The results to our gene therapy clinical trial were published in the Human Gene Therapy Medical Journal. The trial's data scientifically demonstrates that our gene therapy clinical trial was a success in slowing the progression of the disease as well as being safe. The publication proves our therapeutic approach as a safe and viable platform to treat other neurological diseases. The findings supports our therapy and further gives justification for our new version of the drug as it will be safe and expand upon the successes of our initial trial. As indicated before, our new version of the drug gets tremendous enzyme expression in animal models and demonstrates tremendous clinical benefits in these animals. The new drug has curative properties in the animal models. NIH or private funding is all that the new drug is waiting for in order to move forward to a new clinical trail.
Our new drug (AAVhr10-CLN2) is ready and waiting to move forward to clinical trials. All pre-clinical data and toxicology data has been completed. The FDA has given preliminary support of the trial and now funding is the only thing stopping the treatment of children. Cornell has submitted for NIH funding but has been in an holding pattern for some time. Private funding or NIH fund would enable Cornell to move forward with this tremendously promising drug.
The initial AAV2-CLN2 gene transfer clinical trial is completed. The trial data clearly showed that the treatment was effective and was a success. The gene therapy treatment demonstrated clinical improvements in the children based on the LINCL rating scale. Based on the scale, the treatment slowed or stopped the progression of the disease within the CNS. A tremendous amount was learned from the clinical and has positioned us to move the next trial. One main discovery was that we are on the correct tract with introducing the enzyme into the CNS via gene transfer. The new drug AAVrh10-CLN2 will get even more enzyme distribution through out the CNS and allow for more benefit for the patient as also demonstrated in the CLN2 knockout animals.
Cornell has developed a new gene therapy vector to treat Late Infantile Batten disease. This new vector, AAVrh10, has for the first time demonstrated in CLN2-KO mice tremendous clinical benefit. Diseased mice treated with this new vector are able to walk up and down a balance beam and hang on a 'high wire" and climb across a "hire wire". Non-treated animals can not walk and fall off the balance beam. And non-treated animals can not grip or climb on the wire. Treated animals life span are also greatly increased over non-treated animals. This is the first time such remarkable clinical data has been demonstrated in animal models by any proposed therapy. Cornell has begun an effort to mount a clinical program for this new drug and hope to submit an IND to the FDA within the next 12 months.
Stem Cells Inc. receives hospital IRB approval from the Oregon Health & Science University to begin the trial at OHSU Doernbecher Children’s Hospital in Portland, Oregon. Both clearance by the FDA and IRB were necessary to initiate the clinical trial. Subjects are currently being screened for inclusion into the clinical trail. The first patient is scheduled to be treated this Summer. This is the first ever approved trial to transplant human neural stem cells in the brain. No other disease has ever been treated using neural stem cells and the first ever treatment will be attempted in Batten disease.
P.J. goes to NYC for his 18 month follow up visit. The 18 month evaluation is the final short term evaluation point in the clinical trial protocol. The 18 month evaluation was 3 days of testing which included a MRI/MRS study. P.J. did well with all the test and clinical evaluations. His clinical status remains stable and hopes for the future is that this stable status will continue. We hope and believe that we may have stopped or slowed the disease in the brain and the concern for the future would be any potential downstream affects from pre-existing damage to his systemic neurological, pulmonary, or renal systems.
Nathan goes to NYC for his 18 month follow up visit. The 18 month evaluation is the final short term evaluation point in the clinical trial protocol. The 18 month evaluation was 3 days of testing which included a MRI/MRS study. Nathan did very well with all the test and clinical evaluations. Nathan has thrived over the last 18 months gaining over 16 pounds and growing 4-5 inches. His clinical status remains stable and hopes for the future is that this stable status will continue. We hope and believe that we may have stopped or slowed the disease in the brain and the concern for the future would be any potential downstream affects from pre-existing damage to his systemic neurological, pulmonary, or renal systems.
The eighth child was treated in the gene transfer clinical trial at Cornell University. This child was the third "Moderately" affected child to be treated. This child did very well with the procedure. This child was moderately affected but still has many faculties about her and for the most part would be a "normal" little girl. The goal for these treatments is to treat children that have not progressed so treating this little angel has everyone excited about her potential.
