Key Beyond Batten Disease Foundation Fund-raiser Brings in $70,000 in February

A 24-month campaign to raise $6 million toward curing juvenile Batten disease got a $70,000 pop in February, the Beyond Batten Disease Foundation announced in its March newsletter.

The Be Project fund-raising effort, which started in 2016, is about $3.7 million, or two-thirds of the way, toward its goal,  according to a foundation-produced chart. The money will go toward research projects, treatment development efforts — including cutting-edge approaches like gene therapy and gene skipping — and testing potential therapies in clinical trials.

Back in 2009, a $2.5 million gift from the Beyond Batten Disease Foundation and Cherie and Jim Flores enabled Texas Children’s Hospital researchers to come up with a potential treatment they could test in animal models of Batten disease.

Their research has shown that the protein Akt controls transcription factor EB, a master regulator of fat metabolism and lysosomal storage. Lysosomal storage diseases stem from dysfunction of lysosomes, or sacs of enzymes inside cells that digest large molecules. Batten disease is a nervous system lysosomal disease.

Akt, which can modify other proteins, has been studied in detail, and there are drugs that can regulate its activity. The findings of this research could lead to new ways of treating neurodegenerative storage disorders like Batten disease.

Batten research has attracted growing attention from the U.S. government as well as the private sector in recent years. Of the $31.3 billion that the U.S. Department of Health and Human Services awarded for medical research in 2016, $1,670,876 million went to juvenile Batten research.

The funding helped researchers investigate mutations in 12 genes associated with the disease.

Of the $6 million that the Be Project campaign hopes to raise, estimates are that $2.5 million will be necessary to validate initial academic work on Batten therapy development and to fund clinical trials on therapies’ optimal dosing, safety and effectiveness.

Texas Children’s Hospital will be collaborating on this effort by conducting validation studies whose costs are estimated at $1.2 million.

Another $1.6 million go toward developing biomarkers of Batten disease, creating an international patient registry, conducting early-phase clinical trials of potential therapies, and having regulatory consultations with U.S. Food and Drug Administration officials.

About $500,000 will go toward supporting cutting-edge treatment approaches such as stem cell therapy, gene therapy and gene skipping. And $200,000 will provide scientists with Batten-specific research tools.

There are several ways to contribute to the Be Project fund-raising campaign. One is by being a donor, making a one-contribution or pledging a monthly or annual gift. Another is by participating in the amazon component of the campaign. This involves linking an Amazon.com account to the Beyond Batten Disease Foundation, and giving while shopping. Still another way is by being a connector — hosting a gathering of family and friends that want to participate or connecting the foundation to other major donors and foundations.

All of the money donated will go toward research, therapy development, and trials.

To see how the Be Project campaign works, check out this video.