Monday, March 29, 2010

Something in the water? Collaborative R & D picks up steam

At Prize4Life, we constantly (and sometimes obsessively) follow all the trends in research funding. We are pleasantly surprised by how many interesting models are emerging in response to a changing economic and social climate. Two recent blog posts (here and here) discuss some of our favorite examples. And we haven’t seen the end of it: a new article in Nature Medicine discusses how the Michael J. Fox Foundation (MJFF) is “fostering synergies” in Parkinson's research by assembling an international consortium to focus on one particular drug candidate. An excerpt:

Under the terms of the two-year $3.5 million grant design, members of the nine-group LRRK2 consortium, which includes both academic and industry partners, will be compelled to share results and collaborate with one another on an ongoing basis to help accelerate therapeutic discoveries. By working together, “every lab is not reinventing the wheel,” says Todd Sherer, MJFF's vice president of research programs.

“You don't gain much by keeping things under wraps,” says consortium member Patrick Lewis of University College London. “Actually getting different people with different viewpoints and different techniques to work together in an open fashion rapidly speeds up progress.”

The MJFF joins other pharma-biotech-foundation collaborations, including one between the Wellcome Trust and the UK Medical Research Council (read a past Prize4Life blog on that here).

Recently as well, the Global Alliance for TB Drug Development, the Critical Path Institute, and the Bill & Melinda Gates Foundation joined forces to accelerate the discovery of a cure for TB. They have called their new operation the Critical Path to TB Drug Regimens (CPTR); it will test combinations of drugs from various companies to identify new treatment regimens, an approach which Reuters calls “almost radical”. The group has engaged researchers from FDA and companies such as Johnson & Johnson, sanofi-aventis, Pfizer, AstraZeneca, GlaxoSmithKline, Bayer, Otsuka, Novartis, Sequella and Anacor Pharmaceuticals, Inc., reports FierceBiotech.

"This type of collaboration between the public and the private sector is exactly what's needed to help speed the availability of a shorter and more effective treatment for TB," said Dr. Tachi Yamada of the Bill & Melinda Gates Foundation in the FierceBiotech release.

Dr. Paul Stoffels of Johnson & Johnson echoed those sentiments in the same piece: "No single company or institution can do it alone […] Industry has to continue to focus on innovation and accelerate the discovery and development of new compounds with new mechanisms of action, and at the same time work in collaboration with regulators, non-profit organizations, and other partners to accelerate testing of new combination regimens as early as possible in development.” The piece cites the FDA’s “creative regulatory approach” of the 1990s, which was effective in accelerating treatments for the deadly virus.

The Critical Path Institute bills itself as an “independent, non-profit organization whose mission is to serve as the impartial facilitator of collaborative efforts among scientists from government, academia, patient advocacy organizations and the private sector” in order to “significantly improve public health.” Non-profit organizations are increasingly seen in these independent ‘mediator’ roles; indeed, Prize4Life’s strength is built on its lack of affiliation with a specific research agenda or disease model. Disease foundations are taking on ever more crucial--and visible--roles in collaborations.

Acceleration—and collaboration—does seem to be in the air, as the R&D industry struggles in a new economic climate, and is a phenomenon that Prize4Life has been following attentively. It is in everyone’s interest to safely accelerate research for the devastating diseases of the world. While our primary focus is, obviously, ALS, we can learn a lot from the funding and research models deployed for other diseases.

Like what you see? Leave us a comment. Have another great example of a research or funding collaboration? Email us and let us know!

Tuesday, March 16, 2010

Guest writer Elise Michael blogs on the Wellesley Mini-Marathon to End ALS

I remember meeting my great-uncle Ken, for the first and last time, when I was seven years old. My whole family met for his 70th Birthday party in a small restaurant in Westchester, New York, near where we all live. Sadly, my only real memory of my great-uncle was that he was in a wheelchair. I asked my parents why he couldn’t walk. They told me he was suffering from ALS. I did not ask further questions because I was too afraid of what this implied.

Fourteen years later, now that I am a senior at Wellesley College, the disease has much more viscerally returned to my life. On December 14th, when my boyfriend (a senior at Brandeis University) went home to Seattle for his winter break, his family broke the awful news to him that his mother had been diagnosed with ALS. This shocking, gut-wrenching, and painful discovery makes life more difficult for him everyday. The powerlessness and uncertainty of ALS is almost crushing. When he told me, I knew I had to try to do something to bring at least a small amount of control back into his life, as well as a way to find whatever meaning we both could out of this. I called my family for ideas. That was when my brother told me about his amazing and inspiring friend, Avi Kremer. As many of you probably know, Avi is the creator of Prize4Life. He was diagnosed with ALS while he was a classmate of my brother’s at Harvard Business School.

