Success models for curing a diseaseBy Steve Kirsch Foundations doing interesting work
ABC2 Academic approachesDana Farber suggested a comprehensive "Manhattan Project" to cure WM that focuses on the basic science. There is a good case to be made for this. However, others view it as too focused on the genetic causes and a long-term bet, rather than the fastest way to get to either a cure or an effective treatment. Resources/CompaniesNIH Chemical Genomics Center Neil Spector at Duke is looking at doing a non-profit company specializing in developing drugs for orphan diseases that the big drug companies won't touch. He's assembled a team of people who know how to do drug development quickly (within 2 years) against an identified target. He's looking for a few million in a charitable donation to launch his effort. If you have several cell lines in vitro that can be used, and there are small molecule signaling pathways that are known, then his specialty is small molecule drug development to disrupt those pathways. PeopleChris Austin of NCGC John Lazo at Drug Discovery Institute (there are 10 in the country) at the University of Pittsburgh does High Throughput Screening and many other useful things Technologies and processesInteresting medical discoveries Comments from various people (anonymous)The MMRF is by far the best drug development non profit in the space of myeloma and blood cancers. Kathy Giusti is a force of nature and has created a consortium for research with major universities who never play together normally. Kathy has Multiple Myeloma so she is literally fighting for her life through the organization. The Myelin Repair Foundation has a unique research network that accelerates publication and tech transfer in their space but which is very different from your issues. Their network is more focused still on research while MMRF is focused on drug development and collaborations with industry. As for ALS type research, while the Packard Foundation is very good, a simply outstanding and brilliant person you MUST speak to is Jamie Heywood, founder of the ALS Therapy Development Institute in Boston. Jamie founded this project after his brother got ALS in his 30's. Nobody is more creative or entrepreneurial about curing difficult diseases than Jamie. Several of the groups we have brought together in our network have consulted with him to duplicate his approach to data mining and use of information technology. He is a totally disruptive figure which is what you need. He recently stepped down as CEO after the death of his brother last December. He is involved with his brother Ben Heywood in a company called Patients Like Me which is a social networking web site that lets patients compare their course of treatment, drug regimen and outcomes with people like them. Debi Brooks at the MJ Fox Foundation is a phenomenal leader and advocate for innovative research approaches. She is no longer CEO but is still very involved and I can put you in touch with her as well. As for ABC2, they have been re-thinking their mission lately but one of their board members who works often with us and is simply one of the best minds in cancer research is Dr. David Agus from Cedars-Sinai. David has a cutting edge proteomics lab in LA and is working with Danny Hillis to develop new techniques for measuring proteomic profiles of good and bad responders to different cancer therapies. He works often with TGEN in Arizona which is the premier genomic analysis lab to determine the genetic and proteomic basis on individual's cancers and develop personalized therapies. CONCLUSION 1. First, see David Agus to deterine the absolute best doctor and institution for your personal treatment. He is more aware of what is coming down the chute than anyone I know. 2. Then Meet with Kathy Giusti to have her help guide your treatment and to learn more about her work which I believe will and should lead to you providing her support. She is the best investment out there to change the field ... 3. Meet with Jamie Heywood for his opinions on the question you have asked and to find out from him what novel approaches are available to help you manage your disease and to guide your investments toward innovative areas if you like. My 3 years experience of working with them (MJFF) has been very positive.
They run their operations extremely lean and they run their business to go out
of business, meaning that they put funds to research right away and don't spare
budgets to keep operating for following years. We are collaborating with them on
a variety of projects this year. They are a very good group. Related articles Waldenstrom's macroglobulinemia: A new approach |