History of Accomplishments

May-08

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.

May-08

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. 

Nov-07

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.

Jun-06

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.

May-06

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.

Mar-06

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.

Dec-05

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.

Nov-05

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.

Oct-05

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. 

Sep-05

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. 

Aug-05

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.

Jul-05

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.   

Jul-05

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.   

Jun-05

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. 

Apr-05

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.

Jan-05

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.

Dec-04

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.   

Oct-04

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.

Oct-04

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. 

Sep-04

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. 

Sep-04

Nathan had   his 90 day check-up and P.J. had his 60 day check-up.  Both boys are   very stable and doing well!! 

Sep-04

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.

Aug-04

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.  

Jul-04

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. 

Jul-04

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!!!.

Jun-04

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.         

Jun-04

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.   

Jun-04

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.

May-04

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.

May-04

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.         

May-04

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.

Apr-04

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.

Mar-04

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.         

Mar-04

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. 

Mar-04

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. 

Feb-04

The   Indianapolis Business Journal recognizes Nathan's Battle's accomplishments   with their prestigious 40 under 40 award.         

Feb-04

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.       

Feb-04

The Gene   Transfer Clinical Trial at Cornell receives FDA approval to begin treating   humans with the new drug.         

Jan-04

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.         

Jan-04

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.

Dec-03

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.

Dec-03

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.

Dec-03

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.     

Dec-03

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.   

Nov-03

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.   

Oct-03

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). 

Oct-03

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.

Sep-03

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.

Sep-03

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.

Sep-03

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.

Sep-03

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!

Aug-03

The third   Nathan's Battle Family Walk was another success.  Supporters from all   over attended this years walk to continue their support of NBF.

Aug-03

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.

Aug-03

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.

Aug-03

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.  

Jul-03

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.

Jun-03

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.    

Jun-03

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.   

May-03

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.  

Apr-03

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.

Mar-03

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.

Mar-03

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.  

Mar-03

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. 

Mar-03

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

Feb-03

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.

Feb-03

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.

Jan-03

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. 

Jan-03

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.

Dec-02

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. 

Dec-02

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.

Nov-02

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.

Nov-02

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. 

Nov-02

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. 

Nov-02

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".    

Oct-02

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).

Sep-02

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.

Sep-02

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.

Sep-02

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. 

Aug-02

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.

Aug-02

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.  

Jul-02

Max and   Erma's held an all day benefit for NBF.  This years event was very well   attended and beat last years attendance records.  

Jul-02

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. 

Jul-02

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.     

Jul-02

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.

Jun-02

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.

May-02

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.  

Apr-02

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.  

Apr-02

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.

Mar-02

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.    

Mar-02

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.

Mar-02

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.  

Mar-02

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.

Feb-02

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. 

Feb-02

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.

Feb-02

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.

Feb-02

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.  

Dec-01

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.

Dec-01

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