Stem Cells Inc. receives FDA approval to begin a Phase I human clinical trial of Neural Stem Cells to Treat Batten Disease. This is the first ever approved trial to transplant human neural stem cells in the brain. No other disease has ever been treated using neural stem cells and the first ever treatment will be attempted in Batten disease. Click for details.
The seventh child was treated in the gene transfer clinical trail at Cornell University. This child was the second "Moderately" affected child to be treated. This child also did well with the procedure. The team continues to be very excited as they treat more "Moderately" affected children. They are very interested in monitoring the effects of the treatments on these children over time. The belief is the less affected the child is by the disease the more benefit the drug may have for the child.
The Nathan's Battle at Broadmoor Country Club Golf outing was held. This event was well supported by many returning sponsors. This was a great outing for everyone. The organizers did a tremendous job once again.
Nathan goes to NYC to have his 12 month evaluation performed. This is a clinical evaluation to monitor his overall condition and neurological status. The 12 month evaluation part of the trial to gather data to better help determine the effects of the treatment. Nathan's overall condition was viewed as no real change. No Change! Again, given the goals of the treatment to slow or stop the progression of the disease, no change is a POSITIVE result.
P.J. goes to NYC to have his 12 month evaluation performed. The same clinical evaluations are performed on him as they were on Nathan. As with Nathan, P.J.'s overall condition was viewed as no real change. No Change! Again, given the goals of the treatment to slow or stop the progression of the disease, no change is a POSITIVE result.
The sixth child was treated in the gene transfer clinical trail at Cornell University. This child was the first "Moderately" affected child to be treated. This child also did well with the procedure. The team is very excited to follow the progress of this "Moderately" affected child. The belief is the less affected the child is by the disease the more benefit the drug may have for the child.
The fifth child was treated in the gene transfer clinical trail at Cornell University. This child did very well. She was out of intensive care in a couple of days and was released from the hospital as scheduled, shortly after the procedure. This child completes the Severe rated children and now the team will move to less severely affected children.
STEMCELLS, INC. Announces filing of Investigational New Drug (IND) for human neural stem cell transplant treatment for Batten Disease to the U.S. Food and Drug Administration (FDA). This Phase I Clinical Trial at Stanford would Be First-Ever Using Transplantation of Human Neural (Brain) Subject to approval, the Company plans to begin its first clinical investigation of its human neural (brain) stem cells (HuCNS-SC – StemCells’ proprietary neural cell therapy product) in Batten disease. If approved by the FDA, this would mark the first-ever FDA-approved clinical trial to use a purified composition of human neural stem cells as the potential therapeutic agent. On approval of the IND by the FDA, the study will be conducted at Stanford University Medical Center. NBF's efforts to get SCI involved with NCLs has produced another potential treatment for LINCL.
Nathan goes to NYC to have his 6 month evaluation performed. This evaluation includes a MRI and MRS to better help evaluate the effects of the treatment on the CNS. Nathan's MRI shows No Change! Again, given the goals of the treatment to stop the progression of the disease, no change is a POSITIVE result, meaning if the brain doesn't show more degradation then the therapy may be working. MRS results will not be available for several weeks. Nathan continues to be a very happy boy.
The 4th child participates in the gene transfer clinical trial. The most severely affected child with Late Infantile Batten Disease undergoes the treatment procedure. The project team performs the procedure on this very severely affected LINCL child in hopes to determine the full scope of potential benefit in disease ranges of LINCL children. It is unknown how the child will respond with the treatment and if the disease progression is too far advanced for the "drug" to have time to benefit the child. This is another move forward in research with hopes to fully understand the potential benefits or limits of the treatment.
P.J. had his 90 day check-up is still doing well. No change with this treatment is a positive and so far so good.
The national award winning Nathan and P.J.'s Ride was held and it was another huge success. The ride committee did an exceptional job. The event was an all day celebration for Nathan's Battle. Over 800 motorcycles participated in the ride and well over 1100 people. This event had more sponsors, activities and prizes than any previous event. The Milto family continues to be forever indebted to the organizers of this event. The organizers were lead again by Papaw and his right hand Melissa. The ride raised over $78,000 for the Foundation.
Nathan had his 90 day check-up and P.J. had his 60 day check-up. Both boys are very stable and doing well!!
