California Parkinson's Group Strikes Out on Path to Stem Cell Therapy; Clinical Trial, For-profit Company Goals

A San Diego patient advocate group for Parkinson's Disease is making a major financial move as it heads toward creation of a for-profit company to develop a stem cell therapy for an affliction that affects about one million people in the United States.

The group is the Summit for Stem Cell Foundation, which has been deeply involved in seeking funds from the $3 billion California stem cell agency. The Summit organization supports research being conducted by Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute.

Summit says it has raised $5 million as of this spring from a variety of sources. It estimates it will need $8 to $10 million to reach the stage where the federal government approves the beginning of a clinical trial. It also expects to raise more cash by avoiding the high administrative charges involving many research organizations.

Reporter Bradley Fikes of the San Diego Union Tribune wrote yesterday about the latest developments. He said Summit is moving out of its space at Scripps as it prepares to apply for funds for clinical research. Fikes wrote,

"The group has leased about 5,000 square feet, doubling its space, and as a parting gift Scripps Research is donating equipment from its stem cell center, saving the foundation nearly $1 million in start-up costs. At the same time, its overhead is dramatically reduced, said Jeanne Loring, the group’s lead scientist and professor at Scripps Research. (Loring is serving as the unpaid director of research for Summit.)

"A $2.4 million grant the group received from California’s stem cell agency ran out earlier this year, Loring said. Now the group can step up fundraising and add staff. It’s also soliciting more donated equipment."

Fikes continued,

"The group also plans to establish a for-profit company to get funding from investors interested in generating returns, Loring said. By drawing investments as well as philanthropy, the project gains access to capital not otherwise available.

"The group was inspired by the example of the Cystic Fibrosis Foundation, Loring said. In 2000, the foundation gave San Diego’s Aurora Biosciences $40 million to develop a cystic fibrosis drug."

Fikes reported that separation from Scripps has financial advantages dealing with what called "indirect costs." 

"Raising (donations) has been difficult, in large part because of the overhead issue, Loring said.

"Scripps Research imposes overhead of up to 94 percent, meaning that every dollar donated to the group required an additional 94 cents for the institute, Loring said. At the new location close by on Torrey Pines Mesa, overhead is roughly 10 percent.

"Lower overhead means the group can now apply for philanthropic grants that previously weren’t practical, she said, because donors prefer as much as their money as possible go directly to the cause."

Here is a video of Loring describing her work. 

$21 Million Likely for California's Basic Stem Cell Research

Directors of the California stem cell agency tomorrow are expected to approve at least $21 million for basic research into “significant, unresolved issues in human stem cell biology.”

The round was originally slated for $40 million but grant reviewers decided to fund only 20 applications out of 62. The round began with 341 scientists filing pre-applications.

(Here is a link to reviewers' summaries and rankings as well as a link to the CIRM staff's Power Pointpresentation.)

CIRM President Alan Trounson and his staff recommended approval of five additional applications totaling$4.8 million. The rationale in their recommendations could be considered fulsome compared to what Trounson offered on the $40 million genomics round also to be considered tomorrow.

Five additional researchers filed letters with the CIRM governing board seeking its approval. Those letters can be found on the agenda for tomorrow's meeting. Seven researchers filed formal appeals with the CIRM, which are now dealt with behind closed doors by the agency's staff.

The 20 applicants given the nod by reviewers were placed in a tier one category that is virtually certain to be approved by the board with no debate. Others were ranked in a wobbler category called tier two, meaning it could go either way for the scientists. The remainder fell into tier three – not recommended for funding by reviewers.

The board is increasingly turning to proposals that will turn more advanced research into clinical treatments. If directors do not go for spending the entire $40 million budgeted, they would save money that could be used for clinical trials. However, aside from the general arguments for doing basic research, those grants provide large sums to recipient institutions to pay for their overhead. And many of the agency's board members come from institutions that could benefit from payments for those overhead costs. Those board members will not be allowed to vote on applications involving their institutions.

(Editor's note: The number of researchers filing formal appeals was not contained in an earlier version of this article.)

Stem Cell Agency Approves $36 million to Recruit Six Scientists to California

The California stem cell agency today awarded $36 million to six scientists to lure them to the Golden State, in what was the agency's largest-ever recruiting round.

The awards more than doubled the amount of CIRM has spent on recruitment. Until today, the agency had awarded only $23.2 million for four awards.

Today's awards ranged from $7.5 million to $4.8 million. The agency did not immediately identify the recipients. However, testimony at the meeting indicated that two of the institutions involved were UC San Francisco and the Gladstone Institute (the $7.5 million award) and UC Santa Cruz (a $5.4 million award). (The agency later released the list, which can be found here. Here is a link to the CIRM press release.)

The differences in the size of the awards had to do with the overhead charges that are levied by the institutions at which the scientists would work, CIRM staff said. The "direct costs" of the research for each grant was $4.5 million.

Six applications were considered in the latest round, including one that was scored at 75 that was rejected by grant reviewers. However, CIRM staff recommended that application, which involved UC San Francisco and Gladstone, be funded. (See here and here.)

Previous winners of the recruitment awards were Robert Wechsler-Reya, Sanford-Burnham; Dennis Steindler, The Parkinson's Institute; Andrew McMahon, USC, and Peter Coffey, UC Santa Barbara.