Monday, April 26, 2010

“Benefit Corporations”

It’s a well-known fact that most non-profits struggle with finding sufficient funding, and that most for-profit businesses are penalized for considering anything but their bottom line in the development of business strategy. Now Maryland, leading the way in what may become a national effort, has created a new legal structure for an entity that is not-quite-business, not-quite-charity. Signed into law by Governor O’Malley, the bill requires that the newly named benefit corporations create a positive impact on society. Decisions made by these entities must take into consideration potential impact on employees, localities, and the environment, rather than profit only. The law also offers legal protections to its Board members for considering social and environmental issues in decision-making processes (a similar bill recently passed in Vermont’s senate).

"For the first time, we have a market-based solution supporting investors and entrepreneurs who want to make money and make a difference," Andrew Kassoy says in an article from the Chronicle of Philanthropy. Kassoy’s non-profit, B Lab, administers a certification program for socially responsible businesses.

“The new law tackles a major concern of entrepreneurs who need to raise money to expand their social-purpose businesses but fear losing control of the companies' social or environmental mission,” the Chronicle of Philanthropy notes.

Russell Sullivan, staff director for the US Senate Finance Committee, thinks that new tax structures could create a “blurring of the lines” between charities and for-profit companies generally. Historically, the tax code endeavors to group all entities into either the for-profit or the non-profit bucket.

"We might see the emergence of some proposals to establish what I'll call, for the lack of a better term, a for-benefit corporation -- something that is in between a private taxable company that's not under our rules of C corporations or S corporations and partnerships but also not under our rules having to do with charities," he said in another Chronicle of Philanthropy article. Mr. Sullivan continued: "But I see even more blurring of the lines over the past decade." Green energy companies in some cases intend "actually to develop or promote a cleaner environment -- they are just doing it through a corporate structure."

This is an interesting approach, indicative of a changing national tax climate and approach to doing business in many industries. Prize4Life has blogged extensively on collaboration between non-profits and pharma/biotech recently (see posts here and here) in the context of drug discovery and development; could the creation of a benefit corporation be the next step for disease research? Would it follow the model of, for example, the ALS Therapy Development Alliance? What promise might such a national change hold?

Tuesday, April 13, 2010

ALS Awareness Day

On Wednesday, April 7, Prize4Life participated in the ALS Association’s ALS Awareness Day, working to raise awareness of ALS among legislators and to promote the funding of the ALS registry. Being a so-called ‘Orphan Disease’, ALS does not get the attention and funding that many feel it deserves, and this day was an opportunity to both push for awareness, and to focus priorities on a specific program goal. Above: Prize4Life staff Dr. A. Sheila Menzies, Dr. Melanie Leitner, Meghan Kallman, and Brooke Larimer at the Statehouse.

As noted in the ALS Association’s 2010 policy priorities, the Massachusetts ALS Registry is a state-wide ‘surveillance mechanism, enabling researchers to look at patterns of disease in the Commonwealth in their search for a cure (see a release on the project here). This registry is intended to help researchers study both the incidence and prevalence of the disease, identify trends, and develop additional research studies related to better understanding ALS. In the words of the ALS association: “The ALS Registry in Massachusetts brings us one step closer to a cure.”

The Registry was first authorized in Massachusetts by Section 26 of Chapter 140 of the Acts of 2003 (ie, the State budget). The budget line established a program for identifying all the ALS cases in Massachusetts through the Department of Public Health. The program was funded at $150,000 annually since it was initially authorized in 2003. However, the 2010 budget reflected a cut to less than half of that amount. The ALS Association believes that this project is too critical to let fall by the wayside, and rallied to lobby for funding in the original amount to maintain the ALS Registry.

The day opened with speeches by Debra Sharpe, president of the ALS Association. ALS advocate and patient Steven Saling, an architect working closely on the Leonard Florence Center for Living, gave a moving speech on his involvement in the center and his approach to life with ALS (the Center is a home designed especially to enable ALS patients to live independent, fulfilling lives—very cool. Check it out here). Suzanne Condon, Associate Commissioner of the Massachusetts Department of Public Health, updated the audience on the status of the state ALS Registry in Massachusetts. Dr. Tracie Caller, a researcher at Dartmouth Hitchcock, spoke about the current research taking place on the epidemiology of ALS in New England, specifically in New Hampshire.

Prize4Life believes that this is an important advocacy effort, and we were honored to participate. Following the introductory speeches, Prize4Life met with State Senator Patricia Jehlen, as well as Senator Anthony Petruccelli and his aid Donna LoConte. We also spoke with Representative Frank Smizik's staff, and left materials for Representative Lida Harkins. Additionally, Prize4Life’s Chief Scientific Officer, Melanie Leitner, was fortunate to speak with representatives from the Department of Public Health, including epidemiologist Dr. Christine Fischetti, who is working closely on the ALS registry (she signed up for our ALS Forum! Have you?) All meetings discussed the importance of supporting ALS research through this Registry effort, and the importance of focusing resources on finding a cure. Above: Brooke Larimer and Meghan Kallman with Senator Patricia Jehlen.