The Nathan's Battle at Broadmoor Country Club Golf outing was held. This event was well supported by many returning sponsors. This was a great outing for everyone. The organizers did a tremendous job once again.
P.J. has his 14 and 30 day check ups and everything is as planned. The 14 day check was performed locally and the 30 day check up was done in NYC. P.J. is back to his pre-surgery condition, smiling and laughing often. Nathan also had his 60 day check up and everything is fine with him as well.
Nathan has his 14 day and 30 day check ups and is doing well. Nathan's 14 day check up was performed locally and his 30 day check was done back in NYC. Nathan remains very stable.
P.J. GETS TREATED! P.J. participates in the human clinical trial at Cornell. The procedure goes as planned and P.J. does very well. P.J. is a very strong little boy. This is another major accomplishment for NBF. Focusing on winning the battles and the war!!!.
NBF makes HISTORY. The human clinical trial begins. The first ever treatment for LINCL is administered to a child. The first child was treated with AAV2cuCLN2, the drug we have been developing for the last 3.5 years. This is a historical accomplishment which would not of been possible without all the supporters of NBF. Everyone who ever supported NBF in anyway should be proud of this initial accomplishment. This is a major step in winning the war against this disease. We are winning the battles to win the war.
Stem Cells Inc. receives $20 million in new equity financing. This infusion of new capital is one critical factor toward enabling them to initiate their first clinical trial in Batten disease in 2005.
NATHAN GETS TREATED! Nathan participates in the human clinical trial at Cornell. Nathan does very well through the procedure. The procedure goes as planned. Nathan is a true Champion in more ways than one. This is a significant accomplishment for everyone involved.
Phil attended the Lysosomal Diseases and the Brain Conference in Washington D.C. He was invited to help propel LSD's toward therapeutic applications. The focus of the conference was to move research from the bench to the clinic. Something in which NBF has first hand experience.
Stem Cells Inc. publicly commits to moving their technology to the clinic for INCL and LINCL. Stem Cells Inc. is well positioned to move to the clinic by Q1 2005. NBF's efforts to get SCI involved with NCLs is producing measurable results. Their data has continued to be extremely promising and they have made some key hires to guide the program to the clinic.
Genzyme, a major biotech company who focuses on lysosomal storage diseases, submits an abstract to the Society of Neuroscience demonstrating the use of our vector (AAV2cuCLN2) successfully impacts the disease in CLN2 knockout mice which have the disease. The abstract's title is: Intracranial Gene Delivery of Human CLN2 Reduces Brain Pathology in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinoses (LINCL). This abstract is authored by scientists from Genzyme, Cornell, Iowa, Robert Wood Johnson and supported by NBF. Just one of several successful studies being carried out with the CLN2 KO mouse and the above collaborators using our vector.
The Gene Transfer Clinical Trial at Cornell receives final formal approvals from all regulatory levels to begin the "Treatment" protocol. Previously approved by the FDA.
Phil meets with Executive Hospital Director of NewYork-Presbyterian, The University Hospital of Columbia and Cornell, to discuss arrangements to fund the expenses associated with performing the clinical trial. The Director was supportive of the effort and offered to assist NBF by allowing for the Foundation to pay for the procedures at reduced rates.
The Gene Transfer Clinical Trail at Cornell receives formal IBR approvals to begin the "Screening" protocol. Patients begin enrolling in the "Screening Protocol" for ultimately inclusion into the Gene Transfer Clinical Trial.
Nathan and P.J. go to New York to participate in the "Screening Protocol". The screening protocol included a battery of tests and evaluations including a MRI. Both boys were in NYC for 3 days to participate with hopes of meeting the Clinical Trials inclusion criteria to be able to participate in the trial.
The Indianapolis Business Journal recognizes Nathan's Battle's accomplishments with their prestigious 40 under 40 award.
Nathan's Battle Foundation wins the Health Care Heroes Advancements in Health Care Category. This award was given based on the tremendous advancements NBF has made in novel treatment technologies. NBF was honored to be place in the company of some of the country's leading physicians and medical professionals.
The Gene Transfer Clinical Trial at Cornell receives FDA approval to begin treating humans with the new drug.
Gene transfer IND receives approval from RAC (Recombinant Advisory Committee). Receiving formal approval from the RAC is a major milestone and is a testament to the quality of data that has been produced over the last 3 years. The Cornell team has done a tremendous job developing the IND with quality data and an outstanding clinical plan.
