons and astrocytes, P7 OPCs, P14 GC+MOG– immature OLs, and P14 MOG+ mature OLs (left), or in OPCs cultured for 7 DIV in indicated media: ±PDGF ±T3 (right). Error bars show ±SEM, n = 2–8 samples.
For more information you can request to read the whole paper by emailing info@myelinrepair.org or megan@myelinrepair.org. Please feel free to contact us with any questions you might have. We hope that you are as optimistic about this progress as we are here at the Myelin Repair Foundation. Fall Dates Set for Online "Idea Sourcing" With the support of a grant from the Robert Wood Johnson Foundation, this fall, the MRF will be hosting two online “idea sourcing” events designed to explore creative ways to accelerate the pace of medical research and shorten the time to market for new medicines. “The evolution of the ARC (TM) model over the last six years has taught us that there are many, many discrete places in the value chain of research and drug discovery that can be accelerated,” said MRF President Scott Johnson. “Reaching out to a broader universe-- individuals who work within the current system of medical research as well as others with a bent for problem solving in any arena--for out-of-the-box thinking is certainly in line with our values. “While we think this activity may afford us some additional strategies to enhance the ARC (TM) model," continued Johnson, “we also think it’s important to broaden the base of individuals and organizations who understand the problems faced by the industry and may have process ideas for how to improve outcomes for patients.” The MRF will use a game-style “idea sourcing” technology tool developed by the Institute for the Future in Palo Alto, CA, to engage as many as 600 individuals in online conversation threads that are expected to identify a wide range of new strategies for accelerating research. The events will take place September 16-17 and November 9-10 and are open to the public. If you are interested in registering to play the game, watch for updates in this monthly newsletter. MRF's Second Patent: A Core Component of the ARC (TM) Model This month the Myelin Repair Foundation has received news of its second US patent. The patent, entitled “Animal Models for Demyelination Disorders” was granted to Brian Popko and Wensheng Lin as the 7,754,941 patent in the United States. Our first patent, number 7,423,194, covered compositions of matter, specifically, the mouse with the integrated IFN-gamma transgene and the PERK knock-out and a cell having those features. The new patent covers methods of testing agents that modulate demyelination by using the mouse. Patents like these are a core component of MRF's Accelerated Research Collaboration (TM) model. In fact, without clear ownership of the intellectual property, pharmaceutical companies will not make the $1-plus billion investment to commercialize our targets. Patenting discoveries also represents another revenue source for additional research when a myelin repair treatment does come to market. Congratulations to Brian Popko, Wensheng Lin and all of the other MRF team members who made this happen! Past and Future Events When: July 9 If you couldn't make it to the Aspen Ideas Festival to hear Scott and the others speak, check out the video of the talk here. When: July 29-31 MRF Volunteers are Always Busy... Paul Purcell and Drive for Breakthroughs Raised Over $250,000! On July 19th, 2010, Paul Purcell and his team of dedicated MRF volunteers hosted the 4th annual golf outing called Drive for Breakthroughs. Each year with the support of his father, Phil Purcell, and MRF supporter Lester Knight, Paul, organizes the event to raise money for the MRF. This year, with teams of four registering to play on the grassy greens of the North Shore Country Club in Chicago, Illinois, Drive for Breakthroughs raised more than $250,000! Paul says "Our fourth annual event was easily our most successful and best attended yet. The support we have achieved, highlighted by the élan sponsorship, gives us tremendous momentum moving into our 5 year anniversary in 2011!”
We, at the MRF, want to give a huge thank you to all those involved with the Drive for Breakthroughs event. Without dedicated volunteers like Paul Purcell, his assistant Nicole Rudich and his team, we would never be able to reach the goals we set out when we were first founded. The money raised by Paul will push us closer to the clinical trial we hope to enter by 2014. Thank you Drive for Breakthroughs and may next year be even more successful! For more information or to donate to Drive for Breakthroughs visit their site here. |
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