Organizations and individuals approached their meetings differently. One woman brought along a photograph of her mother, who had passed away from ALS, to show her elected officials, explaining through her tears why she cared so deeply about the project. Others spoke about the loved ones they had lost to the disease, while still others, ALS patients themselves and aided by canes or ventilators, arrived to advocate for themselves.

Be aware that the National ALS Advocacy Day and Public Policy Conference, is coming up, from May 9-11 in Washington, DC. May is also ALS Awareness Month—what will you do to help us find a cure?

Tuesday, April 6, 2010

ALS Clinical Trials Update

Back in January, as part of our coverage of the International ALS/MND Symposium in Berlin, Prize4Life blogged briefly about ALS clinical trials. We also featured clinical trials coverage here by Dr. Amber Dance, the science writer we sponsored through the ALS Forum, our collaboration with the Alzheimer Research Forum. And you all are wondering what has happened since then on the clinical trials front, right? Well, wonder no longer. Here’s the latest (in alphabetical order):

Ceftriaxone is an antibiotic used to treat Lyme disease, however it has also been shown to affect uptake of the neurotransmitter glutamate (which means, in English, that it may help protect brain cells which can be killed by too much glutamate) The Ceftriaxone Phase III study continues to enroll , and there is a potentially promising pre-clinical study looking at the drug’s effects for patients with spinal muscular atrophy (an infant-onset disease which shares many similarities with ALS). A 2005 study suggested that Ceftriaxone could increase lifespan, strength, and neuron survival in ALS model mice (for more information, see this review).

San Francisco, CA based Cytokinetics recently initiated a Phase IIa “Evidence of Effect” (EoE) clinical trial of their small molecule CK-2017357 for patients with ALS. The drug is reported to act on fast skeletal muscles to make them more responsive to signals from motor neurons. This means that the drug could, in essence, help muscles stay stronger and delay the loss of muscle control that accompanies ALS. In March 2010, Cytokinetics was granted orphan-drug designation for this compound by the United States Food and Drug Administration (FDA). The orphan-drug designation carries a number of benefits to help the drug reach the market sooner, including tax credits, and scientific and financial support.

Isis Pharmaceuticals in Carlsbad, California is using antisense technology to try to reduce the amount of a protein, superoxide dismutase (SOD1) that is mutated in some ALS patients. Antisense is a method by which scientists can reduce levels of a given protein and thus theoretically help in diseases thought to be related to mutant proteins, such as ALS. This approach was found to be effective in animal models (data will be presented at the upcoming American Academy of Neurology (AAN) Meeting next week) and the first patient was just enrolled in a Phase I clinical trial in March. When completed, this trial will enroll 32 subjects, and safety results will hopefully be completed in 2011.

Knopp Neurosciences
Knopp is working on a small molecule drug that is thought to protect the function of mitochondria, the energy source for all cells of the body (motor neurons are relatively huge cells so they are very sensitive to reduced energy supply). Nothing new has been reported by Knopp since the results presented at the Berlin Meeting, however, the company is scheduled to attend the AAN meeting next week. So stay tuned!


Neuralstem, Inc. of Rockville, Maryland has begun a Phase 1 safety trial utilizing stem cells derived from fetal tissue in ALS patients. The goal of the trial is to test a novel surgical method, developed by surgeon Nick Boulis, to safely deliver cells into the spinal cord. They have currently enrolled 2 patients and are planning on enrolling a dozen participants total (see ARF related news story).

TCA Cellular
TCA Cellular of Covington, Louisiana, has recently enrolled the first of 6 anticipated patients in a Phase I safety trial of its stem-cell based therapy.

While this progress on the clinical trial front looks promising, Prize4Life believes we still need other potential therapeutics in the pipeline. This is why we are excited about the impressive teams competing for the ALS Treatment Prize and why we are so focused on translational research (that is, the process of translating scientific discoveries into practical application, from the ‘bench’ to the ‘bedside’. See a full blog post on it here.) Those interested in the challenges and promise of translational neuroscience and the drug companies at the forefront of the field may be interested in an upcoming meeting happening in Boston in May:

The 2nd Annual Drug Development for Neurodegenerative Diseases conference will be held May 18-19. Convened by marcus evans, an internationally known conference organizer targeting senior industry decision-makers, the conference will feature a wide-ranging and high quality program covering innovations in pre-clinical studies and clinical trial design, biomarker discoveries, and target identification for therapeutic and drug discovery journey.

The conference will feature speakers from major drug companies, including Novartis, Merck, Pfizer, and BiogenIdec as well as some up-and-comers including Allon Therapeutics, Acorda Therapeutics, and EnVivo Pharmaceuticals (EnVivo is also one of the teams competing for the Avi Kremer ALS Treatment Prize). While the meeting will not focus exclusively on ALS, it will cover many topics and speakers in related fields that will hopefully “translate” into the ALS space. Prize4Life will be blogging live from this conference, so make sure to follow the blog for some hot-off-the-press info!