NBF's Drug Screening program validates three potential compounds that show promise in-vitro to enhance TPP-1 enzyme production. Next step is planning the in-vivo studies in KO animal models.
Continued studies on CLN2 knockout mice (mice that have the disease) demonstrate our vectors clear the harmful storage in the mice brains. These studies are being performed by a major biotech company using our vectors in a collaboration with the Cornell team. These ongoing studies provide additional sound scientific data to support the use of our vectors to treat the disease. The vector has "worked" in the over 300 rodents, in the 40+ primates, and in the knockout diseased animals. It has been tested on all drug development tools with tremendous success. It is now time to move to humans.
Cornell's final study data was completed confirming the drug will work. The data demonstrated robust enzyme expression at an 18-month time point in the animals with no negative side effects. This data is extremely promising and is the first of its kind at an 18-month time interval.
Phil meets with Cornell's imaging center director to discuss partnering with our clinical trial project. The imaging center at Cornell is a newly built multi-million dollar, state of the art facility with all the latest capabilities in the field (anatomical, functional, and spectroscopic imaging technologies). The director of the facility is excited about the opportunity to partner with the the project to provide imaging needs for patients in the trial. While in NYC, Phil was invited to lecture to medical students in a genetic medicine course at Cornell. The students were able to get real life experience with a rare disease and gain an understanding of NBF's efforts.
Phil signs $2.1 million dollar milestone driven pledge agreement with Cornell University to pay for the human clinical trial. The pledge allows the trial to begin without any delay and is based on Cornell's ability to satisfy treatment milestones. NBF must make quarterly payments of $177,000 for the next three years starting January 2004. This is great news so that children will be treated immediately following regulatory approvals. Unfortunately, we do not have these funds secured and need to raise the required funds. We need help raising these funds. We have develop a cure and just need to pay to TREAT these innocent children.
Stem Cells Inc. is moving forward with a clinical program for Batten disease. The company's cell mediated technology has demonstrated strong efficacy data in INCL mice and they have committed to moving forward to the clinic. Key new leadership has been added to direct the program and significant financing has been raised by investors given the promising in vivo data. NBF continues collaborations with the company to aid in the advancement of their technology.
Phil attends Cornell project status meeting in NYC. The Cornell team presented recent data from the primate toxicology studies. The data from the toxicology studies demonstrated the drug is safe and can be used in humans. The human protocols and remaining data are being finalized for the regulatory submissions. All regulatory documentation will be submitted BY December 2003. Patient evaluations will start in January 2004. It is unknown how long the FDA and RAC approval process will take (could take from 1-6 months).
Cornell agrees to allow Nathan's Battle Foundation to enter into a pledge agreement with the University to fund the actual clinical trial. Normally clinical trials require the funding secured before the work can begin but Cornell aids NBF by letting the Foundation pay the expenses over time based on a milestone driven funding schedule. Cornell demonstrates their continued leading role in genetic medicine by not delaying the trial and partnering with NBF to move forward. Funding is still the main issue and is desperately needed to treat these innocent children.
The 5th Nathan's Battle Golf Outing was held at the Hickory Stick Golf Course. This event demonstrates the tremendous support the Milto family has from its closest friends and the community. Outback Steakhouse was a major sponsor and catered the events dinner. The outing was very well attended with over 130 golfers and many corporate sponsors. Just as important as the funds raised, the heartfelt support that the Milto's received helps them continue their fight. This support gives them strength to get through their family's daily struggles and strength to fight the war against Batten disease.
Ringling Bros. and Barnum & Bailey Circus held Nathan's Battle Under the Big Top. Ringling Bros. and Barnum & Bailey graciously approached Nathan's Battle to have a fundraising performance again this year. Ringling Bros. and Barnum & Bailey Circus donated portions of the proceeds to the Nathan's Battle Foundation for tickets sold during the benefit performance at Conseco Field house. Thousands came to enjoy the greatest show on earth and to support Nathan's Battle.
The national award winning Nathan and P.J.'s Ride was held and it was another huge success. The organizing committee did an incredible job with this event. This event had more sponsorship and prizes than any previous event. The biker community showed their huge hearts as they participated in large numbers and raised over $40,000. The Milto family will forever be indebted to the organizers of this event. An army of bikers spent a year organizing this event lead by Papaw and his right hand Melissa.
