Monday, June 28, 2010

ALS Drug Development Update

It can be difficult to keep abreast of all of the news that is relevant to research and treatment of ALS. Because of that, Prize4Life has designed tools to help keep researchers and the public up-to-date on what is happening in the research and drug development worlds (you can sign up for the free Weekly Digest and ALS Forum Newsletter if you want to get these alerts in your inbox). Below is a roundup of the most relevant ALS drug development news from the last six weeks. Check it out!

Invitrogen Contributes New Research for ALS
Invitrogen's stem cell business is participating in a new research program designed to find treatments for ALS. Life Technologies, Invitrogen's parent company, has partnered with the University of California San Diego and the Salk Institute for Biological Studies to research cell transplant therapies as a potential ALS treatment.

FoldRx Pharmaceuticals secures $29 million financing

FoldRx Pharmaceuticals has closed a $29 million financing deal to support a program in neurodegenerative disease. "With the addition of two top-tier venture firms to our already strong investor base, we believe we now have access to the resources necessary to commercialize tafamidis and build FoldRx into an independent, orphan-disease focused company," said Richard Labaudinière, Ph.D., President and CEO of FoldRx, in a FierceBiotech release. FoldRx Pharmaceuticals is focusing on disease-modifying small-molecule therapeutics to treat diseases of protein misfolding and aggregation, key pathological processes for many chronic neurodegenerative diseases, including ALS.

Aukera Therapeutics Named 2010 MIT Business Plan Contest Life Sciences Track Winner
Cambridge-based start-up biotech, Aukera Therapeutics, is developing Angiogenin, a novel protein therapy, to treat ALS. As the winner of MIT's Business Plan Contest, Aukera will receive seed funding and in-kind support from The Cambridge Innovation Center.

Stem Cell Therapeutics Wins US Patent
Stem Cell Therapeutics has received US patent rights for production of neural stem cells, a technology intensively investigated for its therapeutic use in ALS and other neurodegenerative diseases.

Amorfix Life Sciences and PREVENT Sign Licensing Agreement on ALS Vaccine
Amorfix has granted exclusive licensing rights to PREVENT for clinical development of its lead ALS vaccine for therapeutic purposes. Amorfix has retained the rights to develop the antibodies for diagnostic purposes.

Q Therapeutics’ Collaborators at Johns Hopkins Receive $1 Million to Study Glial Cells for Neurodegenerative Conditions
This infusion of funds is expected to accelerate characterization of Q Therapeutics’ proprietary glial cell-based therapeutic (Q-Cells®). This technology is being developed as a treatment for ALS and other neurodegenerative diseases.

ALS TDI: Changes in the Works
ALS TDI’s expertise using the SOD1 based model for target identification and drug discovery has made significant contributions to the ALS field. Recently, the non-profit ALS biotech has announced incorporation of a TDP-43 mouse model into its drug development program. This is a valuable effort that could bear dividends for the entire ALS research community.

Cytokinetics Announces Opening of a Phase IIa "Evidence of Effect" Clinical Trial of CK-2017357
Cytokinetics will begin a Phase II study of its fast skeletal muscle troponin activator, CK-2017357, for patients with peripheral artery disease and claudication. This announcement follows an ongoing Phase IIa trial in ALS patients. Cytokinetics believes this second study will further inform their understanding of possible therapeutic applications for this novel drug candidate for ALS and other skeletal muscle-associated diseases.

Neuralstem Updates Clinical Trial Progress
The first FDA approved stem cell clinical trial for ALS is reported to be on track. Neuralstem has completed safety evaluations of its first cohort of ALS patients and plans to move forward with the second cohort using a more extensive treatment regime.

Pfizer, Washington University announce collaboration
Pharma giant Pfizer grants Washington University unprecedented access to proprietary information regarding 500+ drug candidates. The collaboration is expected to uncover new uses for existing compounds.

Teva Reports Disappointing ALS Phase II Results for Talampanel
Teva’s talampanel is a no-go for ALS after discouraging Phase II data are reported. Despite demonstrating safety, talampanel did not reach its primary endpoint and will not be advanced as an ALS therapeutic.

New Virtual ALS-focused Company Aukera Therapeutics Wins MIT Award
Aukera, a finalist in the MIT $100K Business Plan Contest, wins the audience award for its development of a novel ALS therapeutic. The technology is based out of research from Harvard Medical School.

Repligen Files IND for Neurodegenerative Disease Treatment
Repligen has filed an investigational new drug application with the FDA for a Phase I study of its selective HDAC3 inhibitor, RG2833. While Repligen's initial drug development focus will be Friedreich's ataxia, RG2833 may have a broader therapeutic use for the treatment of ALS and other neurodegenerative diseases.

Monday, June 21, 2010

Global Day for ALS

As a member of the International Alliance of ALS/MND Associations, Prize4Life is pleased to participate in Global Day for ALS. Since 1997, the International Alliance has celebrated 21 June as the global day of recognition for ALS– a disease that affects people in every country of the globe.