I immediately contacted Prize4Life. Everyone involved was beyond kind, generous and helpful. It was during my correspondence with them that I got the idea to organize a fun run. To be honest, my first idea was to assemble a 5k in the town of Wellesley; but after meeting with the Police Commissioner, I rapidly realized that there were just too many regulations and organizational complications to get that done before I graduated. But then I found out by chance that the Athletic Department at Wellesley College runs an annual mini-marathon. That was exactly the opportunity I needed! They agreed to set up the race with a $10 suggested donation that will go directly to Prize4Life. In addition, I found organizations all across Wellesley’s campus who were eager to donate funds, volunteer, and join the run! Everyone has been amazingly benevolent and supportive.

I want to invite you all to come run in the “Wellesley Mini-Marathon to End ALS” being held at Wellesley College on April the 10th! This is going to be a really great event that raises a lot of awareness — and hopefully a lot of money. I would love to see Wellesley, Brandeis, MIT, Harvard, Northeastern students and everyone else who supports ALS research to be there! For all you non-runners out there, don’t worry, you can definitely do it! The race is only 2.62 miles. For all of you serious runners, there is a very large trophy at stake. And for all of you, besides the wonderful reward of helping this extremely important cause, the after-party will be at Punches Alley pub on campus with free pizza for the runners, lots of baked goods, a raffle, and (for those 21 and over), lots of beer. I hope to see you all April 10th! Click here to sign up.

The message I want to leave you all with is that we can all do our part to help and be a positive factor when you rally behind a cause. I am thrilled to be associated with Prize4Life and to have helped assemble something so great and meaningful that will go significantly beyond race day.

Elise Michael

Wednesday, March 10, 2010

Techniques to accelerate the drug discovery process

We at Prize4Life are primarily concerned with accelerating translational ALS research—indeed, our organization and programs are shaped around the mantra of acceleration. ALS patients, like patients of many other diseases, have no time to spare. I want to share some other recent innovative techniques that have caught our attention for helping to propel drug discovery, an infamously difficult, expensive, and lengthy process. We are always looking for new ideas and approaches that we can adapt for ALS, so if you see something that sounds promising and fits within our framework, please shoot us an e-mail. Here’s the latest:

A recent Nature article discusses the Wellcome Trust, the UK’s largest non-governmental funder of biomedical research, which has a 5-year Seeding Drug Discovery Initiative (SDDI). Launched in October 2005, the program is designed to facilitate the development of drug-like small molecules that address unmet medical needs. “We consider projects in all disease areas, from any originating environment,” says Richard Davis, the business development manager at the Trust in the Nature article. “A project could be from an academic institution, a spin-out company or even an established pharmaceutical company because we ultimately base the decision [to grant an award] on the excellence of the science.”

Since 2005, the Trust has given out £80.4 million in 30 different grants (to 19 academic institutions and 11 companies). The support covers a variety of therapeutic areas, and company beneficiaries have primarily been small biotechs who have been struggling in the current unfavorable funding climate. Like Prize4Life, SDDI is a results-based initiative that aims for sustainability so that “projects to progress to a stage whereby there is sufficient evidence to make the project results, intellectual property and outcomes attractive to follow-on developers or investors who may be from the commercial or not-for-profit sectors.”

The Wellcome Trust and SDDI generally encourage the use of Contract Research Organizations (CRO’s) because they are efficient and cost-effective, saving the Trust from having to expend resources on infrastructure for every project they fund. This emphasis on leveraging CRO’s and outsourcing is also an important trend we have blogged on recently: see it here.

Another recent innovative translational research effort we have been following is a joint venture between three large pharmaceutical corporations. Merck, Pfizer, and Eli Lilly have announced plans to create a charity to research cancers in Asia. The new charity will develop a database of data derived from lung and gastric cancer samples, and make it available to cancer patients and scientists worldwide. "Through its work and the subsequent sharing of information, the Asian Cancer Research Group hopes to empower researchers, foster innovation and improve the prognosis and treatment of patients with cancer,’’ said Gary Gilliand of Merck Research Laboratories in an NJ.com article.

Finally, the Chronicle of Philanthropy has recently reported on the increasing number of charities who have gone into the drug-development business, lowering the risk assumed by drug companies in the treatment of rare diseases. Like Prize4Life, the Cystic Fibrosis Foundation and a number of others are increasingly collaborating with pharma and biotech to assume some of the risk of drug development, and hoping that those collaborations propel advancements that may otherwise be a long time in coming.

Let us hope that these creative approaches have the desired effect of hastening drug discovery for ALS and other diseases.