The final large animal toxicology studies were completed in September. This milestone signifies the end of the drug testing process. The data will now be analyzed and prepared to be included in our submissions to the FDA. The project team will submit for regulatory approvals by December 2003. There are 4 agencies that must approve our trial. If the regulatory approval process goes well we could begin the HUMAN CLINICAL TRIALS in January 2004. The approval process could take from 1 to 6 months. We are unsure how long the process will take. We are months away from saving lives!
The third Nathan's Battle Family Walk was another success. Supporters from all over attended this years walk to continue their support of NBF.
Nathan's Battle wins an award from Eli Lilly and Company. Phil received the award from the major pharmaceutical company for the pursuit of health care innovation. Phil was one of 17 winners out of a group of over 6000. Phil was nominated by the global procurement group from Eli Lilly and Comany.
The Million Dollar Duck Race was held in downtown Indianapolis on the canal in White River State Park during the Taste of Indiana. This was a spectacular event attended by thousands of duck adopters. Everyone looked on with excitement as the ducks "raced" down the canal where winners won items like a new Saturn car, a hot tub and J.L. Johnson shopping spree. Several television station were in attendance to cover the exciting event.
A First class golf tournament was held at the Nathan's Battle at Broadmoor Country Club Golf outing. This event was well supported by many return sponsors. Four golfers had a chance to win $1 MILLION and a minimum of 1/2 was to be donated back to the foundation. These golfers were all very close to winning the prize. Three of the four were within 12 feet of the cup. One golfer's shot was tracking right toward the hole but stopped just short. He said it was the ball selection that made it check-up. It was a great effort and all who attended had a great time.
The Cornell gene transfer project holds a key CLN2 clinicians meeting to define patient evaluation criteria for the human clinical trial. CLN2 clinical markers and endpoints will continue to be refined for the upcoming human trials.
Phil is the Key Note Speaker at the Indiana Health Industry conference (www.ihif.org) held in the Indianapolis convention center. This conference was attended by over 600 life science professionals as part of the Indiana Life Sciences Initiative. These life sciences professional heard about the accomplishments of the Nathan's Battle Foundation. NBF is developing therapies as it's own "non-for-profit" biotech company just as the attending parties are hoping to accomplish. Phil shared NBF's approach and business strategies used to enable the drug development process. The underlining message brought to the group was the reason for such an initiative is for the patients that depend on their success. NBF humanizes the industry for what drives our biotech is Nathan and P.J. The presentation was acknowledge by a standing ovation by all who attended.
The Cornell gene transfer project performs the final non human primate surgeries for the toxicity studies. These last animals are the final critical path task necessary to complete the large animal toxicity studies. These studies are the final step in the drug development process. The results to these studies will help complete our application to the FDA to gain regulatory approvals to move to human clinical trials. The project continues to progress toward the clinic.
Phil attends Cornell project status meeting in NYC. The Cornell team presented more promising data from the primate efficacy studies. The primate data meets the "Go" criteria defined with the regulatory agencies to move to humans. The primate toxicology studies began in March (the final major step). All other project tasks continue to move forward on schedule. The project is closing in on clinical trials.
NBF projects are featured presentations at the NCL World conference. Phil attends the International Congress on NCLs. The NCL-2003 world conference is attended by the world's leading experts in NCL research. This conference featured presentations on two of NBF's leading projects.
The Cornell gene transfer project begins non human primate toxicity studies. This is a major milestone for the project. The large animal toxicity studies are the final step in the drug development process. The results to these studies will help complete our application to the FDA to gain regulatory approvals to move to human clinical trials. Another major project milestone is in process.
NBF's drug screening program begins performing PCR studies on the list of previously identified positive enzyme affecting compounds. These studies will definitively analyze the compounds to determine potential therapeutic benefit for CLN2 patients. NBF's earlier drug screening findings identified a group of FDA approved drugs that had an affect on the missing enzyme.
The Nathan's Battle "Lift for Life" was held to raise funds for the foundation. This unique event raised funds as contestants competed in a weight lifting competition. Power Clean, Bench Press, Dead lift and 2 Man Dead events were held demonstrating amazing efforts of strength. NBF continues to get "stronger" with community support. One day we will all be as strong as Nathan and P.J.