June 21st is the summer solstice—a turning point—and every year the ALS community undertakes a range of activities to express its hope that this day will be another turning point in the search for cause, treatment and cure of this disease.

Members of the International Alliance, and those who have been touched by ALS worldwide, do a number of things to call attention to the plight of ALS patients. Some organize meetings of patients and caregivers; they put on social events, create working groups, hold fundraisers, or simply come together to reflect on being part of the global fight against ALS.

The following is a reprint of the annual letter from Gudjon Sigurdsson, the Chairman of the International Alliance of ALS/MND Associations:

Global day 2010

The fight continues for a world free of ALS/MND. This year’s global day is no different from others. We will do our best to spread the word about the damn thing we all fight against. Soon we will celebrate this as the day we remember as the day we won the fight and got rid of ALS/MND.

Until then we all must do our best to enjoy every moment of each day. As well as we possibly can. “A glass full of darkness and sorrow is always by our hand. A bit further is a glass filled with light and opptimism. Reach for it.” These words of an Icelandic poet who died from ALS/MND, are worth keeping in mind.

My wife and I are celebrating our 25 years anniversary of marriage this month. I am very lucky she has put up with me all this time. We have agreed that this is only possible if both do their best to make it last and be enjoyable. Friendship and connections with others does not happen by itself. No, we need to work on it. Communicate and tell people we love, that we do.

In those times of crisis we need to defend our rights, find new ways of doing things and do not forget we are here for all the PALS around the world. Making it possible for them to enjoy life, support research and inprove the law and regulations in our home country.


Gudjon Sigurdsson
Chairman, International Alliance of ALS/MND Associations

Global Day is a day of commemoration and strength-building. It, like ALS Awareness Month here in the United States, is an opportunity to assert the resilience of the human spirit over the ravages of ALS.

Prize4Life believes deeply in this resilience, and makes change our business. We know that it is a priority to assist those in need right now, and our colleagues in patient service programs are doing a spectacular job of that. Other organizations, such as the ALS Association and the MDA, fund researchers upfront, and their support has done incredible things for the progression of ALS science.

Prize4Life’s programs are intended to speed up this work by providing resources and incentives that may not otherwise be available to researchers and to the ALS community. Our prizes are intended to foster creativity and new approaches (see the range of talented teams who are currently competing here). This blog, for example, is designed to bring different stakeholders and opinions together to discuss various elements of the ALS, R&D, and philanthropy spaces. Our ALS Forum is a free news source for patients and researchers alike, a cutting-edge resource that gathers the most up-to-date ALS research information in one place.

We are proud of these contributions, and we are always on the lookout for other ways to bring innovative parties and stakeholders together.

What will you do to help win this battle? For a list of ideas, click here. Or email us!

Thursday, June 10, 2010

Prize4Life holds Art, Life, Spring Gala and Art auction to raise funds for ALS

On Thursday, May 27, 2010, Prize4Life held its inaugural A.L.S. Gala and Art Auction at the Roger Smith Hotel in New York City. Sponsored by Bloomberg LP, Vertex Pharmaceuticals, Biogen IDEC, and Bristol-Myers Squibb, the event raised over $110,000 towards Prize4Life’s innovative efforts to find a cure for this devastating neurodegenerative disease (for more on our programs, click here).

The evening included dinner and an auction, led by Sotheby’s auctioneer Jamie Niven (son of David Niven, the famed actor who passed away from ALS). Mayor Bloomberg presented the evening’s opening remarks, and attendees included Nobel laureate Dr. Torsten Wiesel, Asaf Shariv, the Consul General of Israel, and renowned sports painter Bill Lopa.

Mayor Bloomberg presented the evening's opening remarks

Sotheby's Jamie Niven led the A.L.S. Art auction

A companion cocktail party and art showing, held at Sotheby’s on May 20th, also drew a large crowd, including guest of honor Gabriella Shalev, the Israeli Ambassador to the United Nations, who graciously gave an introduction to the evening. Guests sipped champagne, watched a short video on Prize4Life's co-founder and CEO Avi Kremer, and enjoyed the art that was hung for display.

Prior to the auction, the Huffington Post ran an emotive op-ed by artist Miriam Cabessa on her work's physical qualities, and its relationship with ALS. See that piece here. Photographs from both events are available here.

Works auctioned on May 27th included Gold Landscape, by Miriam Cabessa, At the Beach by Natan Elkanovich, Hanna & Shemu'el, by Boaz Vaadia, Ruth and Gehrig by Bill Lopa, The Unveiling by MuKha, Window Box, 2002 by Tim Prentice, Untitled #100 by Leora Laor, and Boy's Head, by Itmar Jobani.

Thanks to all who attended and helped make both events such a great success!