The California stem cell agency this Thursday is scheduled
to give away nearly $16 million for clinical trials for possible therapies connected
to AIDs and a rare genetic affliction involving immune deficiencies.
The larger of the two awards -- $8.5 million -- will go to
an unidentified recipient for an early stage trial involving AIDS-lymphoma
patients.
The agency’s blue-ribbon reviewers earlier voted 10-0 behind
closed doors to approve the application. On Thursday, the full board of the
agency will ratify the vote in public. The board has almost never overturned a
decision by reviewers to fund an award.
The summary of the review of the application, which was not disclosed, said that the
proposed therapy “would be a significant improvement over standard of care in
the proposed AIDS lymphoma patient population.
“With the proposed patient population (AIDS lymphoma
patients), impact will be somewhat limited. However, demonstration of success
in this limited context would serve as an impetus to solve additional problems
such as non-ablative conditioning to achieve engraftment in the larger HIV
patient population.”
Despite another “paucity of patients,” the stem cell agency
is also expected to approve $7.4 million for an early stage trial involving X-linked
chronic granulomatous disease.
Reviewers voted 5-1-4 to approve the application. The voting
translates to five for the application, one indicating it needs improvement and
four indicating that it is “sufficiently flawed that it does not warrant funding,
and the same project should not be resubmitted for review.”
“Reviewers were…divided as to the feasibility. Some
reviewers did not think that there are sufficient patients afflicted with this
disease who also meet the enrollment criteria to allow for completion of the
trial with enough patients to yield meaningful outcomes. Other reviewers were
optimistic that, given the track record of this team and the willingness of
patients with intractable orphan diseases to participate clinical trials, the
trial could be fully enrolled and yield results to potentially support moving
the therapeutic toward approval by the FDA and/or advance the field.”
The target of the potential therapy is an inherited
condition that weakens the immune system.
According to the Rare Disease Network, it occurs in only one out of every 250,000 persons. Only 20 new cases
a year are reported in the United States,
according to Wikipedia. The review
summary used the expression “paucity of patients.”
Reviewers said, however, the approach has “potential value
in a number of other diseases.”
The application carried with it $4.6 million in co-funding
promised in a publicly unspecified form via the applicant.
Both the applications had been reworked and resubmitted to
the agency under its new CIRM 2.0 award process. However, this week is the
first time the board has considered them.
(Please note that the agency has cobbled together the review summaries for both applications. That means that some readers seeking the summary related to the $8.5 million award could be misled when the immune deficiency award turns up at the top of the document. The full application is also never disclosed by the agency, only the summary.)
Rejected by reviewers was a $2.9 million application from
another unidentified group. The vote was 2-6-3, meaning that the proposal can
be reworked and resubmitted.
The board’s meeting will be in Oakland at the Marriott City
Center. Public teleconference locations will be in San Diego, Stanford, South
San Francisco and two in La Jolla. The public, including scientists, can participate from those
locations as well as in Oakland.