P.J. has surgery performed to have a feeding tube installed. The feeding tube was inserted to keep P.J. healthy and strong. This procedures will keep P.J. strong as he waits for a therapy to be developed
A Nathan's Battle dinner benefit was held. This event was another tremendous display of the overwhelming community support. Near 1,200 supporters attended this heartfelt event. The event was an incredible success raising over $185,000.00 for the foundation. Supporters gave from their hearts knowing that they are making a difference while being inspired by the brave presents of Nathan and innocent smiles of PJ. The Milto's always have dreamed of having a family. Their dream is still about their family but it is now just to keep their family. This community and others around the world now share in the Milto's dream and soon they will be living their dream again.
Nathan's Battle receives a letter from Cornell University outlining the projects successes, remaining steps and required funding needed to get to clinical trials. Cornell believes believe that given the over 22 months of data produced from this effort: including rodent distribution and expression studies, long-term rodent studies, initial non-human primate studies, and initial favorable FDA reviews, that if the biology continues to support our theory in the primates and if the regulatory climate is supportive a human clinical can be initiated once the necessary funding is obtained to perform the final few tasks to gain regulatory approvals.
NBF receives more national exposure as The Learning Channel airs a story on the Milto's. The Learning Channel aired a version of CBS's Medical Mysteries show containing segments from the 48 Hours story.
Phil attends a Cornell project status meeting in NYC. The Cornell team presents more promising data from long-term rat studies and the initial primate studies. The long term rat studies demonstrate exciting news in that the gene is expressing over many months. While the early primate data is even more promising than the team expected. These large animal studies will continue over the next several months with toxicity studies set to begin in March. The project is closing in on clinical trials.
The FDA accepts the Cornell CLN2 Gene Transfer project approach and gives the Pre-IND package a favorable review with minor modifications. The Cornell department Chairman stated, "This is a major leap forward". With the FDA's support, the project team can move quickly into the final stages of drug development.
On December 2, 2002, the Cornell CLN2 Gene Transfer project begins large model and toxicity studies. This starts the final stages of the drug development process. Large animal studies and toxicity studies are the two major milestones left to accomplish before submitting for final regulatory approvals. The FDA was supportive for us to begin both studies concurrently to expedite the process.
Phil was invited to attend his third straight advocacy breakfast meeting at the Society for Neuroscience conference in Orlando. NBF continues to be a cutting edge leader in rare disease therapy development and is sought to share their approach and accomplishments.
The second Kiwanis Battle Run was held to support Nathan's Battle Foundation. The Kiwanis continue to show their support for NBF. Many members and volunteers worked hard to hold this event. Sponsors' support and the many participants demonstrated their overwhelming support of NBF. Another great event by the Kiwanis as they continue helping children with their mission and particularly ones in their own community.
Phil was invited to attend "The Neurobiology of Disease Workshop - Stem cells for Neurodegenerative Diseases" at the Society for Neuroscience conference by the workshop's organizing chairman. Phil participated in the day long cell mediated workshop with many of world's leading stem cell scientists. This workshop set the ground work for future studies of LINCL gaining much interest by scientific groups as LINCL proves to be a great disease target for moving cell mediated research toward clinical applications.
The Milto family attended a Mark Schultz benefit concert in Columbus Ohio for Nathan's Battle. NBF has expanded across the country in research and with many fundraising events. The concert was organized by NBF supports in Ohio and attended by other LINCL families. NBF is gaining more and more national and international support. Mr. Schultz gave a wonderful performance raising money and awareness for NBF. This concert was a moving experience for all that attended. Check Mark's web site to see his support "on my heart".
The Cornell CLN2 Gene Transfer project submits for Pre-IND approvals with the FDA. The Cornell project team reached a major milestone this month by submitting for Pre-IND (Investigational New Drug application) review with the Food and Drug Administration. Phil attended the Cornell project team status meeting in NYC to review the detailed Pre-IND design. This is the beginning of the final stages of the drug development process. Large animal studies and toxicity studies are the two major milestones left to accomplish before submitting for final regulatory approvals. If the FDA is supportive of the Pre-IND, the project team's "goal" is to reach human clinical trials by October 2003. Large animal studies begin in the first week of December 2002 with toxicity studies commencing in April 2003. In less than 12 months we could be saving children's lives. Even with this tremendous scientific news, funding is still our biggest obstacle. Cornell informed Phil that they will need $100K/ month for the next 12 months. NBF's current funding will run out in February 2003 (As of Oct. 2002 NBF's remaining balance is $400K).
Roche Diagnostics Inc. supports Nathan's Battle's fight against Batten Disease. Roche is a leading healthcare company with a uniquely broad spectrum of innovative solutions. For more than 100 years, Roche has been active in the discovery, development, manufacture and marketing of novel healthcare solutions. Roche recognizes the importance of the research NBF is developing and as a responsible corporation actively involved in the community has recently supported our locally based international foundation focused on improving society by developing therapies for rare diseases. Roche and NBF will make a great partnership.
The 4th Nathan's Battle Golf Outing was held at the Valle Vista Country Club. This event demonstrates the tremendous support the Milto family has from its closest friends and the community. The outing was very well attended with over 130 golfers and many corporate sponsors. The outing raised over $10,000 for the fight against Batten disease. Just as important as the funds raised, the heartfelt support that the Milto's received helps them continue their fight. This support gives them strength to get through their family's daily struggles and strength to fight the war against Batten disease. Nathan and P.J. attendance at the event left many of the golfers with a sobering understanding of the cruel affects of the disease and the urgency and necessity of the ground breaking work of NBF.
Nathan's Battle Under the Big top 2 took place. Ringling Brothers & Barnum and Bailey Circus partnered with Nathan's Battle Foundation to hold a special benefit performance. This was a spectacular display of entertainment. The world famous circus made Phil and Tricia the honorary ring masters for the night. The Greatest Show on Earth was simply just that as Indianapolis demonstrated its tremendous support for NBF with attendance of over 10,000 at Conseco Fieldhouse.
Nathan's Battle Race For Life 4 mile Run/Walk was another top notch event. The organizers of this event did a tremendous job orchestrating this large scale event. Near 100 volunteers helped to ensure the success of this event. Over 300 participants pushed themselves to achieve personal best as they continued to show their support for NBF. One of the lead runners actually yelled Nathan's name as he ran the course to help motivate himself. Truly a spine tingling experience to have others draw strength from Nathan's spirit.
Another first class golf tournament was held at the Nathan's Battle at Broadmoor Country Club Golf outing. This event had many corporate and local sponsors. Four golfers had a chance to win $1 MILLION and a minimum of 1/2 was to be donated back to the foundation. Local media were in attendance to cover the event. Unfortunately, the golfers accuracy was not there to cash in on the chances but everyone in attendance had a great time. This event raised over $31,000.
Max and Erma's held an all day benefit for NBF. This years event was very well attended and beat last years attendance records.
Phil attends a Cornell project status meeting in NYC. The project continues to progress as we prepare for large model studies to begin. Dosage and distribution studies are being finalized in small models and the protocol development team will begin drafting the regulatory documentation.
Phil visited with a leading stem cell scientist to go over his latest finding. This leading scientist is preparing to perform preliminary studies on a NCL model for NBF. NCL studies are scheduled to begin within the next few months.
The second Nathan and P.J.'s Ride was another success. The biker community showed their huge hearts as they participated in large numbers and raised near $30,000. The ride was escorted by the Indianapolis Motor Speedway around the Circle of Downtown Indianapolis to Southside Harley Davidson for auctions, food, and music.
Nathan's Battle Family Run/Walk was another success. Supporters from all over attended this years walk to continue their support of NBF. The MOMs Club put on another great event.
Mark Schultz benefit concert held for Nathan's Battle. The concert raised over $20,000 for NBF. The concert was attended by near 1000 concert goers. Mr. Schultz announced that he will continue to raise funds for NBF throughout his tour and introduce his audiences to Nathan's Battle. NBF has given his song, "He's My Son", new meaning for him.
Phil attends a Cornell project status meeting in NYC. The project is still progressing as we continue to demonstrate success of transferring CLN2 gene into the CNS of models. Dosage studies are being performed as the manufacturing team finalizes production techniques. Large model studies are now being initiated.
Phil visits Dr. Peter Lobel's laboratory at the Robert Wood Johnson Medical Center in N.J. on the Rutgers University campus. Dr. Lobel's lab is the world's leading CLN2/TPP1 enzyme scientific group. Dr. Lobel and Phil met to discuss the latest progress on enzyme delivery, model development, and NBF's therapy development projects. NBF is supporting the Lobel laboratories to further CLN2 protein research.
Tricia and Phil have an EEG performed on PJ. They have a great deal of concern as PJ is having trouble with his vision, balance, and walking. The EEG was to evaluate potential seizure activity. PJ's EEG was abnormal and he is now placed on seizure mediation to help control this activity. PJ's innocence of the road in front of him give NBF even more power to continue the battle.
Phil receives an update from two of the Canavan AAV2 gene therapy patient's mothers on their child's progress. These children were both injected with a similar gene therapy as the Cornell project's proposed therapy. The first mother stated "...my child's latest report is great. She has new myelin all over her brain. This clearly correlates with clinical improvement. I am convinced that this study will be very positive...". The second mother exclaimed, "...my son is really doing well, he is still making gains in all areas. He is eating really well, and moving more, and overall just looks very healthy. His MRI is showing new myelin in 25 places in his brain, the doctor is seeing the same type of medical results for the other two kids as well. Also, his scores on neuro-assessments and neurological exams have improved and are higher than those of an untreated child...". This data continues to support the realities of gene therapy for Batten disease.
Tricia and Phil take Nathan and PJ to Duke University to see one of the world's leading clinical experts on Batten disease. This expert will help consult the boys local physicians on the proper care for the boys. Clinically evaluating the children will help her have better insight in determining the proper "cocktail" of medications the boys will need. Getting the children the proper mediation is an ongoing struggle with no clear cut answers. Over the last several months, Nathan has been battling different issues and has been in and out of the hospital. The disease has also been showing it's symptoms in PJ.
Phil travels to the west coast to aid in the initiation of a new cell mediated therapy project. Phil met with the project teams from the academic group and the biotech team to walk through the proposed work plan and to review the current progress. The initial data from this project demonstrates promising results in similar disease models. A detailed study of a NCL model is now underway.
Nathan's Battle Foundation enters into an agreement with a biotech company to greatly expand NBF's high through-put drug screening program. This is a major project to screen several compound libraries (libraries include the FDA-2000 and the GNC libraries). NBF is the first and currently the only organization performing drug screening for CLN2. This screening will actually use Nathan's cells to screen drugs against. This therapy would introduce a drug compound into the body that could cross the blood brain barrier into the Central Nervous System of a patient to either enhance residual enzyme activity or remove the storage material that is harmful to the patient.
The Indianapolis Ice, CHL professional hockey team, host "Pack The House" night at Conseco Fieldhouse to benefit Nathan's Battle Foundation. Nathan was able to go on the ice and perform a ceremonial "puck drop" to start the game on center ice. A new record of over 14,000 Ice fans were in attendance to support the Ice and local charities.
Phil attends gene transfer project status meeting at the Weill Medical College of Cornell in NYC. The project update was extremely encouraging. For the first time, pre-clinical data using our specific vector/gene combination (AAV2-CLN2) demonstrate successful gene delivery into models. This data continues to support the hypothesis under which we work. The whole project team was very upbeat with this new data. Now that we have demonstrated gene delivery, the next steps are to perform dosage and distribution studies and scale up to larger models before moving to humans. The project is still on schedule.
The Nathan' Battle Foundation works with a leading stem cell company and a major west coast University to assist in the coordination of applying new stem cell technologies to NCL disorders. NBF will be supporting a project to evaluate these technologies on a NCL model.
Phil traveled to California to meet with a leading stem cell research company. This company's expertise in stem cell technologies are extremely promising and they are directed by some of the worlds top scientists in the field. The company gained interest in the NCLs by attending the dinner Phil's group hosted in San Diego. Phil met with company leaders to: present background information on CLN2, the benefits of working on CLN2 as a disease target for cell mediated therapies, and NBF's value in facilitating the obtainment of CLN2 reagents, academic collaborations, clinical program experience, and regulatory relationships. The company tentatively agreed to investigate CLN2 using their cell mediated technologies toward the development of therapeutic applications.
From education to healthcare, Nathan's Battle offers a variety of programs to support the community. Learn more about our initiatives and how we make a